Medicaid Tries New Approach With Sickle Cell: Companies Get Paid Only if Costly Gene Therapies Work

February 3, 2026

The federal government has launched an innovative Medicaid payment program for sickle cell disease gene therapies that holds pharmaceutical manufacturers accountable for treatment success through rebates and discounts if therapies fail to work as promised. Two FDA-approved gene therapies, costing between $2. 2 million and $3.

Who is affected

Approximately 100,000 primarily Black Americans with sickle cell disease
About 1,000 Missouri Medicaid enrollees with sickle cell disease (with similar populations in other states)
Roughly half of all Americans with sickle cell disease who are covered by Medicaid
Serenity Cole, an 18-year-old patient from St. Louis who received the gene therapy
State Medicaid programs in 33 states, Washington D.C., and Puerto Rico that have signed onto the initiative
Vertex Pharmaceuticals and Bluebird Bio (the two manufacturers)
Participating hospitals that offer the multimonth therapy (limited number nationwide)

What action is being taken

CMS is negotiating drug costs with pharmaceutical companies on behalf of state Medicaid programs and holding them accountable through rebates if treatments don't work
33 states, Washington D.C., and Puerto Rico have signed onto and are participating in the federal program
Missouri's Medicaid program is providing the therapy to enrollees, with fewer than 10 having received it since the state began offering it in 2025
Maryland is accepting patients for the new sickle cell program (expected to begin this month)
CMS is monitoring patients and tracking results to allow states to seek rebates if treatment is not working
Manufacturers are paying for fertility preservation services as part of the program

Why it matters

This initiative addresses the critical challenge of making potentially life-saving but extremely expensive gene therapies accessible to low-income patients with sickle cell disease, a condition that shortens lifespans by more than two decades and costs the health system nearly $3 billion annually. The outcomes-based payment model represents a fundamental shift from traditional pharmaceutical reimbursement by linking payment to actual treatment success, potentially saving taxpayer dollars while ensuring patients receive effective care. With hundreds of cell and gene therapies in clinical trials and dozens expected to receive FDA approval in coming years, this model could establish a precedent for managing the financial burden of future high-cost treatments, particularly for rare diseases. The program also expands access by removing typical Medicaid restrictions and covering additional costs like fertility preservation that would normally be excluded.

What's next

Maryland is expected to begin accepting patients for the new sickle cell program this month
Missouri plans to provide the therapy to about 30 of its 1,000 Medicaid enrollees with sickle cell disease in the first three years
A federally funded evaluation is expected to track the number of patients in the program and their results
The program could run for as long as 11 years, according to CMS
If the sickle cell payment model works, it will probably lead to similar arrangements for other pricey therapies, particularly for those that treat rare diseases
Serenity Cole expects to earn her high school diploma this spring

Read full article from source: Michigan Chronicle