Revised guideline on clinical studies for Alzheimer’s disease medicines

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Revised guideline on clinical studies for Alzheimer’s disease medicines

Guidance expected to facilitate development of medicines to prevent and treat condition

The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a revised guideline on clinical studies for medicines that target Alzheimer’s disease. This document aims to provide guidance for the development of medicines across all stages of Alzheimer's disease.

Alzheimer’s disease, a condition that destroys brain cells and nerves, disrupting the transmitters which carry messages in the brain, is the most common cause of dementia in the elderly. According to the World Health Organization, 35.6 million people have dementia worldwide and this number is expected to double by 2030. It affects more than five million people in the European Union (EU).

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 before clinical symptoms start to appear. Many of the experimental medicines are therefore investigated in earlier disease stages as certain treatments may be more effective at that stage than later in the illness.

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.

Dementia is a key public health priority for EMA. The Agency promoted several initiatives to encourage a broad range of stakeholders to share their experiences and challenges, which were reflected in the revision of the guideline.

Before revising the guideline, EMA organised 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.

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.

The guideline also builds on scientific advice provided by the Agency to medicine developers on specific products and methodologies, such as the qualification of biomarkers for use in clinical trials and a longitudinal model describing changes in cognition in patients with mild or moderate Alzheimer’s disease.

EMA’s new guideline addresses, among others:

  • impact of new diagnostic criteria for Alzheimer’s disease, including early and even asymptomatic disease stages, on clinical trial design;
  • factors to be considered when selecting parameters to measure trial outcomes at the different disease stages in Alzheimer’s;
  • potential use of biomarkers in the various stages of medicine development;
  • design and analysis of efficacy and safety studies.

The guideline will become effective from 1 September 2018.

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