Analysis of Coverage with Evidence Development (CED) Criteria

CMS evidence review | evidence_type=MEDCAC Meeting | record_type=medcac_meeting | document_id=79 | meeting_date=2023-02-13

On December 7, 2022 February 13 and 14, 2023 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 the requirements for clinical studies submitted for CMS coverage under Coverage with Evidence Development (CED). It has been nearly eight years since the criteria for CED were last evaluated and codified. In that time, not only have technologies become more complex, but there has been growing appreciation and commitment to transparency in decision-making, to making certain that study methodologies are “fit to purpose” as determined by the topic, questions asked, health outcomes studied, and to making certain that the populations studied are representative of the diversity in the Medicare beneficiary population. For example, some questions may be sufficiently answered through analysis of real-world evidence including data from clinical registries, electronic health records, and administrative claims. Any decision about whether an item or service is reasonable and necessary must, minimally, be sensitive to these commitments as well as to ensuring that study participants’ interests are respected and protected. The MEDCAC will evaluate the CED criteria to assure that CED studies are evaluated with consistent, feasible, transparent and methodologically rigorous criteria and advise CMS on whether the criteria are appropriate to ensure that CED-approved studies will produce reliable evidence that CMS can rely on to help determine whether a particular item or service is reasonable and necessary.

Actions Taken: September 12, 2022 CMS announces MEDCAC meeting. October 7, 2022 Posted Federal Register Notice November 10, 2022 Posted ' Analysis of Requirements for Coverage With Evidence Development (CED) – Topic Refinement ' November 17, 2022 CMS will reschedule the December 7, 2022 Coverage with Evidence Development (CED) Medicare Evidence Development and Coverage Advisory Committee (MEDCAC) virtual meeting and extend the meeting from one to two-days, February 13 and 14, 2023, 10am-3pm EST. CMS is rescheduling and extending the meeting to two days because of public comments asking for more time to prepare and respond to the recommended CED revisions and public comments asking for more clarity of the meeting. CMS will publish the MEDCAC voting questions no later than December 31, 2022, to ensure that the public has time to fully prepare. Further, CMS will re-open the registration to allow for additional speakers, presentations and comments. Those that already submitted comments or presentations or requested to speak at the December 7, 2022 meeting, continue to have a reserved time slot and do not need to re-register but you will have to register to attend. We note that this MEDCAC is to discuss and make recommendations to CMS regarding the CED criteria. The MEDCAC panel will use the final AHRQ report (as a basis of its discussion during the MEDCAC meeting. The meetings purpose is neither to discuss technologies, such as devices, drugs or biologics, nor to discuss the CMS’s use of the CED pathway whereby Medicare covers items and services on the condition that they are furnished in the context of approved clinical studies or with the collection of additional clinical data December 7, 2022 Posted updated FR notice. December 23, 2022 Posted panel roster and questions to panel. January 30, 2023 Posted agenda , speakers list , written comments , background materials , and presentations . February 14, 2023 Posted Jodi Segal's presentation from February 13. February 23, 2023 Posted scoresheet and recordings from meeting. April 11, 2023 Posted transcripts from meeting. Registration Agenda: Federal Register Notice Agenda Medicare Evidence Development & Coverage Advisory Committee February 13, 2023 10:00 AM – 3:00 PM Virtual Meeting Joseph Ross, MD, MHS , Committee Chair Sanket Dhruva, MD, MHS , Committee Vice-Chair Tamara Syrek Jensen , Director, Coverage and Analysis Group Tara Hall , MEDCAC Coordinator strong>——————————————————————————————————————————————————————————————— 10:00 – 10:15 AM Opening Remarks – Tara Hall/Tamara Syrek Jensen/Joseph Ross, MD, MHS 10:15 - 10:45 AM TA Presentation- Jodi Segal, MD, MPH -Professor of Medicine, Epidemiology, and Health Policy and Management John Hopkins 10:45 – 11:15 AM Questions from Panel 11:15 – 11:30 AM Break 11:30 – 12:50 PM Scheduled Public Comments ************************************************************************************ 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. Speakers Cybil Roehrenbeck, JD, Hogan Lovells Diana Zuckerman, National Center for Health Research Donnette Smith Jay Reinstein, Voices of Alzheimer’s M Kay Scanlan, JD, Haystack Project Susan Peschin, MHS, Alliance for Aging Research Tara Burke, PhD, AdvaMed Yajuan Lu, Boston Scientific William Padula, PhD, Leonard D. Schaeffer Center for Health Policy & Economics Speakers with Presentations Beena Bhuiyan Khan, Duke-Robert J. Margolis, MD, Center for Health Policy Brian Carey, Foley Hoag LLP Cathy Cutler, PhD, Brookhaven National Laboratory Lindsay Bockstedt, Medtronic, Inc. Ralph G. Brindis, MD, MPH, MACC, American College of Cardiology 12:50 – 1:20 PM Lunch 1:20 – 1:40 PM Open Public Comments Public Attendees who wish to address the panel will be given that opportunity Speakers are asked to state whether or not they have any financial involvement with manufacturers of any products being discussed or with their competitors. 