White Paper
ICER / NEWDIGS White Paper: Managing the Challenges of Paying for Gene Therapy: Strategies for Market Action and Policy Reform
Shadowing the excitement about the transformational potential of many gene therapies has been widespread concern about the combination of uncertainty in the durability of their benefits over the long term and the short-term financial shock of high prices.
The white paper analyzes the intersecting challenges of ensuring patient access for gene therapies, including determining fair prices, managing clinical uncertainty, and mitigating financial shocks.
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Research brief
Analyzing 340B and ASP interactions: Do Federal program rules disincentivize the use of VBCs, despite Medicaid Best Price reform?
Our new work describes the interactions among Medicaid Best Price rebate calculations and those for 340B Ceiling Prices and Average Sales Prices which can create disincentives for providers and therapy developers from the use of Value-Based Contracts.
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Paying for Cures
Take a deep dive into the challenges and solutions, and use our tools and calculators to plan your strategy!
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Research Briefs
Emerging market solutions for financing and reimbursement of gene therapies for sickle cell disease: Why do payment innovation?
February 28, 2024Analyzing 340B and ASP interactions: Do Federal program rules disincentivize the use of VBCs, despite Medicaid Best Price reform?
October 19, 2023FOCUS News
About FoCUS (2016–2024)
Medical research is making significant advances toward treatments that cure major diseases, but these treatments will often entail very high up-front costs. The FoCUS Project targeted the need for new innovative financing and reimbursement models for curative medicines in the US that ensure (1) patient access for needed treatments; (2) affordability for public and private payers; and (3) the sustainability of innovation by manufacturers.
FoCUS activities centered around the design, rapid cycle prototyping, and piloting of financing and reimbursement models for curative therapies in the US, with targeted pilot launches occurring throughout the life of the project. All financing and reimbursement designs derived from work done by disease specific multi-stakeholder Target Area Groups (TAGs) in coordination with NEWDIGS team members, academic faculty and others. Designs were rigorously evaluated through modeling and simulation exercises, evaluated through product specific case studies in NEWDIGS Design Labs and vetted. Lessons were disseminated broadly to inform the stakeholder community, pilot implementations and scale up. Initial priorities for action were curative gene therapies and durable oncology treatments.
FoCUS Partners
FoCUS partners supported the project with financial or in-kind donations. Partners included:
Beth Israel Deaconess Medical Center (BIDMC)
Bill & Melinda Gates Foundation
Blue Cross Blue Shield Association
bluebird bio
Bristol-Myers Squibb
Canadian Agency for Drugs and Technologies in Health (CADTH)
Canadian Organization for Rare Disorders (CORD)
European Federation of Pharmaceutical Industries and Associations (EFPIA)
European Organisation for Rare Diseases (EURORDIS)
European Medicines Agency (EMA)
European network for Health Technology Assessment (EUnetHTA)
Food and Drug Administration (FDA, USA)
Friends of Cancer Research
Harvard Medical School
Harvard School of Public Health
Health Canada
Innovative Medicines Initiative (IMI)
Johnson & Johnson
Massachusetts General Hospital
Medicines and Healthcare products Regulatory Agency (MHRA)
National Healthcare Institute (ZIN, the Netherlands)
National Organization for Rare Disorders (NORD)
National Institute for Health and Care Excellence (NICE)
Pfizer
Sanofi