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How and Why to Involve Patients in Drug Development: Perspectives From the Pharmaceutical Industry, Regulatory Authorities, and Patient Organizations

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How and Why to Involve Patients in Drug Development: Perspectives From the Pharmaceutical Industry, Regulatory Authorities, and Patient Organizations

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Background Despite increasing interest and focus on patient-centric approaches to drug development, there might still be divergent views between key stakeholders in how to perceive patient involvement and how possibly divergent views influence the role of patients in the drug development process. The objective of this study is to explore how the perception of patient organizations, pharmaceutical companies, and regulatory agencies influence the role of patients in drug development. Method A qualitative interview study based on 12 semi-structured interviews with representatives from the 3 stakeholders. Interviews were transcribed, and data were analyzed using a social constructivist approach in the form of systematic text condensation. Results Three main perceptions of patient involvement were identified: “a way to improve quality of life,” “a way to avoid business failure,” and “a way to foster a faster drug approval process.” Transparency, trust, and clarification of expectations and roles were factors perceived as prerequisites for a valuable collaboration. Furthermore, a required cultural mindset change in the pharmaceutical industry, the lack of a common framework, patient organizations having limited resources available, and concerns about what to do with patient responsibility were perceived as the most important barriers for patient involvement. Conclusion Based on the findings, the pharmaceutical industry, patient organizations, and regulatory authorities were labeled as “pioneer/dominant,” “unaware/quiet,” and “hesitant,” respectively. The 3 behavioural descriptors reflect a limited negotiation of the role patients have in drug development. Thus, the pharmaceutical industry appears to be the largest influencer with regard to patients’ role in drug development.
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Original Research
How and Why to Involve Patients in Drug
Development: Perspectives From
the Pharmaceutical Industry, Regulatory
Authorities, and Patient Organizations
Marianne Botoft Hansen, MSc
1
, Lotte Stig Nørgaard, PhD
2
,
and Christine Erikstrup Hallgreen, PhD
1
Abstract
Background: Despite increasing interest and focus on patient-centric approaches to drug development, there might still be
divergent views between key stakeholders in how to perceive patient involvement and how possibly divergent views influence the
role of patients in the drug development process. The objective of this study is to explore how the perception of patient
organizations, pharmaceutical companies, and regulatory agencies influence the role of patients in drug development.
Method: A qualitative interview study based on 12 semi-structured interviews with representatives from the 3 stakeholders.
Interviews were transcribed, and data were analyzed using a social constructivist approach in the form of systematic text
condensation.
Results: Three main perceptions of patient involvement were identified: “a way to improve quality of life,” “a way to avoid business
failure,” and “a way to foster a faster drug approval process.” Transparency, trust, and clarification of expectations and roles were
factors perceived as prerequisites for a valuable collaboration. Furthermore, a required cultural mindset change in the phar-
maceutical industry, the lack of a common framework, patient organizations having limited resources available, and concerns
about what to do with patient responsibility were perceived as the most important barriers for patient involvement.
Conclusion: Based on the findings, the pharmaceutical industry, patient organizations, and regulatory authorities were labeled as
“pioneer/dominant,” “unaware/quiet,” and “hesitant,” respectively. The 3 behavioural descriptors reflect a limited negotiation of
the role patients have in drug development. Thus, the pharmaceutical industry appears to be the largest influencer with regard to
patients’ role in drug development.
Keywords
Patient involvement, patient engagement, drug development, social constructivism, qualitative, regulatory science
Introduction
In recent years, the pharmaceutical industry has made a plea in
favour of a more patient-centric approach to drug develop-
ment,
1-4
which has been echoed by some patient organizations
and regulatory agencies.
5-7
Traditionally, the role of the patient
in drug development has been that of a clinical trial subject.
Direct engagement with patients has not been practiced by the
pharmaceutical industry because of regulatory/legal restric-
tions.
8
Today, an increasing body of literature,
1,3,9-14
as well
as multiple patient involvement initiatives,
6,15-20
indicates that
patient involvement in drug development (PIDD) is a highly
topical issue, and great attention is being paid to how to move
drug development in a more patient-centric direction.
The scientific literature identifies the 3 main reasons for
involving patients and the public in biomedical research.
8,9,21
First, patients have a moral right to be involved in decisions
that may substantially affect their health and lives. Second,
health research should be a democratic process involving all
relevant stakeholders.
22
And last but not least, patient knowl-
edge can improve the relevance and quality of health
1
Copenhagen Centre for Regulatory Science, Department of Pharmacy,
Faculty of Health and Medical Sciences, University of Copenhagen, Copenha-
gen, Denmark
2
Social and Clinical Pharmacy, Department of Pharmacy, Faculty of Health and
Medical Sciences, University of Copenhagen, Copenhagen, Denmark
Submitted 04-Feb-2019; accepted 25-Jun-2019
Corresponding Author:
Christine Erikstrup Hallgreen, PhD, Copenhagen Centre for Regulatory Science,
Department of Pharmacy, Faculty of Health and Medical Sciences, University of
Copenhagen, Universitetsparken 2, DK-2100 Copenhagen Ø, Denmark.
Email: christine.hallgreen@sund.ku.dk
Therapeutic Innovation
& Regulatory Science
1-9
ªThe Author(s) 2019
Article reuse guidelines:
sagepub.com/journals-permissions
DOI: 10.1177/2168479019864294
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research.
10
This development may be good news for the
patient. Nevertheless, it raises the question of different motiva-
tions. Coming from different agendas and cultures, it is reason-
able to assume that the pharmaceutical industry and patient
organizations have different incentives for entering into a col-
laboration and generally hold diverse perceptions of this emer-
ging concept. Despite the increasing awareness of the necessity
of bringing patients into the heart of drug development, patients
are not yet routinely involved.
23-25
Rather PIDD is described as
inconsistent and fragmentary on a broader level.
10,24,26
Current efforts seek to collect good examples of PIDD
4
and
to develop frameworks and recommendations for PIDD
15,16
as
well as explore different stakeholder views on PIDD and their
perspectives on how and when in the drug development phase
patient should be involved.
