Health Outcomes in Heart Failure Treatment Technology Studies

CMS evidence review | evidence_type=MEDCAC Meeting | record_type=medcac_meeting | document_id=73 | meeting_date=2017-03-22

On March 22, 2017, the Centers for Medicare & Medicaid Services (CMS) will convene a panel of the Medicare Evidence Development and Coverage Advisory Committee (MEDCAC). The MEDCAC panel will examine what health outcomes in studies for heart failure treatment technologies should be of interest to CMS. Given the increased focus on the need of patients for new and innovative medical products, medical technologies are receiving market authorization based on less long-term data with greater reliance upon intermediate and surrogate outcomes. As a result, assessments of medical technologies are being made with more frequent evidence gaps with respect to the clinically meaningful health outcomes for CMS beneficiaries. The MEDCAC panel will examine the growing challenges associated with the decreased level of evidence generated prior to market authorization of new and innovative technology. By voting on specific questions, and by their discussions, MEDCAC panel members will advise CMS about the ideal health outcomes in research studies of heart failure treatment technologies and appropriate follow-up duration to ensure transparency of National Coverage Analyses (NCA) and requirements under Coverage with Evidence Development (CED). MEDCAC panels do not make coverage determinations, but CMS benefits from their advice.

Actions Taken: January 18, 2017 CMS posts MEDCAC meeting announcement. January 23, 2017 Posted questions to MEDCAC panel March 16, 2017 Posted agenda , roster and speakers list for meeting. Also posted presentations and written comments . March 21, 2017 Additional materials sent to panel: Utility of Patient-Reported Outcome Instruments in Heart Failure. Kelkar AA, Spertus J, Pang P, Pierson RF, Cody RJ, Pina IL, Hernandez A, Butler J. JACC Heart Fail. 2016 Mar;4(3):165-75. doi: 10.1016/j.jchf.2015.10.015. Review. PMID: 26874386 https://www.ncbi.nlm.nih.gov/pubmed/26874386 Cardiovascular health: the importance of measuring patient-reported health status: a scientific statement from the American Heart Association. Rumsfeld JS, Alexander KP, Goff DC Jr, Graham MM, Ho PM, Masoudi FA, Moser DK, Roger VL, Slaughter MS, Smolderen KG, Spertus JA, Sullivan MD, Treat-Jacobson D, Zerwic JJ; American Heart Association Council on Quality of Care and Outcomes Research, Council on Cardiovascular and Stroke Nursing, Council on Epidemiology and Prevention, Council on Peripheral Vascular Disease, and Stroke Council. Circulation. 2013 Jun 4;127(22):2233-49. doi: 10.1161/CIR.0b013e3182949a2e https://www.ncbi.nlm.nih.gov/pubmed/23648778 Two articles recently accepted and in press at the Journal of the American College of Cardiology: http://www.onlinejacc.org/content/early/recent Reduction in Heart Failure Hospitalizations With Ambulatory Hemodynamic Monitoring Seen in Clinical Trials Is Maintained in the 'Real World'. Akshay S. Desai, Arvind Bhimaraj, Rupinder Bharmi, Rita Jermyn, Kunjan Bhatt, David Shavelle, Margaret M. Redfield, Robert Hull, Jamie A. Pelzel, Kevin Davis, Nirav Dalal, Philip B. Adamson, J. Thomas Heywood. Real World Data on Heart Failure Readmission Reduction: Real or Real Uncertain? Harlan M. Krumholz, Sanket S. Dhruva. March 27, 2017 Posted scoresheet from meeting. May 25, 2017 Posted minutes and transcript from meeting. Webcast of meeting Registration Information about visiting CMS Driving directions to CMS Recommended hotels Agenda: Federal Register Notice Agenda Medicare Evidence Development & Coverage Advisory Committee March 22, 2017 7:30 AM - 4:30 PM CMS Auditorium Rita Redberg, MD, Committee Chair Alan Hirsch , MD, Acting Committee Vice Chair Joseph Chin, MD, Deputy Director, Coverage and Analysis Group Maria Ellis , Executive Secretary 7:30 - 8:00 AM Registration 8:00 - 8:15 AM Opening Remarks - Maria Ellis/Joseph Chin, MD/Rita Redberg, MD 8:15 - 8:35 AM CMS Presentation & Voting Questions - Daniel Caños, PhD, MPH 8:35 - 9:05 AM Ileana L. Piña, MD, MPH, Professor of Medicine, Epidemiology and Population Health, Albert Einstein College of Medicine, Associate Chief, Academic Affairs Cardiology, Montefiore Medical Center 9:05 - 9:35 AM Philip B. Adamson, MD, MSc, FACC, Vice President of Medical Affairs and Medical Director at Abbott formerly St. Jude Medical - Representing: AdvaMed 9:35 - 10:05 AM John D. Carroll, MD, Professor of Medicine, University of Colorado School of Medicine, Director, Interventional Cardiology, University of Colorado Hospital 10:05 - 10:35 AM William Lawrence, MD, Associate Director, Clinical Effectiveness and Decision Science, Patient-Centered Outcomes