Advocacy and the cancer patient viewpoint

IMS HEALTH REAL-WORLD EVIDENCE SOLUTIONS
INSIGHTS PARTNERING IN RWE
Advocacy and the cancer
patient viewpoint
Patient advocacy groups have long played a critical role in
improving the lives of cancer patients by raising awareness and
providing valuable tools and services. However, as they
increasingly seek to inﬂuence the focus of oncology research, the
design of clinical trials and the generation of RWD, their role is
evolving. In the era of patient-centricity, pharma must understand
how best to partner with these groups to generate, access and
leverage RWe that can be used to improve patient lives.
Hammit mistry, PHD
Principal, RWE Solutions, IMS Health
Hmistry@uk.imshealth.com

oncology advocates: evolving areas of interest
The traditional goals of patient advocates in oncology has
been to raise awareness, educate patients and support those
impacted by cancer. Many of the most prominent advocacy
groups have also invested in healthcare advancement and
policy shaping. Collectively, these advocates have an
impressive history of success which has seen them evolve
into crucial stakeholders for pharma and healthcare
decision makers.
The evolution of oncology advocacy has also been driven by
greater professionalism, more accessible information and
advancements in technology that drives connectivity. At the
same time, the mindset of donors is changing, with greater
expectations for social return on investment for their charitable
contributions.1
And finally, the ever-growing focus on
patient-centricity increases the importance of including
patient input at each stage of their journey. Clinicians,
regulators and pharmaceutical companies now regularly
work alongside oncology advocates to better serve patient
needs, supporting broader collaborations than typically
seen in the past.
While these changes are not limited to the oncology space,
the life altering impact of a cancer diagnosis and lack of
effective treatments in some tumor types are drivers for
Advocacy engagement and RWD
EvolvingTraditional
Disease awareness
and patient/caregiver
education
Shaping scientiﬁc
research to accelerate
care improvement
Patient and
caregiver support
Online community
moderation and access
Building clinical
trial awareness
Clinical trial design
and patient recruitment
Campaigning for
access to medicines
Generating datasets
and evidence through
patient registries
Providing the
patient viewpoint to
healthcare stakeholders
Powerful thought
leadership
Fundraising Broader commercial
outlook
continued on next page
Figure 1: The changing focus of patient advocacy
in oncology
ACCESSPOINT • VOLUME 6 • ISSUE 12 PAGE 63
The era of patient-centricity
In recent years, pharma has taken a more holistic view
of its role in improving patient care. The days of simply
manufacturing and marketing a drug have been
replaced by a patient-centric focus with emphasis on
providing holistic support. This stems from a more
complete understanding of how a disease impacts
patients and their caregivers, fully appreciating their
unmet needs and what they value, even if it cannot be
met by drug intervention. Companies are increasingly
keen to deliver innovative solutions (drug + service
combinations) and personalized care that improves
patient outcomes at a cost which is acceptable to
regulators and decision makers. The belief is that truly
impactful solutions will be valued by patients, clinicians
and payers, as well as benefiting the companies that
offer them.
increased patient input. For other tumors, advancements in
targeted therapies and improved cancer survivorship are
important catalysts. Overall, cancer patients want to be
more involved in the treatment and management of their
disease, and share their experiences. They seek better
information, hand pick clinicians and centers based on
reputation, and connect with communities to tell their story.
So what does this mean for today’s oncology advocacy groups?
The RWe intersect
As shown in Figure 1, many of the more progressive oncology
advocacy groups are shifting focus to serve their members in
broader ways. As they do so, they enter a domain where
pharma companies, academics, researchers and economists
are increasingly active: RWD and RWE. Access to first-hand,
anonymized patient medical records, genomics data and
self-reported outcomes allows these groups to drive RWD
collection, influence RWE generation and develop credible
viewpoints that shape the way cancer is managed. This is
apparent in four key areas
1. Actively influencing clinical trial design and driving
trial recruitment
Particularly in oncology, there is a growing advocacy
movement built on concerns that firstly, patients are
treated as guinea pigs in today’s clinical trial processes;
and secondly, that many trials do not offer benefit to the
patients involved.2
Examples include trials of drugs that
offer limited potential improvement over standard of
care or blinded studies where only some patients receive
the newer, potentially more effective treatments – a
common problem in rare cancers where current
treatments are unsatisfactory.

