International Consensus-Driven Recommendations for Patient-Reported Outcome Research Objectives in Early Phase...

Area of Science:

• Oncology
• Clinical Trial Methodology
• Patient-Reported Outcomes

Background:

• Growing interest in integrating patient-reported outcomes (PROs) into early phase dose-finding oncology trials (DFOTs).
• Existing research highlights unclear PRO objectives in DFOTs, hindering effective use.
• The OPTIMISE-ROR project aims to provide guidance for incorporating PROs in DFOTs.

Purpose of the Study:

• To establish clear research objectives for PROs in DFOTs.
• To support trialists in effectively integrating PROs into early phase oncology trials.
• To enhance the assessment of treatment tolerability and inform dose selection.

Main Methods:

• Methodological framework from the EQUATOR Network was utilized.
• Included a review of published DFOTs with PROs, expert consultation, and a two-round international Delphi survey (N=109, N=96).
• A consensus meeting with 31 multidisciplinary experts finalized recommendations.

Main Results:

• Six recommendations were developed, focusing on three core PRO tolerability concepts: side effect impact, symptomatic adverse events, and health-related quality of life.
• Emphasized integrating PROs for dose recommendations in escalation and optimization trials.
• Highlighted the importance of PRO data analysis across time and dose levels, defining PRO objectives, and assessing PRO endpoints.

Conclusions:

• Provides foundational guidance for key PRO research objectives in DFOTs.
• Facilitates systematic PRO integration for patient-centered evidence in tolerability and efficacy assessment.
• Aims to contribute to developing safer and more effective cancer therapies through improved dose finding and regulatory evaluation.