Devices to Manage Tremor in Parkinson’s Disease and Essential Tremor

CMS evidence review | evidence_type=MEDCAC Meeting | record_type=medcac_meeting | document_id=82 | meeting_date=2025-06-25

On June 25, 2025, 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 devices for management of tremor in patients with Parkinson’s Disease or essential tremor. By voting on specific questions, and through discussion, MEDCAC panel members will advise CMS about the ideal clinical endpoints to measure in research studies on this topic, as well as appropriate measurement instruments, definitions of minimal clinically important differences and adequate follow-up duration. This need has arisen because Parkinson's and essential tremor are common among Medicare beneficiaries and because potential clinical endpoints are wide-ranging. Input from the MEDCAC panel will be used by CMS in future relevant National Coverage Analyses (NCAs) of new devices. Studies have often focused on shorter term data with greater reliance upon intermediate and surrogate outcomes. Although these endpoints may help in answering certain questions, 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 management of Parkinson’s Disease and essential tremor. The CER revealed many potential clinical endpoints that were used with varying frequency across the studies. Most of these clinical endpoints can be assessed with formal instruments. Definitions of minimal clinically important difference (MCID) could not be identified for some of the instruments, and useful guidance on follow-up duration was not available from the CER. A neurosurgeon and two neurologists reviewed the completed CER and submitted written responses to review questions. These three physicians also had special expertise in movement disorders. 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 from the three physician specialists who read the completed CER. 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: April 24, 2025 Announced MEDCAC meeting and posted questions to panel. June 9, 2025 Posted materials for meeting. Agenda Roster Speakers list Presentations Written comments July 16, 2025 Posted minutes , transcript , scoresheet and video recording of meeting. 03/26/2025 - MEDCAC subcommittee (Devices to Manage Tremor in Parkinson’s Disease and Essential Tremor) Agenda: Federal Register Notice Agenda Medicare Evidence Development & Coverage Advisory Committee June 25, 2025 10:00 AM – 2:00 PM 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 /Joe Ross, MD 10:05 – 10:10 AM Clinical Endpoint Review Program – Tamara Syrek Jensen, JD 10:10 – 10:25 AM CMS Presentation of Clinical Endpoints Review (CER): Devices to Manage Tremor in Parkinson’s Disease and Essential Tremor– Teresa Rogstad, MPH 10:25 – 10:35AM Summary of Subcommittee Deliberations; Review of Voting Questions and Discussion Guide: Dr. Ross 10:35 – 11:30 AM Scheduled Public Comments (Refer to Speaker List) 11:30 – 11:40 AM Break 11:40 – 12:20 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. ************************************************************************************ 12:20 – 12:35 PM Open Panel Discussion of Domains: Dr. Ross 12:35 – 12:55 PM Voting Questions (Clinical Endpoint Domains) The Chairperson will ask each panel member to state his or her position on the voting questions. 12:55 – 1:55 PM Final Open Panel Discussion: Dr. Ross Discussion of Each Domain Regarding Specific Measures Follow-up Duration Minimal Clinically Important Differences (MCID) 1:55 – 2:00 PM Closing Remarks/Adjournment: Dr. Ross Issue: On June 25, 2025, 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 devices for management of tremor in patients with Parkinson’s Disease or essential tremor. By voting on specific questions, and through discussion, MEDCAC panel members will advise CMS about the ideal clinical endpoints to measure in research studies on this topic, as well as appropriate measurement instruments, definitions of minimal clinically important differences and adequate follow-up duration. This need has arisen because Parkinson's and essential tremor are common among Medicare beneficiaries and because potential clinical endpoints are wide-ranging. Input from the MEDCAC panel will be used by CMS in future relevant National Coverage Analyses (NCAs) of new devices. Studies have often focused on shorter term data with greater reliance upon intermediate and surrogate outcomes. Although these endpoints may help in answering certain questions, 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 management of Parkinson’s Disease and essential tremor. The CER revealed many potential clinical endpoints that were used with varying frequency across the studies. Most of these clinical endpoints can be assessed with formal instruments. Definitions of minimal clinically important difference (MCID) could not be identified for some of the instruments, and useful guidance on follow-up duration was not available from the CER. A neurosurgeon and two neurologists reviewed the completed CER and submitted written responses to review questions. These three physicians also had special expertise in movement disorders. 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 from the three physician specialists who read the completed CER. 