Devices for Self-management of Type 1 and Insulin-Dependent Type 2 Diabetes

CMS evidence review | evidence_type=MEDCAC Meeting | record_type=medcac_meeting | document_id=81 | meeting_date=2024-05-21

On May 21, 2024, 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 clinical endpoints should be of interest to CMS in studies of new devices for self-management of type 1 and insulin-dependent type 2 diabetes in older adults. The goal of the meeting will be to provide recommendations to CMS regarding a guidance document on this topic, to be issued under the Clinical Endpoints Guidance program. This need has arisen because the available devices for monitoring and controlling glucose levels have been studied and used primarily in individuals with type 1 diabetes, who historically have not been Medicare-age adults. Although type 2 diabetes is much more common than type 1 diabetes among older adults, better management of type I diabetes is leading to more prolonged survival among patients with type 1 diabetes and, thus, greater numbers of these individuals among the Medicare population. Furthermore, the prevalence of insulin-dependent type 2 diabetes, especially among older adults, is rising. Relevant clinical endpoints for assessing the effectiveness and safety of diabetes management devices may be unique for older adults. For example, the issue of hypotension as a complication of glucose control is a more significant issue among older compared with younger adults. Studies of devices submitted through the Food & Drug Administration’s Breakthrough Technology pathway have often focused on shorter term data with greater reliance upon intermediate and surrogate outcomes. Although these endpoints may help in answering FDA questions on safety and efficacy, they are generally less helpful for CMS whose focus is on the more ultimate health outcomes associated with the technologies, and particularly for older adults. As a result, there are more frequent evidence gaps in the research literature with respect to the clinically meaningful health outcomes for CMS beneficiaries in assessments of these kinds of medical interventions. For these reasons, CMS commissioned a Clinical Endpoints Review (CER) of the literature to identify endpoints that should be considered when designing studies of devices for self-management of type 1 or insulin-dependent type 2 diabetes. An endocrinologist with expertise in managing patients with diabetes contributed to the CER. The CER revealed little consensus on outcomes specific to older adults and found few definitions of minimally clinically important differences (MCIDs). The MEDCAC panel members will make use of the CER as well as their own professional expertise in considering recommendations for CMS. A subcommittee of the full MEDCAC panel reviewed the CER along with responses to the CER from a second external endocrinologist. The subcommittee then assisted CMS in formulating voting questions and a discussion guide for the full panel meeting. By voting on specific questions, and through discussion, MEDCAC panel members will advise CMS on these issues: 1) clinical endpoints considered important for older adults and suggested instruments for measuring those endpoints, 2) MCIDs, and 3) the duration of patient follow-up necessary to demonstrate effectiveness and safety, device durability, and patient adherence.

Actions Taken: March 26, 2024 Announced MEDCAC meeting and posted materials for meeting, including voting questions . Individuals who want to join the meeting may register online at: https://cms.zoomgov.com/meeting/register/vJItcu6hpj4qHL_IlNFkPTSJOCXDvu2IiGg until May 21, 2024, EDT at 10am. May 2, 2024 Posted materials for meeting. Agenda Roster Speakers list Presentations Comments May 30, 2024 Posted scoresheet and recording of meeting. June 13, 2024 Posted transcript from meeting. June 26, 2024 Posted minutes from meeting. 02/08/2024 - MEDCAC subcommittee (Devices for Self-management of Type 1 and Insulin-Dependent Type 2 Diabetes) Agenda: Federal Register Notice Agenda Medicare Evidence Development & Coverage Advisory Committee May 21, 2024 10:00 AM – 2:15PM Virtual Meeting Joseph Ross, MD , Committee Chair Dhruva Sanket, MD , Committee Vice-Chair Tamara Syrek Jensen, JD , Director, Coverage and Analysis Group Tara Hall , MEDCAC Coordinator 10:00 – 10:05 AM Opening Remarks – Tara Hall/ Tamara Syrek Jensen, JD /Joseph Ross, MD 10:05 – 10:10 AM Clinical Endpoints Guidance Program – Steven Farmer, MD, PhD 10:10 – 10:25 AM CMS Presentation: Clinical Endpoints Review (CER) – Teresa Rogstad, MPH 10:25 – 10:35 AM Summary of Subcommittee Deliberations; Review of Voting Questions and Discussion Guide : Dr. Ross 10:35 – 11:40 AM Scheduled Public Comments (Refer to Speaker List) 11:40 – 11:50 AM Break 11:50 – 12:35 PM Open Public Comments Public Attendees who wish to address the panel will be given that opportunity 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 they have any financial involvement with manufacturers of any products being discussed or with their competitors. 12:35 – 12:50 PM Open Panel Discussion of Domains: Dr. Ross 12:50 – 1:10 PM Voting Questions The Chairperson will ask each panel member to state his or her position on the voting questions. 