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THE RWE SERIESProviding the background,

the research and the insights.

PART I:

REAL WORLD EVIDENCE AND WHY IT MATTERS

What is Real World Evidence (RWE) and what does it bring to the pharmaceutical industry?

RWE Series Part I: Real World Evidence & Why It Matters

© 2015 SVMPharma Ltd. All rights reserved. 2

The Changing NHS

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

advanced stage of planning (1-4).

As a result of these changes in the

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 ‘data collected

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).

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).

T

DATA COLLECTED OUTSIDE OF CONVENTIONAL CLINICAL TRIALS TO EVALUATE WHAT IS HAPPENING

IN CLINICAL PRACTICE

BOTH NEW AND EXISTING DRUGS ARE HAVING THEIR VALUE CALLED INTO

QUESTION

CONTACT US

Real World Evidence

KTL Influence Mapping

Patient Experience

Healthcare Data Analytics

Hospital Episode Statistics

RWE Series Part I: Real World Evidence & Why It Matters

© 2015 SVMPharma Ltd. All rights reserved. 3

Data on a vast scale

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).

Bespoke data collection methods answering the complex questions

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).

Bespoke data collection methods

answering the complex questions

Judging true performance

Data on a vast scaleFollowing the

pseudonymous patient journey

RWE Series Part I: Real World Evidence & Why It Matters

© 2015 SVMPharma Ltd. All rights reserved. 4

A SELECTION OF RECENT UK RWE TRIALS

Effectiveness of

omalizumab in severe

allergic asthma: a

retrospective UK real-world

study (17)

Efficacy and safety of canakinumab

therapy in paediatric patients with

cryopyrin-associated periodic

syndrome: a single-centre, real-

world experience (18)

Obtaining real-world

evidence: the salford

lung study (19)

YEAR 2013 2014 2014

TYPE Audit Observational Pragmatic RCT

LOCATION Ten specialist centres

across the UK

Great Ormond Street Hospital,

London

Salford, Greater

Manchester

SIZE 136 10 7000+

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

towards increased generalisability of the findings and

encompasses a wide range of study designs including

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

AN IMPORTANT CHANGE IN THE ACCEPTANCE AND RECOGNITION

OF REAL WORLD DATA

RWE Series Part I: Real World Evidence & Why It Matters

© 2015 SVMPharma Ltd. All rights reserved. 5

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4. Wise J. NHS spending on drugs is frozen for two years under price deal2013 2013-11-07 15:12:47.

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16. Roche N, Reddel HK, Agusti A, Bateman ED, Krishnan JA, Martin RJ, et al. Integrating real-life studies in the global therapeutic research framework. The Lancet Respiratory Medicine. 2013;1(10):e29-e30.

17. Barnes N, Menzies-Gow A, Mansur AH, Spencer D, Percival F, Radwan A, et al. Effectiveness of Omalizumab in Severe Allergic Asthma: A Retrospective UK Real-World Study. Journal of Asthma. 2013;50(5):529-36.

18. Russo RAG, Melo-Gomes S, Lachmann HJ, Wynne K, Rajput K, Eleftheriou D, et al. Efficacy and safety of canakinumab therapy in paediatric patients with cryopyrin-associated periodic syndrome: a single-centre, real-world experience. Rheumatology. 2014;53(4):665-70.

19. New JP, Bakerly ND, Leather D, Woodcock A. Obtaining real-world evidence: the Salford Lung Study. Thorax. 2014.

20. Sanson-Fisher RW, Bonevski B, Green LW, D’Este C. Limitations of the Randomized Controlled Trial in Evaluating Population-Based Health Interventions. American Journal of Preventive Medicine.33(2):155-61.

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Serum-Free Subcutaneous Interferon β-1a in Patients

With Relapsing Multiple Sclerosis. Clinical Therapeutics.

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