Enhancing the validity and utility of randomized clinical trials in addictions treatment research: I. Treatment implementation and research design

Addiction

Volumn 102, Issue 7, 2007, Pages 1047-1056

  Del Boca, Frances K ^a   Darkes, Jack ^a  

^a UNIVERSITY OF SOUTH FLORIDA   (United States)

Author keywords

Addictions treatment; Controlled clinical trials; Randomized clinical trials; RCTs; Research design; Treatment implementation

Indexed keywords

ADDICTION; ALCOHOLISM; BEHAVIOR THERAPY; CLINICAL PRACTICE; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; COST; COST BENEFIT ANALYSIS; EXPERIMENTAL DESIGN; EXTERNAL VALIDITY; FOLLOW UP; HUMAN; INTERNAL VALIDITY; PSYCHOTHERAPY; RANDOMIZED CONTROLLED TRIAL; REVIEW; VALIDITY;

DELIVERY OF HEALTH CARE; HUMANS; RANDOMIZED CONTROLLED TRIALS; RESEARCH DESIGN; SUBSTANCE-RELATED DISORDERS;

EID: 34250357167     PISSN: 09652140     EISSN: 13600443     Source Type: Journal    
DOI: 10.1111/j.1360-0443.2007.01862.x     Document Type: Review

References (97)

1. Guyatt G., Cook D., Haynes B. Evidence-based medicine has come a long way; the second decade will be as exciting as the first. BMJ 2004 329 : 990 1.
2. Tucker J. A., Roth D. L. Extending the evidence hierarchy to enhance evidence-based practice for substance use disorders. Addiction 2006 101 : 918 32.
3. Chambless D. L., Ollendick T. H. Empirically supported psychological interventions: controversies and evidence. Annu Rev Psychol 2001 52 : 685 716.
4. Weisz J. R., Hawley K. M., Pilkonis P. A., Woody S. R., Follette W. C. Stressing the (other) three Rs in the search for empirically supported treatments: review procedures, research quality, relevance to practice and the public interest. Clin Psychol Sci Pract 2000 7 : 243 58.
5. Platt J. R. Strong inference. Science 1964 146 : 347 53.
6. Preventive Services Task Force. Guide to Clinical Preventive Services: Report of the US Preventive Services Task Force, 2nd edn. Baltimore : Williams & Wilkins 1996.
7. Howard K. I., Cox W. M., Saunders S. M. Attrition in substance abuse comparative treatment research: the illusion of randomization. In : Onken L. S., Blaine J. D., editors. Psychotherapy and Counseling in the Treatment of Drug Abuse. NIDA Monograph no. 104. Washington, DC : U.S. Department of Health and Human Services 1990, p. 66 79.
8. Green J. The evolving randomised controlled trial in mental health: studying complexity and treatment process. Adv Psychiatr Treat 2006 12 : 268 79.
9. Moyer A., Finney J. W. Randomized versus nonrandomized studies of alcohol treatment: participants, methodological features, and posttreatment functioning. J Stud Alcohol 2002 63 : 542 50.
10. Concato J., Shah N., Horwitz R. I. Randomized, controlled trials, observational studies, and the hierarchy of research designs. N Engl J Med 2000 342 : 1887 92.
11. Carroll K. M. New methods of treatment efficacy research: bridging clinical research and clinical practice. Alcohol Health Res World 1997 21 : 352 9.
12. Food and Drug Administration (FDA). Guidance for Industry: Good Clinical Practice: Consolidated Guidance. In Expert Working Group (Efficacy) of the International Conference on Harmonization of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH); 1996. Available at: http://www.fda.gov/cder/guidance/index.htm (accessed 17 April 2007).
13. Schwartz C. E., Chesney M. A., Irvine M. J., Keefe F. J. The control group dilemma in clinical research: applications for psychosocial and behavioral medicine trials. Psychosom Med 1997 59 : 362 71.
14. Borkovec T. D. Control groups and comparison groups in psychotherapy outcome research. In : Onken L. S., Blaine J. D., editors. Psychotherapy and Counseling in the Treatment of Drug Abuse. NIDA Monograph no. 104. Washington, DC : U.S. Department of Health and Human Services 1990, p. 50 65.
15. Borkovec T. D. Between-group therapy outcome research: design and methodology. In : Onken L. S., Blaine J. D., Boren J. J., editors. Behavioral Treatments for Drug Abuse and Dependence. NIDA Mongraph no. 137. Rockville, MD : National Institute on Drug Abuse 1993, p. 249 89.
16. Couper D. J., Hosking J. D., Cisler R. A., Gastfriend D. R., Kivlahan D. R. Factorial designs in clinical trials: options for combination treatment studies. J Stud Alcohol 2005 S15 : 24 32.
17. Kazdin A. E., Nock M. K. Delineating mechanisms of change in child and adolescent therapy: methodological issues and research recommendations. J Child Psychol Psychiatry 2003 44 : 1116 29.
18. Longabaugh R., Wirtz P. W. Causal chain analysis. In : Longabaugh R., Wirtz P. W., editors. Project MATCH Hypotheses: Results and Causal Chain Analyses. Project MATCH Monograph Series, Vol. 8. Rockville, MD : National Institute on Alcohol Abuse and Alcoholism 2001, p. 18 28.
19. Berlin J. A., Rennie D. Measuring the quality of trials: the quality of quality scales. JAMA 1999 282 : 1083 5.
20. Moyer A., Finney J. W., Swearington C. E. Methodological characteristics and quality of alcohol treatment outcome studies, 1970-98: an expanded evaluation. Addiction 2002 97 : 252 63.
21. Rothwell P. M. External validity of randomised controlled trials: 'to whom do the results of this trial apply?' Lancet 2005 365 : 82 93.
22. Project MATCH Research Group. Matching clients with alcohol disorders to treatments: clinical implications from Project MATCH. J Ment Health 1998 7 : 789 602.
23. Leshner A. I. Experimental and clinical psychopharmacology: National Institute on Drug Abuse's clinical research agenda. Exp Clin Psychopharm 2002 10 : 159 61.
24. Medical Research Council. Clincial Trials for Tomorrow: An MRC Review of Randomised Controlled Trials. London : MRC 2003.
25. National Advisory Mental Health Council. Bridging Science and Service. NIH Publication no. 99-4353. Washington, DC : National Institutes of Health 1999.
26. Moos R. H. Addictive disorders in context: principles and puzzles of effective treatment and recovery. Psychol Addict Behav 2003 17 : 3 12.
27. Simpson D. D. A conceptual framework for drug treatment process and outcome. J Subst Abuse Treat 2004 27 : 99 121.
28. Doss B. D. Changing the way we study change in psychotherapy. Clin Psychol Sci Pract 2004 11 : 368 86.
29. Project MATCH Research Group. Matching alcoholism treatments to client heterogeneity: project MATCH posttreatment drinking outcomes. J Stud Alcohol 1997 58 : 7 29.
30. Blumenstein B. A., James K. E., Lind B. K., Mitchell H. E. Functions and organization of coordinating centers for multicenter studies. Control Clin Trials 1995 16 : 4S 29S.
31. McRee B. Project MATCH - a case study: the role of a coordinating center in facilitating research compliance in a multisite clinical trial. In : Zweben A., Barrett D., Carty K., McRee B., Morse P., Rice C., editors. Strategies for Facilitating Protocol Compliance in Alcoholism Treatment Research. Project MATCH Monograph Series, Vol. 7. Rockville, MD : National Institute on Alcohol Abuse and Alcoholism 1998, p. 93 110.
32. Youngblood M. E., Murray K. T., Devine E., Latham P. K., Hubatch S. Coordinating and monitoring multisite clinical trials that combine pharmacological and behavioral treatments. J Stud Alcohol 2005 S15 : 82 91.
33. Carty K., Rice C., Barrett D. Strategies for maintaining compliance. In : Zweben A., Barrett D., Carty K., McRee B., Morse P., Rice C., editors. Strategies for Facilitating Protocol Compliance in Alcoholism Treatment Research. Project MATCH Monograph Series, Vol. 7. Rockville, MD : National Institute on Alcohol Abuse and Alcoholism 1998, p. 9 32.
34. Zweben A., Barrett D., Berger L., Murray K. T. Recruiting and retaining participants in a combined behavioral and pharmacological clinical trial. J Stud Alcohol 2005 S15 : 72 81.
35. Miller W. R., LoCastro J. S., Longabaugh R., O'Malley S., Zweben A. When worlds collide: blending the divergent traditions of pharmacotherapy and psychotherapy outcome research. J Stud Alcohol 2005 S15 : 17 23.
36. Califf R. M., Morse M. A., Wittes J., Goodman S. N., Nelson D. K., DeMets D. L. et al. Toward protecting the safety of participants in clinical trials. Control Clin Trials 2003 24 : 256 71.
37. DeMets D. J., Pocock S. J., Julian D. G. The agonizing negative trend in monitoring clinical trials. Lancet 1999 354 : 1983 8.
38. Del Boca F. K., Mattson M. E., Fuller R., Babor T. F. Planning a multisite matching trial: organizational structure and research design. In : Babor T. F., Del Boca F. K., editors. Treatment Matching in Alcoholism. Cambridge : Cambridge University Press 2003, p. 15 28.
39. Babor T. F., Del Boca F. K., editors. Treatment Matching in Alcoholism. Cambridge : Cambridge University Press 2003.
40. COMBINE Study Research Group. Testing combined pharmacotherapies and behavioral interventions in alcohol dependence: rationale and methods. Alcohol Clin Exp Res 2003 27 : 1107 22.
41. Beutler L. E., Clarkin J. F. Systematic Treatment Selection: Toward Targeted Therapeutic Interventions. New York : Brunner-Mazel 1990.
42. Carroll K. M., editor. Improving Compliance in Alcohol Treatment. Project MATCH Monograph Series, Vol. 6. Rockville, MD : National Institute of Alcohol Abuse and Alcoholism 1997.
43. Carroll K. M., Kadden R. M., Donovan D. M., Zweben A., Rounsaville B. J. Implementing treatment and protecting the validity of the independent variable in treatment matching studies. J Stud Alcohol 1994 S12 : 149 55.
44. Carroll K. M., Rounsaville B. Can a technology model of psychotherapy be applied to cocaine treatment? In : Onken L. S., Blaine J. D., editors. Psychotherapy and Counseling in the Treatment of Drug Abuse. NIDA Monograph no. 104. Washington, DC : U.S. Department of Health and Human Services 1990, p. 91 104.
45. Donovan D. M., Kadden R. M., DiClemente C. C., Carroll K. M., Longabaugh R., Zweben A. Issues in the selection and development of therapie sin alcoholism treatment matching research. J Stud Alcohol 1994 S12 : 138 48.
46. Donovan D. M., Carroll K. M., Kadden R. M., DiClemente C. C., Rounsaville B. J. Therapies for matching: selection, development, implementation, and costs. In : Babor T. F., Del Boca F. K., editors. Treatment Matching in Alcoholism. Cambridge : Cambridge University Press 2003, p. 42 61.
47. Docherty J. P. Implications of the technological model of psychotherapy. In : Williams J. B. W., Spitzer R. L., editors. Psychotherapy Research: Where Are We and Where Should We Go. New York : Guilford Press 1984.
48. Waskow I. E. Specification of the technique variable in the NIMH Treatment of Depression Collaborative Research Program. In : Williams J. B. W., Spitzer R. L., editors. Psychotherapy Research: Where Are We and Where Should We Go. New York : Guilford Press 1984, p. 150 9.
49. Kazdin A. E. Nonspecific treatment factors in psychotherapy outcome research. J Consult Clin Psychol 1979 47 : 846 51.
50. Carroll K. M., Nich C., Rounsaville B. J. Utility of therapist session checklists to monitor delivery of coping skills treatment for cocaine abusers. Psychother Res 1998 8 : 307 20.
51. Carroll K. M., Ball S. A., Fenton L., Frankforter T. L., Nich C., Nuro K. F. et al. A general system for evaluating therapist adherence and competence in psychotherapy research in the addictions. Drug Alcohol Depend 2000 57 : 225 38.
52. Carroll K. M., Connors G. J., Cooney N., DiClemente C. C., Donovan D. M., Kadden R. M. et al. Internal validity of Project MATCH: discriminability and integrity. J Consult Clin Psychol 1998 66 : 290 303.
53. Morgenstern J., Longabaugh R. Cognitive-behavioral treatment for alcohol dependence: a review of evidence for its hypothesized mechanisms of action. Addiction 2000 95 : 1475 90.
54. Longabaugh R., Donovan D. M., Karno M. P., McCrady B. S., Morgenstern J., Tonigan J. S. Active ingredients: how and why evidence-based alcohol behavioral treatment interventions work. Alcohol Clin Exp Res 2005 29 : 235 47.
55. Berglund M. A better widget? Three lessons for improving addiction treatment from a meta-analytic study. Addiction 2005 100 : 742 50.
56. Baron R. M., Kenny D. A. The moderator-mediator variable distinction in social psychological research: conceptual, strategic, and statistical considerations. J Pers Soc Psychol 1986 51 : 1173 82.
57. MacKinnon D. P., Fairchild A. J., Fritz M. S. Mediation analysis. Ann Rev Psychol 2007 58 : 593 614.
58. Muller D., Judd C. M., Yzerbyt V. Y. When moderation is mediated and mediation is moderated. J Pers Soc Psychol 2005 89 : 852 63.
59. Holmbeck G. N. Toward terminological, conceptual, and statistical clarity in the study of mediators and moderators: examples from the child-clinical and pediatric psychology literatures. J Consult Clin Psychol 1997 65 : 599 610.
60. Kraemer H. C., Wilson G. T., Fairburn C. G., Agres S. Mediators and moderators of treatment effects in randomised clinical trials. Arch Gen Psychiatry 2002 59 : 877 83.
61. Longabaugh R., Wirtz P. W. Substantive review and critique. In : Longabaugh R., Wirtz P. W., editors. Project MATCH Hypotheses: Results and Causal Chain Analyses. Project MATCH Monograph Series, Vol. 8. Rockville, MD : National Institute on Alcohol Abuse and Alcoholism 2001, p. 305 25.
62. Wirtz P. W., Longabaugh R. Methodological assessment and critique. In : Longabaugh R., Wirtz P. W., editors. Project MATCH Hypotheses: Results and Causal Chain Analyses. Project MATCH Monograph Series, Vol. 8. Rockville, MD : National Institute on Alcohol Abuse and Alcoholism 2001, p. 296 304.
63. Longabaugh R., Wirtz P. W., editors. Project MATCH Hypotheses: Results and Causal Chain Analyses. Project MATCH Monograph Series, Vol. 8. Rockville, MD : National Institute on Alcohol Abuse and Alcoholism 2001.
64. Carroll K. M., Nuro K. F. The use and development of treatment manuals. In : Carroll K.M., editor. Improving Compliance in Alcohol Treatment. Project MATCH Monograph Series, Vol. 6. Rockville, MD : National Institute on Alcohol Abuse and Alcoholism 1997, p. 53 72.
65. Luborsky L., De Rubeis R. J. The use of psychotherapy treatment manuals: a small revolution in psychotherapy research style. Clin Psychol Rev 1984 4 : 5 14.
66. Project MATCH Research Group. Therapist effects in three treatments for alcohol problems. Psychother Res 1998 8 : 455 74.
67. Crits-Christoph P., Beebe K. L., Connolly M. B. Therapist effects in the treatment of drug dependence: implications for conducting comparative treatment studies. In : Onken L. S., Blaine J. D., Boren J. J., editors. Behavioral Treatments for Drug Abuse and Dependence. NIDA Mongraph no. 137. Rockville, MD : National Institute on Drug Abuse 1993, p. 39 49.
68. Miller W. R., Moyers T. B., Arciniega L., Ernst D., Forcehimes A. Training, supervision, and quality monitoring of the COMBINE study behavioral interventions. J Stud Alcohol 2005 S15 : 188 95.
69. Witte G., Wilber C. H. A case study in clinical supervision: experience from Project MATCH. In : Carroll K.M., editor. Improving Compliance in Alcohol Treatment. Project MATCH Monograph Series, Vol. 6. Rockville, MD : National Institute on Alcohol Abuse and Alcoholism 1997, p. 73 86.
70. Carroll K. M. Compliance and alcohol treatment: an overview. In : Carroll K.M., editor. Improving Compliance in Alcohol Treatment. Project MATCH Monograph Series, Vol. 6. Rockville, MD : National Institute on Alcohol Abuse and Alcoholism 1997, p. 5 12.
71. Mattson M. E., Del Boca F. K., Carroll K. M., Cooney N. L., DiClemente C. C., Donovan D. et al. Compliance with treatment and follow-up protocols in Project MATCH: predictors and relationship to outcome. Alcohol Clin Exp Res 1998 22 : 1328 39.
72. Cramer J. A., Rosenheck R. Compliance with medication regimens for mental and physical disorders. Psychiatr Serv 1998 49 : 196 201.
73. Liu H., Golin C. E., Miller L. G., Hays R. D., Beck C. K., Sanandaji S. et al. A comparison study of multiple measures of adherence to HIV protease inhibitors. Ann Intern Med 2001 134 : 968 77.
74. Del Boca F. K., Kranzler H. R., Brown J., Korner P. F. Assessment of medication compliance in alcoholics through UV light detection of a riboflavin tracer. Alcohol Clin Exp Res 1996 20 : 1412 17.
75. Anton R. F. New methodologies for pharmacological treatment trials for alcohol dependence. Alcohol Clin Exp Res 1996 20 : 3A 9A.
76. Carroll K. M. Enhancing retention in clinical trials of psychosocial treatments: practical strategies. In : Onken L. S., Blaine J. D., Boren J. J., editors. Beyond the Therapeutic Alliance: Keeping the Drug Dependent Individual in Treatment. NIDA Research Monograph no. 165. Washington, DC : Government Printing Office 1997, p. 4 24.
77. Kabela E., Kadden R. Practical strategies for improving client compliance with treatment. In : Carroll K.M., editor. Improving Compliance in Alcohol Treatment. Project MATCH Monograph Series, Vol. 6. Rockville, MD : National Institute on Alcohol Abuse and Alcoholism 1997, p. 15 50.
78. Stanton M. D. Getting reluctant substance abusers to engage in treatment/self help: a review of outcomes and clinical options. J Marital Fam Ther 2004 30 : 165 82.
79. Johnson B. A., Ait-Daoud N., Roache J. D. The COMBINE SAFTEE: a Structured instrument for collecting adverse events adapted for clinical studies in the alcoholism field. J Stud Alcohol 2005 S15 : 157 67.
80. Food and Drug Administration (FDA). Guideline for Industry: Clinical Safety Data Management: Definitions and Standards for Expedited Reporting. In Expert Working Group (Efficacy) of the International Conference on Harmonization of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH); 1995. Available at: http://www.fda.gov/cder/guidance/iche2a.pdf (accessed 17 April 2007).
81. Food and Drug Administration (FDA). Guideline for Industry: Structure and Content of Clinical Study Reports. In Expert Working Group (Efficacy) of the International Conference on Harmonization of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH); 1996. Available at: http://www.fda.gov/cder/guidance/iche3.pdf (accessed 17 April 2007).
82. Holder H. D., Cisler R. A., Longabaugh R., Stout R. L., Treno A. J., Zweben A. Alcoholism treatment and medical care costs from Project MATCH. Addiction 2000 95 : 999 1013.
83. Zarkin G. A., Bray J. W., Mitra D., Cisler R. A., Kivlahan D. R. Cost methodology of COMBINE. J Stud Alcohol 2005 S15 : 50 5.
84. Sobell L. C., Ellingstad T. P., Sobell M. B. Natural recovery from alcohol and drug problems: methodological review of the research with suggestions for future directions. Addiction 2000 95 : 749 64.
85. Fogg L., Gross D. Threats to validity in randomized clinical trials. Res Nurs Health 2000 23 : 79 87.
86. Quitkin F. M. Placebos, drug effects, and study design: a clinician's guide. Am J Psychiatry 1999 156 : 829 36.
87. Smith D. Five principles of research ethics: cover your bases with these ethical strategies. APA Monitor Psychol 2003 34 : 56 60.
88. Berger V. W., Weinstein S. Ensuring the comparability of control groups: is randomization enough? Control Clin Trials 2004 25 : 515 24.
89. Schulz K. F., Grimes D. A. Allocation concealment in randomised trials: defending against deciphering. Lancet 2002 359 : 614 18.
90. Blair E. Gold is not always good enough: the shortcomings of randomization when evaluating interventions in small, heterogeneous samples. J Clin Epidemiol 2004 57 : 1219 22.
91. Stout R. L., Wirtz P. W., Carbonari J. P., Del Boca F. K. Ensuring balanced distribution of prognostic factors in treatment outcome research. J Stud Alcohol 1994 S12 : 70 5.
92. Rosenberger W. F., Lachin J. M. Randomization in Clinical Trials: Theory and Practice. New York : Wiley 2002.
93. Margolin A., Avants S. K., Kleber H. D. Investigating alternative medicine therapies in randomized controlled trials. JAMA 1998 280 : 1626 8.
94. Schlosser R. W. On the importance of being earnest about treatment integrity. Argument Altern Comm 2002 18 : 36 44.
95. Bang H., Ni L., Davis C. E. Assessment of blinding in clinical trials. Control Clin Trials 2003 25 : 143 56.
96. Allen J. A. Measuring outcome in interventions for alcohol dependence and problem drinking: executive summary of a conference sponsored by the National Institute on Alcohol Abuse and Alcoholism. Alcohol Clin Exp Res 2003 53 : 1657 60.
97. Sobell L. C., Sobell M. B., Connors G. J., Agrawal S. Assessing drinking outcomes in alcohol treatment efficacy studies: a yardstick of success. Alcohol Clin Exp Res 2003 27 : 1661 6.