FDA/CMS PARALLEL REVIEW: Streamlining Approval and Reimbursement Lakshman Ramamurthy PhD Director, Avalere Health LLC FDA Entrepreneur-in-Residence March 20, 2014! avalerehealth.net Outline – FDA/CMS Parallel Review • Historical perspec-ve on FDA/CMS Interac-ons • What are the goals of Parallel Review? • Implica-ons of an evolving FDA/CMS landscape • Summary 2 Key Milestones Demonstrate Increasing Formal and Informal Agency Collabora-on Informal collabora-on process endorsed by HHS Medical Innova-on Task Force FDA and CMS joint Federal Register No-ce reques-ng comments on parallel review Parallel Review Pilot Begins FDA and CMS announce Memorandum of Understanding Jan 2005 June 2010 Exact Sciences Announces Cologuard Will Be Part of the Pilot Sept 2010 Oct. 2011 Medtronic Announces Symplicity Will Be Part of the Pilot Dec. 2011 Memorandum Of Understanding Between United States Food And Drug Administra-on and Centers for Medicare & Medicaid Services. MOU 225-‐10—0010. Accessed December 28, 2010. Available at: hSp://www.fda.gov/abouTda/partnershipscollabora-ons/memorandaofunderstandingmous/domes-cmous/ucm217585.htm March 2013 Mo-va-ons for a collabora-ve engagement • FDA and CMS have both expressed their respec-ve mo-va-ons for this collabora-on Addressed concerns voiced by stakeholders § Pilot will be conducted in the device industry, which only eventually may transi-on to the drug industry § Ul-mate number of parallel review candidates will be small due to CMS’ limited capacity § Poten-al divergence in need for parallel review for an established versus a startup manufacturer CAG: Coverage and Analysis Group CDRH: Center for Devices & Radiological Health “One of the things that is extraordinarily frustra-ng for us is some-mes manufacturers get through FDA and they come to us, and it turns out that even they have to admit that the evidence ... is en-rely off the map for CMS. And we kind of sit there and go, ‘If only we had talked two or three years ago’” –Louis Jacques, Director of CAG at CMS “We do hear from venture capitalists that collapsing the -meframes for review and between when a product is approved by FDA and when it would be covered -‐ is one of the things the agencies can do to really encourage investments” –Nancy Stade, Deputy Director for Policy at FDA's CDRH Roll out of the Parallel Review Pilot No-ce and Comment: Key Areas of Requested Comments Voluntary, sponsor-‐ iniCated? How to stagger the start of FDA and CMS review? Extent of public disclosure during review process? Parallel Review Degree of data sharing between CMS and FDA? Which classes of products would benefit most? Most Commentators Have Serious Reserva-ons About Parallel Review Supports, with some reservaCons (8) Serious reservaCons (25) Strong opposiCon, “uncertain that it is necessary” (2) Spectrum of Support for Parallel Review Manufacturers § IBA § Exact Sciences § Sanuwave Health Trade AssociaCons § ABC § Con-nua PaCent Advocacy Groups § REAP Specialty SocieCes § AGA § ASTRO Manufacturers § BIPI § Cepheid § Medtronic § Alcon § BCBSA § Telacris § Genentech § JNJ PaCent Advocacy Groups § Breast Cancer Coali-on § NPAF § CF Independent Providers § Leonie Rose, R.N. § Dr. Raj Rao AACC = American Associa-on for Clinical Chemistry ABC = American Blood Centers AGA = American Gastroenterological Associa-on ARM = Alliance of Regenera-ve Medicine ASTRO = American Society for Radia-on Oncology CF = Cys-c Fibrosis Founda-on Specialty SocieCes § ACR/ SNM/ ASNC/ ASNR/ SIR § AACC Specialty SocieCes § ACC/ ASE/ HRS/ SCMR Trade AssociaCons § BIO § MITA § PPTA § ARM § PhRMA § MDMA § AdvaMed § CORAR, MICAA § NVCA, MedIC Provider Groups § ACCC Other § SBA Advocacy CORAR = Council on Radionuclides and Radiopharmaceu-cals IBA = IBA Molecular Tracers, North America MCW = Medical College of Wisconsin MedIC = Medical Innova-on and Compe--veness Coali-on MITA = Medical Imaging and Technology Alliance