Planning for the Expansion of Precision Medicine · PDF file · 2018-02-23Molecular...
Transcript of Planning for the Expansion of Precision Medicine · PDF file · 2018-02-23Molecular...
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Precision Medicine Adoption Technology Spotlight: Gene TherapyIntegrating Precision Medicine
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Different Name, Same Definition
Aka; DNA-Based, Genomic or Molecular Medicine
Personalized Medicine:An emerging approach for disease treatment and prevention that takes into account individual variability in genes, environment, and lifestyle for each person.
-National Institutes of Health
Precision Medicine: An emerging approach for disease treatment and prevention that takes into account individual variability in genes, environment, and lifestyle for each person.
-National Institutes of Health
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Consumers Expect An Increasing Role for Genetic Testing but Worry About Interpretation and Resources
In 5 years, what change do you expect for the use of genetic testing?
Source: UnitedHeath. Personalized Medicine: Trends and prospects for the new science of genetic testing and molecular diagnostics. March 2012.
Primary consumer concerns:─ Physician ability to interpret test results─ Confidentiality of test results and possible discrimination─ Better education and resources about the risks and benefits
How familiar are you with genetic testing?
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Physicians View Genetic Testing As Essential; Despite Challenges, Genetic Services Continue to Grow
Source: UnitedHeath. Personalized Medicine: Trends and prospects for the new science of genetic testing and molecular diagnostics. March 2012 The Association of Community Cancer Program. 2015 Oncology Trends Report.
5875
47
78 7260
83 8172
Dx Testing Offered Genetic Counseling Offered Molecular Testing Offered2013 Survey 2014 Survey 2015 Survey
Do you have patients that would benefit from genetic testing?
26%
28%
35%
42%
Provider Education/Awareness
Patient Education/Awaress
Resource Intensive
Reimbursement
Implementation challenges:
75%25%
Yes No
…however, physicians report <10% of their patients having received a genetic test
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Personalized Medicines comprise: 27% of the drugs approved
by the FDA in 2016 42% of all medicines in
development 73% of cancer therapies in
development
Pharma Is Betting On Precision Medicine;Integration Into Clinical Practice in Slow
Sources: Pharmaceutical Research and Manufacturers of America. Medicines in Development for Cancer: From Hope to Cure. 2015 Report.; The Personalized Medicine Report, Personalized Medicine Coalition, 2017 Report; FDA. Table of Pharmacogenomic Biomarkers in Drug Labeling. Accessed June 2016.
“… despite the steady increase in the number of clinically useful molecular diagnostics and targeted therapies, healthcare systems have been slow to
integrate personalized medicine into clinical practice.”
0%
25%
50%
75%
100%
FDA-Approved Drugs With Pharmacogenomic Labeling
Medicine (56)
Cancer (41)
Neurosciences (32)
Cardiovascular (10)
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21
66
42
57
92
Strategy to enhance PM
Budget for PM
Offer PM services
Current Within 2 Years
HIMSS 2017 Precision Medicine Survey:
HIMSS 2017 Precision Medicine Survey
31%
31%
32%
33%
37%
41%
Pharmacogenetics
Cardiology*
Pharmacogenomics
Prenatal screening
Neurology
Oncology
Current Areas of Focus:
Source: Future Proofing Healthcare: Precision Medicine, conducted by HIMSS Analytics, on behalf of Intel, September 2017.
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Essential to Support Precision Medicine
20
22
23
26
38
43
51
45
34
41
41
41
32
27
Precision medicine platform
Molecular diagnostics analysis solution
Precision medicine EMR
Outside storage/computing capabilities
Analytics/data-mining platform
Laboratory IT
Data warehouse
Current Within 2 Years
Challenges Around Precision Medicine
72%
Improvement of IT
Adequacy of existing IT
Budget/financial
Unprepared Moderately prepared Prepared
UnsureInvestUse existing IT
HIMSS 2017 Precision Medicine Survey
Source: Future Proofing Healthcare: Precision Medicine, conducted by HIMSS Analytics, on behalf of Intel, September 2017.
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Precision Medicine Adoption Technology Spotlight: Gene Therapy
Integrating Precision Medicine
AVAILABLE DEVELOPING RESEARCH
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First FDA-approved test
Cancer Moonshot calls for “efforts to develop noninvasive tests”
Liquid Biopsy Tests Are Now ‘Mainstream’
CURRENT IMPACT
Noninvasive Inform treatment Repeatable
40 companies
$30B(US) market
2011 20162012 2013 2014 2015
Prenatal diagnostic testing
Initial liquid biopsy tests for lung cancer
Source: Chen C. Bezos, Gates chase dream of a blood test that detects cancer. Bloomberg: April 6, 2016.
