PRO white paper by andaman7

W H I T E P A P E R
Patient Reported
Outcomes (PRO) -
Challenges and
Potential Solutions
Whyandhowcanmedicaldeviceandpharmaceuticalcompanies,as
well as the entire healthcare sector, leverage patient engagement
with next-generation ePRO solutions?

PATIENTREPORTEDOUTCOMES(PRO)CHALLENGESANDPOTENTIALSOLUTIONS
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W H I T E P A P E R A N D A M A N 7
Introduction
Empowerment: the era of
the active, expert patient
Until recently, the vast majority of decisions related to care, health services
and medical research were made without patients being really involved or
even consulted. Added to this, in general, the industry, academia, healthcare
professionals, public authorities and patient associations have mostly tended
to act in isolation (1)
. However, this compartmentalised vision has led to
inefficiencies in terms of treatment processes and outcomes. While this is
sometimes still the case today, a new, more collaborative era is dawning. For
example, the role of the patient has expanded considerably over the past twenty
years. As a user of the healthcare system, not only have their rights been
recognised, but they have also been given an institutional role. At the same time,
we are witnessing the emergence of the expert patient, who takes an active role
in their own treatment, and who is a new partner for medical research, industry
and healthcare professionals. This progress has been made possible by the
development of the Internet, social networks, and mobile information sharing
technologies.

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Evolution of care
Moreover, the management of a serious illness, such as cancer, has significantly
changed. While today a life after cancer is increasingly possible, there are
many problems for survivors in terms of quality of life. “Progress in medical
research has made it possible to shift from a fatal illness to an illness, of which the
treatment allows for recovery or even chronicization, with treatments which are
increasingly administered over long periods of time,” observes Patricia Marino,
health economist at the Institute Paoli-Calmettes in Marseilles, France (2)
. “It has
therefore become clear that the effectiveness of treatments can/should no longer
only consider clinical parameters such as anti-tumour activity, relapse-free survival or
overall survival” she adds.
This evolution is taking place at a time when pharmaceutical innovation is also
under strain. Although research and development investments have increased
by nearly 50% within ten years, the number of new medicinal products approved
by organisations like the FDA has tended to stagnate over the past 20 years.
Advances in treatments, when they occur, produce benefits which are becoming
less and less obvious, even marginal. “It has therefore become essential today
to supplement the traditional assessments, which are based on purely clinical,
physiological and biological parameters, with other types of outcomes that can only be
assessed from the patient’s own perspective.” says Patricia Marino.
This is the concept of Patient Reported Outcome (PROs), which, after their
introduction in oncology, are becoming central to the assessment of treatment
strategies, medical innovation and medical research, which previously have been
dominated by biomarkers or purely clinical parameters. In recent years, some
companies have been able to integrate a new, reinvented form of collaboration
with patients and the valuable information they have at their disposal. The
expected benefits of such a partnership are numerous: development and
approval of new treatments and medical products, new indications for existing
treatments, healthcare personalisation, long-term cost-benefit analysis of an
innovative treatment, identification of unmet needs, better monitoring of side
effects, measurement of the clinical and economic impact of interventions within
the healthcare system with savings at stake, development of new guidelines and
decision support tools for clinical practice, etc.

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PROs: an overview
PROs are actually a modern version of what the famous clinician William Osler
had previously evoked: “If you listen carefully to the patient, they will give you the
diagnosis”! They highlight symptoms that make sense to patients and not just
those that make sense to physicians. Typically, patient reported outcomes are
defined as any information from a patient regarding their health condition which
has not been modified or interpreted by a physician or a third party. These results
may cover a series of facts or feelings considered important by the patient. These
may include, but are not limited to, the patient’s experience with the illness and
their care history, the impact or evolution of their symptoms, diet, quality of life,
satisfaction with the treatment, the consequences of the illness on their social,
professional and family life, etc.
In clinical trials, for example, a PRO can be used to measure the effect of a
medical intervention on one or more endpoints or symptoms. In general,
outcomes measured through a well-defined and reliable PRO in well-designed
studies can be used to support a claim for a medical product, if it is consistent
with the documented measurement capability of the device (3)
.

