1. AUGUST 2014
PEOPLE POWER
Clinical R&D must do far more to
attract talent or face a resource crisis
AT THE CENTRE
From trial subjects to lead customers
– patient voices are key to reform
CHANGING NEEDS
How early use of adaptive designs
can impact success in later phases

2. 10 ICT l August 2014
Empowering Patients
Pharma is wising up to the fact that greater patient engagement adds
significant value across drug development. Yesterday's trial subjects
are now being seen as tomorrow's lead customers – at the heart of
industry reform over the next generation
ICT Trial Engagement
extraneous data,with the average cost of these procedures
being greater than $1 million.On average,study timelines
have to be doubled to meet enrolment levels across all
therapeutic areas,and 37% of all research sites typically
under-enrol patients (1).
In addition,years of valuable drug development time are
lost due to the lack of patient engagement.Enrolment costs
account for up to 40% of total US clinical research budgets,
so the widespread delays that are inflating budgets have
become a high value problem.Estimates put the loss of
The need for disruptive innovation in pharma has reached a
precipice.In order to compete in the era of stratified medicine,
cost-effectiveness considerations, and overall patient
empowerment,the industry must rethink every aspect of the
drug development continuum – enforcing budget controls
and adjusting clinical trials for a notably altered landscape.
A number of recent studies have pointed to the rising
complexity and costs of clinical research.Tufts Center for the
Study of Drug Development found that a fifth of procedures
in later-stage clinical trials are solely conducted to collect
Image:©IvelinRadkov–Fotolia.com
Ulrich Neumann
at eyeforpharma

3. www.samedanltd.com l ICT 11
to treat sick people, but to bring drugs to market that cure
diseases.In the past, trials have been understood purely in
terms of the scientific data value they are designed to deliver.
In terms of disruptive innovation – displacing earlier
technology to create a new market or value network – it is
fair to say that to become patient centred, every aspect of
the clinical trial journey has to be reformed.There needs to
be a paradigm shift, a rethinking of trial procedures as we
know them.
Ken Getz, Director for Sponsored Drug Research at Tufts,
illustrates the difference:the established trial follows a linear,
sequential and compartmentalised model, with vertical
ownership, centralised risk and proprietary clinical data at
the core.Patient-centred trials,in contrast,are multi-directional
and interactive,following an integrated model with horizontal
ownership and shared risk.They rely on flexible, adaptive and
proactive designs.In this context, pragmatic trials and the
use of Bayesian statistical methodologies hold potential for
a more patient-friendly study experience (4).
There should be a reframing of each of the objectives of
trials in the context of participants’needs.That means, at
least in the first instance, to see volunteers primarily as
patients seeking treatment.
According to Colin Scott, Clinical Project Leader for Novartis
Consumer Health, this should lead the industry to recognise
long-term studies in later stages of development, primarily
as an effective medical management tool (across a broad
spectrum of chronic conditions).At the same time,“pharma
needs to better understand how to reach out into largely
untapped, close-knit and underserved patient communities,
and work through independent pharmacies, charity clinics
and community working groups,”adds Scott.
Trial Experience
Practically improving clinical trial experiences might mean
taking rather straightforward steps – for example, creating
a video to explain clinical risks, or getting feedback on the
length of forms so that they are easy to understand.Informed
consent should not be obtained; it should be a collaborative
process.Communicating with patients in an effective way is
critical, as trials involve sharing complex information at a time
when participants and their loved ones are highly vulnerable.
More study visits means more time off work for either
patients or caregivers.There are other hidden costs that may
not even be obvious but can have a significant impact on
a patient’s overall experiences, such as sitting in a waiting
room without hospital wi-fi.For many trials, the accumulation
of slight inconveniences makes patients evaluate their
participation in terms of overall costs and benefits.Unless
the patient is in a very desperate disease status, those small
factors can become decisive.
sales revenues a sponsor incurs per month delay in clinical
trials at $40 million (2). Against this background, patient-
centred drug development has become a hot-button issue.
Patient Inspiration
The overwhelming majority of the 94 clinical executives who
participated in a cross-industry research project carried out
by eyeforpharma over the past six months argue that patient
centricity can no longer just be viewed as an opportunity for
commercial pharma.
