EMA considers new biomarkers, patient populations in proposed Parkinson’s disease guideline update
The European Medicines Agency (EMA) has released a concept paper proposing discussion questions about what should be contained in a future clinical guideline revision for Parkinson’s disease, the first revision in more than a decade.“The Central Nervous System Working Party (CNSWP) recommends drafting a revision of Parkinson's disease guideline including the utility and role of new biomarkers to inform enrolment in clinical trials/enrichment approach and other forms of Parkinsonism and related disorders,” the authors of the concept paper wrote.
When finalized, the proposed guideline will replace the second revision of the Parkinson’s disease guideline released in July 2012.
However, the concept paper states that the existing guideline “predates some recent developments in the field” such as proposed biomarker algorithms for clinical trial selection, considerations for the development of new treatments, and guidance for disease with non-motor symptoms. Another proposal for the guideline revision is to expand it to other neurodegenerative movement disorders such as multiple system atrophy, corticobasal degeneration, and progressive supranuclear palsy.
“Advanced [Parkinson’s disease] and other movement disorders patients are increasingly being treated with treatments such as parenteral levodopa/agonists, deep brain stimulation, and focused ultrasounds (FUS),” the concept paper explained. “These patients have usually been excluded from clinical trials with medicines; discussion on strategies to include this population in studies is needed.”
The revised guideline would not include discussion of the development of drug-device combination, treatments for non-rare neurodegenerative movement disorders, or essential tremor.
EMA discussion questions
EMA noted that interested parties for the guidance document would be other regulatory agencies and members of the pharmaceutical industry as well as organizations such as The Movement Disorders Society, European Academy of Neurology, The International Society for CNS Clinical Trials and Methodology, and The European College of Neuropsychopharmacology.
The agency is seeking answers to several topics surrounding Parkinson’s disease and other neurodegenerative movement disorders, including the discussion of including clinical and non-clinical biomarkers for enrollment in clinical trials, updated primary and secondary endpoints for motor and non-motor symptoms, whether patients with medical devices should be included in clinical trials, randomized clinical trial design and whether that should include underlying pathophysiological processes in addition to symptomatic treatment, decentralized components of clinical trial design, how concomitant medication can impact treatment, and other considerations for treatments of neurodegenerative movement disorders and for essential tremor.
EMA said the draft guidance document is expected to be released no later than the fourth quarter of 2026.
Scientific guideline
