Fourth Symposium on Comparative Effectiveness Research Methods
From Efficacy to Effectiveness
June 12-13, 2012

These recordings are from the fourth invitational symposium on research methods for comparative effectiveness research (CER) and patient-centered outcomes research (PCOR) sponsored by Agency for Healthcare Research and Quality (AHRQ). This 2-day symposium and workshop was held on June 12 and 13, 2012 at the AHRQ Conference Center in Rockville, Maryland. The symposium is a follow-up to the AHRQ conferences on Methods in Comparative Effectiveness and Safety Research hosted in 2006, 2009, and 2011; papers presented at the first two conferences were published in a Medical Care Supplement (copies may be downloaded from http://tinyurl.com/dl59yw or effectivehealthcare.ahrq.gov) and the proceedings of the 2011 symposium were published in a Pharmacoepidemiology and Drug Safety journal supplement (copies may be downloaded from http://onlinelibrary.wiley.com/doi/10.1002/pds.v21.S2/issuetoc or http://www.effectivehealthcare.ahrq.gov/index.cfm/search-for-guides-reviews-and-reports/?pageaction=displayproduct&productid=1050).

The theme of the 2012 symposium was “From Efficacy to Effectiveness”. Invited experts gave podium presentations on innovative research methods to evaluate mechanisms that contribute to the differences in the results of randomized clinical trials (i.e., efficacy studies) and observational studies conducted in real world settings (i.e., outcomes and effectiveness research). The proceedings of the symposium will be published as a special journal supplement in the summer of 2013 in the Journal of Clinical Epidemiology.

MATERIAL IN THESE PRESENTATIONS IS NOT FOR QUOTATION OR CITATION. PLEASE CHECK WITH EACH INDIVIDUAL AUTHOR FOR PERMISSION TO USE ANY INFORMATION PRESENTED.

Presentations and slides do not represent the policy of either the Agency for Healthcare Research & Quality (AHRQ), the US Department of Health & Human Services (DHHS), The Brigham & Women’s Hospital (BWH), or Harvard Medical School (HMS). The views expressed herein are those of the presenter, and no official endorsement by AHRQ, DHHS, BWH, or HMS is intended or should be inferred.

If you have any questions or concerns regarding the DEcIDE Methods Center and its learning activities, please contact Liz Robinson Garry (DEcIDEMethods@partners.org).

 

——————————————————————————————————————————–
DAY 1 – TUESDAY, JUNE 12, 2012
——————————————————————————————————————————–

Video 1: http://www.youtube.com/watch?v=RB5qd4QYnPA

    • PRE-SYMPOSIUM WORKSHOP
    • Michele Jonsson-Funk, PhD – Doubly Robust Estimation (see details below)
      Research Assistant Professor of Epidemiology, University of North Carolina at Chapel Hill

 

  • Estimation of the effect of a treatment or exposure with a causal interpretation from studies where exposure is not randomized may be biased if confounding is not taken into appropriate account. Adjustment for confounding is often carried out through regression modeling of the relationships among treatment, confounders, and outcome. Doubly robust estimation produces a consistent effect estimator as long as one of two component regression models is correctly specified and assuming that there are no unmeasured confounders, giving the analyst two chances to correctly specify at least one of the regression models. This workshop will provide a brief introduction to doubly robust estimators, present sample code using a SAS macro, illustrate the use of the macro with results from analyses of simulated data, and discuss issues including interpretation of estimates, assumptions and limitations of this approach.

Video 2: http://www.youtube.com/watch?v=6m_uFtDQzHg

    • OPENING INTRODUCTIONS
      Scott R. Smith, PhD
      DEcIDE Director, Center for Outcomes and Evidence, Agency for Healthcare Research & Quality

 

    • KEYNOTE ADDRESS
      “The PCORI Methods Report and what it means for future CER methods development”
    • Sherine E. Gabriel, MD, MSc
      William J. and Charles H. Mayo Professor of Medicine and Epidemiology, Mayo Clinic
      Chair, Methodology Committee, Patient-Centered Outcomes Research Institute (PCORI)

 

  • Keynote Panel/Q&A
    Jean Slutsky, PA, MSPH
    Director, Center for Outcomes and Evidence, Agency for Healthcare Research & Quality
  • Philip Wang, MD, DrPH
    Deputy Director and Acting Scientific Director, National Institute of Mental Health
  • Sebastian Schneeweiss, MD, ScD
    Vice Chief, Pharmacoepidemiology, Brigham and Women’s Hospital/Harvard Medical School

Video 3: http://www.youtube.com/watch?v=keHmNV0t2oU

    • SESSION I. EVALUATIONS OF HEALTH CARE DELIVERY SYSTEMS
    • The need for more comparative effectiveness of health care delivery systems was highlighted in the 2009 “Top 100 List of Comparative Effectiveness Research Questions” issued by the IOM Committee.  Nevertheless, many methodological challenges remain in estimating the effectiveness of healthcare delivery systems that are both generalizable and valid.

