New Insights into Early Parkinson’s Disease Progression Highlight Importance of Patient-Centered Drug Development
A new study published in the Journal of Neurology offers critical longitudinal insights into how symptoms and functional impacts evolve for individuals with early Parkinson’s disease (PD). The research, applauded by the Critical Path Institute (C-Path), underscores the vital role of patient-focused drug development and real-world impact assessments.
Tracking Parkinson’s Disease Over Three Years
Led by Jamie Adams, M.D. and Jennifer Mammen, Ph.D., long-time partners of C-Path’s Critical Path for Parkinson’s consortium, the study, titled “Three years later: tracking bothersome symptoms and impacts for people with early Parkinson’s disease,” prospectively followed participants for three years. The research was funded by The Michael J. Fox Foundation for Parkinson’s Research (Grant #MJFF-024503 and # MJFF-022743).
The Patient Voice and Real-World Data
Dr. Adams emphasized the importance of integrating the patient voice into the scientific record, stating, “Reaching this milestone represents a special moment for our collaborative team. We have championed the integration of the patient voice from the very beginning and seeing these perspectives formally shape the scientific record feels like the culmination of a deep and long-term partnership that has fundamentally changed how we approach clinical observation.”
Longitudinal tracking utilized wearable devices to assess the real-world experiences of people living with Parkinson’s disease. The findings revealed that functional impairment continued to worsen over time, even when specific symptoms appeared stable.
Key Symptoms and Impacts
Gait, balance, and posture emerged as the most bothersome issues for participants. The study also highlighted a significant increase in the effort required for daily activities, alongside a growing psychosocial burden. These findings emphasize the demand to incorporate real-world measures of disease progression, in addition to traditional symptom assessments, when designing clinical trials.
Improving Clinical Trial Design
Diane Stephenson, Ph.D., C-Path Vice President of Neurology and Executive Director of the Critical Path for Parkinson’s consortium, stated, “At C-Path, our goal is to put robust and patient-centered measurement tools directly in the hands of drug developers so they can design the very best clinical trials.” She added that validating real-world impacts supports a more informed and efficient development process and streamlines trial design, allowing researchers to focus on innovation.
Digital Health Technologies and Future Research
Cheryl Coon, Ph.D., Vice President of C-Path’s Clinical Outcome Assessment Program, noted the growing opportunities to advance patient-centered measurement through digital health technologies. Integrating measures of gait and balance with longitudinal patient-reported assessment offers a powerful method to monitor disease progression.
Sarah Zenner Dolan, a person living with early-onset Parkinson’s and a research participant, underscored C-Path’s focus on the voice of people living with Parkinson’s disease, stating it is “key in the evaluation of how wearable devices can reliably track our symptoms in real time.”
Yuge Xiao, Clinical Research Lead at The Michael J. Fox Foundation for Parkinson’s Research and co-author of the publication, emphasized the importance of integrating the lived experience of people with Parkinson’s disease into longitudinal research. Combining digital measures with participant interviews ensures a clear link between what digital measures capture and changes that matter to people with Parkinson’s, and informs how these tools can be used meaningfully in clinical trials.