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© 2017 PAREXEL INTERNATIONAL CORP.
THE COLLABORATIVE
ADVANTAGE OF
BIOPHARMA COMPANIES
AND GLOBAL SERVICE
PROVIDERS October 26, LA JOLLA, CA
Ubavka DeNoble
© 2017 PAREXEL INTERNATIONAL CORP. / 2
• Drug development trends
• The growing significance of
Genomics and biomarkers
• The importance of the real-time
data access and predictive
analytics
• How trial design complexity
impacts the service provider
• Future critical alignment elements
between biopharma and the
service provider
AGENDA
© 2017 PAREXEL INTERNATIONAL CORP. / 4
FDA APPROVAL FOR ORPHAN DRUG
SETS RECORD AGAIN IN 2015
• Improving NAS
approval trend;
highest since 1997
• Many first-in-class
medicines
• Orphan therapy
approvals also
increasing
Sources: FDA.gov and Jefferies Report, February 2016
© 2017 PAREXEL INTERNATIONAL CORP. / 5
ONCOLOGY AND SPECIALTY DRUGS GAIN MOMENTUM
• Significant growth in approvals
for targeted oncology products in recent years
• Targeted therapies expected to make up 91% of novel
active substances (NAS) in oncology in 2016–2020
• Biopharma companies’ pipelines are shifting to oncology
and other areas of specialty medicine
– High unmet medical need
– Better margins
– Leaner operating model
• By 2020, >225 new medicines will be introduced,
one-third for cancer
• 2/3 of drug sales will be for specialty drugs vs. 1/3 currently
Sources: Citeline, Express Scripts,2013, Drug Trend Report; IMS Health
Report, 2014; JPM Healthcare Technology Report, 2013; Wells Fargo Report,
2013, PwC Specialty drug report, Fierce Biotech, DCAT, Cowen – Therapeutic
Categories Outlook (Feb 2015), Strategy& analysis Source: “Medicine Use and Spending in the U.S.: A Review of 2015 and Outlook
to 2020,” IMS Institute for Healthcare Informatics, April 2016
© 2017 PAREXEL INTERNATIONAL CORP. / 6
TAIPEI, TAIWAN
THE GROWING
SIGNIFICANCE OF
GENOMIC & BIOMARKERS
© 2017 PAREXEL INTERNATIONAL CORP. / 7
Sources: Citeline’s Trialtrove - data accessed January - April 2015
BIOMARKERS ARE INCREASINGLY BEING UTILIZED
IN STRATIFYING CLINICAL TRIALS
• 73% of all ongoing/planned trials
using biomarkers are oncology
related; 94% of all patient
reselection /stratification trials
are cancer related
• Approvals rose for programs
utilizing biomarkers for patient
selection to 25.9%, 3x higher
than development programs not
utilizing biomarkers (8.4%), per
a BioMedTracker analysis
Top sponsors of ongoing/planned oncology trials
using biosimilars to select or stratify patients
The proportion of stratified trials is increasing Percent oncology trials selecting or stratifying using biomarkers
© 2017 PAREXEL INTERNATIONAL CORP. / 8
GENOMICS ARE BECOMING MORE CRITICAL
IN DRUG DEVELOPMENT
Target Pre-
clinical Clinical Launch
Post-
Market
Differentiated Medicines
Patient Enrichment
Patient Stratification
Efficacy
Safety Target Validation
Drug Metabolism, Transport
Drug Repositioning
Target Validation
Drug Metabolism, Transport
Drug Repositioning
Differentiated Medicines
Patient Enrichment
Patient Stratification
Efficacy
Safety
© 2017 PAREXEL INTERNATIONAL CORP. / 9
REGULATORS SEEK GENOMIC INFORMATION TO
INFORM DECISION-MAKING
Definitions
SAD: single ascending dose
MAD: multiple ascending
dose
PK: pharmacokinetic
PD: pharmacodynamic
DDI: drug-drug interaction
PGx: pharmacogenomic
CYP: cytochrome P450
TQT: Thorough QT study
© 2017 PAREXEL INTERNATIONAL CORP. / 10
IDENTIFYING DRUG REPURPOSING OPPORTUNITIES
Background:
Drug repurposing can accelerate medicine
development. While in pharma, we worked
with our clinical colleagues to identify
additional indications for an existing medicine.