 

  • “Effect of Erythropoietin Stimulating Agents (ESAs) on Survival in ESRD: Using Payment Policy Change Shocks vs. Multiple Imputation using Propensity Score Models
  • Vincent Mor, PhD (Brown University)
    “Implementing Randomized Effectiveness Trials in Large Insurance Systems”
    Niteesh Choudhry, MD, PhD (Brigham & Women’s Hospital)
    “Comparative Effectiveness of Intravenous Iron Formulations in End-Stage Renal Disease”
    M. Alan Brookhart, PhD (University of North Carolina at Chapel Hill)
    Session Q&A
    Moderated by: Robert Glynn, ScD, PhD (Brigham & Women’s Hospital)

Video 4: http://www.youtube.com/watch?v=QbXWH1LRspw

    • SESSION II. CER IN DIVERSE POPULATIONS
      One of the reasons for differences between efficacy and effectiveness is differences in populations studied and their responses to treatment.  This treatment effect heterogeneity can arise from true biologic/genetic heterogeneity, differences in treatment adherence in different populations, differences in access to healthcare services (disparities), and patient complexity.
    • Comparative Effectiveness of Treatments for Depression: Research Strategies to Compare the ‘Almost Incomparable’?
      Michael E. Thase, MD (University of Pennsylvania Perelman School of Medicine)
      “CER in Schizophrenia: The Example of Relapse Prevention”
      John Kane, MD (Hofstra North Shore LIJ School of Medicine/ Zucker Hillside Hospital)
      “Clinical Trials and CER on Mental Health”
      Carlos Blanco, MD (Columbia University College of Physicians and Surgeons)
       “The Back to Health Trials: Comparative Effectiveness Research of Yoga for Chronic Low Back Pain in Predominantly Minority Populations”
      Ama R. Boah, MS (Boston Medical Center/Boston University School of Medicine)
      Session Q&A
      Moderated by: Tobias Gerhard, PhD (Rutgers University)

 

  • CLOSING ADDRESS
    Carolyn M. Clancy, MD
    Director, Agency for Healthcare Research and Quality (AHRQ)

——————————————————————————————————————————–
DAY 2 – WEDNESDAY, JUNE 13, 2012
——————————————————————————————————————————–

Video 5: http://www.youtube.com/watch?v=I1Ze_z3tmhI

    • SYMPOSIUM DAY 2 INTRODUCTIONS
      Sebastian Schneeweiss, MD, ScD
      Vice Chief, Pharmacoepidemiology, Brigham and Women’s Hospital/Harvard Medical School

 

    • OPENING ADDRESS
      “Comparative Effectiveness Research: Just Add PROs?”
      Albert Wu, MD, MPH
      Professor, Johns Hopkins Bloomberg School of Public Health
    • SESSION III. METHODOLOGICAL ISSUES IN CER ARISING FROM REGIONAL VARIATIONS IN SETTINGS OF CARE & IMPLICATIONS FOR CER

 

  • Among the reasons behind heterogeneity are those that relate to the characteristics of the healthcare setting which may have substantial regional variation.
  • “The deleterious effects of matching on provider in CER”
    Alexander M. Walker, MD, DrPH (WHISCON)
    “Exploring a Network of Treatments with Direct and Indirect Comparisons”
    Christopher H. Schmid, PhD (Brown University)
    “Simulating changes to specialty hospital access in comparing system effectiveness”

  • Video 6: http://www.youtube.com/watch?v=SmqQFQOF3b4

  • Charles C. Branas, PhD (University of Pennsylvania Perelman School ofMedicine)
    “Bias in case-crossover studies due to accumulation/disequilibrium of therapies for chronic diseases”
    Jason Roy, PhD (University of Pennsylvania Perelman School of Medicine)
    Session Q&A
    Moderated by: Bruce Fireman, MA (Kaiser Permanente)

Video 7: http://www.youtube.com/watch?v=W2zf0LuJMOs

    • SESSION IV-PART I. COMPARISONS INVOLVING DIVERSE HEALTHCARE INTERVENTION
      Are the efficacy-effectiveness issues different for different treatment modalities and how would we best study the comparison of different treatment modalities? (e.g. vaccines, devices, surgery, diagnostic/imaging, behavioral interventions)

“Targeted Maximum Likelihood Estimation in Safety Analysis”
Mark van der Laan, PhD (UC Berkley)
“Using balance measures to select propensity score methods for comparative effectiveness research”
Elizabeth Stuart , PhD (Johns Hopkins Bloomberg School of Public Health)
“Using Local Area Practice Style Measures as Instruments:  Does the Definition of “Local” Matter?”
John Brooks, PhD (University of Iowa)
Session Q&A
Moderated by: Alan Ellis, PhD (University of North Carolina at Chapel Hill)

Video 8:http://www.youtube.com/watch?v=oVwTStLwyUE

    • SESSION IV-PART II. COMPARISONS INVOLVING DIVERSE HEALTHCARE INTERVENTION
    • “Design of the SCOAP CERTAIN Intermittent Claudication Study: A Case Study”
      Beth Devine, PharmD, MBA, PhD (University of Washington)
      “Immortal Person Time Bias in Comparative Effectiveness Research for Cardiovascular Devices”
      Soko Setoguchi, MD, DrPH (Duke University School of Medicine)
      “Exploring the use of validation sample methods to reduce confounding bias in health care database estimates of influenza vaccination effectiveness in seniors”
      Jennifer Nelson, PhD (Group Health / University of Washington)
      “N-of-1 Trials and Evidence Farming: Community-Based Paradigms for Comparative Effective Research”Video 9:http://www.youtube.com/watch?v=SmqQFQOF3b4Naihua Duan, PhD (Columbia University Medical Center)
      “A Bayesian Adaptive Trial for CER: Case Study in Status Epilepticus”
      Jason Connor, PhD (Berry Consultants)
      Session Q&A
      Moderated by: John Seeger, PharmD, DrPH (Brigham & Women’s Hospital)

 

    • CLOSING ADDRESS
      Sebastian Schneeweiss, MD, ScD (Brigham and Women’s Hospital)
      Scott R. Smith, PhD (Agency for Healthcare Research & Quality)