1:40 – 3:00 PM Questions to Presenters 3:00 PM - Adjourn for the day Agenda Medicare Evidence Development & Coverage Advisory Committee February 14, 2023 10:00 AM – 3:00 PM Virtual Meeting Joseph Ross, MD, MHS , Committee Chair Sanket Dhruva, MD, MHS , Committee Vice-Chair Tamara Syrek Jensen , Director, Coverage and Analysis Group Tara Hall , MEDCAC Coordinator ——————————————————————————————————————————————————————————————— 10:00 – 10:15 AM Opening Remarks/Recap – Tara Hall/Tamara Syrek Jensen/Joseph Ross, MD, MHS 10:15 – 11:15 AM Initial Open Panel Discussion: Dr. Ross 11:15 – 12:15 PM Formal Remarks and Voting Questions The Chairperson will ask each panel member to state his or her position on the voting questions. 12:15 – 12:45 AM Lunch 12:45 – 1:45 PM Formal Remarks and Voting Questions The Chairperson will ask each panel member to state his or her position on the voting questions. 1:45 – 2:30 PM Final Open Panel Discussion: Dr. Ross 2:30 – 3:00 PM Closing Remarks/Adjournment: Tamara Syrek Jensen and Dr. Ross Issue: On December 7, 2022 February 13 and 14, 2023 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 the requirements for clinical studies submitted for CMS coverage under Coverage with Evidence Development (CED). It has been nearly eight years since the criteria for CED were last evaluated and codified. In that time, not only have technologies become more complex, but there has been growing appreciation and commitment to transparency in decision-making, to making certain that study methodologies are “fit to purpose” as determined by the topic, questions asked, health outcomes studied, and to making certain that the populations studied are representative of the diversity in the Medicare beneficiary population. For example, some questions may be sufficiently answered through analysis of real-world evidence including data from clinical registries, electronic health records, and administrative claims. Any decision about whether an item or service is reasonable and necessary must, minimally, be sensitive to these commitments as well as to ensuring that study participants’ interests are respected and protected. The MEDCAC will evaluate the CED criteria to assure that CED studies are evaluated with consistent, feasible, transparent and methodologically rigorous criteria and advise CMS on whether the criteria are appropriate to ensure that CED-approved studies will produce reliable evidence that CMS can rely on to help determine whether a particular item or service is reasonable and necessary. Minutes: Transcripts VOLUME I (February 13, 2023, day one of two) VOLUME II (February 14, 2023, day two of two) Other Material: Transcript 1 Panel Voting Questions: Download scoresheet COVERAGE WITH EVIDENCE DEVELOPMENT PROPOSED REVISED REQUIREMENTS MEDCAC PANEL QUESTIONS BACKGROUND: On February 13 and 14, 2023 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 and discuss the revisions of the requirements for clinical studies submitted for CMS coverage under Coverage with Evidence Development (CED) recommended by the Agency for Healthcare Research and Quality’s (AHRQ’s) Evidence-based Practice Program. It has been nearly eight years since the CED requirements were last evaluated. In that time, not only have technologies become more complex, but there has been growing appreciation and commitment to transparency in decision-making, to making certain that study methodologies are “fit to purpose” as determined by the topic, and to making certain that the populations studied are representative of the diversity in the Medicare beneficiary population. The MEDCAC will evaluate the proposed revisions to the CED requirements to ensure that CED studies are evaluated with consistent, feasible, transparent and methodologically rigorous requirements. The MEDCAC will also advise CMS on whether the proposed revisions to the requirements are appropriate to ensure that CED-approved studies will produce evidence that CMS can rely on to help determine whether a particular item or service is reasonable and necessary. Note: The meeting’s purpose is not to discuss specific services or technologies, such as devices, drugs or biologics or when it may be appropriate to issue a CED National Coverage Determination. For each voting question, please use the following scale identifying the level of importance for each requirement – a score of 0 being not important, a score of 1 point being important, and a score of 2 points being essential. Please score each of the following proposed Requirements as: 0 Not Important 1 Important 2 Essential SPONSOR 1) Current CED Requirements (version 2014) (e) The study is sponsored by an organization or individual capable of completing it successfully, The study is conducted by sponsors/investigators with the resources and skills to complete it successfully COMMUNICATION 2) No existing Requirement, A written plan describes the schedule for completion of key study milestones to ensure timely completion of the CED process. GOVERANCE 3) No existing Requirement, The protocol describes the information governance and data security provisions that have been established. CONTEXT 4) Current CED Requirements (version 2014) (b) and (c) (b)The rationale for the study is well supported by available scientific and medical evidence. (c) The study results are not anticipated to unjustifiably duplicate existing knowledge. The rationale for the study is supported by scientific evidence and study results are expected to fill the specified knowledge gap and provide evidence of net benefit. 