Patient advocates are therefore seeking opportunities to
provide earlier input to study protocol design, shaping
trial endpoints to reflect what really matters to patients
and caregivers. The outcome is that better designed trials
which show benefit to the entire study cohort are more
relevant for patients and this in turn facilitates
recruitment. The challenge for clinicians and pharma is
to ensure that patient demands are realistic and within
the boundaries of modern science. One way to manage
quality control is to work collaboratively with patients to
gather, evaluate and prioritize research questions that
are truly meaningful for them (Figure 2).
2. Moderating online communities and managing access
to patient networks
A number of technology providers have built intelligent
platforms to host patient communities, often partnering
with patient advocacy groups to manage content,
improve the quality of information and drive valuable
dialogue amongst forum members. Two such providers,
HealthUnlocked and Inspire, follow a similar model,
inviting a well respected advocacy group to moderate
content for their specific disease area (Figure 3).
The primary aim of these forums is to provide
information, education and social support to members.
However, the significance of an easily reachable patient
community is not lost on researchers and pharma, who
see great value in patient surveys, PROs and the
possibility to accelerate clinical trial recruitment.
Importantly, the advocacy group moderators play a key
role in managing access to their communities, ensuring
that any requests or push-messages are in the best
interests of patients. These communities have seen
massive growth in the last five years and with this trend
likely to continue, the volume and richness of self-
reported RWD available will also increase further.
3. Establishing prominent thought leaders who shape
healthcare priorities
The call for patient input into healthcare policy and
regulatory decision making is loud and getting louder.
Figure 2: Examples of patient advocacy influencing trial design and recruitment 3–6
Figure 3: Example of a patient advocate moderating
online patient communities 7
Clinical Trial Priority Setting
• The James Lind Alliance established a partnership with carers,
clinicians and patient advocates (including The Brain Tumour
Charity, brainstrust, Brain Tumour Research, Children with
Cancer UK) to identify and prioritize clinical questions for
brain and spinal cord tumors
• 600+ potential research questions were gathered from ~200
patients and carers with the support of advocacy groups;
these were prioritized through a series of consultations
• 10 clinical research priorities were agreed that truly reﬂect
patient needs and quality of life (QoL)
The National Institute for Health Research and Cancer Research
UK are actively seeking researchers to initiate these studies, which
will eventually provide benchmark endpoints for drug research
and QoL measures
Trial Expansion & Recruitment
• Chordoma is a very rare form of bone cancer (1 in every million
people) with no current drug treatments, but mutation of the
brachyury protein is a known risk factor
• Chordoma Foundation (CF) invited a biopharmaceutical company
GlobeImmune to present on an investigational immunotherapy
agent that targets the brachyury protein in other cancers
• GlobeImmune decided to broaden its Phase 1 trial and
partnered with CF to successfully recruit chordoma patients
via the CF community within just a few weeks•
Moderated Discussion & Research
• The UK-based CLLSA set up an online community using the
HealthUnlocked platform with two moderators and more than
3,000 registered members
• Moderators can easily host surveys, collect input on key topics
and contact/screen members for clinical trial eligibility
• New platform features allow for closed groups that, for example,
collect PROs as part of a clinical trial, which can be de-identiﬁed
and linked to EMRs

Bettina Ryll
• Bettina Ryll is a patient advocate, founding member of the
Melanoma Patient Network EU (MPNE), and chair of the
European Society for Medical Oncology (ESMO) patient advocacy
working group
• A qualiﬁed doctor, she took up the cause for melanoma
patients when her husband was diagnosed with
advanced melanoma
• She has key interests in patient-focused clinical trials
and access to novel therapies
• MPNE has developed a melanoma risk-beneﬁt tool with
melanoma patient input
Myeloma Patients Europe
• MPE published a European Atlas of barriers and access to
myeloma treatments across 30 European countries to compare
variability in patient care
• They also collected input from patients and payers to
compare outlooks
• An action plan was then developed to support national advocacy
groups
• KOLs now use study results as a platform to improve patient options
Figure 4: Examples of advocacy involvement in generating patient-focused RWE 9–11
The FDA, EMA and NICE issue guidance on many topics
recommending patient input to decision making – a role
that leading oncology patient advocacy groups often
assume.8
It is becoming increasingly common to see
prominent patient advocates addressing medical and
policy congresses and taking up a seat on their
governance committees/boards.
