PCDD – A Roadmap

www.eyeforpharma.com/clinical
Patient-Centered
Drug Development
A ROADMAP FOR
PHARMACEUTICAL LEADERS

Oral Therapies in the Oncology Marketplace
Growth Potential, Challenges and Trade-offs
Patient-Centered Drug Development
A ROADMAP FOR PHARMACEUTICAL LEADERS
Contents
Foreword from Andreas Koester 3
Foreword from Paulo Moreira 4
Introduction 5
Definition and measurement 6
What patients really want 8
Trial design 12
Systematic engagement of 14
patient advocates
Patient-centered systems and 15
trial technology
Better site management, 16
clearer patient focus
The future of trial communication: 17
Online, social, mobile?
Mobile trials and direct-to-patient 19
(DTP) solutions
The FDA and Patient centricity: 21
What are regulators doing?
Expectations and outlook 24
Conclusion 25
DISCLAIMER
The information and opinions in this paper were prepared by eyeforpharma (a division of
FC Business Intelligence) and its partners. FC Business Intelligence has no obligation to
tell you when opinions or information in this report change. FC Business Intelligence
makes every effort to use reliable, comprehensive information, but we make no
representation that it is accurate or complete. In no event shall FC Business Intelligence
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opportunity or profit caused by the use of the material or contents of this paper.
No part of this document may be distributed, resold, copied, or adapted without our
prior written permission. FC Business Intelligence Ltd © 2015.
Contributors to this paper
Marc Boutin, EVP and COO, National Health Council
Bonnie Brescia, Founding Principal, BBK Worldwide
Elise Felicione, Director, Clinical Trial Innovation, Janssen
Zach Hallinan, Director, Patient Communication and Engagement, CISCRP
Andreas Koester, VP, Clinical Trial Innovation, Janssen
Greg Koski, President and Co-Founder, Alliance for Clinical Research
Excellence and Safety (ACRES)
Rhonda Kost, Clinical Research Officer, The Rockefeller University Center for
Clinical and Translational Science
Paulo Moreira, VP, Head of Global Clinical Operations – External Innovation,
EMD Serono
Susan Sheridan, Director, Patient Engagement at the Patient-Centered
Outcomes Research Institute (PCORI)
Jack Whelan, Cancer Survivor, Warrior and Patient Leader
Javier Zambrano, Director, Biogen Idec.
Joel Beetsch, VP, Patient Advocacy, Celgene
Jennifer Byrne, Chief Executive Officer, PMG
Chris Frega, Sr. Director and Head of Global Feasibility and Patient
Recruitment, Quintiles
Jeremy Gilbert, VP, Product and Strategy, PatientsLikeMe
Sharon Hanlon, Director, Clinical Trial Partnerships, Bristol-Myers Squibb
Michael Jones, Sr. Director, Clinical Operations, Eli Lilly & Co
Richard Klein, Head, FDA Patient Liaison Program,
Office of Health and Constituent Affairs
Paul Kluetz, Acting Deputy Office Director of the Office of Hematology and
Oncology Products, FDA
Tom Krohn, Chief Development Officer, TrialReach, former Lead,
Eli Lilly’s Clinical Open Innovation Team
Theresa Mullin, Director of the Office of Strategic Programs in the Center for
Drug Evaluation and Research, FDA
James O’Leary, Chief Innovation Officer, Genetic Alliance
Jeanne Regnante, Head, CMO Strategy Office, Chief of Staff to the CMO, Merck
Tomasz Sablinski, Founder and CEO, Transparency Life Sciences
Roslyn Schneider, Global Patient Affairs Lead, Pfizer Sciences
Tom Sellers, Sr. Director, Patient Advocacy and Corporate Philanthropy, Takeda
Veronica Todaro, VP, National Programs, Parkinson’s Disease Foundation (PDF)
David Verbraska, VP, Worldwide Public Affairs and Policy, Pfizer
Glen de Vries, President, Medidata
Bernard Vrijens, Chief Science Officer, MWV Healthcare
David Vulcano, AVP & Responsible Executive for Clinical Research,
Hospital Corporation of America
Jeff Williams, SVP Operations, Apple
Lode Dewulf, Chief Patient Officer, UCB

3
www.eyeforpharma.com/clinical CONTENTS / PRINT
Foreword from our Conference Chairmen
Patient centricity
Not a new concept, but an increasingly important one
Dear Colleagues,
From talking to you at conferences like eyeforpharma’s Patient-Centered Clinical Trials, I get reminded once in a while why
many of us joined the life sciences field: following a deep running passion to improve and save patients’ lives. But step into
any pharma or biotech company’s office and you can experience what happens to good intentions once they have to compete
with the demands of tight timelines, budget pressures, and regulatory requirements. It’s easy to forget who we aspire to be
working for.
But there is an obstacle to patient mindedness more insidious than just time pressure: it’s the notion that we – due to
education and experience – know what patients want and need.
But when we look around us, we can see every day that this paternalistic model is falling apart. Thanks to technology and
social media, patients often know as much (or more!) about their disease than their doctor.
A decade ago, it was moribund HIV patients who made their voices heard and created the impetus for pharma and regulators
alike to heighten their attention towards patients’ needs. Maybe we can learn from the HIV/AIDS example and from the
successful approach Genzyme and others have taken in rare diseases, when we try to find and define a new balance between
scientific rigor and patient burden, between risk and benefit, between the need for confidentiality and the mandate to inform.
So, maybe the challenge ahead is not so much about reinventing new approaches, but rather harnessing the technology that is
now at our disposal to incorporate what served us and our patients well in life-threatening diseases into every step of the drug
development process for any and all diseases. I’m convinced if we do this consciously, forcefully, and jointly, we may look back
in just a few years on 2014 and ask ourselves why it wasn’t always second nature for us to check with the very patients
afflicted by the disease we are trying to cure to understand what’s important to them rather than just assuming that we know.
So let’s brainstorm together and share ideas and best practices so that we can truly
deliver “the right thing to do” for our patients throughout the drug development
process. And while we are at it, for those who still need a little convincing
that all the investment in time and resources is necessary and
worthwhile, let’s create the foundation for a business case on how
engaging patients in clinical research can be a cure for many of
the problems (expensive amendments, poor recruitment,
high attrition) that ail the clinical research enterprise today.
Andreas Koester
Vice President, Clinical Trial Innovation & External Alliances
Janssen/Johnson & Johnson
eyeforpharma PCCT Conference Chairman 2014

4
Patient centricity
A comprehensive, holistic endeavor
Dear Colleagues,
We are living in times of unprecedented innovation in the pharmaceutical industry. In the recent past, there has been an
awakening to the important role that patients can play in helping biotechnology and pharmaceutical companies develop
new medicines.
Given the advent of and exponential growth of the internet, the amounts of medical information available is astronomical.
Patient are more educated and better informed than they have ever been. This knowledge has given way to patients that
are engaged and more vested than ever in their treatment options. The role of patient advocacy groups is also expanding in
this area.
We are looking forward to acquire invaluable insights on how some collaborative models between pharmaceutical companies
and patient advocacy groups are having a very positive impact in the industry.
Patient centricity in clinical trials has been interpreted and implemented in many ways. However, patient centricity cannot be
viewed simply as isolated activities that aim directly at the patient. It is a much more comprehensive endeavor. Successful
implementation must rely on a holistic approach that touches all of those with a vested interest in the clinical research and
development enterprise.
We propose to shine a light on these complex interactions and demystify how, sponsors, patients, clinicians, patient advocacy
groups, regulators, innovation and technology can come together to deliver new medicines faster to patients without
compromising the scientific merit of the clinical trial.
Paulo Moreira
Vice President, Head of Global Clinical Operations – External Innovation
EMD Serono
eyeforpharma PCCT Conference Chairman 2015
Foreword from our Conference Chairmen

