intermacs annual meeting presentation 2007

1. First Annual Meeting April 24, 2007 Hilton, San Francisco 333 O�Farrell Street Room: Plaza B 2:00 pm to 6:00 pm

4. www.intermacs.org Please visit the website frequently for INTERMACS updates Slides from this meeting will be posted on the website mid-May Slides from the 4 INTERMACS presentations at ISHLT will be posted on the website mid-May

5. Meeting Agenda INTERMACS Brochure List of Activated Sites Device Brand List Subcommittee Structure CMS: Decision Memo for VADs as Destination Therapy FDA Perspective INTERMACS Presentations at ISHLT Real Life Problem Solving: 2010

9. Registry Create Implement Maintain Scientific Patient selection Analyses of outcomes Best practice guidelines Next generation devices

20. Pre-INTERMACS Industry FDA INTERMACS UNOS

21. International INTERMACS Europe, Asia, etc. Harmonization by Doing

22. Which patients/devices should be entered into INTERMACS?

23. The scope of INTERMACS encompasses those patients and mechanical circulatory support devices (MCSD) for whom discharge from the hospital is feasible. Devices that are not designed for discharge, such as the intra-aortic balloon pump or ECMO, will be excluded. Patients receiving such short-term support could be entered into INTERMACS if they subsequently undergo implantation with an eligible device. Patients receiving an eligible device will be entered in the Registry even if they receive such a device in combination with another device for limited-term support that does not qualify for Registry inclusion. Patients receiving the included devices will be registered regardless of initial intent and anticipated duration of support (see Inclusion Criteria)

25. Approved Devices (Durable, potential for patient discharge): These devices should be entered into INTERMACS except in rare circumstances where a patient with an approved device is in the control arm of an FDA approval study. Position Indication AbioCor TAH TAH BTT HeartMate IP L BTT HeartMate VE L BTT/Dest HeartMate XVE L Dest MicroMed DeBakey VAD - Child L BTT/Ped Novacor PC L BTT Novacor PCq L BTT SynCardia CardioWest TAH BTT Thoratec IVAD L/R BTT Thoratec PVAD L/R BTT

26. Device Brand List Approved Devices (Temporary) Appendix K Approved Devices (Temporary): These devices should not be entered into INTERMACS (unless they accompany a durable approved device listed above). They do not meet the INTERMACS definition for �potential patient discharge�. Position Indication Temporary Abiomed AB5000 L/R BTT Yes Abiomed BVS 5000 L/R BTT Yes Biomedicus R Yes Levitronix Centrimag L/R BTT Yes Tandem Heart L/R BTR Yes

31. Registry Create Implement Maintain Scientific Patient selection Analyses of outcomes Best practice guidelines Next generation devices

36. Section 2.0 What are the benefits of participating in INTERMACS? Current Participation in INTERMACS will require inclusion of all patients implanted with devices designed for chronic implantation, regardless of initial intent. Revised Participation in INTERMACS will require inclusion of all patients implanted with approved devices designed for chronic implantation, regardless of initial intent.

37. Section 4.0 Participation Requirements Current Before you begin entering patient data into INTERMACS, your medical center must complete steps 1 through 5. Step 1: IRB Approval Step 2: Participation Agreement Step 3: Conflict of Interest Disclosure Form Step 4: Training Step 5: Certification Revised Before you begin entering patient data into INTERMACS, your medical center must complete steps 1 through 6. Step 1: IRB Approval Step 2: Participation Agreement Step 3: Conflict of Interest Disclosure Form Step 4: Training Step 5: Certification Step 6: Human Subject Training

38. Section 4.3 Conflict of Interest Current Before you begin entering patient data in INTERMACS, your Principal Investigator is required to complete the Conflict of Interest Disclosure Form. Revised Before you begin entering patient data in INTERMACS, your Principal Investigator and Co-Investigator are required to complete the Conflict of Interest Disclosure Form.

39. Section 4.4 Training Current Before entering patient data in INTERMACS, your center is required to send at least one staff coordinator for training on all aspects of INTERMACS. For more information about INTERMACS training please call the INTERMACS Coordinator at (804) 782-4077. Revised Before entering patient data in INTERMACS, your center must complete the online INTERMACS training by going to www.intermacs.org and clicking on �INTERMACS Membership� to complete the process. For more information, please contact Molly Massey, INTERMACS Registry Lead at (804) 782-4077 or masseymb@unos.org or you may contact INTERMACSSupport at support@intermacs.org.

