Causal inference methods to assess safety upper bounds in randomized trials with noncompliance

摘要

BackgroundPremature discontinuation and other forms of noncompliance with treatment assignment can complicate causal inference of treatment effects in randomized trials. The intent-to-treat analysis gives unbiased estimates for causal effects of treatment assignment on outcome, but may understate potential benefit or harm of actual treatment. The corresponding upper confidence limit can also be underestimated.