The Key to Success: INTERMACS Hospitals

1. Sixth Annual Meeting, March 12, 2012 • The Key to Success: INTERMACS Hospitals • Site Utilization of INTERMACS Data and Reports for local QI • Quality Assurance and data quality • Evaluation of Site Data: Audits, Complete Enrollment, Complete Data, etc. INTERMACS Annual Meeting March 2012

2. Sixth Annual Meeting, March 12, 2012 The Key to Success: INTERMACS Hospitals! INTERMACS Annual Meeting March 2012

3. Sixth Annual Meeting, March 12, 2012 • The Key to Success: INTERMACS Hospitals • Site Utilization of INTERMACS • Data and Reports for local QI Naftel INTERMACS Annual Meeting March 2012

4. Hospitals • What services do the hospitals receive for their participation fee? • Services • Meets CMS/Joint Commission requirement for Destination Therapy Certification • Meets FDA required submission of Medical Device Reports (MDRs) by hospitals • Provides clinical summaries of patients • Provides quality assurance reports • Provides electronic data transfer • Provides standardized datasets • Provides benchmarking • Provides training and continuing education units INTERMACS Annual Meeting March 2012

5. Hospitals (Continued) • What benefits do the hospitals receive for their participation fee? • Benefits • Fulfills CMS DT Certification requirement • Become part of the national dialogue on the evaluation and evolution of MCSDs • Invited to participate in the INTERMACS Annual Meeting • Invited to join the INTERMACS Committees • Coordinators Council and other committees • Select Hospital Administrators will have the opportunity to serve on the Business Advisory Committee INTERMACS Annual Meeting March 2012

6. Sixth Annual Meeting, March 12, 2012 • The Key to Success: INTERMACS Hospitals • Quality Assurance and data quality Naftel INTERMACS Annual Meeting March 2012

7. Fifth Annual Meeting, April 12, 2011 Hospital Perspective – Deliverables Coordinators Clinical Team Quality Assurance Officer Office of Risk Management Financial Officers / Administrators Office of Accreditation Researchers INTERMACS Annual Meeting March 2012

8. A Workshop for Researchers and INTERMACS Hospitals QA Report INTERMACS Annual Meeting March 2012 INTERMACS Data Workshop – April 12, 2011

9. A Workshop for Researchers and INTERMACS Hospitals QA Report INTERMACS Annual Meeting March 2012 INTERMACS Data Workshop – April 12, 2011

10. A Workshop for Researchers and INTERMACS Hospitals INTERMACS Annual Meeting March 2012 INTERMACS Data Workshop – April 12, 2011

11. A Workshop for Researchers and INTERMACS Hospitals INTERMACS Annual Meeting March 2012 INTERMACS Data Workshop – April 12, 2011

12. Sixth Annual Meeting, March 12, 2012 • The Key to Success: INTERMACS Hospitals • Evaluation of Site Data: Audits, • Complete Enrollment, • Complete Data, etc. Naftel INTERMACS Annual Meeting March 2012

13. Sixth Annual Meeting, March 12, 2012 1. Regulatory Requirements Regulatory requirements must be met. Assessment: UNOS collects and the DCC evaluates all regulatory documents Goal: 100% of participating hospitals meet all regulatory requirements Minimal Standard: 100% INTERMACS Annual Meeting March 2012

14. Coordinator Training, March 11, 2012 INTERMACS Annual Meeting March 2012

15. Sixth Annual Meeting, March 12, 2012 2. Timely Follow-up data Assessment: will focus on submission of follow-up forms. Goal: 100% of follow-up forms submitted within 30 days of the date of expected follow-up. Minimal Standard: 90% of follow-up forms must be submitted within 30 days of the date of expected follow-up INTERMACS Annual Meeting March 2012

16. Coordinator Training Session: March 11, 2012 Overview of Data Entry Naftel INTERMACS Annual Meeting March 2012

17. Sixth Annual Meeting, March 12, 2012 3. All Device Implants Complete Accounting of all eligible device implants. Assessment: Matching hospital enrollment to industry counts. Goal: 100% of eligible devices enrolled. Minimal Standard: 90% of eligible devices enrolled. Note1: All eligible DT devices must be entered. Note2: Informed consent is a barrier to the minimal standard. INTERMACS Annual Meeting March 2012

18. Sixth Annual Meeting, March 12, 2012 Days from Implant to Entry into INTERMACS Jun 2006 – Dec 2011 Median (days) n % < 30 50 40% 30 – 59 30 24% 60 – 89 13 10% 90 – 119 6 5% > 120 26 21% Total hospitals 125 100% INTERMACS Annual Meeting March 2012

19. Sixth Annual Meeting, March 12, 2012 Days from Implant to Entry into INTERMACS Jun 2006 – Dec 2011 Median (days) n % < 30 50 40% 30 – 59 30 24% 60 – 89 13 10% 90 – 119 6 5% > 120 26 21% Total hospitals 125 100% INTERMACS Annual Meeting March 2012