25
However, to our knowledge, stud-
ies exploring stakeholders’ perspectives on PIDD have not yet
been based on social theory. Science is per se theory-based,
uses systematic research techniques, and is cumulative and
predictive.
27
Theories are thus bodies of knowledge that allow
a more elaborate/complex or sophisticated framework for
understanding the world, thus providing the researcher with a
framework for understanding and organizing his or her empiri-
cal findings
28
. For these reasons, this study was performed
based on a specific social theory, more specifically a social
constructivist theory. Social constructivist theories are suitable
as theoretical foundations for research aiming to describe and
analyze development processes and the negotiations that take
place between stakeholders.
From a social constructivist perspective, different social
groups’ views and perceptions influence how a technology, a
profession, an existing perception, or any other entity is shaped.
No technology, profession, perception, or entity will develop
influenced solely by a single social group. Social constructivist
theory is therefore useful for describing the social process of
any development, and by using social constructivist theories,
the researcher will be able to uncover difficulties when differ-
ent groups are required to collaborate.
29,30
The objective of this
study is to explore how the perception of 3 important stake-
holders, from a social constructivist perspective, influences the
role of the patient in drug development.
Patients and Methods
Theory
The social constructivist theory providing a theoretical founda-
tion for this study is titled the social construction of technology
theory (SCOT theory).
29
According to SCOT, there is no one
way of “doing things” (running a process, designing a technol-
ogy/an artifact, developing a professional role, etc). There is no
single view or truth, and knowledge is the result of perspectives
that can be regarded as valid in different situations.
31
SCOT has
been used to study a variety of issues, such as information
technologies in pharmacy, development of professional roles,
sustainable constructions, etc.
32,33
In this study, SCOT theory
is used for studying PIDD, a development presumably socially
constructed by different stakeholders.
The SCOT theory consists of 3 key concepts: relevant social
groups (RSGs), interpretative flexibility (IF) and technological
frames (TFs). An RSG represents a group of people involved in
the development of an artifact who associates the same set of
meanings with the specific artifact. An RSG can be identified
through a description of the problems attributed to the artifact.
The IF is the total meaning attributed to the artifact by all the
different RSGs, and the TF describes the shared frame of ref-
erence within a particular RSG, for which the group views a
specific artifact.
30
The development of an artifact may also be characterized in
terms of TFs and different degrees of inclusions in the frame.
Three different configurations guide the interactions
30
: (1) no
clearly dominant TF, (2) 1 dominant TF, and (3) more than 1
TF. When no single TF/RSG dominates, several different
inventions will exist side by side. Stabilization can occur when
an RSG tries to propagate its own solution by “recruiting” other
RSGs to support their perception of a given solution. The con-
figuration with 1 dominant TF guiding artifact development is
the most common one.
30
Choice of Unit
Semistructured interviews served as data collection method for
the study since interviews are suitable for gaining knowledge of
how people understand and perceive a social phenomenon situ-
ated and embedded in natural contexts.
34
The interview guide took its basis in the SCOT TF concepts.
The interview guide opens with questions exploring infor-
mants’ experience and definition of the concept “patient
involvement.” The following interview guide topics also relate
to the TF elements of “goal,” “design” and “key problems.”
The final topic “future scenarios” is inspired by the study of
Nørgaard et al, proposing that in the study of an ongoing tech-
nology/development etc, a minimum of prediction for the
future is only natural.
33
The interview guide was piloted on 2
subjects before conducting the interviews, and small changes
were undertaken because of the existence of leading or closed
questions. One of the subjects used for piloting was a pharmacy
master’s student knowledgeable on the theory and method. The
other subject used for piloting worked in the pharmaceutical
industry. Neither subject had specific experience with PIDD,
and results from the pilot interviews were not included in the
final analysis.
The SCOT theory advocates 2 methodologic techniques to
identify stakeholders for a specific development, namely, “roll
your snowball” and “follow the actors,” which were applied
here. Potential interviewees were identified through “follow
the actor” by looking at authors of scientific literature/com-
mentaries on the topic of PIDD, who represent one of the 3
stakeholders (industry, regulatory, or patient organizations).
For this, the participation list for a workshop held in Copenha-
gen in May 2015 on PIDD
1
was also consulted. Additional
2Therapeutic Innovation & Regulatory Science XX(X)
interviewees were identified through snowballing, asking inter-
viewees to refer to new potential interview subjects. Intervie-
wees were contacted through emails sent out in April-May
2016. A total of 14 individuals were invited to participate, of
which 12 accepted the invitation.
The Research Process
All interviews were performed in the spring of 2016. The first
author conducted the interviews. Ten interviews were con-
ducted face-to-face, and 2 interviews were conducted via
Skype. The interviews were conducted in either Danish or
English, depending on the preference of the interviewee. The
interviews lasted an average of 65 minutes (ranging from 40 to
90 minutes). All interviews were audio recorded and tran-
scribed. Interviews in Danish were not translated in their full
length; only relevant quotes were translated.
Research Ethics
No ethical approval is needed for noninterventional studies
carried out in Denmark. All participants were provided via e-
mail with information about the purpose of the study, protec-
tion of anonymity, and interviewee right to withdraw from the
study. Verbal consent was obtained prior to the interview.
Analysis
Data were analyzed using a systematic text condensation
(STC) approach.
35
STCisananalysisstrategythatshifts
between decontextualization and recontextualization of data.
The analysis was performed using a stepwise approach. The
interviews, a total of 140 pages of transcripts, were analyzed
in series, along with interviewing, to avoid losing overview of
data. The first author was in regular consultation with the
coauthors of the paper in this phase of the project. Finally,
all participants were invited to comment on the analysis. Only
one of the participants accepted the invitation and had no
comments to add.
Results
A total of 12 people were interviewed: 6 from patient organi-
zations, 4 from the pharmaceutical industry, and 2 from the
regulatory authorities (Table 1). Ten of the 12 interviewed
persons were Danish. All of the patient organizations repre-
sented are Denmark based and represent patients with diseases
with viable treatment options. The members of the pharmaceu-
tical industry represented in this study are all from larger phar-
maceutical companies.
