For the approximately 100,000 Americans living with sickle cell disease — and for the Medicaid programs that cover between 50 and 60 percent of them — the era of gene therapy coverage has arrived. But the coverage comes with an innovative condition: the manufacturer only gets paid in full if the therapy actually works.
The Centers for Medicare & Medicaid Services announced that 33 states, plus the District of Columbia and Puerto Rico, will participate in the Cell and Gene Therapy (CGT) Access Model, a bold new approach to delivering cutting-edge treatments for people on Medicaid living with sickle cell disease. Participating states represent approximately 84% of Medicaid beneficiaries with the condition, significantly expanding access to transformative care.
The two covered therapies — Casgevy (exagamglogene autotemcel, $2.2 million per patient) and Lyfgenia (lovotibeglogene autotemcel, $3.1 million per patient) — represent the first FDA-approved CRISPR-based and gene therapy treatments for sickle cell disease, both approved in December 2023. Without the CMS model, their prices alone would make them effectively inaccessible through state Medicaid programs that cannot absorb costs at this scale without a systematic coverage mechanism.
Why This Matters
Sickle cell disease is a genetic blood disorder caused by a mutation in hemoglobin that causes red blood cells to become rigid and crescent-shaped, blocking blood vessels, causing chronic pain crises, organ damage, stroke risk, and death at a median age of 40 to 60 without proper care. It disproportionately affects Black Americans.
Between 50% and 60% of people living with sickle cell disease have Medicaid coverage, and the high price of these treatments may strain state Medicaid agencies' budgets. The CGT Access Model helps make it easier for states to provide access to these therapies.
The outcomes-based agreement structure addresses the fundamental challenge of extremely high-cost gene therapies for publicly funded programs: paying $2.2 million for a treatment that fails to produce expected results is fiscally unsustainable and ethically problematic. Under CMS's model, manufacturers offer rebates if outcomes are not achieved — sharing the financial risk rather than placing it entirely on state Medicaid budgets.
What We Know So Far
From CMS.gov and CMS Innovation Center:
- "This model has the potential to improve health outcomes for patients with sickle cell disease while also ensuring state and taxpayer dollars are being used more effectively," said Abe Sutton, Director of the Innovation Center
- The model is voluntary for states and manufacturers; states began participation between January 2025 and January 2026
- The two covered therapies: Casgevy (exagamglogene autotemcel) and Lyfgenia (lovotibeglogene autotemcel)
- Coverage includes pre-treatment, the gene therapy infusion, and follow-up care for up to one year post-treatment
- Patients must be treated at CMS-designated treatment centers enrolled in the CIBMTR registry
- 33 states + DC + PR are participating — covering 84% of Medicaid beneficiaries with sickle cell disease
- Optional federal support of up to $9.55 million per state is available to help with implementation, outreach, and data tracking
The outcomes-based agreement structure means: if the therapy does not produce the specified clinical outcomes for a given patient, the manufacturer pays back a defined portion of the cost to the state Medicaid program. The specific rebate terms are negotiated between CMS and each manufacturer.
Where the Impact Is Greatest
The states with the largest Medicaid-covered sickle cell populations — and therefore the greatest potential impact from the CGT Access Model — include Georgia, Alabama, Mississippi, and Louisiana (Deep South states with high SCD prevalence among Black communities), as well as New York, Illinois, California, Texas, and Florida.
Daniel Cressy, 23, of Metairie, Louisiana — the first person in the Gulf South to receive Casgevy — received his treatment covered through Louisiana Medicaid after a two-year approval process. The CGT Access Model aims to streamline that access timeline for future patients.
What Doctors and Experts Say
Sickle cell disease specialists have noted that the outcomes-based model represents a significant philosophical shift in how the healthcare system values high-cost, potentially curative therapies. Traditional fee-for-service payment treats a therapy as a product purchased at a fixed price regardless of outcomes. Outcomes-based agreements treat it more like a service with a performance guarantee, aligning the financial incentives of the manufacturer with the health outcomes of the patient.
What the Evidence Shows — and What It Does Not
MedicalDaily Evidence Check
- Program: CMS Cell and Gene Therapy Access Model
- Coverage: Casgevy and Lyfgenia for sickle cell disease through state Medicaid programs
- Participating states: 33 + DC + PR (84% of Medicaid SCD beneficiaries)
- Payment model: Outcomes-based agreements — manufacturers rebate a portion of cost if outcomes are not achieved
- Drug price: Casgevy $2.2M; Lyfgenia $3.1M per patient
- What it shows: A scalable mechanism for covering gene therapy through Medicaid using shared financial risk
- Key limitation: Treatment must be administered at CMS-designated centers, which may be geographically inaccessible for some patients
Who Is Most Affected?
- Medicaid beneficiaries with sickle cell disease — the primary group historically unable to access these therapies due to cost
- Patients with severe, recurrent vaso-occlusive crises — the primary target population for both approved therapies
- Children and young adults, for whom earlier gene therapy could prevent decades of cumulative organ damage
Symptoms and Warning Signs to Watch For
Sickle cell disease patients currently enrolled in Medicaid should watch for:
- Signs that their current disease control is inadequate — increasing frequency or severity of pain crises
- Stroke symptoms: sudden weakness or numbness on one side, difficulty speaking, or severe headache
- Signs of acute chest syndrome: chest pain, cough, fever, or difficulty breathing — a medical emergency requiring hospitalization
What You Can Do Now
- If you or a family member has sickle cell disease and is covered by Medicaid, ask your hematologist whether gene therapy through the CGT Access Model is appropriate for your specific situation.
- Ask whether your state is participating in the CGT Access Model — check CMS.gov/priorities/innovation/innovation-models/cgt for the current participant list.
- Identify your nearest CMS-designated treatment center through the CIBMTR website — treatment must occur at a designated facility to qualify for coverage.
- Connect with the Sickle Cell Disease Association of America at sicklecelldisease.org for current coverage guidance, patient navigation, and clinical trial information.
Cost and Access: What Patients Should Know
Under the CGT Access Model, the gene therapy cost is covered by Medicaid through CMS-negotiated outcomes-based agreements — meaning the $2.2 to $3.1 million cost is not borne by the patient or the state alone. CMS provides federal support to participating states of up to $9.55 million per state for implementation, outreach, and data tracking.
What Happens Next
CMS is collecting outcome data from all treated patients through the CIBMTR registry. This real-world data will determine whether the program is expanded, modified, or potentially extended to other gene therapy indications. The program's effectiveness in increasing access will be evaluated in formal reports expected beginning in 2027.
The Bottom Line
Medicaid will now cover the two FDA-approved sickle cell gene therapies through outcomes-based agreements in 33 states and D.C. — meaning the manufacturer takes on financial risk if the therapy fails to produce expected results for a given patient. For the 50 to 60% of sickle cell disease patients covered by Medicaid, this is a historic expansion of access to potentially curative treatment. The pathway requires working with a hematologist, identifying a designated treatment center, and navigating the prior authorization process — but for the first time, the pathway exists.
