34 Medicaid programs in: CMS widens gene therapy deals

CMS will open its Cell and Gene Therapy Access Model to treatments beyond sickle cell disease, the agency’s Innovation Center said on Oct. 27. In the coming months it will release a request for applications so makers of other cell and gene therapies can negotiate with CMS. Talks can start right after a therapy wins FDA approval, or for one already on the market.

In the model, CMS negotiates with drugmakers on behalf of state Medicaid programs, and each state decides whether to sign. Payment is tied to how patients do. If a therapy fails to deliver agreed results, the maker pays the state a rebate.

What the first round did

The first round covered the two FDA approved gene therapies for sickle cell disease: Casgevy from Vertex Pharmaceuticals and Lyfgenia from Genetix Biotherapeutics. Each costs about $2 million to $3 million per patient. That figure comes from the model’s first evaluation report, written for CMS by Abt Global and dated Aug. 13, 2026.

• Medicaid programs in: 34, counting 32 states, the District of Columbia and Puerto Rico
• Share of Medicaid sickle cell patients covered: about 84%
• Drugmakers in: both of the 2 eligible
• Talks with drugmakers: about nine months in 2024, finished on schedule

Sickle cell disease affects about 100,000 Americans, and about half are covered by Medicaid or CHIP, the report says. Standard care eases symptoms but does not cure the disease. The gene therapies change a patient’s genetic code so the body stops making the faulty protein, and they can be curative, the report says.

Who joined and who didn’t

Big caseloads drove sign ups. Of the 19 Medicaid programs with more than 1,000 sickle cell patients, 16 joined. Of the 17 with 200 or fewer, 6 joined, the report found.

Two states that Abt interviewed stayed out because they had struck their own deals with the drugmakers. Those states believe their deals pay higher rebates or protect them better if a treatment fails. Two others stayed out over legal concerns. States told evaluators the CMS contracts were more complex than the rebate deals they usually sign, and some were surprised by the paperwork.

The rebate amounts are confidential. Abt says it had no access to them, so the report can’t say how much states save.

What CMS gets out of it

Pooling patients across states gave CMS leverage no single state had, the evaluation says. The model also lets drugmakers pay for fertility preservation for Medicaid patients, under a federal safe harbor that only CMS could offer. The chemotherapy used before these treatments usually leaves patients infertile, according to the CMS model page.

The report says states and drugmakers named GLP-1 drugs, cancer drugs and diabetes drugs as candidates for future CMS led talks. The Oct. 27 notice covers cell and gene therapies only.

Why it matters to you

Say you or your child has Medicaid and a rare disease with a new gene therapy. This model decides whether your state has a CMS deal for it.

Today that is true only for sickle cell, and only in the 34 programs on the CMS list. To qualify, a patient needs Medicaid as primary coverage in a participating state and has to meet the model’s prior authorization criteria.

Watch for the manufacturer request for applications. It will show which therapies CMS wants next and how soon states could sign.

Medicare’s own new drug model goes the other way on these treatments. The final GLOBE model leaves cell and gene therapies out; see $2.3 billion to $80 million: CMS shrinks GLOBE drug model.