Clinical Outcome Assessments and Digital Health Technologies Supporting Clinical Trial Endpoints in Early Parkinson's Disease: Roundtable Proceedings and Roadmap for Research

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

This Article

RAND Health Quarterly, 2024; 11(3):1

Abstract

This article 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 article 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.

For more information, see RAND CF-A2550-1 at https://www.rand.org/pubs/conf_proceedings/CFA2550-1.html

Full Text

Numerous novel drug candidates aiming to slow or stop the progression of Parkinson's disease (PD) are under development, offering hope for a disease that currently has no approved treatments to prevent or delay the onset of progressive disabilities. Many promising candidates are being tested for use in early PD. Regulatory approval for such treatments hinges on the achievement of prespecified endpoints in randomized controlled trials, but there are limited outcome measures that are sensitive to statistically and clinically significant changes in early PD and that have demonstrated sufficient evidence to support the meaningfulness of such changes to patients. While many outcome measures for PD exist, they pose challenges for use in early PD, given the clinical heterogeneity, slow rate of disease progression, lack of functional impairment, and combination of motor and nonmotor symptoms present in the population of patients with early PD. There is thus an urgent need to develop clinical outcome assessments (COAs) that can be used in clinical trials of novel treatments in early PD. Digital health technologies (DHTs), such as mobile apps or wearable sensors, are promising tools for developing COAs for early PD.

The Michael J. Fox Foundation for Parkinson's Research (MJFF), Parkinson's UK, and Parkinson Canada co-hosted the PD Endpoints Roundtable on November 2–3, 2022, in Washington, D.C., to discuss these issues. The event brought representatives from academia and industry together with representatives of regulatory agencies, community partners, patient advocates, and research funders to build consensus and collaborate on outcome assessment methods that will support development of new novel treatments, referred to as precompetitive alignment. The stated goals of the roundtable (in the agenda document given to attendees) were as follows:

“To build precompetitive alignment and reduce redundancy on methodologies and regulatory insights for

  1. developing patient-centric COAs and
  2. linking voice of the patient to digital health technologies

so that the Parkinson's field can move faster and cohesively advance development of better tools for measuring change in early-stage Parkinson's disease.”

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 Parkinson's disease (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.
  • Additional opportunities to bring the field together to discuss and collaborate on issues of shared interest may accelerate development of COAs and DHTs in early PD.

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.

RAND Health Quarterly is produced by the RAND Corporation. ISSN 2162-8254.