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The Second Annual Medical Device Regulatory, Reimbursement and Compliance Congress - March 28-30, 2007. “ CATCH-22” INTENDED USE DEFINITION. The FDA and CMS: Changes in Regulatory and Surveillance Proximity Beverly H. Lorell, MD, FACC Senior Medical & Policy Advisor

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catch 22 intended use definition
The Second Annual Medical Device Regulatory, Reimbursement and Compliance Congress - March 28-30, 2007“CATCH-22” INTENDED USE DEFINITION

The FDA and CMS:

Changes in Regulatory and Surveillance Proximity

Beverly H. Lorell, MD, FACC

Senior Medical & Policy Advisor

King & Spalding LLP

Washington, D.C.

fda and cms a changing proximity
FDA and CMS: A Changing Proximity
  • Device and Drug Innovation: Tensions between Regulatory and Public Expectations
  • Ensuring rapid access to new devices and drugs for unmet clinical need, and
  • Freedom from late low-frequency serious safety events (near-complete safety), and
  • Physician use for individual patient benefit through practice-of-medicine, and
  • No additional cost (or, maybe, Value =  benefit /  cost compared to current therapy).
fda and cms a changing proximity1
FDA and CMS: A Changing Proximity
  • The Evolution of CMS Oversight and Authority in the Federal Research Agenda:
  • CMS and FDA interaction past and present
  • Mandates for conduct and evaluation of clinical research
  • Payment for research
  • Coverage with Evidence Development
    • Challenges
    • Outsourcing of responsibilities to external partners
  • The challenge of rapid innovation: Case example
fda and cms a changing proximity2
FDA and CMS: A Changing Proximity
  • The Statutory Basis for FDA and CMS Separation:
  • 1976 Medical Device Amendments – FDA risk-based regulation of devices
    • Premarket Approval (“PMA”) for Class III (highest risk) devices: “reasonable assurance of safety & efficacy”
    • Premarket Notification for Class I and II devices: “Substantial equivalence” to a device already marketed
  • Social Security Act Section 182(a)(1)(a) – CMS National Coverage Decision: determination on a national level of items and services that are “reasonable and necessary”
fda and cms a changing proximity3
FDA and CMS: A Changing Proximity
  • FDA and CMS Interaction in the Federal Research Agenda
  • Categorization of Investigational Devices for Coverage under Medicare: Sept, 1995 - The HCFA/FDA Interagency Agreement; Nov, 1995 - The related HCFA Final Rule.
    • FDA categorizes all IDE devices: Category A (experimental/ investigational) Category B (nonexperimental/ investigational)
    • CMS contractors determine coverage of Category B, whereas Category A = non-coverage
  • CMS Clinical Trial Policy
    • Sept, 2000 - Routine costs implemented through the NCD process following a June 2000 Executive Order
    • July, 2006 - Now under reconsideration: Redefinition and expansion of “good clinical studies” that qualify for coverage
fda and cms a changing proximity4
FDA and CMS: A Changing Proximity
  • Tensions between FDA and CMS Perspectives:
  • FDA “reasonable safety and efficacy” determination does not equal:
    • CMS determination of “reasonable and necessary,” or
    • Beneficiary entitlement to an FDA approved device
    • Determinations can differ from the FDA-approved labeling
  • Coverage with Evidence Development (CED): April, 2005 -Patient data collection as a Condition of Coverage
  • MCAC
    • Dec, 2006 - Now MedCAC (Medicare Evidence Development & Coverage Advisory Committee)
    • New charter and expanded focus on CED collection and evaluation of clinical data
fda and cms a changing proximity5
FDA and CMS: A Changing Proximity
  • Ongoing Changes in the Congressional Agenda:
    • The Medicare Data Access and Research Act (S.3897)
      • Sept 14, 2006 - Introduced by Senators Charles Grassley and Max Baucus (Chairman and Ranking Member of the Finance Committee); Likely to be reintroduced this session.
      • Require the Secretary of HHS to enter annual data release agreements with the FDA, CMS, CDC, AHRQ and NIH;
      • Link hospital and ambulatory care data to CMS data on benefits to Medicare beneficiaries, and permit federal agencies and qualified researchers to analyze drug safety and health care use;
      • “More concretely, analyzing the Medicare claims data can help agencies, such as the FDA, identify situations like the one involving Vioxx more quickly and provide a new valuable tool to enable the FDA to take swifter action to protect the public’s health and well-being.” Senator Grassley, Sept 14, 2006
fda and cms a changing proximity6
FDA and CMS: A Changing Proximity




FDA Regulatory Approval







Clinical Trials



CMS National Coverage Decision

Coverage with Evidence Development

Performance Benchmarks

Clinical Value Enablers:

Does Science Support It?

Economic Value Enablers:

Will Someone Pay for It?

