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Abstract:
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Randomized clinical trials (RCTs) are the gold standard for evaluating the benefits and risks of interventions. However RCTs often fail to provide the necessary evidence to inform medical decision-making. The important implications of these deficiencies are largely absent from discourse in medical research communities. Typical analyses of clinical trials involve intervention comparisons for each efficacy and safety outcome. Outcome-specific effects are tabulated and potentially systematically or unsystematically combined in benefit:risk analyses with the belief that such analyses inform the totality of effects on patients. However such approaches do not incorporate associations between outcomes of interest, suffer from competing risk challenges, and since efficacy and safety analyses are conducted on different analysis populations, the population to which these benefit:risk analyses apply, is unclear. This deficit can be remedied with more thoughtful benefit:risk evaluation with a pragmatic focus in future clinical trials. Critical components of this vision include: (i) using outcomes to analyze patients rather than patients to analyze outcomes, (ii) incorporating patient values.
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