Medicaid has initiated a new payment model for cell and gene therapies targeting sickle cell disease, linking payments to treatment effectiveness. This approach allows the federal government to negotiate costs with pharmaceutical companies on behalf of state Medicaid programs, with provisions for discounts and rebates if treatments do not perform as expected. This differs from traditional payment structures where costs are typically paid regardless of patient outcomes. The specific terms of these agreements, however, remain confidential.
Context of Sickle Cell Treatment
Sickle cell disease is a genetic condition affecting approximately 100,000 primarily Black Americans, which is estimated to shorten lifespans by over two decades. Two gene therapies approved by the Food and Drug Administration (FDA) in December 2023 offer a potential cure for individuals aged 12 and older. These therapies are priced at $2.2 million and $3.1 million per patient, excluding hospital stay costs. Medicaid, which covers about half of Americans with sickle cell disease, faces substantial financial challenges with these high-cost treatments. Government estimates indicate that sickle cell care already costs the health system nearly $3 billion annually.
Program Implementation
The Centers for Medicare & Medicaid Services (CMS) established this model, reaching an agreement with manufacturers Vertex Pharmaceuticals and Bluebird Bio. As of July, 33 states, Washington, D.C., and Puerto Rico have voluntarily joined the initiative. The program aims to increase patient access to these therapies without standard state-imposed restrictions, such as lengthy prior authorization processes or requirements for prior treatments. Additionally, manufacturers will cover fertility preservation costs, which Medicaid typically does not.
Evaluation and Future Impact
Clinical trials for these gene therapies involved fewer than 100 patients and observed them for two years, prompting some state Medicaid officials to seek assurances regarding long-term benefits. An independent research institute described the model as a "worthy experiment" for managing high treatment costs and uncertain long-term outcomes, especially with many more expensive gene therapies anticipated in clinical trials. A federally funded evaluation is expected to track patient numbers and results, enabling states to seek rebates if treatments are ineffective. The program is projected to operate for up to 11 years.
Patient Experience
Serenity Cole, an 18-year-old Medicaid enrollee, completed a gene therapy treatment in May. Prior to the therapy, Cole experienced debilitating pain and frequent hospitalizations due to sickle cell disease. She reported a significant improvement in her condition, with no pain episodes requiring hospitalization since completing the treatment.