Research Institute and Larry A. Allen, MD, MHS, Associate Professor of Medicine, Medical Director, Advanced Heart Failure, University of Colorado Denver 10:35 - 10:45 AM BREAK 10:45 - 11:45 AM Scheduled Public Comments (Refer to Speaker List) ******************************************************************************************* Public attendees, who have contacted the executive secretary prior to the meeting, will address the panel and present information relevant to the agenda. Speakers are asked to state whether or not they have any financial involvement with manufacturers of any products being discussed or with their competitors and who funded their travel to this meeting. ******************************************************************************************* 11:45 - 12:00 AM Open Public Comments Public Attendees who wish to address the panel will be given that opportunity 12:00 - 1:00 PM LUNCH (on your own) 1:00 - 2:00 PM Questions to Presenters 2:00 - 3:00 PM Initial Open Panel Discussion: Dr. Redberg 3:00 - 4:00 PM Formal Remarks and Voting Questions The Chairperson will ask each panel member to state his or her position on the voting questions. 4:00 - 4:20 PM Final Open Panel Discussion: Dr. Redberg 4:20 - 4:30 PM Closing Remarks/Adjournment: Dr. Chin & Dr. Redberg Issue: On March 22, 2017, the Centers for Medicare & Medicaid Services (CMS) will convene a panel of the Medicare Evidence Development and Coverage Advisory Committee (MEDCAC). The MEDCAC panel will examine what health outcomes in studies for heart failure treatment technologies should be of interest to CMS. Given the increased focus on the need of patients for new and innovative medical products, medical technologies are receiving market authorization based on less long-term data with greater reliance upon intermediate and surrogate outcomes. As a result, assessments of medical technologies are being made with more frequent evidence gaps with respect to the clinically meaningful health outcomes for CMS beneficiaries. The MEDCAC panel will examine the growing challenges associated with the decreased level of evidence generated prior to market authorization of new and innovative technology. By voting on specific questions, and by their discussions, MEDCAC panel members will advise CMS about the ideal health outcomes in research studies of heart failure treatment technologies and appropriate follow-up duration to ensure transparency of National Coverage Analyses (NCA) and requirements under Coverage with Evidence Development (CED). MEDCAC panels do not make coverage determinations, but CMS benefits from their advice. Minutes: Download minutes Other Material: Transcript 1 Panel Voting Questions: Medicare Evidence Development & Coverage Advisory Committee (MEDCAC) Health Outcomes in Heart Failure Treatment Technology Studies March 22, 2017 On March 22, 2017, the Centers for Medicare & Medicaid Services (CMS) will convene a panel of the Medicare Evidence Development and Coverage Advisory Committee (MEDCAC). The MEDCAC panel will examine what health outcomes in studies for heart failure treatment technologies should be of interest to CMS. Given the increased focus on the need of patients for new and innovative medical products, medical technologies are receiving market authorization based on less long-term data with greater reliance upon intermediate and surrogate outcomes. As a result, assessments of medical technologies are being made with more frequent evidence gaps with respect to the clinically meaningful health outcomes for CMS beneficiaries. The MEDCAC panel will examine the growing challenges associated with the decreased level of evidence generated prior to market authorization of new and innovative technology. By voting on specific questions, and by their discussions, MEDCAC panel members will advise CMS about the ideal health outcomes in research studies of heart failure treatment technologies and appropriate follow-up duration to ensure transparency of National Coverage Analyses (NCA) and requirements under Coverage with Evidence Development (CED). MEDCAC panels do not make coverage determinations, but CMS benefits from their advice. Voting Questions For each voting question, please use the following scale identifying your level of confidence - with a score of 1 being low or no confidence and 5 representing high confidence. 