svmpharma real world evidence – real world evidence (rwe): getting started with your first project
TRANSCRIPT
SVMPharma Ltd, Landmark House
Station Road,Hook,Hampshire, UK,
RG27 9HA
+44(0)1256962220www.svmpharma.com
REAL WORLD EVIDENCE: GETTING STARTED WITH YOUR FIRST PROJECT
Real World Evidence: Getting Started With Your First Project
2 © 2015 SVMPharma Ltd. All rights reserved.
1. Getting Started What should I know about RWE? Real World Evidence (RWE) is any clinical data
collected outside of a conventional randomised
clinical trial (RCT) in order to find out what is
happening in clinical practice. Compared to data
from traditional RCTs, RWE data is more
representative of a real-life patient population,
better demonstrative of the benefits of a drug in
everyday clinical practice, and is more useful in
covering real-world patient behaviour and
adherence.
RWE is becoming increasingly important because
the NHS and healthcare payors are demanding
more from the resources they have available, and
many drugs are having their value called into
question.
Decision-makers such as NICE now recognise that
assessing the value of a drug requires an
understanding of its impact within a real-life
setting.
Whilst RWE data collection via observational
studies or large datasets are well established,
guidance on planning your own bespoke
programme is scarce, and this is what we will
focus on.
Where do I begin? The ideal starting point for a RWE project is a
written project brief. This will serve a number of
purposes for yourselves, your associates and for
any external agencies pitching for the project.
The project brief is important in defining the
programme objectives, setting expectations,
making sure everyone in the organisation is
aligned, and kick-starting the process.
Whilst the content of a RWE brief includes many
sections you are already familiar with, there are
some important differences and if you have not
worked on such a programme before, you may
feel somewhat overwhelmed. By helping you
understand the processes involved this guide is a
resource which will support you through initiation
and implementation of your first RWE project.
You do not need all the answers at this stage, but
asking yourself the questions will trigger the
discussions and talking points that will move the
project forward.
2. Objectives What is my business objective? You should evaluate the existing situation for your
product, and ask yourself where you want it to be
in the future. RWE can be a powerful driver for
positive commercial outcomes and your business
objective could be one or more of the following:
Project Plan
1. Getting Started
•What should I know about RWE?
•Where do I begin?
2. Objectives
•What is my business objective?
•What is my RWE Objective?
•What is the risk?
3. Methodology
•Where is the data?
•What type of data do I need?
•How much data do I need?
•Have I considered data protection and security?
4. Organisation
•What are my timescales?
•Who will be the project lead?
5. Summary
Critical Success Factors
Project Lead
Internal Alignment
“Real world data will have a role in
future appraisals”
Alexia Tonnel (Director of Evidence Resources at NICE, June 2015)1
Real World Evidence: Getting Started With Your First Project
3 © 2015 SVMPharma Ltd. All rights reserved.
What is my RWE objective? This can include one or more of the following:
What is the risk? Phase III clinical trials are known to have high
failure rates and thus represent a beneficial but
high risk approach.
RWE projects have similar risks but via a thorough
planning and scoping phase, this risk can be
reduced. RWE can offer a relatively low-cost,
expedient, and flexible approach, and these
characteristics help to mitigate the risk. As with
all such projects, you must consider your
contingency plans carefully, but you should ask
yourself, is it better to have the data or not have
the data?
3. Methodology Where is the data? Data contained within the patient notes is
considered to be the ‘richest and purest’ form of
RWE. If you are planning to collect data on your
own drug you will need to identify where you
have above average prescribing rates and good
relations with the lead HCP. You should consider
how you can utilise existing relationships and
build new ones with key clinicians in the disease
area. The accessibility of the data varies between
primary and secondary care.
What type of data do I need? The RWE data collection programme can be
designed to collect anything that is recorded
within the patient notes. This will include
demographics, drug efficacy & safety, resource
utilisation, and patient experience data. There
are some important questions to ask including:
How much data do I need? Numerous successful RWE programmes have
been on relatively small numbers of patients (40-
80 patients) but it is possible to scale up to several
hundred. It is the choice of collection parameters
Key Questions
•Do you want to collect clinical data that is prospective, retrospective or a combination of both?
•What is the patient inclusion and exclusion criteria?
•What assumptions have been made and are these appropriate?
•What definitions are you using and have these been used consistently?
Business Objectives HTA Submission
Re-submission
NHS Demonstrate value
Service Redesign
Competitive Threat
Protecting Share
Market Growth
Market Access Local
Regional
Specialist
Publication Congress Presentation
Journals
RWE
ObjectivesEfficacy of a drug
Cost efficiency
Resource utilisation (e.g. appointments/ bed days/ hospital visits/ procedures)
Safety
Patient outcomes (both quantifiable impact e.g. days off work and patient experience and viewpoints)
50% failure rate for Phase III clinical
trials
Centre for Medicines Research (CMR) International2
Real World Evidence: Getting Started With Your First Project
4 © 2015 SVMPharma Ltd. All rights reserved.
and analysis rather than the patient numbers that
leads to meaningful results.
Have I considered data protection and
security? Data confidentiality will play an important part in
how the data collection programme is designed.
You should ensure that the data collected is
pseudonymised (non-identifiable with a computer
generated ID) and you should consider who will
own the data, will it be your company, the agency,
or the centres? Data collection should be ‘fit for
purpose’; you should only collect the data which
you require, and no patient-level data should be
shared outside of the centre providing it.
4. Organisation What are my timescales? RWE programmes are adaptable and can fit
different scenarios and circumstances. However,
at an early stage it is essential to specify any
deadlines that must be met (e.g. HTA submission)
or any upcoming changes to the market (e.g. new
regulations/ competitor launch). Most RWE
programmes can fulfil objectives and drive
positive outcomes within 6-12 months of
initiation.
Ultimately, there will be a number of factors that
will have an impact on your timelines. Therefore,
you should allow some leeway and consider the
following:
Who will be the project lead? The designated project lead will need to play a
significant role in establishing the project timeline,
accessing the resources and personnel required,
and outlining the communication plan between
each of the stakeholders. There is also the matter
of funding and it is important to put a plan in
place to ensure the project is appropriately
resourced.
5. Summary Embarking on a RWE project for the first time can
be challenging experience for your company, but
one that can drive successful commercial and
clinical outcomes. Preparing a project brief at the
beginning of the process will be valuable to both
yourselves and the agency, initiating discussion of
the relevant issues and enhancing future meetings
and proposals. By considering the topics raised
here, you will be taking a significant first step
towards a successful project.
1 FT Digital Health Summit Europe 10th June 2015 Link 2 Arrowsmith J. Trial Watch: Phase III Failures Nature Reviews Drugs Discovery 2011
Graphics from Freepik (freepik.com) and Flaticon
Key Questions
•What are the internal sign-off and approval processes?
•Do you need ethics and research approval?
•Will this project need approval from global?
External Agencies & Associates Project Lead Global
HCPs PharmacovigilanceHEOR and Statistics/ Modelling
Legal Ethics Marketing
Phase 1
•Define Scope
•Programme Objectives
•Identify Centres
•Collection Parameters
Phase 2
•Data collection tool development
•Data input training
•Data collection
Phase 3
•Data analysis
•Final internal and external reports
Proposed schedule for a RWE data collection project
CONTACT SVMPHARMA
Vaneet NayarDirector SVMPharma
Tel: +44 (0) 1256 962 220
SVMPharma LtdLandmark HouseStation RoadHook, HampshireRG27 9HA
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