The desire to listen has allowed many to broach topics
such as access to medicines, funding for research and
drug risk/benefit evaluations. This has led to their
involvement in generating powerful evidence from
reliable RWD sources that expresses the patient point of
view (Figure 4). Partnering with academics and
regulators to design, conduct and publish evidence-based
research is a trend that is likely to continue.
Some may argue that the evolving patient advocate
agenda takes them away from their traditional focus.
Politically active groups often hold a larger share of
voice, enjoy greater publicity and capture a greater
portion of donations versus their more socially conscious
counterparts. Does this detract from the core philosophy
of patient support and wellbeing? There is certainly room
for both types of oncology patient advocate but finding a
way to work in harmony and place patient interests at
the forefront of activities is vital to ensure positive
advocacy impact.
4. Building rich patient datasets that drive advancement
in scientific research
Stratifying patients by genetic mutation, biomarkers and
histology is becoming more mainstream in cancer, fueling
the momentum to develop targeted therapies and
immunotherapies. A precursor to these clinical efforts is
enabling the collection and dissemination of rich,
anonymous patient-level data which can be interrogated
by data scientists. For some of the main cancer types,
clinicians and scientists (often sponsored by pharma)
have successfully established registries that have
supported the development and launch of targeted
therapies. This is not an easy task in any disease area but
is especially challenging in rare or niche cancers given the
low incidence, lack of physician experts and geographic
spread of patients. It is here where many cancer patient
advocacy groups are becoming more active, acting as a
central body to collect and share clinical, genomic and
PRO data.12
The value of this is already being recognized by
• The clinical community, given the complex nature of
rare cancers and the lack of published research, common
endpoints and stratification tools. Registries can provide
insight into baseline patient care pathways, which can
act as comparative endpoints in trial design and remove
the need for control groups. They also serve to support
post-marketing authorization evidence generation,
particularly in rarer cancers or niche patient subsets
where drugs may reach the market with relatively few
patients included in trials.
• Regulators, who increasingly expect robust data on
patient populations and subgroups. The FDA, for
example, recently issued guidance urging manufacturers
to compile more comprehensive natural histories of rare
disease populations.13
The objective was to understand
disease manifestations, patient subtypes and clinical
markers and to observe the complete disease journey.

As advocacy groups connect disease communities and
collate increasingly more sophisticated patient data
(Figure 5), there are clear reasons for pharma to support
registry building and incorporate related intelligence
within their drug submissions.
In most cases, oncology advocates collaborate with
interested KOLs and clinicians to build registries,
ensuring they hold robust clinical information to sit
alongside any personal history provided by the patients.
Without such rigor there is a danger that registries lack
the structure, completeness and accuracy to be of value
to the scientific community. As yet, there is no common
standard for registry design, structure or management,
making it harder for researchers when exploring
potential sources and evaluating data robustness.
Partnerships: The way forward
As pharma continues to embrace the value of partnering
with advocacy groups, there are emerging challenges that
must be carefully navigated, particularly in relation to RWD
and evidence generation. Most companies already profile
and segment these groups to identify those with shared
goals, solid reputations and collaborative attitudes to
working with clinical and industry partners. However, there
are some common areas of misalignment and related good
practices that they should keep in mind.
• Striking a balance around RWD IP and ownership
While pharma may see patient data as an asset with
commercial value or IP that provides competitive
advantage, advocacy groups take the opposite view.
Believing that knowledge drives advancement, many feel
this anonymized data should be shared in the public (or
at least scientific) domain to accelerate real-world
understanding and R&D productivity.13,18
Striking a balance is critical to any partnership. One option is
to drive towards hybrid registries where the bulk of data
remains in the public domain but certain measures or data
points are reserved for sponsoring companies.
