EMA develops guidance on developing treatments for Alzheimer's 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 European Medicines Agency (EMA) has released a discussion paper on revising its guideline on developing medicines for Alzheimer´s disease (AD) and other dementias.
A key issue is helping guide pharmaceutical companies which are developing medicines for the early stage of such diseases, when there may be a better chance of treating them.
EMA announced last year that it wanted to amend its guidance and a public EMA workshop on the issue was to be staged on 24–25 November this year, after which formal fresh guidance will be proposed.
The discussion paper notes that 'there is now a consensus that treatment options should be evaluated in earlier disease stages before the full picture of dementia is reached'.
Treatment options should be evaluated in earlier disease stages before the full picture of dementia is reached
And while it says general guidance for drug development in mild to moderate and severe Alzheimer dementia is still valid, the Agency wants to integrate its advice for medicines that treat earlier stages of the disease, 'including patients with preclinical and prodromal AD...'.
The discussion paper assesses issues including:
- The impact of new diagnostic criteria for AD, including early and asymptomatic disease stages on clinical trial design;
- Choosing outcome parameters and need for distinct assessment tools regarding different AD disease stages;
- Designing long-term efficacy and safety studies;
- Combination therapy and corresponding study designs;
- Stand-alone symptoms, such as agitation, aggression, depression and sleep-wake cycle disturbances; and
- The potential use of biomarkers and their relationship with different AD phases and associated drug development.
The EMA paper acknowledged 'the current uncertainties regarding the pathophysiology of AD, the relevance of biomarkers and the definition of various stages of AD'.