1 — 2 — 3 — 4 — 5 Low Intermediate High Confidence Confidence * How confident are you that the following are standalone, meaningful primary health outcomes in research studies of heart failure treatment technologies: Heart failure hospitalization; Heart failure hospitalization or heart failure hospitalization equivalent events (i.e., outpatient IV therapy for heart failure); Total Hospitalizations? Discussion: For each health outcome with greater than or equal to intermediate confidence (≥ 2.5), please discuss the appropriate length of follow-up post-heart failure intervention for assessing this outcome; Please discuss important considerations when assessing the merits of composite outcomes in research studies of heart failure treatment technologies which include the combination of mortality, heart failure hospitalization, or heart failure hospitalization equivalent events. How confident are you that surrogate and intermediate endpoints are predictive of standalone, meaningful primary health outcomes (e.g., reduction in mitral regurgitation, cardiac remodeling, ejection fraction, or biomarkers) in clinical research studies of heart failure treatment technologies for: Heart failure with preserved ejection fraction; Heart failure secondary to mitral regurgitation where the focus of therapy is mitral valve repair/ replacement; Heart failure with reduced ejection fraction (e.g., cardiac remodeling, ejection fraction)? Discussion: If greater than or equal to intermediate confidence (≥ 2.5), please identify the specific surrogate or intermediate endpoints and associated disease or therapy which you believe are sufficiently predictive of meaningful health outcomes. Please discuss how these intermediate and surrogate endpoints meaningfully contribute towards the evidence base for heart failure treatment technologies. Please discuss important factors to consider when assessing the utility of surrogate and intermediate endpoints. How confident are you that quality of life measures [e.g., Kansas City Cardiomyopathy Questionnaire (KCCQ), Minnesota Living With Heart Failure Questionnaire (MLWHFQ): Are adequate measures which reflect the patient experience; Should be included as the standalone, meaningful primary health outcomes in research studies; Should be included as a composite standalone, meaningful primary health outcomes in research studies? How confident are you that functional assessments [e.g., 6 min walk test (6MWT), VO2max, ventilator threshold]: Are adequate measures which reflect the patient experience; Should be included as the standalone, meaningful primary health outcomes in research studies; Should be included as a composite standalone, meaningful primary health outcomes in research studies? Discussion: Please discuss whether additional patient-reported measurement [e.g., Short Form-36 (SF-36), EuroQol five dimensions questionnaire (EQ5D)] should be considered to capture burdens associated with the heart failure therapy under study. Please discuss the appropriate length of follow-up post-heart failure intervention for assessing patient-reported measurements. For some studies of heart failure treatment technologies it may not be practical for patients to be blinded. Please discuss the impact of unblinded study participants on patient-reported measurements and functional assessments. Please discuss how to best consider the impact of adverse events associated with heart failure technologies while balancing the potential for improvements to meaningful health outcomes. Please discuss how to balance the benefits and harms of therapies which may improve near-term patient-reported health outcome assessments or clinical measurements (e.g., 6 MWT or symptoms) but may decrease length of life. Additional Discussion Topics: Please discuss health outcomes of interest and appropriate follow-up duration in studies of technologies designed for diagnosis of acute decompensation of heart failure. With the health outcomes and information that we have discussed today, how confident are you that there will there be enough accurate information provided to patients for them to make informed decisions? Please discuss how studies can be designed to accurately capture patient preferences and how their preferences can best be considered and operationalized once the study has concluded. * CMS recognizes the importance of mortality as a meaningful primary health outcome of interest in research studies. We are seeking input on what additional outcomes should be considered. Roster: Committee Chair Rita Redberg, MD, MSC Professor of Medicine UCSF School of Medicine Division of Cardiology University of California, San Francisco Medical Center Acting Committee Vice Chair Alan Hirsch, MD Director Vascular Medicine Quality Outcomes and Population Health Professor of Medicine Epidemiology and Community Health Cardiovascular Division University of Minnesota Medical School MEDCAC Members Salvador Cruz-Flores, MD, MPH Professor and Founding Chairman Department of Neurology Paul L. Foster School of Medicine Texas Tech University Health Sciences Center Michael