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Source: Pin Infographic

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PROs:
Use and Benefits
Patients: primary beneficiaries
The famous Alma-Ata Declaration of September 1978, organised and endorsed
by the WHO, formalised the role of patients as an essential stakeholder. This
Declaration reaffirmed the right and duty of individuals to participate both
individually and collectively in the programming and implementation of their
healthcare. This vision applies not only to the delivery of healthcare but also
to the research, development and marketing of medicinal products. As the
end users of any treatment, patients are, of course, the first to benefit from
an approach that now focuses on the patient and their own experience of the
illness. Indeed, this paves the way for more personalised treatments which
target patients’ individual needs, in terms of efficacy, side effects and quality of
life. It should also make it possible to meet treatment or care needs that are not
currently being met, due to the lack of ongoing dialogue and interaction between
patients, medical research and pharmaceutical companies (1)
. Finally, recent data
suggests that there are clinical benefits, when patients receive medical feedback
on the information they have shared.

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Side effect monitoring
This is certainly one of the areas of use of PROs which is of greatest interest
to researchers. This enthusiasm has been fueled by numerous studies which
have shown a discrepancy between the assessment of side effects observed by
doctors, on the one hand, and self-reported by patients on the other, particularly
in terms of the frequency and intensity of symptoms. In a study involving over
a thousand patients included in three clinical trials, for example, there was little
correlation between the assessments made by physicians and those made by the
patients for the six symptoms assessed (anorexia, nausea, vomiting, constipation,
diarrhoea and hair loss) with systematic underestimation by the physicians,
ranging from 40.7% for nausea to 74.4% for appetite loss. “The analysis of these
studies on physician/patient discrepancy shows that the physician tends to report
more (or even only) side effects that affect patients’ vital prognosis, such as anaemia
and fever, but neglects other side effects that can be a source of real discomfort and
suffering for patients.” says Patricia Marino (2)
.
However, this underestimation of the side effects of treatments poses serious
problems, because from a current regulatory perspective, the benefit/risk ratio of
cancer treatments is precisely based on the toxicity estimation. Comprehensive
information, including patient data, therefore becomes not only useful, but
Patient Satisfaction After Biceps Tenotomy - Scientific Figure on ResearchGate.

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essential both for patients and for the health authorities issuing authorisations
for the use of medicinal products. Yet, for the time being, toxicity is most often
established in clinical trials, based purely on statements made by physicians.
But things are changing in this area, too. Since 2008, in the United States, an
adapted version of the Common Terminology Criteria for Adverse Events (CTCAE)
has been developed to promote patients self-reporting their side effects in the
clinical trials in which they participate. More recently, recommendations have
also been published to establish a minimum number of symptoms to be collected
from patients in clinical oncology trials.
Improving the Quality of Care and Compliance
Taking into consideration the patient reported outcomes not only makes it
possible to improve the treatment toxicity estimation, but it should also lead
to improved quality of care and better compliance with treatment. In order
to better treat a symptom, you need to be aware of it. However, as some
research shows, the inaccurate estimation of certain symptoms often leads to
inadequate management of adverse events, particularly those related to pain
and depression. The use of PROs could therefore be beneficial to patients. This
idea is beginning to gain momentum. In 2009, the UK’s National Health Service
(NHS) introduced a systematic collection system for PROs for certain surgical
procedures in order to improve the service provided to patients.
Compliance is one of the other benefits expected to result from the use of
patient reported outcomes, particularly for those whose treatment is chronic
and for those who are mostly treated as outpatients. Studies, particularly those
conducted within the context of HIV treatment, show that failure to take into
account the side effects perceived by patients is a factor that encourages non-
compliance behaviour. “In the era of the development of oral treatments, patient
involvement is particularly central to this issue.” says Patricia Marino(2)
.
Prognostic Improvement Factor
More surprisingly, several studies now show that the use of PROs not only
influences the patient’s prognosis and survival, but it also reveals better
correlation with the patient’s health status than assessments made by
physicians. For example, in 2016 (4)
, a French remote monitoring application
featuring a questionnaire for patients treated for lung cancer made it possible
for patients to undergo four times fewer scans than others and to see their one-

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year survival increase from 49% to 75%, compared to standard monitoring (with
visits to the physician and regular scans).
PROs and public health insurance
organisations, a winning combination
Public health insurance organisations and private insurers are not the last to
show an increasing interest in patient reported outcomes. These organisations
increasingly require high quality and relevant outcomes to make decisions on
coverage and reimbursement. Today, in particular, health insurance stakeholders
expect real world evidence (RWE) to match the assessed performance when a
treatment is initiated. They also ask for new evidence to be made available later
and, most importantly, they want to be able to regularly evaluate the value for
money of any proposed treatment. All these arguments support the increased
use of patient reported outcomes and RWE, both in clinical and in post-
marketing research.
“The most interesting aspect of the questions asked by health insurance stakeholders
is that they generally deal with issues of comparative effectiveness and treatment
heterogeneity.” says David Thompson, Senior Vice-President of Syneos Health (5)
.
“Does one treatment work better than another, and which subgroups are most likely
to benefit or be adversely affected by it? Public health insurance organisations have
developed more sophisticated evidence requirements as we move towards more
personalised medicine. They want to see evidence of efficacy with active comparators
in various patients treated in real conditions. They want comparators which are
relevant to the populations they cover...”.
However, it should be noted that public health insurance organisations are still
exploring ways to include PROs in their decision-making. In Europe, there seem
to be significant differences in the way they are taken into account. Germany
appears to be the country where this data is best taken into account by health
insurance stakeholders, while France is the country where their impact is the
least significant (6)
.

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Improved Patient-Physician
Relationships
As we often tend to forget, the development of PRO tools does not solely benefit
the patient. It is also beneficial to the relationship they have with their physician.
There is scientific evidence that PROM/PREM questionnaires (measuring
experience and perceived health outcomes) improve communication between
caregivers and patients(7)
. They make it possible to address problems that would
not otherwise be addressed (e.g. the impact of an illness or treatment on the
patient’s sex life). This interaction allows the healthcare professional to identify
what is important to the patient and to choose with them the most appropriate
treatment, taking their point of view into account. “PROs can improve physician-
patient satisfaction, allow for meaningful dialogue, improved workflow and increased
physician satisfaction. They also help to bridge the gap between the clinical world and
patients. They facilitate conversations and increase interaction with the patient.” says
JFA Murphy in a commentary published in the Irish Medical Journal.

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Regulators: between promotion
and supervision
Regulatory authorities such as the Food and Drug Administration (FDA), the
European Medicines Agency (EMA) and the Pharmaceuticals and Medical Devices
Agency (PMDA) have been aware of the issues related to the sharing and use
of PROs or real-world data for some 20 years. They are well aware of the need
to promote the adoption of these new tools, particularly for the patient, who
remains the final beneficiary of this development. However, if regulators are now
encouraging the development and implementation of patient-centred outcome
assessment in clinical trials, it is not only to support the approval of medicinal
products and claims on the registration leaflet, but also to help them more
effectively evaluate the risks and benefits of new treatments.
In 2009, the US Food and Drug Administration
(FDA) issued a final guideline to industry on
the use of patient reported outcomes in the
development of medical products to support
package insert claims (PRO guideline). In 2014,
it also issued additional guidelines for eligible
devices that can be used over time in medicinal
product development programmes.
The European Medicines Agency (EMA) has
produced a discussion paper on the use of
Health-Related Quality of Life (HRQoL) measures. Moreover, an increasing
number of applications for marketing authorisations for new medicinal products
are providing PRO data to support claims. For example, DeMuro et al. reviewed
medicinal product approvals for the years 2006-2010. They showed that of
75 FDA and EMA-approved medicinal products, 35 (47%) had at least one PRO
claim approved by the EMA, compared to 14 (19%) for the FDA. The same study
concluded that the FDA was more likely to approve symptom reduction claims,
while the EMA approved more claims pertaining to function improvement or
HrQoL claims(8)
.

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Collecting PROs
Several hundred validated instruments for measuring PROs have been developed
to date. Others are regularly emerging as this field of research is rapidly evolving.
In addition, there are now specialised journals devoted to this subject, such as
Health and Quality of Life Outcomes, Quality Of Life Research, etc.
Two main types of measuring instruments are available: generic instruments
developed for use in a general population, and specific instruments adapted
to a particular problem (e.g. a specific pathology or population). “However,
these two types of measuring instruments are not mutually exclusive. They can
be combined depending on the objective pursued” says a report from the Haute
Autorité de Santé (HAS) [French Health Authority](9)
. Specific devices are
generally more sensitive than generic devices and are widely used in clinical
trials. In comparison, however, generic questionnaires, such as the World
Health Organization Quality of Life Scale (WHOQOL), “make it possible to assess
the relative weight of various patient groups, on different illnesses or in different
countries.”

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e-PROs: the added value
of digital tools
Historically, these measurements were carried out using traditional paper and
pencil questionnaires and at hospital sites. But the widespread use of laptops,
and even more so of tablets and smartphones, has led to the emergence of an
electronic version of PROs or e-PROs, the use of which is promising, particularly
in the form of virtual clinical trials (VCTs), which are now in the process of
changing pharmaceutical research practices, particularly phase 4 clinical trials.
Less cumbersome from an administrative point of view, faster, and therefore
less costly, these “in silico” studies make it possible to collect a much greater
amount of information which is often of better quality. They allow for real-
time monitoring, for example between site visits, to improve knowledge of the
medicinal product and to better assess its tolerability under the conditions of
use in daily life. They also facilitate patients’ access to clinical trials, promote
greater geographical diversity, including rural areas, as well as better diversity of
patient ethnicity and socio-economic status. Finally, within a context marked by a
significant drop in participation in clinical trials, CTVs make it possible to maximise
Source: FDA

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patient engagement and compliance in the clinical research process, by no longer
simply giving them the role of guinea pigs, but by giving them an active role in
their healthcare. The renowned Patient Centric vision has now become a priority
for all stakeholders involved in medical research, from pharmaceutical companies,
to healthcare professionals and patients themselves, to regulatory authorities and
public health insurance organisations.
It should also be noted that e-PROs also avoid data entry errors, provide
immediate access to data, trigger alerts/notifications, and reduce missing
information compared to paper-based results. They also make it easier for
patients to share sensitive content (10)
. As for the data obtained via e-PROs, it
also allows for the real-time monitoring of study compliance.
Simplified Consent
Another edge which digital technologies have over the “good old” paper method
is their ability to transmit daily patient feedback both easily and quickly. A wealth
of information which is increasingly needed by the pharmaceutical sector to meet
the growing requirements of health product assessment regulations. “Patient
consent is the key element in this regard. It is necessary for the collection and use of
information. However, digital technologies make it easier to organise this consent, in
the form of a dynamic e-consent, which allows the patient to give or withdraw consent
at any time. This undoubtedly makes it easier for companies
to engage in dialogue.” remarks Ingrid Maes, CEO and co-
founder of the Inovigate consulting firm(11)
.
In any event, research seems to confirm
the superiority of e-PROs over their paper
equivalent, at least for patients. A 2015 meta-
analysis shows that while the two methods
are considered equivalent by the authors,
e-PROs are widely (87%) supported by
participants (12)
, which encourages their
large-scale development.
the form of a dynamic e-consent, which allows the patient to give or withdraw consent
at any time. This undoubtedly makes it easier for companies
remarks Ingrid Maes, CEO and co-
(11)
.
the superiority of e-PROs over their paper
equivalent, at least for patients. A 2015 meta-
analysis shows that while the two methods
are considered equivalent by the authors,

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A long-awaited development
Given all of their advantages, there is no doubt that the use of PROs is constantly
increasing in clinical studies. This is what an analysis referenced in the official
ClinicalTrials.gov (United States) trial registry found in 2009. It noted that 14%
of interventional clinical trials conducted between 2004 and 2007 included
elements to measure patient reported outcomes. And to compare with the period
from 1980 to 1997, this figure was previously only 4.2% (13)
. However, although
the usefulness of PROs is widely recognised, their use remains fairly unequal.
Some experts even believe that they are underused in regard to many illnesses
that would benefit from increased attention to how patients feel or function
in relation to their illness. This is the case for chronic illnesses such as cancer,
cardiovascular diseases and diabetes.
The example of adult patients with congenital heart defects is revealing in this
regard. Their prevalence has done nothing but increase in recent years. About
80% of neonates and infants with coronary artery disease can expect to reach
adulthood, and this percentage is likely to increase further in the future due to
continuous improvement in surgical techniques and perioperative care. In the
United States alone, the number of these patients is estimated at three million.
“The concept of quality of life has recently emerged as an important measure of
outcomes related to coronary heart disease.” note the two authors of a recent article
published in the Journal of Thoracic Disease (14)
. “The fact that this feedback comes
directly from the patient, that they describe how they feel, makes this information
valuable.” However, the authors say that “this type of outcome measure remains
underused. While various studies have been published in several countries on the
quality of life of patients with coronary artery disease, most have several limitations,
ranging from the use of poor quality methods to inconsistent results.”

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Pharma sector:
the importance
of developing a
PRO strategy
The issues at stake
Patient reported outcomes (PROs) and other real-life data are being increasingly
used to conduct clinical trials prior to or after the marketing of new treatments
(medicinal products, medical devices and procedures), in order to verify their
cost/benefit ratio or efficacy. The development and consideration of these tools
has thus become a declared objective of regulatory bodies. In the United States,
for example, the FDA has encouraged, for several years, the development
and implementation of patient-centred Accountable Care Organization (ACO)
assessments in clinical trials to support medicinal product approvals and
efficacy claims (15)
. The development of Patient-Focused Drug Development
(PFDD) is also part of the FDA’s commitments as part of the PDUFA V*.
* See the Prescription Drug User Fee Act, enacted in 2012

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More generally, the life sciences market continues to evolve and health insurance
companies and patients play an increasingly important role. Today, companies
are therefore required to provide evidence and demonstrate the value of their
products to multiple stakeholders. Therefore, they must now also rely on this
type of evidence, namely PROs and RWE, to develop new compounds and
products, to cope with the increasing cost of R&D and increasingly demanding
reimbursement procedures.
This is notably included in the conclusion of Deloitte, who produced a White
Paper on the issue as well as a study on the use of RWE in 15 pharmaceutical
companies (16)
. “The use of Real World Evidence (RWE) allows companies to respond
to these trends. It offers opportunities to discover new compounds, prioritise product
development, support decision-making in terms of compound development, more
efficient and judicious clinical trials, and increase the return on R&D investments.
This data also helps to build new business models and provide unique and mutually
beneficial partnerships between life sciences companies and health systems,”
say Deloitte.
Market access and release
to market indications
The development of a PRO and RWE strategy is also becoming increasingly
important for such crucial topics as release to market indications and
market access, for which more and more evidence of clinical efficacy is
required before and after marketing. “Market access is no longer simply about
obtaining authorisation for the product’s release to market.” notes Chitra Lele,
Scientific Director at Sciformix Corporation (17)
. “Its scope includes pricing and
reimbursement, health technology assessment (HTA) and licensing. Today, public
health insurance organisations expect the actual evidence to match the projected
performance at the time of launching, as well as expecting new evidence to become
available as the competitive landscape and environment change. Most importantly,
they evaluate the value for money of the product.” It is therefore understandably
clear that PROs and real world evidence (RWE) can provide significant support
when it comes to, for example, supporting prices after the launch of a product
or promoting its marketing.

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The same applies to the claims contained in the package insert, which constitute
the part of the indications of a medicinal product that manufacturers may legally
use to promote their products and which, as such, may be considered of primary
importance for manufacturers seeking to distinguish their products on the
market. “Patient reported outcomes can be a differentiating factor for new medicinal
products released to the market” confirm Anke Van Engen and Mary New, senior
strategy experts at IQVIA (6)
.
Facilitating innovation, clinical research and post-marketing
studies
The collection and use of patient reported outcomes and real world evidence
(RWE) will undoubtedly increasingly complement the results obtained by
conventional randomized controlled trials (RCTs), both before and after
enrollment. For the pharmaceutical industry, this development heralds many
opportunities and potential progress. “The use of this data will help to achieve
several objectives.” confirms Ingrid Maes, Executive Director and co-founder
of the Inovigate consulting firm.(11)
“For example, it will be possible to set up a
real “Disease Management” i.e. a treatment which is more adapted to the patient,
which is personalised. Real-life evidence can also help to increase the efficacy of a
treatment or be used to monitor its efficacy over time, which will help to meet the

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growing requirements for Outcomes-based Payment.” Ingrid Maes further explains.
“Outcomes management is another possible application of RWEs, as well as the
development of new products and solutions based on the information they collect.
This is the case, for example, for populations who are not currently receiving optimal
treatment. In this area, the feedback that such data will generate is likely to feed
innovation.”
PROs and RWE: factors for commercial success
“Access to this data is becoming essential for companies who are active in the life
sciences sector.”, adds Ingrid Maes. “The race for outcomes will be the new challenge
for pharmaceutical companies. Access to patient data and the opportunity to optimise
treatment will lead to better outcomes. Data will therefore become a key factor in
commercial success. Companies that develop a strategy for this early enough will
dominate the market!”
The rise of PROs in post-marketing studies
Driven by a sustained demand for treatments that are more affordable and
better adapted to patients’ needs, post-marketing research should also be
able to take full advantage of the developments brought about by PROs,
particularly ePROs. Indeed, real-world evidence (RWE) and patient-reported
outcomes (PRO) are now increasingly complementing the data collected through
traditional randomised clinical trials. As a result, real-world data, including
observational studies, pragmatic trials and health management programmes,
are expected to play an increasingly significant role in decision-making. These
studies provide valuable sources of information that can be used to develop
a more accurate picture of the clinical benefits of a medicinal product. In
addition, thanks to technological advances, the latest digital solutions are
helping to improve the efficacy and cost-effectiveness of data entry compared
to traditional paper-based methods. “One of the great hopes for post-market
research is the increased adoption of advanced digital technologies, such as mobile
phone applications and mobile devices.” point out Gary Coward and Gavin Birchnall
(18)
. However, in the future, clinical research services will have to adapt to this
evolution of post-marketing research. For example, it becomes imperative to
ensure that the outcomes of the study population are relevant to the treated
population, as well as to consider the study structure and the strategies used
for patient and site recruitment.

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Broadly speaking, patient-centred treatment development initiatives have
several advantages:
› They can be used as a decision-making aid for medicinal product and medical
device development programmes.
› They enable the identification of specific areas where the needs of some
patient populations are not being met.
› They help determine the outcome measures that can be developed for
clinical trials.

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Challenges
and obstacles
As we have already pointed out, the potential of PROs and RWEs may be
significant, but their development is not yet equal to the hopes they raise.
Experts believe that some obstacles still exist in regard to their generalization in
clinical research. “These are mainly technical barriers, such as problems of connection
and interfacing between different systems, legal constraints related to consent, and
the ease with which data is accessible and reusable. For it to be exploitable, it must
be organised in a practical way.” says Inovigate’s General Manager(11)
. “None of
these obstacles are obviously insurmountable. More and more systems are able to
communicate with each other and make it possible to cross-reference data from
different sources, making it all the more essential for consent management to be
controlled by the patient. This is the case with some platforms, such as Andaman7,
which now include a “consent management” component, and thus allow for better
management of consent.”
For some other stakeholders, the barriers are not so much technical as they
are organisational. “Concretely: who gets the alerts, including on weekends? Who
coordinates the patient’s care process from home to hospital or doctor’s visits? How
can we ensure that the data collected by one organisation can be used by another?”
explains Dr Pierre Heudel, oncologist and medical coordinator of the myCLB
project, the information and liaison portal for patients at the Centre Léon Bérard
in Lyon (19)
.

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Some obstacles also persist at other levels. For example, many illnesses and
situations do not yet benefit from specifically developed and validated PRO
instruments. It should also be noted that developing PRO tools can be time-
consuming and resource-intensive, particularly to ensure that they are adapted
to requirements, i.e. well-defined and reliable (15)
.
In particular, the use of patient reported outcomes and real-life data to support
claims on the product registration package insert is evolving more slowly than
might be desired. However, we know that these are a key source of information
for both prescribing parties and patients. In this regard, it could have been
expected that the guidelines published by the FDA and others would have given a
major boost to the use of this data in medicinal product development and post-
marketing. However, this does not seem to be the case yet. “The interpretation
and implementation of these guidelines have posed significant challenges, arising
from various structural, methodological, logistical and communication issues.” said
experts from the Brookings Institute Engelberg Center for Health Care Reform.
“Overcoming these barriers will require the continued support and collaboration of all
relevant stakeholders, including medicinal product developers, researchers, patients,
PRO device developers and regulatory authorities.” they conclude (13)
.
However, it should be noted that such collaborations are beginning to emerge.
Since 2012, the International Consortium for Health Outcomes Measurement
(ICHOM) has been bringing together experts and stakeholders from around the
world (patients, physicians, researchers) to define global standards for measuring
real-life outcomes, by focusing on the criteria that matter most to patients. At
present, the standards for measuring the quality of care developed by ICHOM
cover around 50% of all pathologies and enjoy a well-deserved aura of credibility
and legitimacy. It should be added that they are available in “open source” format
to promote their dissemination (20)
.

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Andaman7:
A next-generation
ePRO solution
It is by personally encountering both cancer and the consequences of this illness
that Vincent Keunen, founder of Andaman7, had the idea of creating a mobile
application that also functions as a secure medical data exchange platform.
Until 2007, everything seemed to be going well for Vincent Keunen and Manex,
his software development company. But that year, everything changed. Three
months after being diagnosed with leukaemia himself, his 10-year-old son,
Pierre, was diagnosed with bone cancer. While Vincent was fortunate enough to
benefit from a targeted therapy that provided lifelong control for development
of his illness, Pierre had to undergo intensive chemotherapy, a bone marrow
transplant and eventually had a leg amputated. However, both Vincent and
Pierre are fortunate to be in good health today, thanks to the tremendous
advances of modern medicine. This difficult ordeal enabled Vincent Keunen to
see the shortcomings when it comes to patients and physicians having access,
everywhere and all the time, to essential medical information. Not only are
these shortcomings found in several fields, but access to the essential medical
information, when it is at all possible, is often difficult. This results in many
unnecessary medical examinations and a waste of time and money for all
parties involved.

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A solution designed
by patients for patients
It is with this in mind that Andaman7 has been launched in 2014, with the
strong idea of significantly improving patient autonomy and communication
between patients and physicians, to ultimately contribute to more effective and
personalised healthcare and medical research. Free for patients, easy and flexible
to use, this mobile application allows patients to collect medical information
about themselves and their illness to build their own portable medical records.
All kinds of information and documents can be gathered from doctors, hospitals,
the patient themselves or from connected devices. The originality of Andaman7
is that it explicitly allows the user to share medical information within their “circle
of trust” relatives (parents, children, etc.), healthcare professionals or hospitals,
thanks to a secure peer-to-peer system, with no storage on the Cloud, and GDPR
compliance by definition. Therefore there is no risk that this data will be used
for processing without the explicit consent of the user. The privacy of individuals
is also respected in the sense that it is the patient, and only the patient who
decides what is done with their health data.
Today, Andaman7 has more than 20,000 registered users, the solution
is available in over twenty languages, the company has twelve full-time
employees and a technological investment equivalent to more than thirty
working years.
A secure medical data
exchange platform
One of the most important aspects of Andaman7 is that it can be used to meet
the needs of many stakeholders, not just the patient alone. For example, this
solution offers a response to the problems faced by medical and pharmaceutical
companies today, namely the increasing cost of research, the increasing
requirements for outcome-based payment, the weight of imposed regulations
difficulties, and patient enrollment for clinical trials and collecting data about
them. Organised in the form of a secure medical data exchange platform, and
thanks to simplified use of e-consent, Andaman7 allows for real interaction

25
between patients and the various healthcare stakeholders (hospitals, physicians,
researchers, etc.). All types of data can be exchanged with a guarantee of
reliability, traceability and almost total interoperability: connected devices, ePRO
questionnaires, photos, genetic analysis or test results, hospital data, etc. As an
integrated solution, this platform is pertinent for all stages of the development of
a compound or a health device.
A typical ePRO journey
with Andaman7
Patient screening
A cohort is defined on the basis of the inclusion and exclusion criteria defined by
the study sponsor.
Recruitment
Physicians inform selected patients of the objectives of the study and the
benefits they may derive from it. The email address of recruited patients is
recorded via a web page and thus communicated to Andaman7.
Registration process
Andaman7 sends an invitation to the patient via email. The patient downloads
the application to their smartphone or tablet and confirms their registration.
Andaman7 provides the specific service under study in a dedicated space which
is easily identifiable in the application. In the case where the hospital’s Electronic
Medical Record is interconnected with Andaman7, the archives are sent and
integrated into the patient’s mobile application.
E-consent
The patient is asked to sign the electronic consent form. The patient’s circle of
trust extends to the hospital and the study sponsor (or CRO). Data relevant to the
study can be shared, directly or indirectly, according to the determined protocol
and integrated into the Electronic Data Capture (EDC) system.

26
ePRO
The patient can be notified on their smartphone of the arrival of an ePRO
questionnaire, or they can complete a questionnaire spontaneously in the event
of a crisis or the appearance of symptoms related to their illness. A wide variety
of data can be included (pain scale, photos, connected object data, etc.).
Data collection
Once the questionnaire is completed, the data is sent to the hospital or
pseudonymised to be sent to the study sponsor (or CRO). In addition to this, data
can always be tracked and identified.
Feedback
Patients have the opportunity to receive feedback from physicians and
researchers.

27
Use case ePRO: Rheumatoid Arthritis
(Virtual Study)
Thanks to Andaman7, a certain number of symptoms can be monitored very
precisely through various types and sources of data collected: pain, temperature,
stiffness (via questionnaires), patient mobility, physical activity (activity sensors
or pedometer), weight, sleep quality (sensors, connected devices), dermatological
problems (photos taken by the camera integrated into the smartphone), biological
parameters (lab tests), joint swelling, bone deformities (medical imaging), etc.
Other potential or actual use cases
Quality of Life (QoL) studies:
Andaman7 Collaboration - EORTC. The project aims to study the quality of life of
breast cancer survivors and involves sending questionnaires in 10 languages, in
12 countries and pseudonymising the data collected.

28
Disease management
Follow-up study of diabetic patients. Collection of the following information:
blood glucose level via connected glucometer, insulin injection recording, physical
activity measurement, weight evolution, nutrition data capture, etc. The data are
available in near real time to the diabetologist, which allows close medical follow-
up of the patients, and the possibility of advancing the periodic consultation in
case of worsening symptoms or biological parameters.
Continuity of Care
This project is part of a collaboration between Andaman7, the OSCARE aftercare
and research centre on burns and scars of the University Hospital of Antwerp,
and Awell Health for the management of the care pathways. Nurses plan events
and provide remote patient follow-up and review red flags. Direct interaction
with patients through forms and feedback from the patient is done through
Andaman7.
Similar parameters are available for maternity, orthopaedics, cardiology,
gastroenterology, etc.

29
Sources
(1) Travailler de concert avec les groupes de patients [Working in synergy with patient
groups]. Groupe de réflexion sur les patients de l’EFPIA [EFPIA Patient Focus Group],
2017. https://efpia.eu
(2) Rôle et utilisation des patients reported outcomes en oncologie [Role and use of
patients reported outcomes in oncology]. Patricia Marino.
www.lesdebatspublicsdelipc.com
(3) Guidance for Industry. Patient-Reported Outcome Measures: Use in Medical
Product Development to Support Labelling Claims. FDA, 2009. www.fda.gov
(4) Stéphany Gardier. Cancer du poumon : une application améliore le suivi des malades
[Lung cancer: an application improves patient follow-up]. www.pourquoidocteur.fr
(5) Why You Should Care About Real-World Evidence (A Lot). www.clinicalleader.com
(6) Anke Van Engen, Mary New. PROving its worth: how to develop a PRO strategy to
distinguish your product with regulators and payers (Europe). www.iqvia.com
(7) Qu’avez-vous pensé de votre séjour dans notre hôpital ? [What did you think of your
stay in our hospital]? https://kce.fgov.be
(8) Patient-reported outcome. https://en.wikipedia.org
(9) Les études post-inscription sur les technologies de santé (médicaments, dispositifs
médicaux et actes) [Post-registration studies on health technologies (medicinal
products, medical devices and procedures)]. Principles and methods. HAS, 2011.
www.has-sante.fr
(10) Prasanna R. Deshpande, Surulivel Rajan, B. Lakshmi Sudeepthi, C. P. Abdul Nazir.
Patient-reported outcomes: A new era in clinical research. www.ncbi.nlm.nih.gov
(11) Ingrid Maes, Inovigate: le RWE va changer les études cliniques [RWE will change
clinical studies]. www.andaman7.com
(12) Campbell N, Ali F, Finlay AY, Salek SS. Equivalence of electronic and paper-based
patient-reported outcome measures. www.ncbi.nlm.nih.gov
(13) Enhancing the Development and Use of Patient-Reported Outcomes in Drug
Development. www.brookings.edu
(14) Sameh M. Said, Joseph A. Dearani. Quality of life: an underutilized patient-reported
outcome for adults with congenital heart disease. www.ncbi.nlm.nih.gov
(15) Michelle Campbell, Measuring How Patients Feel and Function. www.fda.gov
(16) Getting real with real-world evidence. Deloitte’s Real-World Evidence Benchmark
Survey shows life sciences companies have room for improvement.
www.deloitte.com
(17) Chitra Lele, The Growing Importance of Real World Data. www.pharmexec.com
(18) Gary Coward, Gavin Birchnall. The changing face of post-marketing research.
www.pharmatimes.com
(19) David Bême, Cancer: le patient acteur de sa prise en charge améliore son pronostic
[Cancer: the patient as an actor in their management improves their prognosis].
www.doctissimo.fr
(20) Meeting with Dr Christina Akerman, President of ICHOM, International Consortium
for Health Outcomes Measurement. https://nextep-health.com

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