IND

Origin

The requirement for an Investigational New Drug application in the United States was established by the 1962 Kefauver-Harris Amendments to the Federal Food, Drug, and Cosmetic Act. This legislative change was a direct response to the thalidomide tragedy, which highlighted the urgent need for regulatory oversight before a new drug is tested in humans. The IND process was designed to shift the focus from merely documenting a drug’s safety to actively requiring evidence of its quality and the rationale for its clinical use. This landmark regulation formalized the start of modern clinical research oversight.

Mechanism

The IND mechanism functions as a regulatory gatekeeper, placing a mandatory hold on clinical testing until the agency has reviewed the submission for safety and scientific merit. Once the application is submitted, the regulatory body has a defined period, typically 30 days, to raise any clinical or safety concerns, known as a clinical hold. If no objections are raised, the clinical investigation can proceed, allowing researchers to study the pharmacokinetics, safety, and initial efficacy of the new hormonal compound in human volunteers. This process continues throughout all phases of clinical development.