ArticleBMC medicine2023
Why estimands are needed to define treatment effects in clinical trials.
Article in BMC medicine, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 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
16 citing papers in PubMed.
- Constructing an Evidence-Based Validity Rationale for a Multi-component Endpoint in Heterogeneous Populations.Therapeutic innovation & regulatory science · 2026Article
- Different Clinical Questions Need Different Estimands.Therapeutic innovation & regulatory science · 2026Article
- Ten methodological aspects to look for when critically reading randomized-controlled trials.Intensive care medicine · 2026Article
- Adjusting for intercurrent events using Bayesian joint models for longitudinal outcomes in clinical trials.Contemporary clinical trials communications · 2026Article
- Mendelian Randomization Methods for Causal Inference: Estimands, Identification and Inference.Statistics in medicine · 2026Review
- ChatGPT as a Tool for Biostatisticians: A Tutorial on Applications, Opportunities, and Limitations.Statistics in medicine · 2025Article
- Review
- Review
- Article
- A Perspective on the Appropriate Implementation of ICH E9(R1) Addendum Strategies for Handling Intercurrent Events.Statistics in medicine · 2025Article
- Article
- Testing the Effectiveness of a Mobile Smartphone App Designed to Improve the Mental Health of Junior Physicians: Protocol for a Randomized Controlled Trial.JMIR research protocols · 2024Article
- Hospitalization Endpoint in Clinical Trials of Outpatient Settings: using Anti-SARS-COV-2 Therapy as an Example.Clinical epidemiology · 2024Review
- An explainable machine learning-based phenomapping strategy for adaptive predictive enrichment in randomized clinical trials.NPJ digital medicine · 2023Article
- Assessing safety and efficacy in subpopulations in Alzheimer's disease clinical trials: contextualizing representativeness.Alzheimer's & dementia (New York, N. Y.)Article
- 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
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundThe estimand for a clinical trial is a precise definition of the treatment effect to be estimated. Traditionally, estimates of treatment effects are based on either an ITT analysis or a per-protocol analysis. However, there are important clinical questions which are not addressed by either of these analyses. For example, consider a trial where patients take a rescue medication. The ITT analysis includes data after use of rescue, while the per-protocol analysis excludes these patients altogether. Neither of these analyses addresses the important question of what the treatment effect would have been if patients did not take rescue medication. MAIN TEXT: Trial estimands provide a broader perspective compared to the limitations of ITT and per-protocol analysis. Trial treatment effects depend on how events occurring after treatment initiation such as use of alternative medication or discontinuation of the intervention are included in the definition. These events can be accounted for in different ways, depending on the clinical question of interest.
conclusionThe estimand framework is an important step forward in improving the clarity and transparency of clinical trials. The centrality of estimands to clinical trials is currently not reflected in methods recommended by the Cochrane group or the CONSORT statement, the current standard for reporting clinical trials in medical journals. We encourage revisions to these guidelines.
Indexed as
Identifiers
What OpenQuestion holds
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.