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Appendix C: Comparative Effectiveness Research Priorities: IOM Recommendations (2009)
Appendix C: Comparative Effectiveness Research Priorities: IOM Recommendations (2009)
Pages 469-510
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Appendix C Comparative Effectiveness Research Priorities IOM Recommendations (2009) 1 1 Passage from the Institute of Medicine Report, Initial National Priorities for Comparative Effectiveness Research June 0, 2009.
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committee's statement of task charged the committee with developing a list of priority comparative effectiveness research (CER) topics and presenting those recommendations for the Secretary to consider.
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Therefore, it is important that sponsors design CER studies to ensure adequate numbers of all relevant population and patient subgroups, including all genders and patients representing a wide range of races, ethnicities, levels of health literacy, and ages, as well as those with multiple chronic conditions. The following sections conduct a "portfolio analysis" -- an analysis of the distribution of the committee's final 100 priority topics across the portfolio variables, including (1)
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, and racial and ethnic disparities. In addition, if that research question involved comparing alternative organizational approaches to care, such as coordinated disease management programs or remote monitoring of patients' symptoms, the topic could also be classified under the health care delivery system area.
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3 1 4 Immune System, Connective Tissue, and Joint 1 3 4 Disorders Eyes, Ears, Nose, and Throat Disorders 2 1 3 Trauma, Emergency Medicine, and Critical Care 1 2 3 Medicine Complementary and Alternative Medicine 3 0 3 Kidney and Urinary Tract Disorders 2 1 3 Oral Health 2 1 3 Respiratory Disease 1 2 3 Genetics and Disease 0 3 3 Gastrointestinal System Disorders 1 1 2 Palliative and End-of-Life Care 2 0 2 Sexual Function and Reproductive Disorders 0 2 2 Liver and Biliary Tract Disorders 1 1 2 Total 100 193 293 * Although this category was described as "Safety and Quality of Health Care" in the webbased questionnaire, the category was relabeled by the committee as "Health Care Delivery Systems" to be more accurate.
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FIGURE 5-1 Distribution of the recommended research priorities by primary and secondary research areas.
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The prominence of health care delivery systems in the portfolio primarily reflects the interest of the public in this area, as well as the committee's belief that an early investment in CER should focus on learning how to make services more effective. Nearly one-third of the total recommended topics involve research that addresses racial and ethnic disparities and nearly one-fifth address functional limitations and disabilities.
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Table 5-3 displays the 100 final priority topics categorized by type of intervention or strategy proposed for the CER study. Types of comparators represented in the portfolio range from institutional and organizationbased, such as management and delivery of health care, to patient-centered interventions.
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The interventions most strongly represented in the committee's portfolio are systems of care, pharmacologic treatment, and standard of care comparisons. Other frequently proposed types of interventions include behavioral treatments, disease prevention modalities, medical or surgical procedures (including radiological procedures)
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INTRODUCTION TO FINAL LIST OF PRIORITY TOPICS In preparing the list for presentation in this report, the committee refined the wording of each priority topic to fit a common format that indicates the research area, two or more interventions to be compared, the population, and, where appropriate and feasible, the outcomes of interest. The committee did not attempt to change the essence of the research question, or to change or add specific outcomes, nor did the committee attempt to refine the topics by specifying methodologies or comparators that the nominator did not provide.
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Therefore, the 100 priority topics are presented grouped into quartiles, listed alphabetically by primary area of research.4 The first quartile contains all topics with a mean score between 3.5 and 7.4 (see Box 5-1 for a brief recap of how the voting was conducted)
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HCDS Compare the effectiveness of dissemination and translation techniques to facilitate the use of CER by patients, clinicians, payers, and others. HCDS Compare the effectiveness of comprehensive care coordination programs, such as the medical home, and usual care in managing children and adults with severe chronic disease, especially in populations with known health disparities.
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PEDS Compare the effectiveness of various primary care treatment strategies (e.g., symptom management, cognitive behavior therapy, biofeedback, social skills, educator/teacher training, parent training, pharmacologic treatment) for attention deficit hyperactivity disorder (ADHD)
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RED Compare the effectiveness of literacy-sensitive disease management programs and usual care in reducing disparities in children and adults with low literacy and chronic disease (e.g., heart disease)
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HCDS Compare the effectiveness of accountable care systems and usual care on costs, processes of care, and outcomes for geographically defined populations of patients with one or more chronic diseases. HCDS Compare the effectiveness of different residential settings (e.g., home care, nursing home, group home)
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PSYCH Compare the effectiveness of pharmacologic treatment and behavioral interventions in managing major depressive disorders in adolescents and adults in diverse treatment settings. RD Compare the effectiveness of an integrated approach (combining counseling, environmental mitigation, chronic disease management, and legal assistance)
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ENDO Compare the effectiveness and cost-effectiveness of conventional medical management of type 2 diabetes in adolescents and adults, versus conventional therapy plus intensive educational programs or programs incorporating support groups and educational resources. HCDS Compare the effectiveness of alternative redesign strategies -- using decision support capabilities, electronic health records, and personal health records -- for increasing health professionals' compliance with evidence-based guidelines and patients' adherence to guideline-based regimens for chronic disease care.
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NEURO Compare the effectiveness of comprehensive, coordinated care and usual care on objective measures of clinical status, patient-reported outcomes, and costs of care for people with multiple sclerosis. NUTR Compare the effectiveness of treatment strategies for obesity (e.g., bariatric surgery, behavioral interventions, pharmacologic treatment)
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ENDO Compare the effectiveness of different disease management strategies in improving the adherence to and value of pharmacologic treatments for the elderly. HCDS Compare the effectiveness of care coordination with and without clinical decision supports (e.g., electronic health records)
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for informing patients about proposed treatments during the process of informed consent. HCDS Compare the effectiveness of different disease management strategies for activating patients with chronic disease.
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WH Compare the effectiveness of different strategies for promoting breastfeeding among low-income African American women. NOTE: ADDO = Alcoholism, Drug Dependency, and Overdose; BDEV = Birth and Developmental Disorders; CAD = Cardiovascular and Peripheral Vascular Disease; CAM = Complementary and Alternative Medicine; DIS = Functional Limitations and Disabilities; EENT = Eyes, Ears, Nose, and Throat Disorders; ENDO = Endocrinology and Metabolism Disorders and Geriatrics; GI = Gastrointestinal System Disorders; HCDS = Health Care Delivery Systems; IMUN = Immune System, Connective Tissue, and Joint Disorders; INFD = Infectious Diseases Liver and Biliary Tract Disorders; KUT = Kidney and Urinary Tract Disorders; MS = Musculoskeletal Disorders; NEURO = Neurologic Disorders; NUTR = Nutrition (including obesity)
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, which involve disease management (a comprehensive approach to caring for patients with chronic diseases) , clinical guidelines (as followed by both clinicians and patients)
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HCDS-G Compare the effectiveness of different disease management strategies for activating patients with chronic disease. HCDS-H Compare the effectiveness of alternative redesign strategies -- using decision support capabilities, electronic health records, and personal health records -- for increasing health professionals' compliance with evidence-based guidelines and patients' adherence to guideline-based regimens for chronic disease care.
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. HCDS-N Compare the effectiveness of comprehensive care coordination programs, such as the medical home, and usual care in managing children and adults with severe chronic disease, especially in populations with known health disparities.
