The cell and gene therapy (CGT) landscape is rapidly evolving across regulatory, reimbursement, and access dimensions, presenting both opportunities and challenges for manufacturers, payers, and providers. From shifting Medicaid reimbursement methodologies and the expansion of chimeric antigen receptor T-cell (CAR-T) administration into community-based outpatient settings, to proposed CMS Innovation Center models and increasing US Food and Drug Administration (FDA) engagement in evidence generation, stakeholders must navigate a complex and dynamic policy environment. As innovative contracting arrangements continue to mature and new federal and state initiatives emerge, understanding the interplay between coverage, reimbursement, and access will be critical to ensuring patients can benefit from these transformative therapies.  

Medicaid Reimbursement 

Medicaid reimbursement practices for CGTs vary by state within the parameters of federal guidelines. Historically, states have utilized bundled payment methodologies in the inpatient setting to reimburse for all services provided during an encounter, which often presented risks of under-payments to providers administering the products. Recent policy changes have enabled states to collect rebates on drugs provided in the inpatient setting, so long as the drug is separately identifiable.  

Increasingly, states are opting to reimburse CGTs outside of bundled payment methodologies, allowing them to capitalize on rebates and providing greater assurance of reimbursement for providers. However, this change has increased manufacturer exposure to Medicaid Drug Rebate Program rebates for therapies typically administered in the inpatient setting.  

Community Expansion 

 As the removal of Risk Evaluation and Mitigation Strategies (REMS) requirements for CAR-T therapies continue to shape where and how these products can be delivered, there is growing interest in expanding CAR-T administration into outpatient settings, including by enabling community-based providers to offer treatment. Reflecting this shift, the Foundation for the Accreditation of Cellular Therapy developed new community standards that allow for accreditation of community-based practices. Additionally, many community sites have built clinical experience monitoring and managing risk through the administration of bispecific therapies, which can present adverse events similar to CAR-T. However, key questions remain unresolved, particularly around financing and reimbursement for these high-cost products and whether payers will recognize community sites as in-network providers for CAR-T administration. 

CMS Innovation Center Demonstrations 

 The proposed drug pricing negotiation  Global Benchmark for Efficient Drug Pricing (GLOBE)  and Guarding U.S. Medicare Against Rising Drug Costs (GUARD) Models could have varying impacts on CGT access and reimbursement. These models represent a heightened role for CMS in negotiating drug pricing and access, and will ultimately impact payer reimbursement. If CGTs are included in the models, developers may need to revisit launch planning in ex-US markets and assess liability risk under any model that advances.     

FDA Regulatory Stance 

Recent FDA guidance and public statements signal the agency’s increasing flexibility around how sponsors can generate evidence and engage with regulators, particularly in emerging areas like CGT. However, recent approval outcomes for CGTs in development suggest those signals do not eliminate meaningful regulatory risk, even in cases where developers claim the FDA had previously signaled alignment with their study design. Taken together, this dynamic underscores that while the FDA may be providing additional pathways and clarity, sponsors still need rigorous, regulator-aligned trial designs and robust evidence packages to avoid late-stage interruptions that can derail or delay approvals.  

Innovative Contracting 

In recent years, both manufacturers and payers have developed innovative payment methods the commercial and Medicaid markets. While innovative contracting arrangements have historically been driven by engagement between manufacturers and payers, the Cell and Gene Therapy Access Model has emerged as an opportunity for CMS to be an intermediary between states and manufacturers, and could serve as a model for future expansion if successful.   

Dive Deeper 

Avalere Health convenes a diverse set of policy and regulatory experts focused on CGT across all stages of the product life cycle, carefully monitoring trends in coverage, reimbursement, and access. Please contact us if you would like to connect with our CGT experts.