Happy Sweet 16 months PCORnet! Update on Progress & Promise - - PowerPoint PPT Presentation

Happy Sweet 16

monthsPCORnet!

Update on Progress & Promise

Sarah Greene, MPH, Associate Director CER Methods and Infrastructure Program, PCORI

Health Datapalooza 2015

PCOR...what? Pink Cornet? No, PCORnet!

Patient-Centered Outcomes Research network

PCORnet is PCORI’s flagship initiative to build an efficient and patient-centered platform for

• bservational and interventional research

Why? Hasn’t this ground already been covered?

Challenge-tunity: a reusable national infrastructure for clinical research

Clinical trial coordinating centers, large research networks are often established for one purpose or one funding cycle

• New trial or new condition often begets construction of new infrastructure

Persistent inefficiencies in the current research process, from regulatory and operational, to recruitment and data collection hinder the research enterprise Most research doesn’t harness the full potential of electronic health record (EHR) data

PCORnet: Big Data, Easier Processes, Co-Creation by Stakeholders

A data platform is needed to support PCORnet, but we also need the data to have utility in real-world healthcare Through PCORnet, we will have ongoing input from patients, clinicians, health system leaders, payers and

• ther stakeholders on priority research topics

Engagement is the “special sauce” that will enable PCORnet to provide the answers patients need more quickly and efficiently, and at lower unit cost, than has ever been possible

Vision for PCORnet: enable rapid, large-scale, patient-centered clinical research in our healthcare systems and communities

PCORnet is about Research Infrastructure Done Differently PCORI is about Research Done Differently

Engagement is the cornerstone

PCORnet Facts & Figures

29 networks + Coordinating Center

• 11 Clinical Data Research Networks
• 18 Patient-Powered Research Networks
• Of the 18, nine are focused on rare diseases

155 involved organizations across the US 3000+ collaborators/contributors Millions of patients receive care in the participating systems Phase I = March 1, 2014 – September 30, 2015 Phase II = October 1, 2015 – September 30, 2018 ≈$275M: PCORI investment in infrastructure and initial projects

Complementary and synergistic capabilities in the 2 types of networks

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Diverse Sources + Rich Data = High Potential

Electronic Health Records Patient- Powered Registries Clinical & Translational Science Awardees Federally Qualified Health Centers Health Information Exchanges Integrated Delivery Systems Academic Health Centers Disease Advocacy Groups Pharmacy Data Vendors Data from Payers (e.g., CMS) mHealth / Patient- Generated Data Biospecimen Data

Rapidly Growing Data Capabilities

PCORnet Common Data Model builds on prior data models (HMORN Virtual Data Warehouse, FDA Sentinel) Complementary to and compatible with both OMOP and i2b2 Most networks have harmonized local data to CDM Version 1.0 16 months in, we just ratified Version 3.0 of PCORnet CDM Readying data to be “interrogated” for various research questions PopMedNet will be used as the secure querying engine

• 10 of 11 CDRNs and 8 of 18 PPRNs established on PopMedNet

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CDRN 1 PCORnet DRN Coordinating Center PCORnet Secure Network Portal

1 5 2

Demographics Utilization Etc Review & Run Query

3

Review & Return Results

4 6

CDRN 11

Demographics Utilization Etc Review & Run Query

3

Review & Return Results

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• 1. User creates and

submits query (a computer program)

• 2. Individual

CDRNs/PPRNs retrieve query

• 3. CDRNs/PPRNs

review and run query against their local data

• 4. CDRNs/PPRNs

review results

• 5. CDRNs/PPRNs

return results via secure network

• 6. Results are

aggregated

Rather than bringing the data to the question, PCORnet is designed to bring the question to the data

Guiding principle: make research easier

Analysis ready data

• Standard format
• Harmonized definitions
• Quality checked in advance

Reusable analysis tools Efficient clinical trial enrollment and follow up mechanisms Simple, pragmatic studies integrated into routine care Administrative simplicity (e.g., streamlined contracting) Initial PCORnet demonstration projects will help assess end-to-end functionality: design, implementation, analysis, and reporting

Demonstration projects will move PCORnet from concept to action

Aspirin Clinical Trial (ADAPTABLE) Obesity Observational Studies

• Bariatric Surgery Outcomes
• Antibiotics and Weight Gain in Children

PPRN Research Demonstration Projects Health Systems Demonstration Project NEXT-D Initiative with CDC Discussing topics of interest to health plans

Goals of PCORnet demonstration studies

ADDRESS questions important to patients and clinicians that require multi-site evaluation ASSESS PCORnet’s ability to perform large-scale interventional and

• bservational research

FACILITATEcollaboration between networks GUIDE further development of policies, procedures, infrastructure ROAD-TEST data and networking capabilities ENSURE PCORnet’s privacy protecting data infrastructure and analysis capabilities are sound DEVELOP efficient methods for identifying, enrolling, and following potential clinical trial participants

“Triple Aim” for Research: broad applicability and appeal to researchers, funders, health systems, payers, and patients PCORnet Goal: Capacities in place to support all three types of research

Interventional Trials

Rapid Cycle Research on Healthcare Delivery Observational Studies

Aspirin Dosing: A Patient-Centric Trial Assessing Benefits and Long-term Effectiveness (ADAPTABLE) Trial

PCORnet’s First Pragmatic Clinical Trial

Illustrating the Need for an Aspirin Trial: Case Scenario

Ted had chest pain while working and was taken to the ER where he learned he was having a heart attack. Doctors told him that plaque was building up in his arteries. Upon discharge from the hospital, Ted was advised to take 325mg of aspirin each day (regular strength). Ted compared notes with a friend who said his doctor has him

• n a baby aspirin because it causes less bleeding and bruising.

