Learning What Works Infrastructure Required for Comparative Effectiveness Research: Workshop Summary (2011) / Chapter Skim
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To improve the effectiveness and value of the care delivered, the nation needs to build its capacity for ongoing study and monitoring of the relative effectiveness of clinical interventions and care processes through expanded trials and studies, systematic reviews, innovative research strategies, and clinical registries, as well as improving its ability to apply what is learned from such study through the translation and provision of information and decision support. Several recent initiatives have proposed the development of an entity to support expanded study of the comparative effectiveness of interventions.
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Box S-1 describes the issues that motivated the meeting's discussions: the substantial and growing interest in activities and approaches related to CER; the lack of coordination of key activities, such as the selection and design of studies, synthesis of existing evidence, methods innovation, and translation and dissemination of CER information; shortfalls and widening gaps in the workforce needed in all areas of CER; the opportunities presented by the recent calls for expanded resources for work on the comparative effectiveness of clinical interventions; the growing appreciation of the infrastructure needed to support this work; and the need for a trusted, common venue to identify and characterize the need categories, begin to estimate the shortfalls, consider approaches to addressing the shortfalls, and identify priority next steps. BOX S-1 Issues Motivating the Discussion 1.
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The workshop gathered leading practitioners in health policy, technology assessment, health services research, health economics, information technology (IT) , and health professions education and training to explore, through invited presentations, the current and future capacity needed to generate new knowledge and evidence about what works best, including skills and workforce, data linkage and improvement, study coordination and result dissemination, and research methods innovation.
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• R edesigning the Clinical Effectiveness Research Paradigm: Innova tion and Practice-Based Approaches (December 12–13, 2007)
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This publication summarizes the proceedings of the seventh workshop in the Learning Health System series, which focused on the infrastructure needs -- e.g., methods, coordination capacities, data resources and linkages, workforce -- for developing an expanded and efficient national capacity for CER. A synopsis of the key points from each of the sessions is included in this chapter, with more detailed information on session presentations and discussions found in the chapters that follow.
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Wilensky The Cost and Volume of 2 Comparative Effectiveness Research Erin Holve and Patricia Pitman Intervention Studies That Need to Be Conducted Douglas B Kamerow Clinical Data Sets That Need to Be Mined Jesse A
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Coordination and Technical Assistance That Need to Be Supported Jean R Slutsky Electronic Health Records: Needs, 3 Status, and Costs for U.S.
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Bruce Ferguson, Jr., and Ansar Hassan Practical Challenges and Infrastructure Priorities for Comparative Effectiveness Research Daniel E Ford Transforming Health Professions Education Benjamin K
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Appendix B includes evidence summaries of research questions identified and other materials relevant to discussion in a paper in Chapter 2. Appendixes C and D present the recommendations of two groups for priority studies in CER: Initial National Priorities for Comparative Effectiveness Research, an Institute of Medicine report; and the Federal Coordinating Council for Comparative Effectiveness Research Report to the President and Congress.
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• fficiency and effectiveness compel globalizing evidence and local E izing decisions. decisions for the conduct of CER, systematic reviews, and guideline development; and the need to ensure the consistent translation of developed information.
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-- a first-order priority for effectiveness research is the establishment of infrastructure for a more dynamic, real-time approach to learning. Leveraging new tools, such as health information technology (IT)
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D eveloping and applying tools that foster real-time data analysis • is an important element. The scope and scale of evidence needs suggests that innovation is needed across the range of research methods, from making clinical trials faster and less expensive to moving beyond randomized trials to better address practical cir cumstances, using registries, observational databases, and other emerging data resources.
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This brought clearly into play the need to ensure that, where possible, common work to assess an intervention's clinical effectiveness -- or collective work to assess the body of evidence -- be collaborative and well coordinated across boundaries, while also being mindful that different cultural and policy environments may lead to differ ent decisions at the local level. Key Factors and Needs Workshop speakers described a number of implications of the current state of play for the development of an infrastructure for CER (Box S-3)
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The dimen • sions noted above represent in certain ways the functional dimensions of relevance to the infrastructure that is needed for effectiveness research. There are phasing considerations as well, in part driven by the ability and need to take actions even without additional resources and in part driven by the time required to set in motion the necessary activities.
