A GUIDE TO SPINRAZA REIMBURSEMENT · 2021. 2. 26. · SPINRAZA is a US Food and Drug Administration...
Transcript of A GUIDE TO SPINRAZA REIMBURSEMENT · 2021. 2. 26. · SPINRAZA is a US Food and Drug Administration...
INDICATIONSPINRAZA® (nusinersen) is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
SELECTED IMPORTANT SAFETY INFORMATIONCoagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications.Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
IMPORTANT INFORMATION TO HELP NAVIGATE THE ACCESS AND REIMBURSEMENT PROCESS
A GUIDE TO SPINRAZA REIMBURSEMENT
SELECTED IMPORTANT SAFETY INFORMATIONIn the sham-controlled studies for patients with infantile-onset and later-onset SMA, 24 of 146 SPINRAZA-treated patients (16%) with high, normal, or unknown platelet count at baseline developed a platelet level below the lower limit of normal, compared to 10 of 72 sham-controlled patients (14%). Two SPINRAZA-treated patients developed platelet counts <50,000 cells per microliter, with the lowest level of 10,000 cells per microliter recorded on study day 28.
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
IntroductionWELCOMESPINRAZA is a US Food and Drug Administration (FDA)–approved treatment indicated for spinal muscular atrophy (SMA) in pediatric and adult patients.1 Biogen is committed to providing detailed information to assist in obtaining reimbursement for SPINRAZA, drug administration, and related ancillary services. We have developed this guide in conjunction with our support service, SMA360°*, to provide you with the information you need to help with the reimbursement process for SPINRAZA. SMA360° offers individualized support to help your patients and their families throughout the treatment process.The information in this guide is intended for informational purposes only and does not represent legal or billing advice. For specific guidance in this area, consult your own legal/billing advisor and billing/coding specialist because it remains your responsibility to ensure the accuracy of the claims your office submits. The content herein is based on information current as of August 2020, which may have changed.Any product, ancillary supplies, or services received free of charge cannot be billed to third-party payers because doing so could be a violation of federal and/or state laws and/or third-party–payer requirements.
SPINRAZA SUPPORT AND RESOURCESSMA360° IS HERE FOR YOUR PATIENTSBiogen’s SMA360° program offers comprehensive and individualized support to help patients with SMA and their families navigate nonmedical barriers to access. Services include logistical assistance, product education, insurance benefits investigation, and financial assistance. A complete list of SMA360° offerings can be found at SPINRAZA-hcp.com/support.
SMA is a highly variable disease and each patient will have his or her own unique set of needs. Your patients or their caregivers may feel like they could use a helping hand. Biogen has a team that will be there for them throughout the SPINRAZA journey. Please remember that you should be the primary resource for any questions related to SMA and SPINRAZA. Additional information about the services provided by SMA360° is included in this guide.
* SMA360° services from Biogen are available only to those who have been prescribed SPINRAZA. SMA360° is intended for US residents only.
2
SELECTED IMPORTANT SAFETY INFORMATIONRenal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. SPINRAZA is present in and excreted by the kidney. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 71 of 123 SPINRAZA-treated patients (58%) had elevated urine protein, compared to 22 of 65 sham-controlled patients (34%).
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
3
SMA360°™* is available to assist youSMA360° team members are available to assist your practice or site of care (SOC) by providing nonclinical education and support to help overcome access challenges.
RARE DISEASE REIMBURSEMENT MANAGER (RDRM) The RDRM is responsible for helping you and your staff navigate the reimbursement and administrative processes for SPINRAZA. The RDRM can// Educate you and your staff on SPINRAZA procurement methods
// Provide enhanced education on claims forms and coding/billing
// Support your team’s interactions with health plans
* SMA360° services from Biogen are available only to those who have been prescribed SPINRAZA. SMA360° is intended for US residents only.
Indication and Important Safety Information
INDICATIONSPINRAZA® (nusinersen) is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
IMPORTANT SAFETY INFORMATIONCoagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 24 of 146 SPINRAZA-treated patients (16%) with high, normal, or unknown platelet count at baseline developed a platelet level below the lower limit of normal, compared to 10 of 72 sham-controlled patients (14%). Two SPINRAZA-treated patients developed platelet counts <50,000 cells per microliter, with the lowest level of 10,000 cells per microliter recorded on study day 28.Renal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. SPINRAZA is present in and excreted by the kidney. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 71 of 123 SPINRAZA-treated patients (58%) had elevated urine protein, compared to 22 of 65 sham-controlled patients (34%). Laboratory testing and monitoring to assess safety should be conducted. Perform a platelet count, coagulation laboratory testing, and quantitative spot urine protein testing at baseline and prior to each dose of SPINRAZA and as clinically needed.Severe hyponatremia was reported in an infant treated with SPINRAZA requiring salt supplementation for 14 months.Cases of rash were reported in patients treated with SPINRAZA. SPINRAZA may cause a reduction in growth as measured by height when administered to infants, as suggested by observations from the controlled study. It is unknown whether any effect of SPINRAZA on growth would be reversible with cessation of treatment.The most common adverse reactions (≥20% of SPINRAZA-treated patients and ≥5% more frequently than in control patients) that occurred in the infantile-onset controlled study were lower respiratory infection and constipation. Serious adverse reactions of atelectasis were more frequent in SPINRAZA- treated patients (18%) than in control patients (10%). Because patients in this controlled study were infants, adverse reactions that are verbally reported could not be assessed. The most common adverse reactions that occurred in the later-onset controlled study were pyrexia, headache, vomiting, and back pain. Post-lumbar puncture syndrome has also been observed after the administration of SPINRAZA. Please see accompanying full Prescribing Information.
4
SELECTED IMPORTANT SAFETY INFORMATIONLaboratory testing and monitoring to assess safety should be conducted. Perform a platelet count, coagulation laboratory testing, and quantitative spot urine protein testing at baseline and prior to each dose of SPINRAZA and as clinically needed.Severe hyponatremia was reported in an infant treated with SPINRAZA requiring salt supplementation for 14 months.Cases of rash were reported in patients treated with SPINRAZA. SPINRAZA may cause a reduction in growth as measured by height when administered to infants, as suggested by observations from the controlled study. It is unknown whether any effect of SPINRAZA on growth would be reversible with cessation of treatment.
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
5
SELECTED IMPORTANT SAFETY INFORMATIONLaboratory testing and monitoring to assess safety should be conducted. Perform a platelet count, coagulation laboratory testing, and quantitative spot urine protein testing at baseline and prior to each dose of SPINRAZA and as clinically needed.Severe hyponatremia was reported in an infant treated with SPINRAZA requiring salt supplementation for 14 months.Cases of rash were reported in patients treated with SPINRAZA. SPINRAZA may cause a reduction in growth as measured by height when administered to infants, as suggested by observations from the controlled study. It is unknown whether any effect of SPINRAZA on growth would be reversible with cessation of treatment.
Table of Contents
OVERVIEW OF THE REIMBURSEMENT PROCESS FOR SPINRAZA ..................................6
SITE-OF-CARE CONSIDERATIONS ........................................................................................12
INVESTIGATING BENEFITS AND OBTAINING AUTHORIZATION ...................................18
NAVIGATING FINANCIAL ASSISTANCE OPTIONS .............................................................35
ORDERING SPINRAZA .............................................................................................................40
SUBMITTING CLAIMS FOR SPINRAZA AND RELATED SERVICES ..................................42
MEDICARE AND SMA ...............................................................................................................61
APPENDIX...................................................................................................................................70
// Sample SPINRAZA Start Form
// Sample SPINRAZA Copay Reimbursement Form
// Letter of Medical Necessity/Appeal Template
// References
6
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
6
OVERVIEW OF THE REIMBURSEMENT PROCESS FOR SPINRAZA® (nusinersen)
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
7
SELECTED IMPORTANT SAFETY INFORMATIONThe most common adverse reactions (≥20% of SPINRAZA-treated patients and ≥5% more frequently than in control patients) that occurred in the infantile-onset controlled study were lower respiratory infection and constipation. Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (18%) than in control patients (10%). Because patients in this controlled study were infants, adverse reactions that are verbally reported could not be assessed. The most common adverse reactions that occurred in the later-onset controlled study were pyrexia, headache, vomiting, and back pain. Post-lumbar puncture syndrome has also been observed after the administration of SPINRAZA.
Overview of the Reimbursement Process for SPINRAZA
The following pages highlight key phases of the reimbursement process for SPINRAZA, including steps for starting a patient on therapy, as well as the information needed to submit a claim for reimbursement. Biogen is here to support you in the administration of SPINRAZA and timely submission of claims for adjudication.
This overview also informs various stakeholders involved in the care of patients receiving SPINRAZA. Your Biogen representative is available to assist you with any questions you may have about the process.
2. Financial assistance and insurance counseling
3. Patient treatment scheduling
4. SPINRAZA administration and claim submission
1. Benefits Investigation and authorization
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
SELECTED IMPORTANT SAFETY INFORMATIONCoagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 24 of 146 SPINRAZA-treated patients (16%) with high, normal, or unknown platelet count at baseline developed a platelet level below the lower limit of normal, compared to 10 of 72 sham-controlled patients (14%). Two SPINRAZA-treated patients developed platelet counts <50,000 cells per microliter, with the lowest level of 10,000 cells per microliter recorded on study day 28.
8
A BENEFITS INVESTIGATION WILL HELP DETERMINE PAYER REQUIREMENTS BASED ON THE PATIENT’S SPECIFIC INSURANCE PLAN BENEFITS AND THE INDIVIDUALIZED CARE PLANA. Conduct a Benefits Investigation to help identify the following for SPINRAZA
administration and related services:// Coverage requirements, including precertification and/or medical
documentation; referral restrictions; and observation stay rules// Patient out-of-pocket (OOP) costs such as annual deductible vs amount met
to date, coinsurance and/or copay, and annual OOP maximum vs amount met to date
// State and/or network considerations– Participation status of the institution/practices and participating providers– Coverage restrictions and related exceptions process– OOP costs and related exceptions process– Secondary coverage coordination of benefits and reimbursement/payment
methodology from payers// Billing guidelines
– All documentation required to be submitted with the claim – National Drug Code (NDC) number reporting requirements
APPROVAL OF APPROPRIATE AUTHORIZATION(S) WILL PROVIDE PAYER COVERAGE DOCUMENTATION BEFORE TREATMENT INITIATIONB. Contact the patient’s payer(s) directly to submit necessary documentation in order
to obtain authorization for SPINRAZA administration and related services, such as// Prior Authorization (PA)/precertification form(s) and/or Letter of Medical Necessity // Out-of-state and/or out-of-network exception request and
related documentationC. If your authorization or exception request has been denied, locate the appeal
process and timeline in the denial letter. Contact the payer for instructions if they are not documented for you.
Your RDRM and Family Access Manager (FAM) are available to assist you with any questions you may have about this process.
For additional details, please refer to pages 18-34 of this guide.
1. Benefits Investigation
and authorization
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONRenal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. SPINRAZA is present in and excreted by the kidney. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 71 of 123 SPINRAZA-treated patients (58%) had elevated urine protein, compared to 22 of 65 sham-controlled patients (34%).
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
9
THE SMA360°* TEAM WILL HELP CONNECT THE ENROLLED PATIENT WITH APPROPRIATE FINANCIAL ASSISTANCE PROGRAMS AND PROVIDE INSURANCE COUNSELING, IF NEEDEDA. The SMA360° team identifies appropriate financial assistance options
for eligible patients and assists with program enrollment and any related additional documentation†:// Biogen SPINRAZA Copay Assistance Program// Biogen SPINRAZA Procedure Copay Assistance Program // Third-Party Funding Assistance
B. The SMA360° team offers insurance counseling to the patient’s family (if applicable), including// Summary of current insurance status// Review of potential alternative or supplemental sources of insurance
coverage (eg, Medicaid)
THE SMA360° TEAM CAN COORDINATE SPINRAZA ADMINISTRATION LOGISTICSC. The SMA360° team coordinates logistics with the patient’s family and
the SOC in preparation for the SPINRAZA administration visit
For additional details, please refer to pages 36-38 of this guide.
* SMA360° services from Biogen are available only to those who have been prescribed SPINRAZA. SMA360° is intended for US residents only.
†Other programs may also be available for your patients.
COMPREHENSIVE SUPPORT FOR YOUR PATIENT IS AVAILABLE The SMA360° team will contact the enrolled patient’s family to help set expectations.We understand that for a caregiver or an individual living with SMA, life can be challenging. SMA360° is a support service from Biogen created to help families navigate the following areas of the treatment process with SPINRAZA:// Treatment logistics// Insurance and financial assistance
2. Financial assistance
and insurance counseling
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONLaboratory testing and monitoring to assess safety should be conducted. Perform a platelet count, coagulation laboratory testing, and quantitative spot urine protein testing at baseline and prior to each dose of SPINRAZA and as clinically needed.Severe hyponatremia was reported in an infant treated with SPINRAZA requiring salt supplementation for 14 months.Cases of rash were reported in patients treated with SPINRAZA. SPINRAZA may cause a reduction in growth as measured by height when administered to infants, as suggested by observations from the controlled study. It is unknown whether any effect of SPINRAZA on growth would be reversible with cessation of treatment.
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
10
SCHEDULE PATIENT VISIT FOR SPINRAZA ADMINISTRATION AND MAKE APPROPRIATE PATIENT COORDINATION ARRANGEMENTSA. Considerations
// Clinic visit for preprocedure exam, if needed// Intrathecal injection procedure scheduled with appropriate department// Notify hospital outpatient admission department of PA approval and
treatment date // Notify correct pharmacy department of PA approval and treatment date// Assist family with local overnight accommodations, if needed
CURASCRIPT SPECIALTY DISTRIBUTOR (SD) AND ACCREDO SPECIALTY PHARMACY (SP) ARE THE EXCLUSIVE AUTHORIZED PROVIDERS OF SPINRAZAB. Order SPINRAZA from CuraScript SD or Accredo SP for delivery before the
scheduled patient visit:// The ordering process for SPINRAZA is through your facility’s pharmacy or
procurement department, as it would be for any other treatment
For additional details, please refer to page 41 of this guide.
3. Patient treatment scheduling
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
11
SELECTED IMPORTANT SAFETY INFORMATIONThe most common adverse reactions (≥20% of SPINRAZA-treated patients and ≥5% more frequently than in control patients) that occurred in the infantile-onset controlled study were lower respiratory infection and constipation. Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (18%) than in control patients (10%). Because patients in this controlled study were infants, adverse reactions that are verbally reported could not be assessed. The most common adverse reactions that occurred in the later-onset controlled study were pyrexia, headache, vomiting, and back pain. Post-lumbar puncture syndrome has also been observed after the administration of SPINRAZA.
ADMINISTER SPINRAZA ACCORDING TO THE PRESCRIBING INFORMATION AND INDIVIDUALIZED CARE PLAN// SPINRAZA is administered intrathecally by, or under the direction
of, healthcare professionals (HCPs) with experience performing lumbar punctures1
FOLLOWING PAYER BILLING GUIDELINES CAN FACILITATE CLAIM PROCESSING AND PROMPT PAYMENT A. Submit claim(s) to the patient’s payer(s) for SPINRAZA and related
services according to the billing guidelines identified through the Benefits Investigation
For additional details, please refer to pages 43-58 of this guide.
B. Schedule the next patient visit for SPINRAZA administration
PAYER REMITTANCE MONITORING WILL BE CRITICAL FOR ENSURING APPROPRIATE PAYMENT C. Monitor payer remittance for the submitted claim(s)D. Submit appeal with required documentation within filing timelines if the
claim is deniedE. Submit eligible OOP expenses to copay assistance or charitable funding
programs, if applicable
For additional details, please refer to pages 59-60 of this guide.
If you have any questions throughout this process, call SMA360°* at 1-844-4SPINRAZA (1-844-477-4672), Monday through Friday, from 8:30 AM to 8:00 PM ET, or contact your Biogen representative.
* SMA360° services from Biogen are available only to those who have been prescribed SPINRAZA. SMA360° is intended for US residents only.
4. SPINRAZA administration
and claim submission
12
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
12
SITE-OF-CARECONSIDERATIONS
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
SELECTED IMPORTANT SAFETY INFORMATIONCoagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 24 of 146 SPINRAZA-treated patients (16%) with high, normal, or unknown platelet count at baseline developed a platelet level below the lower limit of normal, compared to 10 of 72 sham-controlled patients (14%). Two SPINRAZA-treated patients developed platelet counts <50,000 cells per microliter, with the lowest level of 10,000 cells per microliter recorded on study day 28.
13
Freestanding ambulatory surgical
center (ASC)
Inpatient hospital facility
Outpatient hospital-based facility*
Physician office
Site-of-Care ConsiderationsSeveral factors can influence the decision to administer SPINRAZA in a particular SOC. In order to ensure that the selected SOC can appropriately address patient needs, it is important that these factors are considered by the key stakeholders involved in patient care, including clinicians, administrators, and the patient’s family.
* Including off-campus clinic, on-campus facility, hospital-based ASC, and other outpatient outlets operated by a hospital.
ADMINISTRATION PROCEDURE AND ANCILLARY SERVICES
// Remember, SPINRAZA is administered intrathecally by, or under the direction of, HCPs with experience performing lumbar punctures
// Ensure the availability of clinical specialists who may need to be involved with SPINRAZA administration (eg, neurologist, anesthesiologist, radiologist)
// Prepare facility or doctor’s office with the necessary equipment (eg, sedation, lumbar puncture, ultrasound, fluoroscopy)
// If needed, ensure that logistical support is in place for the patient’s travel needs, depending on the distance between the SOC and the patient’s home
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONRenal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. SPINRAZA is present in and excreted by the kidney. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 71 of 123 SPINRAZA-treated patients (58%) had elevated urine protein, compared to 22 of 65 sham-controlled patients (34%).
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
14
PAYER REIMBURSEMENT METHODOLOGYYour practice or facility should check directly with the patient’s payer(s) to verify specific coding and billing requirements.
Claim form CMS-1450/UB-04
See page 51 for unique billing considerations
and codes
Claim form CMS-1450/UB-04
See page 58 for unique billing considerations
and codes
Claim form CMS-1500
See page 55 for unique billing considerations
and codes
Claim form CMS-1500
See page 55 for unique billing considerations
and codes
Freestanding ASC
Freestanding ASC
Inpatient hospital facility
Inpatient hospital facility
Outpatient hospital-based facility*
Outpatient hospital-based facility*
Physician office
Physician office
COORDINATION OF CARE
// Some patients may require additional monitoring and/or management in a hospital facility
// Ensure that the facility or doctor’s office can accommodate postinjection monitoring or admit or transfer patient for an inpatient stay
// Some patients may require an inpatient hospital stay for additional monitoring and/or management
// Inpatient administration of SPINRAZA may require a “carve-out” reimbursement agreement with the patient’s payer
// For insight about outpatient observation stays, see page 15
*Including off-campus clinic, on-campus facility, hospital-based ASC, and other outpatient outlets operated by a hospital.
Site-of-Care Considerations (cont’d)
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONLaboratory testing and monitoring to assess safety should be conducted. Perform a platelet count, coagulation laboratory testing, and quantitative spot urine protein testing at baseline and prior to each dose of SPINRAZA and as clinically needed.Severe hyponatremia was reported in an infant treated with SPINRAZA requiring salt supplementation for 14 months.Cases of rash were reported in patients treated with SPINRAZA. SPINRAZA may cause a reduction in growth as measured by height when administered to infants, as suggested by observations from the controlled study. It is unknown whether any effect of SPINRAZA on growth would be reversible with cessation of treatment.
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
15
ADDITIONAL CONSIDERATIONS FOR PAYER REIMBURSEMENT// The facility services may be subject to some form of global payment rule or prospectively set
reimbursement rates (eg, global surgery payment, diagnosis-related group [DRG]–based payment, Enhanced Ambulatory Patient Groups [EAPG] payment)
// The payment for SPINRAZA may be separate or may be bundled within a prospectively set rate (eg, DRG-based rate, EAPG rate per diem rate)
OUTPATIENT OBSERVATION STAY INSIGHTS An observation stay is a hospital outpatient service that can be ordered by physicians to allow for medical evaluation and/or testing in order to determine whether a patient may require an inpatient stay. For example, if a patient experiences a complication after an outpatient surgery, his or her physician may order outpatient observation services to allow for additional monitoring after the postoperative recovery period.
The following are some features of an observation stay that could affect billing:
// A patient may occupy any bed in the hospital, but with outpatient status// Outpatient status has important implications for hospital reimbursement and patient OOP costs// The stay is typically completed within 24 to 48 hours, after which time the patient can be admitted
as an inpatient or discharged// Payers may cover different lengths of outpatient observation stays
– Medicaid may allow up to 48 hours; other private payers may cover only 23 hoursIt is important to verify the requirements for an outpatient observation stay with each insurance carrier.
SOC IMPLICATIONS FOR RELEVANT FINANCIAL ASSISTANCE PROGRAMSThere are several financial assistance programs available to eligible patients to support the administration of SPINRAZA. It is important to note to families that the SOC does not limit the patient’s eligibility for Biogen financial assistance programs.
Biogen has several assistance programs for the SPINRAZA and its administration procedure. See page 37 for more information. For more information on third-party funding assistance, contact SMA360°* at 1-844-4SPINRAZA (1-844-477-4672), Monday through Friday, from 8:30 AM to 8:00 PM ET.
* SMA360° services from Biogen are available only to those who have been prescribed SPINRAZA. SMA360° is intended for US residents only.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
16
SELECTED IMPORTANT SAFETY INFORMATIONThe most common adverse reactions (≥20% of SPINRAZA-treated patients and ≥5% more frequently than in control patients) that occurred in the infantile-onset controlled study were lower respiratory infection and constipation. Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (18%) than in control patients (10%). Because patients in this controlled study were infants, adverse reactions that are verbally reported could not be assessed. The most common adverse reactions that occurred in the later-onset controlled study were pyrexia, headache, vomiting, and back pain. Post-lumbar puncture syndrome has also been observed after the administration of SPINRAZA.
