A distinctive feature of the pediatric voucher program was that it gave the
Government Accountability Office (GAO) a year counting from the
awarding of the third rare pediatric disease priority voucher to “conduct a
study of the effectiveness” of the program.
71 Specifically, GAO was to report
(i) The indications for which each rare disease product for which a priority
review voucher was awarded (…);
(ii) Whether, and to what extent, an unmet need related to the treatment or
prevention of a rare pediatric disease was met through the approval of such
a rare disease product;
(iii) The value of the priority review voucher if transferred;
(iv) Identification of each drug for which a priority review voucher was
(v) The length of the period of time between the date on which a priority
review voucher was awarded and the date on which it was used.
So far, seven vouchers have been awarded for rare pediatric diseases, at a
rate that is five times higher than the one for neglected tropical diseases.
Year Company Qualifying disease
2014 BioMarin Morquio A syndrome
2015 United Therapeutics High-risk neuroblastoma
2015 Asklepion Pharma Bile acid synthesis disorder
Hereditary orotic aciduria
Lysosomal acid lipase
2016 Sarepta Duchenne muscular
Table 3: Priority review vouchers awarded for rare pediatric diseases, 2012-2016
70. 21 U.S. C. § 360ff(g); see also PEDIATRIC VOUCHER GUIDANCE, supra note 68, at 1–2
(discussing how as with the tropical disease vouchers, diseases qualifying for pediatric
vouchers may cumulatively qualify for orphan drug designation and corresponding benefits).
71. 21 U.S. C. § 360ff(i)( A); see also infra Part II. B.