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By Steven E. Hyman, M.D.
Director
National Institute of Mental Health
A fundamental goal of research funded by the National Institute of Mental Health (NIMH) must be to provide the necessary information for psychiatrists and other mental health professionals to take the best possible care of our patients. Achieving this goal, however, is more difficult than it might appear.
The traditional treatment study funded by NIMH, and parallel studies supported by the pharmaceutical industry, adhered to what might be called a regulatory model. Under this approach, the aim is to demonstrate the intrinsic efficacy of a treatment and the trade-off of that efficacy against side effects. Carefully controlled, randomized, double-blind clinical trials remain a cornerstone of research sponsored by NIMH. As practitioners are well aware, however, such studies cannot be the end of treatment research but a beginning. The need for information more relevant to the real world is underscored by managed care organizations and, perhaps more importantly, by the perceptions and experiences of employers who pay the insurance bills for many Americans.
Our traditional clinical trials entail relatively small samples. To ensure rigor and maximize the possibly of detecting a treatment effect, these trials have excluded anyone with comorbid disorders. Thus, the typical clinical trial for an antidepressant might occur in outpatients, usually in an academic health center; patients with any excessive alcohol or drug use or a co-occurring medical disorder would be excluded. The major outcome criterion would be a decrement on a behavioral rating scale such as the Hamilton Depression Scale.
In real life, of course, our patients don't read textbooks, and the patient who typically appears in a psychiatrist's office is quite unlike the patient enrolled in the traditional clinical trial. Accordingly, while NIMH will continue to fund the traditional form of clinical trial in those areas that are complementary to the work of industry, we also must adapt to the changing nature of treatments, patients, and the health care environment. To help psychiatrists and other clinicians provide optimal care to patients, we need to conduct trials with larger sample sizes and with fewer exclusion criteria, ensure that trials occur not only in academic clinics but also in more real-world settings including managed care settings, and assess outcome not only on the basis of symptom reduction but also on measures of functional rehabilitation- the end result that is of greatest interest to employers and families, as well as to patients.
This new type of trial-often called an "effectiveness" trial-need not give up any of the traditional and indispensable emphasis on rigor. Patients should be enrolled prospectively and randomized. Patients and investigators alike can and should be blinded to treatment conditions. In trials of both pharmacotherapies and psychotherapies, the information sought should be geared toward helping clinical decision-making in real-world settings and should demonstrate the types of functional outcomes that managed care organizations and employers find compelling.
The research that is needed will require additional funds, but I believe the expenditure is critical. The shared goals of NIMH, APA, and other clinical professions demand that we do the best that is possible for our patients. Toward this end, I have repeatedly described emerging needs in the area of clinical trials, and our proposed response, in congressional testimony and have found members of Congress to be most receptive; nonetheless, I am committed, if necessary, to redirecting funds within NIMH from less productive areas to enhance our clinical trial capacities. In future columns, I will describe other NIMH priorities.