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Summary bACKGROuND Today, when a patient and physician, perhaps with other clinicians and family caregivers, are discussing the best course of treatment for the patient’s medical condition, they often do not have the scientific evidence they need to make a determination. Although there may be studies that in- dicate that a treatment is efficacious relative to a placebo, there frequently are no studies that directly compare the different available alternatives or that have examined their impacts in populations of the same age, sex, and ethnicity or with the same comorbidities as the patient. Comparative effec- tiveness research (CER) is designed to fill this knowledge gap. CER focuses attention on the evidence base to assist patients and health care providers across diverse health settings in making more informed decisions. They will need useful, practical information concerning the most effective interven- tions and health care services for their particular situation. To help identify which health care services work best, Congress, in the American Recovery and Reinvestment Act (ARRA) of 2009 (P.L. 111-5), appropriated $1.1 billion as a down-payment to provide strong federal sup- port of CER. This provision in the law reflected the legislators’ belief that better decisions about the use of health care resources could improve the public’s health and reduce the costs of care. According to the legislation, CER covers “research that compares the clinical outcomes, effectiveness, and appropriateness of items, services, and procedures that are used to prevent, diagnose, or treat diseases, disorders, and other health conditions.” The law appropriated $400 million to the National Institutes of Health 

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 INITIAL NATIONAL PRIORITIES FOR CER (NIH), $300 million to the Agency for Healthcare Research and Quality (AHRQ), and the remaining $400 million to the Secretary of Health and Human Services (HHS). According to the language of the law, the purposes of the appropriations were • “to evaluate the relative effectiveness of different health care ser- vices and treatment options” and • “to encourage the development and use of clinical registries, clini- cal data networks, and other forms of electronic data to generate outcomes data.” The law also charged the Institute of Medicine (IOM) to form a con- sensus committee and solicit stakeholder input to recommend national priorities for spending the $400 million designated for the Secretary. The legislation imposed a short time frame on this study—the IOM report deadline of June 30, 2009, was 19 weeks after the president signed the legislation into law. The National Academies President’s Fund generously supported the study process until the study’s sponsor, AHRQ, could contract with the IOM; IOM funds entirely paid for the public questionnaire and its analysis. The Robert Wood Johnson Foundation also contributed significantly to this study. This support permitted the IOM to rapidly establish a commit- tee and to commence work. The committee encompassed a broad range of expertise, perspectives, and experience, including members who work with consumers and patients, in clinical care and research, or in health care and government administration. The committee’s principal task was to prepare a list of priorities for CER funding; most of its time was spent developing a process for priority setting, eliciting a wide array of input from the public, and deliberating over a list of nominated research topics. Then, as the complexities of prior- ity setting for CER became apparent, the committee began to outline the development of an infrastructure that would sustain a long-term, national CER effort. The committee provided recommendations to implement that infrastructure required for a sustained CER effort. The main justification for including economic considerations is that the overall value of a strategy can be understood best by considering costs and benefits together. In such a circumstance, value may be judged from the perspective of the patient, provider, or payer. Many stakeholders thought CER might persuade payers to support or improve reimbursement for particular services, but the com- mittee did not discuss leveraging research findings to payment policy. The committee presents its recommended list of 100 top priority CER topics in Table S-1. The individual topics are grouped into quartiles ac- cording to the number of votes each received during the committee’s voting

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 SUMMARY process. Topics within the First Quartile were considered higher priority than those in the Fourth Quartile, but the order within quartiles does not signify rank. Following Table S-1 is a brief discussion of how the commit- tee created the priority list, a section on what the committee learned from the process, and implications and recommendations for establishing a solid foundation for CER in the future. LIST OF PRIORITy CER TOPICS TAbLE S-1 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. continued

