Academic Journal
Inclusive designs that allow the inclusion of a broader population in randomized controlled trials: An evaluation of randomization ratio and analysis strategy.
| Τίτλος: | Inclusive designs that allow the inclusion of a broader population in randomized controlled trials: An evaluation of randomization ratio and analysis strategy. |
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| Συγγραφείς: | Lee KM; Department of Biostatistics and Health Informatics, Institute of Psychiatry, Psychology and Neuroscience, King's College London, London, UK., Wang Z; Statistical Sciences Research Institute, University of Southampton, Southampton, England, UK., Emsley R; Department of Biostatistics and Health Informatics, Institute of Psychiatry, Psychology and Neuroscience, King's College London, London, UK.; King's Clinical Trials Unit, King's College London, London, UK., Stallard N; Warwick Clinical Trials Unit, Warwick Medical School, University of Warwick, Coventry, UK. |
| Πηγή: | Statistical methods in medical research [Stat Methods Med Res] 2026 Oct; Vol. 35 (10), pp. 2095-2115. Date of Electronic Publication: 2026 Aug 21. |
| Τύπος έκδοσης: | Journal Article |
| Γλώσσα: | English |
| Στοιχεία περιοδικού: | Publisher: SAGE Publications Country of Publication: England NLM ID: 9212457 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1477-0334 (Electronic) Linking ISSN: 09622802 NLM ISO Abbreviation: Stat Methods Med Res Subsets: MEDLINE |
| Imprint Name(s): | Publication: London : SAGE Publications Original Publication: Sevenoaks, Kent, UK : Edward Arnold, c1992- |
| Ιατρικοί όροι (MeSH): | Randomized Controlled Trials as Topic*/statistics & numerical data , Randomized Controlled Trials as Topic*/methods , Research Design* , Patient Selection*, Humans ; Computer Simulation ; Meta-Analysis as Topic ; Regression Analysis ; Random Allocation ; Models, Statistical |
| Περίληψη: | In most multi-arm randomized controlled trials, all participants need to be eligible to be randomized to all the arms. This means some participants are excluded from participating in multi-arm studies. We propose to relax this by considering a 'differential randomization' approach that allows the inclusion of participants who are eligible for some but not all arms, as in some innovative trial approaches. We refer to a multi-arm design that employs differential randomization as an 'inclusive design' because it maximizes participant inclusion. Considering superiority comparisons for a normal endpoint, we evaluate the performance of some analysis methods for a three-arm inclusive design by simulation studies. Methods include pairwise or overall regression analysis, an averaging approach that pools summary statistics using prevalence rate, and a meta-analysis type framework. We compare the statistical power when the inclusive design employs different treatment allocation schemes. We find that using equal allocation ratio within each subpopulation leads to a higher disjunctive power than using equal allocation across arms, when the many-to-one comparisons are analysed using the corresponding pairwise data separately as opposed to using all data in a single regression analysis. We find that differential covariate-outcome relations among patients can affect the property of the inference. |
| Competing Interests: | Declaration of conflicting interestThe authors declared no potential conflicts of interest with respect to the research, authorship, and/or publication of this article. |
| Contributed Indexing: | Keywords: Broad enrolment criteria; fixed effect; inclusive; meta-analysis; pairwise analysis; randomized controlled trials |
| Entry Date(s): | Date Created: 20260821 Date Completed: 20261008 Latest Revision: 20261009 |
| Update Code: | 20261010 |
| DOI: | 10.1177/09622802261475687 |
| PMID: | 42627367 |
| Βάση Δεδομένων: | MEDLINE |
| ISSN: | 1477-0334 |
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| DOI: | 10.1177/09622802261475687 |