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CONTExT, PRESENTATION, AND DISCUSSION SUMMARIES Background for workshop discussions was provided by an IOM staffauthored background brief that illustrates the case for expanded CER, provides an overview of current CER activities and needs, and briefly discusses relevant issues not under consideration at the workshop (e.g., financing and structure of a new entity to coordinate CER work)
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The Need and Potential Returns for Comparative Effectiveness Research Enhancing the capacity for CER is not an end in itself but is rather a means to begin guiding the development of a healthcare system in which care is evidence driven and focused on providing care of value to individual patients. The staff-authored issue brief, provided as background for meeting discussions, and two presentations provided an important starting point for workshop discussions by summarizing current CER capacity, outlining a vision for -- and suggesting the potential returns of -- an evidence- and value-driven healthcare system.
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To narrow the rapidly growing gap between the available evidence on clinical effectiveness and the evidence necessary for sound clinical decision making, various organizations and recent public articles have called for the creation of a new entity and a quantum increase -- several billion dollars -- for CER (IOM, 2008a; Kupersmith et al., 2005; Wilensky, 2005)
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TABLE S-2 Prominent Comparative Effectiveness Research Activities and Needs -- Key Challenges Issue Key Challenges Head-to-head studies Scant resources; rapidly increasing need; comparison choice Systematic reviews Few primary studies; inconsistent methods; uncoordinated Comparative value insights Little agreement on metrics or role of costs; cost fluctuation Priority setting Fragmentation; inefficiency; no mechanism for coordination Study designs and tools Clinical trial time/cost/limits; large dataset mining methods Research life-cycle links Efficacy–effectiveness disjuncture; postapproval surveillance Evidence standards Standards not adapted to needs; inconsistency in application Practice guidance Disparate approaches; conflicting recommendations Coverage guidance Narrow evidence base; limited means for provisional coverage Application tools Public misperceptions; incentive structures; decision support SOURCE: IOM, 2007.
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These approaches differ in the degree of insulation between the stakeholder priority setting and the conduct of the scientific studies as well as in the ways the studies would be managed, the involvement of existing agencies, and the reporting of results. To this end, the ACA (2010)
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Additionally, $150 million plus $1 per Medicare part A and B enrollee has been appropriated for 2013 and $150 million plus $2 for each A/B enrollee, each year from 2014-2019. As outlined in the Act, PCORI will set a national agenda for research priorities, fund entities that conduct priority research, improve clinical effectiveness research methods, and ensure transparency and broad dissemination of its findings.
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Such infrastructure may eventually serve as an important component of infrastructure for evidence generation -- by supporting studies that compare the safety and effectiveness of treatments in different subgroups. A third and related need is developing a reliable and relevant evidence base on the comparative effectiveness of treatment options to help physicians and patients make the best possible healthcare decisions.
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Moreover, although cost- and clinical-effectiveness information should be considered in reimbursement and even clinical decisions, Wilensky underscored the importance -- for technical and political reasons -- of keeping these analyses and the places where they are conducted separate. Finally, as important as it is to have information available on clinical and cost effectiveness, the potential gains will not be achieved unless the reimbursement system is changed to make better use of information to reward health care of value rather than just paying more for doing more.
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Cost and Volume of Current Comparative Effectiveness Research As policy discussions about CER gather momentum, there continues to be a lack of awareness of the current scale of CER. Erin Holve, senior manager at AcademyHealth, presented the results of a survey of the costs and volume of current CER.
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The process resulted in the adoption of selection criteria -- including the importance of the conditions being treated or prevented, the current availability of effective treatments or preventive interventions, lack of definitive knowledge about the relative effectiveness of available treatments, research plausibility, and study type heterogeneity. These criteria were used to select among candidates nominated in the following comparative effectiveness categories: diagnostic studies drug–drug comparisons, health services systems studies, preventive interventions, surgical studies, and treatment studies across modalities.
