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5 Priorities for Study abstract: The Institute of Medicine Committee on Comparative Effective- ness Research Prioritization was charged with developing a portfolio of priority topics that reflected balance across research areas, populations, type of interventions, and methodologies. The final list of 00 prior- ity CER topics includes a large number addressing health care delivery systems, and a large number that consider racial and ethnic disparities. All but  of the  originally delineated research areas are represented. Similarly, the priority research topics include studies examining various special population categories, including individuals with rare diseases. This chapter presents the full list of priority CER topics. As explained in detail in Chapter 1, the Institute of Medicine (IOM) 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. To develop the list, the committee obtained substantial public input (described in Chapter 3) and followed a multistage process of individual and collective deliberation (described in Chapter 4). The final portfolio, described in this chapter, contains 100 priority topics. The first half of the chapter is a “portfolio analysis,” which shows the representation of research areas, study popula- tions, comparators, and study methodologies within the final 100 topics. The second half of the chapter presents the specific CER topics prioritized by the committee, together with a description of their relevance. 

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 INITIAL NATIONAL PRIORITIES FOR CER ASSEMbLING A DIvERSE PORTFOLIO As described in Chapter 4, the committee utilized the concept of a diverse research portfolio, meaning that the committee’s priority topics reflect a balance of CER questions across research area (i.e., disorders by organ systems, specific populations, systems of care), study populations (i.e., men, women, children, minority groups), types of interventions (i.e., comparators, such as surgical or pharmaceutical treatments), and study methodologies (i.e., randomized controlled trials, registry studies, system- atic reviews). The committee wanted to ensure that the final list of topics represents not only those diseases and conditions with the greatest effects on the health of the U.S. population, but also that it includes other diseases and conditions that disproportionately and seriously affect subgroups of the population (such as women, minorities, and children and adolescents). In addition, the committee wanted to ensure its priority topics examine a variety of interventions, including studies examining prevention, systems of care, pharmacological treatments, devices, surgery, and monitoring of disease. The committee also sought to achieve balance in the distribution of proposed methodologies so that some answers could be obtained within the 2-year framework specified by the American Recovery and Reinvest- ment Act (ARRA) of 2009, while other research questions would require a longer timeframe. For example, CER conducted from established databases and from systematic reviews of the current literature holds the potential to provide information relatively rapidly, whereas performance of randomized controlled clinical trials or prospective observational trials would extend well beyond the 2-year focus of the ARRA. The committee strongly believes that CER should be conducted us- ing “real-world” patients, so that results are readily generalizable across populations. 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, eth- nicities, 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) research areas, (2) study populations, (3) interventions, and (4) study methodologies. A successful portfolio is one that is widely distributed across these dimensions. It is important to recog- nize that the precision of the information in this section was limited by the procedures that were required to meet the committee’s deadline. In the fu- ture, thorough topic nomination development requires interaction with the nominators and other stakeholders to sufficiently develop the nomination

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 PRIORITIES FOR STUDY and to ensure that the supporting evidence accurately conveys the context and the main points of the nomination (Whitlock et al., 2009). The following sections display the distribution of the committee’s pri- ority list by the portfolio criteria: research area, population, intervention, and methodology. In addition, an interactive electronic file providing search capabilities for priority topics by portfolio criteria is available at www. iom.edu/cerpriorities. This spreadsheet will allow the reader to search, for example, all cardiovascular disease topics affecting women and children, or to study the effectiveness of procedures for their treatment. The search will also indicate which quartile the committee assigned each topic. DIvERSITy OF RESEARCH AREAS As described earlier, one of the committee’s main methods of catego- rizing the proposed priority topics was by research area. The committee identified 32 categories of research areas based on disease classification, other patient conditions, and systems of care.1 However, because many of the conditions co-occur frequently (e.g., obesity and osteoarthritis), and many of the nominated priorities mentioned both a disease and a system of care (e.g., Alzheimer’s disease and nursing home care), most of the priority topics could be classified according to two or more research areas.2 For example, a topic to study alternative strategies for treating heart disease in African American patients with diabetes could have been classified as cardiovascular disease, endocrinology (which includes diabetes care), and racial and ethnic disparities. In addition, if that research question involved comparing alternative organizational approaches to care, such as coordi- nated disease management programs or remote monitoring of patients’ symptoms, the topic could also be classified under the health care delivery system area. In fact, among the final 100 priority topics, the average num- ber of assignable research categories was three. To determine whether the committee’s priority list was balanced across research areas, each priority was categorized by all of the possible research areas that reasonably described it. For the purposes of this exercise, one area was designated as the primary topic. Table 5-1 and Figure 5-1 show the breakdown of the 100 final priority topics categorized by research area. In Table 5-1, the topic’s primary research area is shown with assigned second- ary research areas, if reported. Several areas are prominently represented. 1 Refer to Chapter 3 to see how the committee developed the list of 32 research area categories. 2 In the classification exercise that took place at each stage of the IOM committee’s delibera- tions, however, each nominated recommendation was placed into only one area, which was considered its primary research area.

