Health Outcomes in Cerebrovascular Disease Treatment Studies

CMS evidence review | evidence_type=MEDCAC Meeting | record_type=medcac_meeting | document_id=78 | meeting_date=2021-09-22

On September 22, 2021, 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 should be of interest to CMS in studies for cerebrovascular disease treatment with a focus on new technologies. Given the increased emphasis on new and innovative medical products for treating diseases that have few proven therapies, studies of cerebrovascular disease treatment technologies submitted through the Investigational Device Exemption (IDE) pathway have focused on shorter term data with greater reliance upon intermediate and surrogate outcomes. Although such foci may help in answering Food and Drug Administration (FDA) questions on safety and efficacy, they are generally less helpful for CMS whose focus is on health outcomes associated with the technologies. As a result, there are more frequent evidence gaps with respect to the clinically meaningful health outcomes for CMS beneficiaries in assessments of these kinds of medical technologies. The MEDCAC panel will examine the growing challenges associated with these gaps in the evidence used to support requests for CMS coverage of new and innovative cerebrovascular disease treatment technologies. By voting on specific questions, and through their discussions, MEDCAC panel members will advise CMS about the ideal health outcomes in research studies of cerebrovascular disease treatment technologies, appropriate measurement instruments and follow-up durations to help to provide clarity and transparency of IDE analyses and National Coverage Analyses (NCAs). MEDCAC panels do not make coverage determinations, but CMS benefits from their advice. Although there is general agreement on the importance of using mortality as an outcome measure in cerebrovascular disease clinical research, there is little or no consensus on which other outcomes measures to include, such as stroke status and recurrence, hospitalization and healthcare resource utilization, clinician-reported patient functioning, as well as patient-reported outcome measures (PROMs). CMS is seeking input on whether, how, and when to include these additional outcomes.

Actions Taken: August 9, 2021 CMS announces MEDCAC meeting and posts questions to panel. September 16, 2021 Posted meeting materials: agenda , roster , speakers list , presentations and written comments . September 30, 2021 Posted scoresheet from meeting. November 2, 2021 Posted meeting minutes and transcript . Posted link to Zoom recording . Registration Agenda: Federal Register Notice Medicare Evidence Development & Coverage Advisory Committee September 22, 2021 8:00 AM - 4:30 PM Virtual Meeting Peter Bach, MD , Committee Chair Joseph Ross, MD , Committee Vice-Chair Joseph Chin, MD , Deputy Director, Coverage and Analysis Group Tara Hall , MEDCAC Coordinator 8:00 - 8:15 AM Opening Remarks - Tara Hall/ Joseph Chin, MD /Peter Bach, MD 8:15 - 8:25 AM CMS Presentation - Andrew Ward, PhD - Director, Evidence Development Division 8:25 - 8:50 AM Walter J. Koroshetz, MD - National Institutes of Health/National Institute of Neurological Disorders and Stroke 8:50 - 9:15AM Jeffrey L. Saver, MD - SA Vice-Chair for Clinical Research and Professor Department of Neurology David Geffen School of Medicine at UCLA 9:15 - 10:05 AM Sameer A. Ansari, MD - Professor of Radiology, Neurology, and Neurological Surgery. Director, Neuroendovascular Research and Quality Northwestern University, Feinberg School of Medicine Co-Chair. Adnan H. Siddiqui, MD - Chair Joint Cerebrovascular Section of the AANS & CNS. Secretary Society of NeurointerventionalSurgery. 10:05 - 10:20 AM Break 10:20 - 11:15 AM Scheduled Public Comments (Refer to Speaker List) ************************************************************************************ Public attendees, who have contacted the MEDCAC Coordinator 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. ************************************************************************************ 11:15 - 11:45 PM Open Public Comments Public Attendees who wish to address the panel will be given that opportunity 11:45 - 12:45 PM LUNCH (on your own) 12:45 - 1:45 PM Questions to Presenters 1:45 - 2:45 PM Initial Open Panel Discussion: Dr. Bach 2:45 - 3:00 Break 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. Bach 4:20 - 4:30 PM Closing Remarks/Adjournment: Drs. Chin and Bach Issue: On September 22, 2021, 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 should be of interest to CMS in studies for cerebrovascular disease treatment with a focus on new technologies. Given the increased emphasis on new and innovative medical products for treating diseases that have few proven therapies, studies of cerebrovascular disease treatment technologies submitted through the Investigational Device Exemption (IDE) pathway have