Writing in The Hill, National Organization for Rare Disorders (NORD) President and CEO Peter Saltonstall called upon Congress to act swiftly to maintain an important program encouraging research into treatments for rare pediatric diseases. The Rare Pediatric Priority Review Voucher (PRV) Program is set to expire at the end of the month, unless Congress reauthorizes it.
Developing therapies for people with rare diseases is incredibly difficult. Rare diseases are scientifically and medically complex, often locked away in the genetic code. Patient populations are small and dispersed, making clinical trials difficult. These obstacles are only more pronounced in kids.
In 1983, Congress passed the Orphan Drug Act to incentivize therapeutic development in rare diseases. In the resulting 33 years, over 500 orphan therapies have been developed for rare diseases compared to the only 34 orphan therapies in existence prior to 1983.
Thirty years later, in 2012, Congress recognized that more needed to be done for kids and created the Rare Pediatric Disease Priority Review Voucher Program. Since 2012, the program has spurred drug development for rare pediatric diseases, resulting in six new therapies for seven rare diseases that previously lacked a Food and Drug Administration (“FDA”)-approved therapy.
The voucher program works by awarding companies with a priority review voucher if they develop a therapy for a rare pediatric disease. The company can then use that voucher for one of their subsequent products, or sell it to another company. These vouchers allow companies to move through the FDA review process more quickly and are incredibly valuable; the latest voucher sold for $350 million.
Read the full piece here.