PART I:
RWE SERIES
Providing the background, the
research and the insights.
REAL WORLD EVIDENCE
AND WHY IT MATTERS
Jan 2015
What is Real World Evidence (RWE) and
what does it bring to the pharmaceutical
industry?
In Part I of the RWE series we introduce
the ideas and concepts behind RWE,
providing an overview of its role and how
it fits into the industry. We explain why
pharmaceutical companies are increasingly
adopting RWE programmes.
This is the first part of a regular series
available at svmpharma.com
BY GEM AUDDY
[email protected]
SVMPHARMA LIMITED
WINCHFIELD LODGE
OLD POTBRIDGE RD
WINCHFIELD
HAMPSHIRE
RG27 8BT
(+44) 01252 849071 EXT. 2068
© 2015 SVMPharma Ltd. All rights reserved.
1
The Changing NHS
T
he NHS is going through a period of transition that looks set to continue for
several years to come. In addition to changes in infrastructure and organisation,
there has been a strategic shift with the NHS demanding more value from the
resources it has available and the companies it works with. Controlling expenditure on
drugs and managing the size of the UK Medicines Bill is a key area of action for the
NHS, and this is apparent in a number of policies which have either been recently
implemented by the NHS or are in an
BOTH NEW AND EXISTING DRUGS ARE
advanced stage of planning (1-4).
HAVING THEIR VALUE CALLED INTO
As a result of these changes in the
QUESTION
NHS, both new and existing drugs are
having their value called into question, placing certain pharmaceutical companies in a
challenging position to remain competitive, and opening up new opportunities for others.
Pre-launch randomised controlled trials (RCTs) are sufficient for establishing a drug’s
efficacy, demonstrating safety and gaining a licence. However, it is becoming
increasingly clear that drugs need to supplement this with Real World Evidence (RWE)
in order to gain traction in the market and to then either grow or defend its share (5-7).
Defining Real World Evidence
RWE can be broadly defined as ‘the collection
of data outside of conventional clinical trials in
order to evaluate what is happening in clinical
practice’. RWE and related methods seek to
both enhance and complement existing
methods of data collection (8).
THE COLLECTION OF DATA
OUTSIDE OF CONVENTIONAL
CLINICAL TRIALS TO EVALUATE
WHAT IS HAPPENING IN CLINICAL
PRACTICE
This data is derived from sources including patient registries, routine administrative data,
observational research datasets, electronic health records and population health
surveys. It is with the technological evolution and broadening scope of these data
sources in recent years that RWE is ready to fulfil its potential (9).




Real World Evidence through Service Evaluation
KOL Identification and Mapping
Healthcare Data Analytics
Bespoke Market Research

Hospital Episode Statistics
CONTACT US
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2
Following the
pseudonymous
patient journey
Data on a vast scale
Judging true
performance
Bespoke data
collection methods
answering the
complex questions
Data on a vast scale
Bespoke data collection methods
answering the complex questions
RWE enables analyses of study populations
at a scale far beyond what would be feasible
in a clinical trial. Using powerful data tools,
the filtering and analyses of millions of
records can be undertaken efficiently on
datasets such as Hospital Episode Statistics
(HES) and GP Practice Prescribing Data.
These large datasets are useful for identifying
unmet treatment needs, targeting patient
segments and highlighting prescribing
patterns; these are insights which help both
the NHS & the pharmaceutical industry
(10-13).
Following the pseudonymous
patient journey
Different records from a dataset such as HES
or Clinical Practice Research Datalink
(CPRD) can be linked via a unique identifier
whilst maintaining the patient’s confidentiality.
This allows the patient journey to be followed
across hospitals visits, between primary and
secondary care, and along treatment
pathways. This can uncover valuable
information such as motivators for seeking
care, triggers for switching medicines and
long term compliance issues. This can be
used alongside qualitative datasets such as
Patient Reported Outcome Measures
(PROMS) (14).
Intelligent and customised RWE data
collection programs can be designed
alongside patient groups and healthcare
professionals to focus on selected issues and
fill gaps in knowledge. An online tool allows
flexibility, validation and guidance in data
input with real-time analysis. Complex patient
histories can be streamlined and reduced to
the pertinent information required, and real
world scenarios can be analysed.
Judging true performance
It is difficult to predict how a drug will perform
in the real world clinical setting. A variety of
overriding factors come into play such as poor
adherence, potential drug interactions due to
polypharmacy, the impact of co-morbidities
and how far the disease has progressed, all
of which can diminish the impact of a drug or
distort its effects. The collection and analysis
of RWE enables stakeholders to make
informed decisions throughout the product
lifecycle (9, 15, 16).
© 2015 SVMPharma Ltd. All rights reserved.
3
A S ELECTI ON OF RECENT
YEAR
TYPE
LOCATION
SIZE
UK RW E TRI ALS
Effectiveness of
omalizumab in severe
allergic asthma: a
retrospective UK real-world
study (17)
2013
Audit
Efficacy and safety of canakinumab
therapy in paediatric patients with
cryopyrin-associated periodic
syndrome: a single-centre, realworld experience (18)
2014
Observational
Obtaining real-world
evidence: the salford
lung study (19)
Ten specialist centres
across the UK
136
Great Ormond Street Hospital,
London
10
Salford, Greater
Manchester
7000+
2014
Pragmatic RCT
RWE Success & Recognition
In recent years there has been a notable increase in the publication of RWE in leading journals
worldwide; this shows an important change in the acceptance and recognition of real world data. In
traditional RCTs, patient selection, randomisation to groups and other controlled conditions
maximise internal validity. These RCTs are essential in evaluating the clinical effectiveness and
safety of drugs but there are doubts on how it can be applied to the real world clinical setting (10,
16, 20-23). A move towards RWE shifts the focus
AN IMPORTANT CHANGE IN THE
towards increased generalisability of the findings and
ACCEPTANCE AND RECOGNITION
encompasses a wide range of study designs including
OF REAL WORLD DATA
pragmatic RCTs, modelling analyses and observational
studies (17-19, 24, 25).
RWE consists of a broad and interrelated series of concepts; this introductory
article has focused on insights from big data, the ability to follow a patient
journey, customised data collection methods and the ability to judge true
performance outside of a controlled trial.
Pharmaceutical companies need to adapt to meet the changing requirements
and demands of the NHS, and to effectively show the value of their products.
In this competitive environment, the collection, analysis and presentation of
RWE can drive success and growth. RWE is growing in importance and is set
to become an essential part of the marketing and medical strategy for a
successful drug.
NEXT ON THE RWE SERIES JOIN US FOR PART II IN WHICH WE DETAIL THE DIFFERENT SOURCES
OF RWE AND LOOK AT HOW THIS DATA CAN BE COLLECTED AND ANALYSED. VISIT
SVMPHARMA.COM & FOLLOW US @SVMPHARMA
© 2015 SVMPharma Ltd. All rights reserved.
4
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© 2015 SVMPharma Ltd. All rights reserved.
5