5
Introduction
Patient centricity has become a key priority for leaders in
clinical trials – but few in the industry know how to go about
achieving it. Key questions remain unsatisfyingly unanswered.
For example, how should we even define patient centricity?
How is it measured? Where has it been successful so far?
What are the challenges in coming years? Drawing on
in-depth interviews with clinical leaders who participated in
eyeforpharma’s Patient-Centered Clinical Trials program, this
paper provides answers to these and other crucial questions,
looks at solutions for change and examines those
companies which are putting in place structures to make
patient centricity an organizational reality.
Putting patients at the center of the trial has the potential to
make the development process more effective – which
makes it attractive – but it also requires a paradigm shift –
which makes it difficult. Changing the role of the patient
from subject to participant needs a new culture, mind-set,
framework and language. While some observers use the
term “baby steps” to describe where the industry presently
stands on patient-centered drug development, the case
studies and examples in this paper indicate that more
sophisticated, long-term strategies are now being designed.
“Product development with the help of
end users is common in industries such
as the automobile or food industry – it is
only relatively new to pharma.”
Paulo Moreira
Vice President – GCO Head of External
Innovation, EMD Serono
Ignoring the patient means that critical insights are missed by
all stakeholders. “Historically, patients have not played a
significant role in determining the research questions or the
outcomes that really matter to patients,” says Susan Sheridan,
Director of Patient Engagement at the Patient-Centered
Outcomes Research Institute (PCORI). “I think it has been
assumed that this is only researchers’territory and that it is too
complex.”A recent poll by PatientsLikeMe among 70 clinical
operations leaders at the Avoca Quality Consortium, points to
the dominant thinking: Patients are viewed as having too
little ‘expertise and capabilities’ to be able to really contribute.
A rethink is now a matter of urgency for everyone involved.
“Today, trials are complex, where millions of dollars rest on
patients’ reactions to a trial protocol and how quickly they
can link to the trial and potentially enroll,” says Jeremy
Gilbert, VP, Product and Strategy, PatientsLikeMe. “Despite
that, clinical teams spend months trying to ‘think like a
patient’ in making protocol trade-offs but they rarely
actually ask the patient or study the patient’s own
perspective.” These approaches typically focus on medical
outcomes, which don’t capture the journey the patient takes
en route – involving aspirations for their personal life,
treatment and recovery.
R&D costs have “spiralled out of control”,says Michael
Jones, Senior Director, Clinical Operations, Eli Lilly & Co.
“Focus on the patients can help us to focus on the questions
that matter most. We can streamline the research by
focusing on the patient,” he suggests.
There are some interesting current examples of a practical
move towards patient-centered drug development, such as
Janssen’s MyCentralCare (read more in our section on
patient-centered systems below). But Elise Felicione,
Director, Clinical Trial Innovation at Janssen R&D, warns that
there is no quick fix: “Don’t think that it will be three
months from talking about this to patients actually using
your portal.”
“Patient centricity is not doing the same
non-patient-centric things but with the
addition of a graphic from management,
containing a bunch of call-out boxes,
emanating from a diagram of a patient in
the middle.”
David Vulcano
AVP & Responsible Executive for Clinical
Research, Hospital Corporation of America

6
Definition and measurement
All stakeholders – including patients, HCPs, pharma,
regulators, HTAs, payers, and politicians – accept that
more patient involvement in drug development is needed
although there is no widely accepted standard for what
constitutes patient centricity. While our interviews
revealed various definitions of the term, there are clear
areas of consensus.
It may initially be helpful to define what patient centricity
is not. According to David Vulcano, AVP & Responsible
Executive for Clinical Research, Hospital Corporation of
America, the main reason why patients are often ignored in
drug development is “because we’re trying to do
everything the same for all the other stakeholders and
then add patient centricity in to the mix”. He goes on:
“I see a lot of these slides from multitudes of sponsoring
companies and CROs but not a lot of action here with the
exception of trying to be more creative in recruitment and
retention and calling that ‘patient-centric’.” Vulcano’s point
is clear: Anyone who thinks that simply talking about
patient centricity is enough needs to think again.
So what is it? “In its purest form, patient centricity is the
creation of a direct link between the goals of clinical trials
and the needs of patients on an individual and global
scale,” says James C. O’Leary, Chief Innovation Officer at
Genetic Alliance. “It is not simply designing trials to meet
the needs of participants, but rather creating systems and
tools that allow participants to inform and influence the
trials themselves.” Jeremy Gilbert, VP, Product and
Strategy at PatientsLikeMe, has a similar definition:
“Measuring what matters to the patient in the trial itself,
and designing the trial as much as possible to
accommodate the impact on the patient’s life.” Roslyn F.
Schneider, Global Patient Affairs Lead at Pfizer, thinks it is
helpful to concentrate on three main areas:
• Meaningful involvement, including more direct patient
input at key points such as trial protocols
• Patients receiving data where they need it, in a manner
they can easily interpret to actually improve their health
• Utilizing technology to bring patients closer to what the
industry is doing.
Rhonda Kost, Clinical Research Officer, The Rockefeller
University Center for Clinical and Translational Science adds
that patients’ priorities such as convenience, expense,
pain, risk, and benefit must be taken into account, while
Bonnie Brescia, Founding Principal at BBK Worldwide, sums
it up as “making sure you’ve included the voice and values
of the participant”.
CORE GOALS OF PATIENT CENTRICITY
IN DRUG DEVELOPMENT
• Meaningful inclusion of patients, in particular for
trial protocol design
• Linking needs of patients with goals of clinical trials
• Considering patients’ experience of their disease
throughout the program
• Taking patient priorities such as convenience, pain,
expense and benefit into account
• Measuring what matters to the patient
• Giving patients appropriate, timely and user-
friendly information
• Using technology to include patients more
• Including voice and values of patients
It is one thing to agree on the tenets of patient centricity in
clinical trials, but it is another thing entirely to measure the
concept.“ A barrier to metrics at the moment is that most
people don’t know what we’re trying to measure,” explains
Schneider. “To show it is meaningful, and to test different
models, we need to be able to measure it. Patient
satisfaction alone is important but is not the only answer.”
Soft measures of patient centricity such as the level of trust
that a patient has in a trial or a company are easy to
pinpoint, says Tomasz Sablinski, Founder and CEO of
Transparency Life Sciences. “Once the trial has started we
can measure how many are adherent to the trial protocol,”
he adds. “Just the willingness to participate is an important
measure, and how many are willing to inform their fellow
patients about a trial.”

77
Definition and measurement
EMD Serono is establishing patient advisory boards to
include the ‘patient voice’ while most sponsors use some
form of patient survey to capture perceptions of informed
consent procedures, trust, feeling of partnership, unexpected
pain, convenience, feeling of respect, sense of being listened
to, and overall experience. Michael Jones, Senior Director of
Clinical Operations at Eli Lilly Co, has used such feedback
mechanisms to identify opportunities to improve. Eli Lilly
sought answers to the common questions: “Are we able to
shorten recruitment cycles? Can we better retain patients?”
But since clinical leaders have only recently started to fully
embrace a patient-centered approach, more structured
metrics simply do not yet exist – something particularly true
of return on investment (ROI) measurements. “It is difficult
to quantify because each protocol is unique,” says Paulo
Moreira, VP and Global Clinical Operations Head of External
Innovation at EMD Serono. “You would need to focus on
historical accrual rates, number of patients per site per month,
etc. Then, calculate how much faster it was done under the
new model of patient centricity and assign a price to it.”
Brescia of BBK believes that the question – “what’s the ROI
on being patient-centric?” – is the wrong one to ask anyway.
“I’m concerned about linking the two together – patient
centricity is either a moral imperative or it isn’t,” she states.
But even if cost saving is not the primary issue, proper
management of patient centricity in clinical trials still
depends on valid and reliable measurement – indicators
such as recruitment and retention could fulfill this role.
“In some companies, to get a protocol approved internally,
teams must demonstrate what steps they have taken in
protocol design to incorporate the patient voice – and then
show what aspects of the design have been influenced by
their efforts,” Brescia continues. “If you’re talking about
measuring the ROI of a strategic means to accelerate drug
development, then that’s the patient recruitment discipline.
Recruitment and retention can and should be measured
against ROI; they are two key drivers of research success and
an indication that you have done a good job of partnering
with patients. Patient centricity is demonstrated by your actions
and your ability to improve relations with individuals.”
“Patient centricity is demonstrated by
your actions and your ability to improve
relations with individuals.”
Bonnie Brescia
Founding Principal
BBK Worldwide
Looking to the future, companies should be able to
document the impact of patient centricity in areas like study
design, outreach materials and site performance against
historical trends or a benchmark. This needs to happen,
says Vulcano, because pharma has at some point to prove
patient centricity’s value to the healthcare ecosystem.
“If value can’t be proven, then it is just the latest buzzword in
a competitive public relations stalemate,” he concludes.
“I say ‘stalemate’ as I have never seen a company out there
(and don’t expect to see one) saying they don’t put the
patient first.”

