studies that are typical for common diseases (e.g., by involving many fewer research participants).

The biomedical research enterprise overlaps with many aspects of medical product development. However, the latter typically is viewed as building on the discoveries of basic research and focusing on the preclinical and clinical studies necessary to demonstrate safety and efficacy as required for FDA to authorize the marketing of drugs and certain medical devices. The phrase research and development is commonly used for this spectrum of activity, which is usually undertaken by commercial firms.

Efficacy and Effectiveness

The achievement of desired results in controlled clinical studies (efficacy) is not the same as the achievement of desired results in actual clinical practice (effectiveness). After a product enters clinical use, problems may emerge that were not evident in clinical testing. Although FDA statutes and regulations use the term effectiveness to describe positive results reported in clinical trials, FDA review documents often employ the term efficacy rather than effectiveness in discussing clinical data used in approving a new drug.

When it approves a drug or medical device for marketing, FDA may require the sponsor to undertake postmarketing studies to provide additional evidence of safety or effectiveness or both. This report focuses on research to demonstrate safety and efficacy through the stage of clinical testing prior to FDA approval of a product.



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