SELECT THE SETTING AND THE SOC FOR SPINRAZA ADMINISTRATION
Outpatient Setting Inpatient Setting
Hospital outpatient off-campus clinic Inpatient hospital facility
Hospital outpatient on-campus facility Other Setting
Hospital-based ASC Other facility
Freestanding ASC
Physician office
Patient Care Checklist Below is a sample checklist for your consideration.
DOCUMENT THE ADMINISTRATION PLAN FOR THE DOSING SCHEDULE Dates of loading doses Dates of maintenance doses (if applicable)
EVALUATE THE NEED FOR AND THE FEASIBILITY OF AN OUTPATIENT OBSERVATION STAY POST INJECTION
Observation stay as a possibility in lieu of inpatient admission
DETERMINE IF ANY ANCILLARY SERVICES MAY BE NEEDED TO SUPPORT SPINRAZA ADMINISTRATION VIA INTRATHECAL INJECTION
Anesthesia Fluoroscopy
Lumbar puncture Other
Ultrasound
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
SELECTED IMPORTANT SAFETY INFORMATIONCoagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 24 of 146 SPINRAZA-treated patients (16%) with high, normal, or unknown platelet count at baseline developed a platelet level below the lower limit of normal, compared to 10 of 72 sham-controlled patients (14%). Two SPINRAZA-treated patients developed platelet counts <50,000 cells per microliter, with the lowest level of 10,000 cells per microliter recorded on study day 28.
17
COORDINATE WITH THE PATIENT AND HIS OR HER CAREGIVER TO CONFIRM SITE SELECTION AND LOGISTICS AND TO HELP SET APPROPRIATE EXPECTATIONS
Treatment process and related timelines
Current payer coverage situation and any anticipated changes
Potential financial assistance needs
SMA360°™* support services available for patients and their families
SUGGEST SMA360° SUPPORT SERVICES, WHICH MAY BE AVAILABLE TO HELP THE PATIENT’S FAMILY UNDERSTAND AND NAVIGATE THE TREATMENT PROCESS
Provide the patient’s family with the SPINRAZA Start Form, assist in completing the patient portion, and review caregiver consent (see page 71 for a sample Start Form)// Your practice or facility should complete the HCP portion of the SPINRAZA Start Form.
Be sure to include the provider’s signature in the Prescriber Authorization section. Fax the completed Start Form to 1-888-538-9781 or email it to [email protected]
If signed consent is provided, advise the patient’s family that a FAM from Biogen will assist in coordinating the logistics of treatment, such as insurance and financial considerations, if needed
IDENTIFY WHICH PROVIDERS/PROVIDER PRACTICE GROUPS WILL OFFER PROFESSIONAL SERVICES RELATED TO SPINRAZA ADMINISTRATION
Neurology Radiology Anesthesiology Other
* SMA360° services from Biogen are available only to those who have been prescribed SPINRAZA. SMA360° is intended for US residents only.
Patient Care Checklist (cont’d) Below is a sample checklist for your consideration.
18
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
18
INVESTIGATING BENEFITS AND OBTAINING AUTHORIZATION
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONRenal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. SPINRAZA is present in and excreted by the kidney. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 71 of 123 SPINRAZA-treated patients (58%) had elevated urine protein, compared to 22 of 65 sham-controlled patients (34%).
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
19
Investigating Benefits and Obtaining AuthorizationA Benefits Investigation is an important step to complete for patients prescribed SPINRAZA to determine drug and ancillary procedure medical coverage. It will help define payer requirements based on the patient’s specific insurance plan benefits and his or her individual needs.
BEGINNING THE BENEFITS INVESTIGATIONA Benefits Investigation is a process that enables a provider to determine benefit design, coverage requirements, and coding guidance. It is important to note that there are many variables associated with each patient’s benefits, and there may be differences by payer, state, general benefit design, and SOC. For SPINRAZA treatment, there may be patients who travel to an SOC that is out of state and/or out of network for his or her payer. It is important to capture this information up front during the Benefits Investigation process so that your practice or facility can submit the claim to be reimbursed for acquiring SPINRAZA, as well as for its administration.
The following is basic patient and provider information that your practice or facility will need to gather to initiate the Benefits Investigation process.
BASIC PATIENT INFORMATION
CONTACT INFORMATION
Patient name Date of birth Phone number
Address
INSURANCE INFORMATION
Policyholder name
Policy start and end dates Member number
Group number
Type(s) of plan(s) (eg, HMO, PPO, POS, EPO, Medicaid)
Primary, secondary, and tertiary insurance information (eg, commercial, Medicaid)
COMPREHENSIVE SUPPORT IS AVAILABLE THROUGHOUT THE BENEFITS INVESTIGATION SMA360°* is a support service from Biogen created to help navigate the complexities of treatment logistics, insurance, and financial assistance. We understand that your patients’ needs are unique, and the SMA360° team is here to help.
We can answer any questions you may have about obtaining preauthorization or precertification, and advise you on how to best navigate the complexities of Benefits Investigation or any unforeseen bumps in the road to approval.
EPO=exclusive provider organization; HMO=health maintenance organization; POS=point of service; PPO=preferred provider organization.
* SMA360° services from Biogen are available only to those who have been prescribed SPINRAZA. SMA360° is intended for US residents only.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONLaboratory testing and monitoring to assess safety should be conducted. Perform a platelet count, coagulation laboratory testing, and quantitative spot urine protein testing at baseline and prior to each dose of SPINRAZA and as clinically needed.Severe hyponatremia was reported in an infant treated with SPINRAZA requiring salt supplementation for 14 months.Cases of rash were reported in patients treated with SPINRAZA. SPINRAZA may cause a reduction in growth as measured by height when administered to infants, as suggested by observations from the controlled study. It is unknown whether any effect of SPINRAZA on growth would be reversible with cessation of treatment.
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
20
BASIC COVERAGE INFORMATION Contact the payer to gather the following information:
COVERAGE
Covered PA required Quantity
PATIENT COST
Office visit copay or coinsurance Drug cost copay or coinsurance Deductible
OOP maximum Pharmacy capitation
KEEPING ACCURATE RECORDS OF A BENEFITS INVESTIGATIONIt is important to document each communication exchange that your practice or facility has with insurance companies. You may be communicating with them several times during the Benefits Investigation. When you do, be sure to record the following:
Date of communication Contact information (direct phone line, email)
Time of communication Communication preference (fax, email)
Person(s) you spoke with Reference number for the call
BASIC PROVIDER INFORMATION
PHYSICIAN PRESCRIBING SPINRAZA
Physician name NPI # Tax ID #
PHYSICIAN(S) ADMINISTERING SPINRAZA (IF DIFFERENT FROM THE PRESCRIBER)
Physician name NPI # Tax ID #
SITE OF CARE ADMINISTERING SPINRAZA
Practice/facility name NPI # Site of care/place of service
NPI=National Provider Identifier.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
21
SELECTED IMPORTANT SAFETY INFORMATIONThe most common adverse reactions (≥20% of SPINRAZA-treated patients and ≥5% more frequently than in control patients) that occurred in the infantile-onset controlled study were lower respiratory infection and constipation. Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (18%) than in control patients (10%). Because patients in this controlled study were infants, adverse reactions that are verbally reported could not be assessed. The most common adverse reactions that occurred in the later-onset controlled study were pyrexia, headache, vomiting, and back pain. Post-lumbar puncture syndrome has also been observed after the administration of SPINRAZA.
Preauthorization/ precertification and required
documentation
Payers may require an authorization of coverage prior to treatment with SPINRAZA// Determine if preauthorization/precertification is required for SPINRAZA,
administration services, and/or the SOC
// Establish whether or not specific documentation is required before the plan will approve the product, administration services, and/or SOC
Medical exception/appeal
When a patient does not meet SPINRAZA coverage requirements stated in payer policy or no policy is in place, coverage may be obtained through the medical exception (ME) process and/or appeal, which tends to vary among payers// Determine if there is an ME/appeal process and what documentation is
required to demonstrate medical necessity
Referral restrictions
Depending on the patient benefits, a payer may require a referral from the primary care physician for the SOC and/or the specialists involved in the administration of SPINRAZA// If it is determined that the patient will require a referral for the administration
of SPINRAZA, find out who should provide the referral and what specifications may be needed
Observation stay rules
For patients who may require additional monitoring after SPINRAZA administration, payers may allow an outpatient observation stay of up to 48 hours// Clarify the parameters that the payer may cover for length of stay for
outpatient observation
Out-of-state and/or out-of-network
restrictions
Some patients who receive SPINRAZA may face restrictions from their commercial and/or Medicaid payers because the provider and/or the service facility is out of network or out of state. In these instances, waivers or exceptions can be granted on the basis of medical necessity// Verify the state and/or network participation status for the physician(s) and/or
facility involved in the administration of SPINRAZA// Investigate and record the patient OOP cost implications for out-of-state
and/or out-of-network providers// Find out if there is an exception process for patients seeking care out of state
and/or out of network
KEY CONSIDERATIONS FOR A BENEFITS INVESTIGATION
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
SELECTED IMPORTANT SAFETY INFORMATIONCoagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 24 of 146 SPINRAZA-treated patients (16%) with high, normal, or unknown platelet count at baseline developed a platelet level below the lower limit of normal, compared to 10 of 72 sham-controlled patients (14%). Two SPINRAZA-treated patients developed platelet counts <50,000 cells per microliter, with the lowest level of 10,000 cells per microliter recorded on study day 28.
22
POTENTIAL PROVIDER NETWORK RESTRICTIONSEach payer may have a network of participating providers who have contracted to provide healthcare services under specific terms. As a result, patients may be restricted or incentivized to seek care from in-network, or preferred, providers, or else OOP costs could be higher or might not be covered at all.2,3
For Medicaid beneficiaries, coverage is generally limited to participating providers in the specific state, and individuals enrolled in Medicaid managed care may also be restricted to care from in-network providers within their state.4 However, coverage exceptions can be granted as long as medical necessity can be established, especially if there are no in-network providers with the required expertise.5
Coordination of benefits for
multiple payers
There may be cases where your patient has multiple payers that provide benefit coverage, such as a commercial health plan and Medicaid// In the case of multiple payers, your Benefits Investigation must establish which
payer is primary, which is secondary, and if needed, which is tertiary // Once you have established the order of benefits, follow the instructions from
each payer regarding coordination of benefits for reimbursement/payment
KEY CONSIDERATIONS FOR A BENEFITS INVESTIGATION (cont’d)
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONRenal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. SPINRAZA is present in and excreted by the kidney. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 71 of 123 SPINRAZA-treated patients (58%) had elevated urine protein, compared to 22 of 65 sham-controlled patients (34%).
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
23
Patient financial responsibility
Patient OOP costs may vary based on the specific benefit design, SOC, and out-of-state/out-of-network restrictions// Determine the patient’s annual deductible and how much has been met
to date// Record the coinsurance and/or copay that will apply for SPINRAZA and
related services// Determine the patient’s annual OOP maximum and how much has been
met to date
Coding and claims submission details
Specific coding and billing requirements may vary by payer// Clarify the requirements for reporting an NDC number in
a medical claim// Check if any specific documentation is required to be submitted with the
claim (eg, clinical records, drug invoice)
KEY CONSIDERATIONS FOR A BENEFITS INVESTIGATION (cont’d)
Remember to reverify your patient’s benefits prior to each dose of SPINRAZA, as the insurance coverage may have changed since the patient’s last procedure. Remind your patients of the importance of immediately informing you and SMA360° of any insurance changes or updates to avoid unanticipated delays in therapy.
SMA360°* PATIENT SUPPORT SERVICES AND BENEFITS INVESTIGATIONSMA360° will also investigate the insurance benefits in order to help the patient and/or his or her family understand their current coverage and OOP costs, educate them about the financial assistance options, and offer counseling regarding the possibility of changing or adding insurance benefits, if needed. These services help supplement the Benefits Investigation conducted by your practice or facility. If you have any questions throughout this process, call SMA360° at 1-844-4SPINRAZA (1-844-477-4672), Monday through Friday, from 8:30 AM to 8:00 PM ET, or contact your Biogen representative.
* SMA360° services from Biogen are available only to those who have been prescribed SPINRAZA. SMA360° is intended for US residents only.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONLaboratory testing and monitoring to assess safety should be conducted. Perform a platelet count, coagulation laboratory testing, and quantitative spot urine protein testing at baseline and prior to each dose of SPINRAZA and as clinically needed.Severe hyponatremia was reported in an infant treated with SPINRAZA requiring salt supplementation for 14 months.Cases of rash were reported in patients treated with SPINRAZA. SPINRAZA may cause a reduction in growth as measured by height when administered to infants, as suggested by observations from the controlled study. It is unknown whether any effect of SPINRAZA on growth would be reversible with cessation of treatment.
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
24
Preauthorization/PA
The payer requires that a preauthorization/PA be obtained before the treatment will be approved// Based on the payer’s requirements for authorization, demonstrate that the
treatment is medically necessary based on the patient’s diagnosis, clinical presentation, baseline motor functional testing, duration of symptoms, and current supportive care management
The initial preauthorization/PA
was denied
The payer reviewed your request for a preauthorization/PA and denied it, determining that the treatment was not medically necessary// Determine if the reason for the denial was clerical, clinical, or benefit-driven
– If the denial was for clerical reasons, immediately resubmit the request with the proper information
– If the denial was for clinical reasons, determine what additional information may be required to demonstrate medical necessity
– If the denial was for benefit reasons, call the payer to determine if an exception to the benefit is allowed and the process for such an exception (eg, no out-of-network benefits but only experienced provider is out of network)
// Emphasize in your resubmission that your practice or facility believes the treatment to be medically necessary for your patient
KEY CONSIDERATIONS WHEN MEDICAL NECESSITY IS REQUIREDThe following are situations in which your practice or facility may need to demonstrate medical necessity for SPINRAZA during an appeal. The level of information in the letter will vary based on key areas that the payer requires be addressed to demonstrate medical necessity. Your practice or facility can customize the Letter of Medical Necessity based on the specific needs of the payer and the situation (see page 73 for an example of a Letter of Medical Necessity).
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
25
SELECTED IMPORTANT SAFETY INFORMATIONThe most common adverse reactions (≥20% of SPINRAZA-treated patients and ≥5% more frequently than in control patients) that occurred in the infantile-onset controlled study were lower respiratory infection and constipation. Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (18%) than in control patients (10%). Because patients in this controlled study were infants, adverse reactions that are verbally reported could not be assessed. The most common adverse reactions that occurred in the later-onset controlled study were pyrexia, headache, vomiting, and back pain. Post-lumbar puncture syndrome has also been observed after the administration of SPINRAZA.
Exception based on policy
The payer has a policy for treatment and administration services for SPINRAZA, but your patient does not meet the requirements. However, the prescribing physician feels that the treatment is medically necessary// Point out that the patient requires an exception to the plan’s policy and
provide the clinical rationale demonstrating that treatment with SPINRAZA is clinically appropriate
// Provide documentation or information to demonstrate medical necessity, such as– Diagnostic evidence of SMA, including genetic testing– Clinical presentation and duration of symptoms– Current supportive care management– Expectations of therapy and how efficacy will be measured by the clinician– Other relevant aspects of patient history
Exception based on SOC
restrictions
The payer will not cover the treatment and administration services because it will be administered at an out-of-network or out-of-state facility// Emphasize your opinion that the facility is the most appropriate center
to deliver the highly specialized services that may be provided when administering SPINRAZA
// Point out that the patient’s plan does not, in your opinion, currently have an appropriate specialized center to treat SMA in the network and/or state, and that the patient has no other choice but to go out of his or her current network and/or state
// Point out continuity-of-care concerns for switching the patient to a new provider unfamiliar with the patient’s history
// Indicate that it may be important for the plan to know whether a delay in treatment will impact a patient's function or disability
// Provide documentation or information to demonstrate medical necessity, such as– Name and specialty area of your practice or facility to demonstrate
its level of expertise– Distance the patient needs to travel to your practice or facility because there
are no specialized facilities in his or her network and/or state – Areas of medical specialization and years of experience treating patients with SMA
KEY CONSIDERATIONS WHEN MEDICAL NECESSITY IS REQUIRED (cont’d)
When an ME/appeal is denied due to clinical reasons and the submission of an exception to benefit request has been disallowed or denied, the prescribing physician may contact the insurance carrier directly to speak with a clinical representative, who is typically a medical director or someone with a medical background. This is called a peer-to-peer discussion.// A peer-to-peer discussion can be an effective way to help the health plan understand the patient�s unique
medical history, relevant clinical factors, and how those factors support treatment with SPINRAZA// If the HCP is able to obtain the direct contact information for the clinical representative, consider tracking that
information to use for future peer-to-peer discussions
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
SELECTED IMPORTANT SAFETY INFORMATIONCoagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 24 of 146 SPINRAZA-treated patients (16%) with high, normal, or unknown platelet count at baseline developed a platelet level below the lower limit of normal, compared to 10 of 72 sham-controlled patients (14%). Two SPINRAZA-treated patients developed platelet counts <50,000 cells per microliter, with the lowest level of 10,000 cells per microliter recorded on study day 28.
26
OBTAINING APPROPRIATE AUTHORIZATIONS
When requesting a preauthorization or PA it is important to understand that each payer has different requirements with which your practice or facility must be familiar.
When obtaining details on the preauthorization or PA process, your practice or facility will need to
Determine if the information can be phoned in, faxed, emailed, or submitted through the insurer’s website
Find out how long it will take for a decision to be made
Identify the SOC for SPINRAZA administration, especially if it is in a different state than the patient lives
Keep a copy of everything that is submitted relevant to the authorization
Log any calls your office makes about the request
Follow up with the payer if your practice or facility does not receive notification of the decision in a timely manner
A PREAUTHORIZATION VS PRECERTIFICATIONA preauthorization or PA is a request for the insurance company’s approval of the coverage of a drug and/or treatment before the treatment is administered.3
A precertification is similar to a preauthorization/PA in that the insurance company is requiring approval of the drug and/or treatment before it is provided. A payer may also refer to a precertification as a predetermination. In the case of a precertification, the insurer may require documentation that the treatment is medically necessary6; in most cases, this would be included in the form of a Letter of Medical Necessity (see page 73 for an example of this letter).
The important thing to remember about preauthorization and precertification is that approval is required prior to treatment to indicate there is medical necessity for treatment.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONRenal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. SPINRAZA is present in and excreted by the kidney. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 71 of 123 SPINRAZA-treated patients (58%) had elevated urine protein, compared to 22 of 65 sham-controlled patients (34%).
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
27
EXAMPLES OF AUTHORIZATION DOCUMENTATION AND COVERAGE PARAMETERS
REAUTHORIZATION FOR SPINRAZAFor many drugs that treat rare diseases, insurance carriers may require an authorization renewal after a certain period (typically 1 year, but it may be less). This is true regardless of whether the patient is remaining on the same treatment or transitioning to another treatment.// For patients continuing on therapy, medical plans have different intervals for reauthorization
– It is important to become familiar with the medical policy for SPINRAZA at the patient’s health plan– Be mindful of the duration of coverage at the health plan and the patient's start date
// Health plans vary widely in their requirements for reauthorization, but generally include diagnostic criteria and documentation of efficacy– A diagnosis of SMA Type 1, 2, 3, or 4, which would include genetic testing confirming the diagnosis – Efficacy of SPINRAZA is typically documented in terms of maintenance of or improvements in motor
milestones as documented in functional exam testing// Providing documentation of efficacy to health plans in a timely fashion may help enable the patient to
continue treatment uninterrupted– Clinical evaluations, such as evidence of progress in meeting motor milestones, should be conducted
continually, in line with the patient's health plan– Evidence of efficacy, such as maintenance of or improvement in motor function, can provide critical
support for reauthorization // The dosing schedule for SPINRAZA has implications for reauthorization
– Patients treated with SPINRAZA are given a loading dose on approximately days 0, 14, 28, and 58, and a maintenance dose every 4 months thereafter1
– If reauthorization is not achieved in time, the patient’s coverage may end– For plans requiring renewal every 6 months, initial approval coverage may end before maintenance
dosing begins– For plans requiring renewal every 12 months, initial approval coverage may end while patient is
between maintenance doses
Identify specific documentation thatmust be submitted with the request
// Letter of Medical Necessity// Chart notes// Specific payer preauthorization/PA form// SPINRAZA Prescribing Information// Relevant literature, including previously published standards of care// Clinical documentation related to the disease, including
– Diagnostic evidence of SMA, such as genetic testing– Clinical presentation and duration of symptoms– Current supportive care management– Baseline motor function measurement– Other relevant aspects of patient history
Determine the preauthorization/PA coverage parameters
// Number of doses// Time limits of authorization// Diagnosis limitations// Submission requirements
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONLaboratory testing and monitoring to assess safety should be conducted. Perform a platelet count, coagulation laboratory testing, and quantitative spot urine protein testing at baseline and prior to each dose of SPINRAZA and as clinically needed.Severe hyponatremia was reported in an infant treated with SPINRAZA requiring salt supplementation for 14 months.Cases of rash were reported in patients treated with SPINRAZA. SPINRAZA may cause a reduction in growth as measured by height when administered to infants, as suggested by observations from the controlled study. It is unknown whether any effect of SPINRAZA on growth would be reversible with cessation of treatment.
