The Food and Drug Administration's priority review voucher program offers an expedited review for any drug of a drug makers' choosing if the company develops a drug that treats a neglected tropical disease or a rare pediatric disorder. The agency has granted seven such vouchers since the program began in 2007.
A new Viewpoint in JAMA argues the program has failed to live up to its stated purpose and needs redesigning. “Little reliable evidence that the program's primary intention of spurring novel drug development has been met exists, at least for tropical diseases,” writes Dr. Aaron Kesselheim, an associate professor at Harvard Medical School in the opinion piece published in JAMA this week.
Kesselheim, who directs the Brigham and Women's Hospital Program on Regulation, Therapeutics and Law, notes that the first companies to receive tropical disease vouchers didn't develop new drugs. They won vouchers by gaining approval for new indications for old drugs or secured U.S. approval for drugs for neglected tropic diseases that were already in use elsewhere.
Vouchers for rare pediatric diseases, added to the program in 2012, likewise have been issued for drugs that were developed or in development long before the launch of the voucher program.
What the program has succeeded in creating, as Modern Healthcare reporter Steven Johnson has noted, is a booming side business in the sale of the vouchers, which can be freely traded or sold to the highest bidder once received. In 2014, Sanofi and Regeneron paid $67.5 million for a voucher that helped to speed review of $14,000-per-year cholesterol-lowering drug Praluent, nudging its July 2015 approval about a month ahead of a competing Amgen drug, Repatha.
In recent months, the going price had soared to $350 million for a voucher bought by AbbVie, suggesting there may be concerns in drug industry circles that the program may soon be curtailed because of growing anger by politicians, insurers and think tanks about ballooning drug prices.
Kesselheim notes that the impressive sums being paid for vouchers is still rather small compared to the billions of dollars in extra revenue that can be gained from an expedited review at the agency (the vouchers shortens review times by six or more months). Yet the payoff in terms of generating new drugs for profitable tropical or rare diseases is almost non-existent.
Any solutions for the poorly drawn incentives? Kesselheim suggests Congress should require making a drug company invest a minimum amount in R&D in a new drug for neglected diseases before receiving a voucher.