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50 THE PHARMA REVIEW ■ NOVEMBER - DECEMBER 2015
Data & Technology
in Clinical Trials 2015
An Industry View Point: The Future of Leveraging Data and Technology in Clinical Trials.
eyeforpharma’s State of the Industry Survey investigated how the
pharmaceutical industry are leveraging, or plan to leverage, data
and technology in European clinical trials.
"An Industry View Point: The Future of Leveraging Data &
Technology in Clinical Trials" looks at the Survey findings in
detail, with commentary from industry experts Joseph Kim (Eli
Lilly) and Jeffrey Kasher (DrugDev) on how best to leverage the
latest technologies and data innovations and prioritize patient
need.
The Survey
The survey comprised eight questions covering methods currently
employed in clinical trials, methods to be implemented, the impact
of data collection and analysis methods, current challenges, and
patient-centricity.
Respondents were industry professionals from the areas of
research (22.47%), general management (21.35%), operations
(20.22%), development (17.98%), data (10.11%), design (5.62%),
and outsourcing (2.25%).
Clinical Trial Methods – Risk-Based Strategies
Growing In Popularity
Currently, small patient studies are being deployed or piloted by
65.17% of respondents, while risk-based strategies (49.44%)
Figure 1: Clinical function of respondents
and adaptive trials (48.31%) aren’t far behind. Dropping down
the scale in popularity are applied real-world data in trial design
(33.71%) and data modeling and simulation (28.09%).
Of the clinical trial methods planned for deployment by companies
within the next two years, risk-based strategies moved up to first
place (58.43%), closely followed by adaptive trials (52.81%).
Small patient studies dropped from first to third place at 43.82%.
The picture changed when respondents were asked which
methodswouldhavethemostimpactinfiveyears’time.Remaining
at the top of the list of priorities were adaptive trials and risk-based
strategies. However, small patient studies moved to the bottom of
the list, even below applied real-world data and data modelling
and simulation. In other words, the most frequently deployed
methodology currently – small patient studies - is believed to
offer the least effectiveness in the next 5 years. Still, pharma
are set to address this by focusing on riskbased and adaptive
strategies over the next 2 years, with one respondent, noting,
“The biggest area of investment would be to fully implement risk-
based monitoring principles and remote monitoring.”
This move towards risk-based monitoring (RBM) is supported by
other research within the field1
:
Joseph Kim, Jeffrey Kasher & Nassim Azzi*
*Global Project Director, eyeforpharma, Email: nassim@eyeforpharma.com (Author for Correspondence)

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51THE PHARMA REVIEW ■ NOVEMBER - DECEMBER 2015
Figure 2: Trial methods planned within the next 2 years
Figure 3: RBM utilization, MCC survey 2013
Figure 4: Key reasons for adopting RBM, MCC survey 2013

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52 THE PHARMA REVIEW ■ NOVEMBER - DECEMBER 2015
The necessity to move towards RBM is also supported by Joseph
Kim, Senior Advisor in Clinical Innovation, Eli Lilly: “Currently, we
typically send a human being out to a site and they look at the
data, compare it the source and try to find errors and omissions in
data. But it’s expensive to clean data in this way. Not to mention,
even the smartest human isn’t going to be able to catch patient
or sitewide trends that could pop up. With central monitoring and
more powerful statistical models and algorithms - not to analyze
the data but to analyze the data quality – you can start to ascertain
with greater certainty that: a) there's no foul play; b) people are
well trained and the data is coming in as it should; and, c) patients
are also doing things correctly.” These key reasons for adopting
RBM are supported by other research within the field (Figure 4).
By using RBM, you can identify the necessary data to answer
the specific questions in your trials, while bypassing unnecessary
data. Jeffrey Kasher, Chairman of the DrugDev Advisory Board,
says, "I was fortunate to be sponsor of the TransCelerate working
team that developed and published the RBM guidance. I believe
implementation is going well, especially when you consider how
large of a change it represents.”
He adds, “Initially, there was confusion between sponsors/CROs
and sites as to exactly what RBM was and was not. There has
been a lot of dialogue which has brought people to a common
understanding. The large remaining opportunity is for sponsors
to identify and address risk factors in protocols while in the stage
of protocol design. Modifying trial design or ensuring safeguards
are put in place for the highest risk data elements will yield better,
higher quality, trials."
