Study Rationale: To evaluate treatments in ways that are meaningful to people with Parkinson’s disease (PD), we need to identify measurements that reflect how people with early PD function and feel. The Critical Path (C-Path) Institute provides support for ongoing activities related to engagement with regulatory agencies to optimize patient-centric measures for use in early PD trials. The staff at C-Path include clinical outcome assessment experts who have extensive experience in generating qualitative and quantitative evidence to support regulatory submissions and discussions.
Hypothesis: We hypothesize that including the patient voice in PD trials through appropriate measurement approaches and evidence documentation will facilitate the development and testing of treatments that produce a meaningful impact for people with PD.
Study Design: This work will support all trial stages for early PD populations.
Impact on Diagnosis/Treatment of Parkinson’s disease: The project will better measure how people with early PD feel and function so that treatments can be evaluated in terms of concepts that are meaningful to patients.
Next Steps for Development: Next steps for this work would see selected or modified instruments used in interventional and observational studies so that evidence can be generated to support their use in supporting trial endpoints and so that trial endpoints measure meaningful concepts to people with early PD.