8
What patients really want
The most important question is, of course, understanding
what patients actually expect to get out of their
participation in clinical research – and the Patient-Centered
Outcomes Research Institute (PCORI) is pulling together a
database which will help pharma understand just this.
“Patients and researchers are helping us to build this
Engagement rubric almost like Wikipedia,” says Susan
Sheridan, PCORI’s Director of Patient Engagement. “It’s a
framework for innovation and a sort of crowdsourcing of
engagement activities by the patient and researcher
community in our funded portfolio. We plan to evolve this
framework continually, based on examples from the field in
the future.”
“Patients are eager to hear about
research opportunities, but they do
not want to be infantilized or
subordinated. They want to be afforded
the respect to make their own research
participation decision.”
Rhonda Kost
Clinical Research Officer, Rockefeller University
Center for Clinical and Translational Science
One of the principles in the PCORI rubric focuses on
disruption to patients’ lives. “We want the research and the
research setting itself to be patient-friendly,” Sheridan goes
on. “For instance, with the community of those with physical
disabilities, research should be located next to appropriate
transport facilities, be accessible or use technology to
reduce travel requirements. For a trial involving Latina
women it should all be translated into Spanish. I think
patients will be more demanding about trials being
disruptive to enhance recruitment.”
Part of the problem has been that moves to improve
recruitment for clinical trials have long been limited to
urging doctors to pitch research studies to their patients
and to refer patients to the studies – and this approach has
not worked, says Rhonda Kost of Rockefeller University.
“Only patients, and participants, can tell us what draws them
to, or repels them from research participation,” she says,
suggesting that the first step in the development of that
partnership is to ask the patient or participant what they value
about the research experience. “Patients are eager to hear
about research opportunities, but they do not want to be
infantilized or subordinated,” Kost insists. “They want to be
afforded the respect to make their own research participation
decision, starting from how they hear about research.”
MOTIVATIONS BEHIND CLINICAL
RESEARCH PARTICIPATION
Individual patients have various motivations for
getting involved, and understanding these will bring
pharma closer to achieving the optimization of trial
processes, budgets, and timelines. At the core,
patients want improved treatment prospects – but it
is also very important to bear in mind less tangible
feelings. They can include hope (for their future and
that of their families), altruism (patients want to help
fellow sufferers) and practical considerations (such
as trial duration and convenience, site accessibility,
and transportation needs) which may not be top of
the list of concerns for sponsors but are vital for
patients. The invasiveness or pain of a treatment in
the trial, as well as nature of the disease also have
significant implications for the success of the process.
Do trial participants find the interaction with trial
investigators burdensome? PMG Research, a site
management organization, recently facilitated a patient
panel for one of its key pharma partners.“You might find
this surprising,” says Jennifer Byrne, PMG’s Chief
Executive Officer. “When asked about initiatives and
technology that might lessen the burden of coming on
site, overwhelmingly, the clinical trial participants
expressed that they value the direct contact with
physicians and study staff and see this as one of the
greatest benefits of trial participation.”
Chris Frega, Senior Director and Head of Global Feasibility
and Patient Recruitment, Quintiles, also reports that
gradual progress is being made.“We are increasingly able
to incorporate the patient voice,” he argues. “We have seen
some great results through additional input into protocol

99
development, understanding if patients will actually accept
the design and what their specific motivations may be, and
balancing the views of other experts like investigators,
KOLs, sponsors.”
As part of the Patient-Focused Medicines Development
(PFMD), a new initiative to find common ground between
all stakeholders, Roslyn F. Schneider, Global Patient Affairs
Lead at Pfizer, is one of those pharma industry
stakeholders currently developing what patient-centric
models should look like. Schneider advocates for a
collaborative approach as companies test their models.
“Patients can tell us about their experiences – they are the
experts,” she says. “But they may need a certain amount
of training – for example, on CT protocols – to know what
we’re talking about when it comes to feasibility, and
agreements to protect IP and confidentiality in the context
of drug development.”
The priority over the next couple of years must be to develop
the framework that identifies where patients can be plugged
into the process in a meaningful way, to ensure that they are
not merely token participants. “We also need to ensure that
this extends to a diverse patient population, including those
from under-served communities, and those from different
ethnic and racial backgrounds,” PCORI’s Sheridan adds.
“And we need to measure and evaluate how patients are
making a difference in research. PCORI has created the
WE-ENACT tool to evaluate the engagement in our projects.”
In resource-constrained times, pharma has to figure out how
to become truly patient-centric in a way that is completely
compliant and efficient, thinks Schneider. But while Pfizer
believes it will ultimately produce more revenue as well as
better health, the process of getting to that point cannot
reduce access or slow down the timeline. “That would be
unacceptable,” she insists.
CASE STUDY:
PFMD WORKING GROUP
Patient-Focused Medicines Development
(PFMD): a cross-industry initiative
Getting better patient engagement across the pharma
continuum is the raison d’etre of a new working group
called Patient-Focused Medicines Development (PFMD),
a partnership between pharma and patients seeking to
share ideas and best practice.
It brings together stakeholders across the space via
workshops and meetings in a bid to establish a
“master framework”, setting out how systematic
patient involvement covering the entire medicines
lifecycle would work. The group’s vision is that
medicines will “deliver more relevant and impactful
patient outcomes by addressing unmet patient
needs, and medicine development is faster, more
efficient and more productive”. Currently involving
four patient representatives (two EU and two US),
five sponsor companies and one independent expert,
the informal group focuses on North America and
Europe but insists it has “global intent” and is open
to more members.
PFMD membership
James Anderson GSK
Angelika Joos Merck/MSD
Marc Boutin US National Health Council
Peter Verdru UCB
Lode Dewulf UCB
Jeanne Regnante Merck/MSD
Jan Geissler EUPATI
Roslyn Schneider Pfizer
Anton Hoos M4P (Medicines4Patients) Consulting
Murray Stewart GSK
Diana Hughes Pfizer
Veronica Todaro US Patient Leadership Council
Graeme Johnston UK RA Patient
Gervais Tougas Novartis
“It is a think-tank of like-minded individuals from across the
biopharma eco-system,” explains Jeanne Regnante, Head of
CMO Strategy Office, Chief of Staff to the CMO at Merck.
“We got together to understand the landscape, problems,
best practice, and to chart a course for the future.”

1010
The idea grew out of a CMO roundtable but, crucially, there
was early agreement that discussion should not take place
about patient centricity unless patients themselves were in
the room. “You all start to have opinions about how to do
this,” Regnante goes on. “It’s important to share perspectives
and opinions – we’ll be better together.”The ultimate aim is
helping to create, via a more efficient development process,
medicines which work better for patients.
“You need to bring multiple stakeholders together and
have a conversation around patient engagement,”
agrees fellow PFMD member Marc M. Boutin, Executive
Vice President and Chief Operating Officer, National
Health Council. “How else do we get to a place where it
works for everyone? Collaboration is focused on
interdependence, trying to understand how we’re all
successful in achieving our aims.”
To make all this work, PFMD will have to formalize a consistent
approachtopatientcentricityandhelpshapetheenvironment
externally so this approach in turn becomes the norm. The
challenge will be distilling the range of perspectives from
individual patients, patient advocate groups, pharma
companies – and different departments within them – as
well as regulators. “Perspectives may be different,” he
continues. “Shared definition becomes a starting point.”
Schneider acknowledges that the informal partnership
has to come up with substantial ideas. “We need to work
together across industry groups and patient groups to
develop a framework to include what’s being done
already and give structure that we could all build on,”
she says. “We can’t expect others to embrace and
implement it if we wouldn’t commit to that.”
The need to tackle this issue is apparent because corporate
boilerplate statements are given little credence by the
public – and perhaps even from within their own organizations.
A recent eyeforpharma survey asked industry executives
globally who was spearheading the concept of patient
centricity in their company. 20% said it came from the board,
others said it came from the CMO – but 17% admitted that no
one leads the initiative. “One of the barriers is that companies
think there’s a law against doing it,” says Regnante. “But we
can do it through patient organizations and academia. So the
challenge is cultural, but also finding sponsorship within a
company is critically important to achieve innovation. Cultural
change can happen but you need champions inside.”
One of the biggest challenges around clinical trials is that
they have been designed by researchers mostly removed
from medical management who are using them as an
opportunity to get every possible bit of information
Boutin suggests. While that is understandable, it makes
things arduous for the patient, does nothing for retention
and means there is work to be done in weeding out the
protocols that are not useful at all. “We’re constantly
having to calibrate what we do,” he says. “You’ve got to
be vigilant to get the right balance.”
Issues that are being considered within PFMD at present
include what approaches have been most influential in
transforming the ways trials are designed and various
different examples of involving target groups in this
activity. “Getting patient input should be done in a
variety of ways – we should start now and not wait for
‘perfect’,” insists Regnante. “We are focusing on sharing
best practice and there’s probably ten ways of doing it.
The field is wide open.”
CASE STUDY: PFMD WORKING GROUP continued…

