Economic return of clinical trials performed under the pediatric exclusivity program.


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.







Jennifer Shiunroh Li

Beverly C. Morgan, M.D., Distinguished Professor of Pediatric Cardiology

1. Pediatric hypertension and hyperlipidemia
2. Clinical trials in children with heart disease
3. Thrombosis in patients with congenital heart disease
4. Enzyme replacement in Pompe disease
5. Infective endocarditis

Eric Leo Eisenstein

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, emergency, pulmonary, and vascular medicine and surgery. He also has conducted health technology evaluations making use of innovative research methods designed to better understand key relationships in observation (non-randomized) patient data. This work has included evaluations of the long-term clinical outcomes of coronary artery disease patients receiving drug-eluting vs. bare metal intracoronary stents, and how the use of clopidogrel changes those relationships. He also has conducted several studies assessing factors contributing to the costs of and evaluating different design considerations for multi-center randomized clinical trials.

In addition to his working in traditional health technology evaluation, Dr. Eisenstein has an interest in evaluating information technologies as interventions in health care systems. In this regard, he has collaborated in the design and conduct of large-scale, randomized clinical trials to evaluate clinical decision support systems. The research objective in these studies has been to develop methods for evaluating health information technologies in practice-based settings using a “tool kit” of inexpensive, yet highly scalable methods that make use of data sets created as a byproduct of normal clinical and administrative operations. The use of these evaluation methods has been demonstrated in four clinical trials that include care process, clinical, economic, and quality of life measurements.


Daniel Kelly Benjamin

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. Each of these trials is conducted under an Investigational New Drug (IND) application with guidance from the Food and Drug Administration for labeling.

The Pediatric Trials Network has directly impacted the healthcare of over 90% of American children.

Signature programs of the Network include clinical trials in premature, term infants, breast feeding mothers, and obese children. Over the past 10 years, Danny’s group has enrolled more premature infants, at more sites, in more clinical trials of off-patent anti-infectives under an IND than all other academic medical centers, pharmaceutical companies, and government agencies in the world, combined.

Danny is recognized by the National Institutes of Health as a premiere mentor and educator. His research program serves as a platform to train students and early career investigators. Danny’s group has a clinical research summer program for high school and college students that has a focus on trainees under-represented in medicine, and he is the primary mentor for medical students, residents, subspecialty fellows, and multiple junior faculty. He has been the primary or secondary mentor for 10 faculty who have received career development awards and who have then gone on to secure their own funding.

Danny's service to the community is expressed through his passion for coaching baseball. He has coached over 500 recreation league, travel league, and scholastic baseball games and he is the head coach of Smith Middle School, the 5-year reigning southern conference champions. Danny and his wife own a charitable non-profit that provides athletic and fitness opportunities for disadvantaged school-aged boys and girls.

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