Redesigning the Clinical Effectiveness Research Paradigm Innovation and Practice-Based Approaches: Workshop Summary (2010) / Chapter Skim
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With the information and practice demands at hand, and new tools in the works, a more practical and reliable clinical effectiveness research paradigm is needed. Information relevant to guiding decision making in clinical practice requires the assessment of a broad range of research 1 The planning committee's role was limited to planning the workshop, and the workshop summary has been prepared by Roundtable staff as a factual summary of the issues and presentations discussed at the workshop.
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The overall goal of the meeting was to explore these issues, identify potential approaches, and discuss possible strategies for their engagement. Participants examined ways to expedite the development of clinical effectiveness information, highlighting the opportunities presented by innovative study designs and new methods of analysis and modeling; the size and expansion of potentially interoperable administrative and clinical datasets; and emerging research networks and data resources.
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• Need for innovative approaches leading to a more practical and reli able clinical research paradigm. • Need to build a system in which clinical effectiveness research is a more natural by-product of the care process.
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Noted at the workshop was the fact that, as currently practiced, the randomized controlled and blinded trial is not the gold standard for every circumstance. While not an exhaustive catalog of methods, Table S-1 provides a sense of the range of clinical research approaches that can be used to improve understanding of clinical effectiveness.
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relaxed with the goal of improving the secondary applicability of results for clinical or coverage decision making by accounting for broader patient populations or conditions of real-world clinical practice. For example, PCTs often have fewer patient inclusion/exclusion criteria, and longer term, patient centered outcome measures.
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Interrupted Study design used to determine how Primary or Approach Time Series a specific event affects outcomes of secondary dependent interest in a study population. This design can be experimental or non experimental depending on whether the event was planned or not.
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Disease rates and exposures are measured in each of a series of populations and their relation is examined. Natural Non-experimental design that examines Primary or No Experiment a naturally occurring difference Secondary between two or more populations of interest -- i.e., instances in which the research design does not affect how patients are treated.
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Clinical Effectiveness Research and the IOM Roundtable Formed in 2006 as the Roundtable on Value & Science-Driven Health Care brings together key stakeholders from multiple sectors -- patients, health providers, payers, employers, health product developers, policy makers, and researchers -- for cooperative consideration of the ways that evidence can be better developed and applied to drive improvements in the effectiveness and efficiency of U.S. medical care.
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This publication summarizes the proceedings of the fourth workshop in the Learning Healthcare System series, focused on improving approaches to clinical effectiveness research. The Roundtable's work is predicated on the principle that "to the greatest extent possible, the decisions that shape the health and health care of Americans -- by patients, providers, payers, and policy makers alike -- will be grounded on a reliable evidence base, will account appropriately for individual variation in patient needs, and will support the generation of new insights on clinical effectiveness." Well-conducted clinical trials have and will continue to contribute to this evidence base.
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COMMON THEMES The Redesigning the Clinical Effectiveness Research Paradigm workshop featured speakers from a wide range of perspectives and sectors in health care. Although many points of view were represented, certain themes emerged from the 2 days of discussion, as summarized below and in Box S-22: • Address current limitations in applicability of research results.
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Part of the strategy challenge is choosing the right tool at the right time. For the future of clinical effectiveness research, the important issues relate not to whether randomized experimental studies are better than observational studies, or vice versa, but to what's right for the circumstances (clinical and economic)
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• Encourage innovation in clinical effectiveness research conduct. The kinds of "safe harbor" opportunities that exist in various fields for developing and testing innovative methodologies for addressing complex problems are rarely found in clinical research.
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• Foster the transformational research potential of information tech nology. Broad application and linkage of electronic health records hold the potential to foster movement toward real-time clinical effectiveness research that can generate vastly enhanced insights into the performance of interventions, caregivers, institutions, and systems -- and how they vary by patient needs and circumstances.
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This enhanced flexibility and range of research resources is facilitated by the development of innovative design and analytic tools, and by the growing potential of electronic health records to allow much broader and structured access to the results of the clinical experience. The ability to draw on realtime clinical insights will naturally improve over time.
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in making judgments about efficacy in the early 1970s, clinical effectiveness research has developed rapidly and has helped to transform medical care. However, Fineberg suggested that the resulting research paradigm, with randomized controlled double blind trials at the pinnacle, has often left important evidence needs unmet when combined with the costs, complexity, and lack of generalizability of RCTs.
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Clancy, director of the Agency for Healthcare Research and Quality (AHRQ) , shared perspectives in two broad areas: emerging methods that might be applied to meet current challenges in clinical effectiveness research and approaches to turning evidence into action.