1111
Exchanges must be bi-directional, so any microsite
designed to help patients and caregivers must be
user-friendly. “The industry of yesteryear might have
asked physicians what they thought or used our own
subject matter expertise,” she goes on. “But we’re really
thinking about all different aspects of how we engage
with stakeholders. This is all about patients who are
suffering every day and want solutions to help them and
their families. If they were to say that their engagement
has been valuable then we will have succeeded.”
While the importance of face-to-face interaction is
understood, technology is still likely to be a driver for
patient-centricy, and Schneider expects these issues to
be addressed by PFMD more as the group develops a
framework. “Technology will be critically important
depending on the type of methodology,” Boutin says.
“But the methods will have to be aligned to the questions
and responses that you want to receive.”
As things open up, Regnante believes social media may
be useful some way down the line, for example, but says
there needs to be more one-on-one interaction in these
early stages. “We need to do a better job of garnering
trust,” she says. “We need to start the relationship – I
think we’ll get there in terms of social media but this is a
new relationship and it’s better to talk face-to-face initially.”
PFMD’s members believe trial design is beginning to
change for the better and the emphasis is shifting from
scientific decisions being made about patients towards
issues of judgment about clinical effectiveness with
patients’ input. “This will lead to much higher value
products coming out of the pipeline if we do this right,”
Boutin enthuses.
“You look at how patient engagement is transforming
biopharma, this momentum is becoming embedded and
will spread into delivery models, quality measures, and
reimbursement activities to create new health models.
And I think this will come together in a really nice
eco-system in the next 7-10 years.”
Yet despite all the optimism, no-one in PFMD is under any
illusion about the amount of work to be done. “Resistance
to any change is endemic,” he says. “In all of society”.
CASE STUDY: PFMD WORKING GROUP continued…

12
Patient-centered trials will require new processes to
involve patients in formulating research questions, study
design, trial conduct and disseminating study results,
changes that will we need to be managed in tandem with
clinical needs.“Trials are becoming increasingly complex,”
cautions Chris Frega of Quintiles. “However, as an industry
we need to strive to better balance the complexity and
scientific/data needs with the ability to actually conduct
studies and enroll patients in them in the least invasive
way possible.”
Trials also have to measure outcomes that patients care
about, says Tomasz Sablinski, Founder and CEO of
Transparency Life Sciences, and they should do it in a way
that is least intrusive to patients’ daily lives. “If you can
accomplish both of those things it’s going to be a quantum
leap compared with where we are today,” he suggests.
“We’ve designed seven or eight different protocols: four are
recruiting or are about to recruit patients. We’ve made
several changes to clinical study protocols, several
suggested by patients. Our trial in multiple sclerosis is
based on patients’ feedback – as a result we incorporated
several new tele-monitoring devices into the trial.” But
combining these priorities doesn’t need to require increased
complexity; in contrast it could lead to simplification, says
Paulo Moreira of EMD Serono. “A comprehensive model of
patient centricity will lead to reduced timelines and costs
associated with developing a new drug,” he says.
Pharma companies will also need to be increasingly
flexible in their study plans, and open to perpetual change.
“They could pick two or three ideas such as including
patients in protocol planning and design, implementing
study visit run-throughs, and sharing study results with
patients,” says Bonnie Brescia of BBK Worldwide.
Part of the problem, some experts think, has been that
what is required from studies has also shifted away
from patient needs. Rhonda Kost, Clinical Research
Officer at the Rockefeller University Center for
Clinical and Translational Science, has been
frustrated at the “steady increase” in regulatory and
educational requirements for investigators. Heavy on
theoretical input for human protections, without any
outcome measures from the true user of those human
subject protections – the participant. “How could we know if
consent was truly informed if we didn’t ask the participant
how their experience compared with what they had been
prepared for?” she asks.
The issue of consent is at the heart of making it easier for
patients to become involved in trials in the first place. At
eyeforpharma’s 2014 Patient-Centered Trials Conference,
Tom Krohn, then Business Lead of Eli Lilly’s Clinical Open
Innovation Team outlined a new approach. According to
Krohn, the main problem has been that the industry makes
only incremental improvements in this area. “We build on the
same paradigm over and over,” he says. “We haven’t thought
about the paradigm differently in the consent process.”
Trial design

1313
Trial design
Krohn suggests de-coupling “informed” and “consent” to
create a new frame of reference for working with patients.
The consent process flows out of the clinical study design
protocol, where at present the patient is typically out on the
margins. “They have a real challenge at finding and
understanding what we’re doing,” says Krohn. “We’ve made it
difficult for them, partly because it’s a closed model and also
because of how we engage them.” Krohn suggests making
clinical trial information easy to access online, in the same
way that flight or hotel information has evolved. You would
show patients the entire schedule – for example, is it one
lumbar puncture or three, does a patient have to bring their
mom because they’re going to be knocked out – in advance of
a site visit, rather than the patient having to make a 200-mile
trip to get the document “because we’ve coupled ‘informed’
to ‘consent’. If pharma does not answer these practical
questions upfront, then patients will not understand studies
any better. Krohn notes that “Patients’ questions and
language are very different to ours,” and pharma must make
the effort communicate to patient’s priorities, not their own.
Following on from that, the industry needs to think about
how it can change these points of consent into points of
engagement, says James O’Leary, Chief Innovation Officer at
Genetic Alliance. There are 7,000 diseases and an infinite
number of research questions that could be used to engage
patients – but pharma has too narrow a perspective here.
“We view things differently to real people,” he continues.
“They say: ‘What do I need for me and my family?’ One of the
major missed opportunities in clinical trials is our inability or
unwillingness to allow individuals to use their data effectively,
both in enrolling in trials and using the data generated to
improve their health and contribute to research.”
In a project called PEER (Platform for Engaging Everyone
Responsibly), Genetic Alliance has partnered with
Private Access, a firm which specializes in participant-
centric access controls and privacy management
systems. “Using a technology called Privacy Layer, this
partnership places a user-controlled key in-between
individuals’ data and potential users of that data,”
O’Leary explains. “By giving participants the ability to
activate and share their data, personalized connections
are made and research is accelerated.” This means PEER
is essentially a registry system that looks at things in a
different way, putting control in the hands of people who
can set their own data sharing and privacy settings.
Conversion rates have been promising in people who
click through in this community, O’Leary says. “This is
something people want,” he concludes. “They are
interested in engaging with it.”
Ensuring that patients adhere to their treatments must also
be built in to trial design – otherwise the consequences can
be catastrophic. Bernard Vrijens, Chief Science Officer,
MWV Healthcare and Adjunct Professor of Biostatistics at
the University of Liège warns that poor adherence can lead
to “underestimated efficacy of new drugs to the point of
trial failure, underestimated incidence of adverse effects,
distorted pharma co-economic analyses, and/or
overestimated dosing requirements for marketed
pharmaceutical.” Vrijen and his team have documented the
prevalence of major shortfalls in drug exposure during
clinical trials, something industry must change.“This
situation is no longer sustainable under the current overall
financial pressures on healthcare,” he says.
He suggests electronic compilation of drug dosing history
data is required, and is convinced that smart packages –
which automatically record every time a patient takes a
dose out of the pack – must soon play a central role in trials.
“They provide the means to manage patient adherence and
also enable analyses stratified by reliable measurements of
drug exposure,” Vrijens says. The smart packs record the
time of each package-opening – and are simpler, cheaper
and less intrusive than smart pills.
This innovation has demonstrated success too: Vrijens has
been closely involved in the development programs for new
all-oral HCV treatments and says that electronic
measurement of patient adherence to those treatments has
led to a 97% cure rate – “a level of success that can only be
achieved with almost perfect adherence”.

