Managing the challenges of paying for gene therapy: strategies for market action and policy reform in the United States
budget impact
0302 clinical medicine
payment challenges
clinical uncertainty
White Paper
Public aspects of medicine
RA1-1270
0305 other medical science
gene therapy
fair price
DOI:
10.57264/cer-2024-0118
Publication Date:
2024-11-14T11:56:21Z
AUTHORS (4)
ABSTRACT
Gene therapies delivered through a single administration have revolutionized treatment possibilities for many patients living with serious or fatal conditions such as spinal muscular atrophy, hemophilia and sickle cell disease. However, 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. As the healthcare payment ecosystem prepares for the growing number of gene therapies entering the market, three key interconnected challenges must be addressed: determining a fair price, managing clinical uncertainty and managing short-term budget impacts. This paper identifies specific policy reforms and market-based tools to help the US health system address these challenges to achieve more equitable and affordable access for patients to the growing number of gene therapies expected to be approved in the coming years.
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