Real World Evidence (RWE) is defined by the FDA as 'clinical evidence regarding the usage and potential benefits or risks of medical products derived from analysis of Real World Data (RWD).' RWD is the data collected from sources other than traditional clinical trials. **Disclaimer: This article was previously published. Sciformix is now a Covance company.

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3736 BIG DATA
Bridging the gap
In a world where manufacturers must not only prove their product’s safety and
efficacy prior to launch, but also ensure commercial success, real-world evidence
(RWE) is crucial. Here, Dr Chitra Lele, chief scientific officer at Sciformix Corporation
discusses the RWE’s continuum and how it could bridge the gap between clinical
development and patient access.
Real World Evidence (RWE) is
defined by the FDA as ‘clinical
evidence regarding the usage
and potential benefits or risks
of medical products derived
from analysis of Real World Data
(RWD)’.1 RWD is the data collected
from sources other than traditional
clinical trials. These data are being
obtained from electronic tracking
systems used to capture patient
experiences during care.
RWE is increasingly crucial for
life science companies to ensure
patient access and commercial
success. Generating and
leveraging RWE requires technical
expertise along with strategic
insights for informed healthcare
decisions and optimal outcomes.
However, as the sources of RWD
are disparate and the quality can
vary greatly, accessing, analysing
and drawing conclusions from
the data is more challenging than
ever. Going forward, RWE will
become increasingly more patient
centric, as highlighted through
the growing emphasis by payers
and regulators for advances in
this area.
RWE IN CLINICAL
DEVELOPMENT AND POST
APPROVAL
RWE has a role in both pre-
and post-approval. In the clinical
development stage RWE can
enhance the effectiveness of
randomised clinical trials, for
example in the initial phases
(Phase I and Phase II) it can
aid in the identification of
patient sub-populations and in
understanding patient pathway,
disease progression, treatment
preferences and patient related
outcomes (PRO’s). RWE facilitates
operational aspects of drug
development by optimising patient
recruitment and patient access.
Post-approval RWE can assist
in generating further insights to
improve safety and effectiveness
of products, prescribing patterns
and adherence, along with real
world health outcomes.
A value-based, patient outcomes
approach is driving the role of
RWE in formulating a product’s
evidence requirements across
the development continuum.
RWE may potentially explain
facets like head-to-head
comparative effectiveness,
differentiation in sub-population
and effects of switching.
For example, data from an
observational study among insulin-
naïve patients with uncontrolled
Type 2 diabetes was used to
assess health outcomes of Toujeo
(insulin glargine) compared to
basal insulins.2
RWD pooled from
US insurance claims databases
was used to demonstrate
effectiveness of Eliquis (apixaban)
among non-valvular atrial
fibrillation (NVAF) patients.3
RWE IN PATIENT ACCESS
RWE can play a positive role
in improving patient access by
providing payers with information
on the effectiveness in the real
world and the cost-effectiveness
in comparison to other drugs on
the market. RWD can improve
health outcomes for patients and
help pharmaceutical companies
to develop a more efficient and
streamlined commercialisation
process.
The scope of RWE in market
access includes achieving optimal
price and full reimbursement,
favourable recommendation from
health technology assessment
(HTA) authority and inclusion in
various formularies at hospital,
regional and national levels. As an
example, RWE from US insurance
claims helped Zytiga (albiraterone
acetate) receive recommendation
by National Institute for Health
and Care Excellence (NICE) for
prostate cancer patients.4
Performance-based risk-sharing
arrangements (PBRSA) involve
conditional reimbursement by
payers based on health outcomes
and help improve market
access for manufacturers. RWE
provides insight into the impact
of new intervention in the ‘real’
patient population; focusing on
the efficacy to effectiveness
translation. Smart contracts
can be used to trigger drug
reimbursement from insurers only
for responders, thus enabling risk
sharing between the stakeholders,
based on RWD sharing via
blockchain technology. RWD is
also important for high-priced
therapies; wherein ‘micro-pricing’
agreements are fulfilled based on
attaining continuous outcomes and
quality of life targets. There is an
example of a pay for performance
agreement in the cardiovascular
area; wherein reimbursement
will depend on reduction in heart
failure hospitalisations.5
CLINICAL DEVELOPMENT TO
PATIENT ACCESS: THE RWE
CONTINUUM
Previously, there was a difference
in the evidence requirements for
clinical development and patient
access. The continuum of RWE
looks to bridge this gap with
value-development plans. In the
now saturated pharmaceutical
market, manufacturers must not
only prove their product’s safety
and efficacy prior to launch, but
also present evidence-based
recommendations on the way the
product’s value is determined,
demonstrated, communicated and
realised in the market.6
The plan
should be formulated during the
proof-of-concept phase, and later
include new information
over the
product lifecycle as it evolves.
RWE allows for more accurate
conclusion about medications
to be drawn and provides new
insights.
EVOLVING RWE
Going forward, drug pricing will
be based on health outcomes
rather than payment per pill. The
current constraints of using and
leveraging RWE will be mitigated
by the use of technologies like
big data, machine learning and
blockchain technology. The
evolution of the Internet of
Things (IoT) means that the future
sources of RWE are likely to be
much more diverse. Furthermore,
blockchain technology will
enable more secure storage
of patient health data and the
direct transmission of
anonymised
data to pharma companies,
helping to incentivise the right
health outcomes in the system.
Also, precision health economics
outcomes research (HEOR)
through big data can aid
healthcare decision-making
tailored to specific clusters, so
as to provide the most optimum
and cost-effective individualised
treatment.7
RWD obtained from
wearable technology can be used
by regulatory and HTA authorities
to determine drug effectiveness.
Perhaps the biggest impact of
these future trends will be felt
in the emerging markets by
‘leapfrogging’ the aforementioned
innovations.
SUMMARY
With increasing emphasis on
using RWE seamlessly across
the entire product lifecycle, from
development to patient access
and back to discovery and
development, there’s a distinct
advantage to having cross-skilled
teams. Solid experience in clinical
trials with a strong orientation to
the needs of real world studies
and the peculiarities of RWD is the
need of the hour.
Since the volume of work to
be done in RWE, HEOR and
MA is growing, largely due to
vast amounts of data being
available and the advances
in data analytics, partnering
with service providers with the
required domain understanding
and robust analytical and
scientific writing skills is becoming
more prominent. Partners who
have the right blend of clinical
development and patient access
experience will be well-poised to
provide value-added support to
their clients.
REFERENCES:
1 | FDA. Real World
Evidence. 2018. Available
online: https://www.fda.
gov/ScienceResearch/
SpecialTopics/
RealWorldEvidence/default.
html [accessed 27/02/2018].
2 | Sanofi, 2017. Switching
to Toujeo in a real-world
setting produced a similar
number of severe low blood
sugar events as insulin
Degludec. (Internet) http://
www.news.sanofi.us/2017-
11-30-Switching-to-Toujeo-
R-in-a-real-world-setting-
produced-a-similar-number-
of-severe-low-blood-sugar-
events-as-insulin-degludec
[accessed 27/02/2018]
3 | Bristol-Myers Squibb
Press Release, 2017. Bristol-
Myers Squibb and Pfizer
Present Observational Real-
World Data Analysis on the
Effectiveness and Safety of
Eliquis (apixaban)
4 | Ramaekers, B.L.T.,
et al, 2017. Abiraterone
Acetate for the Treatment
of Chemotherapy-Naïve
Metastatic Castration-
Resistant Prostate Cancer:
An Evidence Review
Group Perspective
of an NICE Single
Technology Appraisal
PharmacoEconomics (2017)
35: 191. (Internet) https://
doi.org/10.1007/s40273-
016-0445-5 [accessed
27/02/2018]
5 | Omar Ishrak. Medtronic.
2018, Jan 8. 36th Annual
J.P. Morgan Healthcare
Conference, San Francisco.
(Internet) file:///C:/
Users/ppal/Downloads/
Release0463fc6e-
d125-459e-a1e0-
4f83a78b9ad9_2324263.
pdf [accessed 28/02/2018]
6 | Business wire, 2016.
Cigna Implements
Value-Based Contract
with Novartis for
Heart Drug Entresto
(Internet) https://www.
businesswire.com/news/
home/20160208005778/
en/Cigna-Implements-
Value-Based-Contract-
Novartis-Heart-Drug
[accessed 28/02/2018]
7 | Richard Borrelli,
2008. Health Economics,
Outcomes Research and
Brand Strategy-Canadian
Pharmaceutical Marketing,
An IMS Viewpoint.
(Internet) http://www.
stacommunications.com/
journals/cpm/2008/04-
April%202008/023-IMS.pdf
[accessed 22/01/2018]
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access.ThecontinuumofRWE
lookstobridgethisgap…