Washington, DC – Rare Disease Company Coalition (RDCC) Executive Director Stacey Frisk released the following statement on the continuing resolution to fund the federal government, which includes a five-year extension of the life-changing rare pediatric disease priority review voucher (PRV) program.
The RDCC strongly urges Congress to advance the continuing resolution to support key programs like the rare pediatric disease priority review voucher (PRV).
The rare pediatric disease PRV program has already spurred extraordinary progress for pediatric rare disease treatments and supported groundbreaking innovation for patients. The RDCC’s latest research found that over the past decade, the program has led to new innovations that benefit over 200,000 rare disease patients and addressed unmet medical needs across 47 rare pediatric indications. Over 90 percent of all rare pediatric disease PRVs were awarded to therapies for indications with no approved therapy on the market.
Now, thanks to our champions on Capitol Hill, Congress has the opportunity to ensure innovative companies like our members can continue to discover, develop, and deliver treatments for children, while reinvesting critical dollars into additional R&D for rare disease treatments. Through the continuing resolution, Congress can bring stability to innovators, encourage further investment and create the conditions for groundbreaking innovations in rare pediatric disease drug development.
Read our letter to Congress here.
Washington, DC – With so much at stake for the 1 in 10 Americans living with a rare disease, the FDA’s Rare Disease Innovation Hub could spur the development of new innovations for the 95 percent of rare diseases with no FDA-approved treatment. It has incredible potential to increase collaboration with patients, leverage cross-agency expertise and advance regulatory science on behalf of individuals living with rare diseases; prioritizing the right activities will ensure that happens.
The comment letter below presents our feedback on the Hub’s priorities. We are confident that with these recommendations in mind, the FDA’s new model for rare disease collaboration can fulfill its mission and promise.
We look forward to continuing to work with FDA to support the Hub and our shared commitment to improving the lives of Americans living with rare diseases.
Read the full letter here.
Washington, DC – On September 12, the Rare Disease Company Coalition sent a letter to Congressional leadership urging them to support the swift passage of S.4583/H.R. 7384, the Creating Hope Reauthorization Act of 2024.
The bill would reauthorize the rare pediatric disease priority review voucher (PRV) program, a proven policy that has benefitted hundreds of thousands of patients living with a rare disease. Over the past 12 years, the program has been an innovation-driving and cost-effective policy that spurs research & development (R&D) in rare pediatric diseases.
If Congress allows it to expire on September 30, this critical program will be out of reach for many rare disease treatments.
Read the full letter below.
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.