gene therapy approvals highlight accelerated change in ......apr 05, 2020 · reducing upfront...
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Gene therapy approvals highlight accelerated change in drug environment
April 21, 2020
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About me….
• Executive Vice-president & Chief Clinical Officer at Real Endpoints• Senior Advisor, MIT Center for Biomedical Innovation FOCUS
project• Board Director, Momenta Pharmaceuticals and TherapeuticsMD
• Former Associate Chief Medical Officer, CVS Health• Former Vice-president medical affairs and clinical innovation,
Medco• Former Director health benefits operations and clinical
performance, IBM
Jane F Barlow MD, MPH, MBA
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Overview of today’s discussion
1. Understand the changing market environment and its impact on specialty drug utilization and spend
2. Explore the impact of approvals for rare disease treatments
3. Demonstrate the effect of gene therapy approvals on contracting strategies
Agenda Learning objectives
01 US market dynamics
02 Evolving specialty drug environment
03 Case study: Gene therapies
04 Implications
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01 US Market Dynamics
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US prescription drug costs have more than tripled in the last 20 years
Peterson-KFF Health System Tracker https://www.healthsystemtracker.org/health-spending-explorer/?display=U.S.%2520%2524%2520Billions&service=Prescription%2520Drug&rangeType=range&years=1999%252C2018&source=Out%2520of%2520Pocket%252CHealth%2520Insurance&tab=1 Accessed 5 April 2020
$72.2
$286.2
$30.5
$47.1
$102.7
$333.3
$0.0
$50.0
$100.0
$150.0
$200.0
$250.0
$300.0
$350.0
1999
2000
2001
2002
2003
2004
2005
2006
2007
2008
2009
2010
2011
2012
2013
2014
2015
2016
2017
2018
US $Billions
Payer Pt OOP Total
$374
$1,026
$0
$200
$400
$600
$800
$1,000
$1,200
1999 2018
Per Capita $
Total Prescription Drug
Prescription Drug Expenditures1999-2018
Per Capita Prescription Drug Expenditures 1999 compared with 2018
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Key trends influencing U.S. market
Innovative, targeted treatments and specialty utilization
Payers are challenged to offer affordable benefits, foster health improvement and manage trend
•Increased payer size enables significant pressure/leverage over pharma•Vertical alignments create end-to-end control of the drug channel
•Potential end to protected classes, Part B reforms, international price indexing, drug importation, and direct negotiation
•Specialty drug trend, driven by price and utilization, steadily increasing, necessitating greater utilization management and restrictions
Drug pricing and affordability
Policy climate
Industry consolidation
•Value-based contracting represents a potential shift away from the deep rebate model
•Challenges persist in finding the right fit, developing the right model and implementing
Increase in innovative contracting and risk sharing
•Amplified demand for price transparency and reform •Greater cost sharing pushed on patients at all levels
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Vertical and horizontal integration Government focus on Rx cost and transparency
Innovators and disruptors
Three areas of exponential change having significant impact on the market environment
State Pricing Legislation
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Nearly 2/3rds of lives are enrolled in one of 9 large health plans
SOURCE: MMIT Payer data January 2020
Smaller plans have significant lives on aggregated basis – but limited opportunity to contract
Kaiser12M
United45M
Anthem 33M
Aetna 21M
Cigna 16M
Centene14M
Humana12M
CMS33M
All other plans (385): 125M lives
Coverage of US Population 2019 (327M covered lives)
HCSC16M
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Specialty is growing in absolute dollars and percent drug spend
Pharmacy Drug SpendMedical Drug Spend
% of Total Drug Spend 30% 38% 45% 50%
Total Industry Specialty Spend
20122014 2016
2018
17% Growth Rate
$92B$127B
$179B$235B
Adapted from: Partners Group
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Payers and Pharmacy Benefit Managers (PBMs) are increasingly utilizing strict approaches to manage drugs
• Formulary exclusions• Strict prior authorization criteria• Documentation
Source: Formulary Exclusion Lists published by CVS and ESI18
…leading to tighter management by payer’s across brands
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New approaches: Innovative contracting falls into several buckets…
Type of Contract
Product/ Company
Disease Contract
Outcomes-based: clinical
Luxturna/ Spark Rare retinal dystrophy
Unrestricted access to label following PA; guaranteed WAC pricing from buy-and-bill provider, additional rebate for non-improvement
Onpattro/ Alnylam hATTRamyloidosis
Unrestricted access with prior authorization following label. Multiple rebates including for discontinuation and liver transplant
Outcomes-based: cost
Jardiance/ Boehringer Ingelheim
Diabetes Larger rebate & 1-of-1 formulary if lowers cost of CV disease at health plan
Pay-over-time/ outcomes-based
Zynteglo/ bluebird bio*
Transfusion-dependent β-thalassemia
20% payment annually for 5 years; if certain number of transfusions required, no additional payments.
Cost-cap/ subscription
CV drug/ Biotech** CV disease Guaranteed payment for treating annually increasing % of defined CV/diabetes population with low-end of ICER pricing range & additional discount to reflect $0 copay
*Unsigned and still not final structure; **MoU but final contract unsigned
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02 Evolving specialty drug environment
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Innovative treatments targeted to rare populations are driving increased drug prices
1950 - 2000: Oral primary care drugs
1995-2015: Biologics for specialty categories
2018… Rare/orphan
Price/ScriptPatientsPayers
The most expensive drug in the world: Zolgensma at $2.1M per patient
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Specialty drug spending is poised to surpass traditional drug spending
Managed Care November 2019 based on IQVIA Medicine Use and Spending in the US, May 2019
Moved from 26% of drug spend in 2009 to 49.5% in 2018
Drug Spending by Traditional and Specialty, 2009 to 2018
738 727 699603 595 595 605 592 546 527
262 279 289318 385 385 438 471 489 517
0
200
400
600
800
1000
1200
2009 2010 2011 2012 2013 2014 2015 2016 2017 2018
Traditional drug spend Specialty drug spend
2018
net
per
cap
ita d
rug
spen
d ($
) 1,000 1,006 988931
922 9811,043 1,064 1,034 1,044
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Rare diseases and cancer dominate pipeline and drive growth
1Citeline’s PharmaProjects, 2FoCUS NEWDIGS Initiative at MIT, 3EvaluatePharma, Orphan Drug Report 2019, 4BioCentury, April 2019
• Oncologics comprise 35% of 2018 pipeline1
• Companies are conducting 908 gene/cell therapy trials (in 140 indications)2
• Orphans will comprise 20% of worldwide sales in 20243
• In 4 of the past 5 years, over 40% of first product launches were orphan drugs, reaching a high of 58% in 20184
0
5
10
15
20
25
30
2014 2015 2016 2017 2018
No.
of P
rodu
cts
Orphan Non-orphan
~58%
~45%~41%~22%~40%
SPECIALTY: FIRST PRODUCT LAUNCHES
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03 Case study: Gene therapies
CONFIDENTIAL
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What is a gene therapy?
• Technique that modifies a person’s genes to treat or cure disease
• Mechanisms include:– Replacing a disease-causing gene with a healthy copy– Inactivating a disease-causing gene that is not functioning properly– Introducing a new or modified gene into the body to help treat a disease
• Regulated by the FDA Center for Biologics Evaluation and Research (CBER)– Biologics license application (BLA) required for marketing
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FDA website: https://www.fda.gov/biologicsbloodvaccines/cellulargenetherapyproducts/ucm573960.htm Accessed 29 September 2018
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How do gene therapies work? Hemophilia A example
• Adeno-associated virus serotype 5 (AAV5)-based gene therapy– Functional copy of gene that codes for Factor VIII protein is inserted into AAV5 capsid that delivers the
gene to a patient’s liver cells– Cells use new functional gene to produce Factor VIII protein– Administered by a single intravenous dose
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Examples of current and near-term pipeline gene therapies
Valrox: Hemophilia A
Zolgensma: hATTR Anyloidosis
Zynteglo: Beta Thalassemia
etranacogenedezaparvovec: Hemophilia B
Luxturna: Leber’s congenital amaurosis
5 patients/M lives $2.1M
10 patients/M lives $1.8M Europe
12 patients/M lives $?M
$?M49 patients/M lives
5 patients/M lives $.85 M
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Emerging one-time therapies create financial challenges
Three financial challenges exacerbated
Payment timingOne-time high cost
Performance risk: Effectiveness & durability
Actuarial risk:Likelihood of encountering a case
0
10
20
Period 1 Period 2 Period 3 Period 4 Period 5 Period 6 Period 6
Classic Medicines
Benefit Cost
0
20
40
60
80
Period 1 Period 2 Period 3 Period 4 Period 5 Period 6 Period 6
Durable Therapies
Durable Therapies Distill Payments Upfront
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New treatment innovations challenge current insurance paradigms, threatening access
New durable
therapies
Payer coverage
Patient access
• Growing clinical pipeline on innovative durable therapies
• High one-time cost will likely create financial challenges
• Sustainability for all stakeholders is at risk
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FoCUS: Multi-stakeholder group dedicated to making innovative cures accessible and sustainable
FoCUS: Financing and reimbursements of Cures in the US
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FoCUS Financing and Reimbursement of Cures in the US
FoCUS NEWDIGS Initiative • MIT Center for Biomedical Innovation 22
• Burden of multiple high cost therapies
• Concern about what this will represent in 3-5 years with respect to total cost
• Potential off label use
• Operational management of a larger pipeline
• Adverse selection
Other reasons for high concern
98%84% 91% 91%
65%
2%16% 9% 9%
35%
0%10%20%30%40%50%60%70%80%90%
100%
Total cost is material formy plans
Payment timingrelevant to benefit
realization
Product performancerisk
Actuarial risk Other
Reasons why managing the financial risk of high-cost durable therapies is a high or extremely high concern (n=65)
Agree Disagree
Survey: US Payers’ concerns reflect all these issues
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FoCUS Financing and Reimbursement of Cures in the US
FoCUS NEWDIGS Initiative • MIT Center for Biomedical Innovation 23
Greatest payer interest lies in addressing performance risks
6%9%
3%
18%14%
1%
29% 27%
13%
31%
40%
34%
16%
9%
49%
0%
10%
20%
30%
40%
50%
60%
Reducing upfront budget impact of the newtherapy by smoothing payments over time
Aligning the timing of the therapy costs withits benefits
Only paying for therapy that works
All Payers (N=77)
Not a benefit Slightly beneficial Moderately beneficial Very beneficial Extremely beneficial
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FoCUS Financing and Reimbursement of Cures in the US
Most payers plan to develop strategies for these therapies over the next 2 years
Payers are inclined to manage these therapies differently
The question is high priority over the next two years
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FoCUS NEWDIGS Initiative • MIT Center for Biomedical Innovation
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Three precision financing solutions are emerging to address reimbursement challenges
FoCUS NEWDIGS Initiative • MIT Center for Biomedical Innovation
• Short-term milestone-based contracts• Usually < 2-year duration• Pay for treatment upfront• Specified rebate payments tied to early outcome
• Multi-period performance-based annuities• Usually > 2 years (likely 3 to 5 years)• Payments spread out over contract period• Payment tied to performance over time
• Orphan Reinsurer and Benefit Manager (ORBM) and Risk Pools–Carve out risk and management of novel breakthroughs
Solutions must be customized for the
therapy/disease AND
satisfy each stakeholder
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Overview of a milestone-based contract (MBC)
FoCUS NEWDIGS Initiative • MIT Center for Biomedical Innovation
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One-year MBC timeline:
Via the provider or
intermediary