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
28
MOTOR MILESTONE ASSESSMENT TESTS FOR SMAThere are a variety of functional tests that can be used to assess patients with SMA. Because motor milestones in infants and children with SMA vary significantly, there is not one standardized functional assessment used in clinical practice.7,8 These tests evaluate a range of motor functions and are appropriate for different populations with SMA.8
SUMMARY OF MOTOR FUNCTIONAL TESTS FOR SMA
The Hammersmith Infant Neurological
Examination Section 2 (HINE-2)
Age 2 months to 24 months
Measures neuromuscular development in infants, including voluntary grasp, sitting, ability to kick, crawling, head control, standing, rolling, and walking.9
A 1-point increase in HINE score represents increased level of ability.10
World Health Organization (WHO) Motor Milestones
Age 4 months to 24 months
Compares the actual windows of childhood development with those used in assessments of motor skills.11
The 6 WHO motor milestones are measured and compared across similar populations in different countries.11
The Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders
(CHOP INTEND)Infants and children
The first SMA-specific test to assess patients with limited motor function; can measure response to gains and losses in motor function over time.8
Includes 16 items that may be graded between 0 and 4, contributing to a total score of 64 points.8
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
29
SELECTED IMPORTANT SAFETY INFORMATIONThe most common adverse reactions (≥20% of SPINRAZA-treated patients and ≥5% more frequently than in control patients) that occurred in the infantile-onset controlled study were lower respiratory infection and constipation. Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (18%) than in control patients (10%). Because patients in this controlled study were infants, adverse reactions that are verbally reported could not be assessed. The most common adverse reactions that occurred in the later-onset controlled study were pyrexia, headache, vomiting, and back pain. Post-lumbar puncture syndrome has also been observed after the administration of SPINRAZA.
HOW OFTEN DOES FUNCTIONAL TESTING NEED TO BE DONE?Functional testing information is extremely important to include with all requests for authorizations and reauthorizations. It is recommended that testing be performed within 60 days of the intended dose, so it is important to remember not to send in authorization requests too early. In the case of a delay that affects the date of treatment, be sure to obtain new baseline functional exam scores prior to starting therapy because the patient’s motor skills may have further declined during that period of no treatment. If changes are not documented, reauthorization may be denied because lower, undetected baseline scores could make the patient’s progress appear inadequate according to the plan’s minimum efficacy standard.
SUMMARY OF MOTOR FUNCTIONAL TESTS FOR SMA (cont’d)
The Revised Upper Limb Module (RULM)
Age >36 months
Assesses upper limb function in ambulatory and nonambulatory patients with SMA.12
Nineteen items are graded on a 3-point scale, with a score of 0 (unable), 1 (able with modification), or 2 (able, no difficulty). The maximum total score is 37, which includes a can/cannot score of 1 or 0 for the first item in the assessment.12
6-Minute Walk Test (6MWT)
Ambulatory patients
Measures the distance in meters a patient can walk unassisted.14
Participants walk unaided for 25 meters; distance walked over 6 minutes, distance covered each minute, (patients can rest without sitting), and time to complete the 25-meter course are recorded. Falls are recorded.14
The Hammersmith Functional Motor Scale—Expanded (HFMSE)Ambulatory patients with SMA
Type 2 or Type 3
Assesses gross motor function of ambulatory patients.7
A 2-point change is clinically relevant (eg, a child who was previously not able to crawl has increased crawling ability).13
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
SELECTED IMPORTANT SAFETY INFORMATIONCoagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 24 of 146 SPINRAZA-treated patients (16%) with high, normal, or unknown platelet count at baseline developed a platelet level below the lower limit of normal, compared to 10 of 72 sham-controlled patients (14%). Two SPINRAZA-treated patients developed platelet counts <50,000 cells per microliter, with the lowest level of 10,000 cells per microliter recorded on study day 28.
30
CLAIM RECONSIDERATION AND APPEALS CHECKLISTIf a claim has been denied, you can request an appeal. There are several common reasons that claims are denied, such as an incorrect patient identification number or omission of a Letter of Medical Necessity. Another reason that a claim for SPINRAZA may be denied is that the product is not yet being covered under the insurer’s coverage benefit. In each of these cases, it is important to consider an appeal.
The following are some considerations for understanding and filing an appeal.
Review the EOB/RA to understand
the reason for the denial
SOME TOP REASONS THAT CLAIMS ARE DENIED: Incorrect codes Missing information Incorrect product information Lack of a Letter of Medical Necessity
If additional information is requested, submit the necessary documentation immediately.
Verify the appeals process
for the payer
CONSIDER THE FOLLOWING TO UNDERSTAND THE APPEALS PROCESS OF EACH PAYER:
Is there a need for a particular form? How should the form be sent to the payer? Can the appeal take place over the phone via a physician-to-physician call with the payer? Who should receive the appeal (name, title, and contact information)? What must accompany the appeal (eg, supporting documentation)? How long does the appeals process usually take? How will I learn about the appeal decision?
RECORD THE CORRESPONDENCE WITH THE PAYER AT EVERY POINT OF THE APPEALS PROCESS
IF YOUR CLAIM IS DENIED A SECOND TIME, DETERMINE IF A NEXT-LEVEL APPEAL IS ALLOWED AND CAREFULLY SUBMIT IT WITHIN YOUR PAYER’S TIMELINES. REQUEST ASSISTANCE FROM YOUR BIOGEN REPRESENTATIVE IF NEEDED
EOB=explanation of benefits; RA=remittance advice.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONRenal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. SPINRAZA is present in and excreted by the kidney. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 71 of 123 SPINRAZA-treated patients (58%) had elevated urine protein, compared to 22 of 65 sham-controlled patients (34%).
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
31
FOR CONTINUED APPROVAL OF MAINTENANCE THERAPY WITH SPINRAZAWhen translating test scores into support for an ME or appeal, highlight all improvements in functional measurements compared with baseline. Because the criteria of the SMA functional tests vary, outline cumulative gain in function that the patient achieved according to earlier tests. For example, if a patient has gained the ability to hold up his/her head according to HINE-2, note this even if the patient most recently achieved hand grip according to CHOP INTEND.
Children with advanced SMA may not express improvements from baseline based on the traditional scales, but rather may show // Subtle improvements or preservation of residual distal muscles// Subtle changes that may impact activities of daily living that are relevant to the patient
Any improvement may be significant to the patient and contrary to the natural history of the disease. Therefore, it is important to document these changes for the health plan to support the medical necessity of treatment with SPINRAZA
FOR INITIAL AUTHORIZATION FOR SPINRAZAInclude the following to help support medical necessity when an initial authorization request has been denied:
Test scores establishing baseline measurements, eg, HINE-2, CHOP INTEND, and WHO
HCP observations
The HCP’s opinion of the anticipated course of SMA for the patient with and without treatment
ME/APPEAL CONSIDERATIONS FOR PEDIATRIC PATIENTS WITH SMA
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONLaboratory testing and monitoring to assess safety should be conducted. Perform a platelet count, coagulation laboratory testing, and quantitative spot urine protein testing at baseline and prior to each dose of SPINRAZA and as clinically needed.Severe hyponatremia was reported in an infant treated with SPINRAZA requiring salt supplementation for 14 months.Cases of rash were reported in patients treated with SPINRAZA. SPINRAZA may cause a reduction in growth as measured by height when administered to infants, as suggested by observations from the controlled study. It is unknown whether any effect of SPINRAZA on growth would be reversible with cessation of treatment.
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
32
ME/APPEAL CONSIDERATIONS FOR TEENAGERS AND ADULTS WITH SMAIt is very important to help the payer understand that the available functional tests for SMA may not be robust enough to translate functional improvement or maintenance for teenagers or adults with SMA. Payers need to understand that when a patient achieves a high score on a functional test at baseline, there may not be enough incremental difference on the scale to demonstrate the improvement that may be required by the payer.
However, there are functions that could either be achieved and/or maintained in older patients that may not be demonstrated on a scale at all, but are extremely important and medically necessary to the patient.
The Letter of Medical Necessity/Appeal Template can be reviewed on page 73 of this guide and is available to download at SPINRAZA-hcp.com or from your Biogen representative.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
33
SELECTED IMPORTANT SAFETY INFORMATIONThe most common adverse reactions (≥20% of SPINRAZA-treated patients and ≥5% more frequently than in control patients) that occurred in the infantile-onset controlled study were lower respiratory infection and constipation. Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (18%) than in control patients (10%). Because patients in this controlled study were infants, adverse reactions that are verbally reported could not be assessed. The most common adverse reactions that occurred in the later-onset controlled study were pyrexia, headache, vomiting, and back pain. Post-lumbar puncture syndrome has also been observed after the administration of SPINRAZA.
IF AN INTERNAL APPEAL IS DENIED, CONSIDER AN EXTERNAL REVIEWAppeals should follow the individual payer’s requirement(s) and include additional information that continues to emphasize the medical necessity of SPINRAZA for your patient. At the end of the internal appeals process, the health plan must provide you and your patient with a written decision, but this does not mean that the appeals process is over. There may be other courses of action, such as an independent external review.
// In an external review process, an independent, accredited medical professional will review your patient's case. The reviewer does not receive any financial incentives to perform the review. The insurance carrier is required by law to accept the reviewer’s decision15
// To request an external review, most plans require that the patient file a written request within 45 days following the insurance carrier’s final determination. The letter sent to you and your patient should describe how to request an external review15
// External review decisions are made as soon as possible and should take no longer than 45 days from receipt of request
EXPEDITED REVIEWS Expedited reviews can be requested in the case of an urgent situation in which a delay in treatment would cause the patient to lose motor function, cause further disability in the patient, or risk the life of the patient.
// Reviews can be expedited by requesting that an external review be done simultaneously with the internal review. An expedited appeal may be granted if your patient is currently receiving or waiting to start a prescribed treatment and you believe a delay would be life threatening, affect the patient�s ability to regain maximum function, or subject him or her to severe pain. The request for an expedited appeal may be made verbally. The health plan must make a decision within 4 business days after your patient�s request is received16
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
SELECTED IMPORTANT SAFETY INFORMATIONCoagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 24 of 146 SPINRAZA-treated patients (16%) with high, normal, or unknown platelet count at baseline developed a platelet level below the lower limit of normal, compared to 10 of 72 sham-controlled patients (14%). Two SPINRAZA-treated patients developed platelet counts <50,000 cells per microliter, with the lowest level of 10,000 cells per microliter recorded on study day 28.
34
YOUR BIOGEN REPRESENTATIVE IS HERE TO HELP
If you have any questions throughout this process, call SMA360°* at 1-844-4SPINRAZA (1-844-477-4672) or contact your Biogen representative.
For more information about an external claim review, go to healthcare.gov/appeal-insurance-company-decision/external-review/ or contact your Biogen RDRM for assistance.†
* SMA360° services from Biogen are available only to those who have been prescribed SPINRAZA. SMA360° is intended for US residents only.
† The link above will take you to a website that is outside the control of Biogen. Links are provided as a courtesy for informational purposes only. We do not make or imply any endorsement of external websites.
3535
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
35
NAVIGATINGFINANCIALASSISTANCEOPTIONS
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONRenal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. SPINRAZA is present in and excreted by the kidney. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 71 of 123 SPINRAZA-treated patients (58%) had elevated urine protein, compared to 22 of 65 sham-controlled patients (34%).
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
36
Patient cost-sharing
considerations
Copay: Typically, a flat fee that patients pay each time they receive medical care. The copay may be in addition to other OOP costs, such as deductibles and coinsurance, and it varies by benefit structure
Coinsurance: A beneficiary cost-sharing amount that begins after the deductible is paid. Coinsurance typically is based on a percentage of the cost of services and varies by payer
Deductible: A predetermined amount of money that the patient must spend before his or her payer benefits take effect
Maximum OOP cost: An annual limitation on all cost sharing that patients are responsible for under a health insurance plan. This limit does not apply to premiums, balance-billed charges from out-of-network HCPs, or services that are not covered by the plan
In addition to the Benefits Investigation conducted by your practice or facility, SMA360° will investigate patient benefits in order to be able to inform the patient’s family about potential cost-sharing responsibility and to discuss potential implications.
Navigating Financial Assistance OptionsThe SMA360°* team can help your patients’ families navigate the cost of treatment with SPINRAZA. Patients may have a copay or coinsurance for the drug and/or for the administration of SPINRAZA after they meet their annual deductible and until they reach the annual limit for their maximum OOP costs.
Biogen believes that cost should not be a barrier to treatment. SMA360° offers personalized insurance and financial assistance to help your patients’ families understand their insurance benefits for SPINRAZA and to identify the most affordable way to start and stay on treatment as prescribed by their doctor.
PATIENT COST-SHARING STRUCTURE CONSIDERATIONSDuring the Benefits Investigation, it is important to determine key elements of the cost-sharing structure under the patient’s insurance benefits, including the following:
* SMA360° services from Biogen are available only to those who have been prescribed SPINRAZA. SMA360° is intended for US residents only.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
37
SELECTED IMPORTANT SAFETY INFORMATIONLaboratory testing and monitoring to assess safety should be conducted. Perform a platelet count, coagulation laboratory testing, and quantitative spot urine protein testing at baseline and prior to each dose of SPINRAZA and as clinically needed.
Biogen SPINRAZA Copay Assistance Program: Generally, all individuals on nongovernment insurance are eligible, regardless of income, and there is no annual maximum on the amount Biogen will cover as part of the program. Insurance will be billed first and must pay before copay assistance will be applicable.
Patients who have primary prescription drug coverage through a commercial insurer (under either a pharmacy benefit or a medical benefit, as determined by the insurer) and secondary coverage through Medicaid or another government payer may be eligible as long as reimbursement for SPINRAZA is not provided in whole or in part by Medicaid or other government payer.
Biogen SPINRAZA Procedure Copay Assistance Program: In addition to the above criteria, individuals are eligible for this program if they meet the following requirements: // They are not a resident of Massachusetts, Michigan, Minnesota, or Rhode Island// The HCP submits a request for treatment using an approved procedure code for
anesthesia, imaging procedures, and/or surgical procedure/drug administration // The following procedure codes approved by Biogen are eligible for the program
// Anesthesia17,18
– Lumbar region—00635– Extreme age—99100– Revenue Code—370
// Imaging Procedure/Guidance17
– Fluoroscopy—77003– Ultrasound—76942– CT guidance—77012
// Surgical Procedure and Drug Administration1
Third-Party Funding Assistance: Financial assistance for patients may be available from a third party if it is determined that a family is not eligible for the Biogen SPINRAZA Copay Assistance Program and/or the Biogen SPINRAZA Procedure Copay Assistance Program.
SMA360°™* FINANCIAL ASSISTANCE AND INSURANCE COUNSELING SERVICESBiogen provides several comprehensive financial support services to help reduce nonclinical barriers to patient access, which may allow commercially insured patients to lower their out-of-pocket costs to as little as $0.
Patients are required to enroll separately in each Biogen financial assistance program. Your Biogen representative is available to provide you with additional information about financial resources for your patients. See page 72 for an example of the SPINRAZA Copay Reimbursement Form, which is used for both Biogen copay programs. A copy of the form is available from your Biogen representative.CT=computed tomography.
– Intrathecal drug administration—96450– Lumbar puncture, diagnostic—62270†
– Lumbar puncture, therapeutic—62272†
– Injection(s), without imaging guidance—62322– Injection(s), with imaging guidance—62323– Spinal puncture, lumbar, with imaging
guidance–62328‡
– Spinal puncture, therapeutic, with imaging guidance–62329§
* SMA360° services from Biogen are available only to those who have been prescribed SPINRAZA. SMA360° is intended for US residents only.
†If imaging guidance is being used, use codes 62328 or 62329 as appropriate.‡ Do not report 62270 or 62328 in conjunction with 77003 or 77012. If ultrasound or magnetic resonance imaging (MRI) guidance is performed, see 76942 and 77021.
§Do not report 62272 or 62329 in conjunction with 77003 or 77012. If ultrasound or MRI guidance is performed, see 76942 and 77021.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONSevere hyponatremia was reported in an infant treated with SPINRAZA requiring salt supplementation for 14 months.Cases of rash were reported in patients treated with SPINRAZA.
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
38
INITIATING THE BIOGEN SPINRAZA COPAY ASSISTANCE PROGRAMThis program generally is available for patients with nongovernmental insurance benefits who have provided consent to Biogen. It covers the amount of cost sharing for SPINRAZA, but does not cover administration-related costs. After conducting a Benefits Investigation, the SMA360°* team will contact eligible patients to introduce the program and to complete enrollment.
WHAT YOUR PRACTICE OR FACILITY NEEDS TO DO
1
Confirm patient enrollment
Confirm that the patient is enrolled in the Biogen SPINRAZA Copay Assistance Program for every treatment dose. At enrollment, the patient and HCP will receive a confirmation letter via fax from Biogen. This information also is available through your Biogen representative.
// Keep the confirmation of enrollment in the patient’s file. If the patient withdraws, Biogen will send a withdrawal letter. This information is also available by calling 1-844-4SPINRAZA (1-844-477-4672)
An EOB is a statement sent by a health plan to a member to describe what medical treatments and/or services were paid on his or her behalf. The EOB may also be called remittance advice and usually is used with Medicare and Medicaid payments. Ask your patient for his or her EOB/RA regarding SPINRAZA treatment.
2
Obtain EOB/RA
Locate the EOB/RA demonstrating the patient’s financial responsibility for SPINRAZA
3
Fill out the Copay Reimbursement
Form
Fill out the Copay Reimbursement Form
4
Submit for reimbursement
// Fax the EOB/RA and the completed Copay Reimbursement Form to Biogen at 1-888-656-4343
// Your practice or facility will receive a reimbursement check for plans that cover SPINRAZA under the medical benefit. For plans that cover SPINRAZA under the pharmacy benefit, Accredo SP manages the adjudication via the Rx BIN, PCN, and Group Number
BIN=bank identification number; PCN=processor control number.
*SMA360° services from Biogen are available only to those who have been prescribed SPINRAZA. SMA360° is intended for US residents only.
If you have any questions throughout this process, call SMA360° at 1-844-4SPINRAZA (1-844-477-4672) or contact your Biogen representative.SMA360° offers insurance counseling services to help patients’ families understand their current insurance benefits for SPINRAZA and to provide assistance with changing or adding supplemental insurance benefits, such as Medicaid.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONSPINRAZA may cause a reduction in growth as measured by height when administered to infants, as suggested by observations from the controlled study. It is unknown whether any effect of SPINRAZA on growth would be reversible with cessation of treatment.
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
39
INITIATING THE BIOGEN SPINRAZA PROCEDURE COPAY PROGRAMThis program generally is available for patients with nongovernmental insurance benefits who have provided consent to Biogen. It covers the amount of cost sharing for the administration procedure that is associated with SPINRAZA, but it does not cover the cost of the drug. After conducting a Benefits Investigation, the SMA360°™* team will contact eligible patients to introduce the program and to complete enrollment.
WHAT YOUR PRACTICE OR FACILITY NEEDS TO DO
The eligibility criteria differ for the Biogen SPINRAZA Copay Program and the Biogen SPINRAZA Procedure Copay Program. For more information, consult with your Biogen representative.
1
Confirm patient enrollment
Confirm that the patient is enrolled in the Biogen SPINRAZA Procedure Copay Program for every treatment dose. At enrollment, the patient and HCP will receive a confirmation letter via fax from Biogen. This information also is available through your Biogen representative
// Keep the confirmation of enrollment in the patient’s file. If the patient withdraws, Biogen will send a withdrawal letter. This information also is available by calling 1-844-4SPINRAZA (1-844-477-4672)
2
Obtain EOB
Locate the provider/facility RA and/or the patient’s EOB demonstrating the patient’s financial responsibility for SPINRAZA
3
Fill out the Copay Reimbursement
Form
Fill out the Copay Reimbursement Form
4
Submit for reimbursement
Fax the EOB/RA and the completed Copay Reimbursement Form to Biogen at 1-888-656-4343
* SMA360° services from Biogen are available only to those who have been prescribed SPINRAZA. SMA360° is intended for US residents only.
4040
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
ORDERINGSPINRAZA® (nusinersen)
40
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
41
SELECTED IMPORTANT SAFETY INFORMATIONThe most common adverse reactions (≥20% of SPINRAZA-treated patients and ≥5% more frequently than in control patients) that occurred in the infantile-onset controlled study were lower respiratory infection and constipation. Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (18%) than in control patients (10%). Because patients in this controlled study were infants, adverse reactions that are verbally reported could not be assessed. The most common adverse reactions that occurred in the later-onset controlled study were pyrexia, headache, vomiting, and back pain. Post-lumbar puncture syndrome has also been observed after the administration of SPINRAZA.
Ordering SPINRAZAThe SMA360°™* team will contact the enrolled patient’s family to coordinate treatment logistics for each SPINRAZA administration visit. It is important for your office to coordinate with your Biogen representative when ordering SPINRAZA so that the patient’s family can be prepared for the visit.
HOW TO ORDER SPINRAZACuraScript SD and Accredo SP are the exclusive authorized providers of SPINRAZA. Ordering SPINRAZA is done in the same way as any other product that is administered at your SOC, whether it is an outpatient hospital-based facility,† physician office, freestanding ASC, or inpatient hospital facility. SPINRAZA can be ordered directly through CuraScript SD or from Accredo SP. Once the order for SPINRAZA has been submitted to your pharmacy or procurement department, the order for SPINRAZA will be placed.
SPINRAZA ORDERING CHECKLIST
CONFIRM THAT YOUR PRACTICE OR FACILITY IS READY TO ORDER SPINRAZA
Benefits Investigation has been conducted
Payer approval of appropriate authorizations has been obtained
(Optional) Patient has been enrolled in available financial assistance program(s)
ORDER SPINRAZA FROM CURASCRIPT SD OR ACCREDO SP
Follow the standard process for placing a prescription drug order in your practice or facility
// The SPINRAZA Start Form includes a prescription for SPINRAZA. However, some states may require a separate prescription to be sent to Accredo SP
Your pharmacy or procurement department will need to submit the order form to CuraScript SD
// 1-855-778-1510 (phone)
// 1-888-538-9781 (fax)
COORDINATE SPINRAZA SHIPMENT DELIVERY WITH THE SCHEDULED PATIENT TREATMENT VISIT
CuraScript SD or Accredo SP will ship SPINRAZA in a temperature-controlled container directly to your practice or facility
Make sure there is a staff member available to accept delivery of SPINRAZA and to transfer the product to a refrigerated space in the pharmacy immediately upon receipt of the drug
Coordinate the treatment procedure for SPINRAZA with your site’s care team, including the pharmacy
For assistance with any step in this process, contact your Biogen representative.
