Medicaid Tries Outcomes‑Based Payments for $2M–$3M Sickle Cell Gene Therapies
CMS has begun operating a national outcomes‑based payment model for two FDA‑approved sickle cell gene therapies, under which Medicaid programs pay multi‑million‑dollar prices only if the treatments work as promised. The model, created under a December 2024 contract with Vertex Pharmaceuticals and Bluebird Bio and continued by the Trump administration, lets CMS negotiate on behalf of states and claw back undisclosed "discounts and rebates" when patients do not achieve agreed‑upon outcomes. Thirty‑three states, Washington, D.C., and Puerto Rico have opted in so far, and the NPR/KFF piece follows 18‑year‑old Medicaid enrollee Serenity Cole of St. Louis, who completed months‑long gene‑therapy treatment in May and has since avoided the near‑constant sickle‑cell pain and frequent hospitalizations that previously upended her life. The therapies, list‑priced at around $2.2 million and $3.1 million per patient before hospitalization costs, offer a potential cure for many of the roughly 100,000 mostly Black Americans with sickle cell disease but pose a massive budget challenge for Medicaid, which covers about half that population. CMS and drugmakers have refused to disclose the specific repayment and outcome terms, drawing transparency criticism even as state Medicaid directors cautiously welcome a federal mechanism to blunt the fiscal shock of these one‑time "curative" treatments.
📌 Key Facts
- CMS has negotiated a national outcomes‑based contract with Vertex Pharmaceuticals and Bluebird Bio for sickle cell gene therapies priced at about $2.2 million and $3.1 million per patient, excluding hospital costs.
- The Medicaid model requires drugmakers to provide "discounts and rebates" if patients’ outcomes fall short, a departure from traditional drug payment where plans pay regardless of benefit.
- As of July 2025, 33 states, Washington, D.C., and Puerto Rico had joined the voluntary model, which covers roughly half of the 100,000 Americans with sickle cell disease who are on Medicaid.
- An 18‑year‑old St. Louis Medicaid enrollee, Serenity Cole, was among the first treated under the program and reports being free of her prior daily sickle‑cell pain and holiday‑season hospitalizations.
📊 Relevant Data
Sickle cell disease affects approximately 100,000 people in the United States, with more than 90% being non-Hispanic Black or African American and an estimated 3%–9% being Hispanic or Latino.
Data and Statistics on Sickle Cell Disease — Centers for Disease Control and Prevention (CDC)
The birth prevalence of sickle cell disease is about 1 in 365 Black newborns and 1 in 16,300 Hispanic newborns in the US, compared to the overall US population where Black individuals make up about 13.6% and Hispanics 19% of the total population.
Birth Prevalence of Sickle Cell Disease and County-Level Social Vulnerability — 11 States, 2016–2020 — Centers for Disease Control and Prevention (CDC) Morbidity and Mortality Weekly Report
The estimated life expectancy for individuals with sickle cell disease in the US is more than 20 years shorter than the average, with average age at SCD-related death around 40 years, compared to 75 years for the general Black population.
Trends in Sickle Cell Disease–Related Mortality in the United States, 1979 to 2017 — Annals of Emergency Medicine
Sickle cell disease is caused by a genetic mutation in the hemoglobin gene, inherited in an autosomal recessive manner, and is more prevalent in populations with ancestry from malaria-endemic regions due to the sickle cell trait providing partial protection against malaria.
Sickle cell disease can be deadly, and the persistent health inequities facing Black Americans worsen the problem — The Conversation
There are fewer comprehensive treatment centers for sickle cell disease compared to similar conditions like cystic fibrosis, which affects fewer people but receives 7 to 11 times more research funding per patient, contributing to disparities in access and outcomes for SCD patients, who are predominantly Black and low-income.
When Actions Speak Louder Than Words — Racism and Sickle Cell Disease — New England Journal of Medicine
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