incentive; and 2) if approved by the FDA, drugs and therapies for the diseases
targeted by the program should be made available to patients at prices they
can afford. Early evidence from the two existing types of vouchers suggests
that the current design of the program might not be conducive to achieving
either of these outcomes.
So far, there is a single formal evaluation of the vouchers—focusing only
on rare pediatric diseases—and that evaluation has contributed little to a
comprehensive assessment of the vouchers as incentives. Pursuant to 21
U.S. C. § 360ff(i)( A),
81 in March 2016 the GAO issued a report that bears the
most self-explanatory of titles: Rare Diseases – Too Early to Gauge
Effectiveness of FDA’s Pediatric Voucher Program.
82 The report raises
important questions, especially with regard to the effect of the program on
the FDA.83 The Agency expressed the view that the program:
adversely affects the agency’s ability to set its public health priorities by
requiring FDA to provide priority reviews of new drug applications that
would not otherwise qualify if they do not treat a serious condition or
provide a significant improvement in safety or effectiveness. Additionally,
FDA officials said that the additional workload from the program strains
the agency’s resources.
However, the main conclusion of the report is that, given the extended life
cycle of drug R& D, the then 3-year-old pediatric voucher program had yet to
generate enough evidence to accurately assess whether the vouchers were
“encouraging the development of drugs for rare pediatric diseases.”
In contrast with the findings of the report, commentators have been more
overt in criticizing the voucher program, in both of its current iterations.
now, sufficient empirical evidence has emerged that it is possible to identify
and analyze trends shared by the pediatric and the tropical disease programs.
These trends suggest that it is unlikely that the vouchers are accomplishing
the goals for which they were designed. The following sections examine the
two-fold criticism of the program, starting with the problem of incentives to
increased R& D in voucher-eligible areas, and then turning to the question of
81. 21 U.S. C. 360ff(i)( 1)( A).
82. See generally U.S. GOV’T ACCOUNTABILITY OFF., GAO-16-319, RARE DISEASES: TOO
EARLY TO GAUGE EFFECTIVENESS OF FDA PEDIATRIC VOUCHER PROGRAM (Mar. 2, 2016),
http://www.gao.gov/products/GAO-16-319 [hereinafter GAO REPORT].
84. U.S. GOV’T ACCOUNTABILITY OFF., HIGHLIGHTS OF GAO-16-319, A REPORT TO
CONGRESSIONAL COMMITTEES (Mar. 2, 2016), http://www.gao.gov/products/GAO-16-319
[hereinafter HIGHLIGHTS OF GAO-16-319].
85. GAO REPORT, supra note 82, at 9.
86. See, e.g., Aaron S. Kesselheim, Drug Development for Neglected Diseases — The
Trouble with FDA Review Vouchers, 359 NEW ENG. J. MED. 1981, 1981 (2008).