* SMA360° services from Biogen are available only to those who have been prescribed SPINRAZA. SMA360° is intended for US residents only.
†Including off-campus clinic, on-campus facility, hospital-based ASC, and other outpatient outlets operated by a hospital.
42
Please see additional Important Safety Information on page 76 and enclosed full Prescribing Information.
42
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
42
SUBMITTING CLAIMS FOR SPINRAZA® (nusinersen) AND RELATED SERVICES
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
SELECTED IMPORTANT SAFETY INFORMATIONCoagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 24 of 146 SPINRAZA-treated patients (16%) with high, normal, or unknown platelet count at baseline developed a platelet level below the lower limit of normal, compared to 10 of 72 sham-controlled patients (14%). Two SPINRAZA-treated patients developed platelet counts <50,000 cells per microliter, with the lowest level of 10,000 cells per microliter recorded on study day 28.
43
Hospital facilities and hospital-based ASCs may submit a CMS-1450/UB-04 claim form19,20
Physician office practices may submit a CMS-1500 claim form either for professional services related to drug administration or for the drug and the services related to drug administration19,20
Freestanding ASCs may submit a CMS-1500 claim form for the medication and the services related to drug administration19,20
Following Payer Billing Guidelines Can Facilitate Claim Processing and Prompt PaymentWhen a patient has been administered the SPINRAZA injection and/or a related service, your practice or facility may submit a claim to the patient’s insurance plan. Items included on your claim may depend on the SOC and the billing entity.
The information within this section reviews some of the billing codes relevant for SPINRAZA and the related administration services, as well as key billing considerations across SOCs. However, coding and billing recommendations may vary by payer. Your practice or facility should check directly with the patient’s payer(s) to verify specific coding and billing requirements. Biogen field representatives are available to answer questions and further support the reimbursement process.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONRenal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. SPINRAZA is present in and excreted by the kidney. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 71 of 123 SPINRAZA-treated patients (58%) had elevated urine protein, compared to 22 of 65 sham-controlled patients (34%).
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
44
HCPCS=Healthcare Common Procedure Coding System; ICD-10-CM=International Classification of Diseases, Tenth Revision, Clinical Modification.
ICD-10-CM CODE EXAMPLES
ICD-10-CM Code21 Description21
G12.0 Infantile spinal muscular atrophy, type I [Werdnig-Hoffmann]
G12.1
Other inherited spinal muscular atrophy Adult form spinal muscular atrophyChildhood form, type II spinal muscular atrophyDistal spinal muscular atrophy (NEW)Juvenile form, type III spinal muscular atrophy [Kugelberg-Welander]Progressive bulbar palsy of childhood [Fazio-Londe]Scapuloperoneal form spinal muscular atrophy
NOTE: Be sure to use the appropriate code for SMA diagnosis so it will be accepted on the claim form by the payer. Using codes G12.8 (other spinal muscular atrophies and related syndromes) or G12.9 (SMA, unspecified) may result in a claim denial because SMA type is not specified.
HCPCS CODE
HCPCS Code22 Description22
J2326 Injection, nusinersen, 0.1 mg
NDC NUMBER
NDC Number1
Description1
10-digit format 11-digit format
64406-058-01 64406-0058-0112 mg/5 mL single-dose vial (contains 12 mg of nusinersen solution for intrathecal injection)
Although the FDA uses a 10-digit format when registering NDC numbers, payers often require an 11-digit NDC format on claim forms for billing purposes.23 It is important to confirm with your payer which NDC format is required. In addition, Medicaid requires that all claims for provider-administered drugs include NDC numbers.
This reporting requirement may also be implemented by some commercial payers.24 Guidelines for reporting the NDC number in the appropriate format, quantity, and unit of measure25 vary by state and by payer and should be reviewed prior to submitting a claim.
Summary of Relevant Codes for SPINRAZA
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONLaboratory testing and monitoring to assess safety should be conducted. Perform a platelet count, coagulation laboratory testing, and quantitative spot urine protein testing at baseline and prior to each dose of SPINRAZA and as clinically needed.
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
45
CPT® CODE EXAMPLES
Procedure Type17 CPT® Code17 Description17
Relevant CPT® Codes for SPINRAZA
IMPORTANT: Note that 62270 (spinal puncture, lumbar, diagnostic) and 62272 (spinal puncture, therapeutic, for drainage of cerebrospinal fluid [by needle or catheter]) are still valid codes. These codes should now be used only if there is no imaging guidance (fluoroscopic or CT) used during the procedure.
Surgical Procedure WITHOUT Imaging Guidance
62270* Spinal puncture, lumbar, diagnostic
62272* Spinal puncture, therapeutic, for drainage of cerebrospinal fluid (by needle or catheter)
62322
Injection(s), of diagnostic or therapeutic substance(s) (eg, anesthetic, antispasmodic, opioid, steroid, other solution), not including neurolytic substances, including needle or catheter placement, interlaminar epidural or subarachnoid, lumbar or sacral (caudal); without imaging guidance
96450 Chemotherapy administration, into CNS (eg, intrathecal), requiring and including spinal puncture
Imaging Procedure/Guidance
76942Ultrasonic guidance for needle placement (eg, biopsy, aspiration, injection, localization device), imaging supervision and interpretation
77003Fluoroscopic guidance and localization of needle or catheter tip for spine or paraspinous diagnostic or therapeutic injection procedures (epidural or subarachnoid)
77012CT guidance for needle placement (eg, biopsy, aspiration, injection, localization device), radiological supervision and interpretation
77021MRI guidance for needle placement (eg, for biopsy, needle aspiration, injection, or placement of localization device) radiological supervision and interpretation
Surgical Procedure WITH Imaging Guidance
62323 Injection(s) of diagnostic or therapeutic substance(s) as described in code 62322; with imaging guidance
62328† Spinal puncture, lumbar, diagnostic; with fluoroscopic or CT guidance
62329‡ Spinal puncture, therapeutic, for drainage of cerebrospinal fluid (by needle or catheter); with fluoroscopic or CT guidance
CNS=central nervous system.* If imaging guidance is being used, use codes 62328 or 62329 as appropriate.†Do not report 62270 or 62328 in conjunction with 77003 or 77012. If ultrasound or MRI guidance is performed, see 76942 and 77021.‡Do not report 62272 or 62329 in conjunction with 77003 or 77012. If ultrasound or MRI guidance is performed, see 76942 and 77021.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONSevere hyponatremia was reported in an infant treated with SPINRAZA requiring salt supplementation for 14 months.Cases of rash were reported in patients treated with SPINRAZA. SPINRAZA may cause a reduction in growth as measured by height when administered to infants, as suggested by observations from the controlled study. It is unknown whether any effect of SPINRAZA on growth would be reversible with cessation of treatment.
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
46
CPT® CODE EXAMPLES (cont’d)
Procedure Type17 CPT® Code17 Description17
Relevant CPT® Codes for SPINRAZA (cont’d)
Anesthesia
00635 Anesthesia for procedures in lumbar region (diagnostic or therapeutic lumbar puncture)
99100Anesthesia for patient of extreme age, younger than 1 year and older than 70 years (list separately in addition to code for primary anesthesia procedure)
Moderate (Conscious) Sedation
99151-99153,99155-99157
Drug-induced depression of consciousness during which patients respond purposefully to verbal commands, either alone or accompanied by light tactile stimulation. Coding is based on total intra-service time and the healthcare professional who is performing the procedure. For descriptions of individual codes, refer to the CPT® 2020 Professional Edition
Outpatient Hospital Observation Status
99218-99220
Initial observation care, per day, for the evaluation and management of a patient which requires these 3 key components: a detailed or comprehensive history; a detailed or comprehensive examination; and varying levels of medical decision-making complexity
99217
Observation care discharge day management (this code is to be utilized to report all services provided to a patient on discharge from outpatient hospital observation status if the discharge is on a day other than the initial date of observation status)
99234-99236
Observation or inpatient hospital care is used for the evaluation and management of a patient who is admitted and discharged on the same date, which requires these 3 key components: a detailed or comprehensive history; a detailed or comprehensive examination; and varying levels of medical decision-making complexity
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
47
CODING GUIDE FOR MODERATE SEDATION OF LESS THAN 52 MINUTES17
Total intraservice time for MS* Patient age Code(s) Code(s)
<10 minutes Any age Not reported separately
Not reported separately
10-22 minutes <5 years 99151 99155
10-22 minutes ≥5 years 99152 99156
23-37 minutes <5 years 99151 + 99153 x 1 99155 + 99157 x 1
23-37 minutes ≥5 years 99152 + 99153 x 1 99156 + 99157 x 1
38-52 minutes <5 years 99151 + 99153 x 2 99155 + 99157 x 2
38-52 minutes ≥5 years 99152 + 99153 x 2 99156 + 99157 x 2
CONSIDERATIONS FOR ADMINISTRATIONSPINRAZA is administered intrathecally by, or under the direction of, an HCP with experience performing lumbar punctures. In addition, providers can consider the following services for the administration of SPINRAZA, as needed1:// Sedation as indicated by the clinical condition of the patient// Ultrasound or other imaging techniques to guide intrathecal administration of SPINRAZA
Moderate sedation (MS) is a drug-induced semiconscious state that allows patients to be comfortable during certain surgical or medical procedures. MS requires no interventions to maintain cardiovascular function or a patent airway, and spontaneous ventilation is adequate.17
Coding for MS is based on total intraservice time and the HCP who performs the procedure.17
SELECTED IMPORTANT SAFETY INFORMATIONThe most common adverse reactions (≥20% of SPINRAZA-treated patients and ≥5% more frequently than in control patients) that occurred in the infantile-onset controlled study were lower respiratory infection and constipation. Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (18%) than in control patients (10%). Because patients in this controlled study were infants, adverse reactions that are verbally reported could not be assessed. The most common adverse reactions that occurred in the later-onset controlled study were pyrexia, headache, vomiting, and back pain. Post-lumbar puncture syndrome has also been observed after the administration of SPINRAZA.
* For MS coding of 53 minutes or longer, or for descriptions of individual codes, please refer to page 751 of the CPT® 2020 Professional Edition.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
SELECTED IMPORTANT SAFETY INFORMATIONCoagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 24 of 146 SPINRAZA-treated patients (16%) with high, normal, or unknown platelet count at baseline developed a platelet level below the lower limit of normal, compared to 10 of 72 sham-controlled patients (14%). Two SPINRAZA-treated patients developed platelet counts <50,000 cells per microliter, with the lowest level of 10,000 cells per microliter recorded on study day 28.
48
CPT® CODE MODIFIER EXAMPLES
Modifier17 Description17
22 Increased procedural services
23 Unusual anesthesia services
25 Significant, separately identifiable evaluation and management service by the same physician or other qualified HCP on the same day of procedure or other service
51 Multiple procedures
52 Reduced services
53 Discontinued procedure
59 Distinct procedural service
Appropriate modifier(s) can help report additional circumstances under which a specific procedure and/or ancillary services were provided.
Unique Billing Considerations for Outpatient Hospital-Based Facilities
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONRenal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. SPINRAZA is present in and excreted by the kidney. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 71 of 123 SPINRAZA-treated patients (58%) had elevated urine protein, compared to 22 of 65 sham-controlled patients (34%).
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
49
REVENUE CODE EXAMPLES FOR OUTPATIENT HOSPITAL-BASED FACILITIES*
Service Type18 Revenue Code18 Description18
Drug Product 0636 Pharmacy (ie, drugs requiring detailed coding)
Drug Administration, Surgical Procedure, Recovery, and Observation
0331 Radiology/therapeutic (ie, chemotherapy injected)
0361 Operating room services (ie, minor surgery)
0499 Ambulatory surgical care (ie, other ambulatory surgical care)
0710 Recovery room permits identification of particular services, if necessary
0760 Specialty services (ie, general classification)
0762 Specialty services (ie, observation hours)
Anesthesia Services 0370 Anesthesia (ie, general classification)
Imaging Services0402 Other imaging services (ie, ultrasound)
0409 Other imaging services (ie, other imaging services)
CPT® CODE EXAMPLES FOR OUTPATIENT OBSERVATION
Procedure Type17 CPT® Code17 Description17
Outpatient Hospital Observation Status
99218- 99220
Initial observation care, per day, for the evaluation and management of a patient, which requires these 3 key components: a detailed or comprehensive history; a detailed or comprehensive examination; and varying levels of medical decision-making
99217Observation care discharge is to be utilized to report all services provided to a patient on outpatient hospital observation status if the discharge is on other than the initial date of observation status
99234- 99236
Observation or inpatient hospital care is used for the evaluation and management of a patient who is admitted and discharged on the same date, which requires these 3 key components: a detailed or comprehensive history; a detailed or comprehensive examination; and varying levels of medical decision-making complexity
* Including off-campus clinic, on-campus facility, hospital-based ASC, and other outpatient outlets operated by a hospital.
Revenue codes are required for hospital outpatient billing and will vary depending on the revenue center to which your hospital maps SPINRAZA. Typically, SPINRAZA and the procedure involved with its administration will be reported using all of the revenue codes listed above.
Following administration, an additional observational period may be required after treatment administration for patients who have complications or need extended observation (beyond typical recovery time) to determine if the patient can be discharged or if he or she will need in patient admission. Use the CPT® codes listed above for observational stay.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONLaboratory testing and monitoring to assess safety should be conducted. Perform a platelet count, coagulation laboratory testing, and quantitative spot urine protein testing at baseline and prior to each dose of SPINRAZA and as clinically needed.Severe hyponatremia was reported in an infant treated with SPINRAZA requiring salt supplementation for 14 months.Cases of rash were reported in patients treated with SPINRAZA. SPINRAZA may cause a reduction in growth as measured by height when administered to infants, as suggested by observations from the controlled study. It is unknown whether any effect of SPINRAZA on growth would be reversible with cessation of treatment.
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
50
CODING SUMMARY FOR ELECTRONIC CLAIM SUBMISSION BY OUTPATIENT HOSPITAL-BASED FACILITIES*The table below provides examples of relevant codes, along with corresponding locations, for paper and electronic claims submitted by outpatient hospital-based facilities for SPINRAZA and related administration services.
Requirements and location of information will vary by payer.
Examples of Relevant Codes for SPINRAZA and Electronic Billing Locations for Outpatient Hospital-Based Facilities26
Information Sample Code(s) or Information
CMS-1450/ UB-04 Locator26
Electronic Loop26
Equivalent Segment26
HCPCS Level II Code J2326 Field 44 2400 SV202-2
HCPCS Level II Code Units 120 Field 46 2400 SV205
Additional Product Information
SPINRAZA 64406-0058-01
12 mg/5 mL, 5 mL intrathecal inj
Field 80 2300 NTE
CPT® Code(s) 96450
Other CPT® codes may apply, as appropriate
Field 44 2400 SV202-2
ICD-10-CM Code (primary) G12.0 Field 67 2300 HI01-2
Bill Type Code Provider specific† Field 4 2300 CLM05-1
Revenue Code(s)
03610636
Other revenue codes may apply, as appropriate
Field 42 2400 SV201
* Including off-campus clinic, on-campus facility, hospital-based ASC, and other outpatient outlets operated by a hospital.† A 4-digit bill type code documents facility type (second digit after the leading zero), care type (third digit), and the bill sequence for the given episode of care (fourth digit). Relevant examples for outpatient facilities include 013X (hospital outpatient), 074X (clinic outpatient physical therapy [OPT]), and 083X (hospital outpatient ASC), where X represents the sequence of the billing in this particular episode of care (eg, “1” for admit through discharge claim).27
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
51
SELECTED IMPORTANT SAFETY INFORMATIONThe most common adverse reactions (≥20% of SPINRAZA-treated patients and ≥5% more frequently than in control patients) that occurred in the infantile-onset controlled study were lower respiratory infection and constipation. Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (18%) than in control patients (10%). Because patients in this controlled study were infants, adverse reactions that are verbally reported could not be assessed. The most common adverse reactions that occurred in the later-onset controlled study were pyrexia, headache, vomiting, and back pain. Post-lumbar puncture syndrome has also been observed after the administration of SPINRAZA.
Sample CMS-1450/UB-04 Claim Form FOR OUTPATIENT HOSPITAL-BASED FACILITIES*
__ __ __
1
2
3
4
5
6
7
8
9
10
11
12
13
14
15
16
17
18
19
20
21
22
23
A
B
C
A
B
C
A
B
C
APPROVED OMB NO.
__
1 2 4 TYPE OF BILL
FROM THROUGH 5 FED. TAX NO.
a
b
c
d
DX
ECI
A B C D E F G H I J K L M N O P Q
a b c
a
b c d
ADMISSION CONDITION CODES DATE 12
OCCURRENCE OCCURRENCE33 OCCURRENCE OCCURRENCE SPAN 35 OCCURRENCE SPAN CODE DATE CODE CODE CODE DATE CODEDATE DATE THROUGH
VALUE CODES 39 VALUE CODES VALUE CODES CODE AMOUNT CODE AMOUNT CODE AMOUNT
TOTALS
41
PRINCIPAL PROCEDURE a. OTHER PROCEDURE b. OTHER PROCEDURE NPICODE DATE CODE DATE CODE DATE
FIRST
c. d.OTHER PROCEDURE
75
e. OTHER PROCEDURE NPICODE DATE DATE
FIRST
NPI
b LAST FIRST
c NPI
d LAST FIRST
UB-04 CMS-1450
7
10 BIRTHDATE 11 SEX 16 DHR 18 19 20 21 22 23
CODE
13 HR 14 TYPE 15 SRC
FROM
25 26 2827
CODE FROM
OTHER
PRV ID
b
. INFO BEN.
29 ACDT 30
31
52 REL
THROUGH 32 34 36 37
38 40
42 REV. CD. 43 DESCRIPTION 45 SERV. DATE 46 SERV. UNITS 47 TOTAL CHARGES 48 NON-COVERED CHARGES 49
51 HEALTH PLAN ID 53 ASG.
54 PRIOR PAYMENTS 55 EST. AMOUNT DUE 56 NPI
57
58 INSURED’S NAME 59 P.REL 60 INSURED’S UNIQUE ID 61 GROUP NAME 62 INSURANCE GROUP NO.
64 DOCUMENT CONTROL NUMBER 65 EMPLOYER NAME
66 67 68
69 ADMIT 70 PATIENT 72 73
74 76 ATTENDING
80 REMARKS
OTHER PROCEDURE
a
77 OPERATING
78 OTHER
79 OTHER
81CC
PAGE OF CREATION DATE
3a PAT. CNTL #
24
b. MED. REC. #
44 HCPCS / RATE / HIPPS CODE
e
a8 PATIENT NAME
50 PAYER NAME
63 TREATMENT AUTHORIZATION CODES
6 STATEMENT COVERS PERIOD
9 PATIENT ADDRESS
17 STAT STATE
DX REASON DX 71 PPS
CODE
QUAL
LAST
LAST
OCCURRENCE
QUAL
QUAL
QUAL
CODE DATE
1
2
3
4
5
6
7
8
9
10
11
12
13
14
15
16
17
18
19
20
21
22
23
A
B
C
A
B
C
A
B
C
THE CERTIFICATIONS ON THE REVERSE APPLY TO THIS BILL AND ARE MADE A PART HEREOF.
National Uniform NUBC™
Billing Committee
LIC9213257
G12.0
SAMPLE
SPINRAZA 64406-0058-0112 mg/5 mL, 5 mL intrathecal inj
0636 SPINRAZA 64406-0058-01 J2326 1200361 Minor Surgery 96450 1
013X
Field 66: Enter the appropriate primary ICD-10-CM diagnosis code; for example:• G12.0, Infantile spinal
muscular atrophy, type I [Werdnig-Hoffmann]
Field 4: Enter the appropriate type of bill code; for example†:• 013X, Hospital outpatient• 074X, Clinic OPT• 083X, Hospital outpatient (ASC)† X represents a placeholder for the fourth digit, which indicates the sequence of this bill in this particular episode of care (eg, “1” for admit through discharge claim).
Field 46: Enter the appropriate number of units of service.
Fields 42 and 43: Enter appropriate revenue codes and corresponding description of service; for example:• 0636, Pharmacy (ie, drugs requiring detailed coding)‡
• 0361, Operating room services (ie, minor surgery)NOTE: Other revenue codes may apply; for example:• 0331, Radiology/therapeutic (ie, chemotherapy injected)• 0370, Anesthesia (ie, general classification)• 0402, Other imaging services (ie, ultrasound)• 0762, Treatment/observation room (ie, observation room)‡For Field 43, NDC reporting requirements may vary by payer.
Field 44: Enter appropriate CPT®/HCPCS codes and modifiers; for example:• J2326, Injection, nusinersen, 0.1 mg• 96450, Chemotherapy administration, into CNS
(eg, intrathecal), requiring and including spinal punctureNOTE: Other CPT® codes may apply; for example:• 62328, Spinal puncture, lumbar, diagnostic; with
fluoroscopic or CT guidance• 62272, Spinal puncture, therapeutic, for drainage of
cerebrospinal fluid (by needle or catheter)• 76942, Ultrasonic guidance for needle placement (eg,
biopsy, aspiration, injection, localization device), imaging supervision, and interpretation
• 77012, CT guidance for needle placement (eg, biopsy, aspiration, injection, localization device), radiological supervision and interpretation
• 99218, Initial observational care, per day, for the evaluation and management of a patient, which requires these 3 key components: a detailed or comprehensive history, a detailed or comprehensive examination, and medical decision-making that is straightforward or of low complexity
*Including off-campus clinic, on-campus facility, hospital-based ASC, and other outpatient outlets operated by a hospital.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
SELECTED IMPORTANT SAFETY INFORMATIONCoagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 24 of 146 SPINRAZA-treated patients (16%) with high, normal, or unknown platelet count at baseline developed a platelet level below the lower limit of normal, compared to 10 of 72 sham-controlled patients (14%). Two SPINRAZA-treated patients developed platelet counts <50,000 cells per microliter, with the lowest level of 10,000 cells per microliter recorded on study day 28.