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 INITIAL NATIONAL PRIORITIES FOR CER TAbLE S-1 Continued 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. MS Establish a prospective registry to compare the effectiveness of treatment strategies for low back pain without neurological deficit or spinal deformity. NEURO Compare the effectiveness and costs of alternative detection and management strategies (e.g., pharmacologic treatment, social/family support, combined pharmacologic and social/family support) for dementia in community-dwelling individuals and their caregivers. NEURO Compare the effectiveness of pharmacologic and non-pharmacologic treatments in managing behavioral disorders in people with Alzheimer’s disease and other dementias in home and institutional settings. NUTR 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 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. ONC Compare the effectiveness of management strategies for ductal carcinoma in situ (DCIS). ONC Compare the effectiveness of imaging technologies in diagnosing, staging, and monitoring patients with cancer including positron emission tomography (PET), magnetic resonance imaging (MRI), and computed tomography (CT). ONC Compare the effectiveness of genetic and biomarker testing and usual care in preventing and treating breast, colorectal, prostate, lung, and ovarian cancer, and possibly other clinical conditions for which promising biomarkers exist. ORAL 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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 SUMMARY TAbLE S-1 Continued 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) in children. PSYCH Compare the effectiveness of wraparound home and community-based services and residential treatment in managing serious emotional disorders in children and adults. RED 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 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). WH Compare the effectiveness of clinical interventions (e.g., prenatal care, nutritional counseling, smoking cessation, substance abuse treatment, combinations of these interventions) to reduce incidences of infant mortality, pre-term births, and low birth weights, especially among African American women. WH Compare the effectiveness of innovative strategies for preventing unintended pregnancies (e.g., over-the-counter access to oral contraceptives or other hormonal methods, expanding access to long-acting methods for young women, providing free contraceptive methods at public clinics, pharmacies, or other locations). Second Quartile (listed alphabetically by primary research area) BDEV 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 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 Compare the effectiveness of diverse models of comprehensive support services for infants and their families following discharge from a neonatal intensive care unit. continued

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 INITIAL NATIONAL PRIORITIES FOR CER TAbLE S-1 Continued CAD Compare the effectiveness of treatment strategies for vascular claudication (e.g., medical optimization, smoking cessation, exercise, catheter-based treatment, open surgical bypass). CAM 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. ENDO Compare the long-term effectiveness of weight-bearing exercise and bisphosphonates in preventing hip and vertebral fractures in older women with osteopenia and/or osteoporosis. HCDS Compare the effectiveness of shared decision making and usual care on decision outcomes (treatment choice, knowledge, treatment-preference concordance, and decisional conflict) in children and adults with chronic disease such as stable angina and asthma. HCDS Compare the effectiveness of strategies for enhancing patients’ adherence to medication regimens. HCDS Compare the effectiveness of patient decision support tools on informing diagnostic and treatment decisions (e.g., treatment choice, knowledge acquisition, treatment-preference concordance, decisional conflict) for elective surgical and nonsurgical procedures—especially in patients with limited English-language proficiency, limited education, hearing or visual impairments, or mental health problems. HCDS Compare the effectiveness of robotic assistance surgery and conventional surgery for common operations, such as prostatectomies. HCDS Compare the effectiveness (including resource utilization, workforce needs, net health care expenditures, and requirements for large-scale deployment) of new remote patient monitoring and management technologies (e.g., telemedicine, Internet, remote sensing) and usual care in managing chronic disease, especially in rural settings. HCDS Compare the effectiveness of diverse models of transition support services for adults with complex health care needs (e.g., the elderly, homeless, mentally challenged) after hospital discharge. 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) in caring for elderly patients with functional impairments.

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 SUMMARY TAbLE S-1 Continued KUT 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. MS Compare the effectiveness of treatment strategies (e.g., artificial cervical discs, spinal fusion, pharmacologic treatment with physical therapy) for cervical disc and neck pain. ONC Compare the effectiveness of film-screen or digital mammography alone and mammography plus magnetic resonance imaging (MRI) in community practice- based screening for breast cancer in high-risk women of different ages, risk factors, and race or ethnicity. ONC Compare the effectiveness of new screening technologies (such as fecal immunochemical tests and computed tomography [CT] colonography) and usual care (fecal occult blood tests and colonoscopy) in preventing colorectal cancer. PELC Compare the effectiveness of coordinated care (supported by reimbursement innovations) and usual care in long-term and end-of-life care of the elderly. 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) with a non-integrated episodic care model in managing asthma in children. SKIN Compare the effectiveness (including effects on quality of life) of treatment strategies (e.g., topical steroids, ultraviolet light, methotrexate, biologic response modifiers) for psoriasis. TEMC 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. WH Compare the effectiveness and outcomes of care with obstetric ultrasound studies and care without the use of ultrasound in normal pregnancies. WH Compare the effectiveness of birthing care in freestanding birth centers and usual care of childbearing women at low and moderate risk. continued

