ArticleContemporary clinical trials2021
Why ITT analysis is not always the answer for estimating treatment effects in clinical trials.
Article in Contemporary clinical trials, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers, 1 of them a synthesis that pooled it.
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, 1 synthesis or guideline pooled it.
- Evaluating how clear the questions being investigated in randomised trials are: systematic review of estimands.BMJ (Clinical research ed.) · 2022Pooled it
- The effectiveness of microlearning-based education on medication adherence and health literacy in elderly individuals: a randomized controlled trial.Scientific reports · 2025Trial
- Comparing Different As-Treated Approaches: A Methodological Study Using Real-World Data on Psoriasis Treatments.Pharmacoepidemiology and drug safety · 2026Article
- A randomized controlled trial comparing Mindfulness and Heartfulness meditation programmes on stress, anxiety, depressive symptoms and wellbeing among distance-learning postgraduates.Frontiers in psychology · 2026Article
- Using the ICH estimand framework to improve the interpretation of treatment effects in internet interventions.NPJ digital medicine · 2025Review
- A Perspective on the Appropriate Implementation of ICH E9(R1) Addendum Strategies for Handling Intercurrent Events.Statistics in medicine · 2025Article
- Statistical methods for clinical trials interrupted by the severe acute respiratory syndrome-coronavirus-2 (SARS-CoV-2) pandemic: A review.Statistical methods in medical research · 2024Review
- Demystifying estimands in cluster-randomised trials.Statistical methods in medical research · 2024Article
- The estimands framework: a primer on the ICH E9(R1) addendum.BMJ (Clinical research ed.) · 2024Article
- Why estimands are needed to define treatment effects in clinical trials.BMC medicine · 2023Article
- Finerenone and effects on mortality in chronic kidney disease and type 2 diabetes: a FIDELITY analysis.European heart journal. Cardiovascular pharmacotherapy · 2023Article
- Marking 2-Years of New Thinking in Clinical Trials: The Estimand Journey.Therapeutic innovation & regulatory science · 2022Article
- A likely responder approach for the analysis of randomized controlled trials.Contemporary clinical trials · 2022Article
- The therapeutic effect of Taijiquan combined with acupoint pressing on the treatment of anxiety insomnia in college students: A study protocol for a randomized controlled trial.Frontiers in psychiatry · 2022Article
- Estimands-A Basic Element for Clinical Trials.Deutsches Arzteblatt international · 2021Article
- 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
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
For many years there has been a consensus among the Clinical Research community that ITT analysis represents the correct approach for the vast majority of trials. Recent worldwide regulatory guidance for pharmaceutical industry trials has allowed discussion of alternatives to the ITT approach to analysis; different treatment effects can be considered which may be more clinically meaningful and more relevant to patients and prescribers. The key concept is of a trial "estimand", a precise description of the estimated treatment effect. The strategy chosen to account for patients who discontinue treatment or take alternative medications which are not part of the randomised treatment regimen are important determinants of this treatment effect. One strategy to account for these events is treatment policy, which corresponds to an ITT approach. Alternative equally valid strategies address what the treatment effect is if the patient actually takes the treatment or does not use specific alternative medication. There is no single right answer to which strategy is most appropriate, the solution depends on the key clinical question of interest. The estimands framework discussed in the new guidance has been particularly useful in the context of the current COVID-19 pandemic and has clarified what choices are available to account for the impact of COVID-19 on clinical trials. Specifically, an ITT approach addresses a treatment effect that may not be generalisable beyond the current pandemic.
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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.