Use of placebos in clinical trials involving children and adolescents.

Abstract

Medications are commonly prescribed to treat child and adolescent psychiatric disorders, despite limited research about the safety and efficacy of their use in this population (1). The lack of data on medication treatment creates a dilemma for the physician. Added to this dilemma are pressures on the physician to treat patients quickly and with the least costly intervention, a circumstance that is often interpreted to mean that medication should be prescribed early in the treatment process. Clearly, additional data are needed to improve our database on the safety and efficacy of psychopharmacologic agents for children and adolescents and to serve as a rational guide for clinical treatment. At the same time, controversy has developed about the use of placebos in psychopharmacological clinical trials involving children and adolescents as subjects. Some investigators have proposed that the use of placebo treatment is unnecessary and unethical, serving only to deprive subjects of active treatment; a version of this position has been outlined by Rothman and Michels (2). In fact, some have proposed that it would be preferable to use an active placebo, that is, a medication or a medication dosage thought to be ineffective in the treatment of the study condition. Unfortunately, child and adolescent psychiatric disorders are not well understood, particularly because of the lack of data on the course and the outcome of these disorders. As such, it is impossible to predict changes in symptoms for individuals or groups of individuals within any sample, much as is the case in research on adult psychiatric disorders (3). Therefore, without a placebo control within the study, researchers have no basis for judging whether a given study medication was associated with improvement or worsening of symptoms. Furthermore, subjects in child and adolescent trials often have a high rate of response to placebo. This paper summarizes the findings of two recent studies—one part of a controlled trial and the other a naturalistic study. The findings highlight the need for clinicians to exercise caution in prescribing medication early in the course of assessment and emphasize the need for placebo in clinical trials.