August 15, 2017, Vol 318, No. 7, Pages 587-676
Balancing the Need for Access With the Imperative for Empirical Evidence of Benefit and Risk
Robert M. Califf, MD
When people use medical products, they generally expect that the benefits of doing so will outweigh the risks in terms of living longer, feeling better, or improving their functional status. However, most drugs and biologics and many medical devices that enter early evaluation in humans are found either to have no benefit or to have risks that outweigh the observed benefits when proper clinical trials are conducted. The complexity and high failure rates of medical product development have appropriately led to a regulatory system that requires compelling evidence of safety and efficacy from clinical trials for drugs, biologics, and high-risk devices before they are allowed on the market.