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 INITIAL NATIONAL PRIORITIES FOR CER TAbLE S-1 Continued Third Quartile (listed alphabetically by primary research area) ADDO 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. CAD Compare the effectiveness of aggressive medical management and percutaneous coronary interventions in treating stable coronary disease for patients of different ages and with different comorbidities. CAD Compare the effectiveness of innovative treatment strategies (e.g., cardiac resynchronization, remote physiologic monitoring, pharmacologic treatment, novel agents such as CRF-2 receptors) for congestive heart failure. CAD Compare the effectiveness of traditional risk stratification for coronary heart disease (CHD) and noninvasive imaging (using coronary artery calcium, carotid intima media thickness, and other approaches) on CHD outcomes. CAD Compare the effectiveness of different treatment strategies (e.g., modifying target levels for glucose, lipid, or blood pressure) in reducing cardiovascular complications in newly diagnosed adolescents and adults with type 2 diabetes. CAM Compare the effectiveness of acupuncture for various indications using a cluster randomized trial. CAM Compare the effectiveness of dietary supplements (nutriceuticals) and usual care in the treatment of selected high-prevalence conditions. EENT 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 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. 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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 SUMMARY TAbLE S-1 Continued HCDS Compare the effectiveness of adding information about new biomarkers (including genetic information) with standard care in motivating behavior change and improving clinical outcomes. HCDS Compare the effectiveness of different quality improvement strategies in disease prevention, acute care, chronic disease care, and rehabilitation services for diverse populations of children and adults. HCDS Compare the effectiveness of formulary management practices and usual practices in controlling hospital expenditures for products other than drugs including medical devices (surgical hemostatic products, radiocontrast, interventional cardiology devices, and others). HCDS Compare the effectiveness of different benefit design, utilization management, and cost-sharing strategies in improving health care access and quality in patients with chronic diseases (e.g., cancer, diabetes, heart disease). INFD Compare the effectiveness of HIV screening strategies based on recent Centers for Disease Control and Prevention recommendations and traditional screening in primary care settings with significant prevention counseling. MS Establish a prospective registry to compare the effectiveness of surgical and nonsurgical strategies for treating cervical spondylotic myelopathy (CSM) in patients with different characteristics to delineate predictors of improved outcomes. NEURO Compare the effectiveness of traditional and newer imaging modalities (e.g., routine imaging, magnetic resonance imaging [MRI], computed tomography [CT], positron emission tomography [PET]) when ordered for neurological and orthopedic indications by primary care practitioners, emergency department physicians, and specialists. 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) on the resolution of obesity-related outcomes such as diabetes, hypertension, and musculoskeletal disorders. ORAL 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. continued

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0 INITIAL NATIONAL PRIORITIES FOR CER TAbLE S-1 Continued PSYCH Compare the effectiveness of atypical antipsychotic drug therapy and conventional pharmacologic treatment for Food and Drug Administration- approved indications and compendia-referenced off-label indications using large datasets. PSYCH Compare the effectiveness of management strategies (e.g., inpatient psychiatric hospitalization, extended observation, partial hospitalization, intensive outpatient care) for adolescents and adults following a suicide attempt. RED 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. SKIN 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. Fourth Quartile (listed alphabetically by primary research area) ADDO 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. CAD Compare the effectiveness of computed tomography (CT) angiography and conventional angiography in assessing coronary stenosis in patients at moderate pretest risk of coronary artery disease. CAD Compare the effectiveness of anticoagulant therapies (e.g., low-intensity warfarin, aspirin, injectable anticoagulants) for patients undergoing hip or knee arthroplasty surgery. DIS Compare the effectiveness of focused intense periodic therapy and usual weekly therapy in managing cerebral palsy in children. 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) in producing good health outcomes in chronically ill patients, including children with special health care needs.