What we did:
Our experts conducted a comprehensive,
clinical transcriptomics analysis across
multiple dermatologic conditions.
Value we added:
Our experts identified additional dermatology
indications for this medicine, discovered
mechanistic connections between skin
diseases and generated new hypotheses
to accelerate medicine development.
Atopic Dermatitis Psoriasis Acne
Drug Discovery Today (2014) 19 (9): 1364-71
PAREXEL Genomic Medicine
can advise clients on lifecycle
management strategies
© 2017 PAREXEL INTERNATIONAL CORP. / 11
BERLIN, GERMANY
THE IMPORTANCE
OF THE REAL-TIME
DATA ACCESS AND
PREDICTIVE
ANALYTICS
© 2017 PAREXEL INTERNATIONAL CORP. / 12
DESCRIPTIVE – REPORTING
PREDICTIVE – SIMULATION
INVESTIGATIVE – DIAGNOSTIC
What happened? What could happen? Why did it happen?
CAPITALIZING ON REAL-TIME DATA
© 2017 PAREXEL INTERNATIONAL CORP. / 13
Speed and Simplify Your Journey to Market
NOVEL
TECHNOLOGY
INDUSTRY
STANDARDS
EXPERT
SERVICES
THE SOLUTION
© 2017 PAREXEL INTERNATIONAL CORP. / 14
LEVERAGE OF SMAC TECHNOLOGIES
IMPACT CLINICAL DEVELOPMENT PLATFORM
About How We Interact Patients
Sites
Clinical Services Teams
About Big Data and Analytics Access to Digitized Information
Combined Insight
Right Decisions
Social Mobile Analytics Cloud
END TO END CLINICAL PROCESS
CONVERGENCE
Patient Reported Outcomes
Randomization
Trial Supply
Management
Clinical Trial
Management
System
Electronic
Data Capture Protocol
Deviations
Site
Start-Up Regulatory
Platform Imaging Data-Driven
Monitoring
Country
Allocation
© 2017 PAREXEL INTERNATIONAL CORP. / 15
Moving from transactional
to transformational
BENEFITS TO THE BUSINESS
DRIVE DOWN
COSTS ACCELERATE
DEVELOPMENT
TIMES
PEOPLE,
PROCESS &
TECHNOLOGY
IMPROVE
COMPLIANCE
Make better
decisions faster
Reduce monitoring
costs while
enhancing quality
Focus on your
core competency
Turning data
into knowledge
© 2017 PAREXEL INTERNATIONAL CORP. / 16
BOSTON, MASSACHUSETTS
HOW TRIAL DESIGN
COMPLEXITY IMPACTS THE
SERVICE PROVIDER
© 2017 PAREXEL INTERNATIONAL CORP. / 17
INCREASING TRIAL COMPLEXITY
• Major shift in
business mix
toward more
complex studies
(e.g., oncology)
• Patients for
targeted indications
more difficult to find
• Specialized centers
with internal IRBs*
delay startup
$194
*Institutional Review Boards
Source: Medidata PICAS database score for therapeutic areas: high>70, medium= 20-70, low<20
© 2017 PAREXEL INTERNATIONAL CORP. / 18
CLINICAL DEVELOPMENT OPTIMIZATION
EXECUTE
SUBMIT
START-UP
DESIGN
Commercialization and Outcomes Plan
Regulatory Strategy
Quantitative Clinical Development
Risk Assessment
Protocol
Optimization
Clinical Study Report Approved
Country and Site
Selection
Last Subject Last Visit (LSLV)
First Subject First Visit (FSFV)
First Protocol Approval
End-to End Data Standards and Trial Master Storage
= PAREXEL Optimization Innovations
DYNAMIC PLANNING
DYNAMIC PLANNING
Regulatory and Ethical Approval
Investigator Grants and
Contracts
Study Training
Clinical Trial Logistics
Site Regulatory
Documentation
Critical Path
Management
Recruitment and Retention
Adaptive
Monitoring
Data Surveillance
Payments
Trial Management
and Analytics
Imaging and
Spirometry
Data Management / Statistics Aggregation
Regulatory
Services
Clinical Study Report
Cross Data Analysis
Publications
Study Transparency /
Disclosure
© 2017 PAREXEL INTERNATIONAL CORP. / 19
PROTOCOL OPTIMIZATION - HOW DOES THIS WORK?
