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 of single-patient trials would also generate a continuously expanding database of prospectively documented effectiveness and safety outcomes that would support population and subgroup risk/benefit assessments, leading to improved prescribing information.
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