EMA Issues Revised Guidance on Biomarkers, Diagnostic Criteria for Alzheimer’s Medicines

The EMA’s Committee for Medicinal Products for Human Use (CHMP) adopted a revised guideline on the clinical investigation of medicines for the treatment of Alzheimer’s disease, focusing on the design and analysis of safety studies and the potential use of biomarkers in various stages of medicinal development.

The revision, which goes into effect on Sept. 1, separates biomarkers in AD clinical trials according to their potential use:

• Diagnostic, for determining diagnosis;
• Enrichment, for population selection;
• Prognostic, for determining the course of illness;
• Predictive, for safety assessment and predicting a future clinical response; and
• Pharmacodynamic, for determining intended or unintended activities

The guidance notes that while most biomarkers still require validation for these purposes, cerebrospinal fluid markers as well as MRI and PET imaging markers “are qualified for the enrichment of study populations” even though the biomarkers have not been qualified for preclinical Alzheimer’s disease.