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00 INITIAL NATIONAL PRIORITIES FOR CER TAbLE 5-1 Recommended Research Priorities by Research Area Primary Secondary Research Research Category Area Area Total Health Care Delivery Systems* 23 27 50 Racial and Ethnic Disparities 3 26 29 Cardiovascular and Peripheral Vascular Disease 8 13 21 Geriatrics 2 19 21 Functional Limitations and Disabilities 2 20 22 Neurologic Disorders 6 11 17 Psychiatric Disorders 7 10 17 Pediatrics 1 15 16 Endocrinology and Metabolism Disorders 2 12 14 Musculoskeletal Disorders 5 7 12 Oncology and Hematology 6 5 11 Women’s Health 5 2 7 Alcoholism, Drug Dependency, and Overdose 2 4 6 Infectious Diseases 3 2 5 Skin Disorders 3 1 4 Birth and Developmental Disorders 3 1 4 Nutrition (including obesity) 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 web- based questionnaire, the category was re-labeled by the committee as “Health Care Delivery Systems” to be more accurate.

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Number of Priority Topics 0 10 20 30 40 50 60 Health Delivery Disparities Disabilities Cardiovascular Geriatrics Psychiatry Neurology Pediatrics Endocrinology Musculoskeletal Oncology/Hematology Women's Health Substance Abuse Infectious Diseases Developmental R01511 Rheumatology Figure 5-1 Nutrition priorities Area Dermatology Complementary/Alternative EENT Genetics Primary Research Area Genitourinary Secondary Research Area Oral Health Respiratory Emergency/Critical Care Gastrointestinal FIGuRE 5-1 Distribution of the recommended research Researchby primary and secondary research areas. Hepatobiliary End-of-Life Care Reproduction 0

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0 INITIAL NATIONAL PRIORITIES FOR CER Half of all topics involve a comparison to some aspect of the health care de- livery system. Research topics categorized in this group focus on comparing how or where services are provided, rather than which services are provided. 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 be- lief that an early investment in CER should focus on learning how to make services more effective. Nearly one-third of the total recommended topics in- volve research that addresses racial and ethnic disparities and nearly one-fifth address functional limitations and disabilities. Other frequently represented areas are cardiovascular disease, geriatrics, psychiatric disorders, neurologic disorders, and pediatrics. Twenty-nine out of the original 32 research areas are represented in the final portfolio. The missing categories include medical aspects of bioterror- ism, pancreatic disorders, and regenerative medicine. The fact that there are no topics from any of these categories in the final list is less of a reflection of these categories’ importance than of the fact that these categories only received 2 nominations out of the total 1,268 topics that entered the first round of voting and that the committee did not score the particular topics nominated within these categories as highly as topics in other categories. The portfolio’s inclusion of 29 out of the original 32 research areas suggests that an investment in CER based on the committee’s portfolio recommen- dations would comprehensively explore a broad spectrum of disease. It is interesting to note that, when asked for input, the public responded with recommendations that spanned a full portfolio of research areas.3 DIvERSITy OF POPuLATIONS A balanced portfolio should include a consideration of the demo- graphic characteristics of the populations and subpopulations to be stud- ied, including minority, racial, and ethnic groups; gender; and different age groups ranging from infancy to the elderly. It should also consider less obvious factors that affect health care, such as geographic location, socioeconomic status, educational achievement, and cultural differences; and it should be proportionately representative of those factors. Table 5-2 displays the 100 final priority topics categorized by study population. Many of the nominators of the priority topics selected more than one population as appropriate for the proposed research. Adults, including the elderly and the general population, are the most frequently represented study popula- tions in the committee’s portfolio. Other populations well represented in 3 As discussed in Chapter 3, 82 percent of the committee’s final priority list were nominated by the public; 18 percent were nominated by the committee during its in-depth discussion of the priority list.

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0 PRIORITIES FOR STUDY TAbLE 5-2 Committee’s Recommended Research Priorities by Study Populations Study Population Number of Topics Adults (including elderly) 36 Population at Large (general population) 28 Women 27 Special Populations (e.g., pregnant women, low income, patients with 24 disabilities) Men 22 Children and Adolescents Only 20 Elderly Only 15 Other 12 Long-Term Care 7 Ethnic Subpopulations Only 5 Adults (excluding elderly) 4 Rare Diseases 2 Total 202 NOTE: The total exceeds the total number of priority topics because respondents were al- lowed to select multiple populations for each topic. the committee’s portfolio are women, special populations (such as pregnant women and low-income families and individuals), men, and children and adolescents. Based on the answers to the open-ended questions given by the ques- tionnaire respondents, the “other” category in the table encompasses a wide variety of study populations, such as those with chronic conditions, cancer survivors, persons with psychiatric and mental disabilities, and persons at risk of developing heart disease. DIvERSITy OF INTERvENTIONS Another component of a balanced portfolio is that it should cover all steps in the trajectory of health care, from prevention and screening to diagnosis and treatment of acute and chronic health problems to palliative and end-of-life care. It should also reflect the full range of care modalities, from behavioral changes to pharmacological treatment to radiation to sur- gery. 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 organization- based, such as management and delivery of health care, to patient-centered interventions. The patient-centered interventions range from completely

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0 INITIAL NATIONAL PRIORITIES FOR CER TAbLE 5-3 Committee’s Recommended Research Priorities by Types of Intervention Types of Interventions Number of Topics Systems of Care 43 Pharmacological Treatment 36 Standard of Care 33 Behavioral Treatment 29 Prevention 24 Procedures 23 Provider-Patient Relationships 20 Treatment Pathways 19 Testing, Monitoring, and Evaluation 17 Devices 13 Alternative Treatment 9 Other 18 Total 284 NOTE: The total exceeds the total number of priority topics because respondents were al- lowed to select multiple interventions to be compared for each topic. noninvasive approaches, such as ways to persuade patients to adopt health- ier behavior, to major surgical procedures. The interventions most strongly represented in the committee’s port- folio 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), provider-patient forms of communication or other features of provider-patient relations, and treat- ment pathways (or clinical guidelines). The list includes a broad array of diagnostic and therapeutic actions taken by primary care physicians and specialists. It also includes actions taken by other health professionals, ancillary service providers, administra- tors, and, importantly, health care leaders—for example, professional as- sociations that develop treatment pathways. The “other” category includes interventions such as complementary care and economic incentives. DIvERSITy OF STuDy METHODOLOGIES Table 5-4 displays the division of the 100 final priority topics by study methodology. The four major methodologies identified by the committee as appropriate for CER are well represented on the committee’s portfolio. Thus, the committee’s portfolio provides a list of CER questions that vary

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0 PRIORITIES FOR STUDY TAbLE 5-4 Committee’s Recommended Research Priorities by Study Methodology Methodology Number of Topics Randomized Trial 49 Prospective Observational Study 46 Database Research 27 Systematic Review 23 TOTAL 145 NOTE: The total exceeds the total number of priority topics because respondents were al- lowed to select multiple methodologies for each topic. widely in terms of resource requirements, timelines, and types of infra- structure necessary to conduct the research. For example, a database study using existing databases could be performed more rapidly and economically than a randomized clinical trial, but its findings and conclusions may be less definitive. The appropriate choice of method depends on the nature of the research, on whether the intervention is currently in use, on whether sufficient data are available to identify a large group of persons receiving the intervention and suitable unbiased comparator groups, and whether a range of patient outcomes is recorded. 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 ques- tion, 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. The committee fully anticipates that funding agencies, when preparing their Requests for Applications based on these priority topics, will provide details on the scope of the clinical problem, the current best practices, and the potential alternative approaches. It is ultimately the responsibility of the research teams applying for funding to propose the precise population, comparators, outcomes, and methodologies to be undertaken in the studies attempting to answer the priority questions. Moreover, a single priority topic is likely to generate alternative designs, so the committee’s 100 priorities will likely provide the opportunity for many more than 100 specific research studies.

