# Guidelines for estimating causal effects in pragmatic randomized trials

arXiv: https://arxiv.org/abs/1911.06030 · HTML: https://mlsanity.com/paper/1911.06030/

## Key facts

- Cited by 1 papers in the map (data through 14 Oct 2026).
- First posted on arXiv 14 Nov 2019.

## Built on by (most cited first, 1 of 1)

- [Pragmatic Clinical Trials in the Rubric of Structural Causal Models](https://mlsanity.com/paper/2204.13782/) (2022)

## Abstract (arXiv)

Pragmatic randomized trials are designed to provide evidence for clinical decision-making rather than regulatory approval. Common features of these trials include the inclusion of heterogeneous or diverse patient populations in a wide range of care settings, the use of active treatment strategies as comparators, unblinded treatment assignment, and the study of long-term, clinically relevant outcomes. These features can greatly increase the usefulness of the trial results for patients, clinicians, and other stakeholders. However, these features also introduce an increased risk of non-adherence, which reduces the value of the intention-to-treat effect as a patient-centered measure of causal effect. In these settings, the per-protocol effect provides useful complementary information for decision making. Unfortunately, there is little guidance for valid estimation of the per-protocol effect. Here, we present our full guidelines for analyses of pragmatic trials that will result in more informative causal inferences for both the intention-to-treat effect and the per-protocol effect.