5) Current CED Requirements (version 2014) (a) The principal purpose of the study is to test whether the item or service meaningfully improves health outcomes of affected beneficiaries who are represented by the enrolled subjects. Sponsors/investigators establish an evidentiary threshold for the primary outcome(s) so as to demonstrate clinically meaningful differences with sufficient precision. OUTCOMES 6) No Existing Requirement, The primary outcome(s) for the study are clinically meaningful and important to patients. A surrogate outcome that reliably predicts these outcomes may be appropriate for some questions. PROTOCOL 7) Current CED Requirements (version 2014) (h) and (j), (h) The study has a written protocol that clearly demonstrates adherence to the standards listed here as Medicare requirements; and (j) The clinical research studies and registries are registered on the www.ClinicalTrials.gov website by the principal sponsor/investigator prior to the enrollment of the first study subject. Registries are also registered in the Agency for Healthcare Quality (AHRQ) Registry of Patient Registries (RoPR). The CED study is registered with ClinicalTrials.gov and a complete protocol is delivered to CMS. POPULATION 8) No existing Requirement, The study population reflects the demographic and clinical diversity among the Medicare beneficiaries who are the intended users of the intervention. This includes attention to the intended users’ racial and ethnic backgrounds, gender, and socio-economic status, at a minimum. GENERALIZABLE 9) Current CED Requirements (version 2014) (m) The study protocol explicitly discusses how the results are or are not expected to be generalizable to affected beneficiary subpopulations. Separate discussions in the protocol may be necessary for populations eligible for Medicare due to age, disability or Medicaid eligibility. When feasible and appropriate for answering the CED question, data for the study should come from beneficiaries in their usual sites of care, although randomization to receive the product may be in place. DATA QUALITY 10) No existing Requirement, The data are generated or selected with attention to completeness, accuracy, sufficiency of duration of observation to demonstrate durability of results, and sufficiency of sample size as required by the question. DATA USE 11) No existing Requirement, Sponsors/investigators provide information about the validity of the primary exposure and outcome measures, including when using primary data that is collected for the study and when using existing (secondary) data. DESIGN 12) Current CED Requirements (version 2014) (d) and (g), (d) The study design is methodologically appropriate, and the anticipated number of enrolled subjects is sufficient to answer the research question(s) being asked in the National Coverage Determination. (g) All aspects of the study are conducted according to appropriate standards of scientific integrity. The study design is selected to generate valid evidence safely and efficiently for decision making by CMS. If a contemporaneous comparison group is not included, this choice must be justified. The sponsors/investigators minimize the impact of confounding and biases on inferences with rigorous design and appropriate statistical techniques. DESIGN-SUBPOPULATIONS 13) Current CED Requirements (version 2014) (l) The study protocol must explicitly discuss beneficiary subpopulations affected by the item or service under investigation, particularly traditionally underrepresented groups in clinical studies, how the inclusion and exclusion Requirements effect enrollment of these populations, and a plan for the retention and reporting of said populations in the trail. If the inclusion and exclusion Requirements are expected to have a negative effect on the recruitment or retention of underrepresented populations, the protocol must discuss why these Requirements are necessary. In the protocol, the sponsors/investigators describe plans for analyzing demographic subpopulations, defined by gender and age, as well as clinically- relevant subgroups as motivated by existing evidence. Description of plans for exploratory analyses, as relevant subgroups emerge, is also appropriate to include, but not required. REPRODUCIBILITY 14) No existing Requirements, Sponsors/investigators using secondary data will demonstrate robustness of results by conducting alternative analyses and/or using supplementary data. REPORTING 15) Current CED Requirements (version 2014) (k) The research study protocol specifies the method and timing of public release of all prespecified outcomes to be measured including release of outcomes if outcomes are negative or study is terminated early. The results must be made public within 12 months of the study’s primary completion date, which is the date the final subject had final data collection for the primary endpoint, even if the trial does not achieve its primary aim. The results must include number