ArticleStatistics in medicine2022
Translating questions to estimands in randomized clinical trials with intercurrent events.
Article in Statistics in medicine, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
12 citing papers in PubMed.
- Article
- Comparative Weight Change With Initiation and Adherence to Common Medications for Type 2 Diabetes.Obesity (Silver Spring, Md.) · 2025Article
- Hazards Constitute Key Quantities for Analyzing, Interpreting and Understanding Time-to-Event Data.Biometrical journal. Biometrische Zeitschrift · 2025Review
- Survivor Average Causal Effects for Continuous Time: A Principal Stratification Approach to Causal Inference With Semicompeting Risks.Biometrical journal. Biometrische Zeitschrift · 2025Article
- An Example to Illustrate Randomized Trial Estimands and Estimators.The American statistician · 2025Article
- Medication-Induced Weight Change Across Common Antidepressant Treatments : A Target Trial Emulation Study.Annals of internal medicine · 2024Observational
- Re-evaluating the mythical divide between traditional and novel cardiovascular risk factors in rheumatoid arthritis.RMD open · 2024Article
- Causal inference with recurrent and competing events.Lifetime data analysis · 2024Article
- A causal roadmap for generating high-quality real-world evidence.Journal of clinical and translational science · 2023Review
- Estimating causal effects in the presence of competing events using regression standardisation with the Stata command standsurv.BMC medical research methodology · 2022Article
- Translating questions to estimands in randomized clinical trials with intercurrent events.Statistics in medicine · 2022Article
- Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Intercurrent (post-treatment) events occur frequently in randomized trials, and investigators often express interest in treatment effects that suitably take account of these events. Contrasts that naively condition on intercurrent events do not have a straight-forward causal interpretation, and the practical relevance of other commonly used approaches is debated. In this work, we discuss how to formulate and choose an estimand, beyond the marginal intention-to-treat effect, from the point of view of a decision maker and drug developer. In particular, we argue that careful articulation of a practically useful research question should either reflect decision making at this point in time or future drug development. Indeed, a substantially interesting estimand is simply a formalization of the (plain English) description of a research question. A common feature of estimands that are practically useful is that they correspond to possibly hypothetical but well-defined interventions in identifiable (sub)populations. To illustrate our points, we consider five examples that were recently used to motivate consideration of principal stratum estimands in clinical trials. In all of these examples, we propose alternative causal estimands, such as conditional effects, sequential regime effects, and separable effects, that correspond to explicit research questions of substantial interest.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.