• Safeguarding advocacy independence
Part of the power of advocacy groups lies in their
independence. As companies seek out areas for
collaboration they must be careful to maintain their own
patient-centric reputation without damaging the
integrity of advocacy partners.
Typical good practice today is to work with a network of
partners (other pharma industry sponsors, KOLs,
academics/researchers, multiple advocacy groups) who share a
common goal. Here, with the resources, capabilities and
know-how to operate across all stakeholder groups, pharma
typically plays a key role in managing agreements, RFPs and
working with regulators to get projects off the ground.15
Registry
• The Pancreatic Cancer Action Network
(PCAN) is seeking patients to join its
registry and “know your tumor” program
• Joiners are asked to transcribe their
medical records into the online system
and are offered complete control in
terms of how that information is shared
• PCAN staff identify patients eligible to
donate tissue samples for genomic
profiling and gauge their interest
• Scientists submit study proposals to
access the database; the focus for
PCAN is to learn more about mutations
related to pancreatic cancer and its
potential therapies
Registry & Biobank
• The MMRF is building CoMMpass,
the richest clinical and genomic
dataset on multiple myeloma patients
anywhere; they recently enrolled their
1,000th patient
• The dataset will include a longitudinal
collection of bone marrow and blood
samples, clinical data, treatments and
responses, and QoL alongside extensive
molecular profiling
• They are also establishing a broader
patient registry to enable further
understanding of patient profiles,
clinical trends and unmet needs
Scientific Advancement
• MMRF has also built a ‘researcher
gateway’ to provide scientists and
clinicians with access to the rich
CoMMpass dataset and a ‘research
consortium’ to encourage collaboration
• The overarching aim is to accelerate
discovery of new targets, pathways and
biomarkers that will help to personalize
therapies for myeloma patients
Figure 5: Examples of advocacy involvement in building rich RWD datasets 14–17
“
”
The richness of cancer RWD being generated and accessed with advocacy
support demands more open collaborative work to ultimately improve clinical
and experience outcomes
“

• Ensuring trial endpoints of value to patients
With the trend for stronger capture of patient
perspectives during trial design, the likelihood of
misalignment between clinical and quality of life (QoL)
endpoints is very real.2,8
While clinically proving extension of life may be very
meaningful for manufacturers, patients may place more
emphasis on being able to carry out daily activities or
avoid side-effects that impact their routine. A further
challenge for pharma is if patients see a trial as being
not “fair”, for example if a particular cohort misses out
on a high-potential investigational drug in order to
provide a control arm.2
Some general principles for pharma are to seek advocate input
early in the trial design, test potential endpoints with a cross-
section of patients and generate a patient-centric mix of
clinical and QoL measures to prove effectiveness. Patient
advocates can be a great sounding board for getting trials right.
• Addressing data quality/completeness issues
On the surface, patient registries and patient-reported
information sources may seem the perfect solution to
understanding a disease, especially when they report on
the number of patients involved/captured in data
collection. In reality, data quality and completeness are
highly variable, even when physicians are inputting data
into medical records. Missing data fields, incomplete
patient histories and lack of real outcomes data are
hugely detrimental for analysis. However, for advocacy
groups this may be superfluous versus covering a large
number of patient lives.
To help evaluate, cleanse and navigate the data chaos, it is worth
employing data scientists and research partners with experience
in these data types who can work closely with data owners to
identify solutions and help improve future data feeds.
• Being transparent on commercial interests
In general, pharma/advocate partnerships run fairly
smoothly. However, there are three areas where views
are likely to differ
1. Price of oncology drugs. This can be a major area of
disagreement, even if both parties share the goal of
improving patient access to new medicines. One
potential route is to lower drug prices which could
obviously cause conflicts in the relationship.
2. Fair weather partners. There is an impression that
pharma is happy to partner with and sponsor advocate
activity while drugs are making money, but that these
activities are the first to be cut if financials worsen or
drugs are at the end of patent exclusivity.
3. Survivorship care. This is a growing area of concern
for cancer advocacy groups and healthcare funders
given the improving life expectancy of many cancer
patients. The perception is that pharma will support
patients while on their drug but forsake longer-term
survivors who may need support as part of their
lifestyle and continue to drive healthcare resource
utilization.
