Signs that the industry is planning to reinvest voucher-money on R& D for
tropical or rare pediatric diseases are tentative at best. When interviewed
during the course of the pediatric program evaluation, representatives of
pharmaceutical companies that made money from the sale of a voucher told
GAO that they planned “to reinvest portions of the proceeds they received
into additional research on rare pediatric diseases.”
95 However, all available
evidence indicates that drug sponsors are not investing in innovative R& D
for voucher-eligible diseases, but rather acquiring fully developed drugs and
bringing them to the FDA for review.
96 To be sure, these companies still bear
the non-negligible costs of acquiring the drugs and of regulatory review, but
these are not the types of costs that the voucher program was designed to
cover. As per current practice, the program is subsidizing the non-negligible,
yet modest costs (by pharmaceutical industry standards) of bringing existing
drugs into the United States market. And, since there is no requirement that
voucher-money be used to fund R& D on voucher-eligible diseases, the drugs
so far approved through voucher priority review do not target niche or
underfunded areas.
97
2. Drug Affordability
Even if we were to settle for a system of vouchers that merely brings
existing drugs into the United States market, the no-strings-attached design
drugs awarded pediatric vouchers were in development prior to the voucher program’s
implementation. Any sponsors motivated by this relatively new program to attempt to develop
drugs for such diseases would likely be years away from submitting their new drug
applications to FDA.” GAO REPORT, supra note 82, at 9–10.
95. GAO REPORT, supra note 82.
96. Id.
97. In 2011, Novartis unsuccessfully redeemed the voucher it was awarded for its
combination therapy for malaria. The company used the voucher to speed up review of a
biologics license application for Ilaris, a canakinumab-based treatment for gouty arthritis. The
Arthritis Drugs Advisory Committee at the FDA voted 11-1 against approving the drug.
Following voucher redemptions fared better; in 2015, Sanofi used a voucher and obtained
approval of Praluent, which is used to treat high cholesterol levels; in 2016, Gilead used a
voucher and obtained approval for Odefsey, used in HIV- 1 infections; and also in 2016, Sanofi
again used a voucher and obtained approval for Soliqua, a combination therapy used in adults
with type 2 diabetes. All three drugs approved through a priority review voucher thus target
chronic and mainstream diseases. See Kurt R. Karst, Priority Review Vouchers – Not Much
Bang for The Buck, FDA LAW BLOG (July 11, 2011),
http://www.fdalawblog.net/fda_law_blog_hyman_phelps/2011/07/priority-review-vouchers-
not-much-bang-for-the-buck.html; Press Release, U.S. Food & Drug Admin., FDA Approves
Praluent to Treat Certain Patients with High Cholesterol (July 24, 2015),
http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm455883.htm; and
Press Release, Sanofi, Sanofi Receives FDA Approval of Soliqua 100/33 for the Treatment of
Adults with Type 2 Diabetes (Nov. 21, 2016), http://www.news.sanofi.us/2016-11-21-Sanofi-
Receives-FDA-Approval-of-Soliqua-100-33-for-the-Treatment-of-Adults-with-Type-2-
Diabetes.
