“Not at Present Indicated for Use in Pediatric Patients:” Pediatric Pharmaceutical Policy in the United States, 1933-1979

In 1972, Harry Shirkey's frustration finally boiled over. The pediatrician and pharmacist felt little closer to his long term goal of assuring that drugs used in children were both safe and effective. He asked a series of provocative questions in speeches, publications, and Capitol Hill testimony: “Was it intended that infants and children be singled out for neglect when two major pieces of twentieth century drug legislation, one in 1938 and one in 1962, were enacted?” Did Congress, he wondered, intend “only those who had passed puberty to benefit” from these statutes? He argued that it was long past the time for those under the age of 18 years to languish as “therapeutic orphans” (a term he had coined a few years earlier) denied their “pharmacological rights” through commonplace disclaimers on labels acknowledging the lack of data regarding the product's safety and efficacy for children. Whose job was it, he asked, to “speak” for children and fix this longstanding problem? This paper, part of a larger study on the history of children and pharmaceuticals in the United States since the 1930s, traces the political, legislative, and regulatory history of pharmaceuticals (therapeutic medicinal chemistry) in the pediatric population in the years between 1933 and 1979. Despite numerous attempts throughout the twentieth century, legislation mandating pharmaceutical safety and efficacy testing in children, the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) was not enacted until 2002. But the issues addressed by these early twenty-first century laws had long been acknowledged, not just by the Food and Drug Administration (FDA), but by pediatricians, parents, elected officials, and industry. This paper situates this history in the context of the larger societal debates during this era surrounding children's health care delivery, their “rights” in terms of research participation, and the renegotiation of decision-making authority between parents and physicians.