The European Medicines Agency (EMA) has released a concept paper outlining proposed updates for its clinical guideline on Parkinson’s disease—the first major revision in more than a decade. The document signals a significant shift toward modernized trial design, expanded patient inclusion, and the integration of emerging biomarkers.
A Needed Update to a Decade-Old Guideline
The current Parkinson’s disease guideline has been in place since 2012. EMA’s Central Nervous System Working Party (CNSWP) noted that substantial advances in biomarker research, diagnostic tools, and therapeutic innovation require a comprehensive update.
The revision will consider how newly developed biomarker algorithms could support patient selection, trial enrichment, and disease characterization. EMA also indicated that the guideline may expand beyond classic Parkinson’s disease to include related neurodegenerative movement disorders such as multiple system atrophy, corticobasal degeneration, and progressive supranuclear palsy.
Including More Diverse Patient Populations
One of the most significant changes under consideration is the potential inclusion of patient groups typically excluded from clinical studies. Individuals with advanced Parkinson’s disease who receive treatments such as deep brain stimulation, focused ultrasound, or continuous levodopa infusions often fall outside traditional study criteria.
EMA stated that these populations represent real-world clinical needs and should be part of future scientific discussions. Including them could strengthen the applicability and generalizability of clinical trial findings.
Evolving Trial Design Questions
To guide the development of the full guideline update, EMA is seeking stakeholder input on several key topics:
• Whether clinical and non-clinical biomarkers should be used for trial enrollment.
• How endpoints for both motor and non-motor symptoms should evolve.
• Whether patients using certain medical devices should be included in drug trials.
• How randomized trial designs can incorporate underlying pathophysiology instead of focusing solely on symptomatic improvement.
• The role of decentralized or hybrid clinical trial components.
• How concomitant medications should be evaluated in study design.
These questions reflect the agency’s focus on modernizing clinical evaluation frameworks for neurodegenerative conditions.
What the Revised Guideline Will Not Cover
EMA clarified that the update will not address drug-device combination products, treatments for non-rare neurodegenerative movement disorders, or essential tremor. These areas fall outside the scope of the planned revision.
Timeline and Next Steps
The draft guideline is expected to be released by the fourth quarter of 2026. EMA is encouraging input from regulators, academic groups, clinical trial experts, and professional organizations such as The Movement Disorders Society and the European Academy of Neurology as it refines the document.
At EMMA International
As regulators incorporate new scientific tools and broaden expectations for clinical trial design, life-science organizations must adapt quickly. EMMA International supports sponsors developing neurological and movement disorder therapies by providing:
• Regulatory strategy and gap assessments
• Endpoint and study design consulting
• Alignment with EU and FDA clinical guidance expectations
• Documentation and submission support
Our team ensures your development program remains compliant, scientifically robust, and ready for evolving regulatory standards.
For more information on how EMMA International can assist, visit www.emmainternational.com or contact us at (248) 987-4497 or info@emmainternational.com.
Reference:
Craven, J. (2025, December 2). EMA considers new biomarkers, patient populations in proposed Parkinson’s disease guideline update. Regulatory Affairs Professionals Society.
European Medicines Agency. (2025). Concept Paper on the Revision of the Guideline on Parkinson’s Disease.
European Commission. (2025). Scientific Guideline Development Timeline.