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• 3 January 2018: Massachusetts insurer Harvard Pilgrim Healthcare announces outcomes-based agreement for vision gene therapy
Treatment&
Payment
30 MonthEye Test
Manufacturer rebate if under-performance
X
Manufacturer rebate if under-performance
30-90 DayEye Test
X
Example milestone-based contract for vision therapy
FoCUS NEWDIGS Initiative • MIT Center for Biomedical Innovation
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Overview of a performance-based annuity (PBA) contract
FoCUS NEWDIGS Initiative • MIT Center for Biomedical Innovation
Pay when patient and healthcare system receive benefits
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Initial Upfront Payment
Assess Outcome Metric
Payer milestone payment if outcome met
Treatment 1 2 3 4 5Years
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NEWDIGS Initiative • MIT Center for Biomedical Innovation • CONFIDENTIAL 29
bluebirdbio company presentation, ready to recode; September 23, 2019. http://investor.bluebirdbio.com/static-files/8c4eb7bb-37d6-4fba-941c-1a154c8bbfd6 Accessed November 18, 2019
• Spread payments over up to 5 years
• Get paid only if the treatment works
Example performance-based annuity for β-thalassemia (Europe)
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Orphan Reinsurer and Benefit Manager (ORBM) concept
• Carve-out actuarial risk
• Enable scale for innovative contracting and financing
• Provide medical management
Source: Trusheim M, Mytelka DS, Warren GL, Han D, Ciarametaro M. Improving Management of Gene and Cell Therapies: The Orphan Reinsurer and Benefit Manager (ORBM). PharmExec 2018 Sept 10. Available at: http://www.pharmexec.com/improving-management-gene-and-cell-therapies
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Source: http://lab.express-scripts.com/lab/insights/specialty-medications/delivering-on-the-promise-of-modern-medicine
Beginning with 2 Gene Therapies
Cigna offering ORBM-Lite: Embarc
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FoCUS Financing and Reimbursement of Cures in the US
Payers may favor different solutions based on their particular dynamics
Approach Commercial Fully Insured
Medicaid Medicare Advantage
Self-Insured Employer
All Payer Segments
Population risk-pooling 69% 40% 52% 50% 55%
Short-term MBC 75% 50% 61% 20% 60%
Long-term MBC 58% 36% 43% 30% 46%
Annuity: Spread payments over >2 years 33% 14% 24% 20% 24%
Performance-based annuity: spread payment over >2 years/tied to performance
47% 21% 35% 30% 35%
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Milestone-Based Contract (MBC): Pay for therapy upfront and receive refunds tied to performance over the short-term (< 2 years) or the long-term (> 2 years)
MIT FoCUS payer survey completed April 2019: 77 participants representing over 280M lives
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Important to start the dialogue on financing solutions early as barriers need to be addressed
FoCUS NEWDIGS Initiative • MIT Center for Biomedical Innovation
Structural Strategic Administrative• Pricing and Reporting
Regulations, esp. Medicaid Best Price
• Insurance Reserve Regulations
• Accounting Cost and Revenue Recognition Rules
• Ability to sign multi-year agreements
• Coverage • Patient mobility• Reinsurance strategy
• Tracking patients over time
• Ability to align on relevant outcomes measures and milestones
• Ability to align on terms• Program administration
effort
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FoCUS Financing and Reimbursement of Cures in the US
FoCUS insights and resources available onlinehttps://www.payingforcures.org/toolkit
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FoCUS Financing and Reimbursement of Cures in the US
Purpose
Target Audience
Format
• Payer and biopharma teams who have not designed a precision financing solution and are considering it • For specific products• For the coming pipeline
• Policy-makers who wish to understand key FoCUS insights
Toolkit: Purpose, Audience, Format
• Web-based• Self-directed• Simple -- “peeling the onion “ iteratively
• Resource for practitioners considering offering or adopting a precision financing solution:• Easy access to FoCUS learnings• Jump start own solution design • Accelerate use of precision financing
NEWDIGS Initiative • MIT Center for Biomedical Innovation
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04 Implications
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Summary
US drug trend continues to grow fueled by specialty and increasingly drugs for orphan conditions
Market consolidation leaves few payers with most of the negotiating power
Gene therapies introduce new areas of uncertainty: Payment timing, performance risk and actuarial uncertainty
New reimbursement models are under consideration to address gene therapy risks
New tools are emerging to support stakeholders in their assessment of gene therapies
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Actions
Educate• Stay abreast of the changing environment• Identify resources and tools to help
Assess
• Understand the impact of orphan drugs including gene therapies on your plan
• Consider cumulative impact in addition to the individual drug impact
Innovate• Explore new reimbursement models• Define a strategy to achieve negotiating power
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