Sharing Clinical Trial Data

Robin Jenkins

De-identification Symposium: Preserving Privacy AND

Advancing Data Analytics

October 21, 2014

1

Disclaimer

• The opinions expressed in this presentation are my own, and not those of either Sanofi US or Project Data Sphere, LLC. The presentation is for informational purposes only.

• There are no representations or warranties expressed or implied, with respect to the usefulness or sufficiency of the information for the purposes of clinical trial sharing.

1: Savage & Vickers, 2009. PLoS One

FDA sees huge opportunities in opening up

drug dataLONDON | Mon Dec 5, 2011 9:23pm IST

(Reuters) - Regulators and drugmakers need to find ways to make

more clinical data openly available, since vital knowledge about

fighting disease is often locked away in confidential databases, the

head of the U.S. drugs watchdog said on Monday. Food and Drug

Administration (FDA) Commissioner Margaret Hamburg said opening

up data to public scrutiny needed to be done selectively, given

legitimate concerns among companies over commercial

confidentiality, but more could still be done.

The Data Sharing Landscape

3

The Data Sharing Landscape

● Public pressure has been

mounting for the pharma

industry to be more ‘transparent’

about the results of clinical trials

● “Transparency Campaigners”

● 2013 - EMA proposed to release

all data they hold once a drug

has been approved

● Industry concerned about the

implications of this for

competitiveness, IP, data

protection

4

The Data Sharing Landscape

● PhRMA working group drafted set of voluntary

guiding principles for the Responsible Sharing of

Clinical Trial Data.

● Adopted by PhRMA and EFPIA in July 2013.

● Came into effect Jan 1, 2014

● Five ‘commitments’

1. Enhancing Data Sharing with Researchers

2. Enhancing Public Access to Clinical Study Information

3. Sharing Results with Patients who Participate in Clinical Trials

4. Certifying Procedures for Sharing Clinical Trial Information

5. Reaffirming Commitments to Publish Clinical Trial Results

| 5

The Data Sharing Landscape

● EMA, after stakeholder

feedback from the industry,

has re-thought it’s data

sharing proposals

● Policy final Oct 2014 and will

come into effect Jan 1 2015

● Clinical Study Reports will

become publically available

upon decision of the

application

6

Access Models: One size doesn’t fit all..

1. Black Box, or Database Query model:

● May be suitable for some types of very sensitive health information

2. Gatekeeper Model:

● PhRMA / EFPIA principles

● Independent review board

● Limited access, and broad data

● Protection of Confidential Commercial Information

3. Broad Access Model:

● Immune Tolerance Network, Sage Bionetworks, and Project Data Sphere

● No Independent Review Panel

● Responsible-use attestation

● Broader access, but less data. Data integration. Crowd

7

The Data Sharing Landscape

● Sage Bionetworks - Synapse

● Immune Tolerance Network

● ImmPort

● PhRMA / EFPIA

8

● Lead to a better understanding of disease progression and

endpoints

● Promote more efficient research:

● Improved trial design and statistical methodology

● Secondary hypotheses & epidemiology

● Disease model development

● Smaller trials sizing

● Reduced duplication & increased transparency

● Real world corroboration with trial data

● Data standards & meta-analysis

● Analytics and insights

● Unknowns2

How may sharing data accelerate researchto benefit patients?

1: Vickers 2006

2: www.cardia.dopm.uab.edu: 475 publications from a single large dataset

Benefits of Sharing Data

www.clinicalstudydatarequest.com

Multi-sponsor Consortium

11

www.clinicalstudydatarequest.comHigh-level Process

● Request access to data via Scientific Proposal

● Technical requirements met – e.g. statistician, publication plan, statistical

plan, etc…

● Reviewed by Independent Panel of Experts

● If approved, will sign data use agreement and will have access to the data

for 12 months

12

Researcher

selects required

trials from list of

those requestable.

