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3 Obtaining Input to Identify National Priorities for Comparative Effectiveness Research Abstract: Stakeholder input was one of the express requirements of the comparative effectiveness research priority study that Congress requested from the Institute of Medicine (IOM) in the American Recovery and Reinvestment Act of 2009. The committee concluded that such input should be invited and analyzed from direct communications to the IOM from an in-person stakeholder meeting before the committee and from a web-based questionnaire. Policy recommendations, general comments, and opinions were provided in direct communication before, during, and after the public meeting from the biomedical and health care communities and patients and families. More than 2,600 nominations representing a diversity of research topics, respondents, and perspectives from the public and private for-profit and not-for-profit sectors were submitted to the web-based questionnaire. INTRODUCTION In response to the directive of the American Recovery and Reinvest- ment Act (ARRA) of 2009 (P.L. 111-5) to consider input from stakehold- ers, the committee focused on three mechanisms for obtaining input on comparative effectiveness research (CER) from as wide an array as possible of stakeholders including patients, families, and consumers. This chapter â American Recovery and Reinvestment Act of 2009, P.L. 111-5, 111th Congress, 1st ses- sion (February 17, 2009). 61
62 INITIAL NATIONAL PRIORITIES FOR CER describes the process of soliciting the inputs that informed the committee in preparing its report. INVITATIONS TO PROVIDE INPUT More than 20,000 email announcements describing the Institute of Medicine (IOM) study and inviting stakeholder input were sent out the first week of March 2009. These announcements were directed to general lists maintained by the IOM, composed of IOM members and other individuals who signed up for the listserv because of an interest in the IOM and its work. The lists include members of the media, policy makers, academics, re- searchers, health care industry, physicians and other health care providers, students, former Robert Wood Johnson Foundation Health Policy fellows, and others in the public and private sectors interested in health policy, as well as those on listservs to congressional staff and congressional agency staff. Announcements were also sent specifically to seven categories of stakeholders (Table 3-1) with broad constituencies; they were requested to forward the announcement to their memberships. The committee concluded that these organizations, and their memberships and constituencies, were relevant to CER and could initiate further dissemination of the announce- ments to similar individuals and groups thus providing an opportunity for increased input to the committee. Invitations provided three distinct avenues for submitting advice on national priorities for CER to the committee: 1. ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ Direct correspondence with the IOM committee 2. ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ Oral and written presentations at an open stakeholdersâ meeting scheduled at the National Academy of Sciences Building in Wash- ington, DC 3. ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ï¿½ Submission of specific CER topics, as well as general comments on the process of conducting CER via a web-based questionnaire The committeeâs goal was to receive the most extensive advice and recommendations possible for national CER priorities from the widest possible array of stakeholders within the time and resources available. In the aggregate, useful advice and a list of national priorities emerged from these three steps that related well to selection criteria and 32 research areas as described below. COMMUNICATIONS DIRECTLY TO THE COMMITTEE The committee received approximately 90 emails and letters from a wide variety of stakeholders that ranged from pharmaceutical manufacturers to health profession associations, patient and consumer organizations, health
OBTAINING INPUT TO IDENTIFY NATIONAL PRIORITIES 63 TABLE 3-1â Solicited Stakeholder Groups (including, but not limited to the examples shown) Categories Stakeholder Groups Consumers/Patient â¢ AARP Advocacy Groups â¢ Center for Advancement of Health â¢ Consumers Union â¢ National Health Council â¢ National Minority Quality Forum Federal Government â¢ Agency for Healthcare Research and Quality Agencies â¢ Centers for Disease Control and Prevention â¢ Centers for Medicare & Medicaid Services â¢ Department of Veterans Affairs â¢ Food and Drug Administration â¢ National Institutes of Health Health Care Providers and â¢ American Academy of Family Physicians Researchers â¢ American Academy of Pediatrics â¢ American College of Physicians â¢ American Medical Association â¢ American Nurses Association â¢ American Psychological Association â¢ National Medical Association Insurers â¢ Americaâs Health Insurance Plans â¢ Blue Cross and Blue Shield Association â¢ CIGNA Integrated Health Systems â¢ Geisinger â¢ HealthPartners â¢ Kaiser Permanente Manufacturers â¢ Advanced Medical Technology Association (including drugs, devices, and â¢ Biotechnology Industry Organization biotechnology) â¢ Pharmaceutical Research and Manufacturers of America State Government Agencies â¢ Association of State and Territorial Health Officials â¢ National Governors Association
