Treatment research at the crossroads: the scientific interface of clinical trials and effectiveness research.

OBJECTIVE Policy and clinical management decisions depend on data on the health and cost impacts of psychiatric treatments under usual care, i.e., effectiveness. Clinical trials, however, provide information on treatment efficacy under best-practice conditions. An understanding of the design, analysis, and conventions of both efficacy and effectiveness studies can lead to research that better informs clinical and societal questions. METHOD This paper contrasts the strengths and limitations of clinical trials and effectiveness studies for addressing policy and clinical decisions. These research approaches are assessed in terms of outcomes, treatments, service delivery context, implementation conventions, and validity. RESULTS Clinical trials and effectiveness research share problems of internal and external validity despite more attention to internal validity in clinical trials (e.g., randomization, blinding, standardized protocols) and to external validity in effectiveness studies (e.g., community-based treatments, representative samples). CONCLUSIONS To develop research at the interface of clinical trials and effectiveness studies, research goals must be redefined, and methods, such as cost-utility and econometric analyses, must be shared and developed. Development of hybrid designs that combine features of efficacy and effectiveness research will require separation of conventions such as frequency of follow-up, intensity of measurement, and sample size from the central scientific issues of aims and validity.

[1]  M. Weinstein,et al.  Recommendations of the Panel on Cost-effectiveness in Health and Medicine , 1997 .

[2]  J. Newhouse.  Free for All?: Lessons from the RAND Health Insurance Experiment , 1993 .

[3]  J. Hornberger,et al.  The economic impact of psychotherapy: a review. , 1997, The American journal of psychiatry.

[4]  J. Lave,et al.  Treating major depression in primary care practice. Eight-month clinical outcomes. , 1996, Archives of general psychiatry.

[5]  W. Katon,et al.  A multifaceted intervention to improve treatment of depression in primary care. , 1996, Archives of general psychiatry.

[6]  R. Russell,et al.  Psychotherapy research in historical perspective. Implications for mental health care policy. , 1996, Archives of general psychiatry.

[7]  Bruce G. Link,et al.  A comparison of two levels of family-aided assertive community treatment. , 1996, Psychiatric services.

[8]  W. Katon,et al.  Initial antidepressant choice in primary care , 1996 .

[9]  D. Antonuccio,et al.  Psychotherapy versus medication for depression: Challenging the conventional wisdom with data. , 1995 .

[10]  K. Schulz,et al.  Subverting randomization in controlled trials. , 1995, JAMA.

[11]  Joseph P. Newhouse,et al.  Does More Intensive Treatment of Acute Myocardial Infarction in the Elderly Reduce Mortality? Analysis Using Instrumental Variables , 1995 .

[12]  M. Tinetti,et al.  The effects of exercise on falls in elderly patients. A preplanned meta-analysis of the FICSIT Trials. Frailty and Injuries: Cooperative Studies of Intervention Techniques. , 1995, JAMA.

[13]  W. Katon,et al.  Collaborative management to achieve treatment guidelines. Impact on depression in primary care. , 1995, JAMA.

[14]  R. J. Hayes,et al.  Empirical evidence of bias. Dimensions of methodological quality associated with estimates of treatment effects in controlled trials. , 1995, JAMA.

[15]  K. Wells,et al.  How can care for depression become more cost-effective? , 1995, JAMA.

[16]  D G Altman,et al.  Assessing the quality of randomization from reports of controlled trials published in obstetrics and gynecology journals. , 1994, JAMA.

[17]  L. Gurel.  Research Designs and Methods in Psychiatry , 1994 .

[18]  E Frank,et al.  Cost utility analysis of maintenance treatment for recurrent depression. , 1992, Controlled clinical trials.

[19]  N. Lurie,et al.  Does capitation affect the health of the chronically mentally ill? Results from a randomized trial. , 1992, JAMA.

[20]  James J. Heckman,et al.  Randomization and Social Policy Evaluation , 1991 .

[21]  D. Neuhauser.  Modern Methods of Clinical Investigation , 1991 .

[22]  S D Imber,et al.  National Institute of Mental Health Treatment of Depression Collaborative Research Program. General effectiveness of treatments. , 1989, Archives of general psychiatry.

[23]  D. Rubin,et al.  Statistical Analysis with Missing Data. , 1989 .

[24]  Chris Chatfield,et al.  19. Statistical Analysis with Missing Data , 1988 .

[25]  P. Holland.  Statistics and Causal Inference , 1985 .

[26]  M S Kramer,et al.  Scientific challenges in the application of randomized trials. , 1984, JAMA.

[27]  R. Lalonde.  Evaluating the Econometric Evaluations of Training Programs with Experimental Data , 1984 .

[28]  S. Pocock,et al.  Clinical Trials: A Practical Approach , 1984 .

[29]  K. Rickels,et al.  Long-term diazepam therapy and clinical outcome. , 1983, JAMA.

[30]  D. Rubin,et al.  The central role of the propensity score in observational studies for causal effects , 1983 .

[31]  D. Sackett,et al.  Controversy in counting and attributing events in clinical trials. , 1979, The New England journal of medicine.

[32]  K. Wells.  The design of Partners in Care: evaluating the cost-effectiveness of improving care for depression in primary care , 1999, Social Psychiatry and Psychiatric Epidemiology.

[33]  C. Sherbourne,et al.  Caring for depression , 1996 .

[34]  B J Burns,et al.  Assertive community treatment: an update of randomized trials. , 1995, Psychiatric services.

[35]  K. Wells,et al.  Issues and approaches in evaluating managed mental health care. , 1995, The Milbank quarterly.

[36]  E. Keeler,et al.  Quality of care for hospitalized depressed elderly patients before and after implementation of the Medicare Prospective Payment System. , 1993, The American journal of psychiatry.

[37]  Ethan A. Halm,et al.  The Changing Economics of Medical Technology , 1991 .

[38]  Annetine C. Gelijns,et al.  Modern Methods of Clinical Investigation , 1990 .

[39]  R. Liberman,et al.  Quality-of-care research in mental health: responding to the challenge. , 1988, Inquiry : a journal of medical care organization, provision and financing.

[40]  James J. Heckman,et al.  Longitudinal Analysis of Labor Market Data: Frontmatter , 1985 .

[41]  T. Louis,et al.  Critical issues in the conduct and interpretation of clinical trials. , 1983, Annual review of public health.