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. These databases can provide relatively inexpensive and rapid access to clinical data and analyses of pharmaceutical exposures within a quantifiable source population, and these data reflect healthcare decisions and outcomes as they were actually made (versus the artificial constructs of an RCT)
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2 LEARNING WHAT WORKS TABLE S-4 The Comparative Effectiveness Studies Inventory Project Identified 16 Candidate Topics for Comparative Effectiveness Research Study Topic Study Type Age Group Condition Treatment of attention Comparative Children Mental diseases deficit hyperactivity effectiveness disorder in children: treatment studies drugs, behavioral across modalities interventions, no prescription Treatment of acute Comparative Adults Heart and thrombotic/embolic effectiveness vascular diseases stroke: clot removal, treatment studies reperfusion drugs across modalities Treatment of chronic Comparative Adults Heart and atrial fibrillation: effectiveness vascular diseases drugs, catheter treatment studies ablation, surgery across modalities Treatment of chronic Comparative Adults Neurological low back pain effectiveness diseases treatment studies across modalities Gamma knife surgery Comparative Adults Neurological for intracranial lesions effectiveness diseases vs. surgery and/or treatment studies whole brain radiation across modalities Treatment of localized Comparative Adults Cancer prostate cancer: effectiveness watchful waiting, treatment studies surgery, radiation, across modalities cryotherapy Diagnosis and Diagnostic studies Adults Cancer prognosis of breast cancer using genetic tests: human epidermal growth factor receptor 2 and others Over-the-counter drug Drug–drug Children Respiratory treatment of upper and drug–placebo disorders respiratory tract treatment studies infections in children Drug treatment of Drug–drug and drug– Adults Mental disorders depression in primary placebo treatment care studies
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2 SUMMARY TABLE S-4 Continued Study Topic Study Type Age Group Condition Drug treatment of Drug–drug and drug– Children Neurological epilepsy in children placebo treatment diseases studies Use of erythropoiesis- Drug–drug and drug– Adults Cancer stimulating agents placebo treatment in the treatment of studies hematologic cancers Outcomes of Health services/ Adults Heart and percutaneous systems studies vascular diseases coronary interventions in hospitals with and without onsite surgical backup Screening hospital Preventive Adults Infectious inpatients for interventions diseases methicillin-resistant Staphylococcus aureus infection Tobacco cessation: Preventive Adults Preventive nicotine replacement interventions interventions agents, oral medications, combinations Prevention and Surgical studies Adults Dermatological treatment of pressure diseases ulcers Inguinal hernia repair: Surgical studies Adults open vs. minimally Surgical invasive disorders NOTE: Study topics are categorized by study type, age group, and condition.
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It also recommended the appointment of a Clinical Effectiveness Advisory Board to oversee the program and the appointment of a Priority Setting Advisory Committee to identify high-priority topics. The report further prescribed the development of evidence-based methodological standards for systematic reviews, including a common language for characterizing the strength of evidence.
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For the many instances in which even RCTs are not feasible or sufficient to meet information needs, methods for conducting approximate randomized trials using balancing strategies and real-world observational clinical data have become increasingly common -- although a number of their important features remain to be explored and understood. Many trials focus on early outcomes or else introduce medicines or devices that bring additional complications.
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) √ √ √ √ √ √ √ colonoscopy Disease management Ambulatory blood pressure √ √ √ √ √ √ √ monitoring Intermittent intravenous insulin √ √ √ √ √ therapy Diagnosis CT angiography for suspected √ √ √ √ √ √ √ coronary artery disease Microvolt T-wave alternans √ √ √ √ √ √ √ Wireless capsule endoscopy √ √ √ √ √ √ √
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Treatment Brachytherapy for various cancers: √ √ √ √ √ √ √ breast, ovarian, and prostate cancer and brain tumors Dysfunctional uterine bleeding and √ √ √ √ √ √ √ fibroids Fallopian tube occlusion for permanent √ √ √ √ √ contraception Growth factor–mediated lumbar spinal √ √ √ √ √ fusion Intracoronary brachytherapy √ √ √ √ √ √ √ Minimally invasive surgery for low √ √ √ √ √ √ √ back pain Photodynamic therapy for Barrett's √ √ √ √ √ √ esophagus and esophageal cancer Vagus nerve stimulation for intractable √ √ √ √ √ √ √ depression Devices Artificial total disc replacement for √ √ √ √ √ √ √ lumbar and cervical spine Cochlear implants √ √ √ √ √ √ Total artificial heart √ √ √ √ √ √ Total hip resurfacing arthroplasty √ √ √ √ √ √ √ NOTE: Not all reviews are comprehensive assessments. Agency for Healthcare Research and Quality evidence-based practice centers have reviewed 5 of the 20 topics listed (ambulatory blood pressure monitoring, CT angiography, proteomic testing for ovarian cancer, spinal fusion for low back pain, and uterine fibroids)
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Moving beyond randomized trials to the real world, exploiting emerging semantic technology in order to integrate currently disparate medical data, using the knowledge generated for strategic decision support, and developing the next generation of statistical tools for analysis of clinical data are but a few concrete examples of the methods that need to be developed to provide an infrastructure for learning which is the right treatment, for the right patient, at the right time.
