The original sunset provision established that the pediatric voucher
program would come to an end one year after the FDA issued the third
voucher for a rare pediatric disease.
73 That third voucher was granted to
Asklepion on March 17, 2015 for Cholbam, a drug that treats patients lacking
enzymes to synthesize bile acid.
74 In January 2016, Congress reauthorized
the program through September 201675 and a second short-term
reauthorization prolonged it until December 31, 2016.76 The 21st Century
Cures Act now extends the program until 2020.77 Drugs receiving rare
pediatric designation before October 1, 2020, will be eligible for a voucher if
approved before October 1, 2022.78
B. Criticism of the Voucher Program
In theory, the revenue generated, either by direct redemption or sale of a
priority review voucher, functions as a reward in areas where traditional
incentives—such as patents—have failed to generate substantial innovation.
The prize-like benefit is justified as a mechanism to incentivize costly R& D
in areas with small disease populations, rendering them attractive to
pharmaceutical companies who would otherwise avoid markets that offer few
prospects of covering their investment. The particular appeal of the voucher
system is that, unlike other types of incentives,
79 vouchers do not require any
direct financial support from the government or tax-based contributions. As
seen in the previous section, this feature renders them politically attractive
and has helped extend the life of the program.
To accomplish its goals,
80 the voucher program must however produce two
interlinked yet distinct outcomes: 1) pharmaceutical companies should invest
in R& D for neglected and/or rare diseases as a direct result of the voucher
73. 21 U.S. C. § 360ff(b)( 5) (“TERMINATION OF AUTHORITY.— The Secretary may
not award any priority review vouchers under paragraph ( 1) after the last day of the 1-year
period that begins on the date that the Secretary awards the third rare pediatric disease priority
voucher under this section.”).
74. See Press Release, U.S. Food & Drug Admin., FDA Approves Cholbam to Treat Rare
Bile Acid Synthesis Disorders (Mar. 17, 2015),
(explaining rare bile acid synthesis disorders and the hoped effect Cholbam will have on them).
75. Advancing Hope Act of 2016, Pub. L. No. 114-229, § 1878, 130 Stat. 943, 946 (2016).
76. Press Release, Kids V Cancer, President Obama Signs the Advancing Hope Act to
Extend the Creating Hope Act to December 31, 2016 (Sept. 30, 2016),
80. See TROPICAL DISEASE PRIORITY REVIEW VOUCHERS, supra note 23; see also Ridley
et al., supra note 16.