New research presents clear guidelines on how patient-reported outcomes (PROs) can be used more effectively in early-stage cancer drug trials, helping ensure that patient experiences meaningfully inform treatment decisions around dosing.

The Institute of Cancer Research, London, led an international effort to develop practical recommendations for incorporating PROs into early phase dose-finding oncology trials. These trials are designed to identify safe and effective doses of new treatments, but until now, they have lacked clear standards for defining PRO research objectives.

The study introduces OPTIMISE-ROR (incOrporating PaTIent-reported outcoMes In doSE-finding trials–Research Objectives Recommendations), the first foundational guidance to outline critical PRO objectives for dose-finding trials. The recommendations were developed through a large international consensus meeting involving clinicians, statisticians, regulators and patient advocates.

Published in the Journal of Clinical Oncology, the work was supported through a PhD studentship awarded to Emily Alger, a PhD student in the Early Phase and Adaptive Trials Group in the Clinical Trials and Statistics Unit at the ICR (ICR-CTSU), as part of the Medical Research Council and the National Institute for Health and Care Research Trials Methodology Research Partnership. Programme infrastructure funding from Cancer Research UK and the Experimental Cancer Medicine Centre also supported the study.

Why patient-reported outcomes matter

PROs capture how patients themselves experience treatment, including the impact of side effects, their symptoms and overall quality of life. While PROs are being increasingly implemented in early phase trials, previous research has shown that many studies either do not clearly define PRO objectives or rely on vague statements such as ‘assessing quality of life’.

As a result, trial reports often fail to demonstrate the clinical relevance of PRO findings, limiting their usefulness for guiding dose decisions or informing later-stage studies.

The OPTIMISE-FOR project was established to address this gap by providing trial teams with a minimum set of clear, stakeholder-informed recommendations to support the meaningful use of PROs in early-stage cancer trials.

Clear recommendations for dose-finding trials

Using a structured international survey followed by a consensus meeting, the research team agreed on six core recommendations that focus on three key aspects of treatment tolerability – overall side effect impact, patient-reported symptoms and overall health-related quality of life.

The guidance emphasises the importance of analysing PRO data over time and across dose levels, and of defining whether PRO objectives are descriptive or designed to support formal statistical testing. Importantly, it also highlights how PRO data can be used to inform dose escalation, dose optimisation and the final dose selected for later-stage trials.

Supporting better decision-making

In the short term, OPTIMISE-ROR provides trialists with a practical framework to systematically integrate PROs into dose-finding trials, making better use of the early insights data can offer.

First author Emily Alger said: “In the longer term, we expect the guidance to promote more patient-centred approaches to assessing treatment tolerability and effectiveness. We also foresee it strengthening the clinical interpretation of PRO findings and improving the quality and transparency of reporting in early phase trials.

“Ultimately, our work supports higher-quality research while helping ensure that the patient experience plays a meaningful role 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.”