$3M Gene Therapy—With a Catch

Medicaid is testing a pay-for-performance deal that makes drugmakers refund millions if new sickle cell gene therapies fail.

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Serenity Cole, an 18-year-old with sickle cell disease, underwent gene therapy that reprograms stem cells to produce healthy red blood cells.
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The new Medicaid payment model allows states to negotiate gene therapy costs and receive rebates if treatments fail to work as promised.
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The gene therapies approved for sickle cell disease cost between $2.2 million and $3.1 million per patient, excluding hospital stay expenses.
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The Biden administration's initiative aims to tackle high drug costs and could lead to similar payment models for other expensive therapies.
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Gene therapy offers a potential cure for sickle cell disease, which affects primarily Black Americans and significantly shortens lifespans.

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