Program limitations pertaining to winners of vouchers (drugs for neglected and rare diseases):

  • The prize rewards manufacturers of some drugs that would have been developed even in the absence of the program. In this way, the voucher program is like tax breaks for charitable donations that would have been made even without the tax incentive.
  • The prize rewards manufacturers of some drugs already available elsewhere in the world. Some drugs for these diseases are available in other countries, but are not registered in the United States, and thus might be eligible for vouchers. Some have called on Congress to make drugs ineligible for the voucher, if the drug has been available outside the United States for more than 3 years. Advantages of registering a drug in the U.S. include making the drug eligible for insurance payment and making the drug available to U.S. soldiers who often encounter neglected diseases when deployed.
  • The prize might be too small. Diseases with incredible burdens might merit more resources. The Advance Market Commitment, proposed by Michael Kremer and colleagues, calls for creating a $3 billion market for neglected diseases with high burdens, such as malaria, tuberculosis, and HIV/AIDS.
  • The prize does not ensure access to existing therapies. Some developers, such as Medicines Development for Global Health have access plans for their drugs. Some recommend that all manufacturers have such plans. In the absence of such plans, funding from governments or foundations might be needed to purchase treatments for poor people.

Program limitations pertaining to users of vouchers (drugs which would have had standard review now receiving priority review):

  • The prize might tie up FDA resources. Fortunately, however, the law includes an extra fee paid by manufacturers to the FDA and requires that voucher bearers provide FDA with 90 days’ notice before using a voucher.
  • Priority review might not be safe. Priority review should not, however, be confused with accelerated approval or fast track. Priority review does not omit safety or efficacy studies or require approval within a given time frame. It sets a target of 6 rather than 10 months for FDA review. Nevertheless, if the FDA feels pressure to meet deadlines faster, it might be more likely to err.
  • The program is costly for insurers. A drug using a voucher is available 4 months earlier, so insurers begin paying 4 months earlier. Some of the sales come from the competitive effect of taking market share from competing products (Ridley and Régnier 2016). However, some of the sales would not have otherwise been made. While this is a cost to society, presumably the benefit outweighs the cost. If it does not, then we must question whether approving any drug designated for standard review is beneficial at any time.

This web page is maintained by David Ridley, one of the authors of the priority review voucher program.