52
Unique Billing Considerations for Professional Services OnlyCPT® CODE MODIFIER FOR THE PROFESSIONAL COMPONENT
Modifier17 Description17
26 Professional component
For procedure codes with professional and technical components, physician office practices may use the 26 modifier to bill for the professional services component of the procedure (eg, interpretation and report of a radiology service) performed in the hospital inpatient or outpatient setting.17
CODING SUMMARY FOR ELECTRONIC CLAIM SUBMISSION FOR PROFESSIONAL SERVICESThe table below provides examples of relevant codes, along with corresponding locations, for paper and electronic claims submitted by physician office practices for professional services associated with SPINRAZA administration.
Requirements and location of information will vary by payer.
Examples of Relevant Codes for SPINRAZA and Electronic Billing Locations for Professional Services28
Information Sample Code(s) or Information
CMS-1500 Location28
Electronic Loop28
Equivalent Segment28
CPT® Code(s)
964507694200635
Other CPT® codes may apply, as appropriate
Field 24D 2400 SV101
ICD-10-CM Code (primary) G12.0 Field 21A 2300 HI01-2
Place of Service Code Provider-specific* Field 24B 2300 CLM05-1
* A 2-digit place of service code documents site of care. Relevant examples for professional services include 19 (off-campus outpatient hospital), 22 (on-campus outpatient hospital), and 21 (inpatient hospital).29
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONRenal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. SPINRAZA is present in and excreted by the kidney. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 71 of 123 SPINRAZA-treated patients (58%) had elevated urine protein, compared to 22 of 65 sham-controlled patients (34%).
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
53
APPROVED OMB-0938-1197 FORM 1500 (02-12) PLEASE PRINT OR TYPE
APPROVED OMB-0938-1197 FORM 1500 (02-12) PLEASE PRINT OR TYPE
G12.0
9645022
22
22 00635
76942 26
Field 24D: Enter appropriate CPT®/HCPCS codes and modifiers; for example:• 62272, Spinal puncture, therapeutic, for drainage
of cerebrospinal fluid (by needle or catheter)• 96450, Chemotherapy administration, into
CNS (eg, intrathecal), requiring and including spinal puncture
• 76942, Ultrasonic guidance for needle placement (eg, biopsy, aspiration, injection, localization device), imaging supervision, and interpretation• 26, Professional component
• 00635, Anesthesia for procedures in lumbar region (diagnostic or therapeutic lumbar puncture)
NOTE: Other CPT® codes and modifiers may apply.
Field 24B: Enter the appropriate place of service code; for example:• 19, Off-campus outpatient hospital• 21, Inpatient hospital• 22, On-campus outpatient hospital
Field 21A: Enter the appropriate primary ICD-10-CM diagnosis code; for example:• G12.0, Infantile spinal
muscular atrophy, type I [Werdnig-Hoffmann]
SAMPLE
Sample CMS-1500 Claim Form FOR PROFESSIONAL SERVICES
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONLaboratory testing and monitoring to assess safety should be conducted. Perform a platelet count, coagulation laboratory testing, and quantitative spot urine protein testing at baseline and prior to each dose of SPINRAZA and as clinically needed.Severe hyponatremia was reported in an infant treated with SPINRAZA requiring salt supplementation for 14 months.Cases of rash were reported in patients treated with SPINRAZA. SPINRAZA may cause a reduction in growth as measured by height when administered to infants, as suggested by observations from the controlled study. It is unknown whether any effect of SPINRAZA on growth would be reversible with cessation of treatment.
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
54
Unique Billing Considerations for Physician Offices and Freestanding ASCsCODING SUMMARY FOR ELECTRONIC CLAIM SUBMISSION BY PHYSICIAN OFFICES AND FREESTANDING ASCs The table below provides examples of relevant codes, along with corresponding locations, for paper and electronic claims submitted by physician office practices or freestanding ASCs for SPINRAZA and related administration services.
Requirements and location of information will vary by payer.
Examples of Relevant Codes for SPINRAZA and Electronic Billing Locations for Physician Offices and Freestanding ASCs28
Information Sample Code(s) or Information
CMS-1500 Location28
Electronic Loop28
Equivalent Segment28
HCPCS Level II Code J2326 Field 24D 2400 SV101
HCPCS Level II Code Units 120 Field 24G 2400 SV101
Additional Product Information
SPINRAZA 64406-0058-01 12 mg/5 mL, 5 mL
intrathecal injField 19 2300 NTE
CPT® Code(s) 96450
Other CPT® codes may apply, as appropriate
Field 24D 2400 SV101
ICD-10-CM Code (primary) G12.0 Field 21A 2300 HI01-2
Place of Service Code Provider-specific* Field 24B 2300 CLM05-1
* A 2-digit place of service code documents site of care. Relevant examples for professional services include 19 (off-campus outpatient hospital), 22 (on-campus outpatient hospital), and 21 (inpatient hospital).29
For patients on Medicare being treated in an ASC, be sure to refer to Addendum AA and BB to ensure that the CPT® codes for the drug administration and for the drug itself are eligible for payment in the ASC setting.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
55
SELECTED IMPORTANT SAFETY INFORMATIONThe most common adverse reactions (≥20% of SPINRAZA-treated patients and ≥5% more frequently than in control patients) that occurred in the infantile-onset controlled study were lower respiratory infection and constipation. Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (18%) than in control patients (10%). Because patients in this controlled study were infants, adverse reactions that are verbally reported could not be assessed. The most common adverse reactions that occurred in the later-onset controlled study were pyrexia, headache, vomiting, and back pain. Post-lumbar puncture syndrome has also been observed after the administration of SPINRAZA.
Sample CMS-1500 Claim Form FOR PHYSICIAN OFFICES AND FREESTANDING ASCs
APPROVED OMB-0938-1197 FORM 1500 (02-12) PLEASE PRINT OR TYPE
SAMPLEG12.0
SPINRAZA 64406-0058-01 12 mg/5 mL, 5mL intrathecal inj
J2326
96450
120
1
24
24
Field 21A: Enter the appropriate primary ICD-10-CM diagnosis code; for example:• G12.0, Infantile spinal muscular
atrophy, type I [Werdnig-Hoffmann]
Field 24G: Enter the appropriate number of units of service.
Field 24D: Enter appropriate CPT®/HCPCS codes and modifiers; for example:• J2326, Injection, nusinersen, 0.1 mg• 96450, Chemotherapy administration, into CNS (eg, intrathecal), requiring
and including spinal punctureNOTE: Other CPT® codes and modifiers may apply; for example:• 00635, Anesthesia for procedures in lumbar region (diagnostic or therapeutic
lumbar puncture)• 76942, Ultrasonic guidance for needle placement (eg, biopsy, aspiration, injection, localization
device), imaging supervision, and interpretation
Shaded areas for fields 24A-D: NDC reporting requirements may vary by payer.
Field 24B: Enter the appropriate place of service code; for example:• 11, Office• 24, ASC
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
SELECTED IMPORTANT SAFETY INFORMATIONCoagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications.
56
Unique Billing Considerations for Inpatient Hospital FacilitiesREVENUE CODE EXAMPLES FOR INPATIENT HOSPITAL FACILITIES
Service Type18 Revenue Code18 Description18
Drug Product 0636 Pharmacy (ie, drugs requiring detailed coding)
Room and Board 0101 All-inclusive rate (ie, all-inclusive room and board)
Drug Administration, Surgical Procedure, Recovery, and Observation
0272 Medical/surgical supplies (ie, sterile supply)
0331 Radiology/therapeutic
0360 Operating room services (ie, general classification)
0369 Operating room services (ie, other operating room services)
0710 Recovery room permits identification of particular services, if necessary
Anesthesia Services 0370 Anesthesia (ie, general classification)
Imaging Services0402 Other imaging services (ie, ultrasound)
0409 Other imaging services (ie, other imaging services)
Revenue codes are required for hospital inpatient billing and will vary depending on the revenue center to which your hospital maps SPINRAZA. Typically, SPINRAZA will be reported using the revenue codes listed above.
ICD-10-PCS PROCEDURE CODE EXAMPLES
Procedure Type30 ICD-10-PCS Code30 Description30
Intrathecal Drug Administration 3E0R3GC Introduction of other therapeutic substance into spinal
canal, percutaneous approach
Imaging Procedure/ Guidance
BR13YZZ Fluoroscopy of lumbar disc(s) using other contrast
BR49ZZZ Ultrasonography of lumbar spine
Inhalation Anesthesia 3E0F7BZ Introduction of anesthetic agent into respiratory tract,
via natural or artificial opening
When SPINRAZA is administered in the inpatient setting, appropriate inpatient procedure codes will need to be reported on the claim. Typically, SPINRAZA administration procedures will be reported using the ICD-10-PCS codes listed above.
ICD-10-PCS=International Classification of Diseases, Tenth Revision, Procedure Classification System.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
SELECTED IMPORTANT SAFETY INFORMATIONIn the sham-controlled studies for patients with infantile-onset and later-onset SMA, 24 of 146 SPINRAZA-treated patients (16%) with high, normal, or unknown platelet count at baseline developed a platelet level below the lower limit of normal, compared to 10 of 72 sham-controlled patients (14%). Two SPINRAZA-treated patients developed platelet counts <50,000 cells per microliter, with the lowest level of 10,000 cells per microliter recorded on study day 28.
57
CODING SUMMARY FOR ELECTRONIC CLAIM SUBMISSION BY INPATIENT HOSPITAL FACILITIESThe table below provides examples of relevant codes, along with corresponding locations, for paper and electronic claims submitted by inpatient hospital facilities for SPINRAZA and related administration services (as part of a planned inpatient stay).
Requirements and location of information will vary by payer.
Examples of Relevant Codes for SPINRAZA and Electronic Billing Locations for Inpatient Hospital Facilities26
Information Sample Code(s) or Information
CMS-1450/ UB-04 Locator26
Electronic Loop26
Equivalent Segment26
HCPCS Level II Code J2326 Field 44 2400 SV202-2
HCPCS Level II Code Units 120 Field 46 2400 SV205
Additional Product Information
SPINRAZA 64406-0058-01 12 mg/5 mL, 5 mL
intrathecal injField 80 2300 NTE
ICD-10-PCS Code(s)
3E0R3GCBR13YZZ3E0F7DZ
Other ICD-10-PCS codes may apply, as appropriate
Fields 74-74E 2300HI01-2
through HI05-4
ICD-10-CM Code (primary) G12.0 Field 67 2300 HI01-2
Bill Type Code Provider-specific* Field 4 2300 CLM05-1
Revenue Code(s)
0101, 0272, 0360, 0370, 0409, 0636
Other revenue codes may apply, as appropriate
Field 42 2400 SV201
* A 4-digit bill type code documents facility type (second digit after the leading zero), care type (third digit), and the bill sequence for the given episode of care (fourth digit). Relevant examples for inpatient hospital facilities include 011X (hospital inpatient part A) and 014X (hospital other part B), where X represents the sequence of the bill in this particular episode of care (eg, “1” for admit through discharge claim).27
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONRenal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. SPINRAZA is present in and excreted by the kidney. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 71 of 123 SPINRAZA-treated patients (58%) had elevated urine protein, compared to 22 of 65 sham-controlled patients (34%).
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
58
Sample CMS-1450/UB-04 Claim Form FOR INPATIENT HOSPITAL FACILITIES
__ __ __
1
2
3
4
5
6
7
8
9
10
11
12
13
14
15
16
17
18
19
20
21
22
23
A
B
C
A
B
C
A
B
C
APPROVED OMB NO.
__
1 2 4 TYPE OF BILL
FROM THROUGH 5 FED. TAX NO.
a
b
c
d
DX
ECI
A B C D E F G H I J K L M N O P Q
a b c
a
b c d
ADMISSION CONDITION CODES DATE 12
OCCURRENCE OCCURRENCE33 OCCURRENCE OCCURRENCE SPAN 35 OCCURRENCE SPAN CODE DATE CODE CODE CODE DATE CODEDATE DATE THROUGH
VALUE CODES 39 VALUE CODES VALUE CODES CODE AMOUNT CODE AMOUNT CODE AMOUNT
TOTALS
41
PRINCIPAL PROCEDURE a. OTHER PROCEDURE b. OTHER PROCEDURE NPICODE DATE CODE DATE CODE DATE
FIRST
c. d.OTHER PROCEDURE
75
e. OTHER PROCEDURE NPICODE DATE DATE
FIRST
NPI
b LAST FIRST
c NPI
d LAST FIRST
UB-04 CMS-1450
7
10 BIRTHDATE 11 SEX 16 DHR 18 19 20 21 22 23
CODE
13 HR 14 TYPE 15 SRC
FROM
25 26 2827
CODE FROM
OTHER
PRV ID
b
. INFO BEN.
29 ACDT 30
31
52 REL
THROUGH 32 34 36 37
38 40
42 REV. CD. 43 DESCRIPTION 45 SERV. DATE 46 SERV. UNITS 47 TOTAL CHARGES 48 NON-COVERED CHARGES 49
51 HEALTH PLAN ID 53 ASG.
54 PRIOR PAYMENTS 55 EST. AMOUNT DUE 56 NPI
57
58 INSURED’S NAME 59 P.REL 60 INSURED’S UNIQUE ID 61 GROUP NAME 62 INSURANCE GROUP NO.
64 DOCUMENT CONTROL NUMBER 65 EMPLOYER NAME
66 67 68
69 ADMIT 70 PATIENT 72 73
74 76 ATTENDING
80 REMARKS
OTHER PROCEDURE
a
77 OPERATING
78 OTHER
79 OTHER
81CC
PAGE OF CREATION DATE
3a PAT. CNTL #
24
b. MED. REC. #
44 HCPCS / RATE / HIPPS CODE
e
a8 PATIENT NAME
50 PAYER NAME
63 TREATMENT AUTHORIZATION CODES
6 STATEMENT COVERS PERIOD
9 PATIENT ADDRESS
17 STAT STATE
DX REASON DX 71 PPS
CODE
QUAL
LAST
LAST
OCCURRENCE
QUAL
QUAL
QUAL
CODE DATE
1
2
3
4
5
6
7
8
9
10
11
12
13
14
15
16
17
18
19
20
21
22
23
A
B
C
A
B
C
A
B
C
THE CERTIFICATIONS ON THE REVERSE APPLY TO THIS BILL AND ARE MADE A PART HEREOF.
National Uniform NUBC™
Billing Committee
LIC9213257
SAMPLE
G12.0
SPINRAZA 64406-0058-0112 mg/5 mL, 5 mL intrathecal inj
0101 All-Inclusive Room and Board 0272 Sterile Supply 0360 Operating Room Services/General 0370 Anesthesia/General 0409 Other Imaging Services 0636 SPINRAZA 64406-0058-01 J2326
3E0R3GC BR13YZZ 3E0F7DZ
0111X
Field 66: Enter the appropriate primary ICD-10-CM diagnosis code; for example:• G12.0, Infantile spinal
muscular atrophy, type I [Werdnig-Hoffmann]
Fields 42 and 43: Enter appropriate revenue code and description of service; for example:• 0101, All-inclusive rate (ie, all-inclusive room and board)• 0272, Medical/surgical supplies (ie, sterile supply)• 0360, Operating room services (ie, general classification)• 0370, Anesthesia (ie, general classification)• 0409, Other imaging services (ie, other imaging services)• 0636, Pharmacy (ie, drugs requiring detailed coding)†
† For Field 43, NDC reporting requirements may vary by payer.
NOTE: Other revenue codes may apply.
Field 44: Enter the appropriate HCPCS code corresponding to the revenue code 0636 (Pharmacy: drugs requiring detailed coding) in order to provide detailed level coding; for example:• J2326, Injection, nusinersen, 0.1 mg
Fields 74-74e: Enter appropriate principal and other ICD-10-PCS procedure codes (along with corresponding dates); for example:• 3E0R3GC, Introduction of other therapeutic substance into spinal
canal, percutaneous approach • BR13YZZ, Fluoroscopy of lumbar disc(s) using other contrast • 3E0F7DZ, Introduction of inhalation anesthetic into respiratory
tract, via natural or artificial opening NOTE: Other ICD-10-PCS procedure codes may apply; for example:• BR49ZZZ, Ultrasonography of lumbar spine
Field 4: Enter the appropriate type of bill code; for example*:• 011X, Hospital inpatient• 014X, Hospital other* X represents a placeholder for the fourth digit, which indicates the
sequence of this bill in this particular episode of care (eg, “1” for admit through discharge claim).
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONLaboratory testing and monitoring to assess safety should be conducted. Perform a platelet count, coagulation laboratory testing, and quantitative spot urine protein testing at baseline and prior to each dose of SPINRAZA and as clinically needed.Severe hyponatremia was reported in an infant treated with SPINRAZA requiring salt supplementation for 14 months.Cases of rash were reported in patients treated with SPINRAZA. SPINRAZA may cause a reduction in growth as measured by height when administered to infants, as suggested by observations from the controlled study. It is unknown whether any effect of SPINRAZA on growth would be reversible with cessation of treatment.
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
59
Claim Submission and Tracking ChecklistsCompleting timely and accurate claims can help facilitate prompt payment. In order to help proactively prevent denials and underpayment, it is important to review claims before submitting them to a payer.
CLAIM SUBMISSION CONSIDERATIONS CHECKLIST
Confirm payer requirements
DURING THE BENEFITS INVESTIGATION PROCESS, CONFIRM THAT YOU HAVE IDENTIFIED THE FOLLOWING:
Coverage and any PA restrictions Coding and billing guidelines Required medical documentation
Check claim for accuracy and completeness
WHEN FILLING OUT THE CLAIM FORM, PLEASE DOUBLE-CHECK THE FOLLOWING:
Patient information (eg, patient name, insurer, subscriber name, date of birth, member ID) Provider information (eg, NPI number, name, address, place of service) Coding (eg, ICD-10, CPT®, revenue, and/or HCPCS codes along with appropriate modifiers) Billing units (consistent with the descriptors for the reported CPT® and/or HCPCS codes) Additional information required by the payer (eg, PA, tax ID and/or drug NDC number) (If clinical documentation is required) Confirm with the payer how documentation should be submitted with the initial claim submission
Confirm compliance with claim
submission rules
WHEN SUBMITTING THE CLAIM, BE MINDFUL OF THE FOLLOWING:
Required standards for electronic claims Punctuation and character limit requirements Time frame for submitting claims
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
60
SELECTED IMPORTANT SAFETY INFORMATIONThe most common adverse reactions (≥20% of SPINRAZA-treated patients and ≥5% more frequently than in control patients) that occurred in the infantile-onset controlled study were lower respiratory infection and constipation. Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (18%) than in control patients (10%). Because patients in this controlled study were infants, adverse reactions that are verbally reported could not be assessed. The most common adverse reactions that occurred in the later-onset controlled study were pyrexia, headache, vomiting, and back pain. Post-lumbar puncture syndrome has also been observed after the administration of SPINRAZA.
CLAIM TRACKING CONSIDERATIONSTracking payer reimbursement for therapies and treatments is key for facilitating appropriate payment. It is important for offices to check payers’ EOB/RA statements for accuracy in order to detect any claims processing errors or inappropriate adjustments and to monitor for denials.
THE FOLLOWING ARE CONSIDERATIONS FOR TRACKING CLAIMS:
ESTABLISH A ROUTINE PROCEDURE FOR MONITORING THE STATUS OF CLAIMS
MAINTAIN A LOG OF ALL CORRESPONDENCE WITH EACH PAYER FOR EACH CLAIM. THIS WILL ENABLE YOUR OFFICE TO MONITOR PAYER CONTRACT COMPLIANCE
MONITOR THE CLAIM FOR PAYERS WHO REQUEST ADDITIONAL INFORMATION (CLINICAL OR OTHER) AND SUBMIT PROMPTLY TO AVOID PROCESSING DELAYS
REVIEW PAYER EOB/RA AGAINST CONTRACTED FEE SCHEDULES
EVALUATE PAYER RESPONSIVENESS IN ADDRESSING REIMBURSEMENT ISSUES
ESTABLISH A PROCEDURE FOR ADDRESSING CLAIMS DENIALS AND SUBMITTING APPEALS
Consider using the Biogen Reimbursement Tracking Log, located at SPINRAZA-hcp.com, to reconcile drug and procedure claims for SPINRAZA.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
MEDICARE AND SMA
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
6161
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
SELECTED IMPORTANT SAFETY INFORMATIONCoagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications.
62
Medicare Eligibility for Patients With SMA*†
WHO IS ELIGIBLE?
// Children who were receiving benefits as a minor child on a parent’s Social Security record (via SSI) may be eligible to continue receiving benefits on that parent’s record upon reaching age 18 if they are disabled31
– Marriage of the disabled “adult child” may affect eligibility for this benefit// Medicare covers about 1 in 4 adults with SMA (aged 18 years and older)
HOW PATIENTS APPLY FOR MEDICARE // Most people with SMA who are receiving SSDI benefits are automatically enrolled in Original
Medicare (Parts A and B)32
– Patients who receive SSDI get Part A at no cost, but may have to pay a premium for Parts B and D. If patients do not want the Part B premium automatically deducted from their SSDI, they can call Medicare to opt out of Part B. However, please note that in certain situations, opting out of Medicare Part B may affect Medigap coverage and cause detrimental financial consequences for the patient. Be sure to discuss with your Medicare contact prior to opting out of Medicare Part B.