1:10 – 2:10 PM Final Open Panel Discussion: Dr. Ross Discussion of Each Domain Regarding Specific Measures Minimal Clinically Important Differences (MCID) Follow-up Duration 2:10 – 2:15 PM Closing Remarks/Adjournment: Dr. Ross Background Materials: Overview of Clinical Endpoints Review Clinical Endpoints Review Clinical Endpoints Review, Executive Summary Issue: On May 21, 2024, 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 clinical endpoints should be of interest to CMS in studies of new devices for self-management of type 1 and insulin-dependent type 2 diabetes in older adults. The goal of the meeting will be to provide recommendations to CMS regarding a guidance document on this topic, to be issued under the Clinical Endpoints Guidance program. This need has arisen because the available devices for monitoring and controlling glucose levels have been studied and used primarily in individuals with type 1 diabetes, who historically have not been Medicare-age adults. Although type 2 diabetes is much more common than type 1 diabetes among older adults, better management of type I diabetes is leading to more prolonged survival among patients with type 1 diabetes and, thus, greater numbers of these individuals among the Medicare population. Furthermore, the prevalence of insulin-dependent type 2 diabetes, especially among older adults, is rising. Relevant clinical endpoints for assessing the effectiveness and safety of diabetes management devices may be unique for older adults. For example, the issue of hypotension as a complication of glucose control is a more significant issue among older compared with younger adults. Studies of devices submitted through the Food & Drug Administration’s Breakthrough Technology pathway have often focused on shorter term data with greater reliance upon intermediate and surrogate outcomes. Although these endpoints may help in answering FDA questions on safety and efficacy, they are generally less helpful for CMS whose focus is on the more ultimate health outcomes associated with the technologies, and particularly for older adults. As a result, there are more frequent evidence gaps in the research literature with respect to the clinically meaningful health outcomes for CMS beneficiaries in assessments of these kinds of medical interventions. For these reasons, CMS commissioned a Clinical Endpoints Review (CER) of the literature to identify endpoints that should be considered when designing studies of devices for self-management of type 1 or insulin-dependent type 2 diabetes. An endocrinologist with expertise in managing patients with diabetes contributed to the CER. The CER revealed little consensus on outcomes specific to older adults and found few definitions of minimally clinically important differences (MCIDs). The MEDCAC panel members will make use of the CER as well as their own professional expertise in considering recommendations for CMS. A subcommittee of the full MEDCAC panel reviewed the CER along with responses to the CER from a second external endocrinologist. The subcommittee then assisted CMS in formulating voting questions and a discussion guide for the full panel meeting. By voting on specific questions, and through discussion, MEDCAC panel members will advise CMS on these issues: 1) clinical endpoints considered important for older adults and suggested instruments for measuring those endpoints, 2) MCIDs, and 3) the duration of patient follow-up necessary to demonstrate effectiveness and safety, device durability, and patient adherence. Meeting Recording: https://cms.zoomgov.com/rec/share/7IiRIp1GfS8wVNjoIY6ALGYFZ6sorCEihTL54gWYj4RXzfLy0KWdCsh3rixJHUpD.l1K-8Av9HCBwq48q Passcode: C29bU1!w Minutes: Download meeting minutes Other Material: Transcript 1 Panel Roster: Committee Chair: Joseph Ross, MD, MHS Professor Medicine and Public Health Section of General Internal Medicine Department of Medicine Yale University School of Medicine Committee Vice Chair: Sanket Dhruva, MD, MHS, FACC Assistant Professor of Medicine UCSF School of Medicine Affiliate Faculty, Philip R. Lee Institute for Health Policy Studies Staff Physician (Cardiology), San Francisco VA Medical Center MEDCAC Members: Brian Isetts, RPh PhD, BS-Pharm Professor with Tenure Department of Pharmaceutical Care & Health Systems University of Minnesota College of Pharmacy Alexander Fanaroff, MD, MHS Assistant Professor of Medicine Division of Cardiology University of Pennsylvania Perelman Center for Advanced Medicine Melissa M. Garrido, PhD, BS Research Professor Department of Health Law, Policy & Management Boston University School of Public Health Director Partnered Evidence-Based Policy Resource Center (PEPReC) Boston VA Healthcare System Fred Kobylarz MD, MPH Associate Professor Geriatric Medicine Department of Family Medicine and Community Health Rutgers, The State University of New Jersey Joy H. Lewis, DO, PhD, FACP Professor Internal Medicine and Public Health A.T. Still University School of Osteopathic Medicine Eric Wall, MD, MPH Clinical Associate Professor of Family Medicine, University of Washington Heather Young Professor and Founding Dean Emerita of the Betty Irene Moore School of Nursing at the University of California Industry Representative Mark D. Carlson, MD, MA Chief Medical Officer Sr. Vice President for Clinical Affairs Covanos, Inc. Patient Advocate Naftali Z. Frankel, MS, MBEE