Economic return of clinical trials performed under the pediatric exclusivity program.
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CONTEXT: 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. DESIGN AND SETTING: 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. MAIN OUTCOME MEASURES: 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.
SubjectClinical Trials as Topic
Costs and Cost Analysis
United States Food and Drug Administration
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Kiser-Arena Distinguished Professor
Dr. Danny Benjamin is the Principal Investigator and Chair of the National Institute of Child Health and Human Development’s Pediatric Trials Network. The Network is responsible for designing and leading clinical trials of off-patent medicines in children of all ages across all therapeutic areas. The team has established, or is actively studying, the correct dosing and safety of more than 70 of the most commonly used medicines in children. E
Donald F. Fortin, M.D. Distinguished Professor of Cardiology, in the School of Medicine
Robert Califf, MD MACC, is the Donald F. Fortin, MD, Professor of Cardiology. He is also Professor of Medicine in the Division of Cardiology and remains a practicing cardiologist. Dr. Califf was the Commissioner of Food and Drugs in 2016-2017 and Deputy Commissioner for Medical Products and Tobacco from February 2015 until his appointment as Commissioner in February 2016. Prior to joining the FDA, Dr. Califf was a professor of medicine and vice chancellor for clinical and translational
Associate Professor Emeritus in Medicine
Research Interests: Dr. Eisenstein is a member of the Duke Clinical Research Institute’s Outcomes Research and Assessment Group, with a special interest in understanding the relationships between complex interventions in health care systems and the long-term clinical and economic outcomes of patients. He has served as Principal Investigator for phase II, III, and IV economic and quality of life studies conducted alongside randomized clinical trials in cardiovascular, emerge
Professor Emeritus of Economics
Professor Grabowski specializes in the investigation of economics in the pharmaceutical industry, government regulation of business, and the economics of innovation. His specific interests within these fields include intellectual property and generic competition issues, the effects of government policy actions, and the costs and returns to pharmaceutical R&D. He has been publishing research papers for over four decades, from his earlier work, “The Effects of Regulatory Policy on the Incentives
Beverly C. Morgan, M.D., Distinguished Professor of Pediatric Cardiology in the School of Medicine
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Adjunct Professor in the Department of Medicine
Kevin A. Schulman, MD, MBA, is a professor of medicine and the Gregory Mario and Jeremy Mario Professor of Business Administration (2010 - 2016) at Duke University. He is a visiting professor of business administration at Harvard Business School. He holds several leadership appointments at Duke. He is an associate director of the Duke Clinical Research Institute in the School of Medicine, the country's largest academic clinical research organization. In Duke's Fuqua School of Business, he s
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