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 SUMMARY TAbLE S-1 Continued HCDS Compare the effectiveness of coordinated, physician-led, interdisciplinary care provided in the patient’s residence and usual care in managing advanced chronic disease in community-dwelling patients with significant functional impairments. HCDS Compare the effectiveness of minimally invasive abdominal surgery and open surgical procedures on post-operative infections, pain management, and recuperative requirements. HCDS Compare the effectiveness of traditional behavioral interventions versus economic incentives in motivating behavior changes (e.g., weight loss, smoking cessation, avoiding alcohol and substance abuse) in children and adults. HCDS Compare the effectiveness of diagnostic imaging performed by non-radiologists and radiologists. HCDS Compare the effectiveness of different techniques (e.g., audio, visual, written) 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. HCDS Compare the effectiveness of different delivery models (e.g., home blood pressure monitors, utilization of pharmacists or other allied health providers) for controlling hypertension, especially in racial minorities. INFD Compare the effectiveness of alternative clinical management strategies for hepatitis C, including alternative duration of therapy for patients based on viral genomic profile and patient risk factors (e.g., behavior-related risk factors). MS Compare the effectiveness of different treatment strategies in the prevention of progression and disability from osteoarthritis. MS Compare the effectiveness (e.g., pain relief, functional outcomes) of different surgical strategies for symptomatic cervical disc herniation in patients for whom appropriate nonsurgical care has failed. NEURO Compare the effectiveness of different treatment strategies on the frequency and lost productivity in people with chronic, frequent migraine headaches. NEURO Compare the effectiveness of monotherapy and polytherapy (i.e., use of two or more drugs) on seizure frequency, adverse events, quality of life, and cost in patients with intractable epilepsy. ONC Compare the effectiveness of surgical resection, observation, or ablative techniques on disease-free and overall survival, tumor recurrence, quality of life, and toxicity in patients with liver metastases. continued

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 INITIAL NATIONAL PRIORITIES FOR CER TAbLE S-1 Continued PELC Compare the effectiveness of hospital-based palliative care and usual care on patient-reported outcomes and cost. PSYCH Compare the effectiveness of different treatment approaches (e.g., integrating mental health care and primary care, improving consumer self-care, a combination of integration and self-care) in avoiding early mortality and comorbidity among people with serious and persistent mental illness. PSYCH Compare the effectiveness of traditional training of primary care physicians in primary care mental health and co-location systems of primary care and mental health care on outcomes including depression, anxiety, physical symptoms, physical disability, prescription substance use, mental and physical function, satisfaction with the provider, and cost. PSYCH Compare the effectiveness of different treatment strategies (e.g., psychotherapy, antidepressants, combination treatment with case management) for depression after myocardial infarction on medication adherence, cardiovascular events, hospitalization, and death. SKIN Compare the effectiveness of different long-term treatments for acne. 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 Develop- mental Disorders; CAD = Cardiovascular and Peripheral Vascular Disease; CAM = Comple- mentary 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 Sys- tems; 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); ONC = Oncology and Hematology; ORAL = Oral Health; PEDS = Pediatrics; PELC = Palliative and End-of-Life Care; PSYCH = Psychiatric Disorders; RD = Respiratory Disease; RED = Racial and Ethnic Disparities; SKIN = Skin Disorders; TEMC = Trauma, Emergency Medicine, and Critical Care Medicine; WH = Women’s Health.

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 SUMMARY DEFINING COMPARATIvE EFFECTIvENESS RESEARCH An agreed-upon definition of CER is an essential first step for setting priorities and developing a sustainable national CER Program. It informs the public of the focus of this research and its importance in their lives, and it informs investigators of the characteristics of the research to be supported by CER funds. It provides a basis for judging research proposals to perform CER and for evaluating the impact of that research and the success of a national CER Program. In formulating its definition, this committee drew upon definitions by several government agencies and other IOM commit- tees (see Chapter 2): Comparative effectiveness research (CER) is the generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care. The purpose of CER is to assist consumers, clinicians, purchasers, and policy makers to make informed decisions that will im- prove health care at both the individual and population levels. CREATING THE PRIORITy LIST OF CER STuDIES The committee received several broad directives. The legislative lan- guage directed the IOM to solicit the opinions of stakeholders. The IOM’s charge from the contracting agency, AHRQ, stipulated that the committee provide a well-balanced portfolio of research topics for the list of priorities. The committee’s approach to priority setting included the following: • Extensive consultation with and input from stakeholders. The committee widely solicited input through three mechanisms: (1) an invitation to the public and key stakeholders to testify at a 1-day public meeting in Washington, DC, at which the committee heard 54 speakers and received additional written testimony (available on the report’s website at www.iom.edu/cerpriorities); (2) a web-based nomination process through which 1,758 respondents, mostly phy- sicians and representatives of professional organizations, but also many members of the general public nominated a total of 1,268 unique research topics (see questionnaire in Appendix B); and (3) the project’s website, which received emails and letters (see Chapter 3). • Development and consideration of written priority-setting criteria. To guide judgments about each nominated topic, the committee formulated priority-setting criteria to identify high priority target conditions, such as their prevalence, mortality, aggregate costs,