64 INITIAL NATIONAL PRIORITIES FOR CER services researchers, health plans, complementary and alternative medicine providers, patient advocates, and individual patients and consumers. Many of these made recommendations to include specific topics nominated as research priorities, as well as suggestions about the general process. A sum- mary of the general stakeholder recommendations follows: â¢ Transparency. Many stakeholders recommended using a systematic and open process of setting priorities that would include patients and consumers, and would avoid conflicts of interest. â¢ Research Design. Some stakeholders favored encouraging new types of studies, adopting certain techniques to minimize research bias, incorporating quality measures and patient preferences in effectiveness studies, and focusing on real-world clinical situa- tions rather than ideal conditions. Related suggestions called for improving existing databases, making databases more accessible to researchers, protecting patient privacy, and investing in health information technology designed to produce a robust, scalable, and open architecture capable of providing real-time data. They also suggested that CER should take into account special populations defined by such factors as race and ethnicity, gender, age, and so- cioeconomic status. â¢ Translation and Dissemination. Many stakeholders addressed issues of disseminating CER findings and converting them into changes in health care practice. These suggestions included provid- ing feedback to physicians, improving decision support for clini- cians, encouraging physicians to use best practices and clinical guidelines, conducting research on medical and surgical devices, enhancing patient adherence to regimens, developing user-friendly guides, testing alternative patient decision-making tools, and al- lowing public comment periods for comparative effectiveness study reports. Long-term issues that were addressed included revisiting re- search results when new information becomes available, expanding training programs for CER, and partnerships among professions. â¢ Economics. A number of stakeholders suggested expanding âcov- erage with evidence developmentâ (i.e., Medicare reimbursement is conditioned on reporting results of use) to other payers in both the public and private sectors. They also suggested including con- sideration of the cost of an intervention as a secondary factor in evaluation. Some correspondents urged that incentives for innova- tion be preserved.
OBTAINING INPUT TO IDENTIFY NATIONAL PRIORITIES 65 â¢ Advocacy. Some stakeholders suggested advocating for specific groups, providers, conditions, or organizations; the committee re- viewed and acted on these suggestions as appropriate. PRESENTATIONS AT AN OPEN MEETING OF STAKEHOLDERS An open stakeholders meeting was held March 20, 2009 in the audito- rium of the National Academy of Sciences Building on Constitution Avenue in Washington, DC. Fifty-four speakers made 3-minute presentations (or 5- minute presentations if the individual represented a large membership orga- nization) to the committee and a large public audience with approximately 25 percent of the day reserved for the committee to address questions to the presenters. Box 3-1 displays the list of speaker organizations and a full agenda from the meeting is available in Appendix A. Written statements from all 54 speakers were made publicly available on the committee website and are provided as an electronic appendix at www.iom.edu/cerpriorities. Word limits were not imposed on these written statements. Virtually all of the presenters addressed issues of public policy or methodology involving CER, rather than proposing specific research top- ics. Many presenters expressed support for CER in principle, while none expressed direct opposition. The representative from Americaâs Health In- surance Plans asserted that some health care interventions are used without evidence or without recent reevaluation, and there is a need to know first, what works, and second, what works best. Many presenters, such as the representatives from the Biotechnology Industry Organization and several physician societies, stated that evidence- based medicine should guide CER, and that CER should be accurate and rigorous. They emphasized that CER needs to be grounded in âreal-worldâ conditions and that the public will require evidence of this as investments in CER increase. The representative from the Association of American Medi- cal Colleges urged the committee to recommend investments in training researchers, in data resources, and in other CER infrastructure. Some presenters, such as the representatives from the National Medical Association and the National Minority Quality Forum, expressed concern that CER might be used to generalize approaches to therapy, in a so-called one-size-fits-all approach to health care. Others, such as the representa- tive from the National Health Council, strongly advocated for a patient- â Federal agencies listed in Table 3-1 did not make presentations before the committee. In- stead, these agencies participated in the federal response to the CER mandate of ARRA under the aegis of the Federal Coordinating Council on Comparative Effectiveness Research. This council heard 3-minute presentations and/or received a written statement from stakeholders over 3 hours on April 14, 2009, at a âpublic listening sessionâ held in Washington, DC, and on May 13, 2009, in Chicago, IL.
