FDA’s long-awaited draft guidance on "enrichment strategies" or "enriched studies" aims to help drug developers "exclude poor potential candidates and select those more likely to show a clinical benefit."
Center for Drug Evaluation and Research Deputy Director Bob Temple, MD, explained that the study designs are "potentially powerful strategies for the pharmaceutical industry because appropriate use of enrichment could result in smaller studies, shortened drug development times, and lower development costs." He said conducting clinical trials within a "patient population that has a larger than average response to treatment can greatly reduce the number of patients needed in the study."
For example, the FDA "approved the cystic fibrosis drug ivacaftor (Kalydeco) which works in just 4% of CF patients with a specific genetic abnormality." But if "all CF patients were included in a trial," Dr. Temple said an "effect would have been impossible to detect."
