Appendix A
Workshop Agenda

Redesigning the Clinical Effectiveness Research Paradigm: Innovation and Practice-Based Approaches

A Learning Healthcare System Workshop IOM Roundtable on Evidence-Based Medicine


December 12-13, 2007

Lecture Room

The National Academy of Sciences Building

2100 C Street, NW

Washington, DC 20037

Issues motivating the discussion:

  1. Need for substantially improved understanding of the comparative clinical effectiveness of healthcare interventions.

  2. Strengths of the randomized controlled trial muted by constraints in time, cost, and limited applicability.

  3. Opportunities presented by the size and expansion of potentially interoperable administrative and clinical datasets.

  4. Opportunities presented by innovative study designs and statistical tools.

  5. Need for innovative approaches leading to a more practical and reliable clinical research paradigm.

  6. Need to build a system in which clinical effectiveness research is a more natural by-product of the care process.

Goal: To explore these issues, identify potential approaches, and discuss possible strategies for their engagement.



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Appendix A Workshop Agenda Redesigning the Clinical Effectiveness Research Paradigm: Innovation and Practice-Based Approaches A Learning Healthcare System Workshop IOM Roundtable on Evidence-Based Medicine December 12-13, 2007 Lecture Room The National Academy of Sciences Building 2100 C Street, NW Washington, DC 20037 Issues motivating the discussion: 1. Need for substantially improved understanding of the comparative clinical effectiveness of healthcare interventions. 2. Strengths of the randomized controlled trial muted by constraints in time, cost, and limited applicability. 3. Opportunities presented by the size and expansion of potentially interoperable administrative and clinical datasets. 4. Opportunities presented by innovative study designs and statistical tools. 5. Need for innovative approaches leading to a more practical and reli- able clinical research paradigm. 6. Need to build a system in which clinical effectiveness research is a more natural by-product of the care process. Goal: To explore these issues, identify potential approaches, and discuss possible strategies for their engagement. 

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2 REDESIGNING THE CLINICAL EFFECTIVENESS RESEARCH PARADIGM DAY ONE 8:30 Welcome and opening remarks Denis A. Cortese, Mayo Clinic and Chair, IOM Roundtable on Evidence-Based Medicine 9:00 keynote: clinical effectiveness research—past, present, and future Overview of the evolution of clinical effectiveness research, current state of play, key challenges (e.g., keeping pace, inference gap, cost, policy), and future opportunities to generate reliable insights. Harvey V. Fineberg, Institute of Medicine 9:30 session 1: cases in point—learning from experience This session will present several case examples of high profile issues—some linked to delayed applications of effective treatments or to premature adoption of unwarranted treatments—that hold important lessons for future work in the design and interpretation of clinical effectiveness studies. Chair: Joel Kupersmith, Veterans Health Administration and IOM Roundtable on Evidence-Based Medicine • Hormone replacement therapy JoAnn E. Manson, Harvard Medical School • Drug-eluting coronary stents Ashley B. Boam, Food and Drug Administration • Bariatric surgery David R. Flum, University of Washington [10:15 to 10:30 Break] • Antipsychotic therapeutics Philip S. Wang, National Institute of Mental Health • Cancer screening Peter B. Bach, Memorial Sloan-Kettering Cancer Center Respondent: Brian G. Firth, Cordis General discussion 12:00 lunch (Box lunches are available in the Executive Dining Room)

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 APPENDIx A 12:45 session 2: taking advantage of neW tools and techniques Where might we expect improvements from analytic tools, sample size, and data quality and availability? What novel techniques could researchers use in conjunction with new information, models, and tools? Chair: Donald M. Steinwachs, Johns Hopkins University and IOM Roundtable on Evidence-Based Medicine • Innovative approaches to clinical trials Robert M. Califf, Duke University • Innovative analytic tools for large clinical and administrative databases Sebastian Schneeweiss, Harvard Medical School • Adaptive and Bayesian approaches to study design Donald A. Berry, University of Texas, M.D. Anderson Cancer Center • Simulation and predictive modeling Mark S. Roberts, University of Pittsburgh, Archimedes Inc. • Emerging genetic information Teri A. Manolio, National Human Genome Research Institute Respondent: Michael Lauer, National Heart, Lung, and Blood Institute General discussion [2:45 to 3:00 Break] 3:00 session 3: organizing and improving data utility What are the research implications of the development of much larger, electronically accessible health records and administrative and clinical databases? How might they most appropriately be applied to clinical effectiveness research? What rules of engagement are needed to harness emerging data sources? Chair: Denis A. Cortese, Mayo Clinic and IOM Roundtable on Evidence-Based Medicine • Electronic health records/point of care data Ronald A. Paulus, Geisinger Health System

