MARCH 2017
REAL WORLD STUDIES LANDSCAPE
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REAL WORLD STUDIES
TABLE OF CONTENTS
MARCH 2017
TABLE OF FIGURES
Executive Summary
0i
Introduction
01
The number of real world studies is accelerating
01
1: Real world studies in the 1990s, 2000s and 2010s 2: Number and cumulative number of real world studies, 1991 to 2017
01
The US and Europe are hubs for real world studies
02
Real world studies are being run for a range of conditions and technologies
03
4: Real world studies by ICD chapter
03
Major investment
04
5: Interventions subject to real world studies
04
The impact of real world studies
05
References
06
3: Location of real world studies by regions across the globe
01
02
6: Number of years real world studies run/expected to run
04
7: Number of enrollees in real world studies
04
8: Funders of real world studies
04
9: Number of sponsoring organisations for real world studies
05
This report contains information from numerous sources that Leela Barham Economic Consulting Ltd believes to be reliable but for which accuracy cannot be guaranteed. Leela Barham Economic Consulting Ltd does not accept any responsibility for any loss incurred by any person who acts or who fails to act as a result of information published in this document.
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Executive Summary
MARCH 2017
Real world studies are a major investment for those involved. That’s driven by both the time they take –
“Let’s get real” seems to be a rallying cry as almost
almost 60% are expected to last at least three years –
everyone wants to know how a technology works in
but also the collection of data relating to hundreds
the messy real world, and outside of the controlled
to thousands of patients too. Most real world studies
trial. There is substance and not just a buzz around
collect data on 1 to 500 patients, but they can be as
Real World Data (RWD) and Real World Evidence
vast as collecting information on over 100,000.
(RWE) as data from ClinicalTrials.gov – a web resource that aims to provide accessible information
Industry is paying for just over 40 per cent of real
on clinical studies from around the world – reveals.
world studies, but ‘others’ are paying for almost the same proportion (37.4%). It’s Pfizer within the
Even if the data in ClinicalTrials.gov is incomplete, it
pharmaceutical industry who is most busy. They
illustrates the significant acceleration in the number
must surely be gathering a wealth of experience
of real world studies that has been taking place over
with 41 real world studies completed, or in train. The
the last three decades. There were 700 real world
industry is also working with others with just over 40
studies started in the years 2011 to February 2017.
per cent of real world studies involving two co-
That compares to just seven in 1990s.
sponsors or more.
The US and Europe are hubs for real world studies.
It’s clear that the number of real world studies is
Perhaps reassuring - and something that the
accelerating, being run around the world and for
industry in the UK can use as they lobby for the best
many conditions and different technologies. But just
deal as part of Brexit - is the UK’s strong position in
what is the impact? That won’t come from analysis
Europe (with 89 real world studies), second only to
of ClinicalTrials.gov data, but by looking more
Germany. Will the UK’s position change though, in
widely.
light of Brexit? There are examples where real world studies seem Any condition can be subject to the real
to have played a role to help companies secure
world approach although popular is to explore real
those increasingly important positive
world issues for cardiovascular disease (29% of the
recommendations from Health Technology
968 real world studies included in the data). So too
Assessment (HTA) agencies.
can a range of interventions, although drugs are most often studied (22.9% of the 968 studies in the
Building an evidence base on the impact of real
data) where the intervention is known.
world studies on decisions from regulators, HTA, payers and of course clinicians and patients too is likely to be a fruitful area for further research. Optimising the impact of real world studies will be the next challenge.
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Real World Data (RWD) and Real World Evidence (RWE) are “buzz words”. RWD is data collected from outside of trials; that means it can be anything from data abstracted from patient records to tweets that discuss side effects. RWE are the insights that such RWD can generate. Almost everyone, it seems, wants to know how a
MARCH 2017
The number of real world studies is accelerating The number of real world studies listed in ClinicalTrials.gov has seen a dramatic increase since the 2000s (Figure 1) and looks to be accelerating (Figure 2). Figure 1: Real world studies in the 1990s, 2000s and 2010s
technology works when it’s used in the messy real world, and outside of the controlled trial. But what’s behind the buzz? ClinicalTrials.gov is a web-based resource to provide easy to access information on publicly and privately supported clinical studies. The database covers all 50 states in the US and 196 countries.[1] The data is not complete as not all studies are required to be registered, and may not be up to date, although it provides an objective lens through which to explore the real world study landscape. The following analysis draws on data on 968 studies identified using the search term “real world” in
Source: Analysis of ClinicalTrials.gov data. Note 2017 is up to 22 Feb 2017.
ClinicalTrials.gov data downloaded on the 22 February 2017. Figure 2: Number and cumulative number of real world studies, 1991 to 2017
Source: Analysis of ClinicalTrials.gov data. Note 2017 is up to 22 Feb 2017.
