The Biden-Harris Administration is taking significant steps to enhance access to treatments for sickle cell disease (SCD), a condition that disproportionately affects Black Americans. The Administration’s Cell and Gene Therapy (CGT) Access Model, set to begin in 2025, aims to improve health outcomes and lower healthcare costs for vulnerable populations, with SCD being the initial focus.
SCD, a painful genetic blood disorder, affects over 100,000 individuals in the U.S. and leads to a reduced life expectancy and frequent hospitalizations. The CGT Access Model, developed by the Centers for Medicare & Medicaid Services’ (CMS) Innovation Center, will implement outcomes-based agreements (OBAs) with drug manufacturers to ensure that pricing for SCD treatments is tied to the effectiveness of the therapies for Medicaid patients.
HHS Secretary Xavier Becerra emphasized the Administration’s commitment to increasing access to high-quality, affordable healthcare. CMS Administrator Chiquita Brooks-LaSure highlighted the transformative potential of gene therapies for SCD, which could lead to healthier lives and reduced long-term healthcare costs.
The CGT Access Model responds to President Biden’s executive order to lower drug costs and will negotiate with manufacturers to ease the financial burden on state Medicaid programs. Liz Fowler, CMS Deputy Administrator and Director of the CMS Innovation Center, noted that the model would simplify administrative processes for states, allowing them to focus on improving access and health outcomes for people with SCD.
CMS will also address care delivery gaps, including fertility preservation services for patients undergoing gene therapy, and offer funding to states that promote equitable access to these therapies. The model could expand to other CGTs in the future, reflecting the Administration’s broader efforts to reduce prescription drug costs.
See “Biden-Harris Administration Announces Action to Increase Access to Sickle Cell Disease Treatments” on the US HHS website (January 30, 2024)
