Clinical trials in Alzheimer disease: Debate on the use of placebo controls

During the past 10 years, there has been a rapidly growing number of pharma ceutical industry-sponsored drug trials for treatment of Alzheimer disease (AD) and other neurodegenerative diseases. As public awareness and concerns about AD have grown, so has interest in developing drug therapies for reta rding symptom progression, delaying onset, and ultimately curing the diseas e. Ethical debate on the use of placebo control trials in AD research has c ome of age in the United States with the availability of treatments approve d by the Food and Drug Administration. The experts and the public agree tha t more effective therapies are necessary, and new therapeutic options are b eing developed as rapidly as possible. The arguments on each side of the de bate are provocative and important but do not provide unequivocal justifica tion for either the abandonment or the maintenance of placebo-controlled tr ials in all AD research. Clinical trials differ with respect to scientific and practical goals, and these factors inherently affect the ethical priori ties of each study. We present these contrasting points of view to delineat e some of the issues rather than to make specific recommendations other tha n to urge that all clinical trials in AD should be designed with careful co nsideration of the ethical issues surrounding the use of placebo controls. As new and more effective treatments emerge, the ethical framework for plac ebo use in AD studies will require frequent re-examination. To make wise ch oices, patients, caregivers, physicians, and ethicists (among others) must have a voice in this continuing discussion.