Hospital Pharmacy - May 2017 - 324

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Editorial

For Sale: FDA Priority Review Vouchers

Hospital Pharmacy
2017, Vol. 52(5) 324-325
© The Author(s) 2017
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https://doi.org/10.1177/0018578717715330
DOI: 10.1177/0018578717715330
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Danial E. Baker1

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.
References
1.

US Food and Drug Administration. Rare pediatric disease
priority review vouchers, draft guidance for industry. https://
www.fda.gov/downloads/RegulatoryInformation/Guidances/
UCM423325.pdf. Published November 17, 2014. Accessed
April 7, 2017.

Washington State University, Spokane, USA

Corresponding Author:
Danial E. Baker, Director, Drug Information Center, College of Pharmacy,
Washington State University, PO Box 1495, Spokane, WA 99210-1495,
USA.
Email: danial.baker@wsu.edu

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