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Regulatory News | 28 September 2016 | By Zachary Brennan
The House late Tuesday followed its Senate counterparts and sent to President Barack Obama a re-authorization of the rare pediatric disease priority review voucher (PRV) program that will last through the end of December.
A spokeswoman for Sen. Bob Casey (D-PA), a sponsor of the bill, told Focus that between now and 30 December, Congress will discuss ways to amend the program and extend it again.
“Our policy changes are in S. 1878, which is headed to the president, so the remaining negotiations will be on the length of the extension. The bill sponsors were pushing for permanency but did not get agreement for that in either the House or the Senate as part of 21st Century Cures/Innovations; HELP [Senate Committee on Health, Education, Labor & Pensions] agreed to 2022 with an option to earn a voucher through 2027 if the drug sponsor gets a drug in development designated as a rare pediatric disease product when we marked up S. 1878 in March. We’ll keep pushing for that in the negotiations, as the House version was a shorter extension,” she added.
FDA, however, is not pleased with the idea of reauthorizing the PRV program.
Spokeswoman Deborah Kotz told Focus last week 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."
In addition to the short-term extension, the bill, called the Advancing Hope Act, also requires drug sponsors to notify the US Food and Drug Administration (FDA) of their intent to seek a priority review voucher and it prohibits sponsors from collecting more than one priority review voucher per drug.
The bill also clarifies the definition of “rare pediatric diseases” from:
“The disease primarily affects individuals aged from birth to 18 years, including age groups often called neonates, infants, children, and adolescents.”
“The disease is a serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years, including age groups often called neonates, infants, children, and adolescents.”
Sen. Casey said in a statement that the new definition ensures that “the devastating treatment gap can be closed for all kids with rare diseases.”
S.1878 - Advancing Hope Act of 2016
Tags: rare pediatric priority review voucher, PRV program, PRV extension, Bob Casey