So far, the FDA has awarded four vouchers for neglected tropical diseases.
The 2012 voucher awarded to Janssen (Johnson & Johnson) for Sirturo,
bedaquiline-based drug used to treat multidrug-resistant pulmonary
tuberculosis, was the first tuberculosis drug to receive FDA approval in 40
Year Company Qualifying disease
2009 Novartis malaria
2012 Janssen(J&) tuberculosis
2016 PaxVax Bermuda cholera
Table 2: Priority review vouchers awarded for neglected tropical diseases, 2007-
Five years after the neglected tropical disease program was created,
Section 529 of the FDCA introduced a similar voucher-based incentives
program for rare pediatric diseases.
66 FDCA defines rare pediatric disease as
one that “primarily affects individuals aged from birth to 18 years,”
67 and an
FDA guidance on the pediatric voucher program further clarifies that a drug
qualifies for a pediatric voucher “if the entire prevalence of the disease or
condition in the U.S. is below 200,000 and if more than 50% of patients with
the disease are 0 through 18 years of age.”
From the start, sponsors of qualifying pediatric drugs were required to
notify the FDA 90 days before using the voucher, and the transfer of pediatric
vouchers was never subject to limitations, as was initially the case with
69 Pediatric vouchers may be used in conjunction with other
64. Press Release, Johnson & Johnson, FDA Grants Accelerated Approval for
SIRTURO™ (Bedaquiline) as Part of Combination Therapy to Treat Adults with Pulmonary
Multi-Drug Resistant Tuberculosis (Dec. 31, 2012), https://www.jnj.com/media-center/press-releases/fda-grants-accelerated-approval-for-sirturo-bedaquiline-as-part-of-combination-therapy-to-treat-adults-with-pulmonary-multi-drug-resistant-tuberculosis.
65. See Katie Thomas, F. D. A. Approves Drug for Resistant Tuberculosis, N.Y. TIMES
(Dec. 31, 2012), http://www.nytimes.com/2013/01/01/business/fda-approves-new-
tuberculosis-drug.html (discussing the FDA’s approval for Situro as a new treatment for multi-drug resistance tuberculosis).
66. 21 U.S. C. § 360ff.
67. 21 U.S. C. § 360ff(a)( 3).
68. U.S. FOOD & DRUG ADMIN., RARE PEDIATRIC DISEASE PRIORITY REVIEW VOUCHERS,
GUIDANCE FOR INDUSTRY, 3 (2014),
[hereinafter PEDIATRIC VOUCHER GUIDANCE].