Future Investments – Data Leads The Way
Findings from the survey indicate that future investments in the
next 2 years will be largely directed towards data, with order
of potential investment then being: research, development,
operations, design, and general management. A total of 85.39%
of respondents agreed that improving the collection, analysis
and management of data would have the biggest impact on how
future clinical trials will be conducted.
Qualitative responses demonstrated some clear strategies for
how pharma could better invest in data in the future, including
utilizing real-world and big data, as well as remaining focused on
patient-centricity.
Real-World Data
Survey respondents felt that, “Data needs to be as close to real-
world as possible,” and that the collection of real-time data is one
of the biggest opportunities for better use of clinical data in the
near future. Real-world data provides faster data generation,
demonstrates value to patients quickly, and increases patient
recruitment. It was noted that, “Collecting real-time data relevant
topeoplehastobeanimprovementonscalesbasedonpopulation
norms.” Indeed, it was expressed that communication in real-time
could facilitate finding urgent answers to unmet needs.
Big Data
A significant number of respondents believed that big data is
where the opportunities are for future clinical trials. Big data
facilitates the challenge of finding innovative therapies, by
identifying sub-groups of patients to target the right therapy to
the right patient. It also allows for the integration of patients and
patient associations into clinical research to help define the right
biomarkers. “As R&D becomes more expensive and there are
less new innovative molecules in development, the new way to do
R&D will be looking into big data and getting the most out of it to
develop new drugs and/or target specific needs and populations,”
offered one respondent.
It appears from responses that one way of achieving better
utilization of big data is through “data mining and meta-analyses”
and “data sharing between companies for the common good.”
It was pointed out that, “Everyone has these information-rich
datasets that could be used for other trials.” Data sharing isn’t
just about sharing positive results, however, the need to include
failed studies within any data sharing initiatives was emphasized.
Patient-Centricity
Running more patient-focused trials was currently the biggest
focus for 66.29% of respondents. Patient-centricity was referred
Figure 5: Consensus that the collection, analysis and management of data will have the biggest impact on how future trials are conducted

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to in a variety of ways, including the need for “personalized
data,” “specialized medicine,” and the recommendation to “use
data from reimbursement and individualized treatment.” The
need to embrace patient reported outcome measures PROMs
was also highlighted in efforts to achieve “data collection directly
from the patient” and “improve patient-reported outcome quality
and collection.” It was believed that one way forward was the
“integration of PROMs data into clinical trials.”
"Participants aren’t an amalgamation of what you read in
scientific papers. They aren’t a faceless person. They are
your wife or brother. If your loved ones are about to enroll
in a clinical trial, what would you want for them to make it
patient-friendly?"
Joseph Kim, Senior Advisor in Clinical Innovation, Eli Lilly
It was highlighted within the survey that, “The priority is patient-
centric trials,” and that, “Evidence collected in clinical trials has
to go down to the individual patient. Personalized medicine may
further evolve as evidence/data grows. This may mean better
efficiency in treatments, cost-effectiveness, and possibly a
reduction in adverse events.”
Patient-centricity remains somewhat of a buzzword, so how
can pharma move it from being a buzzword to an actual way of
working? According to Kim, it isn’t brain science – “Put yourself
in the role of the patient. What would you want as a patient?” He
adds, “Participants aren’t an amalgamation of what you read in
scientific papers. They aren’t a faceless person. They are your
wife or brother. If your loved ones are about to enroll in a clinical
trial, what would you want for them to make it patient-friendly?”
Patient-centricity in trials holds many benefits, not least of which
includes enhanced recruitment and retention. It can often unearth
areas of unmet patient need. The National Cancer Research
Institute Clinical Study Group has patient representatives to help
inform discussions on trial design at their meetings. Another
example of the effectiveness of patient involvement discovering
unmet needs was brought forth by Julie Flygare of Project
Sleep. By getting together with other narcolepsy patients, a new
symptom was identified as a new, potential clinical endpoint to
pursue – ‘brain fog’ – which wasn’t on anyone’s radar. Flygare
did this without a Pharma partnership, “But imagine the value of
being able to purposely harness patients insights so they can help
come up with the new endpoints and unmet needs,” says Kim.
