ReviewBMJ open2021
Comprehensive review of statistical methods for analysing patient-reported outcomes (PROs) used as primary outcomes in randomised controlled trials (RCTs) published by the UK's
Review in BMJ open, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 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
10 citing papers in PubMed, 1 synthesis or guideline pooled it.
- PARP Inhibitor Maintenance After First-Line Chemotherapy in Advanced-Stage Epithelial Ovarian Cancer: A Systematic Review and Meta-Analysis.JAMA network open · 2025Pooled it
- Patient-Reported Outcomes Used for Regulatory Approval of Medicines Indicated for the Treatment of Dermatological Diseases Authorized in the European Union Between 1998 and 2024.Therapeutic innovation & regulatory science · 2026Article
- Comparison of statistical methods for the analysis of patient-reported outcomes (PROs), particularly the Short-Form 36 (SF-36), in randomised controlled trials (RCTs) using standardised effect size (SES): an empirical analysis.Health and quality of life outcomes · 2025Article
- Knowledge map of self-reported outcomes in patients with prostate cancer: a bibliometric analysis (2014-2023).Frontiers in urology · 2025Article
- Comparison of statistical methods for the analysis of patient-reported outcomes in randomised controlled trials: A simulation study.Statistical methods in medical research · 2024Article
- Application of Patient-Reported Outcome Measurements in Adult Tumor Clinical Trials in China: Cross-Sectional Study.Journal of medical Internet research · 2024Article
- Striving for affordable medicine: Lessons in price negotiation learned from the United Kingdom.Journal of managed care & specialty pharmacy · 2024Article
- Association analysis of self-reported outcomes with a validated subset.Statistics in medicine · 2024Article
- Endpoint selection and evaluation in hematology studies.Best practice & research. Clinical haematology · 2023Review
- The use of cognitive task analysis in clinical and health services research - a systematic review.Pilot and feasibility studies · 2022Review
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
objectivesTo identify how frequently patient-reported outcomes (PROs) are used as primary and/or secondary outcomes in randomised controlled trials (RCTs) and to summarise what statistical methods are used for the analysis of PROs.
designComprehensive review.
settingRCTs funded and published by the United Kingdom's (UK) National Institute for Health Research (NIHR) Health Technology Assessment (HTA) Programme. DATA SOURCES AND ELIGIBILITY: HTA reports of RCTs published between January 1997 and December 2020 were reviewed. DATA EXTRACTION: Information relating to PRO use and analysis methods was extracted. PRIMARY AND SECONDARY OUTCOME MEASURES: The frequency of using PROs as primary and/or secondary outcomes; statistical methods that were used for the analysis of PROs as primary outcomes.
resultsIn this review, 37.6% (114/303) of trials used PROs as primary outcomes, and 82.8% (251/303) of trials used PROs as secondary outcomes from 303 NIHR HTA reports of RCTs. In the 114 RCTs where the PRO was the primary outcome, the most used PRO was the Short-Form 36 (8/114); the most popular methods for multivariable analysis were linear mixed model (45/114), linear regression (29/114) and analysis of covariance (13/114); logistic regression was applied for binary and ordinal outcomes in 14/114 trials; and the repeated measures analysis was used in 39/114 trials.
conclusionThe majority of trials used PROs as primary and/or secondary outcomes. Conventional methods such as linear regression are widely used, despite the potential violation of their assumptions. In recent years, there is an increasing trend of using complex models (eg, with mixed effects). Statistical methods developed to address these violations when analysing PROs, such as beta-binomial regression, are not routinely used in practice. Future research will focus on evaluating available statistical methods for the analysis of PROs.
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.