Finding ways to partner with advocacy groups without
jeopardizing the corporate position is an area for
improvement. One tried and tested approach is building trust
through transparency and authenticity; being honest about
where interests align with advocacy groups, where they
potentially misalign and what it is reasonable to expect from
an industry partner.
Conclusion
A new paradigm is emerging where oncology advocacy
groups with strong patient backing and fundraising
capabilities are poised to transform into organizations that
sponsor drug research, engage pharma partners as peers
and take control of RWD collection and evidence generation.
For pharma, this creates a need to better understand how
oncology patient advocacy is evolving and how best to
partner with groups on mutually beneficial projects.
Organizationally, changes are already underway with
“patient” roles becoming more common in the C-Suite and
patient input being gathered more routinely at key decision
points in the cancer drug lifecycle. However, more can be
done to embed good ways of working across the industry
and to support oncology advocates in their game-changing
activities. The richness of cancer RWD being generated and
accessed with advocacy support demands more open
collaborative work with these groups to ultimately improve
clinical and experience outcomes.
“
”
For pharma, this creates a need to better understand how oncology patient
advocacy is evolving and how best to partner with groups on mutually
beneficial projects
“

References
1
Return on Donations, A white paper on Charity Impact Measurement,
Lukas O Berg and Charlotte Månsson, 2011.
http://www.charitystar.org/wp-content/uploads/2011/05/Return_on
_donations_a_white_paper_on_charity_impact_measurement.pdf
2
Welcome to the revolution! The changing role of patient advocates
within research, Anna Wagstaff, 2015.
http://www.cancerworld.org/Articles/Issues/66/May-June-
2015/Patient-Voice/718/Welcome-to-the-revolution-The-changing-
role-of-patient-advocates-within-research.html
3
Welcome to the NCRI Clinical Studies Groups. http://csg.ncri.org.uk/
4
UK patients and healthcare professionals work together to put brain and
spinal cord tumours at the top of the clinical research agenda, Laura
Macdonald on behalf of the JLA Neuro-Oncology Group; International
Brain Tumour Alliance; World Edition 2015; 16: 41-43.
https://issuu.com/ibta-org/docs/ibta-2015
5
Chordoma Foundation. http://www.chordomafoundation.org/
6
The Man Working to Cure His Own Cancer, Aimee Swartz, 2015
http://www.theatlantic.com/health/archive/2014/03/the-man-
working-to-cure-his-own-cancer/284091/
7
CLL Support Association site on Health Unlocked.
https://healthunlocked.com/cllsupport
8
ESMO Workshop Cancer Patient Advocate Networks Driving Research,
2016. http://oncologypro.esmo.org/Meeting-Resources/ESMO-Patient-
Advocate-Workshop
9
The risk of NOT taking risks in Melanoma, Melanoma Patient Network
Europe, Bettina Ryll & Kristin Bryon. http://www.melanomapatientnetworkeu.org
/press-release-mpne-2015.html
10
European Atlas of Access to Myeloma Treatment: a new approach to
advocacy; Ananda Plate.
http://oncologypro.esmo.org/content/download/76233/1395454/file/201
6-Patient-Advocate-Workshop-PLATE.pdf
11
European Atlas on Access to Myeloma Treatment, Myeloma Patient
Europe. http://fadq.net/myeloma/map/
12
Advocacy Groups Raise Cancer Patient Awareness of Genomic Profiling,
Help Manage Testing Complexity, Turna Ray, 2015.
https://www.genomeweb.com/sequencing-technology/advocacy-
groups-raise-cancer-patient-awareness-genomic-profiling-help-
manage
13
Sharing the Mission to Conquer Rare Diseases; Heather Gartman;
inVentiv Health http://www.rarescience.org/wp-
content/uploads/2016/02/Sharing-the-Mission-to-Conquer-Rare-
Diseases.pdf
14
Welcome to the Pancreatic Cancer Action Network’s Patient Registry.
https://www.pancan.org/section-facing-pancreatic-cancer/patient-
registry/
15