Current Challenges – it’s not All About Budget
Budgetary pressures are by far the biggest challenge, according
to survey findings, followed by identifying and maintaining
patients, managing timelines, integrating new technologies into
the trials process, finding and implementing new systems and
software, developing trials that answer initial questions, reducing
silos between different functions/departments, establishing and
maintaining relationships with CROs, and CRO selection.
Kasher observes, “The top 3 challenges in the survey build on
and compound each other. Project teams are under pressure to
get a new trial started (timeline), which results in limited time for
real-world data feasibility analysis, patient input, and site input.”
According to him, this means that trials get started and due to
design flaws, it is hard to recruit and/or retain patients in the
trial. This leads to additional sites being added and prolonged
timelines. He advises,
"Slow down prior to starting the trial; it will result in
completion of the trial on time, on budget, and with quality."
Jeffrey Kasher, Chairman of the DrugDev Advisory Board
Kim also offers some advice: “Budgeting is a multi-variable
discipline. Ultimately, the speed of clinical trials is the most
important metric to improve. Patient enrollment is the leading
cause of delays in drug development. People choose not to
participate. They're not inspired to participate. The health care
system isn’t incentivized to make that happen. So, if we can
accelerate the call to action from patients that is probably the
best way to reduce budgets and the costs of developing drugs.”
We provide some strategies for overcoming the challenges of
patient recruitment and retention, and managing timelines, all of
which can have a direct impact on cost-effectiveness. At the core
of these strategies is the leveraging of technology.
Kim points to the Internet as one key way to enhance enrollment
in clinical trials. However, he also cautions that the real question
isn’t about how to leverage technology, but, “How do we leverage
the Internet as it applies to the real brick and mortar world, so that
more patients can actually participate? And what does that mean
for study design?”

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54 THE PHARMA REVIEW ■ NOVEMBER - DECEMBER 2015
Figure 6: Challenges to respondents’ current role, ranked according to biggest impact
Mobile Technology
Respondents indicate that “mobile technology” might offer such
convenience, as well as, “Integration of new technologies and
implementing new systems to manage data.” Kim agrees that, “If
we want to increase the ability of more patients to participate, we
need to think about more creative ways to let them participate.
Just like online banking has changed the way banking is, can we
virtualize some part of the clinical trial or activate retail health care
providers in conjunction with the investigator, so that patients can
participate in a way that's really convenient for them?”
Wearable technology is another way of enhancing data capture,
data quality and facilitate data sharing to cut costs and drive
efficiency. According to Kim, wearables promise the ability to
provide better objective data than, for example, questionnaires.
Giving an example of how depression is frequently measured
via questionnaires and structured interviews, Kim asks, “Could
an activity monitor be a better indicator of whether a patient’s
depression is improving? One could argue yes, since wearables
can measure real-world data such as physical activity, which
might not just be a good indicator of improvement for depression,
but for pain.” However, he does highlight that just assuming
wearables are better than questionnaires isn’t enough. “You’ve
still got to do the validation studies to prove it,” he says.
Apps
According to Kim, Apple’s ResearchKit is a milestone in clinical
research. He says, “One of the most valuable companies in the
world has taken a first step in trying to create a platform where
anybody with a smart phone can start to participate in research.
One of the first bright spots was the Stanford MyHeart health
study, which enrolled 10,000 patients in one day. To do that in
a day is unheard of.” He adds, “It’s only a matter of time before
smart phones are the dominant channel to the Internet, which
also means access to health information and participation in
health care.” Kim and others believe this could be as soon as two
to three years’ time.
Social Media
Social media is another key player in leveraging technology
to improve patient retention. Kim says, “Social media can be
powerful in terms of accelerating health related information and
empowering patients.” Despite this, the top companies on social
media for 2014 don’t include any pharma companies. Exceptions
to this are Novartis; they recently leveraged the power of Twitter
(@NovartisCancrUS) to keep their 10,000+ followers engaged
and motivated during cancer trials in the US. In this campaign,
Tweets comprise promotions of cancer trials, with most tweets
achieving engagement with patients, healthcare providers, or
organizations. Appeals to retweet information on trials are also
made, meaning that their reach is much wider than their followers
alone. Also, hashtags are used to target tweets to certain cancer
types (i.e. #breastcancer). Patient engagement is also enhanced
by tweets about trials being linked to relevant information
sources, where patients and professionals can investigate further
and make informed decisions. One of the information sources
some tweets link to is the Novartis Clinical Trials website, which
offers more global support and helps visitors find clinical trials
anywhere in the world.

