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The fourth workshop of the EBM Roundtable's Learning Healthcare System series, entitled, Redesigning the Clinical Effectiveness Research Paradigm: Innovation and Practice-Based Approaches, was held on December 12-13, 2007, in The National Academy of Sciences Building, Lecture Room, located at 2100 C Street, NW, Washington, DC 20037. The workshop agenda, common themes and presentations are included below.
Participants examined the role of innovative research designs and tools that can expedite the development of evidence on clinical effectiveness by streamlining approaches and bringing research and practice closer together. In the context of existing evidence gaps and a need for a substantially improved understanding of comparative clinical effectiveness of healthcare interventions, this workshop explored opportunities presented by emerging research networks and data resources, innovative study designs, and new methods of analysis and modeling that will address the existing evidence gap and the need for a substantially improved understanding of comparative clinical effectiveness of healthcare interventions. Presentations and discussion focused on how these tools and methods can be engaged in a new generation of studies that better address current challenges in clinical effectiveness research.
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:
- Need for substantially improved understanding of the comparative clinical effectiveness of healthcare interventions.
- Strengths of the randomized controlled trial muted by constraints in time, cost, and limited applicability.
- Opportunities presented by the size and expansion of potentially interoperable administrative and clinical data sets.
- Opportunities presented by innovative study designs and statistical tools.
- Need for innovative approaches leading to a more practical and reliable clinical research paradigm.
- 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.
Day One
Welcome & opening remarks
Denis A. Cortese, Mayo Clinic and Chair, IOM Roundtable
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
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
Ø Hormone replacement therapy
JoAnn E. Manson, Harvard Medical School
Ø Drug-eluting coronary stents
Ashley B. Boam, Food and Drug Administration
Ø Bariatric surgery
David R. Flum, University of Washington
Ø Antipsychotic therapeutics
Philip S. Wang, National Institute of Mental Health
Ø Cancer screening
Peter B. Bach, Memorial Sloan-Kettering Cancer Center
Respondent: Brian G. Firth, Cordis
General discussion
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
Ø Innovative approaches to trials
Robert M. Califf, Duke University
Ø Innovative analytic tools for large clinical and administrative data bases
Sebastian Schneeweiss, Harvard Medical School
Ø Adaptive and Bayesian approaches to study design
Donald A. Berry, University of Texas, Anderson Cancer Center
Ø Simulation and predictive modeling
Mark S. Roberts, University of Pittsburgh, Archimedes Inc.
Ø Emerging genetic information
Teri Manolio, National Human Genome Research Institute
Respondent: Michael Lauer, National Heart, Lung and Blood Institute
General discussion
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 data bases? 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
Ø Electronic health records/point of care data
Ronald A. Paulus, Geisinger Health System
Ø Administrative and claims data
Alexander M. Walker, Harvard School of Public Health & WHISC
Ø Registries
Alan J. Moskowitz, Columbia University
Ø Distributed research model
Richard Platt, Harvard Pilgrim Health Care and Harvard Medical School
Respondent: William H. Crown, Ingenix
General discussion
General discussion of the key points of the day
Denis A. Cortese, Mayo Clinic and Chair, IOM Roundtable
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Day Two
Welcome & short re-cap of day one
J. Michael McGinnis, Institute of Medicine
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?
Carolyn M. Clancy, Agency for Healthcare Research and Quality and IOM Roundtable
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
Ø Large data streams and the power of numbers
Sharon-Lise Normand, Harvard Medical School
Ø Observational studies
Wayne A. Ray, Vanderbilt University
Ø Experimental and hybrid studies
John Rush, University of Texas Southwestern Medical Center
Ø Accommodating genetic variation as a standard feature of clinical research
Isaac S. Kohane, Harvard Medical School
Ø Phased introduction and payment for interventions under protocol
Wade M. Aubry, Center for Medical Technology Policy
Ø Research networks
Eric B. Larson, Group Health Cooperative
Respondent: Joe V. Selby, Kaiser Permanente
General discussion
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
Ø Course of care data
Greg Pawlson, National Committee for Quality Assurance
Ø Manufacturer data
Peter K. Honig, Merck
Ø Regulatory requirements and data generation
Mark B. McClellan, Brookings Institution
Ø Publicly sponsored health data
J. Sanford Schwartz, University of Pennsylvania
- Presentation (soon to come)
Ø Building the research infrastructure
Alan M. Krensky, National Institutes of Health
Ø Engaging consumers
Kathy Hudson, Johns Hopkins University
Respondent: Jerry Avorn, Harvard Medical School
General discussion
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), John E. Niederhuber (NCI), Garry Neil (Johnson & Johnson), Lewis Sandy (United HealthCare)
Concluding summary remarks and adjournment
J. Michael McGinnis, Institute of Medicine
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