ESHS/HSS Annual Meeting

Transforming Systems for Better Clinical Evidence: Creating National Clinical Trial Frameworks in Mid-Twentieth Century America

• In Event: Public Health in the Americas

English Abstract

In 1947, Dr. Sidney Farber started a clinical trial to treat children with acute leukemia using antifolate drugs, producing temporary remissions and being the first to show that chemotherapy could change the course of childhood leukemia for the better. However, the trial also revealed that the tools available for therapeutic research at the time were not adequate to study rare pediatric diseases. Results were recorded as narrative case descriptions, patient selection depended on clinician judgment, and individual institutions treated too few children to allow comparison. Researchers in the United States and abroad faced these similar constraints, raising a broader question: how could systematic evidence be generated for the cases of rare pediatric diseases? Drawing on archival materials from the National Institutes of Health Office of History and Stetten Museum, this presentation will examine how the United States addressed mid-twentieth-century limitations in assessing drug efficacy and safety by building one of the earliest national infrastructures for systematic, statistically powerful evaluation of chemotherapy protocols. By which, the National Cancer Institute created the Cancer Chemotherapy National Service Center and multiple Task Forces, which set fixed eligibility criteria, standardized dosing schedules, formalized toxicity reporting, and defined remission in uniform terms. Investigators submitted data through staged review processes that organized early evidence on biological activity, formulation, and safety. Planning documents also referenced the European Group for Cancer Chemotherapy, suggesting awareness of parallel efforts abroad, though the depth of collaboration remains unclear. Altogether, these findings show how mid-twentieth-century national infrastructures reshaped the coordination and evaluation of clinical research and raise new questions about how similar challenges were addressed in Europe and around the world.

Author

• Khanh Bui, National Cancer Institute