This document discusses how real-world data can be used to better understand patient outcomes. It notes that legislation, technology advances, and a shift toward valuing outcomes over volume are accelerating the focus on using real-world data to determine what treatment approaches improve patient outcomes. The document outlines challenges to using real-world data like data quality issues and privacy concerns, and proposes strategies like using hybrid study designs that combine electronic medical record data with traditional clinical trial data collection to help overcome these challenges. Finally, it emphasizes that solutions in this area will need to be developed incrementally while protecting patient privacy and gaining regulatory acceptance.

1. © 2017 PAREXEL INTERNATIONAL CORP.
ALIGNING ON PATIENT
OUTCOMES – HOW
MARKET DYNAMICS CAN
FACILITATE RWD (REAL
WORLD DATA) SOLUTIONS
IN RESEARCH
Michelle Hoiseth
Corporate Vice President
Real-World Data Services
14th November 2017

2. © 2017 PAREXEL INTERNATIONAL CORP. / 2
OUTCOMES ARE THE
SINGLE COMMON
DEFINITION OF VALUE TO
PATIENTS, HEALTHCARE
PROVIDERS AND
PHARMA
• Identify actions that can be taken today to
leverage RWD while larger, structural
changes take root
• Become more agile in the complex regulatory
environment to ensure RWD strategy remains
relevant to review requirements
• Accelerate the progression of RWD use by
giving patients the ownership of their data
AGENDA AND DISCUSSION OBJECTIVES

3. © 2017 PAREXEL INTERNATIONAL CORP. / 3
MARKET DYNAMICS ACCELERATING THE FOCUS ON PATIENT OUTCOMES
Proliferation
of data with
shared value
Convergence of health care
industry and pharma around
patient outcomes
21st Century Cures Act,
PDUFA VI, EMA Guidance
2018 have time
commitments
Dependency on common data
models and manual curation of
data are reducing
10-20% improvement in
patient outcomes have
been reported by
healthcare providers
through use of big data
Top 10 drug launch
failures due to value gaps
cost industry $12B
Legislation
Technology
advances
available to life
science
Volume to
Value Shift

4. © 2017 PAREXEL INTERNATIONAL CORP. / 4
• Proliferation of data
• Changes in the environment are
permitting true access to the data
and improved utility
• Evolving regulatory positions on
RWD
• Recognition of data value by data
owners
• Technology – advances in data
stores, analytics and visualization
• Moves toward data standards
• Heterogeneous data – including free
text + lack of standards
• The 3 “I’s”: incompleteness,
inconsistency and inaccuracy
• Data ownership and data-use
agreement status
• Data privacy / patient-level authorization
• Lack of standard unique patient
identifiers to enable linking
• The need for re-identification of data
• Data are rarely present in one, single
data source – requires data aggregation
from multiple sources
DYNAMICS OF REAL WORLD DATA IN DETERMINING PATIENT
OUTCOMES

5. © 2017 PAREXEL INTERNATIONAL CORP. / 5
VALUE OF REAL WORLD DATA – DRUG DEVELOPMENT VIEW
• Health economic modeling
• Market access strategy
• Optimizing operational models and
protocols for studies
• Informing corporate and product
development strategies
TRADITIONAL USE CASES
• Hybrid data delivery models
• Randomized studies within EMR
systems
• Direct-with-patient study designs
• Mobile health device-based studies
EMERGING USE CASES
Identify the Outcomes (Endpoints) Measure the Outcomes (Endpoints)

6. © 2017 PAREXEL INTERNATIONAL CORP. / 6
REAL WORLD RESEARCH STUDY MODELS
PRAGMATIC ‘SPECTRUM’
Primary Interventional
Studies
Chart Abstraction
Secondary Database
Analyses
EMR Data Analysis
Primary NIS /
Observational Studies /
Registries
Hybrid Studies
Interventional Observational
Minimally Interventional
Studies
Today’s field of play

7. © 2017 PAREXEL INTERNATIONAL CORP. / 7
THE CURRENT STATUS OF THE EMERGING USE CASES
LOW BARRIERS
TO USE
• Multi-system or country studies
• Data of interest in narratives / notes requiring
NLP
• Patient-authorized access to data
HIGHER
BARRIERS
TO USE
• Single country studies
• Significant amount of key data in structured fields
• Intended use of results can tolerate probabilistic
matching of data
LOW
BARRIERS
TO USE

