South African Clinical Research Association | June 2015 | 1

Zaher El-AssiPresident

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

South African Clinical Research Association | June 2015 | 2

Four game-changing moments in clinical trial history

South African Clinical Research Association | June 2015 | 3

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

South African Clinical Research Association | June 2015 | 4

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

South African Clinical Research Association | June 2015 | 5

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

South African Clinical Research Association | June 2015 | 6

Key TrendsRegulatory Shifts

South African Clinical Research Association | June 2015 | 7

Richard Moscicki, M.D.

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

A bellwether?

South African Clinical Research Association | June 2015 | 8

“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

South African Clinical Research Association | June 2015 | 9

Speed to Market

Monitor

Assess

Pivot

Adaptive Trial Design

South African Clinical Research Association | June 2015 | 10

Key TrendsTechnology Shifts

South African Clinical Research Association | June 2015 | 11

Population-based

South African Clinical Research Association | June 2015 | 12

Wearable technologies (biometric apparel)

PART I

South African Clinical Research Association | June 2015 | 13

Source: Scanadu; NASA Ames Research Center

Star Trek-Style, Non-Invasive

Tricorder (scanning + mobile data

capture)

South African Clinical Research Association | June 2015 | 14

Source: Athos

Apparel – Biosignals to

Mobile Devices via Bluetooth

South African Clinical Research Association | June 2015 | 15

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:

South African Clinical Research Association | June 2015 | 16

Source: Google and Novartis

Google + Novartis –

Smart Contact Lenses

South African Clinical Research Association | June 2015 | 17

Implantables:The ultimate inside edge

PART II

South African Clinical Research Association | June 2015 | 18

Source: Texas A&M + MIT

Under-Skin (dermally implantable)

Sensors

South African Clinical Research Association | June 2015 | 19

Source: University of Illinois at Urbana-Champaign

Straight-to-Skin Printed Sensors

South African Clinical Research Association | June 2015 | 20

Source: Proteus Digital Health

Digital Pills

South African Clinical Research Association | June 2015 | 21

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

South African Clinical Research Association | June 2015 | 22

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

South African Clinical Research Association | June 2015 | 23

Enter “Big Data”

South African Clinical Research Association | June 2015 | 24

South African Clinical Research Association | June 2015 | 25

The Four V’s of Big Data

Volume Variety Velocity Veracity

South African Clinical Research Association | June 2015 | 26

Volume

South African Clinical Research Association | June 2015 | 27

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…)

South African Clinical Research Association | June 2015 | 28

South African Clinical Research Association | June 2015 | 29

South African Clinical Research Association | June 2015 | 30

Variety

South African Clinical Research Association | June 2015 | 31

Variety

• Structured data

• Unstructured data

• Semi-structured data

South African Clinical Research Association | June 2015 | 32

Velocity

South African Clinical Research Association | June 2015 | 33

Velocity

• Collection

• Organization

• Analysis

• Action

South African Clinical Research Association | June 2015 | 34

Veracity

South African Clinical Research Association | June 2015 | 35

Veracity

• Assumes simultaneous scaling up in granularity and performance

• Architectures, platforms, algorithms, methodologies and tools

• “Divide and process” across multiple servers

South African Clinical Research Association | June 2015 | 36

More data

More questions

More inputs

require Improved analysis

Improved action

Improved systems

South African Clinical Research Association | June 2015 | 37

South African Clinical Research Association | June 2015 | 38

The Opportunities• Strengthening health economics and outcomes

research

• Targeting drugs at specific patient populations

• Accelerating drug and device development

• Improving patient recruitment and retention

South African Clinical Research Association | June 2015 | 39

However…

South African Clinical Research Association | June 2015 | 40

However…

South African Clinical Research Association | June 2015 | 41

South African Clinical Research Association | June 2015 | 42

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

South African Clinical Research Association | June 2015 | 43

Q & A

South African Clinical Research Association | June 2015 | 44

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

South African Clinical Research Association | June 2015 | 45

Zaher El-Assizaher@eclinicalos.com

+1 919.653.3420