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Most of this work has built upon an appreciation for the role of technology assessment, comparative study designs, and HIT in the gathering and dissemination of best evidence to clinical practice; however, the development of the infrastructure needed for an expanded national capacity for CER has received less attention. To plan for such capacity rationally and strategically, one must have an understanding of the range of organizations currently conducting CER activities as well as some idea of which functions might benefit from either centralized or local approaches.
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Act, enacted as part of ARRA in 2009, allocates $20 billion to be used as incentive payments to promote the adoption and "meaningful use" of electronic health records. Along these lines, presentations summarized in Chapter 3 discuss key current issues and needed capacity for networks to support the generation, synthesis, and the application of evidence, as well as for providing opportunities to support learning from clinical practice.
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, clinical decision 32.9% support system (error checking) , PACS available outside radiology Stage 2 Clinical data repository, controlled medical vocabulary, 33.2% clinical decision support system inference engine, may have document imaging Stage 1 Ancillaries -- lab, radiology, pharmacy 12.5% Stage 0 All three ancillaries not installed 17.1% SOURCE: HIMSS Analytics, Hospital IT Expenses and Budgets Related to Clinical Sophistication.
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Rather than clinicians relying solely on large databases, centralized research centers, and analysis outside of healthcare delivery that can take months and years, Diamond presented a scenario in which clinicians access, in real time, current research and evidence syntheses as well as information provided via local networks on factors relevant to treating a particular patient (e.g., individual physician's patient outcomes versus his peers, community outbreaks, sensitivity patterns)
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Integrative Vehicles Required for Evidence Review and Dissemination The essential functions of any system dedicated to developing a robust evidence base for medical practice are synthesizing information derived from relevant trials and studies with insights emerging from clinical practice and ensuring that this information is continually updated. As clinical information systems are increasingly deployed, and as research increasingly draws upon connected and distributed data and information networks, the demand for synthesis work -- to ensure studies are appropriately reviewed, vetted, and incorporated into the evolving evidence base -- will also grow.
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Becker suggested that international efforts should focus on increasing efficiency of evidence syntheses through improved coordination -- perhaps with the formation of a registry of systematic reviews -- and fostering rapid progress in methods and standards development -- for the conduct, reporting, and assessment of evidence syntheses and guidelines. Such work will benefit all healthcare decision makers, as well as accelerate the development of key infrastructure elements needed for expanded CER capacity in the United States.
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For each domain, the skill sets and competencies, training and education approaches, and issues related to expanding current capacity were reviewed. The authors concluded that quantifying the needs of the overall workforce requires a better sense for the scale of expansion for the various CER activities (e.g., systematic reviews, trials, studies, guideline development, data mining)
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0 Clinical Epidemiology/Pharamcoepidemiology/ Biomedical Informatics Evidence-Based Medicine Electronic Clinical Data Clinical Other Clinical Information - Electronic Health Record Data into Data Warehouses Trials Studies Needs - Clinical Decision Support - Public Health Informatics Systematic Reviews Data Mining and Analysis - Prioritization - Validation Methods Development Health Services Research Clinical - Outcomes Research Guidelines Guideline - Decision Science Development Development - Economics - Benefits Design and - Coverage Decisions Implementation - Formulary Decisions Guideline Communications Implementation Dissemination - Translation for Clinicians - Translation for Patients/Consumers FIGURE S-3 Key activity domains for comparative effectiveness research. Workforce development will be critical to support the many primary functions within each of these domains as well as to foster the cross-domain interactions and activities identified (e.g., methods development, identifying information needs)
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Important lessons relevant to U.S. efforts to build CER capacity include establishing a stable funding source to support CER that, unlike the standard grant review cycle time, can fund rapidly evolving research needs; ensuring a timely process focused on the needs of decision makers to increase the likelihood that data generated by a study will be relevant; designing programs independent from government and industry, and ensuring a transparent decision-making process; increasing the efficiency and effectiveness of research by creating partnership between universities and those conducting field evaluations; and leveraging Medicare's influence on private payers to more broadly support coverage with evidence development.
