A practical toolkit with recommendations for analysing and visualising patient-reported outcomes in early phase...

Patient-reported outcomes (PROs) are increasingly recognised for their role in assessing tolerability in dose-finding oncology trials (DFOTs). However, analysis and reporting of PRO data within DFOTs are often unclear and inconsistent. OPTIMISE-AR (Incorporating Patient-Reported Outcomes in Dose-Finding Trials-Analysis Recommendations) establishes a practical toolkit supporting the statistical analysis, visualisation, and reporting of PRO data within DFOT publications. International, multidisciplinary, cross-sector statistical analysis and data visualisation working groups identified analytical and visualisation approaches for PROs data, addressing key DFOT PRO research objectives. Informed by existing literature, case studies and recommendations are provided in this Policy Review for analysing binary, ordinal, and continuous PRO data to assess tolerability across doses and timepoints, and to integrate PROs into interim and final dose-decision processes. The OPTIMISE-AR toolkit is structured around four methodological domains aligned with key DFOT PRO research objectives, providing statistical analysis and data visualisation recommendations for (1) PRO endpoints across timepoints, (2) PRO endpoints between timepoints, (3) time-to-event PRO endpoints, and (4) PRO endpoints for formal dose-decision making in model-based dose-finding designs. As PROs have an increasing role in tolerability assessment, this Policy Review promotes analysis and data visualisation of PRO data, facilitating robust, patient-centred tolerability conclusions and supporting the broader development of tolerable and effective treatments.