Medicaid has a new way to pay for costly sickle cell treatment: Only if it works

"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.""

"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, or CMS."

Serenity Cole, 18, experienced frequent hospitalizations and chronic daily pain from sickle cell disease that forced her to cancel plans and miss school. In May, Cole completed a several-months-long gene therapy that reprograms stem cells to produce healthy red blood cells and offers a potential cure for many of the roughly 100,000 primarily Black Americans with sickle cell disease. The therapy's high cost poses a steep financial challenge for Medicaid, the joint state-federal insurer for people with low incomes or disabilities. The federal government negotiated a new payment model to buy therapies on behalf of state Medicaid programs and tie payments to outcomes, requiring discounts and rebates from drugmakers when treatments do not deliver promised results, though CMS has not disclosed full contract terms.

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