Objective: To develop a selection process for digital measures (DMs) to include as exploratory outcome measures of Parkinson’s disease (PD) progression in a platform disease modification trial.

Background: There is currently no accepted DM of PD progression. The Edmond J Safra Accelerating Clinical Trials in Parkinson’s Disease (EJS ACT-PD) initiative is designing a multi-arm multi-stage trial for disease modification in PD. Set to recruit 1600 participants, this presents an opportunity to expedite the validation of DMs as trial outcomes capitalising on objective data capture to detect sensitive surrogate markers of PD progression that are relevant to patients.

Method: 20 EJS ACT-PD consortium members including clinicians, DM experts and patient and public involvement and engagement (PPIE) representatives convened bi-monthly to prioritise concepts of interest (COI) and devise a DM selection process, informed by review of relevant literature, regulatory guidance, validation frameworks, practicalities for deployment and data management.

Results: Mobility was prioritised as a COI which is insufficiently captured within existing clinical outcome assessments with the potential of increased sensitivity to treatment effects. A framework comprising 5 steps was devised: 1) DM identification from literature search and expert recommendation; 2) Shortlisting against essential criteria (relevant to COI, technical and clinical validity demonstrated); 3) Independent expert scoring of evidence compiled by DM developers, in the domains of: participant burden/experience, site staff acceptability, technical and clinical validity, reliability, performance, clinical meaningfulness, scalability, regulatory readiness, data processes, and technical specifications; 4) In-person PPIE review consisting of a live-demonstration and discussion followed by scoring of usability, burden, accessibility/inclusivity, adherence/compliance and data privacy; 5) Selection panel review of expert and PPIE scores to inform final DM selection.

Conclusion: Our selection process, based on an evidence-based framework and informed by regulatory guidance, integrates PPIE and expert review to select the most appropriate DMs for the EJS ACT-PD trial, and realise our potential to support validation of DMs as meaningful clinical outcome assessments of progression in PD modification trials.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/developing-a-selection-framework-for-digital-measures-of-parkinsons-disease-progression-for-the-ejs-act-pd-trial/