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. In less than seven years, the number of US Food and Drug Administration (FDA) approved single-dose gene therapies has grown from zero to 17. 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.
Share the research