(P-195) Parkinson's Disease Experience from a Patient's Perspective: Systematic Review on Patient Reported Outcomes

Background: Patient focused drug development (PFDD) is an approach recommended by regulatory agencies to rigorously capture patient experiences, perspectives, and priorities to inform drug development and regulatory decisions. Parkinson’s disease (PD) remains a condition with unmet needs including an in-depth understanding of the patient experience. In keeping with the FDA’s 21st-Century Cures Act PFDD recommendations, drug development programs for PD seek to incorporate patient experiences and perspectives into clinical trial protocols. To inform the selection of patient reported outcome (PRO) measures useful as endpoints in forthcoming clinical trials, a systematic literature review was performed to recognize and recapitulate PRO measures that have been used in published studies of Parkinson’s disease.

Objectives: 1) Identify PROs being used to assess disease status in US patients with PD. 2) Identify the most common measurement domains of PROs in use.

Methods: A systematic search of computerized databases PubMed and PsycINFO was conducted to identify relevant publications. Restrictions limited articles to human studies published in English between 2016 and 2020, and those with full text access. Specific criteria for inclusion were: 1) PD ≥18 years of age; 2) “patient reported” outcome measures and experiences; 3) sample size >50; 4) US patients.

Results: The search yielded a total of 650 articles and 36 articles met the criteria, encompassing a total of 5695 patients. Most included studies (80.55%) were observational designs. A small number of randomized controlled trials, clinical validation, and qualitative case studies were also included. Overall, 53 PRO measures with 11 domains were identified. A large number of PROs utilized belonged to the emotion/psychological measurement domain (30%), with validated scales of depression accounting for 37.5%. Health related quality of life measures comprised the next largest domain (23%) with PD specific measures accounting for the rest. This review illustrates the interest in understanding the mental and emotional health of PD patients.

Conclusions: This systematic review provides a recent overview of the PD patient reported outcome measures used in studies in the United States published from 2016-2020. The most common PROs identified across 36 studies were in the emotional and psychological domain thus demonstrating the research interest to better understand these experiences in PD patients. Incorporating these and other PROs in future PD clinical trials would meet PFDD recommendations to identify areas most important to patients and provide additional context for drug development and evaluation.