J. Fisch, MD, MPH, FACP National Medical Director Medical Oncology AIM Specialty Health Fred Kobylarz MD, MPH Associate Professor Geriatric Medicine Department of Family Medicine and Community Health Rutgers, The State University of New Jersey Marcel Salive, MD, MPH Medical Officer National Institute on Aging National Institute of Health Art Sedrakyan, MD, PhD Professor of Healthcare Policy and Research Director Patient-Centered Comparative Effectiveness MDEpiNet Science and Infrastructure Center Weill Cornell Medical College Cornell University Jodi B. Segal, MD, MPH Professor of Medicine Division of General Internal Medicine Department of Medicine Johns Hopkins University School of Medicine Julie Ann Swain, MD Vice Chair for Clinical Performance Director of Clinical Research Department of Cardiovascular Surgery Icahn School of Medicine at Mount Sinai New York City Director, Center for Medical Devices (CMeD) at Mount Sinai Heart Diana Zuckerman, PhD President National Center for Health Research Cancer Prevention and Treatment Fund Representatives Eileen Hsich, MD Associate Medical Director Cardiology Department, Heart Failure Heart Transplant Program Cleveland Clinic Lynne Warner Stevenson, MD Professor of Medicine Cardiovascular Division Brigham and Women's Hospital Industry Representative Adi Renbaum, MBA Principal/Owner ANR Consulting, LLC Guest Panel Members Elise Berliner, PhD Director Technology Assessment Program Center for Outcomes and Evidence Agency for Healthcare Research and Quality Patrice Desvigne-Nickens, MD Medical Officer Heart Failure and Arrhythmias Branch Division of Cardiovascular Sciences National Heart, Lung, and Blood Institute Clyde W. Yancy, MD, MSc, MACC, FAHA Vice Dean, Diversity & Inclusion Magerstadt Professor of Medicine Professor of Medical Social Sciences Chief, Division of Cardiology Northwestern University, Feinberg School of Medicine Associate Director Bluhm Cardiovascular Institute Northwestern Memorial Hospital Bram Zuckerman, MD Director FDA Division of Cardiovascular Devices Office of Device Evaluation Center for Devices and Radiological Health Invited Guest Speakers Philip B. Adamson, MD, MSc, FACC Medical Director and Vice President Global Research and Development Abbott Larry A. Allen, MD, MHS Associate Professor of Medicine Medical Director Advanced Heart Failure John D. Carroll, MD Professor of Medicine University of Colorado School of Medicine Director, Interventional Cardiology University of Colorado Hospital William Lawrence, MD Associate Director Clinical Effectiveness and Decision Science Patient-Centered Outcomes Research Institute Ileana L. Piña, MD, MPH Professor of Medicine Epidemiology and Population Health Albert Einstein College of Medicine Associate Chief Academic Affairs Cardiology Montefiore Medical Center CMS Liaison Joseph Chin, MD Deputy Director Coverage and Analysis Group Executive Secretary Maria Ellis Coverage and Analysis Group Speakers List: Medicare Evidence Development & Coverage Advisory Committee March 22, 2017 SPEAKER LIST *7 MINUTES PER SPEAKER* Clinton A. Brawner, PhD , Clinical Exercise Physiologist, Division of Cardiovascular Medicine, Henry Ford Hospital – Representing: The Cardiovascular Research Foundation Biykem Bozkurt, MD, PhD, FACC, FAHA , The Mary and Gordon Cain Chair & W.A. “Tex” and Deborah Moncrief, Jr., Chair, Professor of Medicine, Medicine Chief, DeBakey VA Medical Center, Director of Winters Center for HF Research, Associate Director, Cardiovascular Research Institute, Director of Winters Center for Heart Failure, Baylor College of Medicine, Houston, TX – Representing: American College of Cardiology Srihari S. Naidu, MD, FACP, FACC, FAHA, FSCAI , Director, Hypertrophic Cardiomyopathy Program & Interventional Cardiologist, Westchester Medical Center, Associate Professor of Medicine, New York Medical College, Past Trustee, Society for Cardiovascular Angiography & Interventions (SCAI), Past President, Brown University Medical School Board of Directors, Trustee, Brown University Corporation - Representing: Society for Cardiovascular Angiography & Interventions Nancy Sweitzer, MD , Sarver Heart Center Director, University of Arizona – Representing: American Heart Association Christopher M. O’Connor, MD, FACC, CEO and Executive Director, Inova Heart and Vascular Institute, Professor of Medicine (adj.) Duke University, Editor in Chief, JACC: Heart Failure, President-Elect, Heart Failure Society of America – Representing: Heart Failure Society of America Eran Kurman , Vice President of Sales and Marketing, Sensible Medical Innovations Due to unforeseen circumstances, Mr. Kurman is unable to attend the meeting. Dan Schaber, PharmD, Vice President, Heart Failure Clinical Research, Medtronic plc