Clinical research designs for emerging treatments for Alzheimer disease

Moving beyond placebo-controlled trials

David Knopman, Jeffrey Kahn, Steven H Miles

Research output: Contribution to journalArticle

21 Citations (Scopus)

Abstract

The design of clinical trials must evolve as new therapies become available. The demonstrated efficacy and clinical use of donepezil and vitamin E for Alzheimer disease (AD) has shifted the options for AD research design. There is now a compelling case for alternatives to trials that include a treatment arm with no active therapy (ie, a placebo control). With an existing therapy, such as donepezil or vitamin E, new drugs that are clearly superior to those drugs should be sought. Combination therapy is a likely strategy for the future, implying that clinical trials, if possible, should replicate actual practice. The long duration of future AD trials also will make placebo-controlled trials more difficult to justify and more difficult to recruit for. Add-on or active-control designs represent the alternative approaches. We believe that definitive clinical trials of new AD drugs that use one or the other of these designs would be more likely to bring about therapeutic advances than would comparisons with inactive treatments. Our argument is not a general rejection of placebo-control designs. Our recommendations apply only to the circumstances in which the field of AD drug therapy now finds itself.

Original languageEnglish (US)
Pages (from-to)1425-1429
Number of pages5
JournalArchives of Neurology
Volume55
Issue number11
DOIs
StatePublished - Jan 1 1998

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Alzheimer Disease
Research Design
Placebos
Clinical Trials
Vitamin E
Therapeutics
Pharmaceutical Preparations
Controlled
Therapy
Placebo
Alzheimer's Disease
Clinical Research
Drugs
Drug Therapy
Vitamins
donepezil

Cite this

Clinical research designs for emerging treatments for Alzheimer disease : Moving beyond placebo-controlled trials. / Knopman, David; Kahn, Jeffrey; Miles, Steven H.

In: Archives of Neurology, Vol. 55, No. 11, 01.01.1998, p. 1425-1429.

Research output: Contribution to journalArticle

Knopman, David ; Kahn, Jeffrey ; Miles, Steven H. / Clinical research designs for emerging treatments for Alzheimer disease : Moving beyond placebo-controlled trials. In: Archives of Neurology. 1998 ; Vol. 55, No. 11. pp. 1425-1429.
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