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Lookup NU author(s): Professor Camille CarrollORCiD
This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License (CC BY-NC 4.0).
© 2023 - The authors. Published by IOS Press. Background: Design of disease modification (DM) trials for Parkinson's disease (PD) is challenging. Successful delivery requires a shared understanding of priorities and practicalities. Objective: To seek stakeholder consensus on phase 3 trials' overall goals and structure, inclusion criteria, outcome measures, and trial delivery and understand where perspectives differ. Methods: An international expert panel comprising people with Parkinson's (PwP), care partners (CP), clinical scientists, representatives from industry, funders and regulators participated in a survey-based Delphi study. Survey items were informed by a scoping review of DM trials and PwP input. Respondents scored item agreement over 3 rounds. Scores and reasoning were summarized by participant group each round until consensus, defined as≥70% of at least 3 participant groups falling within the same 3-point region of a 9-point Likert scale. Results: 92/121 individuals from 13 countries (46/69 PwP, 13/18 CP, 20/20 clinical scientists, representatives from 8/8 companies, 4/5 funders, and 1/1 regulator) completed the study. Consensus was reached on 14/31 survey items: 5/8 overall goals and structure, 1/8 Eligibility criteria, 7/13 outcome measures, and 1/2 trial delivery items. Extent of stakeholder endorsement for 428 reasons for scores was collated across items. Conclusions: This is the first systematic multi-stakeholder consultation generating a unique repository of perspectives on pivotal aspects of DM trial design including those of PwP and CP. The panel endorsed outcomes that holistically measure PD and the importance of inclusive trials with hybrid delivery models. Areas of disagreement will inform mitigating strategies of researchers to ensure successful delivery of future trials.
Author(s): Zeissler M-L, McFarthing K, Raphael KG, Rafaloff G, Windle R, Carroll CB
Publication type: Article
Publication status: Published
Journal: Journal of Parkinson's Disease
Year: 2023
Volume: 13
Issue: 8
Pages: 1343-1356
Print publication date: 19/12/2023
Online publication date: 18/11/2023
Acceptance date: 23/10/2023
Date deposited: 30/01/2024
ISSN (print): 1877-7171
ISSN (electronic): 1877-718X
Publisher: IOS Press BV
URL: https://doi.org/10.3233/JPD-230109
DOI: 10.3233/JPD-230109
Data Access Statement: The raw data supporting the findings of this study are available on request from the corresponding author. The data are not publicly available due to privacy or ethical restrictions.
PubMed id: 38007672
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