// Patients will be enrolled by the 25th month of receiving SSDI. They will get their Medicare card in the mail32
// Patients also get a notice in the mail about their Part D drug plan. It will tell them how to review or change it
If patients are not disabled, they will need to enroll in Medicare. They can sign up in 2 ways32:
// Call the Social Security office at 1-800-772-1213 // Sign up online at https://www.ssa.gov/benefits/medicare/
* Please note that Maryland follows the terms of the Maryland All-Payer Model and some of the above information may not apply.† Medicare providers and suppliers are not permitted to bill people enrolled in the Qualified Medicare Beneficiary program for items such as Medicare copays, deductibles, or coinsurance.32
A PERSON CAN QUALIFY FOR SSDI BENEFITS IF
// SMA Types 0 and 1 qualify as compassionate allowances for Social Security disability (1 of more than 200 conditions); it is automatic in many states34
// State-based qualification parameters also apply// To learn about disability benefits through Social Security, call 1-800-772-1213 or
visit https://www.ssa.gov/benefits/disability/
He or she has a past work history33
He or she can no longer work due to his or her disability33
People younger than 65 years who have received Social Security Disability Insurance (SSDI) benefits for at least 2 years (24 months)31
// For these patients, enrollment in Medicare is automatic32
People aged 65 years or older31
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
SELECTED IMPORTANT SAFETY INFORMATIONIn the sham-controlled studies for patients with infantile-onset and later-onset SMA, 24 of 146 SPINRAZA-treated patients (16%) with high, normal, or unknown platelet count at baseline developed a platelet level below the lower limit of normal, compared to 10 of 72 sham-controlled patients (14%). Two SPINRAZA-treated patients developed platelet counts <50,000 cells per microliter, with the lowest level of 10,000 cells per microliter recorded on study day 28.
63
Part A is hospital insurance. It covers inpatient care in a hospital, nursing home care, hospice care, and home healthcare. Part A covers SPINRAZA when patients receive it as an inpatient during a hospital stay.
Part B is medical insurance. Part B covers outpatient care (doctor’s office visits and visits to treatment centers for injections, some home healthcare services, medical equipment, wellness services, lab tests, and select preventive screenings). Part B covers SPINRAZA when patients receive it as an outpatient, for instance, at an SMA hospital outpatient treatment center.
Parts A and B are known as “Original Medicare.” Patients will have Original Medicare unless they choose a Medicare Advantage Plan or other type of Medicare health plan.
Part C is also called Medicare Advantage. Part C plans are sold by private insurance companies approved by Medicare. These plans include all benefits covered under Part A and Part B. Part C may offer additional benefits, as well. Medicare Advantage plans will often include Part D (prescription drug coverage). See the following page for more detailed information about Medicare Advantage plans.
Part D is prescription drug coverage. Part D covers drug costs (pills, self-administered injections, and inhaled treatments). Part D plans are sold by private insurance companies approved by Medicare. These plans do not cover SPINRAZA, but they may cover other drugs patients may need.
A
B
C
D
Medicare BasicsHOW DOES MEDICARE WORK?Medicare has 4 parts to help cover services32:
SMA360°* can help patients navigate Medicare options. Patients can call 1-844-4SPINRAZA (1-844-477-4672), Monday through Friday, from 8:30 am to 8:00 pm ET.
* SMA360° services from Biogen are available only to those who have been prescribed SPINRAZA. SMA360° is intended for US residents only.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONRenal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. SPINRAZA is present in and excreted by the kidney. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 71 of 123 SPINRAZA-treated patients (58%) had elevated urine protein, compared to 22 of 65 sham-controlled patients (34%).
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
64
UNDERSTANDING THE DIFFERENT TYPES OF MEDICARE ADVANTAGE PLANS (PART C)There are many types of Medicare Advantage plans. Each type of plan may keep certain copayments low (ie, office visits), provide varying levels of coverage, and operate in a unique way. Medicare Advantage plans offer the same benefits patients get under original Medicare (Part A and Part B), except hospice care. Medicare Advantage plans may also include other benefits such as prescription drug coverage, hearing, dental, and vision.32
Medicare Advantage plans may help keep copayments low. Each plan comes with a maximum OOP limit on how much patients will spend on health costs each year. Once patients reach the limit, they will pay nothing for the services covered. Each plan has different limits, and the OOP amount can change each year.32
The OOP maximum for Medicare Part C varies by plan. Patients paying higher monthly premiums may have lower maximum yearly OOP costs.35
The different types of Medicare Advantage plans include those outlined in the table below.
HOW EACH MEDICARE ADVANTAGE PLAN WORKS
Types of commercial health plans
Has a network of providers?
Need a referral to see a specialist?
What happens if a patient needs out-of-network care?
HMO36 Yes Yes
HMOs may cover out-of-network care if// The HMO’s network of HCPs does not have the
experience to treat a certain health problem// The patient has an emergency
PPO37 Yes No
PPOs provide out-of-network care, but may not pay for the full cost of treatment. If the patient chooses to see an out-of-network HCP, he or she may have to pay for some of the treatment, even if it is an emergency
Private Fee-for-Service (PFFS)38
Depends on plan No
These plans are also offered by private insurance companies. PFFS plans aren’t the same as Original Medicare or Medigap. PFFS plans determine how much they will pay HCPs, other healthcare providers, and hospitals, and how much the patient will pay when receiving care
Special Needs Plan (SNP)39 Yes Yes
These plans limit membership to people with certain characteristics or specific diseases such as SMA. SNPs tailor their benefits, provider choices, and drug formularies to best meet the specific needs of the groups they serve
HMO Point of Service (HMOPOS)40
Yes NoHMOPOS plans provide out-of-network care, but patients may have higher costs for out-of-network providers
Medical Savings Account (MSA)41
No No
These plans combine a high-deductible health plan with a bank account. Medicare deposits money into the account (usually less than the deductible). Patients can use the money to pay for healthcare services during the year, but only Medicare covered expenses count toward the deductible
Less
Com
mon
Mor
e C
omm
on
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONLaboratory testing and monitoring to assess safety should be conducted. Perform a platelet count, coagulation laboratory testing, and quantitative spot urine protein testing at baseline and prior to each dose of SPINRAZA and as clinically needed.Severe hyponatremia was reported in an infant treated with SPINRAZA requiring salt supplementation for 14 months.Cases of rash were reported in patients treated with SPINRAZA. SPINRAZA may cause a reduction in growth as measured by height when administered to infants, as suggested by observations from the controlled study. It is unknown whether any effect of SPINRAZA on growth would be reversible with cessation of treatment.
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
65
A MEDIGAP POLICY MAY HELP PATIENTS PAY FOR TREATMENT COSTS NOT COVERED BY ORIGINAL MEDICAREMedicare Supplement Insurance policies, also known as Medigap policies, can help pay some of the costs Original Medicare does not. This includes copayments, coinsurance, and deductibles. Medigap policies are sold by private insurance companies. They must follow federal and state laws.32
Medigap policies are not available to people covered
by a Medicare Advantage Plan (Part C) or to patients with
traditional Part D plans32
Patients must have Medicare Part A and Part B to have a Medigap Policy32
Patients cannot purchase a Medigap policy along with
a Medicare MSA42
CONTINUING SPINRAZA TREATMENT WHEN INSURANCE CHANGESIt is important to track and understand changes in health insurance for your patients with SMA, including primary and secondary insurance plans.// For example, patients with SMA may transition to Medicare from Medicaid; Medicare becomes the
primary insurer and Medicaid is the secondary insurer// Patients with SMA may require authorization for treatment due to the change in health insurance// Insurance claims submitted to the wrong primary insurer will likely be rejected and will need to be
resubmitted to the correct insurer, causing a significant delay in reimbursement
FACTS ABOUT MEDIGAP POLICIES
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
66
SELECTED IMPORTANT SAFETY INFORMATIONThe most common adverse reactions (≥20% of SPINRAZA-treated patients and ≥5% more frequently than in control patients) that occurred in the infantile-onset controlled study were lower respiratory infection and constipation. Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (18%) than in control patients (10%). Because patients in this controlled study were infants, adverse reactions that are verbally reported could not be assessed. The most common adverse reactions that occurred in the later-onset controlled study were pyrexia, headache, vomiting, and back pain. Post-lumbar puncture syndrome has also been observed after the administration of SPINRAZA.
THE EXTENT OF WHAT MEDICARE COVERS DEPENDS ON WHETHER IT IS PAYING AS A PRIMARY OR SECONDARY PAYER // Medicare as the primary payer pays up to the limits of its coverage32
– Hospital coverage (Part A) – Physician visits, outpatient services, and physician-administered drugs like SPINRAZA (Part B) – Self-administered prescription drugs, which do not include SPINRAZA (Part D)
// Coverage gaps may still exist with Medicare as the primary payer32,35
– Patients can cover gaps with secondary payer/supplemental insurance (eg, employer-sponsored health plan, Medigap policy)
// Medicare as the secondary payer – Main role is to close the gap in OOP expenses32
– Pays only if there are costs not covered by the primary insurer32
– Medicare coinsurance will still apply32
– Sometimes covers claims when the primary payment is delayed or in dispute43
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
SELECTED IMPORTANT SAFETY INFORMATIONCoagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 24 of 146 SPINRAZA-treated patients (16%) with high, normal, or unknown platelet count at baseline developed a platelet level below the lower limit of normal, compared to 10 of 72 sham-controlled patients (14%). Two SPINRAZA-treated patients developed platelet counts <50,000 cells per microliter, with the lowest level of 10,000 cells per microliter recorded on study day 28.
67
Hospital Reimbursement for SPINRAZA SPINRAZA 340B REIMBURSEMENT UPDATE As of January 1, 2018, Medicare pays an adjusted amount of the average sales price (ASP) minus 22.5% for certain separately payable drugs or biologics acquired through the 340B program. Furthermore, these products are furnished to a Medicare beneficiary by a hospital paid under the Outpatient Prospective Payment System (OPPS) that is not exempt from the payment adjustment policy.44
// As of July 1, 2020, SPINRAZA is reimbursed at ASP minus 22.5% (instead of ASP+6%) for 340B-purchased drugs only for Medicare patients treated at affected sites of care44,45
// For more information about billing 340B-acquired drugs (and use of modifiers), visit https://www.cms.gov/medicare/medicare-fee-for-service-payment/hospitaloutpatientpps/downloads/billing-340b-modifiers-under-hospital-opps.pdf
UNDERSTANDING APCs VS DRGs (BUNDLED PAYMENTS)// Ambulatory Payment Classifications (APCs) are the government’s way to pay facilities for outpatient
services under the Medicare program46
– Hospital-only, OPPS – SPINRAZA is allowed separate payment in the hospital outpatient department setting – APC payments are made to the hospital when a Medicare outpatient is discharged or is transferred
to another hospital or facility not affiliated with the initial hospital where the patient received outpatient services
// Inpatient stays are paid under DRG methodology rather than APC.46 DRG payments do not allow separate payment for drugs administered during an inpatient stay47
– Medicare inpatient stays are subject to the 3-day rule. All outpatient services during the 3 days prior to an inpatient stay need to be incorporated into the inpatient DRG stay47
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONRenal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. SPINRAZA is present in and excreted by the kidney. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 71 of 123 SPINRAZA-treated patients (58%) had elevated urine protein, compared to 22 of 65 sham-controlled patients (34%).
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
68
Did you know
ORIGINAL MEDICARE PATIENTS’ OOP COSTS ARE CAPPED IN THE HOSPITAL OUTPATIENT SETTING// OOP costs for patients receiving SPINRAZA in this treatment setting may
be limited to the Medicare Part A deductible – Typically, outpatient facility claims process under Part B benefits, so
beneficiaries who do not have supplemental insurance (eg, Medigap) are responsible for a 20% coinsurance (Medicare Part B covers 80%)35
– However, in the outpatient facility setting, if the patient’s coinsurance under Medicare Part B exceeds the Medicare Part A deductible, then the patient pays the Part A deductible and Medicare pays the difference.35,48 This may significantly reduce OOP costs
// Although hospital outpatient department claims for SPINRAZA are still covered under Medicare Part B, the claims are submitted to the Part A Medicare Administrative Contractor (MAC)
?
If your patient has Original Medicare and is being treated in the hospital outpatient setting, you can contact Medicare to find out the current Part A deductible amount he or she would pay. Call 1-800-MEDICARE or 1-800-633-4227.
Resources to determine the actual cost savings for patients treated in the outpatient setting are available from your Biogen representative.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONLaboratory testing and monitoring to assess safety should be conducted. Perform a platelet count, coagulation laboratory testing, and quantitative spot urine protein testing at baseline and prior to each dose of SPINRAZA and as clinically needed.Severe hyponatremia was reported in an infant treated with SPINRAZA requiring salt supplementation for 14 months.Cases of rash were reported in patients treated with SPINRAZA. SPINRAZA may cause a reduction in growth as measured by height when administered to infants, as suggested by observations from the controlled study. It is unknown whether any effect of SPINRAZA on growth would be reversible with cessation of treatment.
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
69
Medicare Considerations Nationally and Locally
CMS-ISSUED NCDs APPLY TO ALL MACs NATIONWIDE49
// If an NCD does not exist, MACs can issue a Local Coverage Determination (LCD)
// An LCD is a coverage policy detailing the MAC’s coverage criteria for items of services within their jurisdiction(s). MACs may choose to cover items and services without developing an LCD
MACs CAN IMPACT COVERAGE POLICY50
// Establish LCDs
// Handle first-stage appeals process
// Review medical records for selected claims
NATIONAL CONSIDERATIONS// The Centers for Medicare & Medicaid Services (CMS) contracts with private companies known as MACs
to process and pay claims49
// CMS issues National Coverage Determinations (NCDs)49
// An NCD describes the circumstances under which a particular item or service (eg, a drug) is covered nationally under Medicare49
// NCDs apply to all MACs nationwide49
// CMS has not issued an NCD for SPINRAZA
LOCAL MEDICARE CONSIDERATIONSMACs process Medicare Part A and Part B (A/B) claims for a defined geographic area or “jurisdiction.”50
// A/B MACs process Part A and Part B claims for a defined geographic area servicing institutional providers, physicians, practitioners, and suppliers50
// Durable Medical Equipment MACs process Medicare Durable Medical Equipment, Orthotics, and Prosthetics (DMEPOS) claims for a defined geographic area servicing suppliers of DMEPOS50
There are 7 A/B MACs covering a total of 12 jurisdictions51
// Claims for SPINRAZA are processed by A/B MACs. Each MAC may have its own rules for coverage, billing/coding, etc49
// Healthcare insurers can be awarded more than 1 jurisdiction to process claim51
MACs AND MEDICAL POLICY
// Regional MACs may review SPINRAZA claims on a case-by-case basis until they issue an LCD or until CMS issues an NCD with conditions for coverage
7070
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
7070
APPENDIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
71
SELECTED IMPORTANT SAFETY INFORMATIONThe most common adverse reactions (≥20% of SPINRAZA-treated patients and ≥5% more frequently than in control patients) that occurred in the infantile-onset controlled study were lower respiratory infection and constipation. Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (18%) than in control patients (10%). Because patients in this controlled study were infants, adverse reactions that are verbally reported could not be assessed. The most common adverse reactions that occurred in the later-onset controlled study were pyrexia, headache, vomiting, and back pain. Post-lumbar puncture syndrome has also been observed after the administration of SPINRAZA.
Sample SPINRAZA Start Form
The SPINRAZA Start Form must always be accompanied by the consent information. Please contact your Biogen representative for a copy of this document or download it from SPINRAZA-hcp.com.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
SELECTED IMPORTANT SAFETY INFORMATIONCoagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 24 of 146 SPINRAZA-treated patients (16%) with high, normal, or unknown platelet count at baseline developed a platelet level below the lower limit of normal, compared to 10 of 72 sham-controlled patients (14%). Two SPINRAZA-treated patients developed platelet counts <50,000 cells per microliter, with the lowest level of 10,000 cells per microliter recorded on study day 28.
72
Sample SPINRAZA Copay Reimbursement FormPlease see your Biogen representative for a copy of the SPINRAZA Copay Reimbursement Form.
MED
ICARE
AN
D SM
ACO
DIN
G A
ND
CLA
IMS
ORD
ERING
SPIN
RAZA
FINA
NCIA
L A
SSISTAN
CEBEN
EFITS IN
VESTIGATIO
NSITE-O
F-CARE
CON
SIDERATIO
NS
REIMBU
RSEMEN
T PRO
CESS OVERVIEW
APPEN
DIX
SELECTED IMPORTANT SAFETY INFORMATIONRenal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. SPINRAZA is present in and excreted by the kidney. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 71 of 123 SPINRAZA-treated patients (58%) had elevated urine protein, compared to 22 of 65 sham-controlled patients (34%).
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
73
Letter of Medical Necessity/Appeal TemplateThis Letter of Medical Necessity/Appeal Template is intended to be used as a guide to help you structure your letter. Remember to include the relevant health plan information and patient information (eg, policy number and/or claim number).
Remember to tailor each section of the Letter of Medical Necessity/Appeal template as appropriate to align with the letter’s purpose and the patient about whom you are writing.
REFERENCES1. SPINRAZA [Prescribing Information]. Cambridge, MA: Biogen. 2. Rapaport C. An Introduction to Health Insurance: What Should a Consumer Know? https://www.fas.org/sgp/
crs/misc/R44014.pdf. Published April 30, 2015. Accessed August 24, 2020. 3. Glossary of health coverage and medical terms. Centers for Medicare & Medicaid Services website.
https://www.cms.gov/CCIIO/Resources/Files/Downloads/uniform-glossary-final.pdf. Accessed August 24, 2020. 4. The Lewin Group, Inc. Medicaid Non-Emergency Out-of-Network Payment Study. http://www.lewin.com/
content/dam/Lewin/Resources/Site_Sections/Publications/OutofNetworkStudyReport.pdf. Published July 13, 2009. Accessed August 24, 2020.
5. Biogen, Data on file. SMA payer and channel strategic issues. Published January 28, 2016. 6. Little T. Referral, predetermination, authorization, precertification: what’s the difference? MGMA website.
https://www.mgma.com/resources/operations-management/guide-to-closing-a-private-equity-transaction-pre. Published July 16, 2019. Accessed August 24, 2020.
7. O’Hagen JM, Glanzman AM, McDermott MP, et al. An expanded version of the Hammersmith Functional Motor Scale for SMA II and III patients. Neuromuscul Disord. 2007;17(9-10):693-697.
8. Glanzman AM, Mazzone E, Main M, et al. The Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND): test development and reliability. Neuromuscul Disord. 2010;20(3):155-161.
9. Haataja L, Mercuri E, Regev R, et al. Optimality score for the neurologic examination of the infant at 12 and 18 months of age. J Pediatr. 1999;135(2 pt 1):153-161.
10. Biogen, Data on file. Nusinersen brand book. Volume 1. 11. WHO Multicentre Growth Reference Study Group. WHO Motor Development Study: windows of
achievement for six gross motor development milestones. Acta Paediatr Suppl. 2006;450:86-95. 12. Mazzone ES, Mayhew A, Montes J, et al. Revised Upper Limb Module for spinal muscular atrophy:
development of a new module. Muscle Nerve. 2017;55(6):869-874. 13. SO-SMART readies for clinical trials workshop. National Institute of Neurological Disorders and Stroke
website. https://www.ninds.nih.gov/News-Events/Workshop-Conference-Proceedings/SO-SMART-Readies-Clinical-Trials-Workshop. Published June 11, 2014. Accessed August 24, 2020.
14. Dunaway Young, S, Montes J, Kramer SS, et al. Six-minute walk test is reliable and valid in spinal muscular atrophy. Muscle Nerve. 2016;54(5):836-842.
15. Appealing a health plan decision: external review. Healthcare.gov website. https://www.healthcare.gov/appeal-insurance-company-decision/external-review/. Accessed August 24, 2020.
16. Patient Advocate Foundation. A Patient’s Guide to Navigating the Insurance Appeals Process. https://www.patientadvocate.org/wp-content/uploads/Navigating-the-insurance-appeals-guide-pages.pdf. Accessed August 24, 2020.
17. American Medical Association. CPT® 2020 Professional Edition. Chicago, IL: American Medical Association; 2019. 18. Centers for Medicare & Medicaid Services. CMS Manual System: Uniform Billing (UB-04) Implementation.
1767. https://www.cms.gov/Regulations-and-Guidance/Guidance/Transmittals/Downloads/R1767CP.pdf. Published July 10, 2009. Accessed August 24, 2020.
19. Ambulatory surgical center. HMSA Provider Resource Center website. https://hmsa.com/portal/provider/zav_pel.fh.AMB.500.htm. Accessed August 24, 2020.
20. Centers for Medicare & Medicaid Services. Important guidance on the new CMS-1500 and UB-04 forms. MLN Matters. https://www.cms.gov/Outreach-and-Education/Medicare-Learning-Network-MLN/MLNMattersArticles/downloads/SE0729.pdf. Accessed August 24, 2020.
21. ICD-10-CM 2020 ICD-10-CM. Centers for Medicare & Medicaid Services website. https://www.cms.gov/Medicare/Coding/ICD10/2020-ICD-10-CM. Accessed August 24, 2020.
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
74
REFERENCES (cont’d)22. Centers for Medicare & Medicaid Services (CMS) Healthcare Common Procedure Coding System (HCPCS)
application summaries for drugs, biologicals and radiopharmaceuticals. https://www.cms.gov/Medicare/Coding/MedHCPCSGenInfo/Downloads/2017-05-17-HCPCS-Application-Summary.pdf. Published May 17, 2017. Accessed August 24, 2020.
23. Academy of Managed Care Pharmacy. AMCP Guide to Pharmaceutical Payment Methods, 2013 Update.Version 3.0. https://www.amcp.org/sites/default/files/2019-03/Full-Pharmaceutical-Guide-%283.0%29.pdf. Accessed August 24, 2020.
