NHLBI Workshop
Comparative Effectiveness Research in Lung Diseases Workshop

Executive Summary

Introduction

Recent congressionally mandated federal efforts to establish a “robust comparative effectiveness enterprise” have generated definitions, priorities, and organizational mechanisms for conducting CER. The National Institute of Health’s commitment to CER is reflected in its history of comparative clinical trials that have profoundly influenced clinical practice as well as its current involvement in shaping the CER enterprise. The NIH currently uses the Federal Coordinating Council definition of CER: “...the conduct and synthesis of research comparing the benefits and harms of different interventions and strategies to prevent, diagnose, treat, and monitor health conditions in “real world settings.” Operationalizing this definition to shape a specific research agenda poses numerous challenges, including defining where CER would best fill the gaps in clinical evidence for particular lung diseases or sleep disorders, understanding how to use innovative study designs, obtaining large sample sizes and data sets to examine subgroup variations in responses, using electronic health records and integrated data systems to increase efficiency and identifying the role for observational studies.

Workshop Description

The Division of Lung Diseases (DLD), National Heart, Lung, and Blood Institute convened a workshop in September 2010 to discuss the role of CER in DLD supported research and to make recommendations to help assure that CER research in lung diseases and sleep disorders meets the challenges and opportunities noted above. Participants included clinical investigators in asthma, COPD, ARDS, pediatric lung diseases, and sleep disorders, as well as methodologists with expertise relevant to CER. Before the workshop, participants completed a survey asking about priority topics for CER lung diseases overall and in their respective fields, the barriers to conducting CER, and the most important steps to advance CER in their fields. Results were used to provide a framework for the meeting agenda. At the workshop, participants were divided into subgroups to design a mock CER intervention trial and a mock CER observational study. This activity provided a practical consideration of the themes that emerged from the morning’s more general discussion of CER in lung disease. Group discussion of the mock trials and the key issues and concerns that emerged in their development led to the specific recommendations to advance CER in lung diseases presented in this report.

Recommendations

• Increase efforts to engage stakeholders in developing CER questions and study designs
• Invest in further development of databases and other infrastructure, including efficient methods for data sharing
• Make full use of a broad range of study designs
• Increase the appropriate use of observational designs and the support of methodologic research
• Ensure that committees that review CER grant applications include persons with appropriate perspective and expertise
• Further develop the workforce for CER by supporting training opportunities that focus on the methodologic and practical skills needed.

Workshop Roster

Chair

• Tracy Lieu, M.D., M.P.H., Harvard Medical School, Harvard Pilgrim Health Care Institute, Children's Hospital Boston
• Alfred Connors, M.D., Case Western Reserve University School of Medici

Participants

• David Au, M.D., M.S., VA Puget Sound Health Care System, University of Washington, Department of Veterans Affairs
• Jerry A. Krishnan, M.D., Ph.D., University of Chicago
• Marc Moss, M.D., University of Colorado School of Medicine
• Harry Selker, M.D., M.S.P.H., Tufts Medical Center Institute for Clinical Research and Health Policy Studies
• Andrea Apter, M.D., M.A., M.Sc.,University of Pennsylvania
• Thomas F. Boat, M.D., University of Cincinnati College of Medicine
• Noreen Clark, Ph.D., University of Michigan at Ann Arbor
• Dennis Drotar, PhD, Children’s Hospital Medical Center
• Bruce C. Marshall, M.D., Cystic Fibrosis Foundation
• Susan Redline, M.D., M.P.H., Brigham and Women’s Hospital
• Shelby D. Reed, Ph.D., Duke University
• Michael Schatz, M.D., M.S., Kaiser Permanente Medical Center
• Stephen B. Soumerai, Sc.D., Harvard Medical School and Harvard Pilgrim Health Care Institute
• B. Taylor Thompson, M.D., Massachusetts General Hospital
• Virginia Taggart, M.P.H., Division of Lung Diseases
• Carol Blaisdell, M.D., Division of Lung Diseases
• Andrea Harabin, Ph.D., Division of Lung Diseases
• Daniel Lewin, Ph.D., Division of Lung Diseases
• Antonello Punturieri, Ph.D., M.D., Division of Lung Diseases
• Robert Smith, Ph.D., Division of Lung Diseases

Last Updated June 2011