intermacs collaborators: nhlbi: sustainability – are we there yet?

INTERMACS Collaborators:

• NHLBI: Sustainability – Are we there yet?• CMS: The blurred lines among DT, BTC, BTT• FDA: Benchmarks for next device

approval• Industry: INTERMACS – The ultimate post

market study• Hospitals: Utilizing the Quality Assurance

Reports

Sixth Annual Meeting, March 12, 2012

INTERMACS Annual Meetin

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NHLBI: Sustainability–Are we there yet?

M Miller



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National Heart, Lung, and Blood Institute:

INTERMACS-Looking Forward to Full Sustainability

Marissa A. Miller, DVM, MPHProject Officer

Chief, Advanced Technologies and Surgery BranchNational Heart, Lung, and Blood Institute

INTERMACS Annual Meeting

March 12, 2012


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INTERMACS: From an Idea To…..

2005 0 patients/0 centers Complete NHLBI funding

No pre/post market studies 0 publications 0 citations 0 spin-offs

2012 6492 patients/131 centers Sustainable Public Private

Partnership Pre/post market studies 40+ publications & abstracts 700+ citations MEDAMACS, PEDIMACS,

IMACS, ‘JAPANMACS’, ‘CANADAMACS’…..

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eeting

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Financial 131 hospitals engaged Device manufacturers integrated—sound business plan Longstanding NHLBI commitment

Scientific Where were we before the INTERMACS patient profiles,

INTERMACS survival curves, INTERMACS comparator data? Public Health Relevance

The trend toward rigorous registries—AHRQ publication, TVR Registry

Regulatory and reimbursement decisions being based on INTERMACS data

INTERMACS: Sustainability?

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eeting

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Expand and Improve the Data MEDAMACS! & PEDIMACS! Augment collection of functional capacity & health status Streamline data fields New site monitoring paradigm Rigorous hospital standards

Strategically align with other programs PumpKIN REVIVE-IT International data collection efforts

Don’t get complacent!

INTERMACS What’s Left To Do To Assure Sustainability?

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eeting

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Enhance as research tool and expand access De-identified data sets Semi-annual research fairs Web-based data requests

Formulate New Hypotheses Connect with transplant registry Link to CMS/reimbursement /insurance data Examine quality improvement/best practices

Ongoing Safety Surveillance (for the future) Link registry data to other data sources through UDI Implement automated safety surveillance tools to

prospectively monitor for failures and complications

INTERMACS What’s Left to Do To Assure Sustainability?

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eeting

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INTERMACS Partnerships

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ResearchersPublic

International

Organizations

Industry

Hospitals Academia

CMS

FDA

NHLBI

The Next Frontier—Strategic Partners?


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CMS: View of INTERMACS

Jyme Schafer, MD



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Jyme Schafer, MDDirector, Division of Medical and Surgical

ServicesCoverage and Analysis Group

Medicare and INTERMACS:

Past, Present and Future

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Brief History of Medicare Coverage

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VAD Postcardiotomy – 1993 Bridge to transplant – 1996 Destination therapy with registry

requirement – 2003* INTERMACS was written into policy in 2007

* Joint Commission hospital certification effective 2009

Artificial Heart Bridge to transplant in approved

studies – 2008 Destination therapy in approved

studies - 2008


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Social Security Act 1862(a)(1)

Notwithstanding any other provision of this title, no payment may be made under part A or part B for any expenses incurred for items or services—

(A) which, except for items and services described in a succeeding subparagraph or additional preventive services (as described in section 1395x(ddd)(1) of this title), are not 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

section 1142, which is not reasonable and necessary to carry out the purposes of that section

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What is the definition of R&N?

Congress has not defined it in statute. For practical uses, CMS has operationalized the following definition:

Adequate evidence to conclude that the item or service improves clinically meaningful health outcomes for the Medicare population

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Health Outcomes of Interest

More Persuasive Longer life and

improved function/participation

Longer life with arrested decline

Significant symptom improvement allowing better function/participation

Reduced need for burdensome tests and treatments

Less Persuasive

Longer life with declining function/participation

Improved disease-specific survival without improved overall survival

Surrogate test result better

Image looks better Doctor feels confident

Historically, Medicare has stated publicly as a matter of policy that it does

not consider cost in making national coverage determinations.

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What is an NCD?

National Coverage Determination (NCD) Policy for the Medicare FFS program Continuity in coverage across the country May include clinical indication, patient

population, centers of excellence, provider qualifications, etc.

NCD Manual Section 20.9, Artificial Hearts and Related Deviceshttp://www.cms.gov/manuals/downloads/

ncd103c1_Part1.pdf17


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Artificial Hearts and Related Devices (20.9)

Each VAD indication has different coverage criteria

Postcardiotomy (1993): limited to FDA device label

Bridge to transplant (1996): patient approved and listed for transplant, transplant center (some exceptions)

Destination therapy (last updated 2010):

detailed patient selection (NYHA, OMM, LVEF,

peak VO2); facility criteria (procedure volume, third party certification, INTERMACS, focus on informed consent for procedure)

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Staff Review

ProposedDecision

Memorand

um Posted

National Coverage Request

MEDCAC

External

Technology

Assessment

Reconsideration

Staff Review

Public

Comment

Final DecisionMemorandum

andImplementati

on Instructions

Benefit Category

Departmental Appeals

Board

MEDICARE NATIONAL COVERAGE PROCESSMEDICARE NATIONAL COVERAGE PROCESS

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Preliminary

Discussions


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Medicare and INTERMACS

Past Supported registry innovation and data

collection Present

Continuous communication with UAB Information sharing, status updates Monitoring of participation NCD requires registry participation Consider INTERMACS an example for others

Future INTERMACS data and policy development

(e.g., terminology, patient selection, facility selection)

Policy changes???20


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FDA: Benchmarks for next device approval

S Patel



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INTERMACS and FDA:The Clinical Goal Line

Sonna Patel-Raman, Ph.D.March 12, 2012

Acting ChiefCirculatory Support and Prosthetics Branch

Division of Cardiovascular DevicesCenter for Devices and Radiological Health


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Financial Disclosures

None


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Post-Market - Where Have We Been? Post-market is not intended to demonstrate

safety and effectiveness of the device Provides sponsors opportunity to design &

conduct post-approval studies (PAS) in cost efficient way to meet FDA requirements

Track real world usage of the device Adverse events Labeling and/or product modifications

Satisfies CMS reporting requirement for DT


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Pre-Market – Where Have We Been?

BTT = Performance Goal based upon published literature

DT = Randomized Controlled Trial

FDA’s thinking is evolving as the distinction becomes extinct


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Clinical Trial Design – Reasons to Conduct an RCT for VADs Not enough historical data to serve as control

group Unknown risk factors due to limited

experience with device Practice of medicine has changed Produce study groups comparable with

respect to known and unknown risk factors


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Clinical Trial Design –Reasons Not to Conduct an RCT for VADs

Extensive historical reporting, experience and/or literature available for a given therapy/device

Limited patient enrollment Longer trials resulting in unreasonable

burden to sponsors Increase in technology – fewer patients to

study per device


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Post-Market - Where Are We Now? Adverse event reporting that may lead to

minor modifications to labeling, device, or other interventions when appropriate

Publications of aggregate data allowing for interpretation of certain datasets

Balancing pre-market and post-market expectations of a registry


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Pre-Market – Where Are We Now?

FDA has approved: DT = RCT FDA has approved:

BTT = Concurrent/Contemporaneous Control – INTERMACS

Trial design evolving Appropriate amount of data to provide valid

scientific data to support a pre-marketing application

How much data is enough data? How much analysis is enough analysis?


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Where Are We Going?

Agree that distinction is changing due to practice of medicine


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Where Are We Going?

Mechanical CirculatorySupport

Very Short Term(< 6 hours)

Short Term(> 6 hours, ≤ 14 days)

Intermediate Term(> 14 days, < 1 year)

Long Term“forever”(2 years)

During CPB

Failure to weanCardiogenic shock

Bridge to next therapyPending recovery (?)

Bridge to transplant “Destination therapy”

All-comers?


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Where Are We Going?

Agree that BTT/DT lines are becoming obsolete The most robust clinical trial is still an RCT Ongoing discussions about an “all-comers” trial to

understand what is the appropriate comparator Need robust data that are appropriately audited and

adjudicated FDA believes in maximizing its role in the review and

mining of registries to analyze and interpret trial data Trial conduct and registry conduct of critical

importance


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Emphasis on Trial Conduct

Avoid missing data! Prospective plan to account for it Report amount of missing data at specific

intervals FDA encourages sponsors to closely monitor

patients and collect ALL appropriate data Data integrity and collection (audit/adjudicate) Safety and effectiveness evaluated on

TOTALITY of ALL the data


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Conclusions

FDA evolving with the field of MCSD RCT = gold standard for an all-comers trial FDA willing to accept alternative designs and

controls when based on sound, scientific rationale

Emphasis on trial conduct and appropriate data collection


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QUESTIONS?

[email protected]


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Industry: INTERMACS – The ultimate post market study

D Naftel



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Device Manufacturers

• What services do the device manufacturers receive for their fee?

I. Registry Data• Data contained in the Registry that relate only to MCSD’s

manufactured by the Company• Information is de-identified• Dataset includes all pre-implant medical conditions,

implant details, re-hospitalizations, adverse events, quality of life information, neurocognitive assessments and transplant and survival status


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Device Manufacturers (Continued)


II. Standard Reports• Provided as an aggregate basis and are de-identified • Reports include pre-implant medical conditions, implant

details, re-hospitalizations, adverse events, quality of life information, neurocognitive assessments and transplant, recovery and survival status

• Allows device manufacturers to benchmark its MCSDs against the national data for comparison purposes


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III. Company MCSD Reports• Provided as an aggregate basis and are de-identified• Report contains summaries of outcome events including

actuarial depictions and competing outcome analyses and will include all pre-implant medical conditions, implant details, adverse events, re-hospitalizations, quality of life information, neurocognitive assessments, and transplant and survival status


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IV. Device Malfunction Notifications: Upon Occurrence• Company will be notified within 72 hours if a device

malfunction occurs with respect to a MCSD manufactured by the Company

• A complete report will be sent within 45 days of the event to the Company providing full details, sufficient to enable the Company to fulfill its CFR reporting requirements to the US Food and Drug Administration.


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Hospitals: Utilizing the Quality Assurance Reports

D Schmuhl



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DARLENE SCHMUHL, RNDARLENE SCHMUHL, RN

CLEVELAND CLINICCLEVELAND CLINIC


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UTILIZING THE QUALITY UTILIZING THE QUALITY ASSURANCE REPORTSASSURANCE REPORTS

MD’S,NURSE MANAGER

PI REVIEWS ALL DATA

SITE ADMINISTRATORREVIEWS AND ANSWERS TO

DISCREPANCIES AND COMPLIANCE

DCC SENDS REPORTFEEDBACK TO DCC WITH

COMMENTS, QUESTIONS, SUGGESTIONS

PRESENTATION AND DISCUSSION OF REPORT AT MCS MEETING

ADDITIONAL DATA COLLECTIONAND ANALYSIS OF NON-CONSENTED

MCS PATIENTS


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4:00 Final Comments and Adjournment

James Young



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intermacs collaborators: nhlbi: sustainability – are we there yet?

Documents

intermacs data intermacs

intermacs partnerships

intermacs collaborators

intermacs comparator

intermacs survival curves

intermacs patient profiles

mdsixth annual meeting

millersixth annual meeting