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. In addition, the AHRQ Effective Health Care program, Healthy People 200, and the Cochrane Collaboration rank cardiovascular disease among the highest national priorities for health (Doyle et al., 2005; HHS, 2000; Whitlock et al., 2009)
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CAD-F Compare the effectiveness of treatment strategies for atrial fibrillation including surgery, catheter ablation, and pharmacologic treatment. CAD-G Compare the effectiveness of treatment strategies for vascular claudication (e.g., medical optimization, smoking cessation, exercise, catheter-based treatment, open surgical bypass)
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. Cancer is also listed as a national priority by the AHRQ Effective Health Care Program and Healthy People 200 (HHS, 2000; Whitlock et al., 2009)
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NEURO-B Compare the effectiveness of different treatment strategies on the frequency and lost productivity in people with chronic, frequent migraine headaches. NEURO-C Compare the effectiveness of comprehensive, coordinated care and usual care on objective measures of clinical status, patient-reported outcomes, and costs of care for people with multiple sclerosis.
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WH-D Compare the effectiveness of birthing care in freestanding birth centers and usual care of childbearing women at low and moderate risk. WH-E Compare the effectiveness of different strategies for promoting breastfeeding among low-income African American women.
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MS-C Compare the effectiveness of treatment strategies (e.g., artificial cervical discs, spinal fusion, pharmacologic treatment with physical therapy) for cervical disc and neck pain.
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Chronic infections with HIV and hepatitis C can now be treated so that people live decades. However, identifying optimal treatment strategies, particularly in African American populations and at-risk populations, such as intravenous drug users, require more research.
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As such, the committee recommended it as a priority (ENDO-A) , as did AHRQ's Effective Health Care Program and Healthy People 200 (AHRQ Effective Health Care Program, 2009; HHS, 2000)
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. The final list includes two priority topics focused on identifying effective treatment strategies for these disorders (BDEV-A–B)
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Some minority populations, such as African Americans, Asian Pacific Islanders, Latinos, and Native Americans, have higher rates of chronic diseases and also experience greater barriers to obtaining care. Together, these factors contribute to creating disparities in health status and clinical outcomes.
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to reduce health disparities in cardiovascular disease, diabetes, cancer, musculoskeletal diseases, and birth outcomes. RED-B Compare the effectiveness of literacy-sensitive disease management programs and usual care in reducing disparities in children and adults with low literacy and chronic disease (e.g., heart disease)
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SKIN-C Compare the effectiveness (including effects on quality of life) of treatment strategies (e.g., topical steroids, ultraviolet light, methotrexate, biologic response modifiers)
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. The committee recommended one priority focus on treatment strategies for hearing loss among those with diverse cultural/linguistic and medical/developmental backgrounds (DIS-A)
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Palliative and TABLE 5-25 Oral Health Priority Topics ORAL-A Compare the clinical and cost effectiveness of surgical care and a medical model of prevention and care in managing periodontal disease to increase tooth longevity and reduce systemic secondary effects in other organ systems. ORAL-B Compare the effectiveness of the various delivery models (e.g., primary care, dental offices, schools, mobile vans)
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. Both AHRQ's Effective Health Care Program and Healthy People 200 list arthritis and non-traumatic joint disorders as national research priorities (HHS, 2000; Whitlock et al., 2009)
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. Trauma, Emergency Medicine, and Critical Care Medicine Accidents are a leading cause of death for all ages in the United States, and trauma-related disorders are listed as one of the most prevalent and TABLE 5-29 Pediatric Disorders Priority Topics PEDS-A Compare the effectiveness of various primary care treatment strategies (e.g., symptom management, cognitive behavior therapy, biofeedback, social skills, educator/teacher training, parent training, pharmacologic treatment)
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TABLE 5-31 Trauma, Emergency Medicine, and Critical Care Medicine Priority Topics TEMC-A Compare the effectiveness of treatment strategies (e.g., cognitive behavioral individual therapy, generic individual therapy, comprehensive and intensive treatment) for Post-traumatic Stress Disorder stemming from diverse sources of trauma.
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2009. Identifying, selecting, and refining topics for comparative effectiveness systematic reviews: AHRQ and the Effective Health Care Program.
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