Institute of Cancer Research Leads International Effort to Define Use of Patient-Reported Outcomes in Early-Stage Cancer Trials

Institute of Cancer Research Leads International Effort to Define Use of Patient-Reported Outcomes in Early-Stage Cancer Trials

(IN BRIEF) The Institute of Cancer Research, London, has led the development of OPTIMISE-ROR, the first structured guidance outlining how patient-reported outcomes should be used in early-stage cancer dose-finding trials. Published in the Journal of Clinical Oncology, the recommendations provide clear objectives for collecting and analysing PRO data so that patient experiences can meaningfully inform dose decisions. The framework is expected to improve the quality, transparency, and clinical relevance of early-phase oncology trials while supporting more patient-centred drug development.

(PRESS RELEASE) LONDON, 27-Jan-2026 — /EuropaWire/ — The Institute of Cancer Research, London (ICR), London, has led new international research that sets out clear, practical guidance on how patient-reported outcomes (PROs) can be more effectively used in early-stage cancer drug trials. The recommendations aim to ensure that patients’ real-world experiences of treatment meaningfully inform critical dose-finding decisions during the earliest phases of drug development.

Early-phase oncology trials are designed to identify safe and effective doses of new treatments, yet until now they have lacked clear standards for defining the role and objectives of PROs. To address this gap, researchers at the ICR coordinated an international effort to develop foundational guidance that clarifies how PRO data should be collected, analysed, and applied in dose-finding trials.

The study introduces OPTIMISE-ROR (incOrporating PaTIent-reported outcoMes In doSE-finding trials – Research Objectives Recommendations), the first structured framework to define critical PRO research objectives specifically for early-stage dose-finding oncology trials. The recommendations were developed through a large international consensus process involving clinicians, statisticians, regulators, and patient advocates, ensuring that multiple perspectives were reflected.

Published in the Journal of Clinical Oncology, the research was supported through a PhD studentship awarded to Emily Alger, a doctoral researcher in the Early Phase and Adaptive Trials Group within the ICR Clinical Trials and Statistics Unit. The work also received infrastructure support from Cancer Research UK and the Experimental Cancer Medicine Centre, as well as funding through the Medical Research Council and the National Institute for Health and Care Research Trials Methodology Research Partnership.

Patient-reported outcomes capture how patients experience treatment, including side effects, symptoms, and overall quality of life. Although PROs are increasingly included in early-phase trials, previous studies have shown that objectives are often poorly defined or limited to broad statements such as “assessing quality of life.” This lack of clarity has reduced the clinical relevance of PRO findings and limited their influence on dose selection and later-stage trial design.

The OPTIMISE-ROR guidance addresses these shortcomings by setting out a minimum set of clearly defined, stakeholder-informed objectives. The recommendations focus on three key dimensions of treatment tolerability: overall side-effect impact, patient-reported symptoms, and health-related quality of life. They emphasise analysing PRO data over time and across dose levels, and distinguishing between descriptive objectives and those intended to support formal statistical testing.

Importantly, the guidance highlights how PRO data can directly inform dose escalation, dose optimisation, and the selection of doses for subsequent trial phases. By clarifying how and why PROs are collected, the framework is designed to strengthen clinical interpretation, improve transparency, and promote more patient-centred decision-making in cancer drug development.

Senior author Professor Christina Yap, Professor of Clinical Trials Biostatistics and Group Leader of the ICR-CTSU Early Phase and Adaptive Trials Group, said: “If PROs are to genuinely inform early decision-making, we need to be clear about why we are collecting them and how they will be used. This guidance provides a practical starting point for doing that.”

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SOURCE: The Institute of Cancer Research