20. Sixth Annual Meeting, March 12, 2012 4. Completeness of data elements Assessment: The web-based application requires that all elements be addressed (either a data value entered or “not done” selected) before the form can be submitted. The proportion of captured data values will be calculated. Goal: 100% completion of data elements. Minimal Standard: To be determined after consultation with HSC. INTERMACS Annual Meeting March 2012

21. Sixth Annual Meeting, March 12, 2012 Completeness of Quality of Life Data will be addressed by Dr. Grady INTERMACS Annual Meeting March 2012

22. Sixth Annual Meeting, March 12, 2012 • The Key to Success: INTERMACS Hospitals • Risk adjustment: • Will require an in-depth, collaborative effort among the INTERMACS Collaborators including hospital representatives. The approach will be modeled after the SRTR evaluation of post heart transplant survival. INTERMACS Annual Meeting March 2012

23. Sixth Annual Meeting, March 12, 2012 • Update on New INTERMACS/NIH Initiatives • MedaMACS • PumpKIN • PediMACS • IMACS • Revive-IT INTERMACS Annual Meeting March 2012

24. Sixth Annual Meeting, March 12, 2012 • Update on New INTERMACS/NIH Initiatives • MedaMACS (15 min) Stewart INTERMACS Annual Meeting March 2012

25. MEDAMACS Update 2012:Medical Arm of Mechanical Circulatory Support INTERMACS - 6th Annual Meeting March 12, 2012 INTERMACS Annual Meeting March 2012

26. The MEDAMACS Mission • Map terrain of ambulatory advanced heart failure • Improve selection and timing of MCS by studying outcomes with medical therapy • Support IOM mandate for patient-centered care and shared decision making • Design integrated endpoints that move beyond survival alone • Define a broader context for the next generation of MCS clinical trials INTERMACS Annual Meeting March 2012

27. MEDAMACS Study 2012-2015 n=350 12 centers 2 yrs follow-up Screening Pilot 2010-12 n=168 10 centers 1 yr follow-up Evolution of MEDAMACS INTERMACS Specific Aim 2006, 2011 INTERMACS Annual Meeting March 2012

28. MEDAMACS Study Sites INTERMACS Annual Meeting March 2012 Brigham and Women’s U. of Alabama Birm. U. of Pittsburgh Cedars-Sinai U. of Colorado U. of Pennsylvania Cleveland Clinic U. of Iowa U. of South Florida Duke U. of Michigan U. of Texas Southwestern

29. INTERMACS Annual Meeting March 2012 • clinicaltrials.gov/NCT01452802 • clinicaltrials.gov/NCT01369407

30. INTERMACS Annual Meeting March 2012 • clinicaltrials.gov/NCT01452802 • clinicaltrials.gov/NCT01369407

31. MEDAMACS Encounters 1 Month Re-Look Baseline Telephone Contact Study Site Phone Calls 6 and 18 mos Events (hosp, stroke, transplant, vad, inotropes, death) Meds, Euroqol, NYHA/INTERMACS profile A B Inpt or outpt outpt C D Time Zero One month 1 Yr Face-to-face 18m 2 Yrs Phone Interview 6 mos Phone Interview End Baseline A Baseline B 1-Yr Visit C 2-Yr Visit D Consent 1mo. 12mos 24mos Face-to-face encounters 6MW Gait speed Euroqol+KCCQ VAD Survey Events Treatments 6MW Gait speed Euroqol+KCCQ VAD Survey Risk Scores 6MW Gait speed Euroqol+KCCQ VAD Survey Events Risk Scores Treatments 6MW Gait speed Euroqol+KCCQ VAD Survey Events Risk Scores Treatments INTERMACS Annual Meeting March 2012

32. MEDAMACS Timeline Enrollment N=350 x 12sites 2 Year Visit & Closeout 1 Year Visit STUDY SITE Spring Training IRB 6M Telephone 18M Telephone Annual Mtg @ISHLT Annual Mtg @ISHLT Annual Mtg @ISHLT 2012 2013 2014 2016 2015 ISHLT Final Pilot Analysis 1 Yr Data Complete Baseline Data ISHLT/ACC WBDE Programming DATABASE CLOSED DCC + STEERING Late-Breaking Submission OSMB Review & DCC IRB (11-12/2011) Late-Breaking Submission Double Baselines Completed 6M Data Complete Pilot Complete INTERMACS Annual Meeting March 2012 Design MedaMACS 2.0 MedaMACS Pilot Estimated # Active Pts 0 90 160 250 270 260 250 235 210 180 150