MICAA = Medical Imaging Contrast Agent Associa-on NPAF = Na-onal Pa-ent Advocate Founda-on PPTA = Plasma Protein Therapeu-cs Associa-on REAP = Regulatory Educa-on and Ac-on for Pa-ents Majority of Comments Focused on Five Key Issues Voluntary, Sponsor DisCnct Missions and IniCated Review Processes Should Not Subvert Local Coverage Process Which Products or Classes Subject to Parallel Review ConfidenCality of Sponsor Data Consensus Disagreement Volume of Comments What happens ajer FDA review? FDA Decision Default: Paid Review Evaluate outcome and cost data to support value proposition Similar = Certainty Different = Uncertainty $ FDA’s Entrepreneur-in-Residence Program – Innovation Pathway! • Problem Statement: Reimbursement for innova-ve medical devices is inconsistent at cri-cal -mes, such as during IDE clinical studies or immediately following FDA clearance/ approval. • As a result, some technologies, with the poten-al for important public health impact, do not reach US pa-ents. hSp://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDRH/CDRHInnova-on/Innova-onPathway/ucm286138.htm FDA EIR -‐ Drivers for Parallel Review Ini-a-ve • Currently no forum exists to prospec-vely nego-ate terms for data required to support reimbursement • Fragmented and Inconsistent reimbursement process • Ini-a-on of Na-onal Coverage Determina-on (NCD) poten-ally damaging to companies • FDA pre-‐market and post-‐market data may be insufficient to support reimbursement • Lack of Clarity on evidence requirements for reimbursement • Early adop-on of reimbursement by Ini-a-ve par-cipants may lead to expansion of payer and user adop-on 10 Separate Mandates of FDA and Payers Require a Comprehensive Approach to Study Evolving Evidence Standards Medicare Coverage (National & Local) “reasonable and necessary” § Review rationale and works cited in NCD decision memorandum » Evidence thresholds not explicitly stated, vary by benefit and product, require a case-law approach § CMS frequently provides discretion too MACs » Map variations in Medicare local coverage decision making and associated evidence (when available) Private Payers “medically necessary” FDA (“safe & effective”) § Coverage frequently varies by individual plan » However, many large payers publish clinical policy bulletins, some with evidentiary rationale § Supplement abstraction of published rationale with primary research on evidence standards § FDA label most commonly cited by payers as source of initial product information » Published pivotal trial results § Increasing payer focus on evidence and data generation beyond traditional requirements for FDA approval TherapeuCc landscape assessments and product characterisCcs to determine which factors are most important for a coordinated regulatory & reimbursement strategy Un-‐Intended Consequences -‐ What happened? Our Analysis • Per FDASIA 2012, the agency was disallowed from rejec-ng IDEs for any other reason than safety concerns; i.e., even if the study would not support approval FDA may not disapprove the IDE • We predicted that this would have a bearing on how CMS would pay for such IDEs. • That CMS would centralize IDE reimbursement; and may require that the trials be pivotal! 12 • • On July 9, 2013 CMS proposes to centralize CMS’ review of Category A & B IDE trials rather than leave coverage for Category B devices at the discre-on of local contractors. Addi-onally, CMS proposes to “automa-cally cover the costs of rou-ne items and services in the a Category A study or trial, and the costs of the inves-ga-on device and the rou-ne items and services in a Category B study or trial” if the following two characteris-cs are met: – The study is a pivotal study; – The study includes a superiority study design hSp://avalerehealth.net/exper-se/life-‐sciences/insights/centralized-‐review-‐of-‐inves-ga-onal-‐device-‐ exemp-ons-‐at-‐cmsan-‐idea-‐whose hSp://www.ofr.gov/OFRUpload/OFRData/2013-‐16547_PI.pdf 13 Implica-ons of new MFPS Rules: • CMS, specifically Coverage and Analysis Group will have a say in IDE reimbursement; and wants to provide inputs on study designs • CMS prefers that device trials include superiority study design; while not ruling out reimbursement for non-‐superiority study design trials! • A more ac-vely engaging CMS agency in pre-‐ approval device trials! 