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Last week, an article published in Biocentury confronts the perspectives that a member of New Jersey’s congressional delegation has expressed regarding the Rare Pediatric Priority Review Voucher (PPRV) program. Congressman Pallone (D, NJ6), Ranking Member of the crucial House Energy and Commerce Committee, recently accused the PPRV program of being misused to be redeemed for me-too drugs — proposing an amendment that would have caused the program’s vouchers essentially worthless. Fortunately, the amendment was abandoned.
The article notes how the PPRV program has represented a lifeline for children suffering from rare diseases, and highlights a recent study conducted by the National Organization for Rare Disorders (NORD) that analyzed data through April 2024. NORD found that 54 PRVs have been awarded for treatments for 40 rare pediatric diseases. Only three of these diseases already had an FDA-approved treatment prior to the creation of the PPRV program.
Last week, the House passed the Give Kids a Chance Act by a voice vote, and the bill included reauthorization of the PPRV program. This follows the inclusion of a short extension of the program in a continuing resolution until December 20.
The Senate Health, Education, Labor & Pensions Committee (HELP) was slated to consider a bill that would reauthorize the program last Thursday, but the hearing has been postponed. The Committee was also slated to consider several other relevant bills, including the Biosimilar Red Tape Elimination Act (S. 2305) and Medication Affordability and Patent Integrity Act (S. 2780).
BioNJ will continue to convey the virtues of the PPRV program, and particularly how it has ultimately redounded to the benefit of young people confronting rare diseases.
To read the article in Biocentury, click here.
To read the PPRV study conducted by NORD, click here.
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