Health Datapalooza IV: June 3rd-4th, 2013 Illuminating Disease at the Speed of Light: How Big Data Is Accelerating Biomedical Research Convener: Marcia A. Kean, Chairman, Strategic Initiatives, Feinstein Kean Healthcare Moderator: Kevin Davies, Author, “The $1,000 Genome”; Founding Editor, Nature Genetics and Bio-IT World magazine; and Vice President Business Development, American Chemical Society Speakers: Amy P. Abernethy, Director, Center for Learning Healthcare (CLHC), Duke Clinical Research Institute Michael Cantor, Senior Director, Information Strategy and Analytics, World Research Development, Pfizer Dave King, Founder, Exaptive Robert McBurney, Chief Executive Officer, Accelerated Cure Project for Multiple Sclerosis Dietrich Stephan, Founder and Chief Executive Officer, Silicon Valley Biosystems (SV Bio) Until recently, biomedical research was conducted in small silos, separated by huge cultural and technical walls and inability to exchange data facilely. But the field is now exploding: massive amounts of complex, multi-dimensional clinical, imaging, and genomic data are being collected, aggregated, integrated, analyzed, and shared. The pioneers driving this digital transformation are working in novel collaborations among patients, providers, and scientists. In this session, speakers will showcase multimodal, computational, and analytic tools for biomarker discovery, patient stratification, and intelligent clinical decision solutions; high-speed genome profiling, with algorithms of individual and population profiles, for real-time molecular-based diagnoses of ‘mystery’ diseases; a point-of-care quality monitoring program used by clinicians, in which data are used for important research questions and then transitioned into clinical trials; and collection and aggregation of data and samples from patients with a neurological disease, enabling queries into markers of disease origins and discovery of new therapies.

1. Confidence in Data • Driving the Portfolio • Fueling Innovation
eControls: Unlocking the Value of Placebo
and Control-arm Clinical Trial Data
Michael Cantor, MD
June 4, 2013

2. Background: Secondary Use of Placebo, Comparator,
and Medically Stable Patient Data
• Pooled placebo data has been explored and incorporated
into ongoing internal and collaborative R&D efforts.
Selected secondary control data projects include:
• Critical Path Institute (C-Path) Coalition Against Major
Disease (CAMD)
• Ongoing placebo data sharing effort in Alzheimer's
disease to support biomarker development
• EU Innovative Medicines Initiative (IMI) EuroPain
Initiative
• Pooling placebo data to understand the placebo
effect in Pain studies
• Pfizer Vaccine Development [Pilot]
• Control database to provide context to observations
• Pfizer Pain Research Unit: Enhanced Clinical Trial
Design by leveraging patients from prior clinical trials
with common end points
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3. ePlacebo: Objectives
• Estimate background incidence of AE’s and SAE’s in different age
populations
• To estimate the frequency of adverse events in different age populations
occurring within 6 months of having received a study intervention (placebo or
active)
• Rapid turnaround
• Flexibility to do subset analysis as needed
J Am Med Inform Assoc doi:10.1136/amiajnl-2012-001257
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4. ePlacebo to eControls
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Placebo
Data
Filters
ePlacebo
(20k)
Filters +
Additions
eControls
(50k)
• Parallel Design
• Completed within the last 11
years
• No Concomitant drugs (i.e.
aspirin)
• Treatment duration > 6
weeks
• Vaccine-specific filters (e.g.
absence of autoimmune
disease)
• Added medically stable
patients from control arms

5. eControls Consortium
• Convened by Critical Path Institute
• Goal: Establish a pooled, multi-therapeutic area, standardized,
web-enabled database of clinical trial control arm data
• 10 interested pharmaceutical companies
• Members must contribute data to participate
• Logistics in process
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6. Goals for eControls
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• Comparison of placebo/control effects across trials and
therapeutic areas
• Generation of disease models, clinical outcome assessment
instruments, new biomarkers
• Development of historical controls, especially for rare/orphan
diseases, with the potential for reduction in number of patients
needed for placebo/control arms
• Broad database with multiple contributors will provide an
increased likelihood of observing subjects with common
endpoints
• Large and diverse set of research questions

7. eControls Future: Beyond Clinical Trial Data?
• Non-patient volunteers who would contribute data
• More representative of healthy population
• 100,000+ patients from diverse age groups and backgrounds
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8. Acknowledgements
• Ed Bowen
• Prakash Bhuyan
• Martin Carlsson
• Jigar Desai
• Owen Fields
• Mike Maher
• Matt St. Louis
• Critical Path Institute
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9. Demo
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10. Interested in Contributing?
michael.cantor@pfizer.com
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