14
Systematic engagement of patient advocates
The preceding discussion has underlined the importance of
involving the patient, however patient groups have long
complained that their voices are not heard enough
throughout the clinical trial process. “Many patient groups
have voiced that they are often only approached post-
research to help disseminate research findings, but patients
are now calling for greater involvement throughout the
process not just after research is complete,” says Susan
Sheridan, Director, Patient Engagement, Patient-Centered
Outcomes Research Institute (PCORI). “Patient advocates
really have a role reshaping the research to reflect what’s
really important to these groups.”
Joel Beetsch, Vice President of Patient Advocacy, Celgene
argues that pharma is perfectly capable of getting patients
into two-way communication, fostering successful
partnerships from research design and protocol development
onwards. Beetsch states, “Patients and patient advocates can
get involved as part of patient-reported outcome
development and selection, and they can be involved in trial
results interpretation across all phases of a trial,” he says.
Pharma’s benefits should be clear: involving patients in the
design and conduct of research means the research can be
more patient-centered, useful, and relevant. It will also
establish trust and a sense of legitimacy in the findings and
make more likely the successful use and uptake of research
results by the patient community. PCORI has attempted to
achieve this through its Patient-Centered Clinical Research
Network Program, a large, representative, national network
with a focus on conducting comparative effectiveness
research (CER).
As the opportunities for patient advocacy groups are
changing, their role and structure is adapting too. Early
patient advocacy groups worked to protect patients from
over-reach by drug developers, says Bonnie Brescia,
Founding Principal, BBK Worldwide. “What’s happening now
is that these groups are being supplemented by more
disease-specific organizations looking to have their own
voices heard,” she goes on. “There are a number of
organizations looking to develop expertise within patient
communities, giving patients a stronger training and
background on things like ‘what is drug research?’, an
understanding of the differences between Phase II and
Phase III and so on. Patient advocacy in clinical research is
becoming a specific sub-specialty within these groups.”
“There are several ideal roles for patient
advocates. Legislative advocates are
perhaps the most effective voice to help
educate lawmakers about the need to
update out-of-date regulations.”
Jack Whelan
Cancer survivor, warrior and patient leader
These changes in the role of advocacy are also reflective of
the fact that patients themselves do not come ‘one size fits
all’. “There are several ideal roles for patient advocates
because there are several types of patient advocates,” says
patient leader Jack Whelan. “Legislative advocates are
perhaps the most effective voice to help educate lawmakers
about the need to update out-of-date regulations.” They can
also influence government on funding to support clinical
research, he adds, while research advocates – patients who
work on behalf of biopharma firms, for instance – also help
educate physicians about what treatment options are
available in trials. “These physicians are the first contact for
most patients,” says Whelan. “Very few participate in
research because they are not asked about it.”

15
CASE STUDY: ELI LILLY’S COLAB
Eli Lilly Co’s CoLAB initiative is a study design platform
and process which Lilly hopes will make its programs
more patient centric, says Tom Krohn, when speaking as
Business Lead of Lilly Clinical Open Innovation Team.
“CoLAB is a dress rehearsal of a study,” he explains.
“What would it be like to execute it? There isn’t a site I
have visited that didn’t say ‘I wish we can be more
involved in study design’. If we did that right, we have
less rework, fewer amendments, and the patients’
experience would be better.” Through CoLAB, the
company has in effect made trials part of a digital
design process, allowing investigators to do scenario
analysis – from a patient burden, cost and time
perspective, for example. “One of the things we’re trying
to do in complexity management is to make it real-time,
fast, easy,” says Krohn.
Lilly takes a draft study design from its digital canvas and
puts it into an internal collaboration platform such as
SharePoint in order to open it up to various stakeholders
such as study teams and sites. These come together in
virtual ‘jam’ sessions. “It’s about enabling people to
have a different type of conversation,” he says. The idea
is to begin discussions about the protocol, such as
whether the schedule of events works or if there are
eligibility issues in a specific country, with the CoLAB
initiative bringing study coordinators, investigators,
patients and clinical staff into the process. Scientific
perspectives are clearly important, but then so are
operational ones. “It’s not that the sites can’t follow
protocols,” says Krohn. “It’s just that we make them too
complicated.” The purpose of CoLAB is to iron out the
kinks, avoiding late amendments, allowing teams to flag
up concerns about dosing or screening and so on, and
challenging assumptions. Scenario analyses just take
minutes, with CoLAB enabling teams to physically
simulate the space so that it will be possible to see what
it is going to be like when you enter the clinic.
Lilly measures whether participants found it useful to
be involved in CoLAB and seeks to establish if sites gain
the ability to execute studies better and improve
engagement levels. “We find many things that we
assumed were fine but were not,” Krohn says. “You end
up with a bunch of protocol improvements and
adjustments.” The results have been impressive: Lilly
has made 189 protocol changes via simulation prior to
eight studies – all because it put “the right people in
the room”.
Patient-centered systems and trial technology
Not every patient group is representative of all patients’ needs and desires – however not all pharma companies are at present
set up in a way which makes engagement with individual patients a viable alternative. One industry initiative that is making
patient-centered trial management more of a reality is Eli Lilly Co’s CoLAB.

16
Bettersitemanagement,clearerpatientfocus
Talking about the patient focus, a large part of the success
depends on the trial sites themselves. Strategic partnerships
can enhance operational oversight, drive efficiencies and
speed up the resolution of issues between sponsors and
sites. “We need to help sites engage effectively with
potential subjects, supporting sites with methods, tools and
materials for easily uncovering and answering patient
resistance to enrollment,” says Greg Koski, President and
Co-Founder, Alliance for Clinical Research Excellence and
Safety (ACRES). “This, combined with integrating electronic
health records and trials databases, more effectively
applying geographic information systems and creating a
global network of sites of excellence, will result in a much
more effective approach to patient recruitment, retention
and overall conduct of clinical trials.”
However, moves towards greater patient centricity are likely
to see traditional roles changing. Will there be a role for
clinical trial sites in an era of ‘direct-to-patient’ efforts?
“There are going to be some forms of research that lend
themselves to direct-to-patient,” explains Michael Jones,
Senior Director, Clinical Operations, Eli Lilly Co. “Other
types of studies will continue to require participation of a
healthcare practitioner. We are not setting out to expressly
disintermediate the trial sites. Our aim is to relieve the
burden. I see that there will be a spectrum of opportunities
to remove that burden and increase convenience for the
patient and the site.”
In order to do so Sharon Hanlon, Director of Clinical Trial
Partnerships at Bristol-Myers Squibb, says it is important to
identify steps to establish a reliable, collaborative system of
sharing metrics. These would monitor start-up and
enrollment activities as well as site-patient relationships,
tapping into the potential of patient advocacy involvement
to support and enhance site relationship management.
“The sites are our key collaborators,” she says of her work
developing the relationship between BMS and its sites.
The company wanted to ensure it had adequate criteria to
evaluate unique skills and talents that sites have, as well
as to identify future partners. The ability to sit down with
site representatives and talk about issues such as
electronic medical records, resources, and changes in
development plans is key – but the most important thing is
to have core sets of metrics on performance and quality
that can be used.
BMS set up research advisory councils, bringing sites
together to look at what they are doing and how they could
better help patients. These councils found that sponsors
tended to want to improve the start-up experience and
increase access while sites wanted awareness of what is
coming – something better collaboration would help with.
“They see our book of work from the time that it’s internally
approved,” says Hanlon. This gives them the opportunity to
input into the design of study, with teleconferences set up
with KOLs at sites on issues of recruitment and so on.
She adds that having a single point of contact has made a
significant difference to the relationship, allowing her to
partner with sites more effectively on improvement. One site
which had an average 18-month start-up time has now
trimmed its processes with BMS’s help and is down to an
average three-month lead time. “They’ve been the best
recruiter in some of our studies,” says Hanlon.
The message is clear: with effective management, sites can
improve their performance. Hanlon and the sites put
together five different criteria and success factors to be
judged by qualitative and quantitative measures:
• Strategic alignment
• Operational excellence
• Resources
• Information technology
• Process compatibility
Increased transparency and frequency of communication
between site and sponsor can help identify potential new
areas of collaboration based on joint interest and capability,
as well as the implementation of process improvements
based on performance measures and qualitative feedback.

