For Sale: FDA Priority Review Vouchers

Did you know that the US Food and Drug Administration (FDA) has a voucher system related to new drug approvals? The voucher system was implemented to encourage drug development for “neglected” tropical and rare pediatric disease. Both of these represent markets that may not allow the pharmaceutical company to recoup its development cost. Recently, the development of medical countermeasures was added to the list of drugs covered by the voucher system.^1,2

Diseases eligible under the tropical disease priority review voucher system include blinding trachoma, Buruli ulcer, Chagas disease, cholera, cuevavirus, dengue/dengue hemorrhagic fever, dracunculiasis (Guinea-worm disease), Ebola virus, fascioliasis, human African trypanosomiasis, leishmaniasis, leprosy, lymphatic filariasis, malaria, Marburg virus, neurocysticercosis, onchocerciasis, schistosomiasis, soil-transmitted helminthiasis, tuberculosis, yaws, and Zika virus. The FDA also has the authority to add other infectious diseases that are defined within the regulation.^2,3 The drugs (also known as orphan drugs) used in the treatment of rare pediatric disease priority voucher system include those that affect fewer than 200 000 persons in the United States.^1,2

Unlike a lot of vouchers or coupons a consumer receives from a business as a reimbursement or “reward,” which are generally not transferable or cannot be sold, these priority vouchers can be sold. This means that the pharmaceutical company that submits the product to the FDA that qualifies for one of these voucher programs and gets FDA approval can market the new drug in the United States and receives a priority review voucher. The pharmaceutical company then has the right to use or sell the voucher to another company. In addition, if the FDA classifies the drug as an orphan drug, there are other financial incentives.

A pharmaceutical company with one of these priority review vouchers can use it to expedite the review of new drug product. For a pharmaceutical company to use one of these vouchers, it has to inform the FDA of its intent to use the voucher on an upcoming submission. When that application is submitted, the FDA accepts the voucher and agrees to review the data in the drug application within 6 months.^2,3 In addition to surrendering the priority review voucher, the pharmaceutical company has to pay the FDA the priority review fee plus the normal new drug user fee. In 2017, the priority review fee was $2 706 000.^1-5

These vouchers can be sold or transferred an unlimited number of times; they require a 90-day notification prior to its use and have no expiration date.^1-4 Published information from 2016 indicates that the FDA has distributed 13 of these vouchers. Fifty-four percent of these vouchers have been transferred and used by another company. Examples of how some of these vouchers were used include the use of a tropical disease voucher for the review of canakinumab (Ilaris); a rare pediatric disease voucher for the review of alirocumab (Praluent); a tropical disease voucher for the review of rilpivirine, emtricitabine, and tenofovir alafenamide (Odefsey); and a rare pediatric voucher for the review of basal insulin glargine/lixisenatide (Soliqua).^2,5-7

Use of a voucher does not guarantee that the drug will be approved, but it does speed up the time of the FDA’s review process, assuming the new drug or biologics license application is complete and provides sufficient efficacy and safety data to support the drug’s approval. If the drug is approved, the pharmaceutical company may be able to market their drug earlier than if it had been reviewed under the standard process. If that happens, it means the pharmaceutical company may be able to beat to the market a similar product or a different type of treatment for the same medical condition from another company or they can just get a head start on generating revenue from the sale of the drug.^2,7

The biggest unanswered question about this voucher system is whether it has accomplished its goal of increasing drug development for the treatment of these types of medical conditions or has become a means for a few companies to make money by selling these vouchers or purchasing these vouchers to speed up the approval process of other drugs. There appears to be no answer in sight.