CMS announces plans to broaden Cell and Gene Therapy Access Model beyond sickle cell disease

CMS intends to expand its Cell and Gene Therapy Access Model following an initial evaluation of centralized manufacturer negotiations. The findings support the contracting approach, while highlighting administrative challenges and the importance of aligning outcomes measurement with evidence needs across reimbursement, regulatory decision-making, and health technology assessment.

The Baseline

• CMS has unveiled expansion of the CGT Access Model beyond sickle cell disease, inviting additional manufacturers to participate.
• The first evaluation found that centralized negotiations strengthened bargaining leverage and secured participation covering approximately 84% of Medicaid beneficiaries with SCD.
• Findings highlighted the value of shared administrative support and identified opportunities to streamline negotiations and state onboarding.

The US Centers for Medicare & Medicaid Services (CMS) has announced plans to broaden its voluntary Cell and Gene Therapy (CGT) Access Model beyond sickle cell disease (SCD), following positive findings from its first evaluation. The model allows CMS to negotiate outcomes-based agreements (OBAs) directly with manufacturers on behalf of participating state Medicaid agencies.

Through the CMS Innovation Center, the initiative will invite manufacturers of additional cell and gene therapies to apply through a forthcoming request for applications. CMS intends to enable negotiations  to begin sooner following FDA approval, while also making previously approved therapies eligible.

Launched in 2025, the model initially focuses on Casgevy (Vertex Pharmaceuticals/CRISPR Therapeutics) and Lyfgenia (bluebird bio, Inc.) for SCD. The agreements link financial terms, including supplemental rebates, to patient outcomes and establish common access policies and coverage criteria. By coordinating negotiations and sharing administrative responsibilities, CMS aims to improve treatment access and reduce the financial and administrative burden on state Medicaid programs.

The first evaluation report examined whether CMS-led negotiations were an efficient and effective alternative to separate state agreements. Drawing on documentation and interviews with CMS, manufacturers, and Medicaid agencies in 10 states, it assessed manufacturer negotiations and state participation decisions.

Following approximately nine months of discussions, CMS secured terms covering rebates, fertility preservation services, access policies, and coverage criteria. Agreements were subsequently established with 32 states, the District of Columbia, and Puerto Rico, which together account for approximately 84% of Medicaid beneficiaries with SCD.

The evaluation found that pooling beneficiaries strengthened CMS’ negotiating leverage and provided capacity and resources that individual state agencies might lack. Manufacturers welcomed the opportunity to reach a larger Medicaid population without negotiating separately with each state, whilst agencies valued CMS support for collecting outcomes data and managing disputes over rebate determinations.  They also valued assurances of timely reimbursement to qualified treatment centers. Both manufacturers and state agencies viewed the model’s provision for manufacturer-funded fertility preservation services as an incentive to participate.

However, manufacturers reported that negotiations were more protracted than anticipated, although they still considered the process more efficient than separate state negotiations. CMS noted that discussions concluded within the established timeframe.

Both manufacturers and agencies described the agreements as more detailed and administratively demanding than their typical value-based contracts. Agreeing on outcomes measurement requirements was particularly complex, while some states reported unexpectedly extensive documentation requirements.

The report identified clearer negotiating parameters, earlier communication of requirements, and additional state onboarding support as areas for improvement. Its findings concern agreement development and participation, rather than demonstrated changes in patient outcomes or healthcare spending.

The findings overlap with priorities outlined in a recent white paper published in the Journal of Comparative Effectiveness Research. Simmons and colleagues drew on insights from a multidisciplinary advisory council of 21 healthcare professionals involved in CGT delivery, alongside their own experience, to examine operational and financial barriers from the provider perspective.

The authors identified greater standardization of manufacturer onboarding and contracting, more predictable reimbursement, and closer coordination among manufacturers, providers, and payers as priorities. CMS’ coordinated negotiations and assurances of timely reimbursement address aspects of these concerns, although the evaluation also documented substantial administrative requirements.

“Addressing these challenges may help reduce administrative burden, mitigate financial risk, and support streamlined implementation of CGTs across both academic and community settings,” the authors stated.