Washington, DC – The Rare Disease Company Coalition (RDCC) has submitted a response to the 21st Century Cures and Cures 2.0 Request for Information, offering recommendations for policy improvements to facilitate the discovery, development, and delivery of treatments for people living with a rare disease.

RDCC applauds the longstanding leadership of Rep. DeGette and Rep. Bucshon in developing and advancing policy solutions on behalf of the 1 in 10 Americans living with a rare disease. 21st Century Cures and Cures 2.0 yielded notable progress in advancing the development and availability of treatments for rare diseases, but there is a critical need for further action to address the unmet needs associated with rare disease. In particular, the RDCC recommends that the 21st Century Cures initiative ensures:

By supporting these initiatives, Congress can help create an environment where effective treatments for rare diseases are developed and delivered without delay. We look forward to working with policymakers to advance legislation to accomplish these goals.

Read the full letter here.

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.