14 Factors to Consider When Embarking on Launching a New Device: A role for FDA and CMS? InvesCgaConal Device ExempCon Approval Medicare Local and NaConal Coverage DeterminaCons Post-‐Approval Study Design and Enrollment Sponsors Must Consider Appropriate Engagement Strategy with Each Agency at Every Step Pre-‐Market Approval Study; Clinical Endpoints Registries to Capture Clinical and AdministraCve Claims Data Closing thoughts … • Reimbursement and coverage for devices and diagnos-cs is non-‐uniform and, in some cases, unclear • Payers will increasingly require stronger evidence of clinical u-lity and clinical validity for devices and diagnos-cs • The CMS may play an increasing (and incen-vized) role in the regulatory and reimbursement environment for devices and diagnos-cs • Engaging FDA and CMS early during a device trial may result in more efficient clinical study designs that meet the needs of regulators and payers ensuring faster path to market. 16 Avalere Provides Unparalleled Expertise in Executing Solutions! Evidence-Based Medicine – Quality Measures Strategies – Guideline Assessments – Stakeholder Mapping Providers and Facilities – Care Transitions – Payment & Delivery Models – Health Information Technology Health Economics & Outcomes Research – Value Proposition Support – Economic Analyses – Evidence Generation Strategies Strategy & Communications – Stakeholder Analysis – Strategic Planning – Multimedia Design & Development Payer Markets and Coverage – Private Payers – Medicare & Medicaid – Policy/Legislation Surveillance Data Analytics – Medicare & Commercial Data Analysis – Budget impact models – Customer payer tools Market Access – Integrated Launch Planning – Market Access Strategies and Payer Planning – Commercialization Support 17 Understanding the Market / Avalere’s Experts are Highly Respected Thought Leaders! Pharmaceutical & Biotechnology Providers & Facilities Financial Industry Rami Armon, JD Lehman Brothers, Chief Political Strategist Rick Chapman, PhD IMS Health, Principle Center for Risk Analysis at the Harvard School of Public Health Tanisha Carino, PhD CMS, Coverage and Analysis Group Fulbright Scholar, International Health Technology Assessment Ed Drozd, PhD RTI International, Senior Health Economist Eric Hammelman, MBA JP Morgan, Healthcare Analyst Nora Hoban, MPA CMS, Medicare Payment Policy GAO, Medicare Payment Systems Medical Devices & Diagnostics Managed Care/PBMs Medical Associations Research Foundations Kathy Hughes, MBA Holy Cross Health Corporation, COO Erik Johnson, MBA Advisory Board Company, Director HIT OMB, Deputy Branch Chief for Health Finance Mike Johnsrud, PhD Center for Pharmacoeconomic Studies, UT Austin, Director Jenny Levinson Medtronic Diabetes, Reimbursement Schering-Plough, CMS Strategy Kristi Mitchell, MPH American College of Cardiology, Chief Science Officer Dianne Munevar, MPP RTI International, Health Services Researcher Patient Groups Government Emil Parker, MPA Washington, D.C., Dept of Health, Capital Projects Lakshman Ramamurthy, PhD FDA, Center for Devices and Radiological Health" GlaxoSmithKline, R&D scientist " ! Erin Sullivan, PhD Abt BioPharma Solutions, Senior Director Boston Scientific, Director Anne Tumlinson, MMHS OMB, Medicaid and LTC Policy Office of Rep. John Lewis (D-GA) – Legislative Assistant Bonnie Washington, MSc Novartis, Director Government Affairs United Healthcare, Policy and Government Affairs OMB, Medicaid and SCHIP Policy 18
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