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 INITIAL NATIONAL PRIORITIES FOR CER gaps in knowledge, and small area variation in rates of tests and treatments of top conditions as well as criteria focused on specific research topics (see Chapter 4). • Commitment to developing a broad-based portfolio of high priority topics. The committee’s criteria for creating a balanced portfolio considered four dimensions: (1) clinical category (e.g., cardio- vascular and peripheral vascular disease), (2) study population, (3) categories of interventions, and (4) research methodology (e.g., randomized trial, cohort study) suggested by the nominator (see Chapter 5). • A three-round voting process to narrow the nominated CER topics to a final list of 00. Members voted independently based on the committee-specified criteria and their own values; votes were tallied to rank each nominated topic (see Chapter 4). • Committee discussion of the highest-scored topics. After the second round of voting, the committee had a detailed discussion of the highest-scored topics. The objective of this discussion was to see if the committee agreed on the nominator’s intent and also to reframe some of the nominations to adhere to a common format. The com- mittee also reached consensus on topics to fill or eliminate gaps in the portfolio representation. A total of 26 topics were nominated by the committee. These topics were incorporated into the 129 remaining submitted topics without distinguishing them, providing a total of 155 unique nominated research topics for consideration in the third round of voting. PORTFOLIO DISTRIbuTION OF THE PRIORITy TOPICS The committee’s goal in examining the list of priority research topics as a portfolio was to include balance across the four dimensions previously mentioned. A balanced CER portfolio not only studies those diseases and conditions with the greatest effects on the health of the U.S. population, but also includes rare diseases and conditions that disproportionately and seriously affect subgroups of the population (such as women, minorities, and different groups across the age continuum). The committee sees great value in extending the concept of drug-to-drug comparisons to a variety of interventions including tests to screen for or monitor disease (e.g., imaging for cancer or during normal pregnancy), surgical techniques (e.g., closed vs. open procedures), and therapeutic alternatives (e.g., medical therapy vs. surgery vs. radiotherapy for prostate cancer). Additionally, CER that

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 SUMMARY examines different means of delivering health care was considered to be an important determinant of quality and was incorporated into the options for intervention. Finally, CER priorities should be balanced in the primary method- ologies employed to conduct them: systematic reviews, database research, observational studies, and randomized trials. There are some studies that can be completed in the short term with relatively minimal resources, but other studies will require a longer time frame and a substantial investment of resources. The committee was charged with developing a portfolio of topics that would lead to an appropriate expenditure of the $400 million for CER under the ARRA time frame. Determination of the specific design, questions to be answered by each individual research project, and meth- odology, as defined by the potential researcher, will determine the research costs; however, this task is well beyond the scope of this committee. The committee sought balance in the methodologies proposed by the nomina- tors for all 100 priority topics and determined that they were reasonably well balanced across the four major study methodologies. Systematic review of existing literature is a relatively inexpensive and rapidly performed methodology when compared with other methods. It can identify both information gaps requiring new data generation as well as areas in which sufficient data exist to establish best practices. Research using established databases and registries can be undertaken in a reasonable time frame, inexpensively, and can generate new hypotheses and identify major health care gaps. The generation of new information, either through initiation of new databases or prospective observational studies or through prospective, randomized controlled trials is far more expensive and time consuming, but is often necessary to provide sufficient evidence of what works best and for whom. Thus, the committee balanced the types of study designs so that many studies could be conducted within the time period identified in ARRA. An interactive file of the list of priority topics is available on the report website at www.iom.edu/cerpriorities. Using this file, readers can sort the list of topics by various portfolio characteristics such as research area, study population, or type of intervention. RECOMMENDATIONS FOR A RObuST NATIONAL CER ENTERPRISE Based on stakeholder input and its own deliberations, the committee concluded that the country needs a robust CER infrastructure—referred to throughout as the “CER Program”—to sustain CER well into the future, including carrying out the research recommended in this report and study- ing new topics identified by future priority setting. The committee’s list of