The meaning attached to PIDD by the interviewees ranged
from “subjects in a clinical trial” to “equal players.” Two
industry interviewees distinguished between the term “patient
involvement” and the terms “patient engagement” and “patient
partnership,” with the latter 2 implying a closer degree of col-
laboration. However, the majority of the informants considered
PIDD to extend beyond being only the patient subject in a
clinical trial.
Involvement means, it can mean different things, but for me, it’s a,
it’s not commitment. ...Whereas a partnership is ...where you
have a shared objective, something that should help my research-
ers, something which has to help the chairman in his life in the role
of a chairman. [PI3]
Although there was a relatively large span in the meaning
attached to PIDD, the 3 stakeholder groups appeared to a high
degree to share one common TF, described through the 3 com-
ponents “goals,” “design methods and criteria” and “key prob-
lems (risks and barriers).” See the overview of the elements of
the common TF in Table 2.
Goals
“Creating better treatment solutions” was a goal for all 3 sta-
keholder groups. PIDD was seen as a way to understand what
constitutes a relevant drug for patients. Expressions such as an
“eye-opener for the pharmaceutical industry,” closing
Table 1. Characteristics of the 12 Interviewees.
ID
a
Organization Function Experience With PIDD
PO1 Patient organization Volunteer board member (patient) Yes
PO2 Patient organization Volunteer board member (patient) Yes
PO3 Patient organization Secretary employee (patient) No
PO4 Patient organization Patient—chairman (patient) No
PO5 Patient organization President Yes
PO6 Patient organization President No
PI1 Pharmaceutical industry Manager Yes
PI2 Pharmaceutical industry Senior management Yes
PI3 Pharmaceutical industry Senior management Yes
PI4 Pharmaceutical industry Senior management Yes
RA1 Regulatory authority Senior medical officer Yes
RA2 Regulatory authority Senior medical officer Yes
Abbreviation: PIDD, patient involvement in drug development.
a
The ID is used within the article to identify the origin of particular quotes.
Hansen et al 3
knowledge gap,” and “a more holistic focus” were used to
describe how PIDD could contribute to better treatment
solutions.
So I think that it can help to change the focus to what a good drug
actually is, besides the specific treatment effect. [PO1]
It is about bringing better solutions to them, and what does that
mean, it means solutions that are more relevant to them and have a
bigger impact on their lives and that come faster, so there are three
elements. [PI4]
The common goal, described above, was complimented by
the following 3 additional motivations: “improve the quality of
life”; “avoid business failure”; and “faster drug approval” for
patient organizations, industry, and regulatory agencies,
respectively. The total interpretative flexibility of the goals for
patient involvement is presented in Table 3.
The view that PIDD was a way to “improve quality of
life” was expressed by the patient organizations. Achieving
“improved quality of life” contained both a personal and an
altruistic element, the latter being very closely related to
the first goal of achieving better treatment solutions. On a
more personal level, improved quality of life could be
achieved merely by the feeling of being empowered.
Empowerment might be in the form of increased knowl-
edge and the feeling of contributing with knowledge to
new drug solutions.
Well this is also about new knowledge, it’s nice to be a part, if you
can help creating something that could provide a better treatment,
it could be nice with a treatment that makes life easier. [PO2]
The additional goal associated with PIDD for industry was
“avoid business failure.” Working directly with patients can
enable the medical industry to build further business strate-
gies, based on insights into true consumer needs rather than
bare assumptions about consumer needs. The wish to innovate
and evolve with the need of patients in mind and to avoid
business failure is a key driver for the pharmaceutical indus-
try. This was a general perception expressed by all industry
informants.
I would say that basically they [patients] should have as much
influence as we can possibly do, because I believe that if we can
cover the unmet needs of patients, then you will also generate a
lucrative business, it’s as simple as that. [PI2]
Finally, the possibility of fostering a “faster drug approval
process” was seen as an incentive for PIDD because it was
meant by one of the regulatory authority interviewees (RA2)
to benefit not only the pharmaceutical industry but also
patients. A company demonstrating end points based on patient
preferences will be in a much better position for a faster drug
approval process.
If you can demonstrate that you have made an analysis showing
that patients are interested in this and this, and that you have that as
a secondary or primary endpoint if you do that early you will get
your development programme on the right track ...you will also
have a better case, meaning that it will help you achieve the
approval faster. [RA2]
Design Methods and Criteria
Three elements emerged to describe the interpretative flexibil-
ity of criteria for PIDD (see Table 4). The themes
“transparency and trust,” “clear roles and expectations,” and
“patient education” constitute a convergent TF for all of the
stakeholders.
Table 2. The Common Technological Frame for All 3 Stakeholders.
Common Technological Frame
Goal
Design Methods and
Criteria
Key Problems (Risks
and Barriers)
Better treatment
for patients
Transparency
and trust
Clear roles and
expectations
Patient education
Conflict of
interest
Reputation
damage
Lack of
framework
Table 3. The Total Interpretative Flexibility of the Goal of Patient
Involvement in Drug Development for the 3 Stakeholders.
Stakeholders
Terms Used to Describe
Goals for Patient
Involvement in Drug
Development
Key Overall Goals for
Patient Involvement in
Drug Development
Patient
organization
Eye-opener for the
pharmaceutical
industry
Create better treatment
solutions
Patient-relevant
treatment solutions
Empowerment Improve quality of life
Influence
Improve quality of life
Pharmaceutical
industry
Understanding of true
consumer needs
Create better treatment
solutions
Better treatment
solutions
An eye-opener for the
pharmaceutical
industry
Identifying unmet
medical needs
Avoiding business failure
Create lucrative business
cases
Regulatory
authorities
Patient-relevant
treatment solutions
Create better treatment
solutions
Foster a better dialog Faster drug approval
processFaster approval
4Therapeutic Innovation & Regulatory Science XX(X)
Patient education was considered pivotal for PIDD, to pro-
vide patients with a better understanding of the drug develop-
ment process, to ensure that patients could represent a broader
perspective and not just their own personal view, and finally,
also to empower patients with a stronger voice in collaboration
with the pharmaceutical industry.
I think that in order to be a relevant partner, several things have to
be learned. You have to learn something about the process that you
are going to be a part of in order to make your suggestions more
relevant, but patient education is also about being able to go in and
represent others’ experiences and present a view that is wider than
your own experiences. [PO1]
All of the stakeholders found that “transparency and trust”
were important for PIDD. Transparency and trust were consid-
ered to go hand in hand, with transparency being a prerequisite
of trust. The focus for transparency was about being transparent
about how patient input would be used in the development and
decision processes.
So it all starts by making it transparent; the objective from both
parties it is like in a true partnership and then everything flows
from there, so the ideal for me is to start with that open transparent
discussion and saying, “Hey I am reaching out to you because what
I need is the following, what do you want from me, is there a place
where we can work together”—yes or no? And you both have the
freedom to say yes or no. [PI4]
The participants also agreed that effective PIDD required
“clarification of roles and expectations,” and having a trans-
parent dialog between patients and industry was deemed
necessary for having clarified roles and expectations. One facet
was the importance of understanding that experiential and sci-
entific knowledge exist side by side and of recognizing the
complementarity between those 2 as well as the limitation of
the 2 forms of knowledge. A number of informants (4) felt that
the pharmaceutical industry still lacks acknowledgement of
experiential knowledge as being valid knowledge.
I believe we must use all the competencies we have on this know-
how, we just need to acknowledge that there is also an experiential
patient knowledge which is valid and important knowledge on an
equal level as professional knowledge. [PO1]
Patient organization informants also expressed that to take
part in defining which role patients should play in drug devel-
opment, patient organizations have to take on a more active role.
So I think that we, as a patient organization must have the courage to
take initiative and invite the industry because then I think that there
is a greater chance that we are a part of setting the agenda. [PO1]
Risk and Barriers
A high degree of convergence toward one TF in the risks and
barriers associated with PIDD was found in the study. Three
potential risks and barriers were identified, consisting of
“conflict of interest,” “reputation damage,” and “lack of
framework.” See Table 5 for an overview of the total interpre-
tative flexibility of risks and barriers associated with PIDD.
Table 4. The Total Interpretative Flexibility for the Technological
Frame Element “Design Methods.”
Stakeholders
Terms Used to Describe
Key Design Features and
Criteria for Patient
Involvement in Drug
Development
Key Criteria for Patient
Involvement in Drug
Development
Patient
organizations
Feedback to patients
Ongoing collaboration
Win-win focus
Transparency and trust
Clear roles and
expectations
Patient education
Transparency and trust
Clear roles and
expectations
Patient education
Pharmaceutical
industry
Transparency and trust
Clear roles and
expectations
Patient education
Win-win focus
Transparency and trust
Clear roles and
expectations
Patient education
Regulatory
authorities
Transparency and trust
Clear roles and
expectations
Patient education
Transparency and trust
Clear roles and
expectations
Patient education
Table 5. Total Interpretative Flexibility of Risks and Barriers
Associated With Patient Involvement in Drug Development.
Stakeholders
Total Interpretative
Flexibility of Barriers
Associated With Patient
Involvement in Drug
Development
Total Interpretative
Flexibility of Risks
Associated With Patient
Involvement in Drug
Development
Patient
organizations
Lack of knowledge on the
patient side
Lack of resources
Lack of framework
Patient responsibility
Lack readiness
Conflict of interest
Reputation damage
Give drug “seal of
approval”
Pharmaceutical
industry
Culture change
Lack of framework
Pharma reputation
Tools to measure added
value of PIDD
Finding the right patients
Lack of readiness
Conflict of interest
Reputation damage
Base future strategy on
“wrong” patient input
Regulatory
authorities
Lack of knowledge on the
patient side
Lack of framework
Patient responsibility
Conflict of interest
Reputation damage
Abbreviation: PIDD, patient involvement in drug development.
Hansen et al 5
The risk of “conflict of interest” and “reputation damage”
made stakeholders hesitate to venture into a collaboration for
the purpose of involving patients in drug development. In par-
ticular, one patient organization informant was worried that
venturing into a collaboration with the pharmaceutical industry
for PIDD could potentially compromise their political judge-
ment, policies, and activities and possibilities to influence pol-
icy decisions.
We want to have political influence and we cannot have that if
we—forgive me—engage with the industry; the politicians natu-
rally would say that we are coming on behalf of a pharmaceutical
company, because we know that you have so many projects
together and you receive money from them or something along
those lines, then we would not be “clean,” so I do not see that this
would happen. [PO3]
Common to all of the stakeholders was a need for a common
framework to govern PIDD. Such a framework should also
involve both a methodological and a legal frame for PIDD,
including how to identify the “right” patients, how to collect
and interpret data, ethical considerations, legal requirements,
and responsibilities. In particular, the industry informants
found that regulatory authority involvement was needed to
develop such a framework for PIDD.
We do not have a formal system of how to work with patients, not
at all, how do we work with patients we do not know. I mean it is a
trial and error process I would say ...we need a regulatory envi-
ronment which can make a good framework. [PI1]
I think that the biggest problem right now is that we do not have
a framework, we lack statistical methods to analyse these data.
[RA2]
While the regulatory authorities also recognize a need for a
framework for PIDD, they did not believe that it was their
responsibility to develop such a framework.
In long term it may be that these three groups together must figure
out how to set up a framework. But it is in the early stage right now,
and we should not put up some rules that say “in regard to this drug
you need to involve patients,” if it is not relevant for that specific
drug. [RA1]
A patient organization informant expressed that the pharma-
ceutical industry currently initiates the collaborations and
invites patient organizations to generate ideas and give feed-
back on the design of devices, patient information material, etc.
This approach might leave the patient organization without any
responsibility. It was further stressed that this outreach raises
questions about the future of a collaboration between patient
organizations and the pharmaceutical industry.
