Abstract: Creating the "Therapeutic Orphan": Pediatric Pharmaceutical Policy in the United States, 1933-1979

Cynthia Connolly

Abstract

In 1972, pediatrician Harry Shirkey's frustration boiled over. The nation's leading advocate for stronger pediatric drug regulation felt stymied in his long-term goal of assuring that drugs used in children were safe and effective. He raised a provocative question: "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?" He argued that it was long past the time when it was considered acceptable for children to languish as "therapeutic orphans," denied their "pharmacological rights" through label disclaimers acknowledging the lack of pediatric safety and efficacy data. Whose job was it to fix the problem, he asked, industry, pediatricians, or the Food and Drug Administration (FDA)? This paper, part of a larger study on the history of children and pharmaceuticals in the United States since the 1930s, traces negotiations among the FDA, Congress, industry, health care providers, and parents in the years between 1933 and 1979 as new drugs poured on the market, the regulatory environment shifted dramatically, and the costs of drug development skyrocketed. It draws on numerous primary sources: FDA records, National Archives; United States Pharmacopeia records and archival holdings at the American Institute for the History of Pharmacy, both in Madison, Wisconsin; the Helen Taussig papers at Johns Hopkins; American Academy of Pediatrics' Committee on Drugs records in Elk Grove, Illinois; and oral histories drawn from industry, FDA, and clinicians. Findings are contextualized in the historiography of twentieth-century children's health care. The narrative is also situated in recent scholarship on the history of pharmaceuticals in the United States, little of which has focused on pediatrics.