TY - JOUR
T1 - International Consensus-Driven Recommendations for Patient-Reported Outcome Research Objectives in Early Phase Dose-Finding Oncology Trials
T2 - OPTIMISE-ROR
AU - Alger, Emily
AU - Aiyegbusi, Olalekan Lee
AU - Dueck, Amylou C.
AU - Minchom, Anna
AU - Pe, Madeline
AU - Peipert, John D.
AU - Snyder, Claire
AU - Symeonides, Stefan N.
AU - Wilson, Roger
AU - Basch, Ethan
AU - Qiao, Yu
AU - Bates, Susan E.
AU - Bulbeck, Helen
AU - Dean, Lizzie
AU - Di Maio, Massimo
AU - Hansen, Aaron R.
AU - Kholmanskikh, Olga
AU - Kobayashi, Ken
AU - Landers, Dónal
AU - Le Tourneau, Christophe
AU - Lee, J. Jack
AU - Ma, Brigette B.Y.
AU - Marshall, Lynley V.
AU - Patel, Sheetal
AU - Petrie, Joan
AU - Pond, Gregory R.
AU - Prior, Kieran
AU - Rantell, Khadija R.
AU - Reeve, John F.
AU - Solovyeva, Olga
AU - Wages, Nolan A.
AU - Weber, Harald A.
AU - Calvert, Melanie J.
AU - Yap, Christina
N1 - Publisher Copyright:
© 2026 by American Society of Clinical Oncology
PY - 2026/1
Y1 - 2026/1
N2 - PURPOSE – There 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.METHODS – Using 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.RESULTS – Consensus 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.CONCLUSION – This 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.
AB - PURPOSE – There 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.METHODS – Using 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.RESULTS – Consensus 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.CONCLUSION – This 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.
UR - https://www.scopus.com/pages/publications/105032008630
UR - https://www.scopus.com/pages/publications/105032008630#tab=citedBy
U2 - 10.1200/JCO-25-01625
DO - 10.1200/JCO-25-01625
M3 - Article
C2 - 41576310
AN - SCOPUS:105032008630
SN - 0732-183X
SP - 709
EP - 719
JO - Journal of Clinical Oncology
JF - Journal of Clinical Oncology
ER -