Using negative controls to identify causal effects with invalid instrumental variables.

Many proposals for the identification of causal effects require an instrumental variable that satisfies strong, untestable unconfoundedness and exclusion restriction assumptions. In this paper, we show how one can potentially identify causal effects under violations of these assumptions by harnessing a negative control population or outcome. This strategy allows one to leverage subpopulations for whom the exposure is degenerate, and requires that the instrument-outcome association satisfies a certain parallel trend condition.

Generalizing the intention-to-treat effect of an active control from historical placebo-controlled trials: A case study of the efficacy of daily oral TDF/FTC in the HPTN 084 study.

In many clinical settings, an active-controlled trial design (e.g., a non-inferiority or superiority design) is often used to compare an experimental medicine to an active control (e.

Covariate adjustment in randomized controlled trials: General concepts and practical considerations.

There has been a growing interest in covariate adjustment in the analysis of randomized controlled trials in past years. For instance, the US Food and Drug Administration recently issued guidance that emphasizes the importance of distinguishing between conditional and marginal treatment effects. Although these effects may sometimes coincide in the context of linear models, this is not typically the case in other settings, and this distinction is often overlooked in clinical trial practice.

Ensuring valid inference for Cox hazard ratios after variable selection.

The problem of how to best select variables for confounding adjustment forms one of the key challenges in the evaluation of exposure effects in observational studies, and has been the subject of vigorous recent activity in causal inference. A major drawback of routine procedures is that there is no finite sample size at which they are guaranteed to deliver exposure effect estimators and associated confidence intervals with adequate performance. In this work, we will consider this problem when inferring conditional causal hazard ratios from observational studies under the assumption of no unmeasured confounding.