Improving Pediatric Drug Development: Challenges, Opportunities and Lessons Learned

Abstract

Pediatric drug development has been challenging for more than 5 decades. Two recent books; one a workshop report and the other a volume prepared from the perspective of the pharmaceutical industry offer modest hope that clinical therapeutics within this vulnerable population is beginning to be addressed. Both unfortunately focus predominantly on pediatric drug development within the US, however, parallel activity within the World Health Organization (WHO) offers hope that pediatric drug development will improve in both developed and developing countries (see the WHO website for more information http://www.who.int/mediacentre/factsheets/fs341/en/index.html, accessed 25 August 2010). The Institute of Medicine's (IOM) Forum on Drug Discovery, Development and Translation held a workshop in 2006 to explore issues in pediatric drug development. Topics addressed included; regulatory framework, challenges in developing and prescribing drugs, models for enhancing pediatric drug development and challenges and opportunities for the future. Several legislative activities have stimulated pediatric drug development in the United States beginning with the Food and Drug Administration Modernization Act, Best Pharmaceuticals for Children Act and Pediatric Research Equity Act. Despite the recent legislative, regulatory and research activity in the US and Europe, barriers to pediatric drug development are substantial, and the workshop identified many of them. See the following web sites for current information: US NIH: http://bpca.nichd.nih.gov/ FDA: http://www.fda.gov/ScienceResearch/SpecialTopics/PediatricTherapeuticsResearch/default.htm EMEA: http://www.ema.europa.eu/ema/index.jsp?curl=pages/special_topics/general/general_content_000302.jsp&murl=menus/special_topics/special_topics.jsp&mid=WC0b01ac058002d4ea DG Research: http://imi.europa.eu/index_en.html Published after the IOM report is a volume with potential solutions to some of the challenges identified by the IOM Forum. While pediatric pharmacology began in earnest in the late 1960s, the struggle to place drug development for infants, children, and youth on the same footing as adult drug development remains far from satisfactory. The editors of Pediatric Drug Development have created a volume which reviews earlier work in pediatric pharmacology while providing a resource for learning about pediatric drug development from a pharmaceutical industry perspective. They summarize the history of pediatric drug development, explore ethical aspects, describe regulatory environments, and then consider the traditional steps in drug development: preclinical evaluation, clinical studies across the diverse ages and developmental stages. The two final sections focus on manufacturing and case studies.