Advancing patient-centric care: integrating patient reported outcomes for tolerability assessment in early phase clinical trials - insights from an expert virtual roundtable.
Yap, Christina Lee Aiyegbusi, Olalekan Alger, Emily Basch, Ethan Bell, Jill Bhatnagar, Vishal Cella, David Collis, Philip Dueck, Amylou C Gilbert, Alexandra
Yap, Christina
Lee Aiyegbusi, Olalekan
Alger, Emily
Basch, Ethan
Bell, Jill
Bhatnagar, Vishal
Cella, David
Collis, Philip
Dueck, Amylou C
Gilbert, Alexandra
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2024-09-24
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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.
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Yap C, Lee Aiyegbusi O, Alger E, Basch E, Bell J, Bhatnagar V, Cella D, Collis P, Dueck AC, Gilbert A, Gnanasakthy A, Greystoke A, Hansen AR, Kamudoni P, Kholmanskikh O, King-Kallimanis BL, Krumholz H, Minchom A, O'Connor D, Petrie J, Piccinin C, Rantell KR, Rauz S, Retzer A, Rizk S, Wagner L, Sasseville M, Seymour LK, Weber HA, Wilson R, Calvert M, Peipert JD. Advancing patient-centric care: integrating patient reported outcomes for tolerability assessment in early phase clinical trials - insights from an expert virtual roundtable. EClinicalMedicine. 2024 Sep 24;76:102838. doi: 10.1016/j.eclinm.2024.102838.
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