The National Organization for Rare Disorders (NORD) released a new report showing the undeniable effectiveness of the Rare Pediatric Disease Priority Review Voucher (RPD PRV) program, designed to incentivize the development of therapies for hard-to-study pediatric rare diseases. The RPD PRV has spurred the development of more than 50 safe and effective treatment options for rare pediatric diseases in the first 12 years of the program.
Patient Organization Leaders are Speaking Out:
Check out an op-ed from NORD’s President and CEO, Pam Gavin: The rare pediatric disease voucher program creates new treatments. I have new data to prove it.
Watch this four-minute video by Dr. Leslie Gordon, Co-Founder and Medical Director of the Progeria Research Foundation, a NORD Member organization, explaining the importance of the RPD PRV program for progeria research and other rare pediatric diseases.
Watch this three-minute video by Dr. Annette Bakker, CEO of the Children’s Tumor Foundation, also a NORD Member organization, explain the impact of the RPD PRV program for childhood cancer research, specifically neurofibromatosis.
Watch this 30-second video by Gina Glass, mom to a child with sickle cell disease and Executive Director of the Dreamsickle Kids Foundation, also a NORD Member organization, where she explains the need to reauthorize the RPD PRV program.
Watch our Community Webinar: Why Saving the Rare Pediatric Disease Priority Review Voucher Program Matters
This webinar was held Wednesday, July 24, via Zoom, for all stakeholders in our rare disease community. Watch the webinar here.
Nearly 200 Patient Organizations Sign Letter to Congress Supporting Reauthorizing the Rare Pediatric Disease Priority Review Voucher Program
Legislation has been introduced in the House and Senate to reauthorize this important program that has helped spur a safe and effective treatment option for almost 40 rare disease patient communities. To show the rare disease patient community’s strong support for the program, NORD led a patient organization sign-on letter to House and Senate leaders, urging their swift reauthorization of the program for at least five years through H.R. 7384 / S. 4583, the Creating Hope Reauthorization Act. Nearly 200 patient organizations signed on to the letter, which you can view here.
Make Your Voice Heard
Contact your Representative TODAY and urge them to cosponsor the Creating Hope Reauthorization Act! Take action here.
As many as half of all people living with a rare disease are children, and the Rare Pediatric Disease Priority Review Voucher (RPD PRV) program offer a crucial incentive for companies to develop therapies for these particularly challenging patient populations. Reauthorizing this vital program before the September 30, 2024 deadline would maintain an important tool in ongoing efforts to address the significant unmet treatment needs of the rare pediatric disease community.
If you would like to get involved or share your story of how the pediatric PRV program helped you or someone you love, please email [email protected].
Background on Rare Pediatric Disease Priority Review Vouchers
The RPD PRV has helped spur rare disease drug development in pediatric populations and brought over 50 therapies to market with critical safety and dosing data specific to children.
The PRV program began in 2007 to address the unmet need of developing drugs for tropical diseases and was expanded in 2012 to include rare pediatric diseases. Under this program, companies that develop novel therapies for rare pediatric diseases can be awarded a PRV, which allows a sponsor to obtain priority review for a New Drug Application (NDA) or Biologic License Application (BLA) that would otherwise not qualify for priority review; it can also be sold or transferred to another manufacturer to obtain a priority review for their product.
This program’s authorization ends on September 30, 2024, and without a timely reauthorization, FDA will no longer be allowed to initiate the process necessary to issue new RPD PRVs.
Timely reauthorization is needed to support rare disease drug development
As of April 30, 2024, 53 PRVs have been awarded for 39 different rare pediatric diseases. Prior to the creation of the RPD PRV program, only three of these 39 rare pediatric diseases had any FDA–approved treatments. Additionally, more than half of all RPD PRV designations, awards and redemptions occurred after 2019, which is when the most recent Government Accountability Office (GAO) analysis of the PRV program ended.[1]
It is important to note that at the time of the GAO analysis, the program had only been in place for 7 years; on average, it takes 10+ years to bring a new rare disease therapy to market.[2] The significant uptick in rare pediatric disease drug approvals in recent years[3] demonstrates the benefit of this incentive to the rare disease patient community, where more than 95% of rare diseases lack an FDA approved treatment.
NORD would like to thank Senators Bob Casey (D-PA), Markwayne Mullin (R-OK), Sherrod Brown (D-OH), Susan Collins (R-ME), as well as Representatives Michael McCaul (R-TX), Anna Eshoo (D-CA), Gus Bilirakis (R-FL), Nanette Barragán (D-CA), Lori Trahan (D-MA), and Michael Burgess (R-TX) for introducing S.4583/H.R. 7384, the Creating Hope Reauthorization Act, which would reauthorize the critical RPD PRV program for at least five more years.
Footnotes
[1] U.S. Government Accountability Office. (2020, January 31). Drug development: FDA’s Priority Review Voucher Programs. Drug Development: FDA’s Priority Review Voucher Programs | U.S. GAO. https://www.gao.gov/products/gao-20-251
[2] Orphanet: About orphan drugs. (n.d.). https://www.orpha.net/consor/cgi-bin/Education_AboutOrphanDrugs.php?lng=EN
[3] Center for Drug Evaluation and Research. (n.d.). CDER continues to advance rare disease drug development. U.S. Food and Drug Administration. https://www.fda.gov/drugs/our-perspective/cdercontinues-advance-rare-disease-drug-development-new-effortsincluding-accelerating-rare-disease#:~:text=New%20Drug%20Approvals%20for%20Rare,for%20patients%20with%20rare%20diseases
