FDA Priority Review Vouchers for Neglected Diseases: A Little Goes a Long Way

The global health community is an ambitious group; we often gravitate to the "big ideas" that revolutionize the way that things are done. But sometimes you don't need to change the rules to make a difference - you only need to apply them creatively. In a 2006 Health Affairs article (subscription required), Duke University professors David Ridley, Henry Grabowski and Jeffrey Moe did just that by proposing the US Food and Drug Administration grant transferable "priority review vouchers" to companies that successfully obtain approval for a drug or vaccine against neglected diseases, which can then be used to jump the queue for a review of another of the company's FDA applications - shaving up to a year off the time to market for a potential blockbuster product. This accelerated approval could be worth over $300 million to major pharmaceutical companies; moreover, this also benefits smaller biotechnology companies (who could sell such a voucher to a larger company) and generic firms (who can also enter the market sooner given the earlier patent expiration). All in all, these priority review vouchers could act as a "pull mechanism," like Advance Market Commitments, to create incentives for manufacturers to increase their R&D investments in diseases of the poor. (These are complementary ideas: While the more generous AMCs are targeted at developing and delivering vaccines for which there are sizable donor resources, the priority-review voucher can be applied to a wider class of neglected diseases by encouraging developers to salvage existing projects that were initiated for other diseases.)

Instead of calling for a new global initiative or massive injections of donors funds, the Duke team sensibly called for a mere tweak to the existing rules - so small and simple that it almost slipped below the global health radar. But thankfully, it didn't quite make it past Senator Sam Brownback, who recognized the concept's true lifesaving potential and incorporated it into the Elimination of Neglected Diseases amendment to the FDA Revitalization Act (.pdf), which was co-sponsored by Senators Sherrod Brown and Joseph Lieberman and ultimately signed into law on September 27, 2007. This is a truly powerful example of how policy research can be translated into action, and it just goes to show that it really is the small things that matter.

See also: The Health Affairs blog shares a letter from Senator Brownback about the amendment, which is also featured in a Duke MBA newsletter. Finally, BIO Ventures for Global Health has an excellent summary of the HA proposal and the resulting legislation.