Economic return of clinical trials performed under the pediatric exclusivity program.
BACKGROUND
In 1997, Congress authorized the US Food and Drug Administration (FDA) to grant 6-month extensions of marketing rights through the Pediatric Exclusivity Program if industry sponsors complete FDA-requested pediatric trials. The program has been praised for creating incentives for studies in children and has been criticized as a "windfall" to the innovator drug industry. This critique has been a substantial part of congressional debate on the program, which is due to expire in 2007.


OBJECTIVE
To quantify the economic return to industry for completing pediatric exclusivity trials.


METHODS
A cohort study of programs conducted for pediatric exclusivity. Nine drugs that were granted pediatric exclusivity were selected. From the final study reports submitted to the FDA (2002-2004), key elements of the clinical trial design and study operations were obtained, and the cost of performing each study was estimated and converted into estimates of after-tax cash outflows. Three-year market sales were obtained and converted into estimates of after-tax cash inflows based on 6 months of additional market protection. Net economic return (cash inflows minus outflows) and net return-to-costs ratio (net economic return divided by cash outflows) for each product were then calculated.


METHODS
Net economic return and net return-to-cost ratio.


RESULTS
The indications studied reflect a broad representation of the program: asthma, tumors, attention-deficit/hyperactivity disorder, hypertension, depression/generalized anxiety disorder, diabetes mellitus, gastroesophageal reflux, bacterial infection, and bone mineralization. The distribution of net economic return for 6 months of exclusivity varied substantially among products (net economic return ranged from -$8.9 million to $507.9 million and net return-to-cost ratio ranged from -0.68 to 73.63).


CONCLUSIONS
The economic return for pediatric exclusivity is variable. As an incentive to complete much-needed clinical trials in children, pediatric exclusivity can generate lucrative returns or produce more modest returns on investment.
Li
Jennifer S
JS
Department of Pediatrics, Duke Clinical Research Institute, Duke University, Durham, NC 27705, USA. jennifer.li@duke.edu
Eisenstein
Eric L
EL
Grabowski
Henry G
HG
Reid
Elizabeth D
ED
Mangum
Barry
B
Schulman
Kevin A
KA
Goldsmith
John V
JV
Murphy
M Dianne
MD
Califf
Robert M
RM
Benjamin
Daniel K
DK
D002986:Clinical Trials as Topic / Q000191:economics / Q000331:legislation & jurisprudence / Q000592:standards; D015331:Cohort Studies; D003365:Costs and Cost Analysis; D017277:Drug Approval; D016527:Drug Costs; D004345:Drug Industry / Q000191:economics*; D040541:Marketing; D010372:Pediatrics; D014481:United States; D014486:United States Food and Drug Administration