The Regulatory Burden Of The Priority Review Voucher Program

In a recent interview, an official from the Food and Drug Administration (FDA) expressed concern about the costs to the FDA of the priority review voucher program. The director of the Office of New Drugs said he's worried the additional priority reviews are taking resources away from other important work. Furthermore, an FDA spokesperson wrote that "These programs allow sponsors to 'purchase' a priority review at the expense of other important public health work in FDA's portfolio."

But it doesn't have to be that way, and it's important to tackle these concerns because the FDA is critical to the success of the voucher program in encouraging the development of drugs for neglected diseases and rare pediatric diseases.

Officials from the FDA are busy with drug reviews and the voucher program makes them busier. But the voucher program also gives the FDA more resources. Each time a voucher is redeemed, the FDA receives a user fee ($2.7 million in fiscal year 2016) from the company redeeming the voucher, in addition to the user fee ($2.4 million in fiscal year 2016) required of all new drug applications with clinical data ($5.1 million total per drug redeeming a voucher).

In anticipation of three vouchers being redeemed in 2015, Congress allocated $8 million in extra funding to the FDA so the FDA had the resources in advance. Is the user fee sufficient? If not, the FDA has the power to increase it. Indeed, the voucher user fee was $5.3 million in fiscal year 2012, and the FDA cut it in half.

Part of the challenge is that work at the FDA is so variable. Some days are especially busy because of the requirements for priority review. However, more vouchers could actually reduce the variability and provide more resources for the FDA.

Another challenge for the FDA is that, while the financial resources are available to hire new staff, Federal pay constraints make it hard to offer a competitive salary to new reviewers. However, this is a broader challenge for the FDA that should be addressed.

The priority review voucher program is based on an article that Jeffrey Moe, Henry Grabowski, and I wrote for Health Affairs in 2006 and which became law in 2007. The program is a reward for registering at FDA a drug for a neglected disease or rare pediatric disease.

The developer receives priority review for that drug, as well as a bonus priority review voucher that may be sold. Hence, two drugs are involved: the drug that wins a bonus priority review that may be sold, and the drug that uses the bonus priority review. Thus far, seven vouchers have been awarded, four have been sold, and two have been redeemed.

Fortunately, the FDA has abided by their mandates under the voucher program. The FDA has an impressive record of completing priority review in six months. Furthermore, the FDA recently expanded the voucher program to include treatments for Chagas and Neurocysticercosis.

To reassure the FDA that their hard work will pay off in encouraging new drugs for neglected and rare diseases, we need to provide more evidence of the impact of the program. We are currently collecting data on clinical trials by companies developing drugs for voucher-eligible diseases. In the meantime, we can report that companies like Kineta, NanoViricides, and non-profit Medicines Development are working on drugs for neglected diseases, because of the voucher program.

I look forward to the day when their medicines reach the market. The FDA will deserve credit for encouraging development of these new drugs through the priority review voucher program.

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