FDA draft guidelines suggest route to approval for early-stage Alzheimer's drugs

FDA draft guidelines suggest route to approval for early-stage Alzheimer's drugs

The Food and Drug Administration issued draft guidance Thursday on clinical trials for early-stage Alzheimer's drugs.

Alzheimer's disease involves subtle changes in the brain and few overt symptoms in its early stages. Therefore, choosing clinical trial participants and measuring whether a drug treatment results in improved feeling or function can be difficult.

Measuring levels of biomarkers such as β-amyloid and tau proteins has emerged as one way to track the progress of the disease. However, the FDA notes a lack of “widespread evidence-based agreement in the research community that an effect on a particular biomarker is reasonably likely to predict clinical benefit.”

The agency will therefore not consider biomarkers a primary outcome measure in clinical trials. However, the agency says it is “open to considering” biomarker levels as a secondary outcome measure, in conjunction with a primary clinical change. The FDA urged drug developers to conduct biomarker analysis, saying it is open to changing its guidelines.