Evidence mapPaperPMID 41576310Full record

ArticleJournal of clinical oncology : official journal of the American Society of Clinical Oncology2026

International Consensus-Driven Recommendations for Patient-Reported Outcome Research Objectives in Early Phase Dose-Finding Oncology Trials: OPTIMISE-ROR.

Emily Alger, Olalekan Lee Aiyegbusi, Amylou C Dueck, Anna Minchom, Madeline Pe, John D Peipert, Claire Snyder, Stefan N Symeonides, Roger Wilson, Ethan Basch and 24 more

Abstract readConsensus Statement
In one paragraph

Article in Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
field-weighted citation impact
1 · What the graph read from 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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

4 citing papers in PubMed.

  1. Article
  2. Generalized Pairwise Comparisons in Dose Optimization Oncology Trials: Beyond Safety to Multi-outcome Dose Selection.Clinical cancer research : an official journal of the American Association for Cancer Research · 2026
    Article
  3. Article
  4. Advancing oncology innovation under persistent constraints.The Lancet regional health. Europe · 2026
    Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

34 authors.

Emily AlgerClinical Trial and Statistics Unit, Institute of Cancer Research, London, United Kingdom.ORCID 0000-0002-5378-7439
Olalekan Lee AiyegbusiCentre for Patient-Reported Outcomes, University of Birmingham, Birmingham, United Kingdom.ORCID 0000-0001-9122-8251
Amylou C DueckMayo Clinic, Scottsdale, AZ.ORCID 0000-0002-9912-1085
Anna MinchomRoyal Marsden Hospital/Institute of Cancer Research, London, United Kingdom.ORCID 0000-0002-9339-7101
Madeline PeEuropean Organisation for Research and Treatment of Cancer, Brussels, Belgium.
John D PeipertCentre for Patient-Reported Outcomes, University of Birmingham, Birmingham, United Kingdom.
Claire SnyderJohns Hopkins School of Medicine, Baltimore, MD.ORCID 0000-0001-8952-4561
Stefan N SymeonidesEdinburgh Cancer Centre, NHS Lothian, Edinburgh, United Kingdom.ORCID 0000-0002-9892-9314
Roger WilsonCancer Research Advocates Forum UK, Stevenage, United Kingdom.ORCID 0000-0002-6043-7306
Ethan BaschThe University of North Carolina at Chapel Hill, Chapel Hill, NC.ORCID 0000-0003-3813-9318
Yu QiaoClinical Trial and Statistics Unit, Institute of Cancer Research, London, United Kingdom.ORCID 0009-0002-3615-0177
Susan E BatesColumbia University Irving Medical Center, New York, NY.ORCID 0000-0001-6708-0330
Helen BulbeckBrainstrust-The Brain Cancer People, Cowes, United Kingdom.ORCID 0000-0002-6775-5251
Lizzie DeanCancer Research Advocates Forum UK, Stevenage, United Kingdom.
Massimo Di MaioDepartment of Oncology, University of Turin, AOU Città della Salute e della Scienza di Torino, Turin, Italy.ORCID 0000-0001-8906-3785
Aaron R HansenDivision of Cancer Services, Princess Alexandra Hospital, Brisbane, Australia.ORCID 0000-0002-2363-8707
Olga KholmanskikhFederal Agency for Medicines and Health Products, Brussel, Belgium.ORCID 0000-0002-9530-356X
Ken KobayashiSmall Woods Biopharma Consulting, LLC, San Diego, CA.ORCID 0000-0002-2904-5435
Dónal LandersEarly Clinical Development, Dukes Street Bio Ltd, London, United Kingdom.ORCID 0000-0001-8376-9779
Christophe Le TourneauDepartment of Drug Development and Innovation (D3i), Institut Curie, Paris-Saclay University, Paris, France.
J Jack LeeUniversity of Texas MD Anderson Cancer Center, Houston, TX.ORCID 0000-0001-5469-9214
Brigette B Y MaState Key Laboratory of Translational Oncology, Department of Clinical Oncology, Phase 1 Clinical Trial Centre, Sir YK Pao Centre for Cancer, Hong Kong Cancer Institute, The Chinese University of Hong Kong, Hong Kong, China.ORCID 0000-0003-4802-1102
Lynley V MarshallChildren & Young People's Unit, The Royal Marsden NHS Foundation Trust, London, United Kingdom.ORCID 0000-0002-6537-2118
Sheetal PatelGenentech, San Francisco, CA.
Joan PetrieCanadian Cancer Trials Group (CCTG), Kingston, ON, Canada.ORCID 0009-0007-0996-7081
Gregory R PondMcMaster University, Hamilton, ON, Canada.ORCID 0000-0003-1033-0882
Kieran PriorCancer Research UK, London, United Kingdom.
Khadija R RantellMedicines and Healthcare Products Regulatory Agency, London, United Kingdom.
John F ReeveCancer Research Advocates Forum UK, Stevenage, United Kingdom.
Olga SolovyevaClinical Trial and Statistics Unit, Institute of Cancer Research, London, United Kingdom.ORCID 0000-0001-7828-4099
Nolan A WagesDepartment of Biostatistics, Virginia Commonwealth University, Richmond, VA.ORCID 0000-0002-2190-4864
Harald A WeberPfizer AG, Zug, Switzerland.
Melanie J CalvertCentre for Patient-Reported Outcomes, University of Birmingham, Birmingham, United Kingdom.
Christina YapClinical Trial and Statistics Unit, Institute of Cancer Research, London, United Kingdom.ORCID 0000-0002-6715-2514

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

purposeThere is growing scientific interest in incorporating patient-reported outcomes (PROs) in early phase dose-finding oncology trials (DFOTs) to assess tolerability, inform dose selection, and guide later stage trial design. However, research indicates that PRO objectives in DFOTs are often unclear. The Incorporating Patient-Reported Outcomes in Dose-Finding Trials-Research Objectives Recommendations (OPTIMISE-ROR) project was established to support trialists to effectively incorporate PROs into DFOTs.

methodsUsing the Enhancing Quality and Transparency of Health Research (EQUATOR) Network's methodological framework, guideline development included the following: (1) a methodological review of published DFOTs incorporating PROs; (2) candidate item generation, refined through expert consultation; (3) a two-round international multistakeholder Delphi survey (N = 109 in Round 1 [October 2024]; N = 96 in Round 2 [December 2024]); and (4) an independently chaired virtual consensus meeting (N = 31; January 2025) where multidisciplinary, international experts reviewed and voted to finalize items for inclusion.

resultsConsensus was reached on six recommendations emphasizing three core PRO tolerability concepts: overall side effect impact, symptomatic adverse events, and overall health-related quality of life. The integration of PROs to inform final dose recommendations in dose escalation and optimization trials should be considered, regardless of trial design. The recommendations highlight the importance of PRO data analysis over time and across dose levels, defining PRO research objectives as descriptive or statistically powered, and assessing PRO-related end points to guide end point selection for subsequent studies.

conclusionThis foundational guidance outlines key PRO research objectives in DFOTs. By facilitating the systematic integration of PROs, this guidance supports the utilization of patient-centered evidence for the tolerability and efficacy assessment of therapies to inform dose escalation, optimization, and regulatory evaluation-ultimately contributing to the development of safer, more effective therapies.

Indexed as

Antineoplastic AgentsClinical Trials as TopicNeoplasmsPatient Reported Outcome MeasuresResearch DesignDelphi TechniqueDose-Response Relationship, DrugHumansMaximum Tolerated DoseAntineoplastic Agents

Identifiers

PMID41576310
PMCPMC12959595

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

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 Socratic graph.