EMA set to revise guidelines on clinical investigation of medicines for the treatment of Alzheimer’s disease

The European Medicines Agency’s (EMA) has announced the revision of its guideline on clinical trial studies for the treatment of Alzheimer’s disease. This document aims to provide guidance for the development of medicines across all stages of Alzheimer’s disease. The guideline will become effective from 1 September 2018.

This guideline provides guidance for the development of medicines across all stages of Alzheimer’s disease. It covers the impact of new diagnostic criteria for Alzheimer’s, including early and even asymptomatic disease stages, factors to be considered when selecting parameters to measure clinical trial outcomes at the different disease stages in Alzheimer’s, the potential use of biomarkers in the various stages of medicine development and  the design and analysis of efficacy and safety studies.

EMA organized a workshop for patients, academia, regulators, representatives from the pharmaceutical industry and independent experts to ensure that it was informed of the most up-to-date scientific developments in understanding and treating Alzheimer’s disease before revising the guideline. This effort was complemented by a series of meetings between EMA and developers of medicines intended to slow down the disease progression, to discuss the issues encountered in their clinical trials.

Currently available medicines for Alzheimer’s disease only treat its symptoms. However, a number of therapies under development are targeting the biological mechanism of the condition to try and modify the course of the disease. There is now a consensus that treatment options should be evaluated in earlier disease stages before the full picture of dementia is reached. The guidance document specifically addresses:

  • The impact of new diagnostic criteria for AD including early and even asymptomatic disease stages on clinical trial design.
  • The choice of outcome parameters and need for distinct assessment tools with regard to the different disease stages in AD (different signs and symptoms, differences in progression rate).
  • Potential use of biomarkers and their temporal relationship with the different phases of AD in different stages of drug development (mechanism of action, target engagement, use as diagnostic test, enrichment of study populations, stratification for subgroups, safety and efficacy markers, etc.).
  • Targets of estimation defining treatment effects of interest for regulatory decision making.
  • Design of long term efficacy (maintenance of effect) and safety studies.

Link: to guidance document: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2018/02/WC500244609.pdf