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 REDESIGNING THE CLINICAL EFFECTIVENESS RESEARCH PARADIGM • Administrative and claims data Alexander M. Walker, Harvard School of Public Health & Worldwide Health Info. Science Consultants • Registries Alan J. Moskowitz, Columbia University • Distributed research model Richard Platt, Harvard Pilgrim Health Care and Harvard Medical School Respondent: William H. Crown, Ingenix General discussion 4:45 general discussion of the key points of the day Chair: Denis A. Cortese, Mayo Clinic and IOM Roundtable on Evidence-Based Medicine 5:15 Reception—great hall DAY TWO 8:45 Welcome and short recap of day one J. Michael McGinnis, Institute of Medicine 9:00 keynote: research that meets evidence needs Evidence gaps and research challenges. Insights on how the approach to clinical effectiveness research can better take advantage of emerging tools and study designs to address the challenges, including those related to generalizability, heterogeneity of treatment effects, and multiple co-morbidities? Chair: Carolyn M. Clancy, Agency for Healthcare Research and Quality and IOM Roundtable on Evidence-Based Medicine 9:30 session 4: moving to the next generation of studies 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—and account for lifecycle variation of the conditions and interventions in play? Chair: Robert M. Califf, Duke University

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 APPENDIx A • Large data streams and the power of numbers Sharon-Lise T. Normand, Harvard Medical School • Observational studies Wayne A. Ray, Vanderbilt University • Experimental and hybrid studies A. John Rush, University of Texas Southwestern Medical Center [10:15 to 10:30 Break] • Accommodating genetic variation as a standard feature of clinical research Isaac S. Kohane, Harvard Medical School • Phased introduction and payment for interventions under protocol Wade M. Aubry, Center for Medical Technology Policy • Research networks Eric B. Larson, Group Health Cooperative Respondent: Joe V. Selby, Kaiser Permanente General discussion 12:00 lunch (Box lunches are available in the Executive Dining Room) 12:45 session 5: aligning policy With research opportunities What policy levers can drive innovative research and progress in practice-based approaches? What barriers need to be addressed to accelerate the progress? Chair: Richard Platt, Harvard Pilgrim Health Care and IOM Roundtable on Evidence-Based Medicine • Course-of-care data Greg Pawlson, National Committee for Quality Assurance • Manufacturer data Peter K. Honig, Merck • Regulatory requirements and data generation Mark B. McClellan, Brookings Institution • Publicly sponsored health data J. Sanford Schwartz, University of Pennsylvania • Building the research infrastructure Alan M. Krensky, National Institutes of Health

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 REDESIGNING THE CLINICAL EFFECTIVENESS RESEARCH PARADIGM • Engaging consumers Kathy Hudson, Johns Hopkins University Respondent: Jerry Avorn, Harvard Medical School General discussion 3:15 session 6: organizing the research community for change—discussion panel What guiding principles are important in refining the clinical effectiveness research paradigm and in fostering the necessary changes? What issues need to be addressed in integrating trials into a lifecycle approach to evidence development? What expertise and training might be needed? What are some of the political challenges? Chair: J. Michael McGinnis, Institute of Medicine Panel discussion Panel: Alan M. Krensky (NIH), Mark B. McClellan (Brookings Institution), Garry Neil (Johnson & Johnson), John Niederhuber (NCI), Lewis Sandy (United HealthCare) 4:30 concluding summary remarks and adjournment J. Michael McGinnis, Institute of Medicine Workshop Planning Committee Robert M. Califf, MD, Duke Clinical Research Institute, Duke University Lynn Etheredge, George Washington University Kim Gilchrist, MD, AstraZeneca LP Bryan Luce, PhD, MBA, United Biosource Corporation Jonathan Perlin, MD, PhD, MSHA, FACP, HCA Inc. Richard Platt, MD, MS, Harvard Medical School and Harvard Pilgrim