started/completed, summary results for primary and secondary outcome measures, statistical analyses, and adverse events. Final results must be reported in a publicly accessibly manner; either in a peer-reviewed scientific journal (in print or on-line), in an on-line publicly accessible registry dedicated to the dissemination of clinical trial information such as ClinicalTrials.gov, or in journals willing to publish in abbreviated format (e.g., for studies with negative or incomplete results). The study is submitted for peer review with the goal of publication using a reporting guideline appropriate for the study design and structured to enable replication. SHARING 16) No existing Requirements, The sponsors/investigators commit to sharing analytical output, methods, and analytic code with CMS or with a trusted third party in accordance with the rules of additional funders, institutional review boards, and data vendors as applicable. The schedule for sharing is included among the study milestones. The study should comply with all applicable laws regarding subject privacy, including section 165.514 of the Health Insurance Portability and Accountability Act of 1996 (HIPAA) LEGAL 17) Current CED Requirements (version 2014) (i), The study is not designed to exclusively test toxicity or disease pathophysiology in healthy individuals. Such studies may meet this requirement only if the disease or condition being studied is life threatening as defined in 21 CFR §312.81(a) and the patient has no other viable treatment options. The study is not designed to exclusively test toxicity, although it is acceptable for a study to test a reduction in toxicity of a product relative to standard of care or an appropriate comparator. For studies that involve researching the safety and effectiveness of new drugs and biological products aimed at treating life-threatening or severely-debilitating diseases, refer to additional requirements set forth in 21 CFR §312.81(a). 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: Michael J. Fisch, MD, MPH, FACP, FAAHPM Clinical Professor, MD Anderson Cancer Center National Medical Director AIM Specialty Health Clinical Trials David Flannery, MD Staff and Director of Telegenetics and Digital Genetics, Center for Personalized Genetic Healthcare Genomic Medicine Institute Cleveland Clinic Genetics Carolyn Ford, PharmD Professor and Director of Community Healthcare Outreach Wingate University School of Pharmacy Health Disparities Genevieve Kanter, PhD Associate Professor Sol Price School of Public Policy Schaeffer Center for Health Policy and Economics University of Southern California Karen Joynt Maddox, MD, MPH, FACC, FAHA Assistant Professor, Washington University School of Medicine Co-Director, Center for Health Economics and Policy, Institute for Public Health at Washington University School of Medicine Cardiology Marc Mora, MD Senior Medical Director Networks and Care Management Kaiser Permanente Washington Evidence based medicine Olorunseun O. Ogunwobi, MD, PhD Director, Hunter College Center for Cancer Health Disparities Research (CCHDR) Associate Professor of Biology Department of Biological Sciences Hunter College of the City University of NY and Adjunct Assistant Professor Joan and Sanford I. Weill Department of Medicine Weill Cornell Medical College Oncology Sally Stearns, PhD Professor, Department of Health Policy and Management Gillings School of Global Public Health The University of North Carolina at Chapel Hill Public Health John Whitney, MD Vice President, Medical Policy Anthem, Inc. Internal Medicine Dru Riddle, PhD, DNP, CRNA Associate Professor of Professional Practice Texas Christian University Genetics Ian N. Kremer, JD Executive Director Leaders Engaged on Alzheimer's Disease Industry Representatives Parashar Patel, MA President & CEO Market Access Strategy, LLC Guest Panel Members Daniel Arthur Caños, PhD, MPH Director, Office of Clinical Evidence and Analysis Office of Product Evaluation and Quality CDRH| Food and Drug Administration Craig A. Umscheid, MD, MS Director, Evidence-based Practice Center Division Senior Science Advisor, Center for Evidence and Practice Improvement Agency for Healthcare Research and Quality Richard J. Hodes, M.D. Director National Institute on Aging National Institutes of Health Speaker List: Medicare Evidence Development & Coverage Advisory Committee February 13, 2023 SPEAKER LIST *5 MINUTES PER SPEAKER* Cybil Roehrenbeck, JD, Hogan Lovells Diana Zuckerman, National Center for Health Research Donnette Smith Jay Reinstein , Voices of Alzheimer’s M Kay Scanlan, JD, Haystack Project Susan Peschin, MHS, Alliance for Aging Research Tara Burke, PhD, AdvaMed Yajuan Lu, Boston Scientific William Padula, PhD , Leonard D. Schaeffer Center for Health Policy & Economics Presentations: Beena Bhuiyan Khan, Duke-Robert J. Margolis, MD, Center for Health Policy Brian Carey, Foley Hoag LLP Cathy Cutler, PhD, Brookhaven National Laboratory Lindsay Bockstedt , Medtronic, Inc. Ralph G. Brindis, MD, MPH, MACC , American College of Cardiology Video Recordings Of Meeting: Day 1- https://cms.zoomgov.com/rec/share/XCtYrtiKxd4FHEx4n6KjJKpU46hcMWXURajVGHjtOcQ1cF-S6u8RQ23Zc66AJQIA.5UlHLc0HjG5Z_41j Passcode: ^74Ywdib Day 2 – https://cms.zoomgov.com/rec/share/w1CHqN4dBZx4Q5WNAEEA3hOIpZ-uj7kEBI_1mpYPOYdtY0TU8Qvpc-3_V1FEW8_-.W539WXpCCxpcsYLo Passcode: HL!?au2b