The Food and Drug Administration is looking to make it easier to bring early-stage Alzheimer’s disease treatments to market.
In an article in the New England Journal of Medicine, which appeared online March 13, two physicians in the FDA’s Center for Drug Evaluation and Research outlined how treatments targeting patients at the very early stages of the disease could be reviewed under an accelerated approval pathway that allows drugs that address an unmet medical need to be approved on the basis of an intermediate clinical endpoint. The approval would be made on the condition that postapproval studies must be conducted to verify the clinical benefit of the drug (N. Engl. J. Med. 2013 March 13 [doi:10.1056/NEJMp1302513]).
Under this proposal, the FDA would consider the impact of a drug based on cognitive outcomes alone. This is a significant departure from the current regulatory framework, which requires any claims of improved cognition to include evidence of improvement in function. In early Alzheimer’s disease patients who have yet to experience the onset of overt dementia, it’s often impossible to show a functional improvement.
"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," Dr. Nicholas Kozauer and Dr. Russell Katz of the FDA wrote.
The agency recently issued draft guidance to the drug industry on how to conduct clinical trials involving early-stage Alzheimer’s patients who do not present with dementia. In the guidance, the FDA suggested evaluating early-stage disease patients using a single scale that combines assessment of cognition and function. For instance, the Clinical Dementia Rating–Sum of Boxes score is a good tool that evaluates patients based on six domains of cognition and daily functioning, according to the FDA.
mschneider@frontlinemedcom.com
On Twitter @MaryEllenNY