Commentary on “Health economics and the value of therapy in Alzheimer’s disease.” Patient-reported outcomes in clinical trials for Alzheimer’s disease

Abstract

a r e m L M a e c [ w m Food and Drug Administration regulatory requirements or outcome data in clinical trials for Alzheimer’s disease AD) have been driven by the biomedical model of the isease [1,2]. Outcomes pertaining to cognition, function, nd global clinical impressions of change have been favored 3] at the expense of data pertaining to emotional (eg, europsychiatric symptoms) or subjective outcomes (eg, uality of life, emotional well-being). It is not difficult to nderstand why biomedical factors took center stage in the arly development of pharmacologic interventions during he late 1980s. At the time, relatively less was known about uality of life (QOL) and emotional functioning in AD elative to cognitive and functional decline. Since that time, however, a great deal has been learned bout emotional [4], neuropsychiatric [5–7], and subjective spects of AD (eg, QOL [8–10]). Furthermore, many perons diagnosed with mild to moderate AD have the ability o provide valid and reliable self-report data about their eeds, preferences, and life quality [8,9,11]. They also can articipate in focus groups about outcomes assessment, ethcs, and QOL and participate in the evaluation and develpment of QOL instruments [8,12–14]. Even some persons ith advanced dementia can complete verbal report scales 7,8] and can report reliably via nonverbal measures [15] or by sing single item QOL rating scales [16]. We are entering an era in which patient-reported outome data are not only desirable but regarded as integral for complete understanding of the economic, social, and psyhological impact (and value) of therapeutic interventions 17] (also article by Frank in this issue). This new emphasis n subjective, patient-report outcomes, paired with the rowing realization that there is more to AD than cognitive nd functional impairment, is empowering for patients, who