Researcher

completes

proposal request

form online

Independent

Review Board

considers request

If approved,

researcher signs

Data Use

Agreement with

Sanofi, data are

de-identified and

uploaded to SAS

hosting site

Technical review

www.clinicalstudydatarequest.comMetrics

● September YTD

● 74 proposals submitted

● 56 met requirements

● 51 IRP approved

● 19 In process

● 32 active projects

13

The Reason Why

● 8.2 million people die everyyear from cancer whilehistorical data sets withuntapped potential sit inwarehouses gathering dust.1

● According to the WHO,more than 10m people willdie from cancer annually bythe year 2020.

15

● Cancer is a major public health concern worldwide: more peopledie every year from cancer than HIV, malaria, and tuberculosiscombined.2

1International Agency for Research on Cancer (IARC) GLOBOCAN 20122Matthers and Loncar (2006) PLoS Medicine 3(11) e44.

16

The Project Data Sphere initiative

● Independent, voluntary, not-for-profit initiative of the CEO Roundtable on Cancer’s Life Sciences Consortium

● One place to broadly share, integrate, and analyze cancer trial data ● from academic and industry Phase III clinical

trials

● historical, comparator arm data

● raw patient level data which are required to be de-identified by the data provider, data dictionary, protocols and CRFs

● State of the art analytic tools provided by SAS to free of charge to registered users within the PDS environment

www.projectdatasphere.org

Key Accomplishments

Our team spent over 2 years addressing historical barriers to data sharing:

Collaboration with privacy, academia, legal,technology, clinical, industry and patientrepresentatives to achieve an optimal platform

PDS ConceptFormed

20

11

Analytics

Legal

Privacy

Resources

300M+ Media Impressions at

Launch:April 8, 2014

17

People, Process and Technology

231 22 21 735

In Preparation

4,300

In Platform

8,921

To Achieve

our Goal:

11,779

Strategic

Pillar Summary Status

+Patients Lives are now at 9,000. We

anticipate 2-4 additional data sets in

October

-Still need to identify data sets representing

11,000 patient lives to meet the 25,000

patient lives goal by April, 2015

+Launch preparations for the Prostate

Cancer Research Challenge are underway.

+Website community tools launched in

September. User communications about the

new functionality are underway.

Nurture

Network

Expand

Use

Grow

Data

At Launch:

4,000

Potential Applications of Comparator-Arm Data

Unused data is an untapped resource and a missed opportunity; the multiple potential benefits include:

● Enable computer simulations and virtual control arm development

● Scientific advances from computer simulations and modeling could evolve clinical development to an era of data-driven decisions

● Optimized trial design and statistical methodology

● Disease modeling - clearer understanding of the natural history of tumor types/subtypes and rare disease

● Quick research – establish baseline rates for safety events

● Streamline protocol development / CRF development

● Build description of timelines of events related to therapies

20

What is the Prostate Cancer Research Challenge?

21

• Innovative, crowd-sourcing research project

• Collates Phase III prostate data from the Project Data

Sphere platform and makes it available to address

key research questions.

• Developed in collaboration with the Prostate Cancer

Foundation, Sage Bionetworks, The Dream Project,

and UNC.

• Novel approaches resulting from the challenge may

hold promise for improving treatments and foster

new collaborations.

National Cancer Institute & Mathematical Models

22

National Cancer Institute (NCI) is developing a significant Comparator-

Arm use case based on the Project Data Sphere initiative

● Early research demonstrates that analysis

of Project Data Sphere prostate cancer

data allows for comparison of treatments

that have not been previously formally

conducted.

● Additionally the research is establishing a

pathway for virtual simulations to inform the

design and development of new drugs and

the clinical trials which enable them.

Cancer Commons

23

● Cancer Commons using the platform as data source to: ● Validate their analytics

● Show patients/physicians what others have done/ experienced with their cancer treatment

● combined with other data sources and educational content to helps patients better understand their treatment options and decisions.

● Help answer the most important question cancer patients want answered: What have other patients like me done and what was the result?

24

Prostate Lung Colon Breast

2

# o

f P

atie

nt

Live

s

4

6

8

10

12

14

16

Pancreas Myelo-fibrosis

Stomach Bladder Head &Neck

LysisSyndrome

Kidney

Data in the Platform

32 Data Sets currently in the platform or being prepared