1717
Better site management, clearer patient focus
“It has been a huge learning experience, listening to the
sites,” she goes on. And it has led to real changes: following
input from various teams, BMS has been able to alter its
SOPs to increase the number of satellite sites it could use,
an example of the sorts of broader benefits that the
collaboration has brought, outside of simply leveraging
synergies in the trial process.
The collaboration has also been leveraged to build connections
with site-specific advocacy organizations, health equity/
disparity groups and to gain the patient perspective. There
has also been increased awareness of processes with the
opportunity to standardize – creating standard pre-filled
documents, for instance – and a positive trend toward
meaningful improvements in key study milestones. “Since
the partnership, one institution has tripled number of
activated studies,” Hanlon points out. “The access to health
equity, minority populations associated with some of these
sites have really been a meaningful difference and have
really helped us in the long run.”
“We need to start the relationship with
patients. We’ll get there in terms of social
media but this is a new relationship and
it’s crucial to talk face-to-face initially.”
Jeanne Regnante
Head of CMO Strategy Office, Chief of Staff to
the CMO, Merck
For pharma, building a network with influencers using online
channels is one thing, but getting insights back from
patients via social media is quite another. Companies worry
this could lead to patients sharing symptoms or speculating
over the treatment assignment and are concerned about
unsolicited safety reporting and privacy violations.
But engaging patients in non-traditional ways is important
as they increasingly use the internet to communicate and to
become better informed. Susan Sheridan, Director, Patient
Engagement, Patient-Centered Outcomes Research Institute
(PCORI), says patient recruiting via social media and patient
groups crafting their own privacy and data-sharing
agreements are both already happening.
“A really simple but important initiative is where a patient group
has rewritten a consent form in collaboration with a patient
group in the UK to make it understandable and patient friendly,
and is now recruiting via Facebook in both countries,” Sheridan
explains. “There are also examples of patient groups suggesting
the use of cell phone technology to report patient reported
outcomes and for research interventions; there are many
examples of patients changing or being the source of the research
questions, such as a research question that was identified by an
adolescent with diabetes. We’re seeing some interesting shifts:
for instance, in one research program patients shortened a survey
tool developed by a research team; it originally included 22 items
and took 45 minutes to complete but with patient intervention
the tool now has 15 items and takes 20-25 minutes.”
“The risk of sharing is sharing – but the
benefit of sharing is sharing too!”
Roslyn F. Schneider
Global Patient Affairs Lead
Pfizer
Use of social media throws up real regulatory problems for
pharma – but there also ingrained cultural barriers to overcome.
The future of trial communication:
Online, social, mobile?

1818
CASE STUDY:
JANSSEN’S MYCENTRALCARE
“The point is: let’s start talking about it,
because it’s going to happen whether
we want it to or not.”
Elise Felicione
Director, Clinical Trial Innovation
Janssen RD
Janssen’s MyCentralCare is a secure, private online resource
to support patients in a Janssen study on obesity in the
US. The idea behind the portal is to help patients easily
find information, and it includes FAQs and a study schedule.
It explains to a patient what visits have been completed,
what is upcoming, study procedures and requirements
(for example, do they need to fast in advance), a link to
Google maps so they can plan directions and the
opportunity to sign up to visit reminders via text or email.
It means that patients can go online rather than calling
the site and Janssen hopes that it will improve
understanding of process in general – allowing patients
to bring up the site on a tablet, for instance, when
discussing their treatment with family or carers.
Elise Felicione, Director, Clinical Trial Innovation, Janssen
RD, oversaw the project and states that the objective was
to put the patient first at the beginning of the design
procedures. Felicione says, “We brainstormed what we
could do to make our studies more patient centric.” In 2013,
the company actually piloted the new scheme as part of a
short clinical trial. There were pros and cons: as Janssen
developed this internally it was cheap to run and they had
complete control over the system – but the burden of
ownership meant that further implementations had to be
resourced by the company alone. Most Institutional Review
Boards (IRB) approved it without making any changes,
which was positive and showed the value of good
preparation – but the project was dependent on the trial
timelines. These were delayed, which meant the pilot itself
also fell behind schedule, by a total of nine months.
The company learned above all that patient-facing innovation
takes time, with adequate build-in required for stakeholder
review and approval. Janssen still plans to make this a global
tool, perhaps with a multi-language, multi-country website
with a web or mobile app. Including dynamic content means
on-going approvals are required but this also creates the
possibility to expand the scope post-trial, through sharing a
study results summary and invitations to join an alumni
community. Felicione believes that this feature brings pharma
to the next logical step for patient centricity: to facilitate
patient-to-patient communication – something she argues
can no longer be ignored (see Social media below).
“The first step is to start the conversation,” Felicione
suggests. “If we’re not talking about it, we’re sticking our
heads in the sand.” This could be disastrous, since
patients are communicating anyway, leaving the conditions
“ripe for a perfect storm”. Patients have no malicious
intent, she says, they just want their study to succeed.
Pharma should consider educational content about the
dangers of sharing too much or too broadly, patient-
authored articles, ‘letters to the editor’ or hosting a closed
and moderated patient discussion forum on Facebook.
The chances are there will be at least one patient who will
tweet or blog about their involvement, so pharma might
be better off embracing this, saying that if patients have
something to share then why not pass it on to the sponsor
or to the IRB for review before posting it on the site. “The
point is: let’s start talking about it because it’s going to
happen whether we want it to or not,” Felicione concludes.
“The key is to put some organization around something that is
meant to be disorganized,” says Roslyn F. Schneider, Global
Patient Affairs Lead at Pfizer. “The risk of sharing is sharing – but
the benefit of sharing is sharing too!The reluctance from pharma
is not because pharma doesn’t think it’s a good thing to do
– but we’re still a science-based industry and we have to ensure
that our engagement is scientific and has a methodology to it.”
Whatever the barriers, Chris Frega of Quintiles thinks that
technologies to share what is happening and keep patients
engaged in a trial have a place. “These can be social media,
online communities and some that are more study specific,”
he suggests. “By building communities of patients who are
engaged in their own treatments, they can more easily be
informed of their options should studies be initiated.”
The future of trial communication: online, social, mobile?

19
Mobile trials and direct-to-patient solutions
The extraordinary potential of mobile health has only
recently begun to be exploited by clinical trial sponsors.
Traditional methods of executing clinical trials could be
thrown out with the proverbial bathwater if the
pharmaceutical industry can get to trips with the myriad of
possibilities that utilise mobile technology.
Sensor technology has exploded in recent years, and the
options provided, in the form of wearables, smartphones
and remote devices, coupled with powerful apps and
pervasive wireless access, means that direct-to-patient
(DTP) solutions are already available.
Yet challenges obviously exist in terms of integrating these
solutions into the current clinical trial set-up. Instrumenting
patients with wearable technology has the power to
transform clinical trials, but pitfalls exist in terms of
potential errors. These include:
• Device failure
• User error
• Privacy/security
• Data integration
• Regulatory compliance
• Site preparedness
• Introduction of bias
• Poor quality data
Scaling represents another challenge. For example, a recent
mHealth initiative generated 18 million data points per
patient per day. Managing data volumes on this scale brings
its own set of difficulties.
The ubiquitous providers of mobile technology, Apple, have
now made a new foray into health. They recently unveiled a
new API called Research Kit, built as an open source
framework and designed to help medical researchers collect
data from research subjects. The new framework was
co-developed by Apple and a group of renowned academic
institutions including Stanford University, Massachusetts
General Hospital, Dana Farber Cancer Institute, and the
University of Oxford.
Essentially Research Kit provides researchers with a
platform that they can use to build a data collection app and
tap into Apple’s 700 million customer base. The API provides
developers with a wealth of data points that they can then
utilize to collect data in support of the study being
conducted. Apple users will retain complete control over
what data is shared, and what data will be kept private.
Researchers can tap into the accelerometer, microphone,
gyroscope and GPS sensors in the iPhone in order to collect
a plethora of data. Five health systems have now developed
apps in support of ongoing research efforts using the
Research Kit framework and many more are sure to follow.
In retrospect, this seems a no-brainer. Apple’s apps “already
help millions of customers track and improve their health,”
said Jeff Williams, Apple’s senior vice president of
operations, in a statement.
“With hundreds of millions of iPhones in
use around the world, we saw an
opportunity for Apple to have an even
greater impact by empowering people
to participate in and contribute to
medical research.”
Jeff Williams
SVP Operations
Apple

