Key Takeaways:
- On January 30, 2024, the Center for Medicare and Medicaid Innovation (CMMI) issued additional details regarding the forthcoming Cell and Gene Therapy (CGT) Access Model, which was initially announced in February 2023.
- The model will test whether a CMS-led approach to negotiating and administering outcomes-based arrangements (OBAs) for CGTs will improve access and health outcomes for Medicaid beneficiaries and reduce health care costs.
- While participation in the model remains voluntary for both states and manufacturers, CMS will act as the negotiator of outcomes-based arrangements (OBAs) on behalf of participating manufacturers and states.
- The model will initially focus on cell or gene therapies for sickle cell disease (SCD) and will include a comprehensive strategy for addressing a range of barriers to equitable access to CGTs.
Model Overview
CMS as Negotiator
Under the model, CMS will negotiate OBAs with participating manufacturers, which will tie pricing for SCD treatments to whether the therapy improves health outcomes for people with Medicaid. Specifically, CMS will negotiate supplemental rebates and standard access policies for the SCD treatments of participating manufacturers, which will then be offered to participating states with “some limited state-to-state variation . . . as may be necessary to comport with state legislative and regulatory requirements.” CMS will then reconcile data, monitor results, and evaluate outcomes with respect to the negotiated OBAs.
Participation Voluntary
CMS confirms that the model will be a voluntary model for both manufacturers and states. However, it appears that participation is “all-or-nothing” for both participant types. That is, participating manufacturers must offer their OBA to all participating states, and participating states must sign an OBA with all participating manufacturers. That said, states will have the opportunity to review the key terms of the OBA between CMS and the manufacturer before deciding to opt in to the model.
Access Protections
Participating states will be required to implement operational and system requirements to support the model, including “changing reimbursement structures to appropriately reimburse for gene therapy; aligning coverage policies with Medicaid managed care organizations, as applicable; and contracting with out-of-state providers as may be necessary.” CMS will provide funding support to states through a cooperative agreement to help states cover the costs of participation in the model, including the state obligations related to provider requirements, data collection, and activities to ensure equitable access to care.
Eligibility Criteria
Manufacturers are eligible if they have an FDA-approved cell or gene therapy for SCD launched by May 2024. All states and U.S. territories that participate in the MDRP are eligible to apply to participate in the model.
Timing
While two gene therapies for SCD were approved in late 2023, the CGT Access Model will not begin until January 2025, and states may choose to begin participation at a time of their choosing between January 2025 and January 2026.
CMS anticipates releasing the request for applications (RFA) to manufacturers in early spring 2024, and a separate RFA and Notice of Funding Opportunity (NOFO) to states in summer of 2024. CMS also released a State Letter of Intent (LOI), for states to express their intent to participate in the model by April 2024. While states are not required to submit the LOI to participate in the model, states would be required to respond to the RFA by February 2025.
Additional Disease States Possible
While the model will initially focus only on SCD, CMS notes that it “might include additional cell and gene therapies for other diseases in the model at a future time” and that “if additional disease conditions are added to the model in future years, states will be permitted to apply for model participation pertaining to that condition.”
Additional Information:
CMS will be hosting webinars on the CGT Access Model on February
6, 2024 (general overview) and February
8, 2024 (overview for states).