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. Minutes: Download meeting minutes . Other Material: Transcript 1 Presentations Written comments Movement Disorders Society United Parkinson’s Disease Rating Scale Video recording of meeting Panel Voting Questions: Download scoresheet . A Preview of the Voting Questions and Discussion Guide After presentations of background information and a time for public comments, the Panel will be asked to provide feedback that CMS will subsequently use to draft a technical report on this topic. To that end, the remainder of the Panel meeting after public comments will be structured as follows: An electronic poll to determine how Panelists rate the importance of 3 clinical endpoint domains under which important clinical endpoints have been organized. A brief statement from each Panelist regarding his or her vote. Open discussion of each endpoint domain. Panelists will be asked to very briefly: Identify the most important and perhaps least important clinical endpoints within the domain Provide guidance regarding minimal clinically important differences (MCIDs) where none have been identified in the literature Suggest an appropriate duration of follow-up when endpoints in these domains are used in clinical studies Panelists may find it helpful to print this document or plan to have it open on their laptops for reference during Panel discussions. VOTING The following endpoint domains and suggested clinical endpoints pertain to studies evaluating medical devices to manage tremor in older adults who have Parkinson’s Disease or essential tremor. These domains and endpoints have been identified through a systematic literature review (the Clinical Endpoints Review [CER} document). The review included input from an external neurologist. Completion of the CER document was followed by feedback from three other external expert reviewers (two neurologists and one neurosurgeon) and deliberations of a Panel Subcommittee on March 26, 2025. In the June 25 full MEDCAC Panel meeting, Panelists will use rating scales, as shown, to indicate the importance of each endpoint domain. During the discussion that follows, Panelists will comment on the reasons for their rating and the relative importance of the endpoints within each domain. 1. Clinician-Assessed Health Outcomes Domain 1 Not at all important 2 Slightly important 3 Somewhat important 4 Very important 5 Extremely important Specific Clinical Endpoints, Parkinson’s Disease Global change in motor symptom severity Motor-related activities (experiences) of daily living Gait function, postural instability, balance and/or falls Global change in nonmotor symptoms Motor complications (motor fluctuations, dyskinesia) Speech function and swallowing Cognitive function Specific Clinical Endpoint, Essential Tremor Reduction in tremor 2. Patient-Reported Outcomes Domain 1 Not at all important 2 Slightly important 3 Somewhat important 4 Very important 5 Extremely important Specific Clinical Endpoints, Parkinson’s Disease Parkinson’s Disease-related quality of life Sleep quality Specific Clinical Endpoint, Essential Tremor Tremor-related quality of life or tremor-related activities (experiences) of daily living 3. Device-Related Safety Domain 1 Not at all important 2 Slightly important 3 Somewhat important 4 Very important 5 Extremely important Several adverse events are possible following device-related treatments of Parkinson’s Disease and essential tremor. The nature and seriousness of these possible adverse events vary by device type. DISCUSSION Please be prepared to discuss the following issues as they apply to each endpoint domain. The importance of specific clinical endpoints within each domain and how importance may differ by device type or patient subpopulation Ideal and minimum duration of follow-up required for detection of an impact within each domain Thresholds for defining an MCID for clinical endpoints identified for studies of devices for patients with essential tremor . No MCIDs were identified for these endpoints in the literature reviewed by the CER, nor were any suggested by the expert reviewers. Panel input can be qualitative and need not be instrument-specific. 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: Yi-Fen Irene Chen, MD Primary Care Physician and Associate Executive Director The Permanente Medical Group Robin Newman, OTD, MA, OTR/L, CLT, FAOTA Program Director: Behavior and Health Program Clinical Associate Professor Department of Occupational Therapy Sargent College of Health and Rehabilitation Sciences Boston University Sally Stearns, PhD Professor, Department of Health Policy and Management Gillings School of Global Public Health The University of North Carolina at Chapel Hill Patient Advocate Amy Goldsmith, MA, CCC-SLP, RAC-CT, CMAC Chief Operating Officer for Reimbursement Reimagined Industry Representatives Laura Mauri, MD, MSc Senior Vice President Chief Scientific and Medical Officer Medtronic Speakers List: SPEAKER LIST *6 MINUTES PER SPEAKER* Presentations: Kelly Lyons, PhD, International Essential Tremor Foundation Pravin Khemani, MD, Swedish Neuroscience Institute Helen Bronte-Stewart, MD , QDG Health Stuart H. Isaacson, MD, Parkinson’s Disease and Movement Disorders Center of Boca Raton Peter A. LeWitt, MD, Wayne State University School of Medicine Joshua M. Rosenow, MD, Northwestern Medicine Ashwini Sharan, MD, Medtronic