The RDCC sent a letter to Senator Cassidy and the Senate Health, Education, Labor, and Pensions (HELP) Committee in response to the recent request for information (RFI) on ways to improve and protect access to gene therapies for Americans with rare ultra-rare diseases. Coverage and reimbursement is a critical issue for all patients living with a rare disease and their families. As the RFI recognizes, if a patient cannot access innovative treatments, to them it is as if that innovation never occurred. 

RDCC members currently have over 200 programs in development, many of which are first-in-class gene and cell therapies. With the appropriate policy framework, these therapies could ultimately prove life changing or life saving to patients with no other treatments available.  

But current coverage and reimbursement policies have not kept pace with the rapidly-evolving science. We urge policymakers to consider solutions to ensure the ecosystem is better-equipped to accommodate tremendous leaps in innovation in the development of gene and cell therapies and ensure timely access for patients living with a rare disease. 

Interested in learning more about the RDCC’s recommendations? Click here.