Study Rationale: This project seeks to address an unmet need for fit-for-purpose, clinical outcome assessments for early-stage Parkinson’s disease (PD) to be used in clinical trials to capture the viewpoint of individuals with PD. The project team recently completed a study to develop measures for this purpose by analyzing an existing dataset to identify symptoms that are important to people with PD, comparing those symptoms with sets of questions already available, and choosing items from these sets to create 13 draft questionnaires for 7 non-motor symptom domains and 20 symptom concepts.
Hypothesis: We hypothesize that further testing will reveal whether these 13 questionnaires are easy to understand and contain questions that are relevant to people with PD in the first three years after diagnosis.
Study Design: This study will be conducted in two stages. In stage 1, we will use small focus groups of people with PD to review and comment on the draft questionnaires and provide feedback to tell us if anything is missing. In stage 2, we will conduct cognitive interviews with 45 people with PD who will complete a subset of the questionnaires and then answer questions about the meaning and relevance of the questions in light of their experience with PD. Based on these interviews, we will make any necessary changes to the questionnaires to improve them for future use in clinical trials.
Impact on Diagnosis/Treatment of Parkinson’s disease: The project will impact PD research and treatment protocols by providing questionnaires to assess non-motor symptoms as patient-centric endpoints for early-stage PD clinical trials. These non-motor symptoms are extremely burdensome to people with PD, impacting their ability to carry out daily activities.
Next Steps for Development: The next steps for development would be to include the questionnaires in clinical trials; if changes are needed, we would conduct additional testing with a larger number of people with PD to confirm the reliability and validity of any new questions in a standalone study before including them in clinical trials.