Funded Studies
Study Rationale: There are currently no validated tools for assessing functional changes in people at the earliest clinical stages of Parkinson’s disease (PD). Available approaches lack sufficient sensitivity to capture these changes and thus are inherently limited in the context of trials to evaluate disease-modifying therapies, in which clinical disease progression is measured. Regulatory agencies have placed the “voice of the patient” at the center of clinical outcome assessments (COA) used in the evaluation of new medicines.
Hypothesis: We propose that a validated, patient-reported COA can be used to assess the functional impact of therapies in people at the earliest stages of PD.
Study Design: Working with disease experts, patient-reported COA developers and clinimetric experts, we will explore the published literature and collect information from focus group sessions with people with PD and their care partners to identify topics that will allow a functional assessment of impairment at the earliest stages of PD. Using a consensus methodology, we will formulate a new COA that will be evaluated for clarity and ease of use through an interview technique. After this iterative process of “cognitive pretesting,” we will arrive at a COA that will be evaluated for its performance in a larger group of people with PD.
Impact on Diagnosis/Treatment of Parkinson’s disease: Development of a patient-reported COA sensitive to the earliest changes in function would improve identification of people with PD who might benefit from early treatment and facilitate the evaluation of therapeutics designed to slow the progression of PD at its earliest stages.
Next Steps for Development: We plan to complete the validation of this new scale by evaluating how it performs as it is administered to people with PD. Ultimately, we will support its use in clinical trials and its endorsement by regulatory agencies that approve new treatments, such as the FDA.