Sunset coming for rare pediatric disease treatment vouchers

(The Center Square) – Government vouchers that fast-track drug approval for rare pediatric diseases are nearing expiration.

Pennsylvania Democratic Sen. Bob Casey, however, wants to revive the program for another decade – the longest renewal period since it was introduced in 2012. Together with Sen. Markwayne Mullin, R-Okla., the lawmakers say the extended time period gives pharmaceutical companies more stability and brings more treatments to market.

“Too many children suffer from rare diseases with few treatment options,” Casey said Thursday. “Our bipartisan bill will keep this critical voucher program going so drug companies don’t stop innovating new treatments to help sick kids. This is an investment in finding treatments and cures for rare diseases so that children get the care they need.”

Only 5% of rare pediatric diseases – defined as “serious or life-threatening” condition that affects less than 200,000 patients in the United States – have an FDA-approved drug or biological treatment, according to researchers from Harvard Medical School and Boston Children’s Hospital.

Approximately 10 million children across the country live with a rare disease, though developing treatments can be costly and complex, especially for pediatric patients. Companies that use vouchers for new drugs will receive an FDA decision in six months, rather than 10.

Drug companies say doing so boosts private investment into treatments that would otherwise be overlooked while giving them more time to recoup the millions – and sometimes billions – of dollars sunk into development.

Cashing in on the vouchers can also be lucrative. Seventeen of 19 vouchers awarded through 2019 were sold for an average of $100 million each. A report from the Regulatory Affairs Professionals Society that was compiled using federal data found that new owners redeemed the vouchers to approve medicines for type 2 diabetes, HIV, cardiovascular disease and rheumatoid arthritis.

A report from the Government Accountability Office found no correlation between the program and boosted drug development.

Researchers note, however, that the news isn’t all bad. Vouchers appear to advance more drugs through clinical trials and could mean more FDA approvals in the future. Lawmakers, however, must consider how the policy will divert resources from treatments that may prove more effective, they said.