Objective: To advance patient-centric endpoints for use in early-stage Parkinson’s disease (PD) clinical trials.

Background: Limitations with currently available patient-centric clinical outcome assessments (COAs) has led to industry sponsors independently working to develop endpoints and advance them for regulatory acceptance. Precompetitive collaboration that engages vested stakeholders – industry, regulatory, academic, research foundations, advocates, and patients and care partners – provides an efficient means to share resources and learnings to find a common solution.

Method: In collaboration with the Critical Path for Parkinson’s (CPP) Consortium of the Critical Path Institute (C-Path), members of industry, FDA, academic experts, disease research foundations, and patient advocacy organizations formed the PD Patient-Centric Endpoints Working Group (WG) in December 2021. The WG is co-chaired by a C-Path COA Scientist and an industry Patient-Centered Outcomes Research Scientist.

Results: The WG has identified a number of goals and challenges to address in the near-term. A primary goal is to align with FDA to seek input and identify expectations for implementation of reliable, valid, and clinically meaningful patient-centric COAs for use in PD clinical trials. Another goal is to share experience, progress, and knowledge on COA development and use among stakeholders.  Engaging people with PD and their care partners is key to aligning with patient-focused drug development. In addition to the need for sensitive, responsive patient-centric COA endpoints, the WG recognizes the measurement difficulties inherent to PD, including the general slow rate of decline in early-stage PD and the impact of symptomatic therapy. Mid-term and long-term goals of the WG will be informed by progress in the first half of 2022.

Conclusion: Formation of the WG is consistent with FDA’s Patient-Focused Drug Development guidance, which encourages the collaboration among multiple stakeholders and the leveraging of learnings and existing data. The WG has the opportunity to make substantive progress towards the successful inclusion of COA endpoints in PD clinical trials and serve as an exemplar approach for other disease areas.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/optimizing-patient-centric-clinical-outcome-assessments-for-use-in-parkinsons-disease-clinical-trials-critical-path-for-parkinsons-multistakeholder-initiative/