Research Profile of Thirty Recent Therapies for the Treatment of Life-Threatening and Severely-Debilitating Illnesses

摘要

The report describes the research and development process for 30 vitally important therapies (VITs) that treat life-threatening and severely-debilitating illnesses. Characteristics of the therapies, descriptions of the pivotal clinical trials, and the durations of the research periods are included. Over one-half (17) of these therapies were originally developed abroad although recent (since 1985) approvals have been predominantly from the United States. Only four pivotal trials were completed with double-blind, placebo-control protocols; most trials used historical or alternate controls. Twelve therapies were approved by FDA on the basis of trials that did not use specific survival/mortality end-points, but surrogate measures instead. The median pivotal study included 222 subjects, although the median cardiovascular trial included 1,395 subjects. The average size of these trials has decreased since 1980 although the number of safety reports has remained constant.