Study Rationale:
There are no validated tools to assess functional changes in people living with PD (PwP) at the earliest clinical stages. Available tools lack sufficient sensitivity to capture these changes and thus are inherently limited in the context of trials evaluating disease modification therapies, in which clinical disease progression is measured. Regulatory agencies have placed the voice of the patient at the core of clinical outcome assessments (COA) used in the evaluation of new medicines
Hypothesis:
The proposed study will develop a validated patient-reported COA able to assess the functional impact in PwP at the earliest stages of PD
Study Design:
We will start by identifying the themes of interest to assess functional impairment at the earliest stages of PD. To accomplish this, a group of PwP, PD disease experts, patient-reported COA developers and clinimetric experts will consider published literature on the topic and information collected during focus group sessions with PwP and care partners. We will adopt a consensus methodology. At the conclusion of this phase, a first version of the new COA will advance to phase 2 (Cognitive pretesting) to be evaluated for clarity and ease of use through an interview technique. Cognitive pretesting is an iterative process that, once successfully completed, will provide a version of the new COA ready for evaluation of its performance in a larger group of PwP.
Impact on Diagnosis/Treatment of Parkinson’s disease:
Development of a patient-reported COA sensitive to the earliest changes in function on PwP would improve identification of PwP who might benefit from treatments earlier and the evaluation of the therapeutic effects for a novel intervention designed for an early disease stage, with particular relevance for the evaluation of treatments with the potential to slow the progression of PD.
Next Steps for Development:
We plan to complete the validation of this new scale by evaluating how it performs as it is administered to PwP, to ultimately support its use in clinical trials and endorsement by regulatory agencies such as the FDA that approve new treatments.