EMA proposes updating guideline for Alzheimer's disease treatments

The European Medicines Agency (EMA) is proposing to develop a new guideline on Alzheimer’s drugs (AD) to reflect recent advances in the field, including the approval of a new Alzheimer’s treatment in the EU and learning from several marketing authorization applications (MAAs).

“Dementia represents one of the most devastating healthcare problems faced by Europe and has been recognized as public health priority by the World Health Organization (WHO) since 2017,” EMA wrote. “Alzheimer´s disease (AD) is by far the most common form, affecting 60 to 80% of the individuals living with dementia. Treatment options are still limited and no treatment has been shown to stop the progression of clinical signs and symptoms to date.”

EMA noted that its current guideline needs to be revised to incorporate recent developments in the definition of AD, which have “important implications” for designing and conducting clinical trials across all stages of sporadic AD.

The update will replace the Guideline on the clinical investigation of medicines for the treatment of Alzheimer’s disease issued in February 2018.

The paper states that “the ongoing development and validation of plasma biomarkers raises the potential of using biomarkers not only in the research but also in clinical settings to assist the diagnostic work-up of patients with suspected AD.”

Recently, there has been a proposal to shift AD diagnosis towards a purely biological model and to replace the previous clinical-biological definition. These biological models of AD, which incorporate recent advances in biomarkers, are suggested as a means to bridge the gap between research and clinical care, aligning with current scientific understanding. However, these new criteria are not consistent with the recommendations of clinical practice guidelines, particularly concerning the asymptomatic stage of the disease.

The recent experience with MAAs for anti-amyloid treatments, including Leqembi, which has now been authorized in the EU, underscores the necessity of planning for long-term data regarding both safety and efficacy, EMA said.

The proposed updated guideline will cover the following areas:

The evolution of diagnostic criteria and its potential implications for defining the target population and setting eligibility criteria for clinical trials;
The selection of appropriate endpoints, especially for early stages of the disease, since currently used scales have been developed for later stages of the disease;
Recommendations for the context of use of biomarkers, including biomarkers for patient selection (diagnostics and for enrichment), and for monitoring treatment response

EMA’s Central Nervous System Working Party (CNSWP) will develop the draft guideline along with the Methodological Working Party (MWP), Scientific Advice Working Party (SAWP) and other relevant WPs and committees.

EMA plans to release a draft CHMP guidance document for public consultation in the fourth quarter of 2026.

The deadline for submitting comments is 28 February 2026. Comments should be provided using this EUSurvey.

Concept paper

EMA proposes updating guideline for Alzheimer's disease treatments

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