Ted is confused about what dose he should take. He does a lot

• f work outdoors and carpentry. He is worried about bleeding

while working but doesn’t want another heart attack either. And now Ted wonders what he should do.

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Distribution of aspirin dosing at time of hospital discharge has been shown to vary

81 mg 36% 162 mg 3% 325 mg 61% Other 0.01%

81 mg 162 mg 325 mg

• ther

Hall et al. Circulation Cardiovascular Quality and Outcomes 2014

Aspirin: “wonder” drug in many ways

Proven clinical benefit in reducing ischemic vascular events Cost-effective Benefit with combination antiplatelet therapies But there are issues:

• Emerging evidence for dose modifiers (e.g., ASA resistance, genetics)
• Equal efficacy across patients?
• Intolerance

Most effective dose uncertain— ADAPTABLE Trial aims to resolve that uncertainty

Trial Logistics: Taking Advantage

• f PCORnet Infrastructure

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*Enrichment factors

• age > 65 years
• creatinine > 1.5
• diabetes
• known 3-vessel CAD
• current CVD &/or PAD
• known ejection

fraction <50%

• current smoker

Study design

Patients with known coronary artery disease (MI, or CAD or Revasc) + ≥1 “enrichment factor”*

Identified through EHR/direct patient consenting in CDRN & PPRN clinics/hospitals (PPRN patients will need to connect through a CDRN to participate)

• Pts. contacted electronically with trial information and eConsent;

treatment assignment will be provided directly to patient ASA 81 mg QD ASA 325 mg QD Electronic follow-up quex at 4 months; supplemented with EHR/CDM/claims data Duration: Enrollment over 24 months; maximum follow-up of 30 months Primary Endpoint: Composite of all-cause mortality, nonfatal MI, nonfatal stroke Primary Safety Endpoint: Major bleeding complications

Goal n = 20.000

Use EHR data to create “computable phenotype” that can identify patients at participating CDRNs

History of CAD

• Past MI

OR

• Past cath showing

significant CAD OR

• Revascularization

(PCI/CABG) At least one

• f the following:
• age > 65 years
• Creatinine > 1.5
• Diabetes,
• Known 3 vessel coronary artery

disease

• Current cerebrovascular

disease and/or peripheral artery disease

• Known ejection fraction <50%,
• Current smoker

Obtain consent, electronically when possible

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Centralized follow-up will also be electronic whenever possible

4 12 16 30 DCRI FOLLOW-UP

• Patient Reported Outcomes
• Medication use
• Health outcomes

Baseline Data

ADAPTABLE Enrollee

8 20 …. OR CMS Virtual Data Warehouse FOLLOW-UP

• Longitudinal health outcomes

HeH FOLLOW-UP

• Patient Reported Outcomes
• Medication use
• Health outcomes

PCORNet Coordinating Center FOLLOW-UP

• Via Common Data Model
• Longitudinal health outcomes

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PCORnet Ethos and ADAPTABLE

This is a novel clinical trial employing novel methods Large group of pioneering networks and people, working together to leverage different experiences, skills, expertise (patients = co-creators) ADAPTABLE needs to be adaptable! If successful, ADAPTABLE will:

• Help solve the challenge and demonstrate the value of a reusable

infrastructure

• Launch a new era for pragmatic clinical trials and observational research to

answer questions with high impact on population health Public Health Impact: Annually, in the US alone, getting the dose of aspirin right could save up to tens of thousands of lives &/or prevent heart attacks (and prevent thousands of major bleeding episodes)

16 months later, a national resource is coming into view

Able to conduct large observational studies affordably using a common data model, distributed querying, sharing of data when needed Able to conduct clinical trials affordably through streamlined contracting, IRB coordination, engagement of clinicians and sites, and rapid identification, recruitment, consenting and follow-up of subjects Able to leap tall buildings in a single bound Openness to data linkage with other databases, (e.g., registries, CMS) for funded studies Open to collaboration with non-PCORnet investigators across a range of topics

Plans for Phase II: 10/1/2015-9/30/2018

Officially “open” for business (Fall 2015) Begin enrollment for ADAPTABLE Launch additional PCORnet studies already in the pipeline Expand the number of PPRNs and CDRNs Identify and initiate studies that are attractive to and/or proposed by other entities (industry, NIH, payers) Fine-tune infrastructure based on demonstration projects Determine business model and business plan that lead to a durable and sustainable research infrastructure Listen and learn from our incredible researchers & stakeholders

In the meantime…

follow us: @PCORnetwork learn more: www.pcornet.org contact me: sgreene@pcori.org visit us: PCORI Booth 503!

Thank you!

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