ABSTRACT
Debate over access to experimental therapy began with passage of the Kefauver Amendments in 1962. These rules, promulgated during the thalidomide scare, ordered the US Food and Drug Administration (FDA) not only to regulate the safety and claims made about drugs, as previously, but also to rule upon their effectiveness. In retrospect, the requirement for proving effectiveness seems oddly unrelated to the events that led to it, since thalidomide presented a question of safety, not efficacy. Thalidomide was no more available under the old rules than under the new ones. Yet we have lived with the Kefauver sanctions for the past 26 years. Supporters say the result is the world’s safest and most effective pharmacy. Critics contend that the sanctions have also stymied development of new drugs in this country and have made Americans second-class citizens in the world’s waiting lines for new therapies.
