Abstract
Through at least the mid-1990s, children were often referred to as ‘therapeutic orphans’ for whom many treatments were administered without the benefit of appropriate studies to guide drug labeling for dosing and other critical therapeutic decisions. At that time, there were no incentives for manufacturers to pursue such work, nor regulatory requirements to compel these studies. Congress addressed this by including an important provision titled the Best Pharmaceuticals for Children Act (BPCA) in the 1997 Food and Drug Administration Modernization and Accountability Act. This was complemented by another key piece of legislation, the Pediatric Research Equity Act (PREA) in 2003. The former Act and its successors created an incentive for firms to study on-patent drugs in pediatric populations by extending the market exclusivity of a medicine by 6 months. The latter was a requirement that provided the US FDA with the authority to require studies of drugs in children if an adult indication also occurs in children.
In the current paper, we consider the effects of both pieces of legislation in terms of the health, societal, and economic benefits they have likely imparted and will continue to provide in the future. We conclude that the gains have been substantial — both in terms of safer and more effective use of medicines in children and in terms of new research that has been incentivized by the BPCA exclusivity provision. We estimate the gross economic benefits from the latter alone to be approximately $US360 billion.
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Acknowledgements
We wish to dedicate this manuscript to the memory of our friend and colleague, John Vernon, who died suddenly on 19 June 2012 during the final stages of publication. John was enthusiastic about this research and the importance of medicines development for children. His intellectual curiosity, creativity, and good nature will be missed.
The authors gratefully acknowledge David Ceryak and Joe Johnston for their insightful suggestions and comments.
Eli Lilly and Company provided funding to support the research for this article.
John Vernon received a research grant for work related to this manuscript in 2011 but not for developing this paper. He has also received consulting fees in years past from the pharmaceutical industry but this work was not related to the subject matter in this paper. Albert Allen, Scott Shortenhaus, and Mark Mayer are employees and stock holders of Eli Lilly and Company. Albert Allen is a member of the Secretary’s Advisory Committee on Human Research Protections. Joseph Golec has received payment from Pfizer for research on a topic unrelated to the one in this paper.
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Vernon, J.A., Shortenhaus, S.H., Mayer, M.H. et al. Measuring the Patient Health, Societal and Economic Benefits of US Pediatric Therapeutics Legislation. Pediatr Drugs 14, 283–294 (2012). https://doi.org/10.1007/BF03262234
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DOI: https://doi.org/10.1007/BF03262234