Rare Disease Supporters Suggest Permanent PRV Program, Even If It May Concern FDA
Executive Summary
Non-profit biotech founder says permanent pediatric rare disease priority review voucher program will boost incentive, but FDA is concerned about resource drain vouchers cause.
Rare disease advocates want to add more certainty to the pediatric rare disease priority review voucher program, although that may put them at odds with the US FDA.
The pediatric rare disease priority review voucher program has generated hundreds of millions for sponsors, but the program will sunset in October 2020, potentially limiting investment in the space.
Robert Selliah, founder and CEO of American MedChem Nonprofit Corp., which deals in pediatric rare diseases, said Feb. 28 that permanent reauthorization of the program would enhance the incentive for his company's industry partners. He said it would ensure sponsors would not lose out on a voucher if the program ends.
"When this is permanent, what it allows industry to do is to plan," Selliah said during a briefing sponsored by the Rare Disease Congressional Caucus.
Selliah's company looks for potential drugs caught in the so-called "valley of death" following the basic research stages and pushes them to the clinical stages. If promising, the company will license the product to industry.
Selliah said that the potential for receiving a priority review voucher, which allows the holder to gain a priority review for any product it chooses, is a strong incentive for industry to take on a product for a very small patient population.
American MedChem does not want the voucher and will give it to the drug company taking over the project in return for a licensing fee.
"With this incentive we can go and have a conversation and say come work with us," Selliah said.
Vouchers also may be sold, which for small drug developers provides the potential for much-needed funding.
Indeed, the market for priority review vouchers skyrocketed to a peak of $350m in 2015. However, their value had dropped in recent years. Recent sales have ranged from $125m to $150m. (Also see "Orphan Incentives: Voucher Price Drop Gives Some Sponsors 'Pause'" - Pink Sheet, 18 Dec, 2017.)
Will FDA Put Its Foot Down On Voucher Extensions?
That presents a problem for permanent reauthorization. As the volume of available vouchers grows, their value, and by extension the value of the incentive, will decrease.
Congress recently created another voucher for medical countermeasures, which will further pressure sale prices when they are awarded. (Also see "Countermeasures May Need More Incentives Than Vouchers, Gottlieb Says" - Pink Sheet, 17 Jan, 2018.)
Add to the potential problem that FDA, among the strongest advocates for rare disease drug development, is not a fan of the priority review voucher idea. Agency officials have said expedited reviews necessitated by vouchers can force resources be directed away from other projects. (Also see "Review Voucher Program For Rare Pediatric Diseases Should Not Be Reauthorized, US FDA Says" - Pink Sheet, 3 Mar, 2016.)
FDA opposition certainly could help derail a push to make the program permanent.
The rare disease community also may be fighting an uphill battle with pending legislation it has championed. The Orphan Product Extensions Now, Accelerating Cures and Treatments (OPEN ACT), which allows for additional exclusivity for approved drugs repurposed for orphan indications, has run into drug pricing concerns on Capitol Hill. Patients and advocates were pushing the bill during Hill meetings as part of their annual Rare Disease Week activities. (See sidebar.)
Congress Not Pushing As Hard To Protect Incentives, Advocate Says
Some rare disease advocates also used the caucus briefing to call out legislators that they believe are not fighting as hard for orphan incentives as they had been.
Deanna Portero, executive director of the Fibrous Dysplasia Foundation, said that some are "taking their foot off the gas" somewhat and that she is seeing "representatives not fighting as hard to keep incentives that are working in place."
She said advocates must fight for protection of current incentives, along with creation of new incentives. That is growing difficult with the pharma industry painted in a negative light.
"I think it is an elephant in the room," Portero said. "I think that it's difficult for our representatives sometimes to champion incentives for rare disease drug development because they are afraid of being painted or seen as being in cahoots with the pharma industry."
Much of the concern likely centers on the cut of the orphan tax credit from 50% of a sponsor's clinical testing costs to 25% in the recent tax reform package. (Also see "Orphan Drug Tax Credit Defense Adds To NORD's Legislative Challenges" - Pink Sheet, 2 Nov, 2017.)
The National Organization for Rare Disorders was not happy with the cut, but was not necessarily ready to criticize policymakers over it. Director of Federal Policy Paul Melmeyer said during the Feb. 26 Rare Disease Legislative Advocates Legislative Conference that it still will provide a stronger tax incentive for companies to develop rare disease treatments than common diseases.
Isabelle Lousada, CEO and president of the Amyloidosis Research Consortium, also indicated the hiring freeze President Trump imposed at the beginning of his term was potentially damaging to implementation of beneficial provisions in the 21st Century Cures Act.
FDA could hire employees for Cures-related positions even though the hiring freeze was in effect. However, the impact of adding those new specialists may have been tempered by the agency's inability to fill some of the hundreds of openings within its rank-and-file. (Also see "The Freeze Thaws: US FDA Allowed To Hire Staff For Cures, User Fee Activity" - Pink Sheet, 22 Mar, 2017.)