Registry Data Collection in Pediatric VADs: Challenges and OpportunitiesINTERMACS Eighth Annual Meeting

May 5, 2014

David Rosenthal, MD

Stanford Children’s Health

Disclosures

• Research support for Berlin Heart

Post-Approval Study

Heart FailureHeart

Failure

Scope• Discussing Pediatric

Cardiology care

environment

(resemblance to

adult circumstances

possible but

unintentional)

• Taking population-

based perspective

rather than just VAD

VADVAD

Transplant

Transplant

Types of Registries

• Procedure/Therapy-based

• Disease-based

• Population-based

Purposes of Registries

• Support Quality Measurement

• Provide Feedback to Clinicians/Institutions

for Quality Improvement

• Conduct Clinical Research

• Evaluate healthcare access

and outcome disparities

Registry Financial Support

• Researchers

• Research Consortia

• NPO’s

• Government Agencies

• IndustryWikipedia.com

Delicate Balance

Michelangelo: artforbreakfast.org

Funding

Investigators

Context

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

RegistryRegistry

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

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)

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

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

Key Questions In Pediatric VADs

Key Questions In Pediatric VADs

Who Should Get a VAD?

• Risk Factor/Patient selection

• Comparison with HF medical group

• Comparison with immediate

transplantation group

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

Robust Registry Environment

• Adequate numbers of registries

• Problems

– Overlapping scope

• proliferating analyses

• Inefficient workflow

– Definitions not coordinated

– Linkages not optimal

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

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