24. Butler K, Davis G, Loftis J, Spector N. A “how to” for reporting NDC in health care claims [presentation]. https://docplayer.net/30789407-A-how-to-for-reporting-ndc-in-health-care-claims.html. Published May 1, 2013. Accessed August 24, 2020.
25. National Drug Code requirements. UnitedHealthcare website. https://www.uhcprovider.com/content/provider/en/viewer.html?file=https%3A%2F%2Fwww.uhcprovider.com%2Fcontent%2Fdam%2Fprovider%2Fdocs%2Fpublic%2Fclaims%2FNDC-Requirement-FAQ.pdf. Accessed August 24, 2020.
26. ASC 837I version 5010A2 Institutional Health Care Claim to the CMS-1450 Claim Form Crosswalk. Palmetto GBA website. https://www.palmettogba.com/Palmetto/Providers.Nsf/files/EDI_837I_v5010A2_crosswalk.pdf/$File/EDI_837I_v5010A2_crosswalk.pdf. Accessed August 25, 2020.
27. Centers for Medicare & Medicaid Services. CMS Manual System: Pub 100-04 Medicare claims processing. Transmittal 1775. https://www.cms.gov/Regulations-and-Guidance/Guidance/Transmittals/Downloads/R1775CP.pdf. Published July 24, 2009. Accessed August 25,2020.
28. ASC 837 v5010 to CMS-1500 Crosswalk. Palmetto GBA website. https://www.palmettogba.com/Palmetto/Providers.Nsf/files/CMS1500_837v5010_Crosswalk.pdf/$File/CMS1500_837v5010_Crosswalk.pdf. Accessed August 25, 2020.
29. Place of service code set. Centers for Medicare & Medicaid Services website. https://www.cms.gov/Medicare/Coding/place-of-service-codes/Place_of_Service_Code_Set.html. Updated October 2019. Accessed August 25, 2020.
30. 2020 ICD-10-PCS codes. ICD10Data.com website. http://www.icd10data.com/ICD10PCS/Codes. Accessed August 25, 2020.
31. Social Security Administration. Benefits for Children With Disabilities. https://www.ssa.gov/pubs/EN-05-10026.pdf. Accessed August 25, 2020.
32. Centers for Medicare & Medicaid Services. Medicare & You – 2020. https://www.medicare.gov/sites/default/files/2020-03/10050-Medicare-and-You_0.pdf. Accessed August 25, 2020.
33. How you qualify. Social Security Administration website. https://www.ssa.gov/planners/disability/qualify.html. Accessed August 25, 2020.
34. Compassionate allowances conditions. Social Security Administration website. https://www.ssa.gov/compassionateallowances/conditions.htm. Accessed August 25, 2020.
35. Medicare costs at a glance. Medicare.gov website. https://www.medicare.gov/your-medicare-costs/medicare-costs-at-a-glance. Accessed August 25, 2020.
36. The ins and outs of seeking out-of-network care. Patient Advocate Foundation website. https://www.patientadvocate.org/wp-content/uploads/Migraine-Out-of-Network-Care.pdf. Accessed August 25, 2020.
37. Preferred provider organization (PPO). Medicare.gov website. https://www.medicare.gov/sign-up-change-plans/types-of-medicare-health-plans/preferred-provider-organization-ppo. Accessed August 25, 2020.
38. Private fee-for-service (PFFS) plans. Medicare.gov website. https://www.medicare.gov/sign-up-change-plans/types-of-medicare-health-plans/private-fee-for-service-pffs-plans. Accessed August 25, 2020.
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
75
Please see additional Important Safety Information on page 4 and accompanying full Prescribing Information.
REFERENCES (cont’d)39. Special needs plans (SNPs). Medicare.gov website. https://www.medicare.gov/sign-up-change-plans/types-
of-medicare-health-plans/special-needs-plans-snp. Accessed August 25, 2020. 40. What’s the difference between Medicare HMO-POS and PPO plans? Priority Health website.
https://www.priorityhealth.com/medicare/medicare-explained/extra-credit/difference-between-hmo-pos-ppo#. Accessed August 25, 2020.
41. Medicare Medical Savings Account (MSA) Plans. Medicare.gov website. https://www.medicare.gov/sign-up-change-plans/types-of-medicare-health-plans/medicare-medical-savings-account-msa-plans. Accessed September 15, 2020.
42. How Medicare MSA plans work with other coverage. Medicare.gov website. https://www.medicare.gov/sign-up-change-plans/types-of-medicare-health-plans/how-medicare-msa-plans-work-with-other-coverage. Accessed August 25, 2020.
43. Congressional Research Service. Medicare Secondary Payer: Coordination of Benefits. Report No. RL33587. https://www.everycrsreport.com/files/20140508_RL33587_a4115c77876940c4301dbae3244c3100d0d926ff.pdf. Published May 8, 2014. Accessed August 25, 2020.
44. Centers for Medicare & Medicaid Services. Medicare-FFS Program Billing 340B Modifiers Under the Hospital Outpatient Prospective Payment System (OPPS). Frequently Asked Questions. https://www.cms.gov/Medicare/Medicare-Fee-for-Service-Payment/Hospital OutpatientPPS/Downloads/Billing-340B-Modifiers-under-Hospital-OPPS.pdf. Published April 2, 2018. Accessed August 26, 2020.
45. Centers for Medicare & Medicaid Services. Medicare program: hospital outpatient prospective payment and ambulatory surgical center payment systems and quality reporting programs; organ procurement organization reporting and communication; transplant outcome measures and documentation requirements; electronic health record (EHR) Incentive programs; payment to nonexcepted off-campus provider-based department of a hospital; hospital value-based purchasing (VBP) program; establishment of payment rates Under the Medicare physician fee schedule for nonexcepted items and services furnished by an off-campus provider-based department of a hospital. Final rule with comment period and interim final rule with comment period. Fed Regist. 2016;81(219):79562-79892.
46. APC (ambulatory payment classification) FAQ. American College of Emergency Physicians website. https://www.acep.org/administration/reimbursement/reimbursement-faqs/apc-ambulatory-payment-classifications-faq/. Accessed April 4, 2019.
47. Centers for Medicare & Medicaid Services. Acute Care Hospital Inpatient Prospective Payment System. https://www.cms.gov/Outreach-and-Education/Medicare-Learning-Network-MLN/MLNProducts/Downloads/AcutePaymtSysfctsht.pdf. Published March 2020. Accessed August 26, 2020.
48. Centers for Medicare & Medicaid Services. Chapter 4 – Part B hospital (including inpatient hospital Part B and OPPS). In: Medicare Claims Processing Manual. https://www.cms.gov/Regulations-and-Guidance/Guidance/Manuals/Downloads/clm104c04.pdf. Revised June 19, 2020. Accessed August 26, 2020.
49. Department of Health and Human Services. MACs Continue to Use Different Methods to Determine Drug Coverage. OEI-03-13-00450. https://oig.hhs.gov/oei/reports/oei-03-13-00450.pdf. Published August 2016. Accessed August 26, 2020.
50. What is a MAC. Centers for Medicare & Medicaid Services website. https://www.cms.gov/medicare/medicare-contracting/medicare-administrative-contractors/what-is-a-mac.html. Accessed August 26, 2020.
51. A/B MAC jurisdictions as of June 2019. Centers for Medicare & Medicaid Services website. https://www.cms.gov/Medicare/Medicare-Contracting/Medicare-Administrative-Contractors/Downloads/AB-Jurisdiction-Map-Jun-2019.pdf. Accessed August 26, 2020.
76
© 2020 Biogen. All rights reserved. 10/20 SPZ-US-0134 V10
77
Reference ID: 4625921
HIGHLIGHTS OF PRESCRIBING INFORMATION
These highlights do not include all the information needed to use
SPINRAZA® safely and effectively. See full prescribing information for
SPINRAZA.
SPINRAZA (nusinersen) injection, for intrathecal use
Initial U.S. Approval: 2016
INDICATIONS AND USAGE
SPINRAZA is a survival motor neuron-2 (SMN2)-directed antisense
oligonucleotide indicated for the treatment of spinal muscular atrophy (SMA)
in pediatric and adult patients (1)
DOSAGE AND ADMINISTRATI ON
SPINRAZA is administered intrathecally (2.1)
Dosing Information (2.1)
• The recommended dosage is 12 mg (5 mL) per administration
• Initiate SPINRAZA treatment with 4 loading doses: the first three
loading doses should be administered at 14-day intervals; the 4th loading
dose should be administered 30 days after the 3rd dose. A maintenance
dose should be administered once every 4 months thereafter.
Important Preparation and Administration Instructions (2.2)
• Allow to warm to room temperature prior to administration
• Administer within 4 hours of removal from vial
• Prior to administration, remove 5 mL of cerebrospinal fluid
• Administer as intrathecal bolus injection over 1 to 3 minutes
Laboratory Testing and Monitoring to Assess Safety (2.3)
• At baseline and prior to each dose, obtain a platelet count, coagulation
laboratory testing, and quantitative spot urine protein testing
DOSAGE FORMS AND STRENGTHS
Injection: 12 mg/5 mL (2.4 mg/mL) in a single-dose vial (3)
CONTRAINDICATIONS
None.
WARNINGS AND PRECAUTIONS
• Thrombocytopenia and Coagulation Abnormalities: Increased risk for
bleeding complications; testing required at baseline and before each dose
and as clinically needed (5.1, 2.3)
• Renal Toxicity: Quantitative spot urine protein testing required at
baseline and prior to each dose (5.2, 2.3)
ADVERSE REACTIONS
The most common adverse reactions that occurred in at least 20% of
SPINRAZA-treated patients and occurred at least 5% more frequently than in
control patients were:
• lower respiratory infection and constipation in patients with
infantile-onset SMA (6.1)
• pyrexia, headache, vomiting, and back pain in patients with later-
onset SMA (6.1)
To report SUSPECTED ADVERSE REACTIONS, contact Biogen at 1-
844-477-4672 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
USE IN SPECIFIC POPULATIONS
Pregnancy: Based on animal data, may cause fetal harm (8.1)
See 17 for PATIENT COUNSELING INFORMATION.
Revised: 06/2020
FULL PRESCRIBING INFORMATION: CONTENTS*
1 INDICATIONS AND USAGE
2 DOSAGE AND ADMINISTRATION
2.1 Dosing Information
2.2 Important Administration Instructions
2.3 Laboratory Testing and Monitoring to Assess Safety
3 DOSAGE FORMS AND STRENGTHS
4 CONTRAINDICATIONS
5 WARNINGS AND PRECAUTIONS
5.1 Thrombocytopenia and Coagulation Abnormalities
5.2 Renal Toxicity
6 ADVERSE REACTIONS
6.1 Clinical Trials Experience
6.2 Immunogenicity
6.3 Postmarketing Experience
8 USE IN SPECIFIC POPULATIONS
8.1 Pregnancy
8.2 Lactation
8.4 Pediatric Use
8.5 Geriatric Use
11 DESCRIPTION
12 CLINICAL PHARMACOLOGY
12.1 Mechanism of Action
12.2 Pharmacodynamics
12.3 Pharmacokinetics
13 NONCLINICAL TOXICOLOGY
13.1 Carcinogenesis, Mutagenesis, Impairment of Fertility
14 CLINICAL STUDIES
14.1 Infantile-Onset SMA
14.2 Later-Onset SMA
14.3 Presymptomatic SMA
16 HOW SUPPLIED/STORAGE AND HANDLING
16.1 How Supplied
16.2 Storage and Handling
17 PATIENT COUNSELING INFORMATION
*Sections or subsections omitted from the full prescribing information are not
listed.
1
Reference ID: 4625921
FULL PRESCRIBING INFORMATION
1 INDICATIONS AND USAGE
SPINRAZA is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and
adult patients.
2 DOSAGE AND ADMINISTRATION
2.1 Dosing Information
SPINRAZA is administered intrathecally by, or under the direction of, healthcare professionals
experienced in performing lumbar punctures.
Recommended Dosage
The recommended dosage is 12 mg (5 mL) per administration.
Initiate SPINRAZA treatment with 4 loading doses. The first three loading doses should be
administered at 14-day intervals. The 4th loading dose should be administered 30 days after the
3rd dose. A maintenance dose should be administered once every 4 months thereafter.
Missed Dose
If a loading dose is delayed or missed, administer SPINRAZA as soon as possible, with at least
14-days between doses and continue dosing as prescribed. If a maintenance dose is delayed or
missed, administer SPINRAZA as soon as possible and continue dosing every 4 months.
2.2 Important Preparation and Administration Instructions
SPINRAZA is for intrathecal use only.
Prepare and use SPINRAZA according to the following steps using aseptic technique. Each vial
is intended for single dose only.
Preparation
• Store SPINRAZA in the carton in a refrigerator until time of use.
• Allow the SPINRAZA vial to warm to room temperature (25o C/77o F) prior to
administration. Do not use external heat sources.
• Inspect the SPINRAZA vial for particulate matter and discoloration prior to
administration. Do not administer SPINRAZA if visible particulates are observed or if
the liquid in the vial is discolored. The use of external filters is not required.
• Withdraw 12 mg (5 mL) of SPINRAZA from the single-dose vial into a syringe and
discard unused contents of the vial.
• Administer SPINRAZA within 4 hours of removal from vial.
Administration
• Consider sedation as indicated by the clinical condition of the patient.
2
Reference ID: 4625921
• Consider ultrasound or other imaging techniques to guide intrathecal administration of
SPINRAZA, particularly in younger patients.
• Prior to administration, remove 5 mL of cerebrospinal fluid.
• Administer SPINRAZA as an intrathecal bolus injection over 1 to 3 minutes using a
spinal anesthesia needle [see Dosage and Administration (2.1)]. Do not administer
SPINRAZA in areas of the skin where there are signs of infection or inflammation [see
Adverse Reactions (6.3)].
2.3 Laboratory Testing and Monitoring to Assess Safety
Conduct the following laboratory tests at baseline and prior to each dose of SPINRAZA and as
clinically needed [see Warnings and Precautions (5.1, 5.2)]:
• Platelet count
• Prothrombin time; activated partial thromboplastin time
• Quantitative spot urine protein testing
3 DOSAGE FORMS AND STRENGTHS
Injection: 12 mg/5 mL (2.4 mg/mL) nusinersen as a clear and colorless solution in a single-dose
vial.
4 CONTRAINDICATIONS
None.
5 WARNINGS AND PRECAUTIONS
5.1 Thrombocytopenia and Coagulation Abnormalities
Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia,
have been observed after administration of some antisense oligonucleotides.
In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 24 of 146
(16%) SPINRAZA-treated patients with high, normal, or unknown platelet count at baseline
developed a platelet level below the lower limit of normal, compared to 10 of 72 (14%) sham-
controlled patients.
In the sham-controlled study in patients with later-onset SMA (Study 2), two SPINRAZA-treated
patients developed platelet counts less than 50,000 cells per microliter, with a lowest level of
10,000 cells per microliter recorded on study day 28.
Because of the risk of thrombocytopenia and coagulation abnormalities from SPINRAZA,
patients may be at increased risk of bleeding complications.
3
Reference ID: 4625921
Perform a platelet count and coagulation laboratory testing at baseline and prior to each
administration of SPINRAZA and as clinically needed.
5.2 Renal Toxicity
Renal toxicity, including potentially fatal glomerulonephritis, has been observed after
administration of some antisense oligonucleotides.
SPINRAZA is present in and excreted by the kidney [see Clinical Pharmacology (12.3)]. In the
sham-controlled studies for patients with infantile-onset and later-onset SMA, 71 of 123 (58%)
of SPINRAZA-treated patients had elevated urine protein, compared to 22 of 65 (34%) sham-
controlled patients. Conduct quantitative spot urine protein testing (preferably using a first
morning urine specimen) at baseline and prior to each dose of SPINRAZA. For urinary protein
concentration greater than 0.2 g/L, consider repeat testing and further evaluation.
6 ADVERSE REACTIONS
The following serious adverse reactions are described in detail in other sections of the labeling:
• Thrombocytopenia and Coagulation Abnormalities [see Warnings and Precautions (5.1)]
• Renal Toxicity [see Warnings and Precautions (5.2)]
6.1 Clinical Trials Experience
Because clinical trials are conducted under widely varying conditions, adverse reaction rates
observed in the clinical trials of SPINRAZA cannot be directly compared to rates in clinical
trials of other drugs and may not reflect the rates observed in practice.
In clinical studies, 346 patients (47% male, 76% Caucasian) were treated with SPINRAZA,
including 314 exposed for at least 6 months, 258 exposed for at least 1 year, and 138 exposed for
at least 2 years. The safety of SPINRAZA was studied in presymptomatic infants with SMA;
pediatric patients (approximately 3 days to 16 years of age at first dose) with symptomatic SMA;
in a sham-controlled trial in infants with symptomatic SMA (Study 1; n=80 for SPINRAZA,
n=41 for control); in a sham-controlled trial in children with symptomatic SMA (Study 2; n=84
for SPINRAZA, n=42 for control); in an open-label study in presymptomatic infants (Study 3,
n=25) and other studies in symptomatic infants (n=54) and later-onset patients (n=103). In Study
1, 58 patients were exposed for at least 6 months and 28 patients were exposed for at least 12
months. In Study 2, 84 patients were exposed for at least 6 months and 82 patients were exposed
for at least 12 months.
Clinical Trial in Infantile-Onset SMA (Study 1)
In Study 1, baseline disease characteristics were largely similar in the SPINRAZA-treated
patients and sham-control patients except that SPINRAZA-treated patients at baseline had a
higher percentage compared to sham-control patients of paradoxical breathing (89% vs 66%),
4
Reference ID: 4625921
pneumonia or respiratory symptoms (35% vs 22%), swallowing or feeding difficulties (51% vs
29%), and requirement for respiratory support (26% vs 15%).
The most common adverse reactions that occurred in at least 20% of SPINRAZA-treated patients
and occurred at least 5% more frequently than in control patients were lower respiratory
infection and constipation. Serious adverse reactions of atelectasis were more frequent in
SPINRAZA-treated patients (18%) than in control patients (10%). Because patients in Study 1
were infants, adverse reactions that are verbally reported could not be assessed in this study.
Table 1. Adverse Reactions that Occurred in at Least 5% of SPINRAZA Patients and
Occurred at Least 5% More Frequently or At Least 2 Times as Frequently Than
in Control Patients with Infantile-Onset SMA (Study 1)
Adverse Reactions
SPINRAZA 12 mg1
N = 80
%
Sham-Procedure Control
N = 41
%
Lower respiratory
infection2
55
37
Constipation
35
22
Teething
18
7
Urinary tract infection
9
0
Upper respiratory tract
congestion
8
2
Ear infection
6
2
Flatulence
5
2
Decreased weight
5
2
1 Loading doses followed by 12 mg (5 mL) once every 4 months 2 Includes adenovirus infection, bronchiolitis, bronchitis, bronchitis viral, corona virus infection, Influenza, lower respiratory tract infection, lower respiratory tract infection viral, lung infection, parainfluenzae virus infection, pneumonia, pneumonia
bacterial, pneumonia influenzal, pneumonia moraxella, pneumonia parainfluenzae viral, pneumonia pneumococcal, pneumonia
pseudomonal, pneumonia respiratory syncytial viral, pneumonia viral, and respiratory syncytial virus bronchiolitis.
In an open-label clinical study in infants with symptomatic SMA, severe hyponatremia was
reported in a patient treated with SPINRAZA requiring salt supplementation for 14 months.
Cases of rash were reported in patients treated with SPINRAZA. One patient, 8 months after
starting SPINRAZA treatment, developed painless red macular lesions on the forearm, leg, and
foot over an 8-week period. The lesions ulcerated and scabbed over within 4 weeks, and resolved
5
Reference ID: 4625921
over several months. A second patient developed red macular skin lesions on the cheek and hand
ten months after the start of SPINRAZA treatment, which resolved over 3 months. Both cases
continued to receive SPINRAZA and had spontaneous resolution of the rash.
SPINRAZA may cause a reduction in growth as measured by height when administered to
infants, as suggested by observations from the controlled study. It is unknown whether any effect
of SPINRAZA on growth would be reversible with cessation of treatment.
Clinical Trial in Later-Onset SMA (Study 2)
In Study 2, baseline disease characteristics were largely similar in the SPINRAZA-treated
patients and sham-control patients except for the proportion of SPINRAZA-treated patients who
had ever achieved the ability to stand without support (13% vs 29%) or walk with support (24%
vs 33%).
The most common adverse reactions that occurred in at least 20% of SPINRAZA-treated patients
and occurred at least 5% more frequently than in control patients were pyrexia, headache,
vomiting, and back pain.
6
Reference ID: 4625921
Table 2. Adverse Reactions that Occurred in at Least 5% of SPINRAZA Patients and
Occurred at Least 5% More Frequently or At Least 2 Times as Frequently Than
in Control Patients with Later-Onset SMA (Study 2)
Adverse Reactions
SPINRAZA 12 mg1
N=84
%
Sham-Procedure Control
N=42
%
Pyrexia
43
36
Headache
29
7
Vomiting
29
12
Back pain
25
0
Epistaxis
7
0
Fall
5
0
Respiratory tract congestion
5
2
Seasonal allergy
5
2
1 Loading doses followed by 12 mg (5 mL) once every 6 months
Post-lumbar puncture syndrome has also been observed after administration of SPINRAZA.
6.2 Immunogenicity
As with all oligonucleotides, there is potential for immunogenicity. The detection of antibody
formation is highly dependent on the sensitivity and specificity of the assay. Additionally, the
observed incidence of antibody (including neutralizing antibody) positivity in an assay may be
influenced by several factors, including assay methodology, sample handling, timing of sample
collection, concomitant medications, and underlying disease. For these reasons, comparison of
the incidence of antibodies to nusinersen in the studies described below with the incidence of
antibodies in other studies or to other products may be misleading.