Social Value Enablers:

Public Health Mandates

fda and cms a changing proximity7
FDA and CMS: A Changing Proximity
  • The CMS Authority to Mandate CED -
    • “The statutory authority for linking coverage decisions to the collection of
    • additional data is derived from Sec.1862(a)(1)(A) of the Act, which states
    • that Medicare may not provide payment for items and services unless they are
    • ‘‘reasonable and necessary’’ for the treatment of illness or injury. In some
    • cases, CMS will determine that an item or service is only reasonable and
  • necessary when specific data collections accompany the provision of the service. In these cases, the collection of data is required to ensure that the care provided to individual patients will improve health outcomes.”
  • The Purpose of CED - CMS Guidance Document July 12, 2006:
  • Document appropriateness of use in beneficiaries;
  • Consider future changes in coverage;
  • Generate clinical information that will improve the evidence base on which providers base medical recommendations.
cms national coverage with evidence development
FDA and CMS: A Changing ProximityCMS National Coverage With Evidence Development

Broad National Coverage


Is existing evidence sufficient to support Medicare coverage?

Limited Coverage with Evidence Development (ICD Registry)

Coverage with Evidence Development (colon cancer drugs)



fda and cms a changing proximity8
FDA and CMS: A Changing Proximity

National Coverage DeterminationCED

  • Cochlear Implantation None scheduled at present

2. Chemotherapy Colorectal Cancer National Cancer Institute: Clinical Trials Covered Under the Medicare Anti-Cancer Drug NCD

3. PET (FDG) for Brain, Cervical, National Oncologic PET Registry

Ovarian, Pancreatic, Small Cell Lung,

and Testicular Cancers

  • Implantable Cardioverter Defibrillators American College of Cardiology- National Cardiovascular Data Registry
  • Carotid Stenting American College of Cardiology-NCDR
  • PET (FDG) for Dementia and Medicare-Approved PET for Dementia

Neurodegenerative Diseases Trial

6. Long term oxygen treatment NHLBI

fda and cms a changing proximity9
FDA and CMS: A Changing Proximity
  • FDA Clinical Standards for “Reasonable Efficacy and Safety” are Better Defined than those of CED
  • Clinical evidentiary standards: Prospective, randomized, blinded clinical trial as highest clinical standard of proof-of-efficacy (predefined questions to be tested and endpoints)
  • Control of bias in the groups to be compared
  • Formal adjudication of events, and stopping rules
  • Evidence needed for decision making: 2 confirmatory studies (drug approval)
  • “ p < .05” Statistical standard – the level of confidence required to conclude that the result is not due to play of chance
fda and cms a changing proximity10
FDA and CMS: A Changing Proximity
  • Clinical Evidentiary Standards for CED Registry Research are Still Rudimentary and Evolving
  • Registry Strengths
    • Demographics and patterns of real-world use
  • Registry Weaknesses - Less consensus regarding
    • Methods of monitoring and analysis
    • Acceptable statistical boundaries of certainty
    • Bias
    • Adjudication
    • Stopping rules - Does a CED registry ever end?
    • Longitudinal patient data and protection of privacy
  • Poor tools to ask the REAL QUESTION: “Does it really work?” or," Does A work better than B?” or, “Is A safer than B?”
fda and cms a changing proximity11
FDA and CMS: A Changing Proximity

Outsourcing of CED data collection,

“American College of Cardiology-National Cardiovascular Data Registry, a confidential quality measurement program for cardiac and vascular facilities. It now operates four national registries.”:


Carotid Artery Stents

Angioplasty and

Drug Eluting Stents

Acute Coronary Syndrome

Drugs, Devices, Processes

fda and cms a changing proximity12
FDA and CMS: A Changing Proximity
  • Drug Eluting Stents - A Case Example
  • Emerging combinatorial science:
    • Novel combination products: drug, polymer, device
    • Reduce coronary restenosis (and need for 2nd invasive procedure) compared with bare metal stents
  • FDA approval: 2003 (Cypher) and 2004 (Taxus)
  • CMS anticipatory reimbursement: New DRG
  • Postmarket surveillance:
    • FDA condition-of-approval: 5-year follow-up of patients enrolled in pivotal premarket trials, and
    • Single-arm real world registries:at least 2000 pts followed for one year (but, no control group)
fda and cms a changing proximity13
FDA and CMS: A Changing Proximity
  • Drug Eluting Stents - A Case Example

Postmarket challenges for FDA and CMS

  • Rapid penetrance: >80% of coronary stent use
  • Practice-of-medicine: >60% of real world use is in complex patients and complex arteries
  • Variable use of adjunctive drugs (aspirin and Plavix)

Detecting late and rare safety signals: late stent thrombosis

  • Determining causality vs. the play of chance
  • FDA - Dec 2006: public panel; Jan 2007: safety statement including consideration of change in product labeling
  • CMS - 2007: Possible reconsideration of coverage policy
FDA and CMS: A Changing Proximity

Life Cycle of Device Approval & Iteration

FDA Postmarket Surveillance

CMS Coverage


FDA Approval

FDA and CMS: A Changing Proximity

Life Cycle of Continuous Change in Benefit–Risk

New Clinical Information

Increased Interagency Interaction





In Patient Choice

Evolution of CMS Policy

Evolution of FDA Policy

Changes in

Practice of


fda and cms a changing proximity14
FDA and CMS: A Changing Proximity
  • What are the Opportunities to Enhance FDA and CMS Interaction to Improve Patient Care and Assure Innovation?
  • Recognize: Strengths and limitations of registries
  • Align: FDA condition-of-approval and CMS CED clinical data collection
  • Innovate: Use the Medicare claims data base to ask specific questions
  • Simplify: Avoid redundant postmarket clinical data collections
  • Assure: Transparency for all stakeholders