Abstract 3624: The Economic Return of Performing Pediatric Anti-Hypertensive Drug Clinical Trials
Background: Congress has authorized the US Food and Drug Administration (FDA) to provide industry sponsors a 6 month extension of drug marketing rights under the pediatric exclusivity provision if FDA-requested pediatric drug trials are conducted. As a result, the pharmacokinetics, safety and efficacy of over 126 drugs used in children ages 0 –17 years have been evaluated since 1997. We sought to determine the cost of performing pediatric hypertension trials and the subsequent economic return to industry sponsors from 6 additional months of marketing exclusivity.
Methods: We evaluated 9 orally administered anti-hypertensive drugs submitted to the FDA under the pediatric exclusivity provisions between 1997 and 2002. Key elements of the clinical trial design and operations were obtained and the costs of performing the studies were estimated and converted into estimates of after- tax cash outflow. Market sales were obtained, converted into after-tax inflows based on 6 months of additional patent protection and net economic return was determined for each drug.
Results: An average of 2 studies was performed per drug, including at least one pharmacokinetics study and a single study evaluating the safety and efficacy of the drug. The median cost of completion for a pharmacokinetic trial was $650,000 (range: $440,000 to $1.4M). The median cost of performing safety and efficacy trials for these agents was approximately $3.2 million (range: $1.6M to $9.1M). The most significant predictor of the cost of performing a trial was the number of patients enrolled. The ratio of net economic return to cost was high with a median net economic return ratio of 17 (range: 4 to 64.7).
Conclusions: The pediatric exclusivity program has been successful in encouraging drug studies in children. Our findings suggest that within a cohort of anti-hypertensive drugs, the pediatric exclusivity program has generated profitable returns to pharmaceutical companies and is an effective measure for improving knowledge of the safety, efficacy and pharmacokinetics of drugs in children.