Consumer, professional, legislative and regulatory organizations are increasingly calling for the development and adoption of evidence-based therapies, based on demands for quality services and expectations that outpouring of dollars and time are rewarded by beneficial outcomes. In child and adolescent mental health, growing public concerns over safety, in particular with psychotropic medications, and the recognition that psychiatric impairment is a major factor within other social service systems has further fueled the demand for empirically based interventions.
Randomized, controlled trials (RCTs) with adequate sample sizes and defined study populations are the standard for characterizing an intervention as evidence-based (Cochrane Collaboration, 2002). A listing of all RCTs in child and adolescent psychiatry is beyond the scope of this commentary (for a review, see McClellan and Werry ). This review will outline interventions with the best research support. Fortunately, although the literature remains limited, the number of well-conducted studies is increasing.
An estimated 6% of young people under the age of 20 in the United States receive prescriptions for psychotropic medication. This represents approximately a threefold increase since 1987 (Zito et al., 2003), and includes a substantial rise in prescriptions for preschoolers (Zito et al., 2000). The majority of prescriptions are off-label (i.e., not U.S. Food and Drug Administration-approved). Pediatricians and family practice physicians issue the majority of prescriptions for psychotropic drugs, in part due to the scarcity of child psychiatrists (Goodwin et al., 2001).
Stimulant medications for attention-deficit/hyperactivity disorder are the best-supported treatment, with more than 160 published, randomized, controlled trials (Greenhill et al., 2002). In addition, well-designed, National Institutes of Health-funded, multisite, randomized, controlled trials support the use of stimulant medications for ADHD (MTA Cooperative Group, 2004); fluoxetine (Prozac) plus cognitive-behavioral therapy (CBT) for major depression (March et al., 2004); risperidone (Risperdal) for behavioral disturbances in youth with autism (McCracken et al., 2002); and fluvoxamine (Luvox) for childhood anxiety disorders (Research Unit on Pediatric Psychopharmacology Anxiety Study Group, 2001). There are also several large trials supporting the use of selective serotonin reuptake inhibitors for obsessive-compulsive disorder (American Academy of Child and Adolescent Psychiatry, 1998). Beyond that, the research literature supporting most pediatric psychopharmacologic practices is limited in terms of the number of studies, sampling limitations and/or variability in results.
Perhaps the greatest area of recent concern is the use of antidepressants. For pediatric depression, published trials report efficacy for fluoxetine (Emslie et al., 1997; March et al., 2004), sertraline (Zoloft) (Wagner et al., 2003) and citalopram (Celexa) (Wagner et al., 2004). Paroxetine (Paxil) was superior to placebo on some, but not all, primary outcome measures of depression in one multisite trial (Keller et al., 2001). High placebo response rates and unpublished negative trials raise questions about whether the use of SSRIs for pediatric depression is clinically justified (see Whittington et al., 2004).