HHS Secretary Announces CGT Access Model

The Centers for Medicare & Medicaid Services (CMS) announced on February 14 that Xavier Becerra, Secretary of the Department of Health and Human Services (HHS), had selected three new models for testing by the Center for Medicare and Medicaid Innovation (the Innovation Center). The Cell and Gene Therapy Access Model has the potential for a far-reaching impact on the CGT field.

The Innovation Center, which is a part of CMS and will test the models, was created to support the development of “various payment and service delivery models that aim to achieve better care for patients, better health for our communities, and lower costs through improvement for our health care system.” It is tasked with improving the quality of care in Medicare, Medicaid, and the Children's Health Insurance Program (CHIP), with an eye toward reducing costs. 

“Yesterday, CMS announced it has selected three new payment models for testing to help lower drug costs and improve quality of care for patients.  If implemented properly, the Cell and Gene Therapy Access Model could help Medicaid beneficiaries gain access to potentially life-changing therapies,” said ASGCT President Hans-Peter Kiem, MD, PhD.

Regarding the Cell and Gene Therapy Access Model, CMS said in their release: “Cell and Gene Therapies are an emerging area of new drug development that holds significant potential, but these therapies can cost upwards of $1 million. Under this model, state Medicaid agencies would assign CMS to coordinate and administer multi-state, outcomes-based agreements with manufacturers for certain cell and gene therapies.” 

Through the model, state Medicaid agencies would assign CMS to coordinate and administer multi-state outcomes-based agreements with manufacturers for certain cell and gene therapies. Rather than 50 different negotiations between states and manufacturers, this approach could give states the option to cede to CMS the authority to negotiate outcomes-based arrangements. Through this model, CMS would also be responsible for reconciling the financial and clinical outcomes of the outcome-based agreements. 

“ASGCT’s mission is to advance genetic and cellular therapies to alleviate human disease.  Therefore, the accessibility of therapies to patients is of paramount importance to the Society’s membership. While the development and science has progressed tremendously, leading to transformative therapies, the current health care ecosystem has not kept pace,” said ASGCT CEO David Barrett, JD.  

The Accelerating Clinical Evidence Model was also selected for demonstration by Secretary Becerra. Through this proposal, CMS, in consultation with the FDA, is considering developing payment methods for drugs approved through accelerated approval to “encourage manufacturers to complete confirmatory trials.” – suggesting differential payment rates for drugs approved through this pathway. Accelerated approval allows the Food and Drug Administration (FDA) to make treatments available earlier based on a surrogate endpoint if they address an unmet medical need for a serious condition, contingent on post-marketing trials that demonstrate the drug offers a clinical benefit and an improved patient outcome. Notably, the 2022 Omnibus included reforms to the accelerated approval pathway that requires greater reporting on post-marketing study progress as well as a refined withdraw pathway. ASGCT is concerned that this approach by the Innovation Center could harm the integrity of the accelerated approval pathway and slow access to the next generation of gene and cell therapies. 

Dr. Kiem said: “Many gene therapies have the potential to be approved through the accelerated pathway, as their outcomes may not be possible to assess within the duration of a traditional clinical trial. ASGCT believes post-market surveillance is necessary to ensure approved products remain safe and efficacious. However, efforts to ensure confirmatory trials should not have the unintended consequence of impeding the development of new therapies and patient access.”

The third model selected for demonstration was the Medicare $2 Drug List. Under this model, CMS will identify a list of generic drugs would be applicable for chronic conditions such as high blood pressure and high cholesterol where cost-sharing can vary widely across insurance plans. Part D plans participating in the model would agree to cap cost-sharing for these products, and CMS would highlight those plans to beneficiaries during open enrollment. Additional information on why these models were selected models can be found in the Secretary’s report.

The three payment models are in response to President Joe Biden’s Executive Order 14087, “Lowering Prescription Drug Costs for Americans,” issued on October 14, 2022. The payment models are intended to complement the goals of the Inflation Reduction Act (IRA). Biden’s order directed the Secretary Becerra to consider whether new payment models would reduce drug costs and promote access to innovative drug therapies for beneficiaries enrolled in the Medicare and Medicaid programs. This included exploring models that could lead to lower cost-sharing for commonly used drugs and could potentially support value-based payments. 

If considered successful by Secretary Becerra, the Cell and Gene Therapy Access Model may expand its duration and scope. ASGCT will continue to monitor these developments, and share information with the Society’s membership.

Margarita Valdez Martínez is ASGCT's director of policy & advocacy.

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