The Centers for Medicare and Medicaid Services (CMS) is initiating a Cell and Gene Therapy Access Model, offering renewed hope for patients with sickle cell disease seeking access to potentially transformative treatments. CMS Administrator Chiquita Brooks-LaSure announced that the manufacturers of the sickle cell disease gene therapies, Lyfgenia (lovotibeglogene autotemcel) and Casgevy (exagamglogene autotemcel), have agreed to participate in this innovative program.
Addressing Access Barriers
Despite the FDA's approval of Lyfgenia and Casgevy in December 2023, only a small number of patients have been able to access these therapies due to high costs, with Lyfgenia priced at $3.1 million and Casgevy at $2.2 million. By August 2024, only 20 patients had initiated Casgevy treatment and just four had started on Lyfgenia. The CMS Access Model seeks to overcome these barriers by allowing CMS to negotiate outcomes-based agreements on behalf of state Medicaid programs, beginning with sickle cell disease treatments.
Outcomes-Based Agreements
The voluntary model, led by the Center for Medicare and Medicaid Innovation, will test outcomes-based agreements for cell and gene therapies. These agreements will link payments to whether each therapy demonstrably improves health outcomes. Sickle cell disease is considered a reasonable test case due to the relative ease of monitoring and measuring outcomes. CMS Deputy Administrator Liz Fowler stated that the model will afford state Medicaid agencies more budget predictability, with risk-sharing through pay-for-performance mechanisms.
Impact on Sickle Cell Disease
Sickle cell disease affects millions worldwide, with approximately 100,000 individuals living with the condition in the United States, predominantly impacting people of sub-Saharan African descent. The disease alters hemoglobin structure, leading to severe pain, anemia, organ damage, and infections, reducing life expectancy by more than 20 years on average. While treatments like pain medications, antibiotics, and hydroxyurea exist, Lyfgenia and Casgevy offer the potential to significantly decrease or even eliminate pain crises. Given that Medicaid is a dominant payer (50-60%) in the sickle cell disease area, these arrangements between manufacturers and state Medicaid agencies are critical.
Future Expansion
The model is set to launch next month, with states having the option to participate between January 2025 and January 2026. The CMS anticipates that this model will increase patient access and improve health outcomes, particularly in areas of unmet need like sickle cell disease. CMS Administrator Chiquita Brooks-LaSure emphasized that this initiative represents a "new frontier" in providing access to potentially transformative treatments for individuals with sickle cell disease. Pending future administrations, this model could expand to include other cell and gene therapies, offering a blueprint for addressing access challenges across multiple disease areas.
