Guidance for defining outcomes in clinical trials
Develops a structured approach for defining clinical-trial outcomes completely and consistently before trials begin.
Published in PLOS Medicine on 21 October 2026 as Guidelines and Guidance. Earlier MetaArXiv versions are retained below.
Open-science resources
- Open access: Published article (open access)
- Preprint: Preprint, version 2
- Preprint: Preprint
- Shared materials: Supporting information
Resource verification & source notes
- Published article (open access): Full final publisher JATS inspected; CC BY 4.0 license, final DOI and complete 19-author byline verified.
- Preprint, version 2: OSF version 2 and primary manuscript retrieved; Crossref confirms is-version-of relationship.
- Preprint: Public preprint record in the saved source catalogue. No final journal article identified as of retrieval.
- Supporting information: All six publisher supplementary DOCX files were downloaded and document XML inspected: registry fields, worked outcome examples, framework comparison, metric definitions, time-point guidance and registry-field table. These are materials, not shared analysis code or a study dataset.
Publication details
Research themes
Author affiliations & identifiers
- Xiangji Ying: 1 Department of Epidemiology, University of North Carolina Gillings School of Global Public Health, Chapel Hill, North Carolina, United States of America · ORCID https://orcid.org/0000-0002-1713-3788
- Tianjing Li: 2 Department of Ophthalmology, University of Colorado Anschutz Medical Campus, Aurora, Colorado, United States of America
- Joanne E. McKenzie: 3 Methods in Evidence Synthesis Unit, School of Public Health and Preventive Medicine, Monash University, Melbourne, Victoria, Australia · ORCID https://orcid.org/0000-0003-3534-1641
- Matthew J. Page: 3 Methods in Evidence Synthesis Unit, School of Public Health and Preventive Medicine, Monash University, Melbourne, Victoria, Australia · ORCID https://orcid.org/0000-0002-4242-7526
- Kiran Ninan: 1 Department of Epidemiology, University of North Carolina Gillings School of Global Public Health, Chapel Hill, North Carolina, United States of America · ORCID https://orcid.org/0000-0002-8085-6751
- Jean-Pierre Oberste: 1 Department of Epidemiology, University of North Carolina Gillings School of Global Public Health, Chapel Hill, North Carolina, United States of America · ORCID https://orcid.org/0009-0003-2075-5267
- Colby J. Vorland: 4 Department of Epidemiology and Biostatistics, Indiana University School of Public Health-Bloomington, Bloomington, Indiana, United States of America · ORCID https://orcid.org/0000-0003-4225-372X
- Andrew W. Brown: 5 Department of Biostatistics, University of Arkansas for Medical Sciences, Little Rock, Arkansas, United States of America; 6 Arkansas Children’s Research Institute, Little Rock, Arkansas, United States of America · ORCID https://orcid.org/0000-0002-1758-8205
- Riaz Qureshi: 2 Department of Ophthalmology, University of Colorado Anschutz Medical Campus, Aurora, Colorado, United States of America · ORCID https://orcid.org/0000-0002-6670-4874
- Nicholas J. DeVito: 7 Nuffield Department of Primary Care Health Sciences, University of Oxford, Oxford, United Kingdom · ORCID https://orcid.org/0000-0001-8286-1995
- Ian J. Saldanha: 8 Department of Epidemiology, Johns Hopkins Bloomberg School of Public Health, Baltimore, Maryland, United States of America · ORCID https://orcid.org/0000-0002-2062-3382
- Martin Offringa: 9 Child Health Evaluative Sciences, The Hospital for Sick Children Research Institute, Toronto, Ontario, Canada · ORCID https://orcid.org/0000-0002-4402-5299
- Nancy J. Butcher: 9 Child Health Evaluative Sciences, The Hospital for Sick Children Research Institute, Toronto, Ontario, Canada; 10 Department of Psychiatry, University of Toronto, Toronto, Ontario, Canada · ORCID https://orcid.org/0000-0002-5152-0108
- Sirui Zhang: 11 Department of Epidemiology, School of Public Health, Brown University, Providence, Rhode Island, United States of America · ORCID https://orcid.org/0009-0002-8410-436X
- Phi-Yen Nguyen: 3 Methods in Evidence Synthesis Unit, School of Public Health and Preventive Medicine, Monash University, Melbourne, Victoria, Australia · ORCID https://orcid.org/0000-0002-0476-3385
- Joe Menke: 12 School of Information Sciences, University of Illinois Urbana-Champaign, Champaign, Illinois, United States of America · ORCID https://orcid.org/0000-0002-7374-7549
- Veronica Ka Wai Lai: 9 Child Health Evaluative Sciences, The Hospital for Sick Children Research Institute, Toronto, Ontario, Canada · ORCID https://orcid.org/0000-0003-3593-9141
- Halil Kilicoglu: 12 School of Information Sciences, University of Illinois Urbana-Champaign, Champaign, Illinois, United States of America · ORCID https://orcid.org/0000-0003-3987-9393
- Evan Mayo-Wilson: 1 Department of Epidemiology, University of North Carolina Gillings School of Global Public Health, Chapel Hill, North Carolina, United States of America · ORCID https://orcid.org/0000-0001-6126-2459
Versions & associated materials
These items belong to this research work. Their individual titles, author lists, and source identifiers are preserved below.