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0 INITIAL NATIONAL PRIORITIES FOR CER BOX 5-1 Round 3 Voting Procedures One hundred fifty-five nominated research topics were considered in the committee’s third round of voting. Each committee member was allocated 300 total points to distribute among the 155 topics but could not award more than 30 points to any one topic. The mean score for each topic was calculated by dividing the total points that each topic received by the number of committee members voting. The raw scores were reviewed by the committee, and the distribution of the scores provided a natural cutoff at 100 topics. The top 100 topics all received a mean of at least 1.0 points. TAbLE 5-5 Results of the IOM Committee’s Final Vote for Priority Topics, by Quartile Range Standard Quartile Mean Score Deviation Low High 1 4.6 1.0 3.5 7.4 2 2.9 0.3 2.5 3.4 3 2.0 0.3 1.5 2.4 4 1.3 0.1 1.0 1.4 The voting process (described in detail in Chapter 4) introduced a sub- stantial degree of subjectivity and variable weighting of topics. The com- mittee felt that this imprecision reduced the reliability of relative rankings. 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). The second quartile contains all topics with a mean score between 2.5 and 3.5. The third quartile contains all topics with a mean score between 1.5 and 2.5. The fourth quartile con- tains all topics with a mean score between 1 and 1.5. Refer to Table 5-5 to see the variability and ranges of the committee’s votes across quartile. Table 5-6 displays the 100 priority topics by quartile. The medical terminology used in the list of priorities is defined in Appendix E. 4 Note that 55 of the 155 nominated recommendations that appeared on the final ballot did not score high enough to be included in the final list. These 55 items are not represented in the quartiles.

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0 PRIORITIES FOR STUDY TAbLE 5-6 Final List of Priority Topics, by Quartile Ratings *display within quartile does not indicate priority rank—topics are listed alphabetically by primary research area First Quartile (listed alphabetically by primary research area) CAD Compare the effectiveness of treatment strategies for atrial fibrillation including surgery, catheter ablation, and pharmacologic treatment. DIS Compare the effectiveness of the different treatments (e.g., assistive listening devices, cochlear implants, electric-acoustic devices, habilitation and rehabilitation methods [auditory/oral, sign language, and total communication]) for hearing loss in children and adults, especially individuals with diverse cultural, language, medical, and developmental backgrounds. ENDO Compare the effectiveness of primary prevention methods, such as exercise and balance training, versus clinical treatments in preventing falls in older adults at varying degrees of risk. GI Compare the effectiveness of upper endoscopy utilization and frequency for patients with gastroesophageal reflux disease on morbidity, quality of life, and diagnosis of esophageal adenocarcinoma. 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. IMUN Compare the effectiveness of different strategies of introducing biologics into the treatment algorithm for inflammatory diseases, including Crohn’s disease, ulcerative colitis, rheumatoid arthritis, and psoriatic arthritis. INFD Compare the effectiveness of various screening, prophylaxis, and treatment interventions in eradicating methicillin resistant Staphylococcus aureus (MRSA) in communities, institutions, and hospitals. INFD Compare the effectiveness of strategies (e.g., bio-patches, reducing central line entry, chlorhexidine for all line entries, antibiotic impregnated catheters, treating all line entries via a sterile field) for reducing health care associated infections (HAI), including catheter-associated bloodstream infection, ventilator associated pneumonia, and surgical site infections in children and adults. KUT Compare the effectiveness of management strategies for localized prostate cancer (e.g., active surveillance, radical prostatectomy [conventional, robotic, and laparoscopic], and radiotherapy [conformal, brachytherapy, proton-beam, and intensity-modulated radiotherapy]) on survival, recurrence, side effects, quality of life, and costs. continued

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 INITIAL NATIONAL PRIORITIES FOR CER disease management strategies on the efficiency and value of pharmacologi- cal treatments (ENDO-D). There are multiple other topics that affect the elderly population; these topics are listed according to the specific organ system or disease area to which they pertain. birth and Developmental Disorders The uncertainty surrounding the root causes of social-emotional dis- orders in infants and toddlers, as well as autism spectrum disorder, has resulted in a lack of effective treatment options for these individuals. As a result, AHRQ’s Effective Health Care Program recommended this as a na- tional priority area for CER (Whitlock et al., 2009). The final list includes two priority topics focused on identifying effective treatment strategies for these disorders (BDEV-A–B) (Table 5-16). With the remarkable improve- ment in survival and attendant costs for premature infants, the impact of support programs on child and family outcomes after a child is discharged from a neonatal intensive care unit (NICU) (BDEV-C) was felt to be of sig- nificant value. For specific topics related to pregnancy, refer to the Women’s Health category. Complementary and Alternative Medicine The widespread use of complementary and alternative methodologies (including yoga, meditation, acupuncture, and nutriceuticals [CAM-A–C]) in managing a broad array of disorders (e.g., anxiety and depression, pain, cardiovascular risk factors, chronic diseases, other prevalent conditions) TAbLE 5-16 Birth and Developmental Disorders Priority Topics BDEV-A Compare the effectiveness of therapeutic strategies (e.g., behavioral or pharmacologic interventions, the combination of the two) for different autism spectrum disorders (ASD) at different levels of severity and stages of intervention. BDEV-B Compare the effectiveness of the co-location model (psychological and primary care practitioners practicing together) and usual care (identification by primary care practitioner and referral to community-based mental health services) in identifying and treating social-emotional and developmental disorders in children ages 0-3. BDEV-C Compare the effectiveness of diverse models of comprehensive support services for infants and their families following discharge from a neonatal intensive care unit.

