Prior to 1997, and almost 30 years after Shirkey’s editorial1 that coined the phrase “therapeutic orphan,” the legislative and regulatory efforts to address the issue of labeling medications for the safe and effective use by children were, by and large, unsuccessful. With an estimated 75% of the drugs marketed in the United States lacking essential pediatric data,2 it would take a new legislative effort, first proposed in 1994 by Senator Nancy Kassebaum of Kansas, that incorporated both an incentive and a requirement for industry to conduct appropriate pediatric studies. In 1997, the Food and Drug Administration Modernization Act (FDAMA), which provided an additional 6 months of marketing exclusivity to industry as incentive, was signed into law, catalyzing the development and conduct of a range of pediatric clinical investigations.3 The FDAMA, the incentive, coupled with the Pediatric Final Rule, the requirement, were subsequently replaced by the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA), now in place for a decade. The Institute of Medicine’s 2012 report on the studies conducted under BPCA and PREA found that important information to guide clinical care for children was emerging from these studies, and noted a trend in improvement in the quality of studies and reporting of results over time.4
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