We have been totally without any form of responsibility, ...it is
still our industry partner’s area we have entered, and it was their
decisions all alone. [PO1]
A few additional risks and barriers related to PIDD, not
common to all stakeholders, emerged in the study, such as lack
of readiness, lack of resources and lack of tools to measure
added value of PIDD. Lack of readiness related to the patient
organization being unaware of the possibilities of PIDD and
waiting until being invited by the pharmaceutical industry.
Patient organizations also expressed concerns about not having
the resources to venture into PIDD activities. For the pharma-
ceutical industry, lack of readiness related to cultural changes
required to accept the validity and relevance of patient input.
So, I do not believe that many patients think that it is obvious. I still
believe that this way of thinking, about using patients and listen to
patients’ knowledge and experience, is relatively new in this con-
text I believe. [PO3]
Discussion
This study showed how different stakeholders attach both dif-
ferent and similar meanings to PIDD. Nevertheless, one com-
mon TF could be identified among the 3 stakeholder groups.
All of the stakeholders saw the goal of PIDD as producing
better treatment solutions by “closing a knowledge gap.” The
common criteria for enabling PIDD were “transparency and
trust,” “clear roles and expectations,” and “patient education.”
Risks and barriers for the future development of PIDD were the
lack of an accepted framework and the risk of conflict of inter-
est and reputation damage, both from an industry perspective
and from a patient organization perspective.
Several of the identified TF elements can also be identified
in the literature. For instance, the motivation or goal for PIDD
was found to be very outcome oriented, which aligns with a
common view reflected in the literature.
1,3,10
This outcome
focus could pose a risk for the development of PIDD if it does
not prove to have any impact on providing better treatment
solutions. This concern was also voiced by one interviewee
from the industry. The expectation of the value of PIDD and
these implications for possible success PIDD is further illu-
strated in recent efforts to provide a model to assess the poten-
tial financial value of PIDD.
13
The criteria for enabling PIDD expressed in this study
(transparency and trust, clear roles and expectations, and
patient education) reiterate the findings from other studies.
15
Multiple voices advocate for patient education—to ensure that
patients have the necessary level of knowledge about the drug
development process—to be able to contribute to drug
development.
1,8,23,36
As described earlier, the constellation with one common TF
in the development of an artifact, in this case, PIDD, is the most
common constellation, which is seen in several empirical
SCOT theory-based studies.
30
The constellation here is that all
stakeholders interviewed in this study appear to be in high
agreement about the goal, enablers (“design methods and
criteria”), and risks and barriers of PIDD. However, the level
6Therapeutic Innovation & Regulatory Science XX(X)
of engagement/involvement appears to be different between
the stakeholders.
The current state of PIDD is characterized by a lack of clear
evidence of the value of PIDD.
13
Godin found that collabora-
tion on technology development may be built on the basis of
expectations and that the innovation process is dependent on
people’s prior ideas and motives.
37
This study also suggests
that what motivates the pharmaceutical industry and the patient
organizations to collaborate is grounded in their expectations of
the value of patient involvement.
Despite expecting that PIDD could help create better treat-
ment solutions for patients, patient organization interviewees
generally lacked awareness of PIDD. Our findings suggest that
the interviewed patient organizations can be considered a less
engaged group in the development of PIDD and might be
labeled an “unaware” and/or “quiet” group. Even patient orga-
nizations aware of the possibilities of PIDD awaited an invita-
tion by the pharmaceutical industry to collaborate and did not
demand to be part of the drug development process. A similar
pattern has been observed for public involvement in research.
25
Patient organizations may have the possibility of influencing
the future role of PIDD, but this possibility requires them to
take an active part in the process, as recognized by one of the
patient organization interviewees. These findings that some
patient organizations are “quiet/unaware” groups in the devel-
opment of PIDD should be seen in light of the fact that patient
organizations represented in this study have access to some
treatment options. It has been suggested that patients without
access to well-established treatment may be more likely to be
involved in drug development.
36
This may explain the lack of
awareness and the lack of incentives among the patient orga-
nizations. Other factors such as patient organizations’ lack of
resources or reservations to venture into a collaboration with
the pharmaceutical industry because of a concern that this
could affect their perceived independence may also contribute
to the patient organizations’ somewhat quiet position in the
development of PIDD. While patient organizations in this study
could be described as a less-engaged group in the TF, the
pharmaceutical industry interviewed in this study could be
described as a highly engaged group. Industry interviewees
viewed PIDD not only as a way to create better treatment for
patients but also as a means to create a better business case, and
PIDD was even expressed as a way to avoid business failure.
The industry interviewees also seemed to think that the phar-
maceutical industry had a high level of responsibility for the
development of PIDD and to invite patients to participate. This
suggests that industry could be labeled a “dominant” or a
“pioneer” group. All stakeholders interviewed considered the
lack of a framework to be a barrier of PIDD. This call for a
framework and recommendations to support PIDD is also
reported in other studies and comes from industry, regulators,
and patient organizations as well as from academia.
15,38
We
found that both the industry and patient organizations appear to
be looking toward the regulatory authorities to provide a legal
and methodological framework for PIDD. This may prove to be
somewhat of a paradox since the regulatory authorities consid-
ered their knowledge and experience with PIDD to be too pre-
mature to be able to, or even willing to, come up with a
framework that sets the boundaries for PIDD. Regulatory
authorities in our study might be labeled “hesitant.” This find-
ing may appear surprising considering efforts from regulatory
authorities inviting patients/patient representatives to provide
perspectives and advice for regulatory decision making.
6,39
These are, however, quite different indicatives than providing
legal and methodological frameworks for PIDD.
To understand the role patients may play in drug develop-
ment and how this will influence the outcome, it might be
worthwhile to consider the levels of lay/citizen participation
that have been laid out elsewhere.
40,41
Arnstein (1969) suggests
with her typology that the degree of decision-making power
defines the level of participation. Simply asking for patient
preferences without giving away any decision-making power,
which was the way many informants defined patient involve-
ment, might be labeled as participation by a “consultative”
character. At this level, the view presented by patients will not
necessarily have an impact on the outcome. Likewise, at this
level, participation is claimed to remain what Arnstein refers to
as a “window-dressing ritual.”
