For the first time in the history of the mental health disciplines, major resources are flowing into treatment research to attempt to determine what works in everyday clinical practice.
Over the last three years, the National Institute of Mental Health (NIMH), by far the largest funder of mental health studies, has instituted what it calls a "public health" emphasis--a veritable sea change to fund studies on what works in practice.
The new emphasis breaks sharply with traditional treatment studies that often look at select patients in academic settings: It will study large numbers of diverse patients in real-world settings, follow them for lengthy periods of time and measure progress by the patients' functioning in school, work and other areas of life.
"This should be revolutionary," says NIMH Director Steven Hyman, MD. "In the next few years, we should be equipping practitioners with things that can really work in their hands. I am hoping we will see a substantial difference in practice areas like adolescent depression and manic depressive illness within five years."
NIMH is directing more than $40 million annually to this public health emphasis and it has rewritten nearly all of its treatment intervention funding announcements to focus future treatment research in this area. It's also offering a large variety of support to help the field build expertise in this research.
Many researchers are hailing NIMH's new emphasis on public health--an idea that's been pushed for more than a decade. The need to move studies toward real-world conditions is "the consensus that has emerged in the field," says Boston University psychologist David Barlow, PhD, a leading researcher in psychopathology.
In fact, say some, this new emphasis could make significant differences for practice fairly quickly.
"My guess is that over the next five years we will get a much better idea of treatments of choice for each disorder, both psychological and pharmaceutical," says former APA President Martin E.P. Seligman, PhD, who has advocated for this change for years.
And if the appropriate rigor is maintained, says Philip Kendall, PhD, professor of psychology at Temple University and editor of APA's Journal of Consulting and Clinical Psychology, this kind of research is likely to "help bridge the gap between practice and science."
Some practitioners do have concerns with the new initiative, however. They worry that the new research focus will have too narrow an entrance point and therefore narrow results.
"My concern," says Dorothy Cantor, PsyD, a practitioner in private practice in Westfield, N.J., and a former APA president, "is that much of this new research may only look at a limited band of manualized-type treatments because only manualized-type treatments lend themselves to typical research designs."
SAMPLING OF PSYCHOSOCIAL CONTENT
The majority of the new "public health" funding is targeted to single-site grants for investigator-initiated studies, often for developmental efforts.
A small sampling of the work the agency has called for over the last year includes:
Work on interventions for disorders that have few good treatments: hypochondriasis, anorexia, trichotillomania and other self-mutilating disorders, dissociative disorders, borderline personality disorder, childhood feeding disorders or autism.
Exploratory testing of common but relatively unproven self-treatments, such as self-help or support groups.
Development of interventions for understudied high-risk behaviors and events, such as suicide attempts or exposure to traumatic events and understudied mental disorders and behavioral problems.
Studies on whether psychoeducational approaches work for all types of families and when individual family psychoeducation should be used instead of group.
Examining risk and protective processes associated with co-morbid mental and medical disorders, such as heart disease and depression, anxiety disorders and cancers.
Research collaborations between academic health centers and health-care organizations oriented to community care, with access to large, stable and diverse patient populations.
Services research on how the social, cultural and psychological characteristics of the provider interact with those of the client and how the economic, social, political and cultural environments of services affect organization and financing, the process and outcomes of care.
Integration of basic behavioral science and public health expertise in collaborative research on mental health and disorders.
Meanwhile, in the other major mechanism by which it is funding the initiative, NIMH has already launched four large clinical trials on real-world effectiveness on a scale never before seen in mental health research. The institute will devote $100 million over five years to studies seeking to improve treatment of about 5,000 patients with bipolar disorder, about 4,000 patients with depression unresponsive to standard medication and about 1,800 patients with schizophrenia and Alzheimer's disease unresponsive to antipsychotic medication. A smaller trial will compare the effectiveness of established treatments in 432 youths with major depressive disorder. These large trials will include both medication and psychosocial treatment approaches.
Conducted at numerous research sites over five years, the studies are of the magnitude one might expect for cancer or heart disease research (see chart, page 32). Previously, the largest mental health treatment study had a few hundred patients and most clinical trials lasted only six to eight weeks.
EXPLAINING THE NEW PUBLIC HEALTH EMPHASIS
This public health emphasis is a pioneering venture in "effectiveness" research. Although the long-established efficacy research looks at patients in inpatient hospital settings--often in academic health centers--the new "effectiveness" research will study patients in diverse settings.
And whereas efficacy research carefully controls the types of patients allowed into the studies to ensure that comparisons are legitimate, the new effectiveness studies will look at people who live in the community, come from diverse backgrounds and have co-occurring disorders or atypical illness patterns. To compensate for that variation in patients, the newer clinical trials will enroll large numbers of people to be sure that the findings have real meaning and apply to entire population groups.
In previous studies of depression, "We would have a fairly narrow age range," says Hyman. "We would exclude anyone with heart disease, anyone who drank more than a certain amount of alcohol, anyone with a co-occurring anxiety disorder. And pretty soon the population in the trials would not be representative for people who come into treatment."
The new clinical trials will follow patients for years rather than weeks to track rates of remission, side effects and adherence to treatment. Rather than measuring improvement only on a symptom-rating scale, the research will rate functional outcomes, such as how the patients are doing at work or school and personal relationships, whether they reduce their use of health care and if they have a better quality of life.
And, of critical importance, the new studies will look at whether treatments will be cost-effective.
"We should be able to make the case that these treatments deserve to be part of any reasonable insurance," says Hyman.
At the same time, he emphasizes that classical efficacy research will not be replaced. The older types of studies help to initially identify effective treatments. Researchers in the field explain that the efficacy research "informs" the effectiveness research.
In addition, beyond the efficacy and effectiveness studies of treatment in patients, the new public health emphasis will look at systems of care. Such work will provide new support for "practice research" to scrutinize what really happens to patients seen by clinical care providers; how health organizations' structures affect patient care; how practice and care systems might be optimized; and how to incorporate interventions into widespread practice.
In addition, "systems research" funding will underwrite studies on topics such as the cost of different kinds of care to an entire system and the effect of legislation and other public policy on organizations and delivery of care.
In one of its calls for applications, NIMH points to growing evidence that for mental health services research "the assumptions and the approaches of the last decade are not significantly improving the quality of care, knowledge assimilation or outcomes."
"We can have all the best treatments in the world, and if nobody ever uses them, what's the point?" says Grayson Norquist, MD, MSPH, head of the new NIMH Division of Services and Intervention Research (DSIR), which is in charge of the public health research area. "We've got to understand what it is that makes a difference, and why both providers and consumers will use treatments. How do we get systems to change their behaviors to ensure that these good things are being done?"
BRIDGING SCIENCE AND SERVICE
NIMH's willingness to tackle these research goals is what makes the institute's new focus seem like a break with history, particularly among practicing psychologists who have long been frustrated by research's inapplicability to their work with patients.
Even though over the last several decades traditional efficacy studies have shown that mental illnesses can be treated successfully with specific interventions, after the research is published, practitioners say they can't replicate the treatment. Often they lack the training or money, their patients don't match the specifications of the studies or their patients have co-occurring health problems.
At the same time, the research literature often hasn't offered practitioners definitive evidence for making some of the most important decisions in daily practice, such as what to recommend when initial treatment fails.
For years, NIMH tried to move some of its research closer to real-world applications, but the pivotal moment came in 1996 when Hyman became NIMH chief and made the shift one of his priorities.
Then, with remarkably fortuitous timing, Congress significantly increased the National Institutes of Health budget over several years. Hyman pushed the new monies for NIMH into a few areas that showed promise, including genetics and functional magnetic resonance imaging for studying brain function in living subjects. But he also emphasized public health research.
Hyman also reorganized NIMH and created DSIR as one of the three grant-making divisions, to focus on treatment research, including traditional efficacy research and the new public health emphasis.
From 1997 to 2000, funding for the division rose from $151 million to $194 million, and most of the new money is in the public health emphasis area, according to Norquist.
Hyman also pulled together a task force from the NIMH advisory council to identify the types of research needed to understand effectiveness and to write a strategy for supporting them. That report, "Bridging Science and Service," called for impressive modifications in how the agency should lead treatment research.
"We are making every effort to do whatever is required to address every single one of those recommendations," says Norquist.
Those recommendations include advice for NIMH to support studies on:
Adherence to treatment procedures on the part of clinicians and service settings. For instance, cognitive therapies have been shown to be efficacious with 12 to 20 sessions. But in settings where only six sessions are permitted, what should clinicians to do?
The various treatment guidelines in the field--including those developed by managed-care organizations--to ensure that the guidelines are scientifically based and sufficiently specific.
Methods for finding the best measurements of whether patients have improved.
Provider and patient treatment preferences, how they are incorporated into treatment decisions and the impact they have.
EXCITEMENT AND WORRY
The NIMH reforms have induced a veritable brew of anticipation and trepidation in psychology researchers and practitioners.
"The NIMH clearly has enormous power to influence the direction of the field," says Marvin Goldfried, PhD, of the State University of New York at Stony Brook.
He is concerned in particular that the research be done in a way that leads to better treatment manuals, but also that the manuals be written to give the therapist sufficient leeway to intervene, depending on the case at hand.
Ronald Fox, PhD, chair of APA's Committee for the Advancement of Professional Practice, also supports the research but has similar worries.
"Explicit protocols are good for research proposals, but in these days of managed care, we should not get hung up on blind adherence to a treatment guideline."
Daniel Abrahamson, PhD, chair of an APA Board of Professional Affairs work group developing criteria for evaluating treatment guidelines, agrees, saying, "All we can hope to do in a research program like this is answer some of the more commonly seen questions."
Kirk Schneider, PhD, a member of APA's Div. 32 (Humanistic) wonders if the initiative will allow studies to address the wide range of therapies and in particular "the longer-term exploratory psychotherapies which have had such difficulties being funded for research."
He fears that the new research will find short-term therapies effective because they help people "adjust" and the studies will never look at "intensity of therapeutic change, sense of passion, aliveness, vibrancy about one's job, education or relationship."
At the same time, some researchers have worried for some time about a loss of control as the research moves to more varied populations and "naturalistic" settings.
"I am both excited and terrified," says Thomas Borkovec, PhD, of Pennsylvania State University. "My terror is that we will lose validity. My main concern is that as a lot of money will start going into effectiveness research and the work will not have sufficient internal validity and that the conclusions drawn from it will not be accurate."
A BRIDGE TOO FAR?
Considering a history of less than perfect communication between practitioners and researchers, some mental health professionals may also question whether the two can be brought together enough to inform this process and really make research applicable to practice.
Some practicing psychologists believe it can be done but don't feel the agency has done enough to encourage it so far. Stephen Ragusea, PsyD, a practicing psychologist in Pennsylvania and a part of a practice-research network, says he wishes practitioners had been pulled in more on NIMH's initial formation of the public health emphasis.
"I applaud this movement, as long as full-time private practitioners are involved from the beginning in choosing what subjects will be researched," says Ragusea.
Fox stresses, "The clinical experience of people who have been working in the field is an important source of data."
NIMH's Norquist is more positive.
"I think this new emphasis is forcing that bridge to develop," he says. "Clinicians understand that if they don't have this kind of information, they are not going to be able to do the right thing by the patients. Also, nobody is going to pay them any more for this unless we show that these things really do make a difference in populations. And researchers understand now that they can't do research just on very select, very homogeneous populations. They have really got to be relevant to the community, or else who's going to pay for their research?"
For his part, Hyman recognizes some researchers' doubts about the amount of money being spent on this type of study and about the ability to maintain scientific rigor in the midst of the real-world complexities.
"But," he says, "I would argue that not to try our best as a community, as a field, would really be to give our people who need treatment, who need preventive intervention, no chance at all."
There are three major reports that lay the foundation for the public health initiative from NIMH. These reports are "A Plan for Prevention Research for the National Institute of Mental Health," "Bridging Science and Service," and "Translating Behavioral Science into Action: Report of the NAMHC." All are available for ordering or downloading at: http://www.nimh.nih.gov/publist/puborder.cfm#long.
In addition to those reports, NIMH officials wrote two commentaries on the public health initiative in the April 1999 issue of the APA electronic journal, Prevention and Treatment. The commentary titles are: "Expanding the Frontier of Treatment Research," and "NIMH Support of Psychotherapy Research: Opportunities and Questions." They are at: http://journals.apa.org/prevention/.