OBJECTIVES: There is increasing momentum for using Patient-Reported Outcomes (PRO) to assess tolerability in early oncology trials. Over the last years regulatory and HTA bodies provide more guidance how to use patient experience data (PED) for their decision making. Sponsors are already considering PED for clinical development and market access purposes, realizing that a suitable data strategy may not start at Phase 3, but should be considered early on. This initiative introduces a range of methodological and logistical complexities that require solutions and global standardization. Our presentation aims to dissect these benefits, challenges, and opportunities to inform future early-phase oncology clinical trial designs.

METHODS: Our session will synthesize current knowledge and stakeholder perspectives on PRO integration in early-phase oncology trials, leveraging insights from published clinical trials and emerging international standards. We will summarize the benefits of PRO integration in early-phase clinical trials as well as critically examine key challenges, including optimizing recruitment methodologies, refining interpretation frameworks for nuanced PRO data collection and dose adaptation, and overcoming practical barriers in PRO implementation. We will address limited and contrasting views regarding resources associated with PRO data collection and assess the value of PRO insights in early-phase oncology trials.

RESULTS: Drawing from an extensive literature review and oncology expertise, we will showcase diverse examples from published clinical trials. We will highlight the development of new measures tailored for early-phase oncology trials and analyze the interpretation challenges posed by multiple trial datasets, including both successes and failures. We will spotlight challenges, opportunities, and strategies for sponsors to effectively implement PRO measures in the early-phase trials.

CONCLUSIONS: Our analyses will equip researchers with insights to navigate the expectations of decision makers, including health regulatory authorities, health technology assessment bodies, regarding PRO utilization. We aim to foster informed debate and promote improved integration of PRO measures.