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The evaluation of disease modifying therapies in Alzheimer's disease: a regulatory viewpoint
Author(s) -
Mani Ranjit B.
Publication year - 2004
Publication title -
statistics in medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.996
H-Index - 183
eISSN - 1097-0258
pISSN - 0277-6715
DOI - 10.1002/sim.1718
Subject(s) - disease , clinical trial , medicine , drug development , dementia , surrogate endpoint , alzheimer's disease , drug trial , drug , food and drug administration , intensive care medicine , modalities , pharmacology , pathology , social science , sociology
Several drugs have received marketing approval in this country for the treatment of dementia of the Alzheimer's type. Their approval has been based on clinical trial designs that do not permit a distinction to be made between an effect of the drug on the symptoms of that disease, and an effect on the pathophysiological mechanisms that underlie that disorder. The latter effect has been referred to as ‘disease‐modifying.’ In recent years there has been considerable interest in developing disease‐modifying treatments for Alzheimer's disease (AD), using either specific clinical designs, or surrogate markers, such as brain imaging modalities. This paper outlines the regulatory framework governing how the Food and Drug Administration addresses new drug claims, the current basis for approving drugs for the treatment of AD, clinical trial designs that have been proposed as a means of demonstrating disease‐modifying effects, a general and regulatory background to the use of surrogate markers in drug development, and, finally, views about the possible role of surrogate markers, especially brain imaging, as outcome measures in clinical trials intended to produce disease‐modifying effects in Alzheimer's Disease. Copyright © 2004 John Wiley & Sons, Ltd.