Stephen Majors, Senior Director for Public Affairs
Cell and Gene Therapy Access Model
ARM applauds the HHS announcement that the Center for Medicare and Medicaid Innovation (CMMI) will test a “Cell and Gene Therapy Access Model” to administer multi-state outcomes-based agreements in Medicaid programs for transformative cell and gene therapies.
ARM has long been a proponent of modernizing payment systems to recognize the unique, durable, and potentially curative nature of these medicines. If designed properly, outcomes-based agreements and other value-based arrangements can improve access to the coming wave of cell and gene therapies for beneficiaries of state Medicaid programs.
ARM forecasts that the FDA could approve at least five new gene therapies to treat rare diseases in 2023, while the FDA has predicted it will approve 10 to 20 cell and gene therapies a year starting in 2025. The Biden Administration’s Cell and Gene Therapy Access Model could be a significant step toward ensuring that the roughly 90 million people covered by Medicaid will have access to the most cutting-edge medicines that science has to offer.
ARM and its 400+ member organizations look forward to working with CMS and Congress on the details of the model and continuing our dialogue to facilitate patient access to cell and gene therapies.
Model Addressing Accelerated Approval
HHS also directed CMMI to test a model that would modify payments for drugs approved under the Accelerated Approval Program (AAP) to encourage developers to complete timely confirmatory trials. ARM believes this approach will undermine the intent of AAP and could discourage developers from investing in potential cures for diseases with high unmet medical need.
Established more than 30 years ago, AAP is a vital pathway for getting promising therapies to patients who have few, or no, other treatment options and could be a lifeline for patients seeking access to cell and gene therapies in the future. ARM believes in the timely initiation, conduct, and completion of confirmatory clinical trials, and supported the changes to AAP to achieve that goal as part of the Omnibus Appropriations bill signed into law in December.
Medicare Bundled Payments for Cell and Gene Therapy
HHS Secretary Becerra has directed CMMI to consider the use of bundled payments as an alternative to traditional fee-for-service for cell and gene therapies in Medicare, to complement the Medicaid-focused Cell and Gene Therapy Access Model. To the extent CMMI moves forward with this model, ARM urges the agency to recognize and account for the unique, personalized nature of cell and gene therapies so that any bundled payments would not undermine access to these therapies.