AVAILABLE
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How Will Liquid Biopsies Impact Lung Cancer Therapy?
SBRT = stereotactic body radiation therapy. Sources: Impact of Change®, 2017; HCUP National Inpatient Sample (NIS). Healthcare Cost and Utilization Project (HCUP) 2014. Agency for Healthcare Research and Quality, Rockville, MD; OptumInsight, 2015; The following 2015 CMS Limited Data Sets (LDS): Carrier, Denominator, Home Health Agency, Hospice, Outpatient, Skilled Nursing Facility; Claritas Pop-Facts®, 2017; Sg2 Analysis, 2017.
Growth in Lung Surgery Sg2 projects 27% growth in
lobectomy (10-year).
Growth in Lung Cancer Treatment
Targeted Therapies Sg2 projects 19% growth
in chemotherapy (10-year).
Radiation Therapy Sg2 projects 72% growth
in SBRT (10-year).
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Procedures Infusion and Transfusion Volumes at Risk
Pharmaceutical Companies Biologic Drug Market
Payers High Cost Cures Compromise Margins and Sustainability
Gene Therapy is Now ‘A Thing’: How Did This Happen
Initial Failures
1990s
VC Influx
2018
Dubious BenefitToxicitiesDeaths
Quiet Progress
2000-2016
SafetyEfficiencyDelivery
FDA Approvals
2017
CAR-TBlindnessClinical Trial Successes $Many Trials
More MoneyDemand
IMMINANT DISRUPTION TO:
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Gene Therapy Will Disrupt 3 Areas
Inherited Conditionshemophilia congenital blindness
Acquired Conditionscancer/car-Tchronic diseases
Augmentative Therapies‘biohacking’, DIY science
AVAILABLE DEVELOPING RESEARCH
Emerging Opportunities for Providers:Expansion of Minor Procedures and BMTPrecision Medicine can Identify/Screen Candidates‘Impatient’ patients will develop complications from unregulated therapies
Sources: DIY = “Do-It-Yourself”. This list is not meant to be exhaustive; “Trials Forbidden” = means ethically/officially forbidden to use public funds to research; number of clinical trials reported by the Alliance for Regenerative Medicine, an Industry group, Sg2 Analysis 2018.
>550 clinical trials
Trialson hold
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DescriptionEnable the immune systems ability to attack B-cell malignant tumors by infusing genetically engineered T-cells back into the patient
What Is the Current Impact? Novartis – ALL; Kite (Gilead) – NHL both FDA Approved Numerous trials in CLL, MM, AML Pending long-term response rates; maybe an alternative to BMT
Novartis and Kite Break Ground with FDAApproved CAR T-cell Therapies
ALL = acute lymphoblastic leukemia; CLL = chronic lymphocytic leukemia; NHL = non-Hodgkin lymphomas; MM = multiple myeloma; AML = acute myelogenous leukemia. Source: American Cancer Society. 2017 Facts and Figures; Sg2 Impact of Change Forecast, 2017.
Tumor Type Annual Incidence
2017 IP/OP BMT Volume
% of Total BMT Volume
Leukemia’s 62,130 4371 28%Hodgkin’s Lymphoma 8,260 842 5%Multiple Myeloma 30,280 6445 42%Non-Hodgkin’s Lymphoma 72,240 3320 21%TOTAL 172,910 15,509 100%
Activated T Cell
Tumor Cell
>50% of BMT volume
AVAILABLE
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Roadblocks to Solid Tumor Adoption Are Not Trivial Finding tumor specific (ie, unique) antigens and tumor heterogeneity Penetrating immunosuppressive microenvironment Delivering T-cells directly to the tumor and extending T-cell persistence
Potential Impact on Future Chemo and Radiation Therapy Volumes
Source: www.ClinicalTrials.gov.