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 PRIORITIES FOR STUDY TAbLE 5-17 Complementary and Alternative Medicine Priority Topics CAM-A Compare the effectiveness of mindfulness-based interventions (e.g., yoga, meditation, deep breathing training) and usual care in treating anxiety and depression, pain, cardiovascular risk factors, and chronic diseases. CAM-B Compare the effectiveness of acupuncture for various indications using a cluster randomized trial. CAM-C Compare the effectiveness of dietary supplements (nutriceuticals) and usual care in the treatment of selected high-prevalence conditions. provides the impetus to compare their effectiveness to more conventional approaches to care (Table 5-17). Nutrition Obesity is a growing epidemic with medical consequences that extend to multiple chronic conditions, such as diabetes, hypertension, heart dis- ease, and arthritis. Within the medical community, there is currently uncer- tainty regarding effective strategies for preventing and treating obesity. The committee recommended priorities that compare strategies for improving social conditions to reduce obesity (NUTR-A), including various school policies (NUTR-B) (Table 5-18). Both of these priorities include a focus on populations with varying risk rates. Identifying effective methods for treat- ing obese populations could significantly improve health in this country. As such, the committee recommends comparing the effectiveness of surgical procedures, such as bariatric surgery (gastric bypass), behavior modifica- tion, and medication (NUTR-C). Racial and Ethnic Disparities Disparities in access to care and in clinical outcomes between different populations were of considerable concern for the committee. Some minority populations, such as African Americans, Asian Pacific Islanders, Latinos, and Native Americans, have higher rates of chronic diseases and also expe- rience greater barriers to obtaining care. Together, these factors contribute to creating disparities in health status and clinical outcomes. The commit- tee recommends comparing the effectiveness of several strategies aimed at reducing these disparities, including community-based and multi-level interventions (RED-A), providing literacy sensitive disease management programs (RED-B), and strategies to improve engagement and retention (RED-C) (Table 5-19).

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0 INITIAL NATIONAL PRIORITIES FOR CER TAbLE 5-18 Nutrition Priority Topics NUTR-A Compare the effectiveness of various strategies (e.g., clinical interventions, selected social interventions [such as improving the built environment in communities and making healthy foods more available], combined clinical and social interventions) to prevent obesity, hypertension, diabetes, and heart disease in at-risk populations such as the urban poor and American Indians. NUTR-B Compare the effectiveness of school-based interventions involving meal programs, vending machines, and physical education, at different levels of intensity, in preventing and treating overweight and obesity in children and adolescents. NUTR-C Compare the effectiveness of treatment strategies for obesity (e.g., bariatric surgery, behavioral interventions, pharmacologic treatment) on the resolution of obesity-related outcomes such as diabetes, hypertension, and musculoskeletal disorders. Skin Disorders Skin disorders across the country are widespread, cause a high degree of morbidity, and are among the most costly disorders in children and ado- lescents between ages 1 and 17 (AHRQ, 2009a,b,c). The committee’s priori- ties on skin disorders include chronic conditions such as lower extremity wounds (common complications in patients with diabetes, peripheral vascu- lar disease, and paralysis) (SKIN-A), and acne—specifically comparing the long-term safety and effectiveness of alternative treatments (SKIN-B) (Table 5-20). Another topic focused on reducing skin disease and comparing treat- ments to improve quality of life for chronic psoriatic disease (SKIN-C). TAbLE 5-19 Race and Ethnic Disparities Priority Topics RED-A Compare the effectiveness of interventions (e.g., community-based multi- level interventions, simple health education, usual care) 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). RED-C Compare the effectiveness of different strategies to engage and retain patients in care and to delineate barriers to care, especially for members of populations that experience health disparities.