66 INITIAL NATIONAL PRIORITIES FOR CER BOX 3-1 Organizations Represented at the Stakeholder Meeting â¢ A Certified Nurse Midwife â¢ Consumers Union â¢ Advanced Medical Technology â¢ Developing Families Center Association â¢ Duke University Medical Center â¢ American Academy of Family â¢ eHealth Initiative Physicians â¢ Focus on Therapeutic â¢ American Academy of Outcomes, Inc. Pediatrics â¢ Friends of Cancer Research â¢ American Association for â¢ Frontier School of Midwifery & Dental Research Family Nursing â¢ American Association of â¢ Health Care Consultancy Neurological Surgeons â¢ HealthPartners Research â¢ American College of Cardiology Foundation â¢ American College of Clinical â¢ International Society for Pharmacy Pharmacoeconomics & â¢ American College of Occupation Outcomes Research and Environmental Medicine â¢ The Lewin Group â¢ American College of Surgeons â¢ National Alliance for Hispanic â¢ American Heart Association Health â¢ American Medical Association â¢ National Alliance on Mental â¢ American Nurses Association Illness â¢ American Psychiatric â¢ National Health Council Association â¢ National Medical Association â¢ American Psychological â¢ National Minority Quality Forum Association â¢ National Pharmaceutical â¢ American Society of Clinical Council Oncology â¢ Network for Regional â¢ Americaâs Health Insurance Healthcare Improvement Plans â¢ Oregon Health and Science â¢ Association of American University and Portland VA Medical Colleges Medical Center â¢ Association of Clinical â¢ Parkinson Pipeline Project Research Organizations â¢ Personalized Medicine â¢ Association of Schools of Coalition Public Health â¢ Pharmaceutical Research and â¢ Biotechnology Industry Manufacturers of America Organization â¢ Society for Cardiovascular â¢ Blue Cross and Blue Shield Angiography and Interventions Association â¢ The Society of Thoracic â¢ California Department of Public Surgeons Health â¢ United BioSource Corporation â¢ Center for Advancement of â¢ United States Pharmacopeia Health â¢ University of Iowa â¢ Center for Science in the Public â¢ Washington State Health Care Interest Authority â¢ CIGNA
OBTAINING INPUT TO IDENTIFY NATIONAL PRIORITIES 67 centered approach to health care delivery and CER. Still, others, such as the presenter from the Lewin Group, offered a set of principles to guide research, which included transparency, public input, a broad scope, careful definitions of comparisons, support for personalized medicine, and evolu- tion of innovation supported by explicit ground rules for review. Speakers identified the inclusion of cost considerations in the outcomes of CER as controversial. At least nine speakers expressly supported the in- clusion of costs in the comparison of health interventions, and many more advocated establishing the relative value of different interventions, services, and care models by comparing costs to clinical benefits for two or more alternatives. At least four others, however, recommended against the use of cost comparisons for fear that the availability of drugs on formularies or coverage decisions would be unduly influenced. One speaker suggested that cost be taken into account only when the alternative interventions are clinically equivalent. Another advocated a focus on enhancing value for patients rather than minimizing costs. Many groups spoke in favor of personalized or individualized care, taking into consideration the varying concerns and clinical and genetic diversity of patients. This concept was expressed in different ways, but it reflected an overall concern that general research results should not be applied in a way that overlooks the specific needs and preferences of indi- vidual patients. Strategic targeting of CER frequently arose as a topic of discussion. Key topics for investigation identified by the speakers included diabetes, coronary heart disease, chronic obstructive pulmonary disease, depression, common