The immunogenic response to nusinersen was evaluated in 294 patients with post-baseline
plasma samples for anti-drug antibodies (ADAs). Seventeen patients (6%) developed treatment-
emergent ADAs, of which 5 were transient, 12 were considered to be persistent. Persistent was
defined as having one positive test followed by another one more than 100 days after the first
positive test. In addition, “persistent” is also defined as having one or more positive samples and
no sample more than 100 days after the first positive sample. Transient was defined as having
one or more positive results and not confirmed to be persistent. There are insufficient data to
evaluate an effect of ADAs on clinical response, adverse events, or the pharmacokinetic profile
of nusinersen.
7
Reference ID: 4625921
6.3 Postmarketing Experience
The following adverse reactions have been identified during post-approval use of SPINRAZA.
Because these reactions are reported voluntarily from a population of uncertain size, it is not
always possible to reliably estimate their frequency or establish a causal relationship to drug
exposure.
Serious infections associated with lumbar puncture, such as meningitis, have been observed.
Hydrocephalus, aseptic meningitis, and hypersensitivity reactions (e.g. angioedema, urticaria,
rash) have also been reported.
8 USE IN SPECIFIC POPULATIONS
8.1 Pregnancy
Risk Summary
There are no adequate data on the developmental risk associated with the use of SPINRAZA in
pregnant women. When nusinersen was administered by subcutaneous injection to mice
throughout pregnancy and lactation, developmental toxicity (long-term neurobehavioral
impairment) was observed at all doses tested (see Data). In the U.S. general population, the
estimated background risk of major birth defects and miscarriage in clinically recognized
pregnancies is 2 to 4% and 15 to 20%, respectively. The background risk of major birth defects
and miscarriage for the indicated population is unknown.
Data
Animal Data
When nusinersen (0, 3, 10, or 25 mg/kg) was administered subcutaneously to male and female
mice every other day prior to and during mating and continuing in females throughout
organogenesis, no adverse effects on embryofetal development were observed. Subcutaneous
administration of nusinersen (0, 6, 12.6, or 25 mg/kg) to pregnant rabbits every other day
throughout organogenesis produced no evidence of embryofetal developmental toxicity.
When nusinersen (1.4, 5.8, or 17.2 mg/kg) was administered to pregnant female mice by
subcutaneous injection every other day throughout organogenesis and continuing once every six
days throughout the lactation period, adverse neurobehavioral effects (alterations in locomotor
activity, learning and memory deficits) were observed when offspring were tested after weaning
or as adults. A no-effect level for neurobehavioral impairment was not established.
8
Reference ID: 4625921
8.2 Lactation
Risk Summary
There are no data on the presence of nusinersen in human milk, the effects on the breastfed
infant, or the effects of the drug on milk production.Nusinersen was detected in the milk of
lactating mice when administered by subcutaneous injection. The developmental and health
benefits of breastfeeding should be considered along with the mother’s clinical need for
SPINRAZA and any potential adverse effects on the breastfed infant from SPINRAZA or from
the underlying maternal condition.
8.4 Pediatric Use
The safety and effectiveness of SPINRAZA in pediatric patients from newborn to 17 years have
been established [see Clinical Studies (14.1)].
Juvenile Animal Toxicity Data
In intrathecal toxicity studies in juvenile monkeys, administration of nusinersen (0, 0.3, 1, or 3
mg/dose for 14 weeks and 0, 0.3, 1, or 4 mg/dose for 53 weeks) resulted in brain histopathology
(neuronal vacuolation and necrosis/cellular debris in the hippocampus) at the mid and high doses
and acute, transient deficits in lower spinal reflexes at the high dose in each study. In addition,
possible neurobehavioral deficits were observed on a learning and memory test at the high dose
in the 53-week monkey study. The no-effect dose for neurohistopathology in monkeys (0.3
mg/dose) is approximately equivalent to the human dose when calculated on a yearly basis and
corrected for the species difference in CSF volume.
8.5 Geriatric Use
Clinical studies of SPINRAZA did not include sufficient numbers of subjects aged 65 and over
to determine whether they respond differently from younger subjects.
11 DESCRIPTION
Nusinersen is a modified antisense oligonucleotide, where the 2’-hydroxy groups of the
ribofuranosyl rings are replaced with 2’-O-2-methoxyethyl groups and the phosphate linkages
are replaced with phosphorothioate linkages. Nusinersen binds to a specific sequence in the
intron downstream of exon 7 of the SMN2 transcript. The structural formula is:
9
Reference ID: 4625921
-
N
N
O
H3C NH
O
H3C
O NH
HO H3C N O
N O O
O R NH
2
NH
O
N O
O
O R
a+-
NH2
N
H3C
NH2
N
N O
Na+-
S H3C
P O N
O
O N O
O R
NH2
Na+-
S
O R O
H3C P O NH
O
O N O
N S P O
O N
O
O R
a+-
N
N
NH2
N
Na+-
S
O
O R O
H3C P O NH
O
Na+-
S P O N
O
O N
Na+-
S
O R
H3C P O
NH2
N
N S P O N
O N
O N
O N O
O
O R
NH2
O R H C
O N O
O
O
a+-
R O
H3C
Na+-
S P O
O
N NH
N
Na+-
S P O 3
N
O N O
O
O R
O
O R
Na+ S P O N
O N
O
NH2
N
N
N S P O NH
O
O N O
O R O
O
O R
Na+-
S P O
N NH2
O
N NH
Na+-
S H
3C
P O NH
O
O N O
O R
O R
Na+-
S P O
O
Na+-
S P O N
O N
O
O R
NH
N NH2
O N
O
OH R
N NH2
Na+-
S P O
O
Na+- S P O
O
R = OCH2CH2OCH3
SPINRAZA is supplied as a sterile, preservative-free, colorless solution for intrathecal use in a
single-dose glass vial. Each 1 mL solution contains 2.4 mg of nusinersen (equivalent to 2.53 mg
of nusinersen sodium salt). Each 1 mL also contains calcium chloride dihydrate (0.21 mg) USP,
magnesium chloride hexahydrate (0.16 mg) USP, potassium chloride (0.22 mg) USP, sodium
chloride (8.77 mg) USP, sodium phosphate dibasic anhydrous (0.10 mg) USP, sodium phosphate
monobasic dihydrate (0.05 mg) USP, and Water for Injection USP. The product may contain
hydrochloric acid or sodium hydroxide to adjust pH. The pH is ~7.2.
The molecular formula of SPINRAZA is C234H323N61O128P17S17Na17 and the molecular weight is
7501.0 daltons.
12 CLINICAL PHARMACOLOGY
12.1 Mechanism of Action
SPINRAZA is an antisense oligonucleotide (ASO) designed to treat SMA caused by mutations in
chromosome 5q that lead to SMN protein deficiency. Using in vitro assays and studies in
transgenic animal models of SMA, SPINRAZA was shown to increase exon 7 inclusion in SMN2
messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein.
12.2 Pharmacodynamics
Autopsy samples from patients (n=3) had higher levels of SMN2 messenger ribonucleic acid
(mRNA) containing exon 7 in the thoracic spinal cord compared to untreated SMA infants.
Cardiac Electrophysiology
10
Reference ID: 4625921
Across the sham-controlled studies in 247 patients with spinal muscular atrophy who received
either SPINRAZA or sham-control, QTcF values >500 ms and change from baseline values >60
ms were observed in 4 (2.4%) patients receiving SPINRAZA. Compared to the sham-control,
there was no increase in the incidence of cardiac adverse reactions associated with delayed
ventricular repolarization in patients treated with SPINRAZA.
12.3 Pharmacokinetics
Absorption
Intrathecal injection of SPINRAZA into the cerebrospinal fluid (CSF) allows nusinersen to be
distributed from the CSF to the target central nervous system (CNS) tissues. Following
intrathecal administration, trough plasma concentrations of nusinersen were relatively low,
compared to the trough CSF concentration. Median plasma Tmax values ranged from 1.7 to 6.0
hours. Mean plasma Cmax and AUC values increased approximately dose-proportionally up to a
dose of 12 mg.
Distribution
Autopsy data from patients (n=3) showed that SPINRAZA administered intrathecally was
distributed within the CNS and peripheral tissues, such as skeletal muscle, liver, and kidney.
Elimination
Metabolism
Nusinersen is metabolized via exonuclease (3’- and 5’)-mediated hydrolysis and is not a
substrate for, or inhibitor or inducer of CYP450 enzymes.
Excretion
The mean terminal elimination half-life is estimated to be 135 to 177 days in CSF, and 63 to 87
days in plasma. The primary route of elimination is likely by urinary excretion for nusinersen
and its chain-shortened metabolites. At 24 hours, only 0.5% of the administered dose was
recovered in the urine.
13 NONCLINICAL TOXICOLOGY
13.1 Carcinogenesis, Mutagenesis, Impairment of Fertility
Carcinogenesis
Long-term studies in animals to evaluate the carcinogenic potential of nusinersen have not been
performed.
Mutagenesis
Nusinersen demonstrated no evidence of genotoxicity in in vitro (Ames and chromosomal
aberration in CHO cells) and in vivo (mouse micronucleus) assays.
Impairment of Fertility
11
Reference ID: 4625921
When nusinersen (0, 3, 10, or 25 mg/kg) was administered by subcutaneous injection to mice
every other day prior to and during mating and continuing in females throughout organogenesis,
no adverse effects on male or female fertility were observed.
14 CLINICAL STUDIES
The efficacy of SPINRAZA was demonstrated in two double-blind, sham-procedure controlled
clinical trials in symptomatic infantile-onset and later-onset SMA patients (Study 1 and Study 2)
and was supported by open-label clinical trials conducted in presymptomatic and symptomatic
SMA patients. The overall findings from these trials support the effectiveness of SPINRAZA
across the range of SMA patients, and appear to support the early initiation of treatment with
SPINRAZA.
14.1 Infantile-Onset SMA
Study 1 (NCT02193074) was a multicenter, randomized, double-blind, sham-procedure
controlled study in 121 symptomatic infants ≤ 7 months of age at the time of first dose,
diagnosed with SMA (symptom onset before 6 months of age). Patients were randomized 2:1 to
receive either 12 mg SPINRAZA or sham injection as a series of loading doses administered
intrathecally followed by maintenance doses administered every 4 months. Patients in this study
were deemed most likely to develop Type 1 SMA.
A planned interim efficacy analysis was conducted based on patients who died, withdrew, or
completed at least 183 days of treatment. Of the 82 patients included in the interim analysis (52
patients in the SPINRAZA-treated group and 30 in the sham-control group), 44% were male,
87% were Caucasian, 2% were Black, and 4% were Asian. Age at first treatment ranged from 30
to 262 days (median 181). Length of treatment ranged from 6 to 442 days (median 261 days).
Baseline demographics were balanced between the SPINRAZA and control groups with the
exception of age at first treatment (median age 175 vs. 206 days, respectively). The SPINRAZA
and control groups were balanced with respect to gestational age, birth weight, disease duration,
and SMN2 copy number. Median disease duration was 14 weeks. There was some imbalance in
age at symptom onset with 88% of subjects in the SPINRAZA group and 77% in the control
group experiencing symptoms within the first 12 weeks of life.
The primary endpoint assessed at the time of interim analysis was the proportion of responders:
patients with an improvement in motor milestones according to Section 2 of the Hammersmith
Infant Neurologic Exam (HINE). This endpoint evaluates seven different areas of motor
milestone development, with a maximum score between 2-4 points for each, depending on the
milestone, and a total maximum score of 26. A treatment responder was defined as any patient
with at least a 2-point increase (or maximal score of 4) in ability to kick (consistent with
improvement by at least 2 milestones), or at least a 1-point increase in the motor milestones of
head control, rolling, sitting, crawling, standing or walking (consistent with improvement by at
least 1 milestone). To be classified as a responder, patients needed to exhibit improvement in
more categories of motor milestones than worsening. Of the 82 patients who were eligible for
the interim analysis, a statistically significantly greater percentage of patients achieved the
12
Reference ID: 4625921
definition of a motor milestone responder in the SPINRAZA group (40%) compared to the sham-
control group (0%). Results from the final analysis were consistent with those from the interim
analysis (Table 3). Fifty-one percent of patients in the SPINRAZA group achieved the definition
of a motor milestone responder compared to 0% of patients in the sham-control group. Figure 1
is a descriptive display of the distribution of net change from baseline in the total motor
milestone score for Section 2 of the HINE for patients in the final efficacy set who did not die or
withdraw from the study.
The primary endpoint assessed at the final analysis was time to death or permanent ventilation (≥
16 hours ventilation/day continuously for > 21 days in the absence of an acute reversible event or
tracheostomy). Statistically significant effects on event-free survival and overall survival were
observed in patients in the SPINRAZA group compared to those in the sham-control group
(Table 4). A 47% reduction in the risk of death or permanent ventilation was observed in the
SPINRAZA group (p=0.005) (Figure 2). Median time to death or permanent ventilation was not
reached in SPINRAZA group and was 22.6 weeks in the sham-control group. A statistically
significant 63% reduction in the risk of death was also observed (p=0.004).
At the final analysis, the study also assessed treatment effects on the Children’s Hospital of
Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND), which is an evaluation
of motor skills in patients with infantile-onset SMA. The CHOP-INTEND results are displayed
in Table 3.
Table 3. Motor Milestone Response and CHOP-INTEND Results of the Final Analysis of
Patients with Infantile-Onset SMA (Study 1)
Endpoint SPINRAZA-treated Patients (n=73)
Sham-control Patients (n=37)
Motor function Motor milestones1
Proportion achieving pre-defined motor milestone responder criteria (HINE section 2)2,3
37 (51%) P<0.0001
0 (0%)
CHOP-INTEND1
Proportion achieving a 4-point improvement
Proportion achieving a 4-point worsening4
52 (71%)
p<0.0001
2 (3%)
1 (3%)
17 (46%) 1At the final analysis, CHOP-INTEND and motor milestone analyses were conducted using the Efficacy Set
(SPINRAZA n=73; Sham-control n=37). 2Assessed at the later of Day 183, Day 302, and Day 394 Study Visit 3According to HINE section 2: ≥2 point increase [or maximal score] in ability to kick, OR ≥1 point increase in the
motor milestones of head control, rolling, sitting, crawling, standing or walking, AND improvement in more
categories of motor milestones than worsening), defined as a responder for this primary analysis. 4Not statistically controlled for multiple comparisons
13
Reference ID: 4625921
Table 4. Survival Results of Patients with Infantile-Onset SMA (Study 1 ) Endpoint SPINRAZA-treated
Patients (n=80) Sham-control Patients
(n=41)
Survival Event-free survival1
Number of patients who died or received permanent ventilation
Hazard ratio (95% CI)
p-value2
31 (39%)
28 (68%)
0.53 (0.32 -0.89)
p=0.005
Overall survival1
Number of patients who died
Hazard Ratio (95% CI)
p-value2
13 (16%)
16 (39%)
0.37 (0.18 – 0.77)
p=0.004
1At the final analysis, event-free survival and overall survival were assessed using the Intent to Treat population
(ITT SPINRAZA n=80; Sham-control n=41). 2Based on log-rank test stratified by disease duration
Figure 1. Percent of Patients Who Died and Net Change from Baseline in Total Motor
Milestone Score (HINE) Among Patients Alive in the Final Efficacy Set of Study
1 *
*For subjects who were alive and ongoing in the study, the change in total motor milestone score was calculated at the later of Day 183, Day 302, or Day 394.
14
Reference ID: 4625921
Figure 2. Event-Free Survival in the Intent to Treat Set
14.2 Later-Onset SMA
Study 2 (NCT02292537) was a multicenter, randomized, double-blind, sham-procedure
controlled study in 126 symptomatic children with later-onset SMA (symptom onset after
6 months of age). Patients were randomized 2:1 to either SPINRAZA 12 mg or sham injection as
a series of loading doses administered intrathecally followed by maintenance doses administered
every 6 months.
The median age at screening was 3 years (range 2-9 years), and the median age of onset of
clinical signs and symptoms of SMA was 11 months (range 6-20 months). Of the 126 patients
included in the study, 47% were male, 75% were Caucasian, 2% were Black, and 18% were
Asian. Length of treatment ranged from 324 to 482 days (median 450 days). At baseline, patients
had a mean Hammersmith Functional Motor Scale – Expanded (HFMSE) score of 21.6, all had
achieved independent sitting, and no patients had achieved independent walking. Patients in this
study were deemed most likely to develop Type 2 or 3 SMA.
The primary endpoint assessed was the change from baseline score at Month 15 on the HFMSE.
The HFMSE evaluates motor function in patients with SMA who have limited ambulation,
comprising of 33 scored activities that give objective information on motor ability and clinical
15
Reference ID: 4625921
progression, such as the ability to sit unassisted, stand, or walk. Each item is scored from 0-2,
with a maximum total score of 66. Higher scores indicate better motor function. The primary
analysis was conducted in the Intent to Treat (ITT) population, which included all subjects who
were randomized and received at least 1 dose of SPINRAZA or at least one sham procedure. At
the final analysis, a statistically significant improvement in HFMSE scores from baseline to
Month 15 was observed in the SPINRAZA-treated group compared to the sham-control group
(Table 5).
Table 5: HFMSE Results in Patients with Later-Onset SMA (Study 2)
Endpoint SPINRAZA-treated
Patients (n=84) Sham-control Patients
(n=42)
HFMSE score
Change from baseline in total
HFMSE score at 15 months1,2,3
Proportion of patients who
achieved at least a 3-point
improvement from baseline to
Month 151
3.9 (95% CI: 3.0, 4.9)
p=0.0000001
56.8% (95% CI: 45.6,
68.1)
p=0.00064
-1.0 (95% CI: -2.5, 0.5)
26.3% (95% CI: 12.4,
40.2)
1Assessed using the Intent to Treat population who received at least one dose of SPINRAZA or at least one sham
procedure (SPINRAZA n=84; Sham-control n=42); data for patients without a Month 15 visit were imputed using
the multiple imputation method 2Least squares mean 3Negative value indicates worsening, positive value indicates improvement. 4Based on logistic regression with treatment effect and adjustment for each subject's age at screening and HFMSE
score at baseline
16
Reference ID: 4625921
Figure 3. Mean Change from Baseline in HFMSE Score Over Time in the Intent to Treat
Set1, 2(Study 2)
1Data for patients without a Month 15 visit were imputed using the multiple imputation method 2Error bars denote +/- standard error
14.3 Presymptomatic SMA
The results of the sham-controlled trial in infantile-onset (Study 1) (NCT02193074) and later-
onset (Study 2) (NCT02292537) SMA patients were supported by an open-label uncontrolled
trial conducted in 25 presymptomatic SMA patients who had a genetic diagnosis of 5q SMA and
2 or 3 copies of SMN2 (Study 3) (NCT02386553). In Study 3, 15 patients (60%) who had 2
SMN2 copies, and 10 patients (40%) who had 3 SMN2 copies; 48% were male, 56% were
Caucasian, 12 % were Asian, 4% were American Indian or Alaska Native, and 28% were of
another race, or had no race reported. Patients ranged in age from 3 days to 42 days (median 22
days) at the time of first dose. Patients received 12 mg SPINRAZA as a series of loading doses
administered intrathecally, followed by maintenance doses administered every 4 months. Patients
were assessed with the World Health Organization (WHO) motor milestones, a set of 6
milestones in motor development that would be expected to be attained by 24 months of age in
healthy children. An interim analysis was performed after all patients had received SPINRAZA
17
Reference ID: 4625921
for at least 14 months (median 25 months, range 14 to 34 months). Patients ranged in age from
14 to 34 months (median age of 26 months) at the time of the analysis. At the time of interim
analysis (data cutoff May 2018), all patients receiving SPINRAZA before the onset of SMA
symptoms survived without requiring permanent ventilation, and beyond what would be
expected based on their SMN2 copy number. All 25 patients (100%) had achieved the WHO
motor milestone of sitting without support, and 22 patients (88%) had achieved the milestone of
walking with assistance. Of the 22 patients who were older than the age expected to have
achieved the ability to walk independently (as defined by the 95th percentile of the WHO
expected age of achievement), 17 (77%) achieved the milestone of walking alone (i.e., walking
independently).
16 HOW SUPPLIED/STORAGE AND HANDLING
16.1 How Supplied
SPINRAZA injection is a sterile, clear and colorless solution supplied as a 12 mg/5 mL (2.4
mg/mL) solution in a single-dose glass vial free of preservatives. The NDC is 64406-058-01.
16.2 Storage and Handling
Store in a refrigerator between 2°C to 8°C (36°F to 46°F) in the original carton to protect from
light. Do not freeze.
SPINRAZA should be protected from light and kept in the original carton until time of use.
If no refrigeration is available, SPINRAZA may be stored in its original carton, protected from
light at or below 30oC (86oF) for up to 14 days.
Prior to administration, unopened vials of SPINRAZA can be removed from and returned to the
refrigerator, if necessary. If removed from the original carton, the total combined time out of
refrigeration should not exceed 30 hours at a temperature that does not exceed 25oC (77oF).
17 PATIENT COUNSELING INFORMATION
Thrombocytopenia and Coagulation Abnormalities
Inform patients and caregivers that SPINRAZA could increase the risk of bleeding. Inform
patients and caregivers of the importance of obtaining blood laboratory testing at baseline and
prior to each dose to monitor for signs of increased potential for bleeding. Instruct patients and
caregivers to seek medical attention if unexpected bleeding occurs [see Warnings and
Precautions (5.1)].
Renal Toxicity
Inform patients and caregivers that SPINRAZA could cause renal toxicity. Inform patients and
caregivers of the importance of obtaining urine testing at baseline and prior to each dose to
monitor for signs of potential renal toxicity [see Warnings and Precautions (5.2)].
18
Reference ID: 4625921
49655-08
Manufactured for:
Biogen
Cambridge, MA 02142
SPINRAZA is a registered trademark of Biogen.
© Biogen 2016-2020
19