Posted on March 14, 2013 · Posted in Brain Injury

Here’s a breakthrough, though not in the lab, that could tremendously boost research on Alzheimer’s drugs: The Food and Drug Administration plans to ease up its requirements to approve such medications.

It was actually more than a month ago, in early February, that the FDA announced its new draft guidance “designed to assist companies developing new treatments for patients in the early stages of Alzheimer’s disease, before the onset of noticeable (overt) dementia.”

In an article in the New England Journal of Medicine Wednesday, the FDA explained its proposed changes regarding Alzheimer’s drugs in more depth.

The bottom line is this: Research on drugs to prevent or halt Alzheimer’s disease keeps having setbacks. One of the problems, according to pharma companies, is that the FDA has been requiring proof that medications help not only patients’ cognitive functions, but also daily functions such as dressing and feeding, according to The New York Times.

But researchers have come to believe that catching the disease in its early stages offers the best chance of halting it. So the problem with the old FDA rules, The Times said, was “how can you show a drug works if patients are so early in the course of the disease that they have no obvious deterioration in their cognitive abilities or daily functioning?”

With the proposed changes in the drug approval process, patients in the early stages of Alzheimer’s could be studied with just cognitive tests, while those with some symptoms would undergo tests for cognition and function, The Times reported.

This loosening of the rules to get Alzheimer’s drugs to market could prompt some pharmaceutical companies to pursue creating such medications, and convince others to continue with their risky R&D.

The FDA’s press release in February about its plans had some details.

“The scientific community and the FDA believe that it is critical to identify and study patients with very early Alzheimer’s disease before there is too much irreversible injury to the brain,” Dr. Russell Katz, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, said in a statement in the agency’s press release. “It is in this population that most researchers believe that new drugs have the best chance of providing meaningful benefit to patients.”

The draft guidance titled, “Guidance for Industry, Alzheimer’s Disease: Developing Drugs for the Treatment of Early Stage Disease,” explains the FDA’s current thinking about the way researchers can identify and select patients with early Alzheimer’s disease, or those who are at risk of developing the disease, for participation in clinical trials.

The release noted that in recent years, scientists have tried to find ways to identify these patients using criteria that are based on biological indicators (biomarkers). Researchers have also tried to develop sensitive clinical measures that can detect subtle mental decline.

“This draft guidance is intended to serve as a focus for continued discussions between the FDA and pharmaceutical sponsors, the academic community, advocacy groups, and the public,” Katz said. “The FDA is committed to vigorously addressing Alzheimer’s disease and will work with industry to help develop new treatments in this early population as expeditiously as possible.”

For drugs designed to treat patients with overt dementia, the FDA currently requires that treatments not only show an effect on abnormal thinking, but also how well patients function, the release said.

“The goal for these trials is to ensure that any beneficial effect on thinking is associated with a clinically meaningful outcome for the patient, e.g., improvement or lack of decline in how patients feel or function,” the release said. “However, because patients with early Alzheimer’s disease have little-to-no impairment of global functioning, it is difficult to assess changes in function in these patients. This can make it difficult to determine if a given treatment’s effect is clinically important.”

The FDA said that it is seeking public comment on the draft guidance for 60 days, so there is about a month left to do so.

The agency said that its proposal is part of U.S. Department of Health and Human Services’ efforts under the National Plan to Address Alzheimer’s Disease, which calls for both the government and the private sector to intensify efforts to treat or prevent Alzheimer’s and related dementias and to improve care and services.

It responds to recommendations from a May 2012 HHS and National Institutes of Health Alzheimer’s research summit to conduct clinical trials in at-risk individuals without symptoms and to develop and validate new measures so that Alzheimer’s can be measured at the earliest possible time in the course of the disease.

About the Author

Attorney Gordon S. Johnson, Jr.
Past Chair Traumatic Brain Injury Litigation Group, American Association of Justice :: 800-992-9447