NORD Report Highlights Benefit of Pediatric Drug Development Incentive Program for Children Living with Rare Diseases

Effective Rare Pediatric Disease Priority Review Voucher (RPD PRV) Program Set to Expire September 30^th without Congressional Action

WASHINGTON, July 23, 2024 /PRNewswire/ -- Today, the National Organization for Rare Disorders (NORD) released a 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.

"This program gives hope to the estimated 15 million children living with a rare disease, of whom the vast majority are currently without FDA approved treatment options for their condition," said NORD CEO, Pam Gavin. "Our research shows that the RPD PRV program is helping to address a substantial deficit in pediatric rare disease drug development. Yet, without Congressional action, this effective program will end September 30, 2024, and this is simply unacceptable."

NORD's analysis found that since the RPD PRV program was created in 2012:

• A total of 53 RPD PRVs have been awarded across 39 rare pediatric diseases. Of these diseases, many typically lead to death before the children reach adulthood.
• It is effective in helping develop new FDA-approved treatments for pediatric patient populations that previously had zero or limited options. Of the 39 rare pediatric diseases granted a PRV, only 3 had any FDA-approved treatment option prior to the creation of the program.
• Utilization of the program has dramatically increased over time, with more than half of all RPD PRV designations, awards, and redemptions occurring in the last four years.
• The vouchers awarded through this program are not typically being utilized for "blockbuster" drugs, as only three vouchers have been redeemed for products in the top 50 in Medicare Part B or D drug spend.

"Without the PRV incentive, it is unlikely companies would have prioritized developing treatments for the relatively small, but profoundly impacted the Lennox-Gastaut Syndrome (LGS) population," said Tracy Dixon-Salazar, Executive Director at the LGS Foundation. "The PRV program has been vital in driving innovation and providing new therapeutic options that have improved lives for LGS families."

NORD and more than 190 patient organizations are urging Congress to swiftly reauthorize this vital program before it expires on September 30, 2024.

Read the full report here.

Background on the RPD PRV Program:

The RPD PRV program began in 2007 to address the unmet need to develop 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 transferred or sold to another manufacturer to obtain a priority review for their product.

Bipartisan legislation in the U.S. House of Representatives and Senate, H.R. 7384/S. 4583, the Creating Hope Reauthorization Act, would extend the RPD PRV program for at least another five years. NORD is grateful to Senators Bob Casey (D-PA), Markwayne Mullin (R-OK), Sherrod Brown (D-OH), and Susan Collins (R-ME) and Representatives Michael T. McCaul (R-TX), Anna G. Eshoo (D-CA), Gus M. Bilirakis (R-FL), Nanette Barragán (D-CA), Lori Trahan (D-MA), and Michael C. Burgess, M.D. (R-TX) for championing this critical program and its continuation.

To learn more about the RPD PRV please visit rarediseases.org/rare-pediatric-disease-prv-program/

About the National Organization for Rare Disorders (NORD):

With a more than 40-year history of advancing care, treatments and policy, the National Organization for Rare Disorders (NORD) is the leading and longest-standing patient advocacy group for the more than 30 million Americans living with a rare disease. An independent 501(c)(3) nonprofit, NORD is dedicated to individuals with rare diseases and the organizations that serve them. NORD, along with its more than 340 patient organization members, is committed to improving the health and well-being of people with rare diseases by driving advances in care, research and policy. For more information, visit rarediseases.org.

SOURCE National Organization for Rare Disorders (NORD)