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The experiences recounted show that, from randomized trials to observational studies, each investigative approach has limitations. These limitations argue against using a particular approach and suggest that the research community needs more experience with the array of methodologies used to generate insights into clinical effectiveness and structured decision rules to guide the study design choice for particular research circumstances.
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Both observational studies and clinical trials have contributed critically important information to elucidate the health effects of HRT with estrogen and progestin and to inform decision making, and they constitute a model suggesting that research findings should be considered in the context of the totality of available evidence and that studies should be designed to complement and extend existing data. Manson noted that observational studies and randomized clinical trials of menopausal HRT and coronary heart disease (CHD)
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While at least two measures obtained from angiography (i.e., late loss and percentage diameter stenosis of vessel) have been identified as biomarkers with a strong correlation to clinical effectiveness -- specifically, the need for a reintervention -- no such marker has been identified as a possible surrogate for safety outcomes, such as death or myocardial infarction.
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However, there is a paucity of evidence to help guide clinical, purchasing, and policy decisions regarding antipsychotic medications. The recently completed, NIMH-sponsored comparative effectiveness trials of antipsychotic medications in patients
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These include new analytic methods, propensity score adjustments, and instrumental variable techniques. Cancer Screening Developing comparative effectiveness information about screening tests is a complex undertaking, as demonstrated by Peter B
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Robert Califf from Duke University emphasizes the importance of focusing discussion about medical evidence on a serious examination of ways to improve the operational methods of both approaches and of building human systems that take advantage of the power of modern informatics on improving both RCTs and observational studies. In particular, the design and conduct of RCTs needs to evolve to take further advantage of modern informatics and to provide a more flexible and practical tool for clinical effectiveness research.
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Trials also could take better advantage of "natural units of care" with cluster randomization, or provide information more relevant to practice by focusing on research questions based on gaps in clinical practice guidelines or being conducted in real-world practice (e.g., pragmatic clinical trials)
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Adaptive and Bayesian Approaches to Study Design Adaptive and, particularly, Bayesian approaches to study design offer opportunities to improve on randomization and to facilitate new ways of learning in health care. Donald A
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In conclusion, Berry notes that although the rigor and inflexibility of the current research paradigm has been important to establishing medicine as a science, new approaches such as Bayesian thinking and methodologies can help to move the field even further by making research more nimble and applicable to patient care, while maintaining scientific rigor. Simulation and Predictive Modeling Certain research questions or evidence gaps will be difficult or impractical to answer using clinical trial methods.
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Two prototypes for applying genomic information from genomewide association studies to clinical effectiveness research are genetic variants related to two traits -- Type 2 diabetes risk and warfarin dosing. Though both have sufficient scientific foundations and clinical availability, they remain many steps away from clinical application.
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Organizing and Improving Data Utility Vastly larger, electronically accessible health records and administrative and clinical databases currently under development offer previously
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Collectively these papers illustrate how these approaches can be applied to improve the efficiency and quality of clinical practice; provide meaningful complementary data to existing research findings; accelerate the capture and dissemination of learnings from innovation in practice; and offer a means to process complex information -- derived from multiple sources and formats -- and develop information that supports clinical practice and informs the research enterprise. The Electronic Health Record and Care Reengineering: Performance Improvement Redefined Ronald A
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Walker of Worldwide Health Information Science Consultants and the Harvard School of Public Health, data from health insurance claims form the backbone of many health analytic programs. Although administrative databases are being used more effectively for research, their development and especially their application for generating insights into clinical effectiveness require careful consideration and attention to potential methodologic pitfalls and hazards.
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Clinical Effectiveness Research: The Promise of Registries The dynamic and highly innovative character of healthcare technologies has been important to improvements in health; however, because intervention capacities often evolve due to iterative improvements or expanded use in practice, assessing their effectiveness presents a substantial challenge to researchers and policy makers. Because clinical registries capture information important to understanding the use of diagnostic and therapeutic interventions throughout their lifecycle, they are particularly valuable resources for assessing real-world health and economic outcomes.
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Clinical registries also provide comparative effectiveness information. Registries offer a powerful means to capture innovation and downstream learning that take place in practice and to develop information complementary and supplementary to that produced by randomized trials.
