Advancing Patient-Centric Care: Integrating Patient Reported Outcomes for Tolerability Assessment in Early Phase Clinical Trials – Insights from an Expert Virtual Roundtable

Christina Yap*, Olalekan Lee Aiyegbusi, Emily Alger, Ethan Basch, Jill Bell, Vishal Bhatnagar, David Cella, Philip Collis, Amylou C. Dueck, Alexandra Gilbert, Ari Gnanasakthy, Alastair Greystoke, Aaron R. Hansen, Paul Kamudoni, Olga Kholmanskikh, Bellinda L. King-Kallimanis, Harlan Krumholz, Anna Minchom, Daniel O'Connor, Joan PetrieClaire Piccinin, Khadija Rerhou Rantell, Saaeha Rauz, Ameeta Retzer, Steven Rizk, Lynne Wagner, Maxime Sasseville, Lesley K Seymour, Harald A Weber, Roger Wilson, Melanie Calvert, Devin Peipert

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

Abstract

Early phase clinical trials provide an initial evaluation of therapies’ risks and benefits to patients, including safety and tolerability, which typically relies on reporting outcomes by investigator and laboratory assessments. Use of patient-reported outcomes (PROs) to inform risks (tolerability) and benefits (improvement in disease symptoms) is more common in later than early phase trials. We convened a two-day expert roundtable covering: (1) the necessity and feasibility of a universal PRO core conceptual model for early phase trials; (2) the practical integration of PROs in early phase trials to inform tolerability assessment, guide dose decisions, or as real-time safety alerts to enhance investigator-reported adverse events. Participants (n = 22) included: patient advocates, regulators, clinicians, statisticians, pharmaceutical representatives, and PRO methodologists working across diverse clinical areas. In this manuscript, we report major recommendations resulting from the roundtable discussions corresponding to each theme. Additionally, we highlight priority areas necessitating further investigation.
Original languageEnglish
Article number102838
Number of pages13
JournalEClinicalMedicine
Volume76
Early online date24 Sept 2024
DOIs
Publication statusPublished - Oct 2024

Keywords

  • Patient-reported outcomes
  • Dose-finding
  • Phase I
  • Phase II
  • Early phase trials
  • Tolerability
  • Patient-centred clinical development
  • Quality of life
  • Trial designs

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