2020
CASE STUDY: MEDIDATA AND GSK
Many mobile health innovation projects are already
underway, however, Medidata, a provider of cloud-based
solutions for clinical research in life sciences, recently
announced the completion of a method development
project conducted in partnership with GlaxoSmithKline
plc (GSK) to evaluate the impact of unifying mHealth
devices with cloud-based technologies in a clinical trial
setting. The joint initiative assessed the capabilities of
mHealth tools and evaluated how they could be used to
enable a new model for clinical trial conduct that aligns
site and patient needs with faster study execution and
reduced costs.
Medidata and GSK provided program participants with
two wearable devices – Vital Connect’s HealthPatch®
MD
and ActiGraph’s wGT3X-BT Monitor – which continuously
measured vital signs, electrocardiogram (ECG) data and
activity levels. Participants used Medidata Patient Cloud®
,
a mobile app for patient-reported outcomes offered as
part of Medidata’s industry-leading technology platform.
The participants carried smartphones that captured data
from the mHealth devices, pulled this data into the
Medidata Clinical Cloud®
and then mapped it to the
clinical record. Participants continued with their usual
daily routine and only checked in with the performance
lab at the project’s beginning and end.
“The effort indicated that mobile devices can support
the long-term goal of lessening the burden on patients
participating in studies by streamlining routine
procedures, eliminating unnecessary ones and
reducing visits to clinical trial sites,” said a statement
from Medidata.
“We gathered data on an unprecedented scale—
collecting more than 18 million data points on activity
and vital signs per participant per day. This is an
extraordinary level of in-life, real-time patient
instrumentation for clinical trials, which will create new
disciplines and new opportunities for life science
companies,” said Glen de Vries, Medidata’s president.
Another project has seen Medidata strike a deal with
Garmin to offer its clients the use of vívofit activity
trackers in clinical trials. The choice of vívofit gives
an indication of the characteristics clinical trial
sponsors may prioritize as wearables start to take off
in research.
This particular fitness wearable device has a remarkable
one-year battery life and water resistance –features that
mean a participant can wear it 24/7 for the duration of
most studies.
The Clinical Trials Transformation Initiative (CTTI) is the
public-private partnership working to identify and promote
practices with the aim of improving the quality and
efficiency of clinical trials.
The CTTI states that currently available remote technologies,
including, for example, mobile health delivery systems,
telemedicine, and remote sensor devices, may increase the
efficiency of clinical trials. The Initiative has now established
a working group to look at “Using Mobile Technologies and
Other Off-Site Methodologies to Facilitate Clinical Trials”.
They say that an increase in the use of mobile/remote
technologies in clinical trials, could potentially lead to
expanded improved patient experience, continuous high
quality data acquisition, reduced costs, increased efficiency,
and fewer losses to follow up.
“Mobile technologies hold the prospect of
reducing or eliminating visits of trial
participants to study sites and may result in
more efficient and reliable data collection.
The program aims to determine how mobile
technologies can be used to improve clinical
trials in areas of remote monitoring/
engagement and new novel data collections
methods to enhance knowledge of disease
trajectory and treatment efficacy”

2121
Clinical trial sponsors have struggled for years with trial
participants forgetting to complete patient-reported
outcome (PRO) forms until the day of their site visit. This
latest collaboration could fulfill the dream of continuous
data collection is unappetizing for sponsors. The device
tracks distance walked, steps taken, hours slept and
calories burnt, is controlled by one button and shows
fitness data on an LCD screen. In Medidata trials, all the
data will be uploaded to its cloud-storage system, on
which data crunchers can integrate the wearables’ feed
with traditional sources of clinical research information.
Regulation is another issue of contention, but providers
of mHealth solutions have been working closely with the
FDA etc in order to ensure that data is FDA compatible.
The Agency recently issued guidance to provide clarity
and predictability for manufacturers of mobile medical
apps, and said “the Agency will continue to evaluate the
potential impact these technologies might have on
improving health care, reducing potential medical
mistakes, and protecting patients.”
Along with understanding key unmet needs from the patient
perspective, and integrating their voice into the
development of new drugs, there is a need for pharma to
work closer with regulatory agencies. “We’re all engaging
with FDA and EMA,” says Marc M. Boutin, Executive Vice
President and Chief Operating Officer, National Health
Council. “It’s hard for FDA to be integrated into these efforts
but they’re extremely interested in this – there is a great
deal of work going on there in how they can facilitate patient
engagement within companies.”
Patient groups are driving regulatory decision-making and
science in directions that are more patient-focused,” says
Tom Sellers, Senior Director, Patient Advocacy and
Corporate Philanthropy, Takeda Oncology. “For example,
we’re doing a patient preference study in multiple
myeloma and have used our multiple myeloma
ambassadors to focus group and develop it, and we are
using the International Myeloma Foundation to field the
survey,” he goes on. “This means we are leveraging the
voice of the patient and when the survey is complete it will
reflect a range of views from patients, KOLs from academia
and our company. That will be a much more powerful and
robust result when you go to the FDA or a payer than a
simple survey would be.”
The Holy Grail for pharma is having patients involved early
on to help accelerate the regulatory process. “Such
regulatory requirements will dictate the extent of patient
centricity in a study,” comments Javier Zambrano, Director,
Medical US Avonex/Plegridy, Biogen Idec.
Regulators must do more to involve patients, demands
patient leader Jack Whelan. “Until educated, engaged
patients are compensated for their time and effort
participating as a thoughtful information resource,
developers will continue to struggle to find reliable patient
voices,” he says. “This is a regulatory issue. Except for their
personal experiences as a patient, most patients are no
more credible than ‘the man in the street’ until they become
seriously involved in the management of their particular
disease and engaged in the subject of drug development.”
The FDA and patient centricity:
What are regulators doing?
CASE STUDY: MEDIDATA AND GSK continued…

2222
Efforts to more effectively incorporate the patients
perspectives throughout the drug development pathway are
growing, and the regulators are not only aware of this, but
have made active progress in formalising this new approach
to patient participation in clinical trials. The US FDA has
made significant moves to give patients a more active role in
medical product development and regulation, with the FDA
Safety and Innovation Act enshrining this commitment to
provide patients with an active role in the drug development
process into legislation.
The FDA’s five-year project Patient Focused Drug
Development (PFDD) was launched in 2012 and reflects a
larger movement to ensure that patients’ perspectives are
meaningfully integrated into the drug development process,
as well as regulatory decision-making. According to the
Administration, the patient perspective will provide “context
in which regulatory decision-making is made, specifically
the analysis of the severity of the condition treatment and
the current state of the treatment armamentarium for a
given disease”.
“We want to learn about the clinical context of each disease
from the patients’ point of view and experiences,” said
Theresa Mullin, Ph.D., director of the Office of Strategic
Programs in the FDA’s Center for Drug Evaluation and
Research (CDER).
The Administration worked to identify some 200 patient
representatives based on their experience, FDA training, and
clearance on conflicts of interest. By the end of 2017, 20 public
meetings will have taken place, each targeting a specific disease,
where patients are asked to assess their available treatment
options and the therapeutic benefits that matter most to them.
Richard Klein, head of FDA’s patient liaison program in the
Office of Health and Constituent Affairs, said at a recent
conference on PFDD that qualified patient representatives not
only have experience with a disease or condition, but are active
in patient advocacy organizations, knowledgeable about
treatment options, and able to grasp basic scientific principles.
These patient representatives are now engaging in
consultations with FDA review divisions and in additional
meetings with sponsors.
Over time, this increased attention to patient perspectives
will “change the way clinical trials are designed and carried
out,” Klein has observed. He has also said that patient input
at pre-IND meetings can help design informed consent to
encourage enrollment. He added, however, that the trickiest
issue for including patients in sponsor meetings is screening
for conflicts of interest.
Public participation has been strong, and the resulting
reports are helpful in developing disease-specific guidance
and new outcomes measurement tools. Patient groups have
been enthusiastic and pro-active, and are now organizing
additional external meetings to continue their discussions.
“We are gratified by the enthusiastic response within the
patient community to PFDD, and we look forward to
continued success with these meetings and the long-term
benefit they can offer for drug development in important
therapeutic areas,” Mullin has said.
She added that the “Voice of the Patient” reports published
after each meeting “serve an important function in
communicating to both the FDA review staff and the regulated
industry what improvements patients would most like to see
in their daily lives”. The FDA hopes that these reports will
strengthen the structured framework for benefit-risk
assessment in the new drug process required by FDASIA.
The FDA’s PFDD initiative has also inspired many companies
within the pharmaceutical industry to re-think their original
approach to patient engagement activities.
The FDA and patient centricity: What are regulators doing?

