ICR Develops First Standardized Guidelines for Patient-Reported Outcomes in Early Cancer Drug Trials
核心洞察
The Institute of Cancer Research, London, has developed OPTIMISE-ROR, the first foundational guidance for incorporating patient-reported outcomes into early phase dose-finding cancer (搜索) trials.
The international consensus-based recommendations address six core areas focusing on treatment tolerability, side effect impact, and quality of life assessment across dose levels.
The guidelines aim to improve clinical interpretation of patient experience data and enhance decision-making for dose escalation and optimization in cancer (搜索) drug development.
The Institute of Cancer Research, London, has published the first standardized guidelines for incorporating patient-reported outcomes (PROs) into early-stage cancer (搜索) drug trials, addressing a critical gap in dose-finding studies that could significantly improve treatment development decisions.
The research team developed OPTIMISE-ROR (incOrporating PaTIent-reported outcoMes In doSE-finding trials–Research Objectives Recommendations), published in the Journal of Clinical Oncology, through an international consensus meeting involving clinicians, statisticians, regulators and patient advocates. The work was led by Emily Alger, a PhD student in the Early Phase and Adaptive Trials Group in the Clinical Trials and Statistics Unit at the ICR.
Addressing Critical Research Gaps
Early phase dose-finding oncology trials are designed to identify safe and effective doses of new treatments, but until now have lacked clear standards for defining PRO research objectives. 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," according to the research team. The OPTIMISE-FOR project was established to address this gap by providing trial teams with a minimum set of clear, stakeholder-informed recommendations.
Six Core Recommendations Framework
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 emphasizes the importance of analyzing PRO data over time and across dose levels, and of defining whether PRO objectives are descriptive or designed to support formal statistical testing. The recommendations also highlight how PRO data can be used to inform dose escalation, dose optimization and the final dose selected for later-stage trials.
Clinical Impact and Future Applications
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, the new guidelines provide the first systematic framework for their meaningful integration.
"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," said first author Emily Alger.
The research was supported through a PhD studentship as part of the Medical Research Council and the National Institute for Health and Care Research (搜索) Trials Methodology Research Partnership, with additional programme infrastructure funding from Cancer Research UK and the Experimental Cancer Medicine Centre (搜索).
Long-term Research Enhancement
Alger noted that the guidance is expected to promote more patient-centered approaches to assessing treatment tolerability and effectiveness in the longer term. "We also foresee it strengthening the clinical interpretation of PRO findings and improving the quality and transparency of reporting in early phase trials," she said.
"Ultimately, our work supports higher-quality research while helping ensure that the patient experience plays a meaningful role in cancer (搜索) drug development," Alger concluded.