OUR GOAL
Help clients develop
multiple study designs
in order to find
the optimal one.
1
2
4
Standard
Building
Blocks in
Every Study
Considerations
for Each
Building
Block
Leverage
30 Years of
PAREXEL
Experience
& Data
Interrogate
Relevant
Building
Blocks
Scenarios
that assist
Clients
with trade-
off
decisions
3
5
A
B
C
D
E
© 2017 PAREXEL INTERNATIONAL CORP. / 20
DATA-DRIVEN INSIGHTS TO YIELD
CONSENSUS ON ACCEPTABLE RISK
THE PHILOSOPHY BEHIND BETTER INFORMED
DECISION MAKING
THE BIG DATA
ENGINE
INVESTIGATOR
& PATIENT VOICE
CLIENT
DISCUSSIONS
& ACTIONABLE
DECISIONS
PREDICTIVE
ANALYTICS
Bringing in
the Investigator
& Patient Voice
The Power to Identify
the “right countries,
sites & patients”
Effective Strategy
Planning by
Understanding Risk
Turning Data
Into Knowledge
© 2017 PAREXEL INTERNATIONAL CORP. / 21
TAIPEI, TAIWAN
FUTURE CRITICAL
ALIGNMENT
ELEMENTS BETWEEN
BIOPHARMA AND THE
SERVICE PROVIDER
© 2017 PAREXEL INTERNATIONAL CORP. / 22
BIOPHARMA
FACE ENORMOUS
PRESSURES • Demonstrating proof-of-concept
• Creating Target Product Profiles
with endpoints that support
regulatory approval
• Identifying the right partner for
protocol design, site selection and
data collection/integrity
• Managing ever-changing regulatory
and reimbursement landscapes
80%
PERCENT OF R&D ORIGINATED
OUTSIDE THE TOP 25
PHARMACEUTICAL COMPANIES
© 2017 PAREXEL INTERNATIONAL CORP. / 23
Whether you seek to license
your compound, reach proof-of-
concept, or enter the clinic
and commercialize, the PAREXEL
BioPharm Unit’s flexible model
will help you extract maximum value
from your compound or portfolio by:
• Enhancing internal capabilities
• Helping reach faster go/no-go
decisions
• Increasing compound value by
demonstrating PoC and understanding
regulatory and reimbursement
landscape
• Accelerating study start-up and
site selection
• Ensuring logistical planning and
execution is integrated into the entire
trial process
KEY ELEMENTS FOR
SUCCESS
© 2017 PAREXEL INTERNATIONAL CORP. / 24
BENEFITS OF
PARTNERING
To reach your goals faster, the
PAREXEL BioPharm unit:
• Assigns a senior PAREXEL executive
to lead a dedicated team
• Develops a strategy to minimize
complexity, accelerate timelines,
and reduce fixed costs
• Designs commercialization solutions to
accelerate development, substantiate
value and vet evidence
• Provides access to sites and patients
across Europe, Asia and the Americas
• Differentiates your company and
compound in the eyes of potential
investors and partners
• Helps you derive the most value from
your compound
© 2017 PAREXEL INTERNATIONAL CORP. / 25
ONGOING VIRTUALIZATION OF BIOPHARMA INDUSTRY
• Small-to-midsize companies,
including virtual companies, rely
heavily on outsourced services:
• Executing clinical trials
• Expanding regulatory and commercial
capabilities
• Managing cost and time to market
• Large, established companies
increasingly willing to extend the
range & breadth of outsourced
services:
• Regulatory
• Commercial
• Clinical IT
Virtualization of Biopharma
© 2017 PAREXEL INTERNATIONAL CORP. / 26
ACCELERATED REGULATORY PATHWAYS
Drug Pipelines and Specialty Products
• 64% used
at least one
accelerated
pathway
• More than a third
used multiple
accelerated
pathways
© 2017 PAREXEL INTERNATIONAL CORP. / 27
INDEXED GROWTH IN HEALTHCARE SPENDING,
GDP AND WAGES IN FOUR COUNTRIES
Note: Index on basis of local currency Sources: BCG Analysis; OECD Health Data 2009; EIU, GBE (Gesundheitsberichterstattung des Bundes),
CMS, INSEE, Office for National Statistics UK; All data indexed to 100
HEALTHCARE SPENDING OUTPACES GDP AND WAGES
Importance of Payers
• Healthcare spend growing at an unsustainable pace
