Cover: Clinical Outcome Assessments and Digital Health Technologies Supporting Clinical Trial Endpoints in Early Parkinson's Disease

Clinical Outcome Assessments and Digital Health Technologies Supporting Clinical Trial Endpoints in Early Parkinson's Disease

Roundtable Proceedings and Roadmap for Research

Published Apr 24, 2023

by Claire E. O'Hanlon, Carrie M. Farmer, Jamie L. Ryan, Natalie C. Ernecoff

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This document summarizes the Parkinson's Disease (PD) Endpoints Roundtable, which was held in Washington, D.C., on November 2–3, 2022, and hosted by The Michael J. Fox Foundation for Parkinson's Research, Parkinson's UK, and Parkinson Canada. This event brought representatives from academia and industry together with those from regulatory agencies, community partners, and research funders to discuss challenges in clinical outcome assessment development for treatments in early PD and to identify priorities for the field and opportunities for collaboration.

This document provides a summary of the presentations given and topics discussed at the roundtable and synthesizes the discussions about the development of clinical outcome assessments and the use of digital health technologies for developing clinical trial endpoints.

Key Findings

  • There are numerous areas where industry, regulatory, clinical, and community stakeholders can advance development of clinical outcomes assessments (COAs) and digital health technologies (DHTs) to underlie endpoints for clinical trials in early PD.
  • Development and regulatory approval of novel therapies to prevent or delay disease progression in early PD requires patient-centered COAs—measures that describe or reflect how a patient feels, functions, or survives. To be used in clinical trials, these measures must be sensitive to change in early PD and have evidence supporting their meaningfulness to patients.
  • Key gaps hindering progress include knowledge of the underlying biology of PD, consensus on a biological staging approach in early PD, harmonization on definitions and standards, development of outcomes reported by knowledgeable informants other than the patient, and diversity in PD research and advocacy.
  • Next steps for the field include publishing a consensus conceptual model of early PD, disseminating data collection and reporting standards for DHTs in PD, establishing collaboratives for sharing qualitative data and DHTs, and creating libraries for concepts of interest and digital data.

Research conducted by

This summary of the roundtable proceedings was funded by The Michael J. Fox Foundation for Parkinson's Research and produced within the Quality Measurement and Improvement Program in RAND Health Care.

This report is part of the RAND conference proceeding series. RAND conference proceedings present a collection of papers delivered at a conference or a summary of the conference.

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