1. Registry Data Collection in Pediatric VADs: Challenges and Opportunities INTERMACS Eighth Annual Meeting May 5, 2014 David Rosenthal, MD Stanford Children’s Health

2. Disclosures Research support for Berlin Heart Post- Approval Study

3. Heart Failure Scope Discussing Pediatric Cardiology care environment (resemblance to adult circumstances possible but unintentional) Taking population-based perspective rather than just VAD VAD Transplant

4. Types of Registries Procedure/Therapy-based Disease-based Population-based

5. Purposes of Registries Support Quality Measurement Provide Feedback to Clinicians/Institutions for Quality Improvement Conduct Clinical Research Evaluate healthcare access and outcome disparities

6. Registry Financial Support Researchers Research Consortia NPO’s Government Agencies Industry Wikipedia.com

7. Delicate Balance Michelangelo: artforbreakfast.org Funding Investigators Context

8. Registries vs. RCT’s RCT – Best for assessment of therapeutic efficacy Registry – Therapeutic effectiveness – Safety/harm of therapy – Generalizability to populations Key Difference – Registries do not randomize Ho, Circulation 2008, 1675-84 Registry

9. VAD RCT Costs Berlin Heart EXCOR trial – $6,200,000 for 109 subjects – 95 non-IDE subjects – $30,000 - $57,000 per patient Berlin Heart PAS – Less rigorous auditing required – Estimated cost $2,000,000 – 50 subjects – $40,000 per subject

10. VAD Registry Costs: Intermacs 28 months from 12/10- 3/13 – $6,170,000 – $2.6M/year Size of registry at that time – 2000 enrollments/year – 5,570 total accruals – 140 active hospitals $350 - $500 per new pt Does not include data entry costs at sites: – ($1,000-$2,000 per patient)

11. Registry Quality What can be done to ensure high quality data in a registry? – Comprehensive enrollment (minimize missing cases) Adherence to well specified enrollment critieria – Ensure data completeness Varies very widely depending upon the field – e.g 1 year QOL vs. Implant date Enhanced by regular audits Data definitions important Workflow critical, compared to clinical workflow Training of those who collect/enter data Both AHA and AHRQ have published guidelines to assist this process. The more boxes you check, the more expensive the process becomes

12. Enhancing Registry Value Data Linkages – Claims data: Hospital utilization Mortality Operates effectively at large scales – Other clinical registries Disease registry linked to procedure registry Integration to EHR – Improved workflow – However, EHR vendor development goals are to differentiate product rather than to enhance interoperability

13. Key Questions In Pediatric VADs Risk Factors – Who should receive a VAD? – How to extend benefits of VAD support to subpopulations such as ACHD, single V Management Refinement – Optimal anticoagulation Device Selection – Device comparison Adverse Events – Rate of VAD failure/replacement – Rate and severity of neurologic events – Other AE’s with long-term consequences such as allosensitization Key Outcomes – Mortality – Transplant rates/outcomes – Recovery – Cost Quality Improvement – How can we improve these outcomes? – How can we ensure that outcomes are consistently good at different centers?

14. Key Questions In Pediatric VADs Risk Factors – Who should receive a VAD? – How to extend benefits of VAD support to subpopulations such as ACHD, single V Management Refinement – Optimal anticoagulation Device Selection – Device comparison Adverse Events – Rate of VAD failure/replacement – Rate and severity of neurologic events – Other AE’s with long-term consequences such as allosensitization Key Outcomes – Mortality – Transplant rates/outcomes – Recovery – Cost Quality Improvement – How can we improve these outcomes? – How can we ensure that outcomes are consistently good at different centers?

15. Who Should Get a VAD? Risk Factor/Patient selection Comparison with HF medical group Comparison with immediate transplantation group

16. Relevant Registries INTERMACS/Pedimacs: VAD procedure and follow-up PHTS: Heart Transplant listing and follow-up PHIS+: Pediatric Hospital data STS: surgical procedures and hospital stay PCMR: cardiomyopathy outpatient registry Pediatric heart failure: not yet

17. Robust Registry Environment Adequate numbers of registries Problems – Overlapping scope proliferating analyses Inefficient workflow – Definitions not coordinated – Linkages not optimal

18. Road Ahead Continue the difficult work of building participation in pediatric VAD, and transplant registries, and of enrolling patients Fill in gaps in pediatric HF Balance privacy and site concerns, while enhancing linkages between registries

19. A Pressing Need Develop infrastructure to coordinate these registries – This is currently the most obvious missing piece in the ecosystem and one that threatens long-term sustainability – Requires support from a convening body, likely a major financial supporter of registries NIH: wikipedia.org