An International Multi-Stakeholder Delphi Survey Study on the Design of Disease Modifying Parkinson’s Disease Trials

Abstract

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.

Patient and Public Involvement and Engagement in the Development of a Platform Clinical Trial for Parkinson’s Disease: An Evaluation Protocol

“…UK based PwP and care partners were recruited to the PPIE working group via advertisement through UK Parkinson’s charities, investigator PPIE groups as well as the UK based PwP and carer contributors to a Delphi study in trial design [ 24 ]. The invitation included a short description of project background, remit of involvement as well as details about reimbursement for time spent on the project.…”