Congress and President Barack Obama have one week to reauthorize the rare pediatric priority review voucher (PRV) program or else it will end on 30 September 2016, after seven pediatric PRVs have been awarded, one of which sold for $350 million last year.
The PRV program offers vouchers that companies can win for gaining approval of new rare pediatric treatments, affecting fewer than 200,000 patients under the age of 18. The vouchers can be used to speed FDA’s review of a new drug application by four months or sold to other companies.
Two bills are currently circulating to reauthorize the program – one from Sens. Bob Casey (D-PA) and Johnny Isakson (R-GA), known as S.1878 - Advancing Hope Act of 2016, and another companion bipartisan one from Rep. GK Butterfield (D-NC) and others, though both standalone bills make changes to the rare pediatric and the rare tropical disease PRV programs.
The Butterfield bill would designate pediatric cancers and sickle cell anemia as rare pediatric diseases, and amend the rare tropical PRV program to contain “an assurance (satisfactory to the Secretary) that the drug for which the application is submitted has not been approved for commercial marketing for any tropical disease indication by a government authority outside of the United States for more than 24 months before the application is submitted.”
The Casey and Isakson bill, meanwhile, would extend the pediatric PRV program until 2022, though if a rare pediatric designation from FDA is authorized by 30 September 2022, that drug remains eligible for a PRV if it is approved by 30 September 2027.
The stand-alone bills may not need to pass to reauthorize the program as Sen. Mitch McConnell's (R-KY) continuing resolution, unveiled Thursday afternoon to fund the government through December, would also re-authorize the rare pediatric PRV program through 9 December.
FDA, however, is not pleased with the idea of reauthorizing the PRV program.
Spokeswoman Deborah Kotz told Focus the PRVs "require the FDA to provide a service (i.e., priority review) that would not otherwise be warranted on the merits for the application for which the voucher is redeemed. This approach is not consistent with FDA’s usual approach to determine priorities for its public health work based on the merits of the application under review.
"In effect, these programs allow sponsors to 'purchase' a priority review at the expense of other important public health work in FDA’s portfolio. The FDA has very limited resources and the more we are mandated to provide special attention to products that do not warrant such special attention, the less any application can be treated as a priority," she added. "The special user fee for redemption of a PRV, while calculated to compensate the FDA for the extra work involved in conducting a priority review on a standard application, does not in practice provide additional review resources to the team responsible for reviewing the application that is the subject of the redeemed voucher."
Speaking on the Senate floor on Wednesday, Sen. Bernie Sanders (I-Vt.) reiterated FDA’s views of the PRV programs outlined in a GAO report from March, in which FDA said that “while they strongly support the goal of incentivizing drug development for rare pediatric diseases, they have seen no evidence that the program is effective.”
Kotz added: "Finally, FDA has expressed concern about what appears
to be the proliferation of PRV programs into additional therapeutic areas,
particularly as the GAO report makes clear it is too early to gauge the
effectiveness of these programs."
When Did it Sunset?
The Food and Drug Administration Safety and Innovation Act (FDASIA) from 2012 legislation makes clear: “TERMINATION OF AUTHORITY.— The Secretary may not award any priority review vouchers under paragraph (1) after the last day of the 1-year period that begins on the date that the Secretary awards the third rare pediatric disease priority voucher under this section.”
The third pediatric PRV was awarded in March 2015 to Asklepion Therapeutics, and three more pediatric PRVs were awarded between March 2015 and prior to Congress extending the PRV program when a budget deal was enacted 18 December 2015.
As the summaries of both bills seeking to reauthorize the pediatric PRV program note, the extension would remove “the provision terminating the program one year after the Food and Drug Administration's (FDA's) issuance of three rare pediatric disease vouchers. is extended by removing the provision terminating the program one year after the Food and Drug Administration's (FDA's) issuance of three rare pediatric disease vouchers.”
The text for the final bills remains to be seen as the Food Drug and Cosmetics Act (FD&C Act) says: “Termination of authority The Secretary may not award any priority review vouchers under paragraph (1) after September 30, 2016.”
In March, the Senate HELP committee advanced the reauthorization bill, but on Wednesday, Sens. Sanders and Elizabeth Warren (D-MA) joined forces to oppose the PRV extension and said, “Republicans accepted a counter-proposal from Sanders and Warren that would only extend the voucher program through the end of the year to allow time to come to an agreement on medical research funding.”
Sanders and Warren are calling for major increases in funding for FDA and the National Institutes of Health (NIH).
Number of PRVs
In May, David Ridley, faculty director of the Health Sector Management Program at the Fuqua School of Business at Duke University, who first proposed the creation of PRVs in a March 2006 paper published in the journal Health Affairs, warned that “if only one priority review voucher is available in a year, it will be worth more than $200 million, but if four vouchers are available, the value could fall below $100 million.”
Four PRVs, including Sarepta’s, appear to be still for sale as Alexion has said that it will not sell at least one of its two PRVs, though the four also include Janssen’s, which was awarded in 2012 and it remains unclear if the company will sell it.
Sarepta's PRV also remains unique in that it was the only PRV awarded to date for a drug that was authorized by FDA under the accelerated approval pathway. However, that distinction does not mean FDA can pull the PRV if Sarepta fails to prove its drug provides a clinical benefit in its follow-up trials.
"FDA would not
have a basis to revoke or withhold a voucher in the circumstances
mentioned (e.g. For a product approved under an accelerated approval
that has yet to confirm benefit.)," Kotz told Focus.
As far as what to expect moving forward, the FDA Law Blog noted this morning: “If efforts to reauthorize the pediatric voucher program before October 1 fail, the program could be reinstated as part of the 2017 user fee reauthorization package or other legislation. Whether this would retroactively extend the program to eliminate a gap between the sunset date and subsequent reauthorization remains to be seen.”