focused on shorter term data with greater reliance upon intermediate and surrogate outcomes. Although such foci may help in answering Food and Drug Administration (FDA) questions on safety and efficacy, they are generally less helpful for CMS whose focus is on health outcomes associated with the technologies. As a result, there are more frequent evidence gaps with respect to the clinically meaningful health outcomes for CMS beneficiaries in assessments of these kinds of medical technologies. The MEDCAC panel will examine the growing challenges associated with these gaps in the evidence used to support requests for CMS coverage of new and innovative cerebrovascular disease treatment technologies. By voting on specific questions, and through their discussions, MEDCAC panel members will advise CMS about the ideal health outcomes in research studies of cerebrovascular disease treatment technologies, appropriate measurement instruments and follow-up durations to help to provide clarity and transparency of IDE analyses and National Coverage Analyses (NCAs). MEDCAC panels do not make coverage determinations, but CMS benefits from their advice. Although there is general agreement on the importance of using mortality as an outcome measure in cerebrovascular disease clinical research, there is little or no consensus on which other outcomes measures to include, such as stroke status and recurrence, hospitalization and healthcare resource utilization, clinician-reported patient functioning, as well as patient-reported outcome measures (PROMs). CMS is seeking input on whether, how, and when to include these additional outcomes. Minutes: Download meeting minutes . Download transcript from meeting. Other Material: Transcript 1 Panel Voting Questions: Download scoresheet 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 each of the following are standalone, meaningful primary health outcomes in research studies of cerebrovascular disease treatment technologies: Major disabling stroke: defined as stroke in the treated vascular territory that results in a Modified Rankin Scale (mRS) ≥ 3; Decrease in mRS of ≥ 2 points compared to baseline; mRS of ≤ 2 or equal to pre-stroke mRS (if the pre-stroke mRS was > 2); Other kinds of stroke, such as major ipsilateral stroke or morbid stroke. Discussion: For each health outcome with greater than or equal to intermediate confidence (≥ 2.5), please discuss the appropriate length of follow-up post intervention for assessing this outcome; For each health outcome with greater than or equal to intermediate confidence (≥ 2.5), please discuss the appropriate cutoff points of mRS and NIHSS for assessing this outcome; Please discuss important considerations when using composite outcomes in research studies of cerebrovascular disease treatment technologies, which may include the combination of mortality, stroke, hospitalization/hospitalization equivalent events, and neurologic functional evaluations. How confident are you that each of the following are standalone, meaningful primary health outcomes in research studies of cerebrovascular disease treatment technologies: Hospitalization length of stay for index procedure; Number of unscheduled re-admissions that are related to cerebrovascular disease; Discharge disposition to rehabilitation (home vs. inpatient facility). Discussion: For each health outcome with greater than or equal to intermediate confidence (≥ 2.5), please discuss the appropriate length of follow-up post intervention for assessing this outcome (applies to “b.” only); Please discuss important considerations when assessing the merits of composite outcomes in research studies of cerebrovascular disease treatment technologies, which include the combination of mortality, stroke, healthcare resource utilization for index procedure, post-procedure and re-hospitalizations, and neurologic functional evaluation. How confident are you that each of the following functional assessments are standalone, meaningful primary health outcome measures in clinical research studies of cerebrovascular disease treatment technologies: The Modified Rankin Scale (mRS); The National Institutes of Health Stroke Scale (NIHSS). Discussion: For each health outcome with greater than or equal to intermediate confidence (≥ 2.5), please discuss the appropriate length of follow-up post intervention for assessing this outcome; For each health outcome with greater than or equal to intermediate confidence (≥ 2.5), please discuss the appropriate cutoff points for assessing this outcome; Please discuss important considerations when assessing the merits of composite outcomes in research studies of cerebrovascular disease treatment technologies, which include the combination of mortality, stroke, hospitalization/ hospitalization equivalent events, and neurologic functional evaluation; Are there any other functional assessments (e.g. the Barthel Index (BI), the Fugl-Meyer (FM) Upper and Lower Extremity scales) that we have not discussed whose use you believe would result in important information pertaining to meaningful primary health outcomes in clinical research studies of cerebrovascular disease treatment technologies? How confident are you that using EQ-5D to measure quality of life: Is adequate measure which reflects the patient experience in the context of cerebrovascular disease studies; Should be included as standalone, meaningful primary health outcome measure in research studies; Should be included as part of a composite, meaningful primary health outcome measure in research studies; Should be included as secondary health outcome measure in research studies. Discussion: Please discuss whether additional patient-reported measurement [e.g., Short Form-36 (SF-36), Stroke Impact Scale-16 (SIS-16)] should be considered to capture quality of life and burdens associated with the cerebrovascular disease treatment under study; Please discuss the minimal clinically important differences (MCIDs) for the instruments; Please discuss the appropriate length of follow-up post intervention for assessing patient-reported measurements. Roster: Committee Chair: Peter Bach, MD, MAPP Attending Physician & Director Center for Health Policy and Outcomes Memorial Sloan-Kettering Cancer Center Committee Vice Chair: Joseph Ross, MD, MHS Associate Professor Medicine and Public Health Section of General Internal Medicine Department of Medicine Yale University School of Medicine MEDCAC Members: Cecelia C. Brewington, MD Professor Radiology UT Southwestern Medical Center Michael P. Cinquegrani, MD Professor Department of Medicine & Radiology Division of Cardiovascular Medicine Director of Heart and Vascular Service Line The Medical College of Wisconsin Ella Annabelle Effat Kazerooni, MD, MS Professor Associate Chair for Clinical Affairs Director, Cardiothoracic Radiology Division Department of Radiology University of Michigan Health System Stephen Lahey, MD Chief Division of Cardiothoracic Surgery Chief (Interim) Division of Vascular Surgery University of Connecticut Health Center Brian J. Miller, MD, MBA, MPH Assistant Professor of Medicine Johns Hopkins University School of Medicine Alan Speir, MD Medical Director Cardiac Surgical Services Inova Health System Director Inova Center for Outcomes Research and Evaluation (ICORE) Cardiac Vascular and Thoracic Surgery Associates Allison Stephens Statewide System of Care Director Nevada PEP Sam Tyagi, MD Assistant Professor of Surgery University of Kentucky College of Medicine Georgetown University Division of General Surgery Gregory Thomas, MD, MPH, FACC, MASNC Medical Director Cardiovascular Program Development MemorialCare Heart & Vascular Institute (MHVI) MemorialCare Health System Steven Waldren, MD, MS Vice President and Chief Medical Informatics Officer American Academy of Family Physicians Guest Panel Members Claudette Brooks, MD Neurological & Physical Medicine Devices Office (OHT5) Invited Guest Speakers Sameer A. Ansari, M.D., Ph.D Professor of Radiology, Neurology, and Neurological Surgery Director, Neuroendovascular Research and Quality Northwestern University, Feinberg School of Medicine Co-Chair, Governing Council Devices Used for Acute Ischemic Stroke Intervention (DAISI) Coordinated Registry Network (CRN) Food and Drug Administration (FDA) Walter Koroshetz, MD National Institutes of Health/National Institute of Neurological Disorders and Stroke Jeffrey L. Saver, MD SA Vice-Chair for Clinical Research and Professor Department of NeurologyDavid Geffen School of Medicine at UCLA Adnan H. Siddiqui, MD, PhD, FACS, FAHA Chair Joint Cerebrovascular Section of the AANS & CNS Secretary Society of Neurointerventional Surgery Professor & Vice Chairman Director Neuroendovascular Fellowship & Research Department of Neurosurgery, Director Canon Stroke and Vascular Research Center SUNY University at Buffalo CMS Liaison Joseph Chin, MD Deputy Director Coverage and Analysis Group MEDCAC Coordinator Tara Hall Coverage and Analysis Group Speakers List: Medicare Evidence Development & Coverage Advisory Committee September 22, 2021 SPEAKER LIST *6 MINUTES PER SPEAKER* Michael Chen, MD , The Society of NeuroInterventional Surgery (SNIS) Joshua Hirsch, MD , The American College of Radiology (ACR) Mahesh Jayaraman, MD , The Society of NeuroInterventional Surgery James Milburn, MD, The American Society of Neuroradiology (ASNR) Clemens Schirmer, MD, American Association of Neurological Surgeons (AANS), The Congress of Neurological Surgeons (CNS) Irene Katzan MD, Neurological Institute, Cleveland Clinic Lourdes R. Carhuapoma, MS, ACNP-BC, Division of Neurosciences Critical Care, Johns Hopkins Hospital, School of Nursing, University of Virginia Noeleen Ostapkovich, MS, Division of Brain Injury Outcomes, Johns Hopkins University Daniel F. Hanley, MD, Division of Brain Injury Outcomes, Johns Hopkins University