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 INITIAL NATIONAL PRIORITIES FOR CER 100 priority topics responds to the requirements of ARRA to advise the Secretary on how to distribute CER funds from the bill. In addition, the list could be useful beyond the $400 million appropriated to the Secretary by influencing the distribution of funds by NIH, AHRQ, and other agencies that fund CER. The list is not sufficient, however, to ensure the needs of a future in which new interventions and new diseases will mandate new pri- orities for CER. The committee’s examination of previous priority-setting efforts and its study of the nominated research topics conveyed through its questionnaire led it to conclude that CER must be an ongoing process. Health care is dynamic; new diseases and health needs can arise suddenly and other health problems might become insignificant when a treatment is found. As new CER produces new evidence and closes gaps in evidence, CER might need to take new directions. A continuous process is necessary to update funding priorities as conditions change and the impact of previ- ous CER becomes evident (see Chapter 4 for discussion of Recommenda- tions 1 through 4). Recommendation 1: Prioritization of CER topics should be a sustained and continuous process, recognizing the dynamic state of disease, inter- ventions, and public concern. The committee acknowledges the critical role that the general public and other stakeholders played in this current report and their potential to enhance CER in the future. CER generates results that bear directly on deci- sions in which individual patients play an active role. Active involvement of consumers, patients, and caregivers is essential to identifying CER topics of real concern to them as well as for suggesting criteria for the prioritization process that reflect public goals and values. Recommendation 2: Public (including consumers, patients, and care- givers) participation in the priority-setting process is imperative to provide transparency in the process and input to delineating research questions. The committee noted that more complete background information about the suggested research topics would have substantially enhanced its prioritization process. A national CER enterprise should, on an ongoing basis, collate national data concerning the significance of diseases and con- ditions as well as information about current research gaps and redundancies related to the specific research topics under consideration. The committee found that the descriptions of research topics were often difficult to under- stand; an opportunity for a priority-setting body to interrogate CER topic nominators would help to clarify the nominator’s intent.

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 SUMMARY Recommendation 3: Consideration of CER topics requires the devel- opment of robust, consistent topic briefs providing background infor- mation, current practice, and research status of the condition and its interventions. The committee concluded that a high level of transparency is essential for setting priorities for expending public funds on research from which the public expects so much. Given the magnitude of public investment in CER, a rolling evaluation of the selection and prioritization processes, as well as the return on investment of prior CER research by application throughout the health system should be incorporated in the prioritization process to ensure quality improvement. Recommendation 4: Regular reporting of the activities and recommen- dations of the prioritizing body is necessary to evaluate the portfolio’s distribution, its impact for discovery, and its translation into clinical care in order to provide a process for continuous quality improvement. The committee’s work, including stakeholder input, revealed the scope of research infrastructure needed to support CER in its goal of improving health care decisions and their implementation. The committee does not attempt to fill in all the details, but it concludes that the country must have a federal organizational infrastructure with appropriate responsibility and authority to coordinate the prioritization process, support the development of necessary databases and registries, fund the training of needed research- ers, conduct the research, and support a vigorous translational effort to help bring research findings into everyday clinical practice. Without federal support for an infrastructure to coordinate the national CER effort, all the CER that the committee identified as high priority is unlikely to occur (see Chapter 6 for a discussion of infrastructure issues). Objectivity will be central to the public’s trust and confidence in the integrity of the CER Program. CER is as vulnerable to bias and conflict of interest as any other area of medical research. A 2009 IOM report, Con- flict of Interest in Medical Research, Education, and Practice, recommends principles to inform the design of policies to identify, limit, and manage conflicts of interest in health care research. The committee urges that the CER Program be constituted and managed in accordance with the recom- mendations of this report. Recommendation 5: The HHS Secretary should establish a mechanism— such as a coordinating advisory body—with the mandate to strategize, organize, monitor, evaluate, and report on the implementation and impact of the CER Program.

