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FDA wants to end pediatric priority review program

Benefits in advancing drugs for rare diseases is questioned

by Ann M. Thayer
March 11, 2016 | APPEARED IN VOLUME 94, ISSUE 11

Credit: Regeneron Pharmaceuticals
The cholesterol-lowering drug Praluent benefited from a priority review voucher.
Credit: Regeneron Pharmaceuticals
The cholesterol-lowering drug Praluent benefited from a priority review voucher.

FDA officials do not support continuing the pediatric voucher program, scheduled to expire on Oct. 1. Created in 2012, the program is meant to incentivize the development of drugs against rare pediatric diseases. A similar program targeting neglected tropical diseases dates back to 2007.

Awarded by FDA upon a drug’s approval, these vouchers can be used on another product to shorten regulatory review to six months—a 40% reduction. If the awardee doesn’t need the voucher, it can sell it, which has occurred five times. In 2015, United Therapeutics reaped a record $350 million when it sold one to AbbVie.

FDA made its opinion known in comments to the Government Accountability Office, which spent eight months studying the effectiveness of the pediatric plan and recently released its report. FDA told GAO that it has seen “no evidence that the program has encouraged increased development of drugs for rare pediatric diseases.” FDA suggested that companies may find the grant of an additional period of market exclusivity a better incentive than the vouchers.

Given that drug development can take a decade or more, GAO concluded that it is too early to gauge the impact of the three-year-old program. In fact, all of the drugs awarded pediatric vouchers were already in development before the program began. Any sponsors motivated by the program “would likely be years away from submitting their new drug applications to FDA,” GAO added.

Meanwhile, FDA says the program saps its resources by requiring it to conduct priority reviews of new drugs that would not otherwise qualify. For example, FDA said it had to defer other work to conduct a priority review of Praluent, a cholesterol-lowering drug for which Sanofi and Regeneron Pharmaceuticals redeemed a purchased voucher.

Drug companies, patient advocacy groups, and trade associations “provided generally positive feedback on the program,” GAO reported. Most drug firms that sold vouchers intend to reinvest some of the money into research on rare diseases. And patient groups noted that the program has yielded important new therapies and could lead to others, in part by transferring needed cash from large companies to small ones.



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