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Utah Life Sciences News & Events

Pediatric Rare Disease Voucher Program Extended

October 9, 2020

Congress recently provided a short-term extension until December 11 of the Rare Pediatric Disease Priority Review Voucher program. The program was set to expire on September 30, but was given a reprieve in the Continuing Appropriations Act, 2021 and Other Extensions Act (H.R. 8337) that kept the government funded. Congress will have to act again to ensure that the FDA can continue to award vouchers after December 11.

Separately, the House passed the Creating Hope Reauthorization Act (H.R.4439) on September 29th, which would extend the program for four more years. The Senate has yet to act on a companion bill (S.4010).

The program incentivizes industry to develop treatments and cures for children with rare life-threatening conditions, including cancer. To date, 22 new drugs for pediatric rare diseases have been approved by the FDA, with more expected in the pipeline. Drug companies face many hurdles in this market, including the high cost of drug development for a limited number of patients.

Of the over 7,000 rare diseases identified in the U.S., 50% affect children. BioUtah, along with a coalition of stakeholders and patient advocates are supporting creation of the Utah Rare Disease Council. During the 2020 session, the state legislature passed House Bill 106 to authorize and fund the council. However, budget cuts in response to the economic impact of COVID-19 has temporarily stalled creation of the council. BioUtah fully expects that funds for the council will be restored next year. 

“We look forward to establishing the Utah Rare Disease Council as soon as possible,” said Kelvyn Cullimore, president and CEO of BioUtah. “The Council will educate policymakers and help us more effectively address issues impacting the rare disease community.”

Drug companies receiving a priority review voucher can have any drug reviewed under priority review (getting an FDA decision in six months as opposed to the standard 10 months).