There is great unmet need in discovering novel treatment for Parkinson's disease (PD) and identifying the new agents potentially causing drug-induced parkinsonism. New indications and contraindications of drugs are typically approved following rigorous randomized controlled trial (RCT) evaluation. However, RCTs have their inherent limitations, since they are usually conducted in ideal conditions, with high cost and limited follow-up periods. In the past decade, large cohort studies with long follow-up outcome data was derived from a PD database in a real-world setting. Studies based on real world data (RWD) can help to augment and extrapolate data obtained in RCTs and provide information about the safety and effectiveness of a medication in heterogeneous, large populations. In the present review, we focus on the published real world studies designed to develop new treatment strategies for repurposing drugs and identifying contraindications for PD. We also outline the challenges and limitations in these studies. Subsequently we introduce PaWei app platform, which hopefully can facilitate PD management and address real-world problems associated with PD. Better understanding of RWD collection and analysis is needed if RWD is to achieve its full potential.
Keywords: Parkinson’s disease; contraindication; randomized controlled trial; real world study; repurposing drug.