Turning to patient centricity“is essential”, says Francisco
Leon,Vice President and Head, Immunology Translational
Medicine at Janssen.“Patients serve as our inspiration and,
in addition, they play an important part in ensuring we
maintain our focus.”
There is a growing recognition that, through technology
and strategic process innovations, patient views and values
must be integrated in new ways in drug development.
Senior executives believe that for reduced development
costs, and faster and more effective trials, patients must
be“front and centre”.
Lead Customers
“Patient-centric innovation is going to be at the heart
of the transformation of healthcare over the next couple of
decades,”argues Miles Ayling,Director of Innovation at NHS
England. As the patient’s role in healthcare has become
more participatory than ever before, drug sponsors are
re-examining traditional approaches.Yesterday’s trial subjects
are increasingly seen as tomorrow’s lead customers.Patients
in trials are becoming partners in research.
“What if patients were viewed as collaborators in drug
development,not sources of data?”asks Tom Krohn,Director
of Clinical Open Innovation at Eli Lilly.Krohn is an advocate
for redefining patient engagement in clinical trials from a
consumer perspective.“What if they could explore what it
was like to be in a study before they visited the site?”
Together with colleagues at Eli Lilly, Krohn has sought to
integrate trials closer into patient lifestyles.In this vein, the
company has improved the trial matchmaking processes,
altered the tone and presentation of communications with
patients, and tried to integrate the patient voice into study
design.Ultimately, patient centricity entails“more than just
the patient’s voice; it involves the patient’s thoughts, values,
preferences, strengths and shortcomings”(3).
Paradigm Shift
Patients do not join clinical trials to help pharma develop
and commercialise new medicines. Analogously, the
pharmaceutical industry does not sponsor clinical trials

4. 12 ICT l www.samedanltd.com
“You cannot just view participants as either in or out,”notes
Paul Wicks,Vice President of Innovation at PatientsLikeMe.
“It is not as though, after they sign an informed consent, they
are yours for the next two years.”Across every touch point,
patients make an active, conscious choice: every time they
take a pill, fill out a questionnaire, or come to the hospital for
a study visit.
Abandoning the Site?
If the safety of a drug is established, trials no longer need
to be conducted in the controlled and sterile setting of
a research site. Indeed, such an environment may very
well provide the opposite of a genuinely patient-friendly
atmosphere. So it is not surprising that eyeforpharma
surveys have noticed an overall movement in clinical
innovation away from a site-centric and towards a patient-
centric environment. Current approaches range from
increased in-home monitoring and tele-medicine, to trial
setting virtualisation and data-driven intervention at the
point of care (patient trial reminders in the doctor’s office).
There are multiple care settings in-between traditional
research sites and patients’homes that could potentially
be leveraged. By bringing the study closer to the patient,
the goal is to reduce the burden on participants and to
boost enrolment, adherence and retention.
One objective of‘direct to patient’is to make clinical trials
accessible to a larger community.The Novartis trial run
at drug retailer Walgreens is the most prominent recent
example of this. In late 2013, Novartis and Walgreens
announced a partnership to run trials out of pharmacies,
launching a study that should ultimately recruit up to
12,000 patients from 300 sites across the US.
The study is an example of pharma learning from
Pfizer’s 2012 virtual study (the REMOTE trial), which was
unsuccessful in the sponsor’s ambition to collect data
directly from trial patients through social media channels
like Facebook.The use of social media as a recruitment tool
for trials has yet to demonstrate any tangible success – it is
currently being used to recruit patients in only about 11%
of all trials (5). More promising for patient engagement are
dedicated sites such as PatientsLikeMe, MyHealthTeams,
CureLauncher, or the tools developed by Transparency
Life Sciences.
Patient-Oriented Data
In November 2013, Pfizer, Eli Lilly and Novartis signed
up as partners in an open innovation initiative to improve
the US online registry, clinicaltrials.gov.The goal is to
make the website more effective at matching patients
to trials.The project marks an important progression from
the era of manually identifying suitable clinical trials.
By using electronic health records (EHRs), the aim is to
devise a target health profile for each trial that is machine-
readable,so that software can match patients to specific
inclusion criteria.
