© CDISC 2014 1

CDISC Essential Standards to Enable

Learning (ESTEL)

Rebecca D. Kush, PhD

President and CEO

IOM Digital Learning Collaborative: EHRs and Research

30 May 2014

© CDISC 2012

Research findings to inform

healthcare decisions

Information from healthcare (private, aggregated)

to enable research

•Discovery of new therapies •Understanding diseases •Testing/comparing therapies (CER) •Assessing efficacy •Monitoring safety •Understanding responses (genomics, biomarkers) •Public health/quality evaluations •Post-marketing surveillance

•Quality healthcare •Informed decisions •Personalized medicine •Patient safety and privacy •Public health •Improved therapies •Efficiencies/reduced costs

Research Healthcare

Currently

Inefficient

~17-year cycle

A National-Scale Learning Health System:

Background from the Institute of Medicine

of the National Academies

© CDISC 2014

National LHS: One Infrastructure that Supports • Research

Clinical

Comparative effectiveness

Translational

• Public Health

Surveillance

Situational Awareness

• Quality Improvement

Health process and outcomes

research

Best practice dissemination

• Consumer Engagement

Knowledge-driven decision making

Source: Dr. Charles Friedman

© CDISC 2014

Learning Health Community

• Infrastructure can enable

necessary virtuous cycle of

study, learning and

improvement

• This requires assembly of data,

analysis, and feedback

Assemble Change

Interpret

Analyze Feedback

CORE VALUES

• Person-focused

• Privacy

• Inclusiveness

• Transparency

• Accessibility

• Governance

• Cooperative and

Participatory Leadership

• Scientific Integrity

• Value

© CDISC 2014

6

© CDISC 2014

The Learning Health Community

• Grew out of the 2012 “Learning Health Summit”

• A self-organizing, multi-stakeholder coalition of the willing

• 60 “endorsers” plus > 600 others expressing interest

• “Summit” Planning Committee became the Community’s

Coordinating Committee

• Catalyzing, leading, and participating in initiatives to

realize a Learning Health System

- Standards (ESTEL)

- Governance

- Technology

© CDISC 2012

Essential Standards to Enable

Learning (ESTEL) Charter

Purpose and Scope:

To define a parsimonious/essential/minimum core set of

standards that could enable a standards-based yet flexible and

scalable LHS in accordance with the following goals:

• a) Ease the burden for any clinician to participate in a research

study or other learning activity;

• b) Increase the capacity for learning from data;

• c) Obtain knowledge and results in an actionable form to

contribute to building the LHS;

• d) Ensure that the data obtained can be readily aggregated

and/or compared; and

• e) Ensure that the data uphold scientific integrity.

8

~ December 2012

© CDISC 2012

Activities to Date Relative to ESTEL

• May 2012 – Learning Health Community Summit

• Q4 2013 – ESTEL Name and Charter

• February 2013 – ESTEL Launch @ CDISC Office

• March 2013 – ESTEL Webinar

• July 2013 – ESTEL “Exec” Group @ IOM

• September 2013 – Second ESTEL F2F @ Duke

• Oct 2013 – March 2014 - Teleconferences

• April 2014 – Third ESTEL F2F @ AHRQ

9

© CDISC 2012

Opportunities/Initiatives to Inform ESTEL

• eSource Data Interchange (eSDI) Initiative – defined

requirements (12) for using eSource (i.e. entering data

electronically - including EHRs, eDiaries) in regulated

research studies (~2006)

• eSource Guidance released by EMA (2010) and FDA

(2013)

• CDISC Healthcare Link Initiative; IHE QRPH Group

• U.S. Health and Human Services (HHS/ONC) HITSP

Interoperability Specification – IS#158 (2009-2010)

• HHS/ONC) Structured Data Capture (SDC) Initiative

(2013-present)

• EU Innovative Medicines Initiative (IMI) EHR4CR and

TRANSFoRm

10

© 2012

ASTER (AE Reporting from EHRs)

30 Ambulatory care physicians at Harvard

and Brigham and Women’s

with Pfizer, CDISC, CRIX

Nov 08 – Jun 09, > 200 Reports Sent to FDA

Physician Reporting:

*91% of participating physicians had

submitted no ADE reports in the prior year

*During the study, participants reported an

average of approximately 5 reports in a 3

month time period

*All participants reported at least 1 AD

* Process: Time to report decreased from

~35 min to < 1 min

Source: Michael Ibara, Pfizer

© CDISC 2012

The “S” Word

What is a standard? • A reference to quality

• An average cut of beef

• In the world of data and technology, there are ‘so many to

choose from’! And, there are many types of standards.

A standard is NOT proprietary.

A standard is NOT a “one-off”.

