Managing the challenges of paying for gene therapy: strategies for market action and policy reform in the United States

• DOI: 10.57264/cer-2024-0118
• Publication Date: 2024-11-14T11:56:21Z
• Authors: Sharon Phares; Mark Trusheim; Sarah K Emond; Steven D Pearson
• Keywords: 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

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 transformational potential of gene has been widespread concern combination uncertainty in durability their benefits over long term short-term financial shock high prices. As healthcare payment ecosystem prepares growing number entering market, three key interconnected challenges must be addressed: determining fair price, managing clinical budget impacts. This paper identifies specific policy reforms market-based tools to help US health system address these achieve more equitable affordable access expected approved coming years.