Happy Sweet 16months PCORnet!Update on Progress & PromiseSarah Greene, MPH, Associate DirectorCER Methods and Infrastructure Program, PCORIHealth 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 observational 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 other 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 & Figures29 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 US3000+ collaborators/contributorsMillions of patients receive care in the participating systemsPhase I = March 1, 2014 – September 30, 2015Phase 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

7

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

9

CDRN 1

PCORnet DRN Coordinating Center

PCORnet Secure Network Portal

1

52Demographics

UtilizationEtc

Review & Run Query

3

Review & Return Results

4

6

CDRN 11

DemographicsUtilization

Etc

Review & Run Query

3

Review & Return Results

4

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 easierAnalysis 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 studiesADDRESS questions important to patients and clinicians that require multi-site evaluation

ASSESS PCORnet’s ability to perform large-scale interventional andobservational 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 on a baby aspirin because it causes less bleeding and bruising. Ted is confused about what dose he should take. He does a lot of 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.

17

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

81 mg36%

162 mg3%

325 mg61%

Other0.01%

81 mg162 mg325 mgother

Hall et al. Circulation Cardiovascular Quality and Outcomes 2014

Aspirin: “wonder” drug in many waysProven 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 of PCORnet Infrastructure

20

*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 CADOR

• Revascularization (PCI/CABG)

At least one of 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

22

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

ADAPTABLEEnrollee

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

23

PCORnet Ethos and ADAPTABLEThis 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 neededAble to conduct clinical trials affordably through streamlined contracting, IRB coordination, engagement of clinicians and sites, and rapid identification, recruitment, consenting and follow-up of subjectsAble to leap tall buildings in a single boundOpenness to data linkage with other databases, (e.g., registries, CMS) for funded studiesOpen to collaboration with non-PCORnet investigators across a range of topics

Plans for Phase II: 10/1/2015-9/30/2018Officially “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: @PCORnetworklearn more: www.pcornet.orgcontact me: sgreene@pcori.orgvisit us: PCORI Booth 503!

Thank you! 27