New medicines — urgent need to assess outcomes in special groups

A few achievable strategies could improve translation of clinical research to practice

Randomised controlled trials (RCTs) provide the most internally valid method of evaluating new pharmaceuticals and, appropriately, provide the core evidence for marketing approval. However, certain patient groups (eg, older people, young people, people with comorbidities, people taking concomitant medications) are typically excluded, and RCTs are often too small and their duration too short to detect uncommon but clinically important adverse drug reactions (ADRs). Consequently, the translation of these internally valid results to the real world of everyday clinical practice is not a trivial task.1