In response to concerns over multimillion-dollar price tags for new gene therapies for sickle cell disease, the U.S. government on Tuesday announced a long-awaited “access model” designed to blunt the cost that state Medicaid programs would pay for these curative treatments.
Two therapies recently approved by health regulators cost $2.2 million and $3.1 million, respectively, and have raised alarms over the ability of financially strapped state Medicaid programs to absorb the expense. Sickle cell disease affects an estimated 100,000 Americans. Estimates vary, but the federal government believes as many as 60% of people with sickle cell disease rely on Medicaid for health care coverage.
To cope, the Centers for Medicare and Medicaid Services will pursue so-called outcomes-based agreements. As envisioned, the agency will negotiate pricing and rebates with the drug manufacturers — Vertex Pharmaceuticals and Bluebird Bio — over the next several months on behalf of an undetermined number of states that, starting in January 2025, will have the option to participate in the program.