In 2007, the Food and Drug Administration Amendments Act (FDAAA) was passed, and with it the Priority Review Voucher (PRV) system was born. Initially created to target neglected tropical diseases, but later extended to pediatric diseases in 2012, this program lets the FDA award a drug manufacturer a voucher that allows it to have any one of its drugs reviewed under the FDA’s priority review system. To be eligible, a manufacturer simply has to be developing a drug to treat any of the tropical diseases on the official list created by the FDA, or any disease that primarily affects individuals from birth to 18 years of age.
Once awarded, the voucher can be redeemed by the manufacturer or sold on to another company. If redeemed, the voucher grants expedited review for the chosen drug, meaning the FDA has to review the drug within 6 months rather than 10. If sold, the voucher can be bought for anything between $25 million to hundreds of millions of dollars, depending on how much the purchaser is willing to pay and how many other vouchers are available for purchase.
It is easy to see how, in theory at least, such a program could be a good idea. For a company developing a drug, the goal is always to be able to sell that drug to as many people as possible, at as high a price as possible, so as to make a good return on investment. But what if the drug being developed is for a small patient population, or for a population that is poor and cannot afford to pay high prices? Manufacturers are likely going to choose to avoid these drugs in favor of drugs that can treat more common diseases. PRVs are supposed to combat this issue by providing an incentive to develop drugs for the diseases that are most commonly overlooked or neglected.
However, some are questioning whether or not this system effectively stimulates innovative drug development, as it is intended. In fact, researchers have suggested that the three vouchers awarded to companies developing drugs for tropical diseases might not have incentivized the development of novel therapies. For example, one voucher was awarded to a manufacturer for a drug that was already being widely used around the world for nine years by the time it was approved in the US.
Furthermore, there is also a question of whether the vouchers are being given to companies that actually require an incentive. For example, six vouchers have been awarded to companies developing pediatric drugs, but all of these drugs were already well into development at the time the vouchers were awarded, so they were expected to come to market with or without FDA assistance.
Then there is the issue of affordability. The program does not go so far as to force the companies that are awarded the vouchers to follow a particular pricing plan. Companies are free to set their price as high or as low as they see fit. So, although the PRV program might help bring novel treatments to market, it does not necessarily ensure their availability to those patients who require them.
The PRV system is definitely well intentioned. Developing a system to help patients who suffer from diseases that often go untreated is commendable. But there have to be measures in place to evaluate the program to make sure that it is actually working effectively. And, if it turns out that it is not, amendments will have to be made.