Advances in single-patient trials for drug treatment optimization and risk management

Experts in evidence-based medicine consider single-patient trials or "n-of-1" studies to be the highest form of evidence for making individual-patient drug treatment decisions. Single-patient trial-based risk management programs,,for chronic care drugs with recognized safety risks, would improve risk/benefit by more reliably identifying individual patients who do not receive benefit and/or who experience undue short-term adverse events. The widespread use a of single-patient trials would also generate continuously expanding database of prospectively documented effectiveness and safety outcomes comes that would support population and subgroup risk/benefit assessments, leading to improved prescribing information.