The Rare Disease Company Coalition

We invite you to the RDCC’s Holiday Rare Reception on Wednesday, December 10. We look forward to an evening of conversation, community, and connection during the holiday season with rare disease stakeholders! RSVP here: https://lnkd.in/euz-EYwg

Today marks a major victory for the #raredisease community! The RDCC applauds the House of Representatives for passing the Mikaela Naylon Give Kids a Chance Act, a critical step to restoring the Rare Pediatric Disease Priority Review Voucher (PRV) program. The PRV program, which lapsed in December 2024, is a life-saving incentive that drives investment in treatments for children with rare diseases. The program has supported treatments across 47 rare pediatric disease indications and benefited more than 200,000 children. For many, these treatments represent hope in a brighter future. Children living with rare, devastating diseases deserve action. We thank the House for their leadership and urge the Senate to follow suit and pass S. 932 without delay. 

Happy #Thanksgiving! The RDCC is deeply grateful to our coalition members for their dedication and hard work in supporting the #raredisease community. It’s through our shared commitment that we can continue to advance a healthy, hopeful future for the 1 in 10 Americans living with a rare disease. We also extend our thanks to the broader community for their tireless work to drive progress for patients living with a rare disease. To our community and beyond, we wish you a restful Thanksgiving filled with joy, gratitude, and love.

The Priority Review Voucher (PRV) program offers hope for children and their families affected by rare diseases by driving innovation for life-changing therapies. Read this op-ed by Ultragenyx CEO Emil Kakkis on why reauthorizing the PRV program matters.

The rare disease innovator community is united in our support for the Rare Pediatric Disease Priority Review Voucher (PRV) Program. This week, the Rare Disease Company Coalition and the Alliance for Regenerative Medicine (ARM) led a joint letter to Congress urging the swift reauthorization of the PRV program. The letter is signed by over 60 biopharmaceutical CEOs at the forefront of rare disease innovation. To date, the PRV program has spurred the development of therapies across 47 indications and benefited more than 200,00 patients. Beyond serving as a crucial source of hope for children and their families, this program drives innovation, standing as a critical resource for companies developing treatments for rare disease patients. Together with ARM and dozens of leaders from across the rare disease ecosystem, we’re eager to work with Congress to reauthorize this vital program and ensure continued hope for children living with rare diseases. Read our letter: https://lnkd.in/ebdXtczT #RareDisease #OneRareVoice

Arya Singh was born with spinal muscular atrophy, a rare genetic disorder. She shared her story at an RDCC briefing, offering a powerful reminder that hope becomes reality only when patients like Arya can access timely diagnoses and innovative treatments. That means supporting continued investment in rare disease R&D. Together, we can spur innovation and expand treatment options for those who need them most.

Thank you FDA Commissioner Makary for reiterating the Administration's support for rare disease innovation, and specifically, for the reauthorization of the rare pediatric disease priority review voucher (PRV) program. This program is a vital incentive that spurs innovation for children living with a rare disease. To date, the PRV program has led to significant advancements for the rare disease community, and your support sends a clear message to rare disease patients and their families that there is hope for a healthier future. We look forward to continuing our work to ensure the program is reauthorized.

What an incredible experience at the National Organization for Rare Disorders Breakthrough Summit! The RDCC was proud to sponsor this event, which brought together over 900 patient leaders, researchers, healthcare advocates, and industry partners in the #raredisease community. Thank you to everyone who stopped by. It was inspiring to connect with you all and have such great conversations around innovation and investment. We're energized to keep up the momentum and deliver hope to patients living with a rare disease. #NORDSummit

When #raredisease therapies receive accelerated approval, the post-approval phase often asks patients to do the impossible: enroll in placebo-controlled trials when an approved treatment already exists. At a recent workshop with the Duke-Margolis Institute for Health Policy Institute and the U.S. Food and Drug Administration, the RDCC’s Executive Director Stacey Frisk discussed the need to rethink how we collect post-approval evidence for rare diseases. Watch her remarks:

As the RDCC grows, so does our impact. We’re excited to welcome Glaukos Corporation, Jazz Pharmaceuticals, Mirum Pharmaceuticals, Inc., and Neurizon as the newest members of the Rare Disease Company Coalition. Each organization is dedicated to advancing treatments for people living with rare diseases, whether through driving innovation, transforming clinical research into therapies, or addressing urgent unmet medical needs. Together we are #OneRareVoice, working to advance policies that accelerate the development of #raredisease therapies and ensure every patient has access to life-changing care.