8. © 2017 PAREXEL INTERNATIONAL CORP. / 8
By carefully managing which data elements are collected via SDA vs. EDC,
hybrid studies can provide a better approach to research
HYBRID DATA DELIVERY APPROACHES OFFER THE POTENTIAL TO
OVERCOME CRITICAL EVIDENCE-GENERATION ISSUES
Research challenges
Site-based data collection (via EDC) for bulk of
study data is expensive
EMR/claims data are intended for
non-research purposes and can lack consistency
or particular data of interest
Patient recruitment timelines are challenging to
predict
Hybrid solutions
EMR/claims can be less expensive (at scale) with
access to some types of health care data that is
difficult to collect via EDC or PRO
Use site-based data collection via EDC to target
select data points where consistency/existence is
critical to the study
Improve predictability/speed by selecting sites
with high volumes
of eligible patients

9. © 2017 PAREXEL INTERNATIONAL CORP. / 9
HYBRID DATA DELIVERY MODELS CAN MAXIMIZE ROI
RWE Platform
Pharma
Medical Affairs
& Commercial
Stakeholder Insights
Payers
Clinicians &
Patients Regulators
Ph 2/3/4+ SDA PRO & COA Genomics & Sensors &
Real-time Data
Access
Integration of
Multiple Data
Streams
Creation of
Longitudinal
Patient ViewsPhV Data Biomarkers Wearables
Analytics Dashboard

10. © 2017 PAREXEL INTERNATIONAL CORP. / 10
MOBILE HEALTH DEVICE OUTCOMES
A LOT TO CONSIDER BEYOND SELECTING THE DEVICE
• Explicit cross functional discipline at PAREXEL
• Led from PAREXEL® Informatics technology business unit
• Supported by operational expertise from PAREXEL’s late stage business
Review Process &
Analytical Methods
Data Context Evolving Regulatory
Environment
Data Volume
CostData Validity Data Privacy and
Ownership
Technology
Evaluation

11. © 2017 PAREXEL INTERNATIONAL CORP. / 11
RWD STRATEGY IN AN EVOLVING REGULATORY ENVIRONMENT
Can’t be hung up on the
common data model
Push for “Qualified Data
Sources”
Regulatory Positions:
EMA appears focused on
structure
FDA appears focused on use
cases
FDA is looking toward:
1) Randomization within EMR
2) Rare disease
3) Label expansions
4) Pediatric data
Open the Dialogue
FDA Mailbox:
CDERMedicalPolicy-
RealWorldEvidence
@fda.hhs.gov

12. © 2017 PAREXEL INTERNATIONAL CORP. / 12
PATIENT PRIVACY PROTECTION AND AUTHORIZATION TO USE
PERSONAL HEALTH INFORMATION
Today’s Environment
• Applicable privacy regulations for patient consent
and patient authorization generally depends on:
− Geography
− Type of research organization
− Sources of research funding
− Types of real world data sources
• Patient authorization and IRB approval to
collect/utilize patient identifiers to link data link are
typically required
• Useful patient identity management tactics
include:
− Hashing
− Tokens
− Trusted third-parties
Rules and regulations
are to protect patients and research participants, not
to prevent legitimate research. While the requirements
may seem daunting, they are not insurmountable
barriers to research.
Near term progress to be made through
increasing opt-In consenting
*Registries for Evaluating Patient Outcomes: A User's Guide. 3rd edition.
Agency for Healthcare Research and Quality
(US); April 2014.

13. © 2017 PAREXEL INTERNATIONAL CORP. / 13
Aligning on Patient Outcomes
• Endpoint selection needs to include patient outcomes that matter to patients,
payers and define treatment value
• Leverage the environmental forces to develop industry standard approaches
• Solutions and capabilities will be developed incrementally – investments must
be made, but with flexibility in delivery in mind
IN CONCLUSION

14. © 2017 PAREXEL INTERNATIONAL CORP. / 14
THANK YOU
© 2017 PAREXEL INTERNATIONAL CORP. / 14
FOR MORE INFORMATION:
Access@PAREXEL.com
www.PAREXEL.com/evidence-wisely