This paper assesses the potential financial impact of novel gene therapies in the United States. The authors develop a simulation model to estimate future spending, considering late-stage clinical trials before January 2020. The simulation factors in disease prevalence, quality-adjusted life years (QALYs), launch prices, and annual spending. Results suggest approximately $20.4 billion in annual spending under conservative assumptions, with roughly half attributed to non-Medicare-insured adults and children. Sensitivity analyses explore various assumptions, and the paper concludes by discussing payment methods and policies to ensure patient access.

Chi Heem Wong, Dexin Li, Nina Wang, Jonathan Gruber, Andrew W. Lo, Rena M. Conti