Presentation (CTI)

OBJECTIVES: This research aims to evaluate the benefits of incorporating patient-reported outcome measures to assess treatment tolerability in early-phase trials, focusing on discrepancies between the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) and CTCAE data. It also examines differences between clinician- and patient-reported symptom severity related to treatment tolerability and the role of embedded trial interviews in capturing comprehensive adverse event (AE) data.
METHODS: A targeted review of Phase 1 and 2 trials incorporating the PRO-CTCAE was conducted using a designated search strategy implemented in MEDLINE® and ClinicalTrials.gov, alongside a meta-analysis of studies comparing PRO-CTCAE and CTCAE data. Studies involving embedded interviews were also analyzed to assess their effectiveness in capturing patient-reported AEs. Data were synthesized to evaluate discrepancies in treatment tolerability reporting and the added value of patient input in tolerability assessments.
RESULTS: The analysis revealed significant differences between patient-reported symptom severity via the PRO-CTCAE and clinician-reported symptom severity via CTCAE reports, with patients consistently reporting higher symptom severity and frequency. The notable divergence suggests potential underreporting and misinterpretation of AEs when relying exclusively on clinician observations. Trials that incorporated embedded interviews demonstrated improved capture of AEs, providing richer contextual insights into patient experiences in Phase 1 and 2 trials.
CONCLUSIONS: Integrating patient-reported treatment tolerability assessments in early-phase trials enhances the accuracy of safety profiling by addressing discrepancies in symptom reporting. Inclusion of the PRO-CTCAE and embedded interviews allows for a more comprehensive understanding of patient experiences and supports the development of patient-centered therapies, aligning with regulatory recommendations and guidance that emphasize the importance of including the patient voice in new drug applications to ensure a successful review process. Capturing patient perspectives in early-phase trials is essential for accurate and comprehensive identification of safety signals early in the drug development process, allowing for earlier dose optimization or modification.