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, Frisse offered suggestions on IT platform requirements and approaches that will help realize significant societal benefit at a realistic marginal cost. With proper design and integration, the current collection of databases, health record systems, health information exchanges, financing, workforce, policies, and governance can evolve into a system that can address a range of needs.
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To remain effective in a practicebased, learning health system, these vertical, procedure-based clinical data will need the longitudinal, medical-condition context important for the development of quality comparative effectiveness information. Resource and analysis development work must translate into a dynamic, real-time learning infrastructure, including built-in feedback processes and a focus on the patient and the point of care.
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Tools for clinical point-of-care application of comparative effectiveness analyses and new analytic tools for CER will affect quality, effectiveness, appropriateness, and efficiency of care. Practical Challenges and Infrastructure Priorities for Comparative Effectiveness Research The process of developing and completing study protocols must be efficient in order for CER to reach its full potential for improving medical care.
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Chu, Southern California regional president for Kaiser Foundation Health Plan and Hospitals, described how these changes will shape the future practice environment and suggested that health professions education should take place in environments that emulate current models of best care. Such an approach would encourage the effective use of new tools by teams of health professionals as well as use and improvement of approaches that achieve the best outcomes across the full continuum of care and patient needs.
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This expansion would include all aspects of health, including biomedical, public health, and multidisciplinary research on the social and environmental determinants of health. Table S-7 outlines an approach to achieving this new research vision, and among the most pressing needs is the development of a new cadre of researchers, clinicians, and health leaders -- a workforce that includes, among others, health professionals, engineers, sociologists, urban planners, policy experts, and economists.
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• raining to provide new skills, including inter T professional training • ncentives within academia to support all types of health I researchers (e.g., academic home, revised promotion, tenure criteria) • nformation technology investments (e.g., electronic New Infrastructure I health records, personal health record, regional health information organizations)
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The involvement of community constituencies affected by research will increasingly be an essential component of health research -- through contributing input into research priorities, helping build trust of community participation in research, or disseminating findings. Particularly critical are national policy makers who will drive this transformation by endorsing the importance of health research in leveraging biomedical discoveries for health improvements; by providing adequate funding for the full range of health research needed, including workforce development; and by helping to address current barriers to research (e.g., Health Insurance Portability and Accountability Act procedures)
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Bocchino, vice president for clinical affairs and strategic planning at America's Health Insurance Plans, discussed several successful public–private partnerships in which health plans and federal agencies have partnered to create databases that are useful in identifying potential safety issues and opportunities to improve care and care delivery. An extension of these activities could contribute to the development of a national data system to serve as a central part of the nation's health research infrastructure.
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Key barriers identified relevant to the CER infrastructure included the need for internal industry champions to drive collaborative work; the need to meet the costs of fulltime equivalent and data management; skepticism by industry, NIH, and academic leadership on the value of such partnerships; and variable legal opinions on intellectual property and medicolegal risks. Moving Forward Although expanding CER capacity offers many potential gains for health care, the scale of needed transformation is also large and spans all healthcare sectors.
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Suggested goals for these areas included the development of capacity to produce relevant data, ensuring maximal value of data through integration and system linkages and making data and information available to appropriate users when and where needed; development of research approaches to meet the needs of CER end users; education of a cadre of professionals -- from across healthcare sectors -- trained to use tools and techniques for developing and applying comparative effectiveness information; prioritization and coordination across the many organizations engaged in various aspects of evidence development -- primary research, synthesis, translation -- to enable more efficient information production; movement from evidence to evidence-based decision making; and sufficient and sustained funding to establish and support CER and its application as an integral part of the U.S. healthcare system.
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Although viewed as an important element of health reform by most healthcare stakeholders, additional work is needed to build support by the public and policy makers for needed investments and potential returns from CER. An open discussion session on this topic was led by Mary Woolley, from Research!
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D evote additional attention to data stewardship issues: Because • the basic resource for effectiveness research is the clinical data system, the Roundtable needs to catalyze more discussion on the integrity of this resource, including issues of maintenance, privacy, and data ownership. I dentify possible incentives: Look at how subsidies and reimburse • ment regulations can stimulate increased use of HIT in medical care, increased use of HIT for application of evidence, and increased use of HIT for the development of evidence.
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2008b. Learning what works: Infrastructure required for comparative effectiveness research.
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2009. Building the training capacity: Implementation priorities In Learning what works: Infrastructure required for comparative effectiveness research.
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