Meeting 4: Redesigning the Clinical Effectiveness Research Paradigm – Innovation and Practice Based Approaches

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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.

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)

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

• 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

• 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 underprotocol

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

• 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

About this Event

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 is 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.


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