40
The extent to which patient
involvement truly is “involvement” and not merely tokenistic
in nature is a concern that has been shared.
14,26
However, the
results from the present study do not illustrate a general focus
on decision-making power as the aim of patient involvement.
The focus was rather on the role that patients can play in
closing the knowledge gap between patients and researchers
in the pharmaceutical industry. A “more holistic focus” and
an “eye-opener for the pharmaceutical industry” were phrases
used to describe what the patient organization informants saw
as the aim, which was a view that was supported by the phar-
maceutical industry and regulatory authority informants. This
result is congruent with findings from the existing literature,
illustrating that lay experts involved in health research see their
role as confirming or supporting the role of the researcher
rather than challenging or questioning the work.
42
To enhance the trustworthiness of this study, the credibility
of the informants was ensured by guaranteeing informants
anonymity and clearly informing them about their right to
withdraw from the study at any time.
43
Informants were also
provided with the opportunity to review and comment on the
final analysis. While the majority of the analysis was per-
formed by the first author, the stepwise analysis approach pro-
vided the opportunity to continuously discuss the results and
interpretation of the data analysis among coauthors.
A common critique of SCOT theory is that it fails to take
into account, for instance, power asymmetries between groups
by implicitly assuming that groups are equal and that all rele-
vant social groups are present in the development of an arti-
fact.
44
In the development of PIDD, power asymmetries are
apparent, and the findings in this study point to this phenom-
enon, namely, the lack of resources of smaller patient organi-
zations, the need for at minimum regulatory acceptance, and
Hansen et al 7
the apparent higher investment in development from the phar-
maceutical industry. Additional power imbalances may exist
between patients and industry related to resources and possibi-
lities to influence policy decisions. In addition, there may be a
risk that only the perception of the “strong” patient (or patient
organization) is heard, while perception of the weaker patient
groups might be absent in the PIDD development process.
The transferability (paralleling representativeness and gen-
eralizability in quantitative research) of the findings in this
study is strengthened by using empirical evidence accounting
for 3 key stakeholders perspective. While the study is con-
ducted with informants of primarily Danish nationality, most
of the informants function in European/global organizations in
their daily lives. In particular, the finding that patient organi-
zations appear to be a rather quiet/unaware group is of general
relevance. Efforts by industry to strengthen PIDD should be
careful to ensure that the patient is fully onboard or, as a min-
imum, aware that the support and enthusiasm about PIDD evi-
dent in some patient organizations
6,15-20
does not necessarily
extend to all patients and patient organizations. Future studies
may look into the following aspects: how widespread is invol-
vement in drug development among patient organizations,
which patient organizations have experience with and actively
pursue opportunities to take part in drug development, and
more importantly which do not.
Conclusion
A large span in the meaning attached to patient involvement in
drug development (PIDD) between interviewees was identified
in this study. This span range from considering PIDD to be
clinical trial participation to considering PIDD to be when
patient and sponsors are equal partners. Despite this finding,
all of the stakeholders emerged to belong to one common tech-
nological frame. All of the stakeholders perceived the goal of
PIDD as something producing better treatment solutions. The
common criteria for enabling PIDD were “transparency and
trust,” “clear roles and expectations,” and “patient education.”
Risks and barriers for the future development of PIDD were the
lack of an accepted framework and the risk of conflict of inter-
est and reputation damage. In turn, it was found that the phar-
maceutical industry, the patient organizations, and the
regulatory authorities could be labeled as “pioneer/dominant,”
“unaware/quiet,” and “hesitant,” respectively. The behavioral
descriptors may reflect a limited negotiation of the role patients
have in drug development today.
Declaration of Conflicting Interests
Christine E. Hallgreen is an associate professor in Regulatory Science
at the University of Copenhagen. In addition, she is also part of the
Copenhagen Centre of Regulatory Science (CORS), based at the same
university. The CORS is a cross-faculty university-anchored institu-
tion involving various public (Danish Medicines Agency, Copenhagen
University) and private stakeholders (Novo Nordisk, Lundbeck, Fer-
ring Pharmaceuticals, LEO Pharma) as well as patient organizations
(Rare Diseases Denmark). The center is purely devoted to the scien-
tific aspects of the regulatory field, with a patient-oriented focus, and
the research is not a company-specific product or directly company
related. The authors have no conflicts of interest to declare.
Funding
No financial support of the research, authorship, and/or publication of
this article was declared.
ORCID iD
Christine Erikstrup Hallgreen, PhD https://orcid.org/0000-0002-
7916-3915
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... Stakeholders expect EUPATI to be a relevant and high-quality training program and thereby to contribute to an increase in the pool of patient experts available for patient involvement activities. It is believed that in addition to their valuable experiences as a patient, substantial knowledge of terminology, and the complex process of development of medicinal products is necessary for patients to be equal partners in the dialogue with stakeholders (9)(10)(11). By focusing on both technical knowledge and relevant skills in general, not only will the quality of the outcomes of the engagement activity benefit (e.g., the patient's advice), but so will the engagement process as such. ...
... Combining the recommendations of several stakeholders in this regard, together with the available international and other guidelines on collaboration with the industry (5) will let us establish an internationally accepted modus operandi, customized for the Dutch situation. Trust, alignment of goals, balancing power, and open communication are among the underlying concepts to be considered (9,17,18). ...
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... Patient preferences can be used to inform the selection and assessment of unmet treatment needs, the treatment outcomes (benefits, risks) and uncertainties related to these outcomes (e.g., regarding their long-term duration and severity). However, approaches and the impact of incorporating patient preferences during medicines development and assessment is presently unsystematic, low and scattered across different phases of the lifecycle of medicines (Janssens et al., 2018;Hansen et al., 2019). Moreover, an array of challenges related to patient preference studies need to be further investigated, including: 1) the need for a systematic and robust preference study methodology; 2) the need for unbiased patient preference studies; 3) insights into how to deal with preference heterogeneity in studies; and 4) insights about whether preference studies need to be designed towards a single medicine or need to be product-"agnostic" (Medical Device Innovation Consortium, 2015; Utens C. et al., 2015;Ho et al., 2016). ...
