Serenity Cole enjoyed Christmas last month relaxing with her family near her St. Louis home, making crafts and visiting friends.
It was a contrast to how Cole, 18, spent part of the 2024 holiday season. She was in the hospital — a frequent occurrence with sickle cell disease, a genetic condition that damages oxygen-carrying red blood cells and for years caused debilitating pain in her arms and legs. Flare-ups often would force her to cancel plans or miss school.
“With sickle cell it hurts every day,” she said. “It might be more tolerable some days, but it’s a constant thing.”
In May, Cole completed a several-months-long gene therapy treatment that helps reprogram the body’s stem cells to produce healthy red blood cells.
She was one of the first Medicaid enrollees nationally to benefit from a new payment model in which the federal government negotiates the cost of a cell or gene therapy with pharmaceutical companies on behalf of state Medicaid programs — and then holds them accountable for the treatment’s success.
Under the agreement, participating states will receive “discounts and rebates” from the drugmakers if the treatments don’t work as promised, according to the Centers for Medicare & Medicaid Services.
That’s a stark difference from how Medicaid and other health plans typically pay for drugs and therapies — the bill usually gets paid regardless of the treatments’ benefits for patients. But CMS has not disclosed the full terms of the contract, including how much the drug companies will repay if the therapy doesn’t work.
…