CBO Outlines Methodology to Estimate Budgetary Impact of Increased SCD Gene Therapy

The Congressional Budget Office (CBO) released a report last month outlining how the agency would estimate the budgetary effects of policies to increase access to gene therapy for sickle cell disease (SCD). The agency would consider treatment costs, savings from improved health and effects on federal spending, including Medicaid, Medicare and disability programs. The report also addressed population and dynamic effects, such as increased tax revenues and labor productivity. However, CBO acknowledged that uncertainties remain due to the limited empirical evidence available on the use, costs, side effects and effectiveness of these relatively new therapies.