Orphan Drugs & Specialized Needs Innomar Strategies

Rare Disease Day 2016 Conference

Agenda

§ Review of Payer Framework

§ Review of Orphan Drug Characteristics- why are they different?

§ Consideration of Current Access Challenges

§ Consideration of alternative models for access:

- Managed Access

- Adaptive Listings

Increased Management of Drug Programs

Private Payer

Value DATA, and Customization will be Key

Manufacturer

Cost-Containment Environment – Price Driven Public Payer

Public Payer

The Payer Framework in Canada

Orphan Drugs

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The “Why” and The “How”

SPECIALTY DRUGS ORPHAN DRUGS

Small Population <500 ~100-300 Patients

Burden of Illness / Population Impact Definitive studies Non-definitive studies

New Born Screening Not Required Required

Delayed Diagnosis / Definitive Diagnosis Definitive Diagnosis Delayed Diagnosis

Genetic Testing / Component Not Applicable ~80% of Rare Disease Population has a genetic component

Reimbursement Challenges Established Reimbursement Framework No clear path, despite other established markets (FDA, EU)

Financial Assistance Yes Yes, with conditions

Health Outcomes Studies Not Required Required

Integrated / Closed Distribution Recommended *Required for Health Outcomes

Measurements and Adherence

Specialty Reimbursement through Private Payers

Timelines

•  Up to 100 days to gain access

•  Variation Private vs. Public

Medical Policy

• Defined criteria for use (e.g. clinical effectiveness, selected specialties, 3° centers, etc.)

• Prior Auth.

• Published

PLA

• Additional conditions to limit exposure for a specific payer (e.g. volume-based price, capped usage, etc.)

• Prior Auth.

• Terms are usually confidential

Case Management

• Reimbursement decision based on individual patient assessment

• Requires prior auth.

• Subject to periodic review

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Cost Containment Is Key PPNs; Capped Mark-Ups; Pooling...

Medavie Blue Cross •  Managing Chronic

Disease Program: 56% of workforce, ≈

•  Self-care approach for chronic disease; targeted health services delivered by specialized HCPs

•  Diabetes Management Program (pharma partnership)

Manulife § Designed to delay listing

& reimbursement until CADTH has reviewed

§ Manulife will review

based on its own schedule

Sun Life Provincial Integration Program

Great West Life •  Monitoring a patient for

a specific period of time to ensure the best health outcome Sun Life Manulife GreenShield Medavie

Private Sector Policy Initiatives

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Green Shield Cooperators Medavie Sun Life Manulife

INSURERS REFERENCING

CADTH/CDR

HEALTH CASE MANAGEMENT PPNs MANAGING CHRONIC

DISEASE

Potential Solutions: Managed Access Programs and Adaptive Listing = Evidence Generation Considerations

Italy

§ Drug-monitoring to asses and track patient eligibility, evaluate utilization in clinical practice, collect epidemiological data (safety data and post marketing info)

§ Meant to guarantee appropriate use of medicines according to its therapeutic indication

§ Providing important information on the tolerability of a new drug and prescribing appropriateness.

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Example of Adaptive Listing Model: Start, Stop Criteria to be negotiated with Manufacturer

Challenges with Managed Access Programs

Defining patient eligibility

•  Collecting data on outcomes

•  Who will be responsible for the patient registries?

•  How will confidentiality be maintained?)

Setting stopping criteria

•  Is it possible to achieve a consensus on stopping criteria when rare diseases are so heterogeneous?

•  How will decisions be made for those who cannot speak for themselves?

Assigning stakeholder roles

and responsibilities

•  What roles will Health Canada, payers, and pharmaceutical companies have?

•  How will patient involvement be organized?

MAPs must work for both patients

and decision-makers.

•  To avoid some of the issues such as backlash when the evidence does not support continued funding

•  There is need to incorporate feedback from all stakeholders, including patients.

MAPs should consider all

support

•  Support for patients but not just the drug alone

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The HealthForward Model in Canada

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Collaborate with the patient and their healthcare team

during all phases of the patient journey from diagnosis to

treatment and beyond

Collecting meaningful insights through well-defined

feedback mechanisms and action planning

Exceptional patient and healthcare

professional quality of care equates to

better overall adherence

Cost-Effectiveness of the HealthForward Model is under Evaluation

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GWL-insuredpatientcohort

HCMpatientcohort

Non-HCMpatientcohort

Fullreimbursementforsixmonths

Fullreimbursementwithoutlimitedtime

Treatmentresponders

Treatmentnon-responders

Continuousreimbursement

Reimbursementdiscontinued

Continuousreimbursement

Reimbursementdiscontinued

Ongoingtreatment

Treatmentdiscontinued

TreatmentadherenceassociatedwithHCM

Ongoingtreatment

Treatmentdiscontinued

TreatmentadherenceassociatedwithoutHCM

Accepted abstract at coming 21st ISPOR international conference: INDEPENDENT FACTORS AFFECTING PATIENT COMPLIANCE TO PRIVATE INSURER-FUNDED HEALTH CASE MANAGEMENT IN CANADA

Opportunities to Leverage Existing Patient Support Program Natural Evidence Generation Approach

PSP Evidence Generation to support market access of orphan drugs Closing the gap between regulatory needs and payer needs

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Considerations for Orphan Program Solutions Cover certain drugs by therapeutic class based on: Adaptive Listing Scheme following a framework based on: •  Agreed upon criteria per Orphan category; •  General framework for post marketing surveillance

requirements

Central Registry/PSP model

•  Testing requirements vary – will not be able to standardize consistent approach

Genetic Testing requirements

•  Link to Payer validated data requirements •  Consider Central Data Collection •  Monitoring tool to provide ability to cover drugs based on

evaluation of outcomes- proactively agreed upon data points

Data Collection criteria based on approved guidelines

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How can We Support the Rare Disease Strategy?

Private Payer

Manufacturer

Public Payer

Public Payer

Provide timely, equitable and evidence-informed care: Right patient, Right drug, Right time

Providing sustainable access to promising therapies Consider offering a unique model based on outcomes for Rare Diseases

Generate real-world evidence to meet market access needs Disease burden studies to demonstrate unmet medical needs and support advocacy of rare disease