The FDA has drafted new guidelines for its Alzheimer’s research drug approval process. In response to the insights learned through decades of research, the FDA is placing a higher importance on early detection and the role of biomarkers in Alzheimer’s.
Learn more about what prompted the FDA to draft new guidelines and how it may affect future clinical trials.
FDA Redefining Success for Alzheimer’s Research
The Food and Drug Administration (FDA) is changing its approach to evaluating drugs for Alzheimer’s disease and other neurological conditions. Since the 1990s, the FDA has required clinical trials to prove that a new drug can improve the cognition and function of a person with the disease. However, as researchers understand more about the nature of Alzheimer’s, it has become clear that this standard will not work if we are to stop Alzheimer’s before it starts.
In the earlier stages of Alzheimer’s and sometimes even into the mid-stages of the disease, there is often no issue with daily function. In fact, researchers agree that functional impairment is often not recognized until Alzheimer’s has progressed into the third stage.
Thus, Alzheimer’s drugs were failing to meet FDA standards because they could not show an improvement in functional impairment since there was no functional impairment present in early Alzheimer’s.
New Approach Applied to Different Stages of Alzheimer’s
In most other cases, the FDA requires only one endpoint. This is now the suggestion being made for Alzheimer’s.
The FDA is striving to modernize its approach, taking into account what we now know about the disease to draft guidelines for three stages of early Alzheimer’s and the drug development for each stage.
According to newly drafted FDA guidelines:
- Individuals with Stage 1 Alzheimer’s show no clinical symptoms of the disease, but do have biomarker evidence of it. Biomarker evidence includes positive PET scans showing beta-amyloid or tau proteins. For this stage, improvements in biomarkers would serve as the basis for drug approval.
- People in Stage 2 of Alzheimer’s show biomarker evidence of the disease and signs of cognitive impairment but are not showing signs of functional impairment. For drug approval in this stage, the FDA will consider “strongly justified arguments” for using only improvements in cognition as a benchmark.
- Individuals in Stage 3 Alzheimer’s have biomarker evidence of the disease and show cognitive and functional impairment. For these trials, approval would be based on improvement in cognition and function.
The revised FDA guidelines reflect decades of research and studies, showing the importance of early identification and the role of biomarkers in diagnosis.
Researchers conducting clinical trials must now specify and identify the stage of Alzheimer’s in each participant at the time of enrollment and use biomarkers for participant selection and to monitor disease progression as well as the effectiveness of the drug.
How do you think the new FDA standards will affect Alzheimer’s research and the search for a cure? We’d like to hear your thoughts in the comments below.