Tumor Type Number of Clinical Trials Phase % of Total
Chemotherapy % of Total
Radiation Therapy
Gyn Onc 10 Phase I/II 5% 3%Head and Neck 2 Phase I/II 2% 9%Pancreatic 15 Phase I/II 4% 1%
Prostate 3 Phase I/II 8% 22%
Colorectal 5 Phase I/II 12% 4%Liver 18 Phase I/II 1% 2%
DEVELOPING
Breast 13 Phase I/II 17% 22%
Lung 12 Phase I/II 10% 9%
Brain 9 Phase I/II 1% 5%
What Is the Future Impact?
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What Does Gene Therapy For HemophiliaLook Like?
Procedural Disruption: TransfusionsInfusion volumes
1. Precision medicine identifies patients2. Design DNA 3. Package DNA into virus4. Intravenous Injection
5. DNA delivered to liver cells6. Cells make DNA into protein7. Protein secreted into blood8. No need for bi-weekly biologic infusions
>$155,000 average annual cost per hemophilia patient DISRUPTED
Sources: Cell. Volume 171, Issue 7, p1478–1480, 14 December 2017; Am J Manag Care. 2016;22:S126-S133; Sg2 Analysis 2018.
DEVELOPING
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Biblical Treatments, Goliath Costs
$850,000 Luxturna (for genetic blindness), one-time treatmentFDA Approved December 19th, 2017
$300,000 Eteplirsen (for Duchenne muscular dystrophy), per year$373,000 Yescarta (CAR-T for blood cancer), one-time drug payment$475,000 Kymriah (CAR-T for blood cancer), one-time drug payment$648,000 Strimvelis (ultra rare immune deficiency), per cure (Europe)$750,000 Spinraza (for spinal muscular atrophy), per year
$1,000,000 Glybera (ultra rare blood disease), per cure (Europe)
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Description Genomic scalpel that cuts or repairs DNA at specific places
Easy to break genes; Feasible to rewrite genes
Impact Improved speed, precision, and ease raise ethical concerns Advances in basic biomedical research and drug discovery Future Impact: Advancement will initially disrupt health care in
non-obvious ways
CASE EXAMPLE: Scripps and Harvard Successful ‘humanization’ eliminated 62 pig viruses from pig
organs (kidney) using CRISPR Off-target effects were minimal; Need to make transgenic pigs!
New Genome Editing Tool Is Quickening the Pace of Research and Drug Discovery
CRISPR Technology (pronounced “CRISP·er”)
RESEARCH
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Gene Therapy Strategic Points
What should you do to prepare?
What will the effect be on volumes?
How will you pay for these therapies?
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Precision Medicine Adoption Technologies Spotlight: Gene TherapyIntegrating Precision Medicine
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Challenge 1: Connect an Ecosystem Around Your Electronic Health Record
Sylvester Cancer Center, University of Miami Miller School of Medicine
SITUATION Providers lack the infrastructure to easily
order, store, and share test results EHRs are not designed to manage or
query genetic information; interoperability has not happened
SOLUTION – SYAPSE Connects clinical, molecular, and
outcomes data into the EHR Facilitate molecular tumor boards
SYLVESTER CC RESULTSModified treatment: ~30% Clinical trial enrollment: +1–2
patients/month
Sources: Trent J. Oncology Issues 2017; Nov/Dec:16-20; .
IMPACT OF INTEGRATION Intermountain Healthcare: doubled
progression free survival without increase total cost of care
Improved access to targeted therapies
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Challenge 2: Precision Medicine Programs Must Prepare for Growing Data Management Requirements
Countless number of
data sources and formats.
Integration of patient-generated
dataNeed a data management
strategy
Dedicated IT Infrastructure
Source: Sg2 Analysis, 2018.
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Challenge 3: How Do Providers Afford Precision Medicine?
Product Champion Pro forma Partnerships Philanthropy Pro bonoChallenge to AMCs: Payers are unconvinced of the benefits of PM; evidence demonstrating its value to healthcare is needed AMCs are positioned to conduct studies to demonstrate value
Design clinical studies to serve multiple purposes such as including the cost and benefits of coverage decisions in areas of unmet need
Develop an effective, universally accepted and user-friendly process to systematically collect and share treatment and outcomes data
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First round• Compassionate use (payers more likely to bend to advocacy pressure)• Rare diseases (orphan drug status confers benefits) • Small markets (once approved, huge barriers to entry, trials harder)
Sells to insurer, not hospital (prevents ‘buy and bill’ practices)
Money back guarantees (outcomes-based rebate agreements”)
Financing options (payment spread over several years)
High Costs Are Changing PhRMA’s Pricing Strategy