On 1 February 2016, the European Medicines Agency (“EMA”) launched a public consultation concerning a “Draft guideline on the clinical investigation of medicines for the treatment of Alzheimer’s disease and other dementias“.

Scope of the draft guideline

The purpose of the draft Guideline is to provide guidance concerning the evaluation of medicinal products for treatment across the Alzheimer’s disease (“AD”) continuum. The document also includes a discussion of development strategies for disease prevention. The usefulness of combination therapy targeting multiple pathophysiological mechanisms and their corresponding study designs is also addressed.

AD is the most common cause of dementia, followed by vascular dementias (“VaD”) or mixed forms of AD and VaD.

New approach to AD treatments

The draft guideline concludes that the biomarker field is evolving and that the possibility to detect disease changes and progression in vivo, opens new regulatory scenarios including the possibility to intervene directly on the neuropathology before the appearance of symptoms. This is the reason why treatment options should be evaluated in earlier disease stages before the full picture of dementia is reached. Therefore, the draft Guideline proposes to address the following points:

  • 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 for the different disease stages in AD (different signs and symptoms, differences in progression rate);
  • Potential use of biomarkers and their 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.);
  • Design of long-term efficacy (maintenance of effect) and safety studies.

The consultation in relation to this draft Guideline runs until 31 July 2016.