US example of how to involve patients as partners to support clinical trials
Note: graphic based on material of
leading patient advocacy groups,
including Parkinson Disease
Foundation, and adapted from CTTI’s
working group on patient advocacy
Develop study
protocol
Secure
funding
Design and
test protocol
Devise study
procedures
Operationalise
study
Monitor study
Analyse and
interpret
findings
Disseminate
study results
Conduct
post-approval
research
FDA review and marketing approval
consent forms
research question in
preference
initiatives

5. 14 ICT l www.samedanltd.com
About the author
These developments open up further advancements.
Right now it is matchmaking from a patient-driven perspective.
For instance, the US Government’s Blue Button standard – a
symbol for patients to view online and download their
own personal health records – only helps to match certain
patient demographics to a certain trial, providing a map
where users can look up a study in their neighborhood.
Ultimately though, this tool could be used by a central
organiser in the reverse.
Organisers that have access to the portal could try to
find patients who would be suitable for a trial and reach
out to them directly.This is highly targeted as there is no
longer the need to post to a university hospital website,
recruitment centre, etc. Instead, an EHR environment is
leveraged. Such a process could be valuable given the
development of personalised health, as well as helping
to find patients in certain demographics.
Any change from web-based clinical trial portals, such as
those being used to receive data, to pushing data out to
physicians,payers or even patients directly,while technically
feasible, is a long way off. At present, regulations restrict
direct recruitment of patients where sponsors actively
identify them through EHRs.With data privacy concerns
growing more acute on a global scale, progress in this area
will likely be gradual. However, the tools now exist to open
up new avenues of patient recruitment.
Optimistic Outlook
A prevalent argument in the debate about patient-centred
clinical trials is that initiatives in the areas outlined here
rarely go far enough. As important as they are to initial
focus areas, they may only result in incremental steps
toward a more fundamental shift in the conception of
drug development.
Furthermore, patient centricity and patient engagement
have become buzzwords in pharma business speak, while
many primarily talk about an updated approach to patient
compliance. However, true patient centricity asks about
how pharma can better comply with patients’needs,
rather than asking them to comply with pharma’s needs.
For too long,patients have felt like the raw material in the
clinical research process – nameless,experimental subjects
generating reliable data.
Nonetheless, the clinical research industry has recently
demonstrated a strong desire to engage with patients on
an equal footing and take innovative steps to incorporate
patient empowerment across the value chain of trials.
The most successful examples of patient centricity place
patients at the top of the trial management hierarchy –
treating them as lead customers, including them in study
design, and giving their decisions actual weight.
Innovation Wave
Some leading sponsors have demonstrated a keen vision
to transform trials and place patients at the core of their
research operations – a shift in mindset that may help fuel
the next wave of patient-focused trial innovation.
In the context of lengthy and complex drug development
schedules, regulatory requirements and ethical constraints,
delivering wholesale transformation of the clinical research
enterprise will be a challenging work in progress. But with
the blockbuster model in question, and individual and
personalised medicine on the horizon, the need for patient-
focused innovation is as inevitable as opportunities for such
change are staggering.
References
1. Tufts Center for the Study of Drug Development, 89% of trials
meet enrolment, but timelines slip, half of sites under-enrol,
Impact Report 15(1), January/February 2013
2. Mintz C, Social media’s impact on clinical trial enrolment,
Life Science Leader, 2010
3. Robbins DA, Curro FA and Fox CH, Defining patient-centricity
opportunities, challenges and implications for clinical care and
research, Therapeutic Innovation & Regulatory Science 47(3):
pp349-355, 2013
4. Mullins CD, Vandigo J, Zheng Z and Wicks P, Patient-centredness
in the design of clinical trials, Value in Health, 2014
5. Tufts Center for the Study of Drug Development, Drug developers
circumspect about social media in clinical research, Impact
Report 16(2), March/April 2014
Ulrich Neumann MSc, MA, BSc, BA, is the
Global Project Director at eyeforpharma.
He leads the Patient-Centred Clinical Trials
Initiative, a collaborative research project
culminating in an executive conference
in Boston on 4-5 September 2014. Ulrich
earned an MSc from the London School of
Economics, and an MA in Global Comms from the University
of Southern California. He also led a research consulting
group supporting cancer trial accrual in Los Angeles.
Email: ulrich@eyeforpharma.com
Any change from web-based clinical trial portals,such
as those being used to receive data,to pushing data out
to physicians,payers or even patients directly,while technically
feasible,is a long way off