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Industry, regulators, health technology assessment (HTA) bodies, and payers are exploring the use of patient preferences in their decision-making processes. In general, experience in conducting and assessing patient preference studies is limited. Here, we performed a systematic literature search and review to identify factors and situations influencing the value of patient preference studies, as well as applications throughout the medical product lifecyle. Factors and situations identified in 113 publications related to the organization, design, and conduct of studies, and to communication and use of results. Although current use of patient preferences is limited, we identified possible applications in discovery, clinical development, marketing authorization, HTA, and postmarketing phases.
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Plain English Summary Patients usually understand their disease and lifestyle needs better than many medical professionals. They also have important ideas about what research would be most beneficial to their lives, especially on how to manage symptoms in a way that improves daily quality of life. In the UK, the National Institute for Health Research has recognised the value of patient insight, and now requires researchers with public funding to involve patients and the public throughout the research process. There are many opportunities for involvement, but these generally focus on improving study design to ensure the trial is acceptable to participants. Some programmes work towards setting research priorities as important to patients, public members, and medical experts, but due to the complexity and cost involved in running clinical trials, the majority of research originates with the pharmaceutical industry or academic institutions. There is a clear mismatch between research ideas that patients prioritise (quality of life), and those actually investigated (drug development). The Patient Led Research Hub (PLRH) is a new initiative hosted by the Cambridge Clinical Trials Unit. The PLRH supports research ideas as proposed by patient organisations, providing resources and expertise in research design and delivery. The PLRH aims to co-produce any technically feasible project, regardless of disease or symptom focus. The proposing patient group maintains ownership of the project with an active role in study management. This method of research has proven to produce credible research studies that are of direct relevance to patients. Abstract Patient and Public Involvement has become an indispensable and expected component of healthcare research in the United Kingdom, largely driven by the National Institute of Health Research and other research funders. Opportunities for patients to become involved in research abound, and many organisations now have dedicated ‘public involvement’ teams. However, its value is often questioned amidst criticism of tokenism and the recognition that a mismatch persists between patient priorities and funded research. Although patients are frequently consulted, evidence that their involvement influences the research agenda remains limited. We propose a novel model that allows patients and the public not only to propose research questions, but to design, initiate and deliver their own research with all the necessary support from research professionals. We demonstrate the feasibility and utility of this approach in reporting the establishment, experiences and progress of the Patient Led Research Hub. Using this resource, patient organisations are now able to initiate and conduct rigorous clinical research unfettered by the constraints of academic or economic agendas.
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Plain english summary: People living with a condition are uniquely positioned to inform the understanding of the therapeutic context for drug development and evaluation. In 2012, the U.S. Food and Drug Administration (FDA) established the Patient-Focused Drug Development (PFDD) initiative to more systematically obtain the patient perspective on specific diseases and their currently available treatments. PFDD meetings are unique among FDA public meetings, with a format designed to engage patients and elicit their perspectives on two topic areas: (1) the most significant symptoms of their condition and the impact of the condition on daily life; and, (2) their current approaches to treatment. FDA has conducted 24 disease-specific PFDD meetings to date. The lessons learned from PFDD meetings range from experiences common across rare diseases to more disease specific experiences that matter most to patients. FDA recognizes that FDA-led PFDD meetings alone cannot address the gaps in information on the patient perspective. Patient-focused drug development is an ongoing effort and FDA looks forward to the next steps in advancing the science and the utilization of patient input throughout drug development and evaluation. Abstract: The U.S. Food and Drug Administration (FDA) has multiple mechanisms for its regulators and staff to interact with patients -- but none quite like its novel Patient-Focused Drug Development (PFDD) initiative. FDA established the PFDD initiative to more systematically obtain the patient perspective on specific diseases and their currently available treatments. Since the initiative's inception in 2012, FDA has held 24 PFDD meetings, covering a range of disease areas and hearing directly from thousands of patients and caregivers. FDA's PFDD meetings have also provided key stakeholders, including patient advocates, researchers, drug developers, healthcare providers, and other government officials, an opportunity to hear the patient's voice. The lessons learned include but are not limited to specific experiences that matter most to patients, patient perspectives on meaningful treatment benefits and how patients want to be engaged in the drug development process. FDA recognizes that FDA-led PFDD meetings alone cannot address the gaps in information on the patient perspective. Further enhancing the incorporation of the patient's voice in drug development and evaluation continues to be a priority for FDA.
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Objective: To identify the elements necessary for successful collaboration between patient groups and academic and industry sponsors of clinical trials, in order to develop recommendations for best practices for effective patient group engagement. Methods: In-depth interviews, informed by a previously reported survey, were conducted to identify the fundamentals of successful patient group engagement. Thirty-two respondents from 3 sectors participated: patient groups, academic researchers, and industry. The findings were presented to a multistakeholder group of experts in January 2015. The expert group came to consensus on a set of actionable recommendations for best practices for patient groups and research sponsors. Results: Interview respondents acknowledged that not all patient groups are created equal in terms of what they can contribute to a clinical trial. The most important elements for effective patient group engagement include establishing meaningful partnerships, demonstrating mutual benefits, and collaborating as partners from the planning stage forward. Although there is a growing appreciation by sponsors about the benefits of patient group engagement, there remains some resistance and some uncertainty about how best to engage. Barriers include mismatched expectations and a perception that patient groups lack scientific sophistication and that "wishful thinking" may cloud their recommendations. Conclusions: Patient groups are developing diverse skillsets and acquiring assets to leverage in order to become collaborators with industry and academia on clinical trials. Growing numbers of research sponsors across the clinical trials enterprise are recognizing the benefits of continuous and meaningful patient group engagement, but there are still mindsets to change, and stakeholders need further guidance on operationalizing a new model of clinical trial conduct.
