Big Data and Clinical Research: Trends, Issues and Considerations

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Zaher El-AssiPresident

Big Data and Clinical Research:Trends, Issues and ConsiderationsSACRA | June 2015

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Four game-changing moments in clinical trial history

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King Nebuchadnezzar

First trial 600BCE

James Lind, M.D.

1747First controlled trial

Sir Geoffrey Marshall

1946First controlled randomized trial

Big Data

2015First trial on steroids

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Today’s clinical trial landscape(What a mess . . .)

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30-fold increase

since 2000

$8 million lost per day of delay

$1 billion to develop a drug

170,000+ registered trials

~75% delayed by one month or

more

94% Experience

delays

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Key TrendsRegulatory Shifts

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Richard Moscicki, M.D.

Deputy Director, Science OperationsCenter for Drug Evaluation & Research, FDA

A bellwether?

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“Quality over compliance”

Office of Pharmaceutical Quality (OPQ)

One voice

Integrate review and inspection across lifecycle

Patient-focused drug development

Apply risk-based approaches

The changing face of the US regulatory environment

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Speed to Market

Monitor

Assess

Pivot

Adaptive Trial Design

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Key TrendsTechnology Shifts

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Population-based

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Wearable technologies (biometric apparel)

PART I

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Source: Scanadu; NASA Ames Research Center

Star Trek-Style, Non-Invasive

Tricorder (scanning + mobile data

capture)

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Source: Athos

Apparel – Biosignals to

Mobile Devices via Bluetooth

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Source: Stanford University, MendlesPod.com, Bloomberg Business, Apple Inc.

Apple Watch + Research Kit (open source

platform)

Signed up 11,000 people in less than 24 hours (contrasted against a year’s time and 50 research sites nationwide)

Stanford Cardiovascular Study:

Signed up 7,406 patients for a Parkinson’s Disease trial in 6 hours (greatest prior study participation was 1,000 subjects)

Sage Bionetworks:

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Source: Google and Novartis

Google + Novartis –

Smart Contact Lenses

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Implantables:The ultimate inside edge

PART II

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Source: Texas A&M + MIT

Under-Skin (dermally implantable)

Sensors

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Source: University of Illinois at Urbana-Champaign

Straight-to-Skin Printed Sensors

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Source: Proteus Digital Health

Digital Pills

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All data, all the time24/7/365 (or 366)

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It means trying to control clinical trials these days is kind of like . . .

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Enter “Big Data”

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The Four V’s of Big Data

Volume Variety Velocity Veracity

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Volume

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Volume

• Paper records • Electronic medical records• Radiology images• Clinical trial data• FDA submissions• Population data genomic sequences• Personal genomic data• 3D imaging• Biometric sensors• Social media• Videography• (to name a few…)

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Variety

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Variety

• Structured data

• Unstructured data

• Semi-structured data

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Velocity

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Velocity

• Collection

• Organization

• Analysis

• Action

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Veracity

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Veracity

• Assumes simultaneous scaling up in granularity and performance

• Architectures, platforms, algorithms, methodologies and tools

• “Divide and process” across multiple servers

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More data

More questions

More inputs

require Improved analysis

Improved action

Improved systems

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The Opportunities• Strengthening health economics and outcomes

research

• Targeting drugs at specific patient populations

• Accelerating drug and device development

• Improving patient recruitment and retention

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However…

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However…

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Looking Forward

“Data and information technology... are the least regulated part of the market, so this area has the most opportunity for really innovative ways in which we can

change the environment.” – Dr. Kevin Schulman

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Q & A

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King Nebuchadnezzar James Lind, M.D. Sir Geoffrey Marshall You

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Zaher [email protected]

+1 919.653.3420