The need to reform the U.S. FDA Priority Review Voucher program for neglected diseases
US Congress needs to fix this incentive program so that it can deliver the treatments and vaccines people affected by neglected diseases urgently need
A reward intended to promote innovation for neglected patients should actually reward innovation for neglected patients. The US Food and Drug Administration (FDA)’s Priority Review Voucher (PRV) is supposed to reward innovation for companies that deliver new treatments and vaccines for diseases, like tuberculosis, Chagas disease, Zika and Ebola, but loopholes in the program mean currently companies can win big, even if neglected patients have nothing to show for it.
How does it work?
Companies that successfully register a product with the FDA to treat certain neglected diseases are rewarded with a voucher (a PRV) that fast-tracks a subsequent product through the crucial FDA review process (with a targeted decision time of six months compared to ten months under standard review).
This voucher can be used by the recipient or sold to another entity. These vouchers are a highly lucrative incentive and have been sold for as much as $350 million.
As of April 2017, four PRVs for neglected diseases and nine PRVs for rare pediatric diseases (issued under a similar program) have been issued. The program has also been recently extended to cover more diseases and a similar program has been created for medical countermeasures.
What’s the problem?
Despite this clear economic value for companies, the PRV program for neglected diseases has been gamed. The conditions attached to the program have so far failed to ensure that the incentive works to reward new therapeutic products for neglected diseases patients and are accessible to the patients and health care providers who urgently need them.
That’s because of two key flaws in the design of the program:
1. A PRV for neglected diseases can still be awarded even when the medical product already exists and is not new to people affected by neglected diseases.
2. A PRV for neglected diseases can still be awarded even if the product is not available to neglected patients, because the program does not include any mechanism to ensure patients, governments and health care providers will have affordable and appropriate access to resulting medical products.
What should US Congress do about it?
This is a program created by US Congress and it is the responsibility of the US Congress to ensure that it works as an incentive for neglected patients. Congress should introduce at least two amendments to close the loopholes in the PRV program:
1. The PRV program for neglected diseases should have a novelty requirement. The product has to be new, it has to offer a new therapeutic or prevention option for a neglected disease. If the product has already been available to treat or prevent the neglected disease for years elsewhere in the world, it shouldn’t be eligible.
2. The PRV program for neglected diseases should have an access strategy. If the company doesn’t intend to register the product in countries where it’s needed most, or doesn’t plan to make it affordable to neglected disease patients and treatment providers, it shouldn’t be eligible to win this US government incentive.
MSF and others have been asking US Congress to fix the program for more than two years, and despite numerous opportunities to do so, Congress has so far failed to act in the interest of patients and neglected disease innovators. Read our latest letter to US Congress here and check out our other resources below for more information.