2323
The FDA and patient centricity: What are regulators doing?
“In the past, the industry’s approach to patient engagement
was primarily anecdotal and ad hoc, with a project here and
there,” said David Verbraska, VP, worldwide public affairs and
policy at Pfizer, agreed. “By being patient-centric and adding
transparency and interaction all along the RD and market
life cycle, patients help us achieve the best public health
outcomes and avoid the worst-case scenario,” he said – this
worst case scenario being a drug receiving approval from the
FDA that does not in fact meet patients’ needs.
Mullin recently proposed important next steps as patient-
centered drug development continues to grow and become
embedded in clinical trial protocol. She said the goal should
be to 1) advance the science of patient input and 2) provide
FDA guidance to patient advocates and drug developers.
Mullin has also commented that companies “could play an
important role in collaborating with patient groups and
researchers in follow-up work to develop clinical outcome
assessment tools or patient-reported outcome measures for
clinical trials that will better capture the patients’
perspectives.”
Paul Kluetz, Acting Deputy Office Director of the FDA Office
of Hematology and Oncology Products, has previously
clarified that the Agency’s Patient-Reported Outcome (PRO)
guidance outlined in 2009 was very necessary, as many
innovators had been trying to develop instruments in the
absence of FDA recommendations of a systematic approach.
As the FDA pursues an approach that is more flexible and
conducive to innovation, it is important that the Agency
continue to make progress to provide timely and robust
feedback to those seeking to develop and use clinical
outcome assessments, and continue to consider ways to
improve communication to stakeholders of complex
regulatory decisions.
In line with the FDA’s patient-centered activities, the
regulators are now reaching out to the public to ask what
more they can do to improve their efforts. Last year the FDA
released a new Federal Register posting indicating that it
will establish a federal docket to allow members of the
public – and in particular patients and patient groups – to
weigh in on “FDA activities performed under the FDASIA
Patient Participation in Medical Product Discussions”.
The intent is to gather input from stakeholders on
“strategies to obtain the views of patients during the
medical product development process and ways to consider
patients’ perspectives during regulatory discussions”,
according to the FDA. It is also seeking feedback from
patients about sponsor meetings.
The FDA has said that the hope is that the long-term
impact of the PFDD program will be a “better, more
informed understanding of how the entire drug
development community might find ways to develop new
treatments for diseases”.

24
Expectations and outlook
Looking ahead at what can be done, Joel Beetsch, Vice
President of Patient Advocacy at Celgene thinks the industry
should focus on three key priorities for the Patient-Centered
Clinical Trial:
• Starting with patient-friendly and patient-focused
endpoints.
• Further identification of patient-reported outcomes and
quality-of-life metrics.
• Major emphasis on data sharing throughout the overall
trial process.
He explains that the last point “can shorten the process –
and saves money – and helps us avoid having to collect
unnecessary data from patients.” Beetch points to the
PhRMA and EFPIA principles on sharing data around clinical
trials which were instituted on January 1, 2014, and another
data sharing project, the Project Data Sphere initiative. “It
enables us to share, integrate, and analyze our collective
historical cancer research data in a single location, so
researchers can share the control arm of Phase III clinical
trials,” he explains.
Electronic health records have the potential to be a
tremendous tool in bringing the right trial to the right
patient, suggests Jennifer Byrne, PMG’s Chief Executive
Officer. “Patients can be significantly empowered in
decision-making regarding their health by having more
information as to what clinical trials might be available to
them as a care option for their condition or disease,” she
says. “In addition, wearable devices will provide real-time
surveillance to health care providers and stand to further
promote patient safety, compliance, and data integrity.”
The migration to a personal health record will
certainly lead to more engaged patients and
thus more engaged trial participants, thinks
David Vulcano, AVP Responsible
Executive for Clinical Research, Hospital
Corporation of America. “Integrating
electronic health records with other
healthcare information systems can be
used to facilitate communication and build
relationships with patients,” agrees Greg
Koski of ACRES.
The development and implementation of standards and
APIs to enable wide-scale integration of information
platforms, especially with regard to drug safety, clinical trial
and health information will empower patients and improve
the clinical trials process, he continues. Koski cites the
example of a project on which ACRES is collaborating with
the Swiss Institute of Technology that enables patients to
‘deposit’ their electronic health records into a secure
repository and retain control over who has access to them
and how they will be used. While the future is notoriously
hard to predict, one thing is certain: patients’ health
information is personal and private, which means patients
will increasingly control access.
“Our customer is the patient. If you’re
serving the patient, the business will
succeed.”
Tom Sellers
Senior Director, Patient Advocacy and
Corporate Philanthropy, Takeda Oncology

25
Conclusion
As the roadmap in this paper exemplifies, patient centricity
is here to stay. Pharma now needs to translate the ideas
into actions, elevating it from the buzzword bubble to
working organizational reality. It is fair to say that the
impact of existing patient engagement activities is rarely
being measured by the industry and more metrics are
required to reinforce the case for doing so. We need a
master framework.
Internally, patient centricity must be deeply engrained in a
company’s values and performance management systems.
Pharma can innovate and readjust organizational structures
and drive cross-functional partnerships to win over patients,
contain budgets, and manage studies more effectively.
However, there is resistance to change legacy systems and a
degree of fear about engaging with the patient (for reasons
including compliance and lack of control over outcomes).
“You have to start in multiple places and bring teams
together”, explains Roslyn F. Schneider of Pfizer. “For
example, later in development, market research, patient
adherence and customer engagement related to many of our
already-marketed products. Many people in teams earlier in
development may not have been exposed to this thinking
and approach.”
There is more work to be done throughout the industry but a
few companies have already shown that change can be
achieved and, through their examples, it is possible to begin
plotting a roadmap for the future. As with anything, change
requires the commitment of leadership, after which the rest
often follows. eyeforpharma’s most recent industry survey
shows that senior management and board level buy-in is
vital for companies which are serious about furthering
patient-centred drug development (n=165). In pharma
companies where no one spearheads patient centricity, only
21% of respondents consider it a top priority. Similarly,
where it isn’t considered a top priority, only one in five
respondents recognize senior leadership efforts. But where
corporate management spearheads patient centricity, 76%
confirm that is has been the top priority for their
organization in 2015.
How can they start the process? “Transparency and
education are the critical first steps to empowering patients,
and new technologies that help the research community
more readily share information and engage with patients
and the public may prove to be of great value,” says Zach
Hallinan, Director of Patient Communication and
Engagement Programs at CISCRP. “However, our focus
should rarely be on the technology itself. Most important is
creating opportunities for patients and research
professionals to interact and learn from one another in
meaningful ways, and the most innovative approaches will
be those that put human connections first.”
If companies are wondering internally what they can “get
out” of patient centricity, they are surely thinking about it in
the wrong way.
“That’s what distinguishes a patient-centric company from a
traditional one,” suggests Tom Sellers, Senior Director,
Patient Advocacy and Corporate Philanthropy at Takeda
Oncology. “A patient-centric company is not starting with
that question per se. If you’re truly putting patients first
then you don’t have to convince the company that there’s a
financial benefit. In most other businesses, if you’re
satisfying the customers’ needs then you’re going to do well.
Our customer is the patient. And if you’re serving the patient
then the business will succeed.”
38%of clinical trials professionals are
planning to leverage e-clinical
technologies and mHealth applications
in the near future
57%of respondents think that
Adaptive Trial Design will have
a huge impact on reducing
clinical trial costs
WHAT THE FUTURE HOLDS:

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