• Limited objective data on value
• Biopharmaceutical companies face external pressures to reduce drug costs
• Biopharmaceutical products help bend cost curve down
100
150
200
250
300
350
19921997200220072012
100
150
200
250
300
350
19921997200220072012
100
150
200
250
300
350
19921997200220072012
100
150
200
250
300
350
19921997200220072012
Healthcare Spending
GDP
Wages
© 2017 PAREXEL INTERNATIONAL CORP. / 28
REAL-WORLD EVIDENCE PLATFORM
Patient Population
from Secondary
Data Assets
Phases
I-III
Post-Approval
Research
Patient Population from Clinical Trials
Approv
al
• Advances in computing power to integrate real-world data
• Allows study of heterogeneous patient populations beyond those studied in clinical trials
• Age groups
• Patient lifestyle variance
• Disease progression
• Compliance
© 2017 PAREXEL INTERNATIONAL CORP. / 29
PERSONALIZED MEDICINES ARE BECOMING
MORE AVAILABLE
Adapted from Pharmaceutical Research and Manufacturers' Association (PhRMA):
Value of Personalized Medicine (2015)
138 FDA approved
drugs with
biomarker
information on their
label
138 FDA approved
drugs with
biomarker
information on their
label
20% of FDA
approvals in 2014
were for targeted
therapies
20% of FDA
approvals in 2014
were for targeted
therapies
© 2017 PAREXEL INTERNATIONAL CORP. / 30
55% OF PERSONALIZED MEDICINES ARE WITHIN
ONCOLOGY/HEMATOLOGY AND PSYCHIATRY
© 2017 PAREXEL INTERNATIONAL CORP. / 31
MARKET DRIVERS INFLUENCING NEED FOR GENOMIC MEDICINE
SERVICES
Decision Maker Decision Drivers
Clients Looking for information to drive drug development
decision making
Regulators Seek pharmacogenomic information to understand who
will benefit or be harmed by the drug
Payers Want companies to demonstrate value over current
medicines
Patients Want medicines that work for them and don’t cause
side effects
© 2017 PAREXEL INTERNATIONAL CORP. / 32
INTEGRATED MULTI-OMICS APPROACHES FOR NOVEL
TARGET DISCOVERY
PAREXEL Genomic Medicine can help clients select and validate drug targets.
Background:
While working in pharma, we partnered
with pre-clinical scientists to develop
and implement a multi-omics strategy to
discover novel, specific drug targets in
the IL23 pathway.
What we did:
Our experts supported multi-omics
strategy design and applied cutting-
edge methodologies (e.g., causal
reasoning, advanced network analysis)
to analyze, integrate, and interpret the
multi-omics experimental findings.
Value we added:
In close collaboration with our bench
scientist colleagues, we discovered
novel drug targets that are validated in
follow-up experiments.
-300 -200 -100 0 100 200 300
Comp.1 [25.66%]
-200
-150
-100
-50
0
50
100
150
200
Co
mp
.2 [
12
.25
%]
Transcriptomics: IL23 vs IL12 Proteomics: IL23 vs. IL12
Integrated Multi-omics Network
Causal Reasoning & Inference
Novel Target Candidates
J Immunol (2014) 192: 2527-8
EBI Immunogenomics Conference 2015
© 2017 PAREXEL INTERNATIONAL CORP. / 33
ADME GENETICS EXPLAINS EXPOSURE DIFFERENCES
• In a Phase I drug-drug interaction study, a
subset of healthy volunteers had elevated
levels of Drug X when administered with the
client’s medicine.
• Drug X is primarily metabolized by CYP2B6.
Our experts assessed genetic variation in
the CYP2B6 gene to determine if genetic
variants leading to reduced CYP2B6
enzyme activity might account for increased
exposure of Drug X.
• The poor metabolizer CYP2B6 *6/*6
genotype was associated with elevated
exposure of Drug X. The client’s medicine
did not impact Drug X exposure; however,
Drug X increased metabolism of the client’s
medicine (data not shown) so dose
adjustments of the client’s medicine are
recommended.
Drug X plasma concentrations over time by
CYP2B6 genotype
© 2017 PAREXEL INTERNATIONAL CORP. / 34
QUANTITATIVE CLINICAL DEVELOPMENT
• Model based development is critical to take the right steps to get into IND –
pave the way for successful Phase I (the right data without surprises)
A fit-for-purpose modeling tool, always
Create definable value in early development
Cost avoidance, focus only on what is truly needed
Enhancement of pharmacology data with focus on exposure response, occupancy
Clear definition of “target” or “candidate”
Establishing high-confidence stopping rules, eliminating clouded judgment
Successful modeling helps sponsors create optimal Proof-of-Principle and
Proof-of-Concept studies, delivering more valuable data earlier in
development cycle
Exposure to Target
Binding to Target
Expression of Pharmacology
© 2017 PAREXEL INTERNATIONAL CORP. / 35
YEAREND CASH AT SMALL/MIDSIZED BIOPHARMA ($ IN BILLIONS)
SMALL AND MIDSIZED CLIENTS
$33 $38
$48
$62
$78
2011 2012 2013 2014 2015
• Despite recent slowdown in funding, smaller biopharma companies have about $78 billion of cash
• Industry as a whole has approximately $350 billion of cash
• New business from segment remains strong
• Willing and ready to deploy capital
Grow the Core Source: Chestnut Partners
© 2017 PAREXEL INTERNATIONAL CORP. / 36
DATA CAPTURE
& MANAGEMENT
Typical challenges
• Timely availability of a data capture
framework prior to first patient in
• Cost effectively capture data
• Leveraging ‘real time’ clinical data
PAREXEL® solution
Robust technology platform and
infrastructure supporting large trials
Allow rapid study-build process with
design specification generation
DataLabs closely integrated with
Data-Driven Monitoring to improve
study oversight
16,000+ SUBJECTS ENROLLED IN
1,900+SITES
Customer results:
Largest study
© 2017 PAREXEL INTERNATIONAL CORP. / 37
SOURCE DATA
VERIFICATION &
SOURCE DATA REVIEW Typical challenges
• Capturing, reviewing and analyzing
patient data in timely manner
• Managing multiple source of information
• Reporting and exporting of data
• Inspection ready SDV and SDR
PAREXEL® solution
Focus SDV within EDC to
critical elements
Efficiently document SDR in CTMS
via customizable checklists
Dashboard view of site and patients
Improved patient data surveillance
enhances data quality safety
20% SDV & SDR THRESHOLD
for some studies
Customer results:
© 2017 PAREXEL INTERNATIONAL CORP. / 38
PAYMENT MANAGEMENT
Typical challenges
• Effective control and management of
contracts and trial budgets
• Greater visibility into committed costs
vs. projected budgets
• Projecting under/over spend areas
throughout the process
PAREXEL® solution
Dynamically forecast and adjust
spend in real time
Easy generation of proposed payments
for hundreds of sites at once
Straightforward interfacing with
Accounts Payable systems
880,000+ SUBJECTS ENROLLED IN
25,000+ STUDIES
Customer results: Impact CTMS
© 2017 PAREXEL INTERNATIONAL CORP. / 39
INVESTIGATOR
PERFORMANCE
Typical challenges
• Lengthy study start-up times
• Difficulty selecting and managing
investigators
• Maintenance of investigator database
impairs planning and study timelines
PAREXEL® solution
Reduce the cost and risk of clinical
trial study start-up
Have confidence that you are using
the right investigators in your study
Expedite site selection and
patient recruitment
380,000+SITES IN
70+ COUNTRIES
Customer results: Impact CTMS
© 2017 PAREXEL INTERNATIONAL CORP. / 40
DATA DRIVEN
MONITORING Typical challenges
• Addressing cost pressure without
compromising data quality
• Regulatory pressure
• Maximizing the value of limited
resources
• Timely ability to monitor quality
and safety issues
PAREXEL® solution
Reduce costs by assessing workload
to drive site visits
Enhances study data integrity and
scientific credibility
Predictability of outcomes that
withstand regulatory scrutiny
10-25% reduction in
Customer results:
STUDY MONITORING COSTS
© 2017 PAREXEL INTERNATIONAL CORP. / 41
TRIAL TRACKING
& MANAGEMENT
Typical challenges
• Site recruitment takes too long
• Large number of global sites
and patients
• Lack of centralized information
management for Clinical Operations
PAREXEL® solution
Timely access to enrollment
and milestones
Track submissions, approvals
and the study start-up process
Focus on the sites that need
attention with on-screen filtering
and drill-through to action items
© 2017 PAREXEL INTERNATIONAL CORP. / 42
EXAMINING RULE BASED VS MODEL BASED DESIGN ACCRUAL NUMBER FOR ILLUSTRATIVE PURPOSES ONLY
1 2 3 4 5 6 7
3
3
3
3 3(15) 40
8 9
T Accrual = 55
No data on
treatment effect
3
3
3
3
3 7 (16)
5 LBM+ | 5 LBM-
+ 15 LBM-
+ 10 LBM+ + 20(?) LBM
T Accrual = 35 T Accrual = 55
Data on treatment
effect + LBM
Biomarker
No MTD
DLT?
Exploring safety
~$43K/Pat*
$2.4M
OBD
All Tox
Enrichment data
Reduced timeline
Exploring efficacy
Reduce P2b $$
~$55K/Pat*
$3.1M
Timeline in Months
* Operation fees only
Rule
Based
Model
Based
© 2017 PAREXEL INTERNATIONAL CORP. / 43
WELL POSITIONED FOR PHASE IIB
1 2 3 4 5 6 7
3
3
3
3
3 7 (16)
5 LBM+ | 5 LBM-
+ 15 LBM-
+ 10 LBM+ + 20(?) LBM
T Accrual = 35
8 9
T Accrual = 55
Data on treatment
effect + LBM
Biomarker
• Reduce timeline to acquire efficacy data
• Reducing risk of 2b design with defined patient enrichment
• Increase Investigator interest in 2b (improved enrolment)
• Reduce $ Investment for Phase 2b and time to Phase 3
OBD
All Tox
Enrichment data
Exploring Efficacy
~$55K/Pat
$3.1M
Prospective
hypothesis testing
Confirm LBM
Response rate
© 2017 PAREXEL INTERNATIONAL CORP. / 44
REGULATORS SEEK GENOMIC INFORMATION TO
INFORM DECISION-MAKING
FDA, EMA and PMDA have all recognized the importance of genomic sampling in clinical
trials: – published guidance to industry on pharmacogenomics
– ICH guideline E18 on genomic sampling and management of genomic data has been released for
public consultation
Graphic courtesy of:
Issam Zineh
Office of Translational Sciences
CDER
U.S. Food and Drug Administration
© 2017 PAREXEL INTERNATIONAL CORP. / 45
DRUG DEVELOPMENT
CHALLENGES
• Clinical studies are becoming
more complex
• Cycle times too long
• Cost pressure
• Lack of real-time and standardized
data and analytics
© 2017 PAREXEL INTERNATIONAL CORP. / 46
PHASE III FAILURES
Source: Korieth, Karyn.. “Facing protocol amendments head-on.” The CenterWatch Monthly 23.4 (April 2016): 1-13. Print
Success Rates by Phase,
2010 vs. 2011 vs. 2012 vs. 2013 (lead and secondary indications)
Approximately
40% fail!
© 2017 PAREXEL INTERNATIONAL CORP. / 47
PHASE III FAILURES
- Recent PAREXEL analysis,
we collected and evaluated
data on 38 Phase III failures
from mid-2012 through 2015
from a variety of publicly
available sources
- Failed to meet primary or
secondary efficacy endpoints
- Phase III trials that failed due
solely to safety issues are
not included
- These 38 failed trials
collectively enrolled nearly
150,000 patients
© 2017 PAREXEL INTERNATIONAL CORP. / 48
ADAPTIVE DESIGNS
How could these designs reduce amendment & failure risk?
Prospectively planned opportunity to revisit initial assumptions based on
interim data –
e.g., Drop a dose arm that is ineffective or adapt sample size
based on observed treatment effect differences
TRADITIONAL DESIGN (CARGO) ADAPTIVE DESIGN (PASSENGER)
© 2017 PAREXEL INTERNATIONAL CORP. / 49
HOW DO WE FIND THE RIGHT INVESTIGATIVE SITES?
A COMPREHENSIVE PICTURE OF THE INVESTIGATOR AND SITE LANDSCAPE
SITE PERFORMANCE DATA IN THE BROADEST SENSE
EXPERIENCE
ENROLLMENT
RESPONSIVENESS
DATA QUALITY
PATIENT POPULATION
INVESTIGATOR VOICE
DIAGNOSTICS
© 2017 PAREXEL INTERNATIONAL CORP. / 50
A SEA OF DATA
Imaging
Geo Profiling
Clinical Trial
Data
IRB
Lab Data
Audits
Validation of
instruments
IVRS
Surveys
CTMS
© 2017 PAREXEL INTERNATIONAL CORP. / 51
• Master the
datasets − Investigator
− Study
− Site
• Get the right sites,
countries and
patients for your
study
INTERNAL &
EXTERNAL
DATASETS INCREASED STUDY
PREDICTABILITY
TURNING
DATA INTO
KNOWLEDGE
• Gain real
insights
NORMALIZE
THE DATA
• Compare to the
median performance
on the same study
in the same country
IDENTIFYING SIGNALS FROM THE NOISE
© 2017 PAREXEL INTERNATIONAL CORP. / 52
Filter for Best Sites in Japan
ACCESSIBLE DATA
REGIONAL AND COUNTRY HEAT MAPS WITH SITE LEVEL DRILL-DOWN
© 2017 PAREXEL INTERNATIONAL CORP. / 53
Electronic Health Records (EHR) Insights
Early identification of potential issues with protocol inclusion
and exclusion criteria will enable a risk based approach to
feasibility and clinical trial management
Most Challenging
Eligibility Criteria
Independent
Criteria Impact
Number of Record
Queries 18,536 (100%)
Criteria 1 16,152 (87%)
Criteria 2 17,996 (97%)
Criteria 3 5,795 (32%)
Potential patient
population
for study
18,536
16,152
15,612
2,871
Identifying the impact certain criteria
will have on the patient pool allows
management of risk sharing
DO THE PATIENTS EXIST?
© 2017 PAREXEL INTERNATIONAL CORP. / 54
CLINICAL TRIAL INTELLIGENCE
MAGNITUDE
OF EXISTING
TRIAL DATA
Competitive
site landscape
Industry
benchmark
data
Study and drug
pipelines
Previous study
outcomes
Study duration
and timelines
© 2017 PAREXEL INTERNATIONAL CORP. / 55
•Analyze conversations
•Understand and advise clients
•Engage patients and/or influencers
•Understand Sentiment – assess what is said, whether good, bad or neutral
DIGITAL LISTENING is Data Collected in Real-time via Social Media
to Support Better Decision-Making
NEWS FORUMS BLOGS
Key Benefits
Develop strategies to mitigate
potential challenges
Accurate insight into
patient/medical staff
experiences
Target bloggers/tweeters to
push information
Cost effective
Global reach
What We Learn from Digital Listening: Examples
Patients speak openly about
illnesses, conditions and drugs on
social media
Understanding Patient Perceptions
Tracking Top Physicians and Patient Influencers
Social media influencers range
from top doctors to recent cancer
survivors to authors and more
DIGITAL LISTENING: PROVIDES UNCENSORED INSIGHT
55
© 2017 PAREXEL INTERNATIONAL CORP. / 56
COUNTRY DECISION MAKING SUPPORTED BY PREDICTIVE ANALYTICS
A data-driven approach to country selection takes into account key parameters
to gain a better awareness of the risks associated with country selection
DATA-DRIVEN
ALLOCATION SELECT THE
RIGHT COUNTRIES
Identify the most desirable
countries from the beginning
Validate protocol feasibility with
local clinical operations
RESULTS
STRATEGY
TRANSPARENCY
Increase quality of country
selection
Use the data to drive allocation
of countries
Enforce indication and protocol
specific assessment
Increase predictability of country
performance
Allow efficient and effective
conversations on country
allocation
Increase sponsor confidence in
country selection capabilities
RESULTS RESULTS
© 2017 PAREXEL INTERNATIONAL CORP. / 57
RISK ASSESSMENT
Feasibility will never be perfect. There will always be risk in the delivery of clinical trials.
Reducing risk to an acceptable level is the goal.
