#ASGCT23 Session Explores Medicaid Strategies to Improve CGT Access

The field of cell and gene therapy (CGT) is a rapidly evolving field of medicine that offers tremendous promise for treating and potentially curing a wide range of diseases. These therapies are often complex and expensive, requiring significant investment in research, development, and manufacturing. While these therapies have the potential to revolutionize health care, ensuring patient access to them remains a challenge. 

Medicaid is a critical source of health care coverage for millions of Americans, particularly those with low incomes, disabilities, and chronic health conditions. Medicaid also is a primary payer for pediatric care as roughly 40% of children are covered by the program. As a result, Medicaid plays a critical role in ensuring patient access to advanced therapies, including CGT. As these therapies become more prevalent, it is important to consider how Medicaid can best support patient access to these life-changing treatments.  

To address the challenges of CGT’s high upfront cost, Medicaid programs are exploring a range of strategies to improve patient access to these therapies. In addition to efforts to negotiate lower prices for these treatments, Medicaid programs may also explore value-based payment models, which tie payment to patient outcomes. This approach can help states recoup some of the costs of therapies that do not provide a long-lasting benefit. At ASGCT’s 26th Annual Meeting, presenters painted a picture of the current Medicaid landscape for CGT, identified barriers to access, and discussed potential solutions from a variety of stakeholder perspectives. 

Rayne Rouce, MD, from Texas Children’s Hospital and Baylor College of Medicine, and member of ASGCT’s Board of Directors and D&I and Comunications committees, shared her perspective as a physician scientist and provider of CAR T therapies, detailing current challenges facing patients seeking access to these therapies and challenges related to coverage and reimbursement under the public payor system. Coverage delays, the individualized manufacturing process, and logistical requirements like travel and lodging all increase patients’ time to treatment. CAR T therapies can be transformative for patients with no other means of disease management, but practical barriers can delay treatment to a point where the therapy can no longer be tolerated as the disease has advanced.  Dr. Rouce also highlighted the potential impact of delayed approval on clinical outcomes and disproportionate effects on special populations including underrepresented minorities and under-resourced patients.   

Diane Berry, PhD, chair of ASGCT’s Government Relations Committee, underscored existing access challenges to CGT under Medicaid using data from a new article titled Medicaid coverage practices for approved gene and cell therapies: Existing barriers and proposed policy solutions, which highlighted inconsistent Medicaid coverage across state lines. She reported data show that where a patient lives can impact coverage and reimbursement decisions, with some states opting to cover based on clinical trial criteria rather than the FDA approved indication. Dr. Berry explained that this practice was not in line with federal requirements for states to cover products to their fully approved FDA-labeled indication, and discussed several federal mechanisms to promote compliance.  

While the budgetary limitations of state Medicaid programs are very real, and these products carry a high upfront cost, speakers discussed possible ways to mitigate this strain including  new rules facilitating the use of value-based payment (VBP) or outcomes-based arrangements (OBA). Denise Pierce, CEO of DK Pierce Consulting, laid the groundwork and explained consistent challenges and opportunities she has seen across states. She outlined hurdles to adoption of these programs such as staffing and tracking success metrics. Ross Hoffman, MD, spoke from the perspective of a large Managed Care Organization (MCO). MCOs contract with states to administer benefits, and may provide services in multiple states. As such, Dr. Hoffman noted his company faces similar challenges  but with the added complication of multi-market administration. Ashley Hume, senior vice president of strategic growth and client engagement, Emerging Therapy Solutions, then provided background on re-insurance for state Medicaid programs and commercial insurers. She explained how re-insurance can mitigate risk at the state level, and how to use historical data appropriately to maximize coverage. Ms. Hume also offered several solutions around improving financing options for GCTs – risk transfer and pooling, accessing refunds/rebates available through value-based contracts, and state and federal pooling. She then tied this back to the topic of value-based payments and discussed several solutions leveraging re-insurance.  

During the panel Q&A session, the presenters engaged with the audience, emphasizing the need for a proactive approach to CGT coverage and reimbursement. They acknowledged the growing attention given to this issue by the Biden administration, as evidenced by recently announced demonstration models from the Center for Medicare and Medicaid Innovation. Efforts to standardize innovative payment approaches were also discussed as a means to improve market predictability and promote broader access to CGT. Overall, the presenters recognized the challenges faced by payors and stressed the importance of innovative models to overcome obstacles associated with VBP/OBA approaches. 

As the session concluded, it was clear Medicaid coverage of CGT is critical for ensuring patient access to these life-changing treatments. While these therapies can be expensive and complex, Medicaid programs and various stakeholders are exploring a range of strategies to improve patient access, including negotiating directly with manufacturers, developing value-based payment models, and partnering with other payers and academic medical centers. By working together, these stakeholders can develop solutions that ensure all patients have access to the latest advances in CGT. 

Francesca Cook is past chair of ASGCT's Government Relations Committee and Vice President, Pricing and Market Access at REGENXBIO. 

Rayne Rouce is an ASGCT Board member; member of the Communications and Diversity, Equity, & Inclusion committees, and an associate professor at Baylor College of Medicine. 

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