The FDA Modernises Approach in Dealing With Alzheimer’s Disease
The week begins with good news after the FDA has finally announced a plan to modernise its approach to evaluating new drugs for Alzheimer’s and other neurologic diseases.
This will no doubt be welcomed by researchers working to develop much-needed medications for these conditions.
According to Stat News, the main change has to do with the endpoints the FDA requires in clinical trials. A loose standard in place since the 1990s required clinical trials to show that a new drug for Alzheimer’s disease improved patient’ cognition and function. However, this isn’t in line with our current understanding of the disease and was likely a contributing factor in the high rate of failure in Alzheimer’s trials.
Under the dual-endpoint standard, the FDA asked drug developers to demonstrate benefits to cognition among Alzheimer’s patients with symptoms of dementia that were “clinically meaningful” by demonstrating improvements in function. But, we are now able to recognise earlier stages of Alzheimer’s disease, including mild cognitive impairment, in which patients have no issues with daily function. In this large group of patients, it’s difficult, if not impossible, to show improvements in function because their function is fine.
In other diseases, the FDA requires clinical trials to focus on one endpoint. It is this that they are now applying to Alzheimer’s disease. In the statement outlining the draft guidelines, they outline three stages of early Alzheimer’s disease and offer guidance on drug development for each. This is a step forward in acknowledging advances in our understanding of Alzheimer’s as a whole.
The real dilemma now is to work out how to design Alzheimer’s clinical trials to meet these guidelines. It’s clear that trials must now specify and define the stage of Alzheimer’s disease among the population being studied at the time of enrollment, as well as the expected stage at its conclusion. More so, it is clear that biomarkers should be used as one criterion for trial enrollment, to monitor disease progression, and to indicate responses to the drug.
Now that the FDA has signaled that it is ready to accelerate the development of new drugs for Alzheimer’s, the challenge for us is to prove that they work.