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Background: While patient groups, regulators, and sponsors are increasingly considering engaging with patients in the design and conduct of clinical development programs, sponsors are often reluctant to go beyond pilot programs because of uncertainty in the return on investment. We developed an approach to estimate the financial value of patient engagement. Methods: Expected net present value (ENPV) is a common technique that integrates the key business drivers of cost, time, revenue, and risk into a summary metric for project strategy and portfolio decisions. We assessed the impact of patient engagement on ENPV for a typical oncology development program entering phase 2 or phase 3. Results: For a pre-phase 2 project, the cumulative impact of a patient engagement activity that avoids one protocol amendment and improves enrollment, adherence, and retention is an increase in net present value (NPV) of $62MM ($65MM for pre-phase 3) and an increase in ENPV of $35MM ($75MM for pre-phase 3). Compared with an investment of $100,000 in patient engagement, the NPV and ENPV increases can exceed 500-fold the investment. This ENPV increase is the equivalent of accelerating a pre-phase 2 product launch by 2½ years (1½ years for pre-phase 3). Conclusions: Risk-adjusted financial models can assess the impact of patient engagement. A combination of empirical data and subjective parameter estimates shows that engagement activities with the potential to avoid protocol amendments and/or improve enrollment, adherence, and retention may add considerable financial value. This approach can help sponsors assess patient engagement investment decisions.
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The heated controversy over “citizen participation,” “citizen control,” and “maximum feasible involvement of the poor,” has been waged largely in terms of exacerbated rhetoric and misleading euphemisms. To encourage a more enlightened dialogue, a typology of citizen participation is offered using examples from three federal social programs: urban renewal, anti-poverty, and Model Cities. The typology, which is designed to be provocative, is arranged in a ladder pattern with each rung corresponding to the extent of citizens’ power in determining the plan and/or program.
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To date, social media has been used predominantly by the pharmaceutical industry to market products and to gather feedback and comments on products from consumers, a process termed social listening. However, social media has only been used cautiously in the drug development cycle, mainly because of regulations, restrictions on engagement with patients, or a lack of guidelines for social media use from regulatory bodies. Despite this cautious approach, there is a clear drive, from both the industry and consumers, for increased patient participation in various stages of the drug development process. The authors use the example of HealthUnlocked, one of the world's largest health networks, to illustrate the potential applications of online health communities as a means of increasing patient involvement at various stages of the drug development process. Having identified the willingness of the user population to be involved in research, numerous ways to engage users on the platform have been identified and explored. This commentary describes some of these approaches and reports how online health networks that encourage people to share their experiences in managing their health can, in turn, enable rapid patient engagement for clinical research within the constraints of industry regulation.
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Purpose: Although there has been more involvement by patients in the drug-development process, there are not a lot of published data that quantify patient-centric activities or that document these activities across a large scale. In order to examine the patient-centricity landscape and to quantify the adoption and implementation of these initiatives, the Tufts Center for the Study of Drug Development and the Drug Information Association collaborated on a research study. The study examined patient-centric activities implemented by pharmaceutical, biotechnology, and contract research organizations, as well as activities being piloted or in the planning stages. Methods: A global industry survey was conducted across pharmaceutical, biotechnology, and contract research organizations, assessing 25 patient-centric activities within clinical research. Some of these initiatives involve the use of social media to engage with patients, or the use of social listening to monitor study activity. Initiatives being implemented, planned, or piloted in addition to those not being considered were evaluated by respondents. Twenty-two unique companies responded to the survey, representing a mix of large, mid-sized, and small organizations. Findings: The most widely adopted patient-centric initiatives, including activities both implemented and piloted across organizations, were patient advisory boards (17/22 companies), professional panels (16), lay-language clinical trial results summaries (13), assessment of the patient-organization landscape (10), and the use of home nursing networks (9). Implications: The results of the study suggest that organizations have a varied approach to the adoption and implementation of patient-centric initiatives, with more activities occurring in the planning stages than are being piloted or implemented. Many factors affect implementation and adoption, including buy-in by senior management, organizational vision, resources, and level of investment.
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A growing number of organizations—including pharmaceutical and biotechnology companies, foundations and associations—are routinely implementing patient advisory boards (PAB) given their high reported value for minimal relative investment. Organizations are typically implementing PABs to solicit patient voices and perspectives on a variety of areas such as protocol designs, clinical trial medicine kit designs, informed consent form designs, technology solutions, and patient communication materials. The Center for Information and Study on Clinical Research Participation (CISCRP) has planned, executed, and facilitated more than 30 PABs. In this article, the authors share lessons learned and best practices with regard to structure, format, and process for organizations wishing to adopt and implement PABs. The authors also provide metrics on the adoption and impact of PABs.
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Background: Obtaining assent from children participating in clinical trials acknowledges autonomy and developmental ability to contribute to the consent process. This critical step in pediatric drug development remains poorly understood, with significant room for improving the clarity, efficiency, and implementation of the assent process. Beyond ethical necessity of informing children about their treatment, the assent process provides the advantages of including children in discussions about their diagnosis and treatment-allowing greater understanding of interventions included in the study. A formalized assent process acknowledges the child as a volunteer and provides a forum for questions and feedback. Legal, cultural, and social differences have historically prevented the development of clear, concise, and accessible materials to ensure children understand the clinical trial design. Published guidelines on obtaining pediatric assent are vague, with many decisions left to local institutional review boards and ethics committees, underscoring the need for collaboratively designed standards. To address this need, 2 surveys were conducted to quantify perspectives on assent in pediatric clinical trials. Methods: Two digital surveys were circulated in the United States and internationally (October 2014 to January 2015). The first survey targeted children, parents, and/or caregivers. The second polled clinical trial professionals on their organizations' experience and policies regarding pediatric assent. Results: Forty-five respondents completed the child and parent/caregiver survey; 57 respondents completed the industry survey. Respondents from both surveys detailed experiences with clinical trials and the impediments to securing assent, offering potential solutions to attaining assent in pediatric patients. Conclusions: An important opportunity exists for standardized practices and tools to ensure pediatric patients make well-informed decisions regarding their participation in clinical trials, using materials appropriate to their level of understanding. These tools would establish a baseline standard for the assent process and be made available to researchers, improving their ability to secure assent from young patients.