GAO report raises questions about reauthorizing two PRV programs
With the rare pediatric disease and medical countermeasure priority review voucher (PRV) programs set to expire by 2022 and 2023, respectively, the Government Accountability Office (GAO) released a new report Friday on the successes and hiccups in all three PRV programs and what stakeholders think.
The PRV programs incentivize companies to develop new rare pediatric disease, tropical disease and medical countermeasure drugs by providing the companies with a voucher when a new treatment is approved. The voucher can be used to speed the review of another drug or sold to another company.
From fiscal year 2009, when the first PRV was awarded, through last September, GAO says the FDA has awarded 31 PRVs, mostly for rare pediatric disease treatments. Of the 31 PRVs awarded by the FDA, 17 were sold to another sponsor for prices ranging from about $67 million to $350 million.
But the report also explains how the FDA, which officially does not have a position on the reauthorization of the two PRV programs, brought in almost $44 million in PRV user fees for the 16 redeemed PRVs but still has issues.
Similar to concerns raised with PRVs in a 2016 GAO report, the agency again told GAO that it “cannot anticipate the therapeutic area for which a PRV will be redeemed, so PRV user fees may not ameliorate the effect of PRV redemptions on the review divisions or provide for rapid hiring of additional review staff with relevant experience and technical expertise.”
The agency also said that the services of over 11 FDA offices are required to work on some aspect of the PRV programs, which may at times require the FDA to shift resources from its public health priorities.
Drug sponsors, researchers and stakeholders, meanwhile, reported “mixed views” to GAO on the reauthorizations, although sponsors “generally indicated support for their reauthorizations, with some noting that PRV program expirations may negatively affect overall drug development and the willingness of drug sponsors to work in these areas.”
But one researcher supported the expiration of the two programs, noting that such decisions could ultimately raise the potential revenue from the sale of an available PRV and could also make the tropical disease PRV program, which does not require reauthorization, more popular.
As far as tweaks to the programs, stakeholders suggested requiring innovation for and guaranteed access to PRV-eligible drugs, among other changes.
“Two researchers and two stakeholders noted that the PRV programs, particularly the tropical disease PRV program, have been criticized for not providing incentives for innovation and suggested PRV awards be limited to drugs new to the global market,” GAO said.
GAO also said it found few studies that examined the PRV programs, and those that did found the programs had little or no effect on drug development.
RAPS: First published in Regulatory Focus™ by the Regulatory Affairs Professionals Society, the largest global organization of and for those involved with the regulation of healthcare products. Click here for more information.
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