Objective: To assemble an international, multi-stakeholder Delphi panel to seek consensus on trial methodology for an adaptive trial platform investigating disease modifying therapies (DMTs) in Parkinson’s disease (PD).

Background: An adaptive trial platform may offer an efficient way to shorten clinical testing of DMTs but requires consensus on a core protocol which we sought through an iterative, survey-based Delphi process.

Method: We recruited an international, multi-stakeholder Delphi panel (13 countries) consisting of 69 people with Parkinson’s (PwP), 18 care partners, 20 clinical scientists, 8 industry representatives, 5 funders and 1 regulator. The Delphi survey comprised 31 questions relating to overall trial goals and structure, inclusion criteria, outcome measures and delivery methodology. Participants were asked to score agreement on a 9-point semantic differential scale and provide reasons for their choice. Questions were administered 3 times or until 70% of at least 3 participant groups (excluding regulators) scored within the same 3-point region of the scale. In each round, participants were shown their own previous scores as well as a summary of scores and reasoning from all participants.

Results: 1 funder, 20 PwP and 5 care partners withdrew from the study during the first 3 surveys. Sharing of opinions was optional, however reasoning was provided for 93% of items across all participants and surveys. Following round 3, consensus was achieved in 13/31 items. Although not always unanimous, there was consensus that the trial should investigate more than one drug, ascertain in which types of PD DMTs work best, be placebo-controlled, and provide a platform to validate new digital measures. Participants agreed phase 3 success could be shown by a delay of disease milestones or effects on quality of life (QoL), activities of daily living (ADL), patient completed questionnaires or passive digital measures. Clinical scientists were less supportive of QoL and ADL measures whilst patients/carers were less sure about questionnaires and digital measures. The panel did not favour the restriction of trials to drug naïve PwP.

Conclusion: We demonstrated consensus relating to some trial design decisions achieved by an international Delphi panel, as well as areas of contention. These findings will be informative for the development of an internationally endorsed protocol for an adaptive trial platform in PD.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/informing-the-design-of-disease-modifying-trials-for-parkinsons-disease-a-delphi-study/