The U.S. House of Representatives has given a unanimous thumbs-up to the Give Kids A Chance Act, which would revive the FDA’s rare pediatric review voucher program following its expiration in December of last year.
Rep. Michael McCaul, R-Texas, introduced the legislation in February alongside support from five other Republican Congressmen and five Democrats. The bill now advances to a Senate vote.
“By strengthening incentives for pediatric drug development, restoring essential programs such as the FDA Rare Pediatric Disease Priority Review Voucher, and ensuring that life-saving therapies reach the children who need them most, we are making meaningful progress,” Rep. Gus Bilirakis, R-Fla., said in a release.
The initiative, which was established in 2012, offered drugmakers a financial incentive in the form of a priority review voucher (PRV) to develop rare disease treatments for children.
PRVs are valued commodities in the biopharma industry because they can slash the FDA’s review time of drug applications from the standard 10 months to six months. In many cases, companies that were awarded PRVs for developing approved rare disease drugs sold them to other drugmakers anxious to bring a lucrative product to the market.
As it previously appeared that the pediatric review voucher program was not going to gain Congressional reauthorization and the availability of PRVs grew scarce, their prices increased from a standard $100 million. In August of last year, Ipsen received $158 million from an unnamed buyer for a PRV it gained from the FDA upon approval of the rare disease drug Sohonos in August of 2023.
Six months ago, an unnamed buyer paid Bavarian Nordic $160 million for a PRV awarded by the FDA under its tropical disease incentive program for the February approval of the chikungunya vaccine Vimkunya. In May, Abeona Therapeutics sold a PRV for $155 million that it had gained just two weeks earlier upon the approval of its gene therapy Zevaskyn.
After the rare pediatric review program expired, there were calls from the industry to reestablish it, while critics raised concerns that the ability to sell PRVs at high prices distorted the program's original purpose.
In an article published in February, lawyers at Hyman, Phelps & McNamara suggested the end of the rare pediatric PRV program could lead to increased value of the tropical disease PRVs, potentially to a level on par with a record $350 million that AbbVie paid United Therapeutics in 2015.
Meanwhile, in June of this year, the FDA introduced a new PRV program designed to shorten drug review time to as little as one or two months. The commissioner’s National Priority Voucher (CNPV) coupon is given to “companies aligned with U.S. national priorities,” the FDA said. Unlike existing PRVs, CNPVs are nontransferable.