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Case Study
Curing Through Connection
Another example of the power of social media is that of e-patient
Dave de Bronkart, who was diagnosed with Stage IV, Grade 4
renal cell carcinoma in 2007. His median survival time at diagnosis
was predicted to be 24 weeks, and his doctor prescribed him to
visit an online social media forum to learn more about the disease
from other patients. He discovered a therapy not widely used but
that had good efficacy with some people. After removal of the
tumor and a Biologic Therapy program, he was able to take part
in a clinical trial for High Dosage Interleukin-2; he was cancer-free
6-months after diagnosis. “That only happened with social media,”
says Kim. “Social media isn't the power of liking and sharing. The
idea is that you're connecting information and aggregating it.
We're all getting wiser due to this wisdom of crowds that can only
be attained when you connect a bunch of people together in a
fast and furious way.”
The State of The Industry
eyeforpharma’s State of the Industry Survey, combined with
case studies and expert insights from Kim and Kasher, highlight
About The Authors
Nassim Azzi is a Global Project Director at eyeforpharma and heads up the Clinical Trials Initiative USA. As
a part of the initiative, he has been bringing together and working closely with clinical thought leaders and
game changers from the Pharmaceutical Industry and other key stakeholders to help implement meaningful
transformations to the clinical trial enterprise. He holds an MBA from the Johns Hopkins Carey Business
School and has lived and worked on three continents and is fluent in three languages.
Joseph Kim serves as a Senior Advisor in Clinical Development Innovation at Eli
Lilly, focusing on developing and implementing innovative patient engagement solutions. He has spent
over 15 years in the pharmaceutical industry utilizing a unique approach that integrates his experiences
working for sponsors such as Shire and Merck, CROs, and technology vendors. He has a robust
combination of experience that includes early and late phase clinical research, and a well-known history
of innovation in the clinical research industry, recognized as one of “Top 100 individuals on the 2015
MedicineMakers Power List, and “20 Innovators Changing the Face of the Clinical Trials Industry” by CenterWatch in 2013.
Follow him on Twitter: @JoPeKim
Jeffrey Kasher, Ph.D., became Chairman of the DrugDev Advisory Board this year, joining a distinguished
team of current and former clinical research executives from many of the leading pharmaceutical companies
in the world. Dr. Kasher is passionate about improving outcomes, bringing patient and research sites into the
developmentprocess,anddramaticallydecreasingthetimetomarketforpromisingdrugcandidates.Heisknown
as a pharmaceutical development change catalyst who brings to the role 28 years of leadership experience at
Eli Lilly, where most recently he managed a portfolio of innovative clinical development approaches to achieve
50% cost and time reductions to be realized over a five-year period beginning in 2017. In addition to his role at DrugDev, Dr.
Kasher is President of Patients Can’t Wait, was a founding member of the TransCelerate Biopharma Operations Committee and
the Avoca Quality Consortium, sits on the DPharm Europe steering committee and faculty, and is a member of Linking Leaders.
In 2013, CenterWatch named Dr. Kasher one of the “20 Innovators Changing the Face of the Clinical Trials Industry.” Follow
DrugDev on Twitter: @DrugDev
the need for pharma to better leverage data and technology
to improve the quality of clinical trials. This can be achieved
through more careful consideration of trial design at the outset,
more effective data collection methods during trials, and greater
transparency with outcomes. One of the key ways to achieve this
is by embedding patient centricity throughout all stages of the
clinical trial process. As Kasher points out, “Patients are the ones
who invest themselves in clinical trials (their bodies, their lives)
and therefore they should have input into the design of the trial,
receive support during the trial, and receive their results (written
so they can be understood) upon completion of the trial.”
The challenge, then, is how to enhance patient-centricity? The
Internet, mobile technology, and social media may be the answer.
Overall, pharma needs to harness the power of data, maximize
technology, and design around the patient.
Sources:
1. Taking the Risk:An Examination of Current OrganizationalApproaches
and e-Data Sources Driving Risk-Based Monitoring Programs. Risk
Based Monitoring Full Report, 2014. Metrics Champion Consortium.
2014.