With the summer recess for Congress next week, lawmakers will have to move quickly in the early fall if they’re going to reauthorize an FDA voucher program that aims to speed up new treatments for rare pediatric diseases.
The program has an expiration date of Sept. 30 unless it’s reauthorized.
Rare disease groups like the nonprofit National Organization for Rare Disorders are pushing for a swift reauthorization. NORD this week released a report highlighting how 53 rare pediatric priority review vouchers (PRVs) have been awarded to treatments for 39 rare pediatric diseases since 2012. Only three of those rare pediatric diseases had an FDA-approved treatment prior to the creation of the voucher program, NORD said.
But critics of the reauthorization say there’s no clear indication that the voucher program actually incentivizes developers to target rare pediatric diseases.
Aaron Kesselheim“I don’t think it should be re-authorized because there’s no high-quality evidence that it has incentivized actual introduction of new treatments for rare pediatric diseases,” Aaron Kesselheim, professor of medicine at Harvard Medical School, told Endpoints News via email.
He pointed to his research from 2019 that found the voucher program was not associated with a change in the rate of new pediatric drugs starting or completing clinical testing. His forthcoming article in the Journal of Pediatrics will show that rare pediatric disease drugs generated similar revenues as other brand-name drugs.
“When you take into account the FDA’s public criticism of the voucher in the past for disrupting its approval process and the fact that the drugs to which vouchers are applied tend to have low added clinical benefits to patients (because they are in the minority of drugs that don’t already qualify for priority review by virtue of the quality of the drug itself), you get a program that offers no clear benefits but has a lot of downsides,” Kesselheim added.
Congress is considering a short-term extension of the PRV program, potentially for four years, but some advocates are calling for it to be a permanent program, like the rare tropical disease PRV program.
Nancy Goodman, founder of the nonprofit Kids v. Cancer who’s advocating for the bill, told Endpoints that she expects the reauthorization of the PRVs will be included in the Give Kids a Chance Act, or HR 3433. “I anticipate it will pass the House before it sunsets,” Goodman said in an email.
She also said it’s “frustrating that the voucher program is not permanent. If it were permanent, then drug developers, starting at the beginning of the clinical development process, could count on the existence of a voucher and would place a much greater value on a possible future voucher.”