Undis
covere
d
Kn
ow
led
ge
What We
Do Know
What We
Don’t Know
What We
Suspect
Feasibility
Fe
as
ibil
ity
Undiscovered
Knowledge
What We
Do Know
What We
Don’t Know
What We
Suspect
Feasibility allows us to maximize what we know, firm up what we suspect, and minimize what we don’t know
© 2017 PAREXEL INTERNATIONAL CORP. / 58
ADAPTIVE TRIAL DESIGN CAN POTENTIALLY REDUCE
COST OF FAILURE
• About 50%
of Phase III
trials fail
• Average cost
of $20 M makes
it an expensive
problem
• Root cause
attributed to
traditionally rigid,
inflexible design
• Adaptive trial
design (ATD)
allows to
revisit initial
assumptions
and change
course
© 2017 PAREXEL INTERNATIONAL CORP. / 59
REAL-TIME DATA AND PREDICTIVE ANALYTICS
TRANSFORMING STUDY EXECUTION
Actionable
Information
Data Driven
Interventions
Silo’ed
Systems
Data Aggregation
Data Driven Interventions
Modelling Normality
Strategic Planning
Adaptive Trials
Proactive Interventions
Raw
Data
Alerts
# People/Study
Monitored
Valu
e
Maturity
Reso
urc
e r
eq
uir
ed
Modelling
Normality
Anomaly
Detection
Proactive
Decisions
© 2017 PAREXEL INTERNATIONAL CORP. / 60
DATA-DRIVEN FEASIBILITY REQUIREMENTS
Medical
Imaging
RTSM /
IRT CTMS EDC
A Single Intelligence Platform
Site Forecast
Patient Feasibility
Survey
Digital Listening
TURNING DATA INTO KNOWLEDGE
External Data
Sources
DATABASE 1
DATABASE 2
DATABASE 3
DATABASE 4
UNDERPINNING BIG
DATA ENGINE
PREDICTIVE
ANALYTICS
Country Allocation
Study
Optimization
Site
Optimization
Analysis and
Scenario Modeling +
Visualization
Power to identify the “right countries, right sites, right
patients”
Risk
Management
What We
Do Know
What
we Risk
EHR INSIGHTS
UNDERSTANDING
RISK
Decisions based on
consensus of
acceptable risk
Investigator
Surveys
Pre-Screening
Platform
CRO
Supporting Solutions
INVESTIGATOR AND
PATIENT VOICE
© 2017 PAREXEL INTERNATIONAL CORP. / 61
THE IMPACTS
COSTS?
The average cost to
develop and gain
marketing approval
for a new prescription
medicine, a process
often lasting longer than
a decade, is now
$2.56 billion dollars.*
* Tuft CSDD Outlook 2015
DELAYS?
On average, 2-3
protocol amendments
can delay a trial by
2 months, with each
amendment adding
$500,000 in
additional costs.*
* Tufts May 12, 2014, International
Journal of Environmental Research
And Public Health.
CHURN?
A big factor
contributing to delay
is the 'churn rate’
among investigators.
In some estimates,
over 50% of
investigators have
only completed
one study.
HOW TO MITIGATE THESE
CHALLENGES WITH BETTER
DATA- DRIVEN DECISIONS
© 2017 PAREXEL INTERNATIONAL CORP. / 62
INDUSTRY CHALLENGES
Site landscape
is highly
fragmented
with variable
performance
40% of
participating
Investigators
never conduct
another FDA
regulated study
About 30% of
sites don’t
enroll a single
patient
Studies don’t
meet their
enrollment
timelines 80%
of the time
Source: CenterWatch Reports
© 2017 PAREXEL INTERNATIONAL CORP. / 63
POSITIVE TREND IN FDA APPROVALS CONTINUES
FIPCOs defined as companies with annual R&D spend of $500 million or over. SEBPCO defined as companies with annual R&D of less than $500 million.
Sources: www.fda.gov. Pink Sheets 2010 -2015. PAREXEL Biopharmaceutical R&D Statistical Sourcebook 2016/2017. Michael Lanthier, Kathleen L. Miller, Clark Nardinelli
and Janet Woodcock; “An Improved Approach To Measuring Drug Innovation Finds Steady Rates Of First-In-Class Pharmaceuticals, 1987-2011,” Health Affairs, 32, no.8
(2013):1433-1439
• Growth in the number of approvals is observed for orphan drugs
18
24 26
21
30
39
27
41
45
0
10
20
30
40
50
2007 2008 2009 2010 2011 2012 2013 2014 2015
Total Orphan First-in-class Breakthroughs
NME Approvals