CMS/Coverage and Analysis Group CMS Requirements for Reimbursement for Innovation: Medicare Coverage & Evidence Development Lori M. Ashby CMS/Coverage and Analysis Group

Disclosure Statement of Financial Interest Within the past 12 months, I or my spouse/partner have had a financial interest/arrangement or affiliation with the organization(s) listed below. Affiliation/Financial Relationship Company Grant/Research Support Consulting Fees/Honoraria Major Stock Shareholder/Equity Royalty Income Ownership/Founder Intellectual Property Rights Other Financial Benefit Company Names All TCT 2018 faculty disclosures are listed online and on the App.

Disclaimers This presentation is a general summary that explains certain aspects of the Medicare Program, but is not a legal document. The official Medicare Program provisions are contained in the relevant laws, regulations, and rulings. This presentation was prepared as a service to the public and is not intended to grant rights or impose obligations. This fact sheet may contain references or links to statutes, regulations, or other policy materials. The information provided is only intended to be a general summary. It is not intended to take the place of either the written law or regulations. We encourage readers to review the specific statutes, regulations, and other interpretive materials for a full and accurate statement of their contents. This presentation was current at the time it was published or uploaded to the web. Medicare policy changes frequently so links to the source documents have been provided within the document. The Centers for Medicare & Medicaid Services employees, agents, and staff make no representation, warranty, or guarantee that this compilation of Medicare information is error-free and will bear no responsibility or liability for the results or consequences of the use of this guide.

Focus on Patients https://www.cms.gov/

Medicare Construct Established by the Social Security Act of 1965, Title XVIII §1862(a)(1)(A) reasonable and necessary for the diagnosis or treatment of illness or injury or to improve the functioning of a malformed body member (E) in the case of research conducted pursuant to §1142, which is not reasonable and necessary Defined benefit program Beneficiaries Age ≥ 65 years Disabled individuals End stage renal disease (557,000) Providers Settings Aged = 48.1 million in 2016 (up from 46.8 million in 2015). Disabled = 9.0 million in 2016 (same as 2015). Million year

Evidence-based Medicare Coverage National Coverage Determination (NCD): Sufficient evident to conclude that the item or service improves clinically meaningful health outcomes for the Medicare population Based on a comprehensive review of the published evidence Focuses on important patient centered outcomes

Medicare Evidence Development & Coverage Advisory Committee (MEDCAC) The MEDCAC: provides independent guidance and expert advice to CMS on specific clinical topics supplements CMS' internal expertise and allows for an unbiased and current deliberation of "state of the art" technology and science reviews and evaluates evidence and examines benefits, harms, and appropriateness of items and services judges the strength of the available evidence and makes recommendations to CMS based on that evidence. https://www.cms.gov/Regulations-and-Guidance/Guidance/FACA/MEDCAC.html

Health Outcomes in Heart Failure Treatment Technology Studies MEDCAC CMS convened a MEDCAC panel on March 22, 2017. The MEDCAC panel examined what health outcomes in studies for heart failure treatment technologies should be of interest to CMS. The MEDCAC emphasized the need for clinical data needed to demonstrate a reduction in the clinical composite endpoint of death/heart failure hospitalization in order to meet the reasonable and necessary standard for coverage following FDA approval.

Coverage with Evidence Development (CED) Coverage in the context of approved clinical studies or with the collection of additional clinical data Allows for positive coverage when evidence is insufficient for a more favorable decision. Evidence gaps may be due to low number of beneficiaries in clinical studies, lack of meaningful health outcomes, limited generalizability, inconsistency of study findings. May involve randomized controlled trials, observational studies and/or registries Specific interventions, benefits and harms, health outcomes

Other Clinical Studies under Medicare Investigational Device Exemptions (IDE) Studies Regulation at 42 CFR 405.201 New centralized process in 2015 Clinical Trial Policy Routine costs in clinical trials funded by certain federal agencies National Coverage Determination (NCD) Pub 100-3, Section 310.1

Medicare Beneficiaries in Clinical Studies Initial studies on new technologies may not include many older adults ≥ 65 years of age for several reasons including: Heterogeneity – may have multiple comorbidities and/or be taking multiple medications Non-adherence - may have difficulty following protocols and/or making all study follow-up visits Other considerations – measurement issues, cognitive function

Study Endpoints and Eligibility Criteria Important to determine the strength and generalizability of published evidence to the Medicare population May assist in establishing parameters of coverage with evidence development (CED)

Inclusion Criteria in National Coverage Determinations Patients eligibility criteria in national coverage determinations (NCDs) may reflect inclusion criteria of the studies forming the evidence base for the item or services, for example: Implantable cardioverter defibrillators Trial Covered Indication Multicenter Automatic Defibrillator Trial (Moss, 1996 and 2006); Sudden Cardiac Death in Heart Failure Trial (Brady, 2005) Patients who have severe ischemic dilated cardiomyopathy but no personal history of sustained ventricular tachyarrhythmia or cardiac arrest due to ventricular fibrillation, and have NYHA Class II or III heart failure, left ventricular ejection fraction (LVEF) ≤ 35%. Multicenter Automatic Defibrillator Trial II (Moss, 2002) Patients with a prior myocardial infarction and a measured left ventricular ejection fraction < .30. Sudden Cardiac Death in Heart Failure Trial (Brady, 2005); Defibrillator Implantation in Patients with Nonischemic Systolic Failure (Kober, 2016) Patients who have severe non-ischemic dilated cardiomyopathy but no personal history of sustained ventricular tachyarrhythmia or cardiac arrest due to ventricular fibrillation, NYHA Class II or III heart failure, and measured LVEF < 35%; been on optimal medical therapy for at least 3 months.

Study Exclusion Criteria Older adults may be excluded from initial studies assessing efficacy. Patients with end stage renal disease (ESRD) are often excluded.

FDA-CMS Parallel Review Program https://www.gpo.gov/fdsys/pkg/FR-2016-10-24/pdf/2016-25659.pdf

Coding CMS uses several code sets: Diagnosis related group (Medicare Severity MS-DRG) Current Procedural Terminology - CPT® is registered trademark of the American Medical Association. International Statistical Classifications of Diseases (ICD) codes are alphanumeric codes on diagnosis, symptoms, signs, diseases. Healthcare Common Procedure Coding System (HCPCS) CMS Level I (CPT), Level II

Payment Prospective Payment Systems Various Fee Schedules Inpatient (IPPS), outpatient (OPPS), long term care /SNF Various Fee Schedules Ambulance Fee Schedule Clinical Laboratory Fee Schedule Durable Medical Equipment, Prosthetics/Orthotics & Supplies Fee Schedule Physician Fee Schedule Resource-Based Relative Value Scale Relative value unit (RVU) physician work, practice expense, malpractice The geographic practice cost indexes (GPCIs) The conversion factor

www.cms.gov/Medicare/Coverage/CouncilonTechInnov/Downloads/Innovators-Guide-Master-7-23-15.pdf

Thank you! Lori.Ashby@cms.hhs.gov