Standards are developed through a robust, authorized,

consensus-based process and maintained by a

recognized standards development organization (SDO).

True standards are mature, useful and broadly adopted.

Standards do NOT inhibit creativity, rather encourage

innovation.

12

Standard

ESTEL Launch – February 2013

© CDISC 2012

Standards and the Learning Health System

13

Essential

Standards

Standards-based

Tool Innovation

The Whole System is

Chaordic, Unmanaged,

approaching Ultra-Large Scale

The Essential Standards

lie between the

Whole System and the Tooling

The Tools come from a

Standards-driven

Innovation Marketplace

ESTEL Exec Group – July 2013

© CDISC 2012

The Hourglass Model

The narrow neck of the hourglass defines

a small set of core abstractions and

protocols (e.g. TCP and HTTP) onto

which many different high-level behaviors

can be mapped (the top of the

hourglass), and which themselves can be

mapped onto many different underlying

technologies (the base of the hourglass).

By definition the number of protocols

defined at the neck must be small.

The Anatomy of the Grid, Kesselman

14

15

The Neck Resource & connectivity

protocols.

The Collective “A wide range of global services

and application-specific

behaviors”

Fabric “Diverse range of resource

types.”

ESTEL

Tools

Healthcare Data

The Hourglass Model: LHS-ESTEL

LHS

ESTEL Meeting – September 2013

April 2014 ESTEL Meeting: Discussed

Standards Requirements and ‘Who?’, ‘What?’

and ‘How?’ relative to the Hourglass Model.

Next Step: What are the Essential Standards

for the LHS---Quality, Research, Public Health,

Consumer Engagement?

© CDISC 2014

TODAY: Research and Healthcare

Medical

Records

(Source

Documents:

Paper or

EHR)

Healthcare Delivery Medical Research

CRFs

(Paper

or

eCRFs)

Data re-entry/transcription

© CDISC 2014

CDISC Healthcare Link

Goal: Optimize the Process

Auto-

Reconciliation for

Source Data Verification

(e)CRFs Source

Documents

EHR

eSource

Healthcare Delivery

~1997

Medical Research

© CDISC 2014

Leveraging Existing Standards at the

Intersection of Healthcare and Research

• Standards FIT FOR PURPOSE, working together

synergistically

• Biomedical Research Integrated Domain Group Model

(BRIDG) (2003-Present) Collaborative, with key stakeholders: NCI, CDISC, HL7, FDA

www.bridgmodel.org

• IHE Profiles for Clinical Research – CDISC Healthcare Link

Developed through the Quality, Research and Public Health (QRPH)

Group with CDISC Leadership

Include Retrieve Form for Data Capture RFD), Research Matching (RM),

Retrieve Process (Protocol) for Execution (RPE), Data Exchange (DEX)

and others

• HL7 Continuity of Care Document (CCDA)->FIHR?

• CDISC Global Clinical Research Standards

Endorsed and/or to be required by FDA and PMDA

18

© CDISC 2014 19

© CDISC 2014

Synergistic Standards Available

eCRFs eSource

Documents

EHR

Healthcare Delivery

2014

Medical Research

ODM

HL7 CCDA

Integration

Profiles

(e.g. RFD)

© 2012

Portal,

Views

Analysis

Tools

and/or

Database

EDC

EHR

Data Sources

Care and/or Research Site (Healthcare Location,

Investigator, Site Personnel)

Study Sponsor

(e.g. ARO, CRO, Vendor, Principal Investigator,

federal agencies…)

Structured,

Pseudony-

mized

Research

Datasets &

Tables OR

Patient-level

data

EHR “A” Site

Research

Archive

CCD

EHR “B”

RFD

CCD RFD

EDC

PRO

eDiary

Potential for Staged Implementation of

Standards-based EHRs for Research

© CDISC 2012

Research findings to inform

healthcare decisions

Information from healthcare (private, aggregated)

to enable research

•Discovery of new therapies •Understanding diseases •Testing/comparing therapies (CER) •Assessing efficacy •Monitoring safety •Understanding responses (genomics, biomarkers) •Public health/quality evaluations •Post-marketing surveillance

•Quality healthcare •Informed decisions •Personalized medicine •Patient safety and privacy •Public health •Improved therapies •Efficiencies/reduced costs

Research Healthcare

To reduce

~17-year cycle and

Enable the LHS for

Research:

Technology

Workflow Enablers

Regulatory Encouragement

Synergistic Standards

Business Case

Staged Implementation

Demos and Pilots

WHAT IS MISSING for

THE REAL THING???

© 2012

• What are the remaining barriers?

• How can we remove these?

• How can we accelerate

Standards-Based EHR-enabled Research?