Last month, colleagues at the Dutch Medicines Evaluation Board (CBG-MEB) published an article in Alzheimer’s & Dementia: Translational Research & Clinical Interventions, entitled “Biomarkers for neurodegenerative diseases in regulatory decision-making”. Led by Audrey Hermans and Marjon Pasmooij (CBG-MEB) and co-authored by our Director for Research, Angela Bradshaw, the article provides a systematic overview of how biomarkers for neurodegenerative diseases (NDDs) are currently utilised in regulatory procedures at the European Medicines Agency (EMA). The analysis encompassed 105 EMA procedures between 1995 and 2023, including 57 scientific advice (SA) procedures, 29 marketing authorisation applications (MAAs), and 19 qualification procedures (QAs/QOs), focusing on biomarker-related content across neurodegenerative disease areas and procedural contexts. The goal of the analysis was to identify how and to what extent biomarkers are integrated into EMA assessments for NDDs such as Alzheimer’s disease (AD), Parkinson’s disease (PD), and multiple sclerosis (MS). Amongst the analysed procedures, AD and MS featured most frequently, with 30 and 33 relevant procedures respectively, while 9 procedures addressed PD.
The authors also observed that biomarker maturity varied significantly between diseases. In MS, biomarkers were more commonly referenced, in particular imaging biomarkers that detect MS lesions in the brain. In contrast, AD biomarkers were more frequently discussed in the context of early-phase clinical development, as diagnostics guiding participant selection. Imaging and cerebrospinal fluid (CSF) biomarkers were the most frequently addressed, although emerging blood-based biomarkers were also noted, particularly in AD. Across the procedures, the majority of biomarkers were used in exploratory or supportive roles rather than as primary endpoints or regulatory surrogates. The authors conclude by stating that, despite the established role of certain biomarkers for MS, there remains a major need for more precise and reliable biomarkers to improve diagnostic accuracy and treatment monitoring for NDDs. Full article:
