|FDA's John Jenkins|
Big Pharma has been willing to pay hundreds of millions of dollars for a shortcut to FDA approval, buying up priority review vouchers created to incentivize new drugs for neglected diseases. But the agency seems less than enthusiastic about honoring its end of the bargain, with one top official expressing concerns about how the voucher program might harm the FDA's core mission.
In an interview with Pharma & MedTech Business Intelligence, FDA Office of New Drugs Director John Jenkins said the agency's long-held problems with priority review vouchers have been "amplified" as more and more companies line up to redeem them. And the market value of the vouchers has skyrocketed over the past year, with one going for $350 million in August, suggesting the issue isn't going away.
The voucher program, created in 2007, works like this: Any drug company that wins approval for a new drug that treats a rare pediatric disorder or neglected tropical disease is given a one-time coupon, which, when redeemed, shortens the FDA review process from 10 months down to 6. And, in a move designed to encourage R&D into such under-served diseases, winning companies can sell their vouchers to anyone they please and at whatever price.
But what's not negotiable is that 6-month timeframe, and that's what worries Jenkins. "In effect, these programs allow sponsors to 'purchase' a priority review at the expense of other important public health work in FDA's portfolio," he told PMBI. The FDA has a finite amount of time and resources available at any given time, and allowing one company to cut in line inevitably affects the agency's ability to do its job elsewhere, he said.
And the universal applicability of a priority review voucher could also create problems, according to Jenkins. A drugmaker could--as Sanofi ($SNY) did--use its coupon on a primary care treatment that would be used by millions of patients. Such drugs require multiple large clinical trials to support approval, and thus their applications are complex and time-consuming. "Reviewing such an application in 6 months is very challenging," Jenkins told PMBI, and the voucher program does not provide any additional staff to follow through.
Despite regulators' concerns, however, the program is a hit with industry, which is paying more and more for the privilege of a quick review.
The first voucher to change hands came from BioMarin ($BMRN), which sold the coupon to Sanofi and Regeneron ($REGN) last year for $67.5 million, helping the pair leapfrog Amgen ($AMGN) in the race to launch a new class of cholesterol treatments. Months later, Gilead Sciences ($GILD) paid $125 million for Knight Therapeutics' voucher in hopes of accelerating its HIV pipeline, followed by Sanofi splurging $245 million for Retrophin's ($RTRX) priority ticket and AbbVie ($ABBV) promising $350 million to United Therapeutics ($UTHR) for the same asset. Last month, AstraZeneca ($AZN) paid an undisclosed sum for a voucher of its own.
- read the interview