title: Designing dementia treatment studies: Diagnosis, efficacy criteria, and duration.
abstract: Development of treatments for dementia is beset by special problems in defining the diagnosis, establishing efficacy criteria, and specifying the necessary duration of study. There is need for agreement among clinicians and scientists on diagnostic subgroups of dementia. Similarly, there is a need for harmonization of the regulatory guidelines in Europe, Japan, and the United States regarding the decision set of variables on which to base efficacy claims. The duration of trials must be based upon the intended claim: transient symptomatic benefit, maintained symptomatic benefit, or a therapeutic effect on disease progression. Claims other than transient benefit require long-term trials, suggested to be of at least six months in duration. Problems with long-term studies include slow patient accrual, high dropout rates, changing milieu, low return on investment, and lack of unanimity regarding regulatory requirements. Regulatory authorities must come to some accord, consonant with current clinical/scientific wisdom and consensus, regarding diagnosis, efficacy criteria, and feasible study duration, in order to attract continued sponsor investment in the development of antidementia treatments.
authors.affiliation: Sandoz Pharma Ltd, Drug Registration and Regulatory Affairs, CH-4002 Basel, Switzerland.
authors.forename: R
authors.initials: R
authors.lastname: Spiegel
authors.forename: P
authors.initials: P
authors.lastname: Irwin