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Prices Rise For Priority Vouchers

Pharmaceuticals: Big pharma is paying more to get chits from neglected disease firms to speed up FDA review

by Ann M. Thayer
September 10, 2015 | A version of this story appeared in Volume 93, Issue 36

Vouchers from FDA offering a faster drug review in exchange for developing a neglected disease treatment are a hot commodity this summer, with three changing hands.

Sanofi paid $245 million to buy one from Retrophin in July, and AbbVie shelled out $350 million to United Therapeutics in August. Then last week, Wellstat Therapeutics transferred its voucher to AstraZeneca for a previously negotiated but undisclosed amount.

Launched in 2007, priority review vouchers are an incentive for developing novel drugs against neglected tropical or rare pediatric diseases. Awarded by FDA upon a drug’s approval, the vouchers can be used on another product to shorten the regulatory review to six months from the average of about 10. If the awardee doesn’t need the voucher, it can reap rewards by selling it.

When Duke University economist David Ridley and two colleagues proposed the idea in 2006 (Health Aff. DOI:10.1377/hlthaff.25.2.313), they thought the vouchers could be worth as much as $300 million to a big pharma firm for the early market access they can offer. The first sale, by BioMarin Pharmaceutical to Sanofi and Regeneron Pharmaceuticals in 2014, was for just $68 million, but prices quickly rose.

“Would-be buyers were very cautious about the first voucher,” Ridley says. “Recent voucher sales have been much more in line with our expectations.” But prices could collapse. “Like any other tradable asset, if supply rises, the market price will fall,” he adds.

FDA has awarded seven vouchers since 2009. Novartis was the first to receive one and used it on an attempt to get the anti-inflammatory Ilaris approved. Using the voucher from BioMarin, Sanofi and Regeneron got approval in July for Praluent, the first PCSK9 inhibitor. And Gilead Sciences recently asked FDA to okay a new HIV drug combination after spending $125 million for a voucher awarded to Knight Therapeutics.

Knight’s voucher caused the Drugs for Neglected Diseases Initiative and Doctors Without Borders to criticize what they saw as shortcomings in the program. They claimed that Knight shouldn’t have qualified because the drug wasn’t new and others had done most of the R&D. They also complained that the companies benefiting were not required to assure patient access to affordable drugs.

Proponents argue that the voucher program does incentivize small firms and can help raise their profiles with investors. “I have seen plenty of data and heard many anecdotes that new drugs are advancing because of the voucher program,” Ridley says.

More vouchers are expected in the next few years, especially since the list of eligible tropical diseases has been expanded. Vouchers for pediatric diseases are in limbo. The pediatric program will end in March 2016 but could be extended if the 21st Century Cures Act passes.

“We need to continue to work on promoting access to these new drugs while preserving the incentives for development,” Ridley says.



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