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Not only are the number of research questions increasing, but current approaches to developing comparative effectiveness information are impractical -- making the need to take better advantage of new sources of data and other opportunities to produce evidence relevant to clinical practice more urgent. Many participants noted that in research today, knowledge is expanding much faster than we can effectively translate
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In a session focused on moving to the next generation of studies, participants considered a set of interrelated questions: What are the key opportunities and needed advancements to improve our approach to clinical effectiveness research? How might we take better advantage of emerging resources to plan, develop, and sequence studies that are more timely, relevant, efficient, and generalizable?
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Although the design of observational studies is a complex subject, opportunities exist to improve their capacity to contribute to clinical effectiveness research. The notion that study design and analysis are quick and inexpensive is misleading.
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As illustrated by John Rush of the University of Texas Southwestern Medical Center, a variety of methods are available for answering such complex and clinically important questions -- such as using observational data obtained when systemic practices are employed (registry/cohort studies) , effectiveness trials (practical clinical trials)
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Although the costs of high-throughput genotyping are rapidly dropping, the cost of assembling, phenotyping, and studying large populations is an estimated $3 billion for 500,000 individuals. Fortunately, the informational by-products of routine clinical care can be used to bring phenotyping and sample acquisition to the same high-throughput, commodity price point as is currently true of genotyping costs.
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Phased Introduction and Payment for Interventions Under Protocol Clinical effectiveness research draws on experience gained in the postmarket setting. Because this research is distinct from that required for FDA approval and market entry, innovative policies that encourage and facilitate these types of investigations will be needed.
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language defining medical necessity, ethical issues, the difficulty in achieving multistakeholder consensus, lack of a clear definition of "adequate" evidence compared to "ideal" evidence, timing of CED in regard to existing coverage without restrictions, and limitations of the number of studies that can be implemented under CED. Research Networks Successful initiatives such as the Cooperative Oncology Groups, HMO Research Network, Center for Education and Research in Therapeutics, the Framingham Heart Study, and others provide useful models for how the clinical effectiveness paradigm might be redesigned to be more timely, relevant, efficient, and generalizable.
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A redesign of the clinical effectiveness research paradigm ideally would address challenges the NIH will face as it aims to reengineer the massive U.S. biomedical research enterprise.
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Pharmaceutical Industry Data As the healthcare system becomes more complex, the pharmaceutical industry is increasingly challenged to meet regulatory, payer, and patient demands for demonstration of the value of their products. Such assessments of risk–benefit, long-term safety, and comparative effectiveness often require postmarket clinical trial and database commitments.
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Regulatory Requirements and Data Generation Data developed and collected to satisfy regulatory requirements offer a rich resource and a driving force for improvements in our capacity for clinical effectiveness research.
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Ensuring Optimal Use of Data Generated by Public Investment Though large amounts of data exist and have the potential to inform clinical and comparative effectiveness assessment, substantial barriers prevent optimal use of these data. Many innovative opportunities are possible from these publicly supported and generated data, such as the ability to inform clinical practice and policy.
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Engaging Consumers Conducting meaningful clinical effectiveness research requires collecting, sharing, and analyzing large quantities of health information from many individuals, potentially for long periods of time. To be successful, this research will need the support and active participation of patients.
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ORGANIZING THE RESEARCH COMMUNITY FOR CHANGE Most issues here require the attention of the research community in order to drive change, with some of the most pressing concerns in areas such as methods improvement, data quality and accessibility, incentive alignment, and infrastructure. Much work is already underway to enhance and accelerate clinical effectiveness research, but efforts are needed to ensure stronger coordination, efficiencies, and economies of scale within the research community.
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Clinical Effectiveness Research • Methodologies. How do various research approaches best align to different study circumstances -- e.g., nature of the condition, the type of intervention, the existing body of evidence?
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Should a venue be established for peri odic convening of primary care and specialty physician groups to explore clinical effectiveness research priorities, progress in practice-based research, opportunities to engage in registry-related research, and improved approaches to clinical guideline develop ment and application? • Academic health center engagement.
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Successful use of clinical data as a reliable resource for clinical effectiveness evidence development requires the development of standards and approaches that assure the quality of the work. How might Roundtable members encour age or foster work of this sort?
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Because shortfalls in coordination and communication impinge on the funding, effectiveness, and efficiency of the clinical research process -- not to mention its progress as a key element of the learning healthcare system -- the notion of working productively together is vital for both patients and the healthcare community. Better coordination, collaboration, public–private partnerships, and priority setting are compelling priorities, and the attention and awareness generated in the course of this meeting are important to the Roundtable's focus on redesigning the clinical effectiveness research paradigm.
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2007. Developments in post-marketing comparative effectiveness research.
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