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 PRIORITIES FOR STUDY TAbLE 5-20 Skin Disorders Priority Topics SKIN-A Compare the effectiveness of topical treatments (e.g., antibiotics, platelet- derived growth factor) and systemic therapies (e.g., negative pressure wound therapy, hyperbaric oxygen) in managing chronic lower extremity wounds. SKIN-B Compare the effectiveness of different long-term treatments for acne. SKIN-C Compare the effectiveness (including effects on quality of life) of treatment strategies (e.g., topical steroids, ultraviolet light, methotrexate, biologic response modifiers) for psoriasis. Alcoholism, Drug Dependency, and Overdose The harms of tobacco smoking are well known and well documented. Yet, roughly one-fifth of the nation’s population continues to smoke. The committee recommended that a national priority for comparative effective- ness should be to examine alternative smoking cessation strategies in un- derstudied populations such as minorities, individuals with mental illness, and adolescents (ADDO-A) (Table 5-21). The Cochrane Collaboration and Healthy People 00 also include tobacco use as national priorities (Doyle et al., 2005; HHS, 2000). The increasing prevalence of abuse of and dependency on pain medica- tions led the committee to recommend an examination of treatment and prescribing practices to reduce substance dependence for patients with non- cancer chronic pain and acute pain (ADDO-B). Functional Limitations and Disabilities While many of the committee’s priority topics affect patients with dis- abilities, the following topics specifically address two populations: (1) the TAbLE 5-21 Alcoholism, Drug Dependency, and Overdose Priority Topics ADDO-A Compare the effectiveness of smoking cessation strategies (e.g., medication, individual or quitline counseling, combinations of these) in smokers from understudied populations such as minorities, individuals with mental illness, and adolescents. ADDO-B Compare the effectiveness of different opioid and non-opioid pain relievers, in different doses and durations, in avoiding unintentional overdose and substance dependence among subjects with acute and non-cancer chronic pain.

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 INITIAL NATIONAL PRIORITIES FOR CER TAbLE 5-22 Functional Limitations and Disability Priority Topics DIS-A Compare the effectiveness of the different treatments (e.g., assistive listening devices, cochlear implants, electric-acoustic devices, habilitation and rehabilitation methods [auditory/oral, sign language, and total communication]) for hearing loss in children and adults, especially individuals with diverse cultural, language, medical, and developmental backgrounds. DIS-B Compare the effectiveness of focused intense periodic therapy and usual weekly therapy in managing cerebral palsy in children. hearing-impaired, and (2) children with cerebral palsy (Table 5-22). The committee recommended one priority focus on treatment strategies for hearing loss among those with diverse cultural/linguistic and medical/de- velopmental backgrounds (DIS-A) and another on usual care compared to focused and intense periodic therapy sessions to manage symptoms re- lated to cerebral palsy (DIS-B). Eyes, Ears, Nose, and Throat Disorders The committee included two topics on eye disorders: (1) comparing the effectiveness of alternative treatment strategies for diabetic retinopathy, macular degeneration, and retinal vein occlusion (EENT-A), and (2) com- paring strategies for treatment of primary open-angle glaucoma (EENT-B), including a focus on minority populations (Table 5-23). Kidney and urinary Tract Disorders The committee identified prostate cancer and renal replacement thera- pies as priority areas for comparative effectiveness research (Table 5-24). Because prostate cancer is the second leading cause of cancer death in men TAbLE 5-23 Ears, Eyes, Nose, and Throat Disorders Priority Topics EENT-A Compare the effectiveness of different treatment options (e.g., laser therapy, intravitreal steroids, anti-vascular endothelial growth factor [anti-VEGF]) for diabetic retinopathy, macular degeneration, and retinal vein occlusion. EENT-B Compare the effectiveness of treatment strategies for primary open-angle glaucoma (e.g., initial laser surgery, new surgical techniques, new medical treatments) particularly in minority populations to assess clinical and patient- reported outcomes.

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 PRIORITIES FOR STUDY TAbLE 5-24 Kidney and Urinary Tract Disorders Priority Topics KUT-A Compare the effectiveness of management strategies for localized prostate cancer (e.g., active surveillance, radical prostatectomy [conventional, robotic, and laparoscopic], and radiotherapy [conformal, brachytherapy, proton- beam, and intensity-modulated radiotherapy]) on survival, recurrence, side effects, quality of life, and costs. KUT-B Compare the effectiveness (including survival, hospitalization, quality of life, and costs) of renal replacement therapies (e.g., daily home hemodialysis, intermittent home hemodialysis, conventional in-center dialysis, continuous ambulatory peritoneal dialysis, renal transplantation) for patients of different ages, races, and ethnicities. (U.S. Cancer Statistics Working Group, 2009), the committee recommended that all aspects of managing the disease be studied (KUT-A). Renal failure is among the leading causes of mortality across all age groups (Kung et al., 2008). It is also one of the most costly diseases in adults over 65 years of age (AHRQ, 2009a). As such, the committee recommended comparing alternative renal replacement therapies with an emphasis on determining the effectiveness differences among different ages, race, and ethnicities (KUT-B). Oral Health The committee recommended two priority topics within oral health for CER, one comparing prevention to surgery in adults with periodontal disease (ORAL-A), and the other in children comparing delivery model ap- proaches for preventing dental caries (cavities) (ORAL-B) (Table 5-25). Palliative and End-of-Life Care Effective management and delivery of palliative and end-of-life care is a challenge as the elderly population grows in the United States. 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) in preventing dental caries in children.

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 INITIAL NATIONAL PRIORITIES FOR CER TAbLE 5-26 Palliative and End-of-Life Care Priority Topics PELC-A Compare the effectiveness of coordinated care (supported by reimbursement innovations) and usual care in long-term and end-of-life care of the elderly. PELC-B Compare the effectiveness of hospital-based palliative care and usual care on patient-reported outcomes and cost. end-of-life care services must be effective for a variety of populations, and in a variety of environments, including hospitals, long-term care facilities, and homes. The committee specifically recommends research comparing strategies to improve delivery of long-term and end-of-life care, including reimbursement models to support coordinated care (PELC-A) and compar- ing hospital-based palliative care services with standard care to standard care alone (PELC-B) (Table 5-26). Gastrointestinal System Disorders Disorders of the upper gastrointestinal tract, such as gastroesophageal reflux disease (GERD), are among the most prevalent disorders in the na- tion, and they are particularly prevalent among the elderly (AHRQ, 2009c). They are also among the most costly conditions for infants less than 1 year old (AHRQ, 2009a). The committee specifically recommends the research of the effects of endoscopy on the management and outcomes of patients with GERD as a priority (GI-A) (Table 5-27). Immune System, Connective Tissue, and Joint Disorders Conditions of the immune system, connective tissue, and joints such as arthritis and connective tissue disorders are some of the most prevalent and costly diseases in all age groups, especially in the elderly (AHRQ, 2009a,c). Both AHRQ’s Effective Health Care Program and Healthy People 00 list arthritis and non-traumatic joint disorders as national research priorities (HHS, 2000; Whitlock et al., 2009). The committee recommended com- paring the effectiveness of different strategies, including biologics, in the treatment of these diseases (IMUN-A) (Table 5-28). TAbLE 5-27 Gastrointestinal System Disorders Priority Topics GI-A Compare the effectiveness of upper endoscopy utilization and frequency for patients with gastroesophageal reflux disease on morbidity, quality of life, and diagnosis of esophageal adenocarcinoma.

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 PRIORITIES FOR STUDY TAbLE 5-28 Immune System, Connective Tissue, and Joint Disorders Priority Topics IMUN-A Compare the effectiveness of different strategies of introducing biologics into the treatment algorithm for inflammatory diseases, including Crohn’s disease, ulcerative colitis, rheumatoid arthritis, and psoriatic arthritis. Pediatrics There are a variety of alternative and pharmacological treatments avail- able for children with attention deficit hyperactivity disorder (ADHD), but more research is needed to compare their effectiveness. In fact, AHRQ’s Effective Health Care Program lists ADHD as a national priority (Whitlock et al., 2009). The committee recommended more research that addresses the comparative effectiveness of these treatments in decreasing the symptoms of ADHD in children (PEDS-A) (Table 5-29). There are a number of other important pediatric topics that are discussed under the research area catego- ries eyes, ears, nose and throat; functional limitations and disabilities; birth and developmental disorders; nutrition; and respiratory disease. Respiratory Disease Chronic Obstructive Pulmonary Disease (COPD) and asthma are among the most prevalent, most costly, and morbid conditions for all age groups (AHRQ, 2009a,c; Kung et al., 2008). Asthma is especially common in children and is the leading condition in terms of cost (AHRQ, 2009a). In addition, AHRQ’s Effective Health Care Program lists asthma as a pri- ority research area (Whitlock et al., 2009). The committee recommended alternative strategies for managing asthma be studied through CER (RD-A) (Table 5-30). 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) for attention deficit hyperactivity disorder (ADHD) in children.

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 INITIAL NATIONAL PRIORITIES FOR CER TAbLE 5-30 Respiratory Disorders Priority Topics RD-A Compare the effectiveness of an integrated approach (combining counseling, environmental mitigation, chronic disease management, and legal assistance) with a non-integrated episodic care model in managing asthma in children. 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. costly (AHRQ, 2009a,c). While there are many disorders that arise from trauma and emergencies, the committee focused on the treatment of Post- traumatic Stress Disorder (PTSD) in all populations and from all sources of trauma. With the large number of veterans returning from the wars in Iraq and Afghanistan, and increased recognition of the inadequacies of the nation’s health system to effectively treat patients with mental health condi- tions, it is important to identify effective treatment strategies. The commit- tee recommended that PTSD be studied as part of a balanced portfolio of CER (TEMC-A) (Table 5-31). TIMELINESS AND LIMITATIONS OF THE COMMITTEE’S PRIORITy LIST The committee believes that the priority list presented in this chapter is relevant to the needs and conditions of today. New questions in CER will continue to appear. However, the balance of topics across the portfolio, the correlation with established priorities by other groups, and the good fit between the topics and the pre-established, pre-specified criteria sug- gest that the process used by the committee was effective. As discussed in Chapters 4 and 6, this process requires modification if it is to be continued in the future. Recognizing the dynamic nature of disease and the rapid technologic and therapeutic advancements in health care, these priorities may very well be answered by ongoing research or be superseded by new disorders in the near future. In fact, that is the committee’s hope and expectation. Recogni- tion of priorities and initiation of new research should provide answers to the clinical dilemmas identified. Therefore, an ongoing and active process

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 PRIORITIES FOR STUDY of priority setting using stakeholder input is imperative. The previous two chapters described systems for continuous stakeholder input, together with methodologies for identifying which of these topics deserve priority. How- ever, the committee emphasizes the importance of repeating this exercise on a regular basis or of integrating aspects of the process described here into the routine determination of CER funding in order to sustain the effort to discover what works best and for whom. REFERENCES AHRQ (Agency for Healthcare Research and Quality). 2009a. Medical Expenditure Panel Survey. Total expenses for conditions by site of service: United States http://www.meps. ahrq.gov/mepsweb/ (accessed March 10, 2009). ———. 2009b. Total number of events accounting for expenditures by site of service: United States, 2006. In Medical Expenditure Panel Survey Component Data. ———. 2009c. Total number of people accounting for expenditures (deduplicated) by site of service: United States, 2006. In Medical Expenditure Panel Survey Component Data. AHRQ Effective Health Care Program. 2009. Effective Health Care: Topic triage cover sheets. Doyle, J., E. Waters, D. Yach, D. McQueen, A. De Francisco, T. Stewart, P. Reddy, A. M. Gul- mezoglu, G. Galea, and A. Portela. 2005. Global priority setting for Cochrane systematic reviews of health promotion and public health research. Journal of Epidemiology and Community Health 59:193-197. HHS (Department of Health and Human Services). 2000. Healthy People 00: Understand- ing and improving health. Place Published: U.S. Government Printing Office. http://purl. access.gpo.gov/GPO/LPS4217 (accessed April 3, 2009). Kung, H.-C., D. L. Hoyert, J. Xu, S. L. Murphy, and Division of Vital Statistics. 2008. Deaths: Final data for 00. National vital statistics reports National Center for Health Statistics. NPP (National Priorities Partnership). 2008. National priorities and goals. Washington, DC: National Quality Forum. U.S. Cancer Statistics Working Group. 2009. United States cancer statistics: 1999–2005 Incidence and mortality web-based report. Atlanta: Department of Health and Human Services, Centers for Disease Control and Prevention and National Cancer Institute. Wennberg, J. E. 2009 (unpublished). Recommendations to the Institute of Medicine on com- parative effectiveness research priorities. Submitted in response to a request from the Institute of Medicine Committee on Comparative Effectiveness Research Prioritization. The Dartmouth Institute for Health Policy and Clinical Practice. Whitlock, E. P., S. A. Lopez, S. Chang, M. Helfand, M. Eder, and N. Floyd. 2009. Identifying, selecting, and refining topics for comparative effectiveness systematic reviews: AHRQ and the Effective Health Care Program. http://effectivehealthcare.ahrq.gov/repFiles/ 20090427IdenttifyingTopics.pdf (accessed June 5, 2009).

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