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The US and Europe are hubs for real world studies Real world studies have been run or are underway across the globe. The US and Europe appear to be hubs (Figure 3). Perhaps reassuringly, given wider uncertainties for the life sciences sector, the UK is a popular choice with 89 real world studies, second only to Germany in the European region. The Brexit question might apply though; will the UK still be a choice for real world studies once it’s outside of the EU?
Figure 3: Location of real world studies by regions across the globe
Source: ClinicalTrials.gov data.
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Real world studies are being run for a range
MARCH 2017
Figure 5: Interventions subject to real world studies
of conditions and technologies Generating real world insights is possible for a range of conditions. Most popular is generating real world insights in diseases of the circulatory system (29% of the 968 real world studies), followed by mental and behavioural disorders and neoplasms (Figure 4). Real world studies are being run across a number of different types of interventions too; perhaps unsurprisingly it is drugs that are the most popular
Source: Analysis of ClinicalTrials.gov data. - = not classified
type of intervention (where it’s specified) (figure 5).
Figure 4: Real world studies by ICD chapter
Source: Analysis of ClinicalTrials.gov data. Note: ICD chapters had to be manually mapped to the information available from ClinicalTrials.gov data. Although best efforts have been made there is a risk of misclassification. - = not classified
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Major investment
The pharmaceutical industry is not alone in
Real world studies represent a significant
their interest in real world studies
investment through both time, and presumably, money too. That time is not just in terms of how long
Figure 8: Funders of real world studies
the studies run for – with almost 60 per cent lasting/expected to last for at least three years but with some expected to run for more than 20 years (Figure 6) – but also the time of those involved in the studies, where collection of the data for hundreds to thousands of patients must be a significant task, even in today’s increasingly digitized health care systems (Figure 7). Figure 6: Number of years real world studies run/expected to run Source: Analysis of ClinicalTrials.gov data.
Industry – not just the pharmaceutical industry but those making devices – is an important funder for real world studies, accounting for 41 per cent of real world studies. However ‘other’ funders are important too, accounting for another 28 per cent. Joint funding is common too (Figure 8). Within the pharmaceutical industry it is some of the big names that are busy with real world studies (Table 1); perhaps it’s no surprise that the world’s Source: Analysis of ClinicalTrials.gov data.
number one by sales, Pfizer, is the busiest on real world studies? It’s also likely that their perspective
Figure 7: Number of enrollees in real world studies
on real world studies will be informed and hence, worth listening to.
Source: Analysis of ClinicalTrials.gov data.
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MARCH 2017
Table 1: The ten companies doing the most real world studies
not the database is updated, plus there is a lag between completion of the study and time for analysis and publication. The crucial question is not just whether the results are available but also their impact. Success with a real world study may mean that a company secures the price and/or reimbursement they are seeking, or perhaps even improving or maintaining their Source: Analysis of ClinicalTrials.gov data.
position following challenge by either competitors or others, whether regulators, Health Technology
Whilst the majority of real world studies are single
Assessment (HTA) agencies or other payers who are
sponsor (572 or 59%), collaboration is popular, albeit
increasingly interested in re-visiting their initial
with most studies being run with just a handful of
decisions and want to be informed by real world
organisations (Figure 9).
experience.
Figure 9: Number of sponsoring organisations for real world
The impact of real world studies won’t come from
studies
analysis of ClinicalTrials.gov data but needs a wider look. Some examples are emerging that suggest that real world studies can help tip the balance from a ‘no’ to a ‘yes’ from HTA agencies; Sanofi used real world studies to achieve a positive recommendation from the Scottish Medicines Consortium (SMC) for Zaltrap (aflibercept) in metastatic colorectal cancer [3] and Johnson & Johnson did too to help achieve a positive recommendation from the National Institute for Health and Care Excellence (NICE) for
Source: Analysis of ClinicalTrials.gov data.
Zytiga (abiraterone) for prostate cancer.[2] Price cuts played a role too. Building an evidence base on the
The impact of real world studies Just 85 (or 9%) of the real world studies listed in ClinicalTrials.gov have results available. That reflects that some studies are still recruiting (277 or 29%), studies that have not yet hit their finish dates (438 or
impact on real world studies on decisions from regulators, HTA, payers and of course clinicians and patients too is likely to be a fruitful area for further research. Optimising the impact of the many real world studies available or in train will be the next challenge.
45% are due to finish in 2017 or beyond) and
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References [1] https://clinicaltrials.gov/ct2/about-site/background [2] Bruce, F. (Nov 2016). Real world evidence and the question for European market access. In Vivo. [3] Liden, D., Jaksa, A. and Ho, Y. (2015). Does real world evidence matter in Health Technology Assessments? Pharmaphorum. Retrieved from: http://pharmaphorum.com/articles/does-real-world-evidence-matter-in-healthtechnology-assessments/
About the author Leela Barham is an independent health economist and policy expert. You can contact her on
[email protected] or +44 (0) 777 927 9511 No funding was received for this work, it was motivated by just a little bit of spare time and curiosity.
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