33. MEDAMACS at ISHLT 2012:Screening Pilot Data • High Event Rates in Medically Managed Advanced Heart Failure Patients Followed at VAD Centers (Stewart et al.) • Prediction of Events in Patients with Advanced Heart Failure: Application of the Seattle Heart Failure Model to the Medamacs Population (Teuteberg et al.) • Patients with INTERMACS 4-7 Heart Failure Have Reduced Quality of Life (Patel et al.) • Uric Acid Elevation is Associated with Severity of Congestion in Advanced Heart Failure (Guglin et al.) • Tricuspid Regurgitation is a Measure of Right Heart Dysfunction and is Associated with Event Free Survival in Stage D Heart Failure (Testani et al.) INTERMACS Annual Meeting March 2012

34. Event-Free Survival in Ambulatory Advanced Heart Failure 100 84% 75 68% 64% Event-Free Survival Event of Interest 50 Death Death, Transplant, or VAD Death, Transplant, VAD or Inotrope 25 INTERMACS Annual Meeting March 2012 0 0 2 4 6 8 Months since Enrollment Stewart et al. ISHLT 2012

35. INTERMACS Profiles Risk Stratify Ambulatory Advanced Heart Failure Patients 100 75 P<0.001 Survival Free of VAD or Transplant 50 INTERMACS 6/7 INTERMACS 5 INTERMACS 4 25 INTERMACS Annual Meeting March 2012 0 0 2 4 6 8 Months since Enrollment Stewart et al. ISHLT 2012

36. REVIVE-IT Event-free Survival by Seattle HFM category 100 80 60 p = 0.0003 Event-free survival (%) 40 20 0 INTERMACS Annual Meeting March 2012 0 3 6 9 Months post-enrollment SHFM < 1.5 SHFM 1.5-2.5 SHFM > 2.5 Teuteberg et al. ISHLT 2012

37. Euroqol VAS Score by HF Severity INTERMACS Annual Meeting March 2012 INTERMACS; Grady K, et al J Heart Lung Trans 2009;28:S269. MEDAMACS: Patel P, et al. ISHLT 2012. HF Action; Flynn K, et al. Am Heart J 2009;158:564-71.

38. Team MEDAMACS UAB/DCC David Naftel Mary Lynne Clark Susan Myers Craig Collum Kathryn Hollifield NHLBI/NIH Monica Shah Tim Baldwin Marissa Miller Thoratec Corporation INTERMACS Annual Meeting March 2012

39. Coordinator Training Session: March 11, 2012 pediMACS David C. Naftel, PhD INTERMACS Annual Meeting March 2012

40. Coordinator Training Session: March 11, 2012 INTERMACS Re-Launch Status • Our Goals: • Reduce the number of elements and forms • Streamline the data entry process • Clarify elements that were confusing in the past • Examine the AE definitions for current clinical relevance • It became clear that the pediatric VAD patients needed a different web-based data entry system. INTERMACS Annual Meeting March 2012

41. Coordinator Training Session: March 11, 2012 pediMACS Launch Status • Pediatric Committee • Betsy Blume, MD - Chair • David Morales, MD • David Rosenthal, MD • Peter Wearden, MD • Christopher Almond, MD • Robert Jaquiss, MD • Jonathan Chen, MD • Dee Dee Epstein, RN • Heidi Moses, MEd, CCRA • David Naftel, PhD • Tim Baldwin, PhD INTERMACS Annual Meeting March 2012

42. Coordinator Training Session: March 11, 2012 pediMACS Launch Status • We (Pediatric Committee, NIH, INTERMACS Co-PIs) have spent 1 year reviewing the WBDE in all aspects: • AE definitions and other definitions • Screens / Forms • Data Elements • Patient Flow through the WBDE INTERMACS Annual Meeting March 2012

43. Coordinator Training Session: March 11, 2012 pediMACS Launch Status • pediMACS will follow the structure of INTERMACS • A few important changes from INTERMACS: • Pediatric patients (< 19 yrs. at time of implant) • Includes both durable and temporary support MCSDs • Modifications of AE definitions • Possible expansion of quality of life instruments INTERMACS Annual Meeting March 2012

44. Coordinator Training Session: March 11, 2012 pediMACS Launch Status • Target Live test site: July 1, 2012 • Testing by INTERMACS Nurse Monitors • Testing by the DCC Data Managers • Testing by the INTERMACS Co-PIs • Testing by 3 Hospitals (Beta Sites) • Testing by Pediatric Committee • Target Launch Date: August 1, 2012 • Online training will be available • Training Session in September 2012 Target: LAUNCH Date Aug 1, 2012 Target: Live Test Site July 1, 2012 Jul 1 - Jul 31 INTERMACS Annual Meeting March 2012

45. Coordinator Training Session: March 11, 2012 pediMACS Launch Status • Training Session for pediMACS • “Mechanical Cardiac Support in Pediatric Heart Disease – State of the Art 2012”: September 20-22, 2012 • The St. Louis Children’s and Washington University Heart Center INTERMACS Annual Meeting March 2012