40. Section 4.5 Certification and Audit Process Current To MAINTAIN CERTIFICATION, a site must 5. Maintain annual IRB approval 6. Maintain annual Conflict of Interest Disclosure 7. Correct UNOS-identified errors in INTERMACS 8. Respond to queries from INTERMACSSupport 9. Participate in annual meetings 10. Comply with data submission requirements outlined in the study protocol Revised To MAINTAIN CERTIFICATION, a site must 5. Maintain annual IRB approval 6. Maintain annual Conflict of Interest Disclosure 7. Maintain Human Subjects Training 8. Correct UNOS-identified errors in INTERMACS 9. Respond to queries from INTERMACSSupport 10. Participate in annual meetings 11. Comply with data submission requirements outlined in the study protocol

42. Section 5.5 - User's Guide Proposed INTERMACS Eligibility Screen

43. Section 5.8.1 Adding a Device Allows for entry of multiple devices for an individual patient. Limited Most recent device for a patient must be removed and all forms related to the implant must be completed and validated. The patient must be alive in order to add a new device. Once the requirements have been satisfied the �Add Device� icon is available for the entry of a new device for the patient.

44. Section 6.8 User's Guide Device Malfunction Form

45. Section 6.8 User's Guide

66. FDA Interests Clinical trial design Patient safety Post-market surveillance (pre-market) Patient outcomes Harmonization by Doing Initiative International effort to promote global regulatory harmonization (Japan/US)

67. Current Situation Majority of VAD/TAH approvals require a post-market study Track real world usage of the device or to monitor safety concerns present during the clinical trial Clinical trial provides valid scientific evidence on a device for device approval Manufacturers collect post-market study data individually

68. INTERMACS: An EffectivePost-market Tool Provides Enhanced Surveillance: AEs, Device Malfunctions QOL Survival Develops �Best Practices� (reducing complications) Provides means for designing & conducting post-approval studies in cost efficient way Allows manufacturers to obtain data from INTERMACS to fulfill post-market requirements

69. Some considerations Current post-market studies stress testable hypotheses, when appropriate �Real world� usage occurs in a patient population extending beyond that circumscribed in a clinical trial Post-market studies could provide data that may lead to modifications to labeling or other interventions when appropriate Which adverse events?... Dependent on indication (BTT or DT)?

70. Key Points Standardized adverse event definitions Manufacturers and user facilities must fulfill their own specific reporting requirements and participation in INTERMACS can help with this process FDA informed participating INTERMACS user facilities that they are exempt from MDR reporting; however devices not collected by INTERMACS follow normal MDR reporting Participating manufacturers may receive extended time to make MDR submissions (pending) the automated INTERMACS MDR reporting process INTERMACS data will be helpful for manufacturers as they develop other clinical trial designs

71. What�s next? What additional benefits to pre-market evaluations will be derived from post-market studies? Can a registry provide valid scientific data to support a pre-marketing application? How should pooling/analyses of post-market study data be performed? Develop guidelines for advanced clinical trial designs using post-market study data (critical path initiative)

72. HBD Working Group 2 To conduct simultaneous post-market studies in Japan and the United States in order to obtain �real world� data on mechanical circulatory support devices (MCSD) usage in patients Continue discussions with all participants to allow Japanese centers to submit MCSD data into INTERMACS

73. HBD Interests Japanese data can help with development of control data Develop global clinical management for MCSD patients Combined review of marketing/investigational application

74. Future Satisfies CMS reporting requirement INTERMACS data can be a source of public information Status of redacted MDRs and post approval studies are available through FDA�s website Can innovative clinical trials be developed from INTERMACS data?

75. Changes to Medicare Coverage:Ventricular Assist Devices for Destination Therapy April 24, 2007 JoAnna Baldwin, MS Coverage and Analysis Group Office of Clinical Standards and Quality joanna.baldwin@cms.hhs.gov

76. Medicare Coverage of VADs: Summary of Coverage Policy Medicare provides coverage for three clinical indications (NCD Manual 100-3, �20.9) Post-cardiotomy (covered since 1993) Bridge to transplantation (covered since 1996) Destination therapy (initially covered in 2003 with updated facility criteria in 2007) Full Policy: http://www.cms.hhs.gov/mcd/index_list.asp?list_type=ncd#PV And scroll to �Artificial Hearts and Related Devices�

77. VAD Destination Therapy Updated Facility Requirements At least one surgeon on the hospital�s VAD team must have experience implanting at least 10 VADs (as bridge to transplantation or as destination therapy) or artificial hearts during the recent 36 months; and Participation in INTERMACS; and Obtain Joint Commission certification under the Disease Specific Certification Program for Ventricular Assist Devices by 3/27/09; and Hospitals listed on the existing approved facilities list must meet the updated criteria

78. Currently Approved VAD Destination Therapy Facilities Letters were sent to the 69 currently approved hospitals Hospitals must provide a written response Including a list of the experience of a least one VAD surgeon with the requisite volume (date of implantation, type of device, clinical indication for which it was implanted) Including a statement attesting to INTERMACS participation Signed by a high level hospital official (e.g., CEO, CFO, etc.) Received within 90 days

79. Hospitals That Are Not Currently Approved Hospitals that are not currently on the Medicare approved list may gain approval at any time by becoming certified through the Joint Commission http://www.jointcommission.org/

80. Additional information is available at: