EMA Highlights: New guideline on Alzheimer’s medicines

by Gary Finnegan: The European Medicines Agency has released a revised guideline on medicines for the treatment of Alzheimer’s disease and other forms of dementia. The publication is part of a public consultation which runs until the end of July.

The move is a sign of the mounting concern at the growing burden of dementia and the lack of a major breakthrough in treating Alzheimer’s despite decades of investment in research.

According to the World Health Organization, 35.6 million people have dementia worldwide and this number is expected to double by 2030.

Recent progress in understanding the pathophysiology of Alzheimer’s disease suggests that the biological changes associated with the disease start to occur as early as 10 to 20 years prior to the emergence of clinical symptoms.

Experimental medicines should therefore be evaluated in earlier disease stages as certain treatments may be more effective at that stage than later in the illness, according to the EU’s drug watchdog.

The Agency says it considers dementia as a key public health priority and follows a multi-stakeholder approach to facilitate research and development of more effective medicines.

The revised guideline takes into account comments received at the EMA’s workshop on the clinical investigation of medicines for the treatment of Alzheimer’s disease in November 2014.

This workshop brought together a wide range of stakeholders, including patient representatives, regulators, pharmaceutical industry and independent experts. The aim of the workshop was to ensure that during the revision of its guideline, EMA would be able to consider the most up-to-date scientific developments in understanding and treating Alzheimer’s disease and views from experts in the field.

The revised guideline also builds on EMA scientific advice provided for a number of specific development plans for Alzheimer’s disease in recent years, as well as the qualification of several biomarkers for the selection of patients in clinical trials.

The revised guideline specifically addresses the:

• impact of new diagnostic criteria for Alzheimer’s disease, including early and even asymptomatic disease stages, onclinical trial design;
• choice of parameters to measure trial outcomes and the need for distinct assessment tools for the different disease stages in Alzheimer’s (different signs and symptoms, differences in changes over time, severity);
• potential use ofbiomarkers and their temporal relationship with the different phases of Alzheimer’s disease at different stages of medicine development (mechanism of action, use as diagnostic test, enrichment of study populations, stratification of subgroups, safety and efficacy markers etc.);
• design of long-termefficacy and safety studies.

After the consultation period, the EMA will review all comments before finalising the new guideline.