spinal disorders, childhood asthma, obesity, early brain devel- opment, family services and midwifery, oral health, minimally invasive surgery, chronic disease in general, and complementary and alternative medicine, among others. Specific modalities of health care delivery that were proposed included behavioral health, medical homes, multi-profes- sional teams, and expanded roles for non-physician health professionals. Some also suggested starting with research that was âshovel-readyââthat is, likely to be accomplished and yield results quickly. There was general agreement that areas to be targeted included high-prevalence conditions, services with high variation in use, conditions with major public health consequences such as those involving health disparities, high-cost condi- tions, conditions not covered by existing clinical guidelines, and research directed at disease prevention. In addition, there was widespread agreement that effectiveness must be assessed in racial and ethnic minorities to help remedy health disparities. A number of suggestions regarding research methods came from groups such as the National Alliance on Mental Illness, the American Heart As- sociation, Friends of Cancer Research, the Association of Clinical Research
68 INITIAL NATIONAL PRIORITIES FOR CER Organizations, and the Washington State Health Care Authority, among others. They recommended improvements in defining the populations used in randomized clinical trials, avoiding treatment and publication bias by considering completed but unpublished studies, and collecting and storing biospecimens. They also suggested the establishment of a process for timely reconsideration of CER results. Many presenters discussed sources of information, databases, and other aspects of CER infrastructure. Some professional groups reported joining together to help develop CER standards, registries, and procedures. Some organizations, such as the Advanced Medical Technology Association and Blue Cross and Blue Shield Association, indicated that they might be willing to support projects materially. The American College of Surgeons and other professional groups said their members would be supportive, participate in studies, and likely to incorporate CER results into their practices. Stakeholders at the meeting generally concluded that there was a need for full transparency in the prioritization process. This encompasses the ongoing participation of stakeholders, the avoidance or strict management of potential conflicts of interest, and the establishment of rigorous scientific standards and methods. INPUT FROM A WEB-BASED QUESTIONNAIRE At a very early stage, the committee concluded that substantial and meaningful stakeholder input and specific recommendations for CER pri- orities to the committee required a web-based questionnaire designed to elicit such recommendations. As described earlier, announcement notices of this questionnaire were circulated through a wide distribution of emails to about 20,000 individuals and organizations, and through these organiza- tions to their memberships. In addition, notice was disseminated through the IOM website (www.iom.edu/cerpriorities). The questionnaire was open for 3 weeks, from March 6 to March 27, 2009, which included 1 week following the open stakeholders meeting. The questionnaire is available in Appendix B. Priority Topic Nominations There were 1,758 respondents to the questionnaire and 2,606 nomina- tions for CER topics. Initial review showed many duplicated entries and â The questionnaire was not requested by the federal sponsor nor was there time for ap- proval by the Office of Management and Budget. This situation did not allow payment for the questionnaire and associated analysis from federal funds, but because the committee concluded it was essential, the IOM took the unusual step of supporting the costs associated with the questionnaire from National Academies funds.
OBTAINING INPUT TO IDENTIFY NATIONAL PRIORITIES 69 Web-based questionnaire open to public March 6-27, 2009 Received 2,606 nominated topics for CER from 1,758 respondents Initial screen of 2,606 nominated topics by clinical review subcommittee (April 2-6) 335 1,268 1,003 Excluded due to Entered into Excluded due nonresponsiveness round 1 of voting to verbatim duplication FIGURE 3-1â Stakeholder response to web-based questionnaire. nonresponsive submissions. The initial entries were screened by staff to identify unique submissions and were confirmed by two committee members in independent review. Figure 3-1 shows the review and screening process of the questionnaire submissions that resulted in the 1,268 topics that entered the first round of voting. Table 3-2 displays the distribution of respon- dents by self-identified stakeholder category. While approximately 75 per- Figure 3-1 cent of respondents identified themselves as either providers or researchers R01311 vector, editable (which includes the following categories: Health Care Provider, Researcher, GovernmentâResearch, GovernmentâPrograms, and Health Plan/Insurance Carrier), there was also representation from other categories in the public and private for-profit and not-for-profit sectors, and nearly 10 percent of respondents identified themselves as patients, families, or consumers (which includes the following categories: Patient/Family, Public/Consumer, and Em- ployer). More than 300 respondents self-identified as members of more than â Nonresponsive submissions were either nonsense answers such as entering âaaaaâ so that the respondent could browse through the questionnaire without inserting answers, were not complete responses, or were just general comments to the committee rather than topics for CER.
70 INITIAL NATIONAL PRIORITIES FOR CER TABLE 3-2â Respondents to the IOM Questionnaire by Stakeholder Category Number of Self-Identified Stakeholder Categories Responders* Health Care Provider 797 Researcher 416 Professional Association 229 Other 212 Nonprofit/Policy Institute 95 Patient/Family (including family caregiver) 77 Public/Consumer 76 Medical Administrator 40 GovernmentâResearch 39 Employer 26 GovernmentâPrograms (e.g., Medicare, Medicaid) 26 Manufacturer (Device) 17 Health Plan/Insurance Carrier 12 Manufacturer (Drug or Biologic) 11 Total 2,073 *315 respondents self-identified with more than one category. one category. Although respondents were primarily from the biomedical and health care communities, the committee concluded that a fair degree of diversity of perspective was represented within those communities. The questionnaire asked each respondent to submit up to three nomi- nations for priority research topics. Respondents were further asked to support each nominated topic with specific information, including data to justify the importance of the proposed research, assignment of the topic to a single primary research area, identification of appropriate study popu- lations, specification of interventions being compared, and the proposed study methodology. In an effort to be as broad and inclusive as possible, the committee identified the primary research areas from the 17th edition of Harrisonâs Principles of Internal Medicine (Fauci et al., 2008). In addi- tion, the committee added the following research areas to the questionnaire: birth and developmental disorders, functional limitations and disabilities, and pediatrics. Stakeholders pointed out during the public meeting on March 20, 2009, that oral health had been omitted as a distinct category. Therefore, the committee reassigned those nominated topics clearly belong- ing in this category. The complete listing of named research area categories totaled 32, with an additional category for âother.â The breakdown of the questionnaire nominations by research area, study population, proposed intervention, and study methodology appear in Tables 3-3 through 3-6. The tables display results for the 1,268 topics that emerged after consolidating
OBTAINING INPUT TO IDENTIFY NATIONAL PRIORITIES 71 TABLE 3-3 Comparative Effectiveness Research Priorities Submitted by Primary Area of Study Category Number of SubmissionsÂ Alcoholism, Drug Dependency, and Overdose 31 Birth and Developmental Disorders 73 Cardiovascular and Peripheral Vascular Disease 50 Complementary and Alternative Medicine 19 Endocrinology and Metabolism Disorders 33 Eyes, Ears, Nose, and Throat Disorders 50 Functional Limitations and Disabilities 55 Gastrointestinal System Disorders 15 Genetics and Disease 11 Geriatrics 35 Health Care Delivery System* 156 Immune System, Connective Tissue, and Joint Disorders 12 Infectious Diseases 37 Kidney and Urinary Tract Disorders 23 Liver and Biliary Tract Disorders 0 Medical Aspects of Bioterrorism 2 Musculoskeletal Disorders 36 Neurologic Disorders 81 Nutrition (including obesity) 47 Oncology and Hematology 57 Oral Health 15 Other 3 Palliative and End-of-Life Care 20 Pancreatic Disorders 2 Pediatrics 89 Psychiatric Disorders 127 Racial and Ethnic Disparities 19 Regenerative Medicine 2 Respiratory Disease 29 Sexual Function and Reproductive Disorders 4 Skin Disorders 19 Trauma, Emergency Medicine, and Critical Care Medicine 79 Womenâs Health 37 Total 1,268 NOTE: Secondary and comorbid conditions were also provided but not included in this table. *Although this category was described as âSafety and Quality of Health Careâ in the web- based questionnaire, the category was relabeled by the committee as âHealth Care Delivery Systemâ to be more accurate.
72 INITIAL NATIONAL PRIORITIES FOR CER TABLE 3-4â Comparative Effectiveness Research Priorities by Proposed Population to be Studied Population Count Adults (excluding elderly) 120 Adults (including elderly) 381 Children/Adolescents Only 448 Elderly Only 193 Ethnic Subpopulations Only 72 Long-Term Care 124 Women 299 Men 242 Population at Large (general population) 336 Rare Diseases 25 Special Populations (e.g., pregnant women, low income, patients with 333 disabilities) Total 2,573 NOTE: The total exceeds the total number of nominations because respondents were allowed to select multiple populations. TABLE 3-5 Comparative Effectiveness Research Priorities by Proposed Intervention Comparators Count Alternative Treatment 171 Behavioral Treatment 421 Devices 114 Pharmacological Treatment 306 Prevention 452 Procedures (including surgery) 136 Provider-Patient Relationships 304 Standard of Care 458 Systems of Care 508 Testing, Monitoring, and Evaluation 398 Treatment Pathways 305 Total 3,573 NOTE: The total exceeds the total number of nominations because respondents were allowed to select multiple interventions.
OBTAINING INPUT TO IDENTIFY NATIONAL PRIORITIES 73 TABLE 3-6 Comparative Effectiveness Research Priorities by Proposed Study Methodology Methodology Count Database Review 295 Prospective Observational Study 593 Randomized Clinical Trial 676 Systematic Review 253 Total 1,817 NOTE: The total exceeds the total number of nominations because respondents were allowed to select multiple methodologies. the 2,606 nominated topics to eliminate verbatim duplicates and nonre- sponsive submissions, described in greater detail in Chapter 4. Public Responses on Their Priority-Setting Process In addition to making specific recommendations for comparative ef- fectiveness priorities and providing supporting information, many of the questionnaire respondents provided information on how they developed their topic nominations, what were their principal priority-setting criteria, and what new or enhanced infrastructure would be needed to sustain a CER enterprise. Regarding the development of CER topics, the largest number of respondents indicated that they nominated topics based on professional experience, both clinical and classroom, and often of many yearsâ duration. Many others developed topics based on literature reviews; conferences attended (such as a National Institutes of Health [NIH] state- of-the-science review); suggestions of specific organizations, such as NIH or the Centers for Disease Control and Prevention (CDC); or their own professional associations such as the American Medical Association, the American Diabetes Association, or Blue Cross and Blue Shield Association. Others reported consulting with colleagues and stakeholders, including a hospital, health system, or consumer group. A small number reported using personal experience to make their nominations, such as being the mother of a deaf child. The most common priority-setting criteria identified by the respondents can be classified into three broad categories: patient need, quality of care, and cost and reimbursement issues. Patient need was ranked as the top criterion; 23 percent of respondents ranked it in first place, and 14 percent ranked it in second place. Patient need referred primarily to disease burden, including prevalence, morbidity, mortality, and family and social impact, as
74 INITIAL NATIONAL PRIORITIES FOR CER well as risk factors such as obesity and substance abuse. Conditions sup- porting the use of this criterion included speech or hearing problems, autism and traumatic brain injury, chronic diseases including HIV, cancer, asthma, cardiovascular disease, birth outcomes, smoking and alcohol abuse, mental health, and dental disease. The remaining specific criteriaâdecreasing vari- ability in care, the potential to act on the information, and cost, all received about 13 percent of the support from respondents. With respect to quality of care-related criteria, many self-identified cli- nicians said they sought better information for making clinical decisions in order to deliver the best or evidence-based treatment, to reduce treatment variation, and to promote quality of care for their patients, including safety and improved outcomes. Clinicians sought better aids in making clinical decisions; they expressed a need for help in delivering the best care when there were many confusing alternatives. Cost and reimbursement issues may reflect that research would lead to cost management, better resource use, a decrease in societal costs, and elimination of waste. Respondents stated that if research could lead to deploying health care resources more effectively, costs would decrease. Oth- ers thought CER might persuade payers to support or improve reimburse- ment for particular services, such as integrative care, complementary and alternative medicine, improved doctor-patient communication, caregiver education, specific diagnostic services, and strategies to improve adherence to treatment regimens, among others. Other comments justifying priority nominations included closing in- formation gaps, countering misinformation, addressing specific areas of research deemed underfunded, assessing new service delivery models, im- proving public interest, minimizing controversy, reducing disproportionate impact on subpopulations, focusing on research that could deliver quick results, that is low cost, and that is feasible to implement, focusing on psy- chosocial and educational factors, including family dysfunction that affects health outcomes, and developing new research methods. In addition, 650 respondents answered the question about enhancing CER infrastructure. Some called for either sufficient, high, or permanent funding for CER, commenting that a public and business case needs to be made to overcome the strong opposition to CER. Important attributes were listed, such as a public-private partnership, methodological rigor, a focus on outcomes, facilitating innovation in interventions and approaches, and broad stakeholder involvement. Some respondents noted that CER is re- lated to health reform or Food and Drug Administration reform, or changes in state licensure of health professions. While some respondents recom- mended privatizing or decentralizing CER or using existing resources, oth- ers suggested that a CER program could be located within the Agency for Healthcare Research and Quality, NIH, or the CDC. It was suggested that
OBTAINING INPUT TO IDENTIFY NATIONAL PRIORITIES 75 funding be available to individually initiated, multicenter, or non-university- based investigators. Simplification of participation in randomized controlled clinical trials, or in institutional review board processes, and prohibition of conflicts of interest were also recommended. Specific proposals were made for openness, including a national conference, a national committee, local research efforts, and scientific input. Identified needs included registries, longitudinal studies, data availability and access provisions, development of clinical guidelines, examination of subpopulations, reduction in disparities, ways of widely disseminating results, and public and professional education and communication of findings. The committee was impressed by the value, breadth, and common themes that characterized these inputs from stakeholders and the public. While the committee concluded that many of the original topics nominated by respondents to the web-based questionnaire were thoughtful and wor- thy, as described in Chapter 4, some topics were edited to be broader and more inclusive of multiple patient populations. In addition, the committee nominated several topics to fill gaps in the portfolio. Through the selection process described in Chapter 4, the topics nominated by the stakeholders, the public, and the committee members were reduced to the final list of 100 national priority topics for CER listed in Chapter 5. The publically nominated topics served as the basis for the majority (82 percent) of the list, and the remaining 18 percent were nominated by the committee. These responses also allayed committee concerns that the 3-week window for the questionnaire that was necessary to meet the short turnaround time for this report may have unduly limited public input. Furthermore, answers to ques- tions on the questionnaire, and input from letters, e-mails, and stakeholder presentations, informed the committee in several other waysâfor example, the suggestions on infrastructure were considered in drafting Chapter 6. Learning from this experience, the committee concluded that an ongo- ing process for citizens to express themselves and provide priorities for CER would be worthwhile. See Chapter 4 for further discussion of how this input could be collected. This process might also serve an educational role by informing the public that CER is aimed to improve the quality of clinical care delivered to patients not a sub rosa scheme to ration it. Reference Fauci, A. S., E. Braunwald, D. L. Kasper, S. L. Hauser, D. L. Longo, J. L. Jameson, and J. Loscalzo. 2008. Harrisonâs principles of internal medicine, 17th edition Place Published: McGraw-Hill Companies. http://www.accessmedicine.com/resourceTOC.aspx?resource ID=4 (accessed May 21, 2009).