Assuring the Safety and Efficacy of Therapies

A brief history and discussion of the evolution of methods for testing and evaluating medical and surgical therapies demonstrates that sophisticated means for such evaluation are presently at hand, primarily in the form of the randomized controlled clinical trial. This article discusses problems in evaluating surgical procedures, increasing drug use, and adverse reactions to therapies, indicating limitations in current practices and pointing out where greater acceptance and application of the controlled clinical trial could improve the overall safety and efficacy of therapies. The example of a minor epidemic of drug-induced cancer is described to illustrate the use of the epidemiologic method in detecting adverse effects and for its implications for the development of a more responsible public policy to prevent such occurrences. Advances in the epidemiologic detection of adverse effects, coupled with the development and application of the controlled clinical trial have provided the necessary evidence on the basis of which new governmental regulations for ensuring safety and efficacy have been developed. It is argued that such evidence suggests that regulation should be expanded to require that manufacturers of new drugs introduced into the market demonstrate, in addition to safety and efficacy, the relative efficacy of their products over existing formulations. It is also suggested that surgical procedures be subjected to forms of scrutiny similar to those to which drugs are now subjected. Finally, some current practices of drug advertising are critically reviewed, and a greater attention to the ecologic consequences of drugs and food additives is recommended.