Patients 2 Trials Consortium

Project Overview

2014-04-01

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What drives us?

We seek to do a better job at informing patients, their providers and caregivers, about the opportunities they have to volunteer for clinical research studies.

A Patient EngagementCall to ActionEngaging patients in sufficient numbers to clinical trials is a recognized challenge. An equally critical challenge is keeping them in the studies for their duration and sustaining the relationship after completion

Hard Truths• Less than 5% of Americans know where to find information about clinical trials relevant

to them(1)

• 2% of the US population and 4% of physicians ever get involved with clinical research(2)

• Between 2003-2006 clinical trial retention rates fell from 69% to 48%(3). They were 30% in 2013

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1) Ken Getz, “The Gift of Participation: A Guide to Making Informed Decisions About Volunteering for a Clinical Trial”, October 20082) Center for Information & Study on Clinical Research Participation (CISCRP)3) Tufts Center for the Study of Drug Development, “Growing Protocol Design Complexity Stresses Investigators, Volunteers” Impact Report 2008

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What’s the problem?

• 75% of the general public state they have little to no knowledge about the clinical research enterprise and the participation process(1)

– What studies do I qualify for?– What does the study do?– How often will I have to take

off from work to participate to this study?

1) Center for Information & Study on Clinical Research Participation (CISCRP)

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– What studies do I qualify for?

– What does the study do?– How often will I have to

take off from work to participate to this study?

Proposed Approach

Clinicaltrials.gov as foundation

“Target Profile” for automated filtering

“Augmented Content” for additional patient centric

content

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Proposed approach - detail

• The Target Profile is a machine readable query, that can be executed against an electronic file (or “record”) with patient health data – such as an Electronic Health Record (EHR), an Electronic Medical Record (EMR) or Personally Controlled Health Record (PCHR)

• Augmented Content is public, IRB approved information about the study that has not been published on clinicaltrials.gov, and that is shared with / targeted for patients with a matching Target Profile.

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Proposed Architecture

Closer Look at Target Profiles

Ct.GovNCT02020616

Trial : NCT02020616Indication: Type 2 Diabetes

Machine Readable Target Profile(xQuery)

Human Readable Target Profile

Inclusion21 years or olderT2DMGender

Men includedPost-menopausal womenWomen surgically sterilized

A1C: 7-10.5% Exclusion

Active insulin Rx Active TZD RxDx of renal insufficiency

SCr 1.4 mg/dL in womanSCr 1.5 mg/dL in men<60 for eGFR

Eligibility

Proof-of-Concept Approach

Machine Readable Target Profile(xQuery)

api.lillycoi.com

s MatchProcess

Human Readable Target Profile

Inclusion21 years or olderT2DMGender

Men includedPost-menopausal womenWomen surgically sterilized

A1C: 7-10.5% Exclusion

Active insulin Rx Active TZD RxDx of renal insufficiency

SCr 1.4 mg/dL in womanSCr 1.5 mg/dL in men<60 for eGFR

683 SampleCCDA EMR Records

Results

123 SampleDiabetic Records

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PoC Results

Predicted Results (Free Text)

Positive Negative

Actual Results (xQuery)

Positive a b

Negative c d

Evaluation Method Positive Matches Negative Matches Total RecordsFree Text (Human Readable) 19 104 123

Confusion Matrix

NCT02020616

xQuery (Machine Readable) 19 104 123

Evaluation Method Positive Matches Negative Matches Total RecordsFree Text (Human Readable) 59 64 123

xQuery (Machine Readable) 59 64 123

NCT01394952

Patient App Prototype

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Proposed End State

• An open platform, where:1. Study sponsors can login, and upload Target Profile,

Augmented Content for their research studies

2. Public matching services are available, to which patients or organizations can send de-identified electronic health data and find matching studies

3. Open standards for those who wish to develop their own matching services against the Target Profiles

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Our approach to Standards

• This project is not about building standards, but about market enablement.

• Therefore, while it is not the purpose to design new standards, we have to adopt them to allow “the market” to use the platform.

• We are using common standards where they are available, supplemented with pragmatic, open definitions where no standards are available yet.

• The platform itself is agnostic to standards, and can easily adopt new standards as they become available in the future.

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Guiding Principles

• We are starting small, and we will scale up as we learn through experience - “done is better than perfect”.

• We have not yet defined governance around the content of Target Profile and Augmented Content.

• We have an open transparent learning environment with robust analytics showing what’s going on.

• Fundamentally, the role of this project is to explore whether and how Study Sponsors can provide rich and patient centric information about clinical trials to enable the marketplace to provide better information to patients, providers and caregivers.

TimelinesOct’

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Project Start

April – May’14

Test version of platform1. Initial controlled testing2. Target Profiles for ~ 50 studies3. Ready for market testing

Identify partner organizations and test drive the platform.- Does it work for them?- Does it help them match patients to

trials?- How can we improve the platform?

July – Oct 14?

Q2 201

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Broad SponsorSupport

Long Term Sustainable Model Development- Who will host?- Who will pay?- Content governance?

Q1 201

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Platform/API Publically available: does this help better inform patients about trials?

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Consortium

• Novartis• Eli Lilly and Company• Pfizer, Inc.• Corengi

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Experiences to date“Not all Eligibility Criteria are created equal”:We are finding there are different types of eligibility criteria, e.g.

1. Things that the patient knows

2. Things that the doctor knows (and you could expect to find in the patient electronic health record)

3. Things that are assessed during screening

So our process for developing Target Profile, is to:4. Sit down with the Study Responsible Physicians

5. Find out which of the eligibility criteria are in category 2

6. Discuss whether and how criteria from the other categories can be replaced or approximated by additional criteria in category 2

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Actions Amongst Members

• We are looking for Studies within our organizations that are fit for purpose:– Studies that will be recruiting, or will still be recruiting, June through December

2014, and possibly beyond– Studies and study teams who are willing and able to contribute to mid-term, yet

potentially fundamental improvements in connecting patients to trials .– Study teams that are willing to participate in targeted 30 min meetings to help

define Target Profile and Augmented Content.

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Getting Involved and Q&A

• Ask us Anything – Panel Discussion