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 INITIAL NATIONAL PRIORITIES FOR CER A central focus on the patient is fundamental to high-quality health care. To meet the requirement of patient-centeredness, respect for individual patients’ unique needs, beliefs, and values must drive the development of the field of CER and the application of its findings to patient care. Con- sumers, patients, and caregivers have a key role to play in informing and framing CER. They typically have different perspectives from researchers, and there is strong evidence that many consumers—but not all—want to be involved in decision making about their care. Involving them in CER will help to keep the research relevant and applicable to real-world set- tings. Also, if consumers, patients, and caregivers are engaged and informed about CER activities, they are more likely to trust the research findings and insist that their own care take account of the results. Recommendation 6: The CER Program should fully involve consum- ers, patients, and caregivers in key aspects of CER, including strategic planning, priority setting, research proposal development, peer review, and dissemination. • The CER Program should develop strategies to reach out to, engage, support, educate, and, as necessary, prepare consumers, patients, and caregivers for leadership roles in these activities. • The CER Program should also encourage broad participation in CER in order to create a representative evidence base that could help identify health disparities and inform decisions by patients in special population groups. CER comprises a broad spectrum of established and emerging research methods including clinical trials, observational studies, and systematic reviews of existing evidence. There is a significant need for better research methods. Current study designs—experimental and nonexperimental—must be refined to ensure scientific rigor. Clinical trials will always be essential to CER, but more efficient, larger, simpler, and pragmatic designs are needed. In systematic reviews, for example, research is needed on how to identify and use evidence from observational studies on intervention effectiveness, and also on how to assess a heterogeneous body of evidence. New analytic tech- niques are needed to evaluate the effects of bias due to confounding when assessing comparative effectiveness using large observational datasets. Recommendation 7: The CER Program should devote sufficient re- sources to research and innovation in the methods of CER, including the development of methodological guidance for CER study design such as the appropriate use of observational data and more informa- tive, practical, and efficient clinical trials.

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 SUMMARY CER should also draw from analyses of existing data, such as that held by payers, health care delivery systems, and electronic health records. How- ever, if the CER enterprise is to harness the rich potential of these data, it must protect the privacy and maintain the security of patient data, develop efficient means for linking data from multiple databases, and engage hold- ers of large datasets such as health insurers, health care delivery systems, and health care providers. Recommendation 8: The CER Program should help to develop large- scale, clinical and administrative data networks to facilitate better use of data and more efficient ways to collect new data to inform CER. • The CER Program should ensure that CER researchers and institu- tions consistently adhere to best practices to protect privacy and maintain security. • The CER Program should support the development of methodolo- gies for linking patient-level data from multiple sources. • The CER Program should encourage data holders to participate in CER and provide incentives for cooperation and maintaining data quality. ARRA’s infusion of federal funds into CER will stress the limited ca- pacity of the current CER workforce. AHRQ’s CER appropriation alone increased tenfold. Whether the current research workforce can meet the human resource demands of the $1.1 billion ARRA appropriation for CER is uncertain. A significant increase in CER activity will certainly cre- ate a substantial need for experts in biostatistics, epidemiology, systematic reviews (including meta-analysis), clinical trials (including head-to-head effectiveness trials), statistical modeling, observational analytic methods, use of analysis of large datasets, cost-effectiveness analysis, clinical out- comes research, and communication of research findings. The methods of CER must advance, which will require training and career support for methodologists. Recommendation 9: The CER Program should develop and support the workforce for CER to ensure the nation’s capacity to carry out the CER mission. Important next steps include the following: • Development of a strategic plan for research workforce development. • Long-term, sufficient funding for early career development including expanding grants for graduate and postgraduate training opportuni- ties in comparative effectiveness methods as well as career develop- ment grants and mid-career merit awards.

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0 INITIAL NATIONAL PRIORITIES FOR CER The substantial geographic variability in health care delivery suggests that physicians differ in what they consider to be “best practice.” By discov- ering what works best, for whom, and under what circumstances, CER has the potential to narrow the spectrum of what health professionals consider to be best practice. Health care professionals and patients should be able to use CER results to make informed decisions based on the best available evi- dence, the patients’ preferences, and the patient’s unique characteristics. However, an ambitious research enterprise alone will not improve health care in the United States without significant attention to high fidel- ity translation of knowledge into practice. At present, the translation of research findings into practice is slow and incomplete. Many barriers exist: perverse reimbursement incentives, physician perceptions about patients’ expectations, and patients’ concerns about denials of care or their reluc- tance to question clinicians. The CER Program should require researchers to publish all federally funded CER studies and make the research avail- able to the public. Moreover, research into knowledge translation must be a high priority. Recommendation 10: The CER Program should promote rapid adop- tion of CER findings and conduct research to identify the most effective strategies for disseminating new and existing CER findings to health care professionals, consumers, patients, and caregivers and for helping them to implement these results in daily clinical practice.