Consumer & Patient Advocate Director of Precision Medicine Innovation, CMR Adjunct Professor, Johns Hopkins University Panel Voting Questions: Download scoresheet . DIABETES SELF-MANAGEMENT DEVICES MEDCAC MEETING (May 21, 2024) A Preview of the Voting Questions and Discussion Guide Following presentations of background information and a time for public comments, the Panel will be asked to provide feedback that CMS will use to draft a Clinical Endpoints Guidance document on this topic. To that end, the remainder of the Panel meeting will be structured as follows: An electronic poll to determine how Panelists rate the importance of 4 endpoint domains under which specific measures can be organized Discussion of each endpoint domain with respect to A prepared list of specific measures Appropriate duration of follow-up Guidance regarding minimal clinically important differences (MCIDs) Panelists may find it helpful to print this document, or plan to have it open on their laptops for reference during Panel discussions. Voting: Endpoint Domain Ratings The following endpoint domains have been identified through a literature review (the Clinical Endpoints Review document) and deliberations of a Panel Subcommittee. They represent the types of endpoints that should be addressed in the body of evidence regarding devices for self-management of type 1 or insulin-dependent type 2 diabetes in older adults . In the meeting, panelists will use rating scales, as shown, to indicate the importance they attach to each endpoint domain. Surrogate markers, i.e., indirect assessments using biomarkers, physiologic measures, or imaging intended to predict or act as a proxy for a target outcome of interest . For example, percentage of time in hypoglycemia. Not at all important Slightly important Somewhat important Very important Extremely important Health outcomes, i.e., direct assessments of a target outcome of interest. For example, complications of diabetes. Not at all important Slightly important Somewhat important Very important Extremely important Quality of life, i.e., patient-reported assessments of symptom burden or function. For example, the Diabetes Distress Scale. Not at all important Slightly important Somewhat important Very important Extremely important Device-related safety . For example, hypoglycemia-related emergency department visits. Not at all important Slightly important Somewhat important Very important Extremely important Discussion Please be prepared to discuss the following issues as they apply to each endpoint domain. The appropriateness of specific endpoint measures within each domain (listed in the Appendix , p. 3 of this document) . Ideal duration of follow-up required for detection of an impact on various endpoint measures. Any conventional or validated thresholds known to Panel members for defining a Minimally-Clinically Important Difference ( MCID) for the endpoints listed in the Appendix. Appendix: Specific Outcome Measures by Endpoint Domain Surrogate markers Number of hypoglycemic episodes (<70 mg/dL), especially episodes of Level 2 hypoglycemia (<54 mg/dL) Percentage of time in level 2 hypoglycemia (<54 mg/dL) Impact on A1C (MCID = 0.5% change)* Percentage of time in acceptable glucose range (70-180 mg/dL) Percentage of time in hyperglycemia (>180 mg/dL) Percentage of time in hypoglycemia (<70 mg/dL) *According to Endocrine Society practice guidelines, A1C should be used for glucose monitoring only on the basis of shared-decision making when patients are not in good health, i.e., they have ≥ 3 nondiabetes chronic illnesses, mild cognitive impairment, dementia (any stage), ≥ 2 ADL impairments, end-stage medical condition, or residence in a long-term nursing facility. The American Diabetes Association also cautions against using A1C to monitor patients in very poor health. Health outcomes Restoration of hypoglycemia awareness Cognitive function changes Diabetes-related emergency department visits Diabetes-related hospitalizations Complications of diabetes, e.g., kidney disease, major adverse cardiovascular events (MACE) Quality of life Audit of Diabetes-Dependent Quality of Life (ADDQoL) questionnaire Diabetes Distress Scale Diabetes Impact Measurement Scales (DIMS) Diabetes Treatment Satisfaction Questionnaire Hypoglycemia Fear Survey Problem Areas in Diabetes Device-related safety Hypoglycemia-related emergency department visits Harms such as tissue damage, if appropriate Device discontinuation rates Patient preferences (comparing the device with conventional self-management) and adherence Speaker List: Medicare Evidence Development & Coverage Advisory Committee May 21, 2024 SPEAKER LIST *5 MINUTES PER SPEAKER* Candace DeMatteis, Partnership to Fight Chronic Disease Susan Peschin, MHS , Alliance for Aging Research Aaron Turner-Phifer , JDRF Laura Friedman , American Diabetes Association Presentations: Jessica Castle, MD, Dexcom Gregory Forlenza, MD, Barbara Davis Center for Diabetes Davida Kruger, MSN , Henry Ford Health System Janet B. McGill, MD, Individual expert Laurel Messer, PhD , Tandem Diabetes Care, Inc. Medha Munshi, Duke-Robert J. Margolis, MD, Center for Health Policy Robert Alan Vigersky, MD, Medtronic Dr. Rifka Schulman-Rosenbaum, MD, Long Island Jewish Medical Center and Dr. Sethu Reddy, MD , CMU College of Medicine