CADTH 2015 Symposium

1. Gwendolyn Ryals, Look at Me
Artwork from The Creative Center
Julia Brown, Vice President
Government Affairs and Market Access
CADTH Symposium
April 2015
RWE is the New Black:
A Perspective from Industry

2. 1
What is Real World Evidence (RWE)?
Generated using data from usual care setting in a range of
non-interventional (observational) studies, including:
 Primary data collections
 Analyses of secondary data
RWE can be generated using so called “pragmatic”
randomized trials that are conducted in more usual care
settings that employ broad patient inclusion criteria and that
investigate effectiveness (rather than efficacy) of different
treatments.
A working definition of RWE…

3. 2
Growing demand and increasing data
availability moving RWE close to tipping point
RWE is growing in importance and relevance at accelerated pace
Growing demand – Focus on
value and transparency
Increasing supply – Easier
availability of data/analytics
 Societal trends (e.g., reform,
aging population, emerging middle
class) increasing utilization
 Markets becoming increasingly
demanding as health care
systems seek meaningful
innovation, value and
transparency
 Substantial economic / pricing
pressure; increased legal,
regulatory, and business scrutiny
 More data from new data
sources, e.g., sensors, self-
reported data
 Better insights from structured
and unstructured data
 Governments (e.g., US, UK,
Denmark) playing an active role
making health data liquid
 Engaged patients – more
involved in care, sharing and
generating data

4. 3
RWE is becoming increasingly important
IMS Health

5. 4
Stakeholders responding globally
Regulators
▪ Mainly focused on
pharmacovigilance
▪ Adaptive licensing concepts
and corresponding use of
RWE data
Payors
▪ Selected payers use RWE for
formulary decisions (US) or
cost/quality analyses (GER)
▪ Emerging outcomes
based/risk-sharing contracts
Providers/clinicians
▪ Usage of internal RWE
datasets for cost/quality
monitoring and physician
incentives
▪ Creating registries to
generate RWE
HTA bodies
▪ Broad variation of use, some
systematic (e.g, UK, Italy)
others more research focused
(e.g., US)
Patients
▪ Interest in real-world
evidence mixed with
questions on data privacy
▪ Starting to “create” real-world
data via forums
Academia
▪ Broad use of RWE as source
of insight to medical research
▪ Partnerships with
other stakeholders

6. 5
But there are significant geographic variations
RWE used
for national
market
access and
general
reimburse-
ment
RWE mainly
used for
utilization/
safety or
formularies
(e.g.,
payer/reg-
ional/hos-
pital)
Sporadic use Frequent use
▪ Limited RWE use for benefit
assessments and
reimbursement negotiations
– RWE has low weight vs.
clinical data
▪ Some payors using cost-
benefit assessment (e.g.,
WellPoint)
▪ Major investments in RWE
analytics
Use of RWE by stakeholders in health system
MainapplicationofRWEdata
France
Germany
ItalyUK
US
Spain
 RWE data used for conditio-
ned reimbursement and ma-
naged entry agreements
(especially for high cost
innovative drugs)
▪ RWE mainly used to identify
potential post-marketing
safety concerns
Japan

7. 6
Going forward, RWE use will increase – albeit at
different speeds
RWE used
for national
market
access and
general
reimburse-
ment
RWE mainly
used for
utilization/
safety or
formularies
(e.g.,
payer/reg-
ional/hos-
pital)
Sporadic use Frequent use
▪ Slower - RWE likely to play a
somewhat larger role during
drug re-evaluations post
launch
▪ Faster - Higher number of
payers and providers conducting
HTA-type assessments (driven
by data availability)
Use of RWE by stakeholders in health system
MainapplicationofRWEdata
▪ Faster - RWE expected to play
significantly larger role during
drug evaluations (3-5 yrs post
launch)
Faster - RWE likely to be used
more frequently at national
level for access and during re-
evaluations at regional level
▪ Faster - RWE use likely to
increase due to increasing data
availability
▪ Slower – role of HTA and RWE
for market access and reimbur-
sement likely to increase slowly
Outlook: At what speed will RWE
usage increase?
France
Germany
ItalyUK
USSpain
Japan
▪ Slower - opportunity to increase
use of RWE via Public Know-
ledge Assessment process

8. 7
What do we need to do in Canada to catch up?
From… …To
Mindset ▪ Systematic and proactive;
earlier and patient-centric
▪ Ad hoc and reactive; post-
launch and product-centric
Capabilities ▪ Broad awareness,
collaboration and
information sharing
▪ Low awareness, variable
capabilities and no
framework for incorporation
into decision-making
Process ▪ Iterative and strategic use of
RCT and RWE
▪ Fragmented and serial
lacking cohesive approach
Data/infra-
structure
▪ Greater access, coordination
▪ Limited access and
coordination
Partnerships
▪ Coordinated and integrated
partnerships
▪ One-off collaborations
Funding ▪ Limited funding ▪ Pre-planned and coordinated

9. 8
In Summary
 Engagement and collaboration from all stakeholders:
– Patients, HTA bodies, payers, healthcare professionals, academia,
industry…
 Framework for RWE generation and incorporation into HTA
reviews, public policy and formulary decision-making
 Generation of data that balances commercial and public interests
 Appropriate allocation of resources and willingness to risk-share
 Prioritization of patient outcomes over short-term cost-
containment