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16 SUPPORTING THE SCIENTIFIC INFRASTRUCTURE FOR TREATMENT RESEARCH Steady progress has been made over the last decade in increasing the alcohol research budget. In 1980, NIAAA's research support was $22.2 million, which included 167 research projects. By fiscal year 1989, the institute's total research funding had grown to $107.1 million with 485 grants supported. Linked to this growth has been a significant improvement in the knowledge base dealing with the biomedical and psychosocial aspects of alcohol problems (IOM, 1987~. NIAAA supports programs of intramural and extramural research that span basic, clinical, and epidemiological studies. Unfortunately, not until 1988, when NIAAA created its new Division of Clinical and Prevention Research, was treatment research given a prominent place in the research portfolio. This previous lack of support is reflected in the relatively small portions of the intramural (14 percent) and extramural (12 percent) research budgets that are devoted to treatment-related research. Until now NIAAA has relied on two mechanisms to fund treatment research: the investigator-initiated (R01) grant and the research center. NIAAA funds a treatment research center at the University of Connecticut Health Center in Farmington. The Veterans Administration (VA) also funds a clinical research center at the San Diego VA Hospital and has provided some support for treatment trials through its Merit Review Program and through two multicenter collaborative studies. The NIAAA and VA research support mechanisms have together led to improvements in methodology and theory and to a slowly expanding pool of researchers and facilities. However, one recent development that provokes serious concern is the cutback in the fiscal year 1989 budget for VA health care programs. This cutback has already resulted in the closing of alcohol treatment programs that were linked to important NIAAA- and VA-funded research. Moreover, the new impediments that have been raised to the admission to VA hospitals of non-se~vice-connected or indigent veterans will have a disastrous effect on a number of major clinical and basic alcohol research programs. Recently, the ADAMHA Office of Substance Abuse Prevention (OSAP) initiated support through large-scale grants for a high-risk-youth demonstration project and community-based programs, but these funding mechanisms have not provided adequate support for systematic evaluation. For the federal government to begin to exploit the opportunities in treatment research described in this report, it must address the need within the alcohol field to develop a scientific infrastructure appropriate to the task. It is a challenge whose dimensions have been previously charted by treatment and prevention research efforts at the National Institutes of Health (NIH)--particularly the National Cancer Institute; the National Heart, Lung, and Blood Institute; and the National Institute on Allergy and Infectious Diseases in its program of AIDS research. Maintaining support for agencies and programs that provide this infrastructure is essential. Recent reviews of treatment efficacy (Saxe et al., 1983; Lettieri, Sayers, and Nelson, 1984; Tims and Ludford, 1984; Miller and Hester, 1986) identified approximately 200 controlled studies. Few of these studies, however, employed advanced clinical assessment rr.ethodologies (e.g., assessments of comorbid psychopathology or severity of dependence). Similarly, although 150 random assignment trials have been conducted, few have been -313

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devoted to comparisons of treatment modalities or settings in relation to outcome. Studies of treatment efficacy are needed to define the dimensions of treatment outcome in relation to the expected effects of treatment. Research should be designed and funded in such a way that treatment effects can be examined not only at the termination of treatment but (at least) one year beyond in order to investigate the potential enduring benefits of certain treatment modalities and settings. Although clinical trials were introduced as a method of scientific investigation before 1945 and have been used extensively in psychiatry and medicine since then, it is only recently that large-scale clinical trials have been conducted in alcoholism treatment research (Fuller et al., 1986~. The relative dearth of controlled trials and treatment matching research (see, for example, Skinner, 1981) is in part a result of the lack of funding in this area and the formidable practical and methodological challenges to researchers. In the early development of new treatment modalities (e.g., a new pharmacotherapy for the treatment of alcohol withdrawal), the investigator-initiated grant application (Rot) can be utilized effectively to compare the new modality with established treatment practice. Random assignment to treatment is highly desirable in this type of study. The investigator-initiated grant may also be the mechanism of choice for studies of well-specified treatment populations (e.g., minorities, women, military personnel, specific age cohorts) in which the investigator has special access to the particular patient or client group. One major disadvantage of the R01 application for these types of studies has been the traditional limited funding period associated with this grant. Three years of funding may not be adequate to initiate a study, collect a cohort of clients/patients who meet the research criteria, and conduct adequate follow-up of a sufficient number of subjects at least 12 months after the treatment intervention. Treatment research grant announcements should strongly encourage four- and five-year awards, with the requirement that the proposal include adequate follow-up of a sufficient number of subjects for at least one year after the treatment intervention. Treatment research methods are often shaped by the requirements of the treatment program, a factor that may limit the rigor of the research design. These studies are of necessity scientifically less exact and more complex and difficult than other projects reviewed by the existing grant review committees. There is a need to increase the number of qualified treatment researchers through increased support for postdoctoral training in relevant specialities (e.g., psychiatry, psychology, internal medicine, family medicine, epidemiology, sociology, health care economics). The Scientist Development Award for Clinicians appears to be an excellent vehicle for the career development of young investigators interested in treatment (and prevention) research, but the present maximum stipend of $45,000 per year is not adequate salary support for young physicians. Center grants offer another vehicle for studying treatment research. For example, the National Institute on Drug Abuse (NIDA) recently established three treatment research centers with the stated goal of "systematically testting] existing and new treatment strategies in well-controlled designs." Because of the need to develop large subject samples, NIDA urged applicants to "consider the advantages of coordination between related studies, and between Centers doing similar work." NIDA emphasized its interest in developing treatment process as well as outcome data; it also urged the funded centers to develop research training opportunities. In some respects, the new NIDA program is modeled after NIAAA's highly successful, but more broadly based, alcohol research centers program. -314

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Only one of 12 NIAAA centers conducts alcohol treatment research. The latter program includes studies at three sites: a VA hospital, a treatment program within a university hospital, and a large outpatient program within a mental health center at a neighboring university. Affiliations with several other institutions in the area have been essential to this center in its efforts to maintain an adequate and diverse patient base for its treatment research programs. (A postdoctoral research training program is also part of the core activities of that center.) The committee suggests that NIAAA consider funding one or more additional centers devoted to treatment research in collaboration with another federal agency (e.g., the VA or the Department of Defense) or in conjunction with one of the state-funded alcohol research centers. A significant proportion of the alcohol treatment in this county takes place in freestanding inpatient units that are generally not affiliated with major academic and research centers. The rapid growth in the number of inpatient beds in proprietary and nonprofit facilities over the past 15 years (Yahr, 1988) has stimulated a strong interest in research on treatment settings and the matching of patient subtypes to specific treatments. This interest has been expressed in many quarters: the Congress, the executive branch of the federal government, the insurance industry, and the treatment community. Since the introduction of the block grant program, NIAAA has lost its ability to evaluate community-based treatment programs as it did in the 1970s through contracts with the Rand Corporation (Armor, Polich, and Stambul, 1978; Polich, Armor, and Braiker, 1981~. The Rand studies were generally limited by the sparseness of patient assessments, but the large number of subjects who were studied across a wide variety of treatment settings provided important information on differential routes to recovery among patients/clients who differed in the severity of their alcohol dependence. The committee commends the recent policy decision to set aside 5 to 15 percent of funds within block grants to evaluate alcohol and drug abuse treatment programs and to determine the quality and appropriateness of various forms of treatment (including the effect of living in the types of housing provided in these programs). The evaluations are to be funded through grants, contracts, or cooperative agreements provided to public and nonprofit private entities. This policy could well encourage linkages among university-affiliated researchers, state agencies, and not-for-profit treatment facilities. A potentially important initiative was launched recently by a programmatic decision by the leadership at NIAAA and NIDA to utilize the funds appropriated through Section 1923 of the Public Health Service Act (an amendment added by the Anti-Drug Abuse Act of 1986) to fund grants for up to five years to evaluate alcohol and cocaine treatments. Unfortunately, the conditions of the appropriation mandated annual awards, with continuation contingent on the availability of funds "and progress achieved." New grant money in the amount of $2.3 million was made available for this purpose in fiscal year 1988. This program could be limited in achieving its stated goals because of uncertainties regarding long-term funding and a failure to address the need to develop treatment research networks of adequate size and diversity. This uncertainty about long-term support could have a negative impact on the scientific dimensions of these evaluations and will also make it more difficult to attract competent investigators to this important research area. To systematically examine questions that relate to treatment setting and treatment matching, it is essential to develop an adequate number of treatment facilities willing to participate in controlled treatment trials. In this context, specific funds must be set aside for thorough patient assessments at intake and at the follow-up point. These assessments must be conducted with a high degree of reliability within and between sites. The optimal -315

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mechanisms for this type of study would appear to be the Cooperative agreements within the Public Health Service and the "collaborative study within the VA As an example of the latter, the conduct of the VA collaborative study on disulfiram (Fuller et al., 1986) has been especially instructive. This study involved nine sites and 14 investigators. Out of an initial pool of 6,629, researchers recruited 605 subjects. The small pool of eligible subjects for this study highlights the formidable logistical problems involved in well-controlled treatment research. The results also highlight the importance of rigorous experimental design in these studies (Fuller et al., 1986~. Table 16-1 compares regular research grants with cooperative agreements. Under a cooperative agreement, institute staff work with the scientific community in a cooperative venture. Institute staff and consultants assist colleagues in the scientific community in the design of the study, project coordination, and the dissemination of research results. As a mechanism for ensuring close cooperation between treatment facilities and investigators, and as a means of securing large treatment samples from multiple sites, the cooperative agreement is preferable to investigator-initiated grants. Experience at NIH suggests that the cooperative agreement is an especially useful instrument for the rapid transfer of knowledge from research to clinical practice. Examples of cooperative agreements include the new network of AIDS clinical studies being funded by the National Institute on Allergy and Infectious Disease and the Clinical Trials Cooperative Groups supported by the National Cancer Institute (NCI). In the NCI program, each project typically includes physicians from multiple medical centers who cooperatively design and conduct clinical trials to evaluate treatment of various types and stages of cancer. The data are collected and analyzed in a coordinating center. Most of the participants are located at major medical centers in urban areas. The program was further extended in 1976 to include the participation of physicians at community hospitals and in private practice. Similarly, the National Heart, Lung, and Blood Institute has funded placebo-controlled trials in a multicenter program for the treatment of cardiac arrhythmia. Cooperative agreements have also been utilized to test new diagnostic instruments. The National Institute of Mental Health (NIMH), an ADAMHA institute, has used the cooperative agreement mechanism to fund a multicenter collaborative study of depression to determine whether different aspects of the disorder respond to different treatments (pharmacotherapy versus psychotherapy). NIAAA is initiating cooperative agreements in fiscal year 1989, including a multisite trial of alcoholism treatment that involves patient-treatment matching. The use of the cooperative agreement to fund large-scale alcohol treatment research should be encouraged. Similar to the VA collaborative study of disulfiram (Fuller et al., 1986), the data from such treatment studies will benefit both the research and the clinical communities. NIAAA staff could play a critical role in bringing together a network of investigators and a variety of treatment facilities from the public and private sectors. In this context, the requirements both of research design and of clinical practice can be considered. NIAAA, the VA, the Health Care Financing Administration, and the Department of Defense should also consider developing cost-sharing arrangements with interested states, universities, service providers, and third-party payers. -316

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Table 1~1. Distinguishing Between Grants and Cooperative Agreements Element Grant Cooperative Agreement Intent Support or stimulation to Support or stimulation to accomplish a accomplish a public purpose public purpose Scope of work Any program activity Any program activity eligible under eligible under NIH/ADAMHA legislation NIHJADAMHA legislation Initiation Applicant initiated; may be Awarding a component initiated through an in response to an RFA or RFA or PA PA NIH/ADAMHA role Normal programmatic and Normal stewardship responsibilities plus administrative stewardship substantial programmatic involvement responsibilities; no during performance of award substantial programmatic involvement Examples of staff Providing technical Participating in the design of activities participation assistance at the recipient's request Close monitoring of an Advising in the selection of contractors, external organization trainees, staff, etc. - Ensuring compliance with Coordinating or participating in the policy requirements and collection and/or analysis of data - terms of award Evaluating performance Advising in training and selection of project through progress reports staff and site visits Reviewing and approving each stage of a clinical trial Advising on management and technical performance Participation in the preparation of publications Participation in all responsibilities required by grants -317

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It should be recognized that multisite clinical trials are difficult to implement. Problems- in this type of research include control over the therapies being delivered, quality control over the selection and training of therapists, and developing an effective means of monitoring day-to-day implementation of the research and clinical protocols. Cooperative studies are best carried out after smaller, single-site studies have been conducted; such smaller-scale research allows for careful specification and testing of procedures. Cooperative efforts are easiest to implement in drug and assessment studies; they are most feasible when the treatments under investigation are well specified, time limited, and relatively easily taught. Finally, in any consideration of scientific infrastructure, the federal government needs to consider the personnel requirements that are essential to the conduct of treatment research. In 1986, NIAAA supported four postdoctoral and two career development stipends that could be classified as treatment research. In conjunction with the centers program and the proposed cooperative agreements, NIAAA should target funding for additional scientists in treatment research careers including epidemiologists, social and behavioral psychologists, physicians (psychiatrists, internists. family nhv~im~n~N health C'~-C ~-c-~h~rc OVA biostatisticians. __7 ~J I__ ~All ~-__ v_^ ~ ^__, ~ _ ~1- Within the institute, new initiatives in the area of treatment research should be complemented by the active use of outside consultants, planning panels, and other mechanisms, such as the proposed Clinical Staff Program for Prevention and Treatment Research. This program is designed to provide greater interaction among the intramural research program, the extramural research program, and clinicians/researchers in the field. This mechanism should also be used to attract visiting scientists to the institute for one- or two-year periods. Over the years NIAAA has developed a number of programs and activities that are capable of stimulating research, facilitating technology transfer, and providing expert advice to the field. These include not only research grants, training grants, the Alcohol Research Centers program, and cooperative agreements but also planning panels, expert committee meetings, research conferences, technical reviews, bilateral international agreements, interagency agreements, contracts, and the intramural research program. NIAAA is also using a developmental grant mechanism (R21) that offers up to two years of support ($40,000 in direct costs per year) to assist institutions in building their capacity to do alcoholism treatment research; conducting pilot studies that lead to expansion, enhancement, or modification of existing treatment research programs; and planning and conducting pilot research that can lead to the development of clinical trials. Projects aimed at the treatment of alcoholism in special groups such as women, minorities, and defined age groups (e.g., adolescents and the elderly) have been encouraged under this mechanism. There appears to be sufficient flexibility in NIAAA's operational mechanisms to permit the implementation of the treatment research opportunities outlined in Part II of this report. As new funding and funding mechanisms are brought into play, NIAAA should evaluate the experience of other agencies that have been involved in clinical trials and large-scale collaborative studies. For instance, NIDA has devoted a substantial proportion of its research budget to treatment grants and has supported a longitudinal evaluation of drug treatment programs (called the Treatment Outcome Perspective Study) since 1978. NIMH has sponsored the NIMH Treatment of Depression Collaborative Research Program through cooperative agreements with seven participating sites. The VA supports investigator-initiated treatment grants through its Merit Review System and has sponsored the VA collaborative study of disulfiram. -318

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International research may also offer some guidance. The alcohol research program of the World Health Organization (WHO) has initiated international collaborative projects in the areas of screening, early intervention, community responses to alcohol problems, and the comparison of alcoholism treatment systems. These projects are typically funded through cooperative agreements among collaborating research centers and health ministries. One WHO program that has been beneficial to treatment research in the United States is the WHO/ADAMHA program on nomenclature and classification of mental disorders and alcohol- or drug-related problems. This program has facilitated the standardization of nomenclature and diagnostic procedures with respect to alcohol-use disorders. It has also led to the development of new diagnostic instruments that promise to assist treatment planning and epidemiological research. These programs not only serve as models for achieving some of NL\AA's goals for treatment research, they also indicate that there may be large areas of common interest among the various national and international agencies engaged in treatment research. These indications take on added vitality in light of the growing participation of state and private agencies in alcohol treatment research and program evaluation. A number of state agencies now engage in treatment evaluation through their own information systems or in cooperation with state-supported research centers. In addition, several private hospital groups have established treatment evaluation components. What is needed is for NIAAA to coordinate activities, divide responsibilities, and share data among the agencies and organizations involved in similar pursuits. Given the recognized leadership position that NIAAA has achieved in the field of treatment, the institute is in a unique position to form a network of mutually beneficial partnerships among various agencies and organizations that are involved in similar activities. By coordinating activities among such interest groups as research centers, pharmaceutical companies, hospital chains, insurance companies, single state agencies, and federal agencies, NIAAA may be able to guide the field toward a more coherent course of action. The committee makes the following recommendations for funding treatment research: The present research program of investigator-initiated grants should be enhanced to encourage the funding of treatment research for periods up to five years. This expansion will allow for start-up work and at least one year of posttreatment evaluation. There is a need to increase the pool of treatment research investigators. This goal can be achieved by a variety of mechanisms including support of M.D./Ph.D. programs in medical schools targeted toward clinical investigators in alcoholism. The newly initiated Scientist Development Award for Clinicians should help to create a pool of young investigators interested in treatment research. NIAAA should play a leadership role in bringing interested groups together to support treatment research in the alcohol field. The National Heart, Lung, and Blood Institute is interested in the effects of abstinence or reduced drinking on hypertension secondary to excessive alcohol use. NIDA is interested in the treatment of polysubstance abuse including cocaine, opiates, and alcohol. NIMH is interested in the comorbidity of psychotic disorders and substance abuse. The VA and the Department of Defense have a strong interest in alcoholism treatment. The Department of Transportation also has an interest in alcoholism treatment because excessive alcohol use can affect safety on roads and railroads, in sea lanes and rivers, and in the air. Insurance companies and state, not-for-profit, and proprietary alcoholism treatment programs all have a stake in treatment outcome research. -319

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~ Recent actions by the VA to curtail the availability of alcoholism treatment will have a devastating impact on treatment research in the alcohol field. Given the high percentage of veterans with alcohol-related pathology who are hospitalized on medicaUsurgical units in VA hospitals, the committee recommends that the VA reverse these actions and renew its commitment to alcohol treatment and treatment research. The program to use funds appropriated in the 1986 Anti-Drug Abuse Act to support controlled trials of alcohol and cocaine treatment should be stabilized, extended, and used as a model for the evaluation of all federally supported treatment initiatives. Similarly, the recent decision to allocate a percentage of block grant funds to evaluation provides an opportunity to advance carefully designed process and outcome evaluation as an integral component of treatment programs supported by the federal government and implemented at the state level. This initiative should be used to encourage cooperation between academic and research institutions on the one hand and publicly supported treatment programs on the other. Cooperative agreements should be implemented as a mechanism for funding complex, multisite treatment outcome studies and clinical trials. NIAAA should be provided with the personnel resources to provide proper staffing of these initiatives. NtAAA should encourage the inclusion of salary support on grants for clinical facility personnel involved in treatment evaluation research as a means of encouraging the participation of such facilities in treatment trials. Salary support should be identified on individual grant proposals and within cooperative agreements. NIAAA should continue to highlight the state of the art in treatment research studies by establishing and upgrading guidelines for quality designs and methods of treatment research. REFERENCES Armor, D. J., J. M. Polich, and H. B. Stambul. Alcoholism and Treatment. New York: John Wiley and Sons, 1978. Fuller, R. K, L. Branchey, D. R. Brightwell et al. Disulfiram treatment of alcoholism. A Veterans Administration cooperative study. J. Am. Med. Assoc. 256:1449-1255, 1986. Institute of Medicine. Causes and Consequences of Alcohol Problems: An Agenda for Research. Washington, DC: National Academy Press, 1987. Lettieri, D., M. Sayers, and J. Nelson. Summaries of Alcoholism Treatment Assessment Research. NIAAA Treatment Handbook Series~vol. 1. Washington. DC Government Printing Office, 1984. O , ~ Miller, W. R., and R. K Hester. Inpatient alcoholism treatment: Who benefits? Am. Psychol. 41~7~:794-805, 1986. Polich, J. M., D. J. Armor, and H. B. Braiker. The Course of Alcoholism: Four Years After Treatment. New York: John Wiley and Sons, 1981. Saxe, L., D. Dougherty, K Esty, and M. Fine. The effectiveness and costs of alcoholism treatment. Health Technology Case Study 22. Office of Technology Assessment, U.S. Congress. Washington, DC: Government Printing Office, 1983. -320

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Skinner, H. ~ Different strokes for different folks: Differential treatment for alcohol abuse. Pp. 349-368 in Evaluation of the Alcoholic: Implications for Research, Theogr, and Treatment. NIAAA Research Monograph No. 5. Rockville, MD: NIAAA, 1981. Tims, F., and I. Ludford. Drug Abuse Treatment Evaluation: Strategies, Progress and Prosper is. National Institute on Drug Abuse Monograph Series, vol. 51. Washington, DC: Government Printing Office, 1984. Yahr, H. T. A national comparison of public and private-sector alcoholism treatment delivery system characteristics. J. Stud. Alcohol 49:233-239, 1988. . -321

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