Biomarkers, which are measurable indicators of biological processes, play a crucial role in the development and evaluation of treatments for neurodegenerative diseases.​ The article "Biomarkers for Neurodegenerative Diseases in Regulatory Decision‐Making by the European Medicines Agency" , just published by our partners CBG-MEB in the Alzheimer's & Dementia TRCI journal, explores the extent to which biomarkers are utilised in the development of treatments for neurodegenerative diseases, as well as topics of discussion regarding biomarkers in regulatory advice‐ and decision‐making processes.

​The European Medicines Agency (EMA) has increasingly incorporated biomarkers into the regulatory evaluation of neurodegenerative disease treatments. These biomarkers serve various roles, including supporting mechanism of action, diagnosing conditions, enriching study populations, and monitoring treatment responses.​ In their analysis, the authors identified and analysed over 100 EMA procedures related to neurodegenerative diseases that discussed biomarkers.

The analysis revealed that acceptance and role of biomarkers differed between neurodegenerative diseases. For example, 20 marketing authorisation applications (MAAs) for multiple sclerosis discussed biomarkers, whereas few MAAs for Parkinson's disease were identified, none of which discussed biomarkers. The authors discuss how biomarkers are viewed and employed in MAAs across diseases, and that an understanding of the disease mechanisms, and the accuracy of biomarkers in relation to these machanisms, forms the basis for regulators’ trust in biomarkers. Even if the disease mechanism is not fully elucidated, evidence for the relation between a biomarker and the clinical outcome, sufficiently validated in appropriate studies, could contribute to the evidence base supporting regulatory acceptance of its use as a surrogate for clinical outcome.

Biomarkers for neurodegenerative diseases in regulatory decision-making by the European Medicines Agency

Hermans AMM, Bakker E, Starokozhko V, den Otter L, Elferink AJA, Bradshaw AC, Guizzaro L, Mol PGM and Pasmooij AMG

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