This article is more than 5 years old. Information may no longer be current.

FDA plans to relax rules on Alzheimer’s drug approvals

The FDA is set to ease restrictions on drug approvals for the treatment of Alzheimer’s disease, proposing that companies will not have to demonstrate in clinical trials evidence of improved daily functioning in patients in the early stages of the disease — only improvements in cognition.

The proposal, recently published in The New England Journal of Medicine, constitutes a shift in the FDA’s position on the development of Alzheimer’s drugs. The agency has long required that studies show clinically meaningful outcomes, including improvements in how patients feel and function, besides improvements in cognition.

“However, the premise that effective cognitive improvement will be manifested in the functional assessment of patients is untenable in the case of early-stage Alzheimer’s disease, which is increasingly the target of drug-development efforts,” the FDA said in the proposal. “We simply do not yet have drug-development tools that are validated to provide measures of function in patients with Alzheimer’s disease before the onset of overt dementia.”

The accumulation of amyloid plaque in the brain has been the pathologic hallmark of Alzheimer’s disease progression, and recent drug development efforts have focused on this biomarker. However, amyloid deposition has been associated more with late-stage Alzheimer’s disease. Attempts to intervene with patients with overt dementia may be too late, which could account for a number of failed drug trials, according to the FDA.

However, easing restrictions on drug approvals may present new challenges, including how to measure improvements in cognition in patients who are in the early stages of Alzheimer’s disease. Last month, the FDA issued a draft guidance on the development of new drugs for the treatment of Alzheimer’s disease. The draft also included recommendations on clinical outcome measures, trial designs and the role of biomarkers.

The draft guidance is still open to public comment and can be accessed here.

“As the focus of drug development has shifted to earlier stages of Alzheimer’s disease, many new and challenging scientific questions have emerged, and the regulatory framework under which such therapies are evaluated should evolve accordingly,” the agency said. “The FDA remains committed to innovative approaches to the evaluation of drugs that are in clinical development.”