Drugmakers are holding on to priority review vouchers: GAO

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Trine K. Tsouderos HRI Regulatory Center Leader, PwC US February 07, 2020

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An FDA program that awards priority review vouchers in three special drug categories is little studied and may not hasten medication development, according to a new US Government Accountability Office (GAO) report to Congress.

Under the program, drugmakers can receive vouchers that cut review times from the typical 10 months to six months for treatments for tropical diseases and rare pediatric conditions, and for drugs used as countermeasures to US health security threats. Awarded vouchers can be sold to another drug sponsor or redeemed for priority review of a drug of the sponsor’s choice, regardless of whether the medication would otherwise qualify for rapid review.

The idea is that the potential for revenue generated by voucher sales or faster FDA review would spur drug development in the three categories, despite their small markets and limited profit potential.

Since 2009, when the first arm of the program began, the FDA has awarded 31 vouchers. Sixty-one percent were for rare pediatric conditions, 32% for tropical diseases, and 7% for medical countermeasures. Seventeen of the vouchers were sold, for prices ranging from $67 million to $350 million, according to available data.

As of Sept. 30, 2019, drug sponsors had redeemed 16 vouchers to obtain shorter review times for drugs that target a range of conditions, from HIV to type 2 diabetes, the GAO found. When they redeem a voucher, drug sponsors must pay the FDA a special user fee, about $2.5 million last fiscal year, to help offset the extra cost of priority review.

In its program review, the GAO found just three studies—one for each drug category—that examined and offered conclusions about the vouchers. Only one analysis found evidence that the program affected drug development. That 2019 study concluded that the rate at which new drugs for rare pediatric diseases started or completed clinical trials did not increase under the program, compared with drugs for rare adult diseases, but that the rate of progress from phase 1 to phase 2 trials increased.

HRI impact analysis

Although few studies have evaluated the program’s performance, seven drug sponsors interviewed by the GAO said the initiative was a factor in drug development decisions. They also said their decision on when to redeem vouchers is based on strategic factors, including whether priority review would help a drug reach market before its competition, the likelihood that the medication ultimately would gain FDA approval, and whether the drug could earn priority review without a voucher.

The drug sponsors generally supported renewal of the rare pediatric disease and medical countermeasure arms of the voucher program—set to expire in 2022 and 2023, respectively. Researchers and other stakeholders, however, offered mixed views on the entire initiative’s impact and on program renewals.

All stakeholders offered program improvement recommendations, ranging from requiring drug sponsors to guarantee access to voucher-eligible drugs to limiting the program to sponsors with financial need. They also offered a variety of alternatives to the vouchers, including tax credits, direct federal funding, market exclusivity and patent extensions.

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Trine K. Tsouderos

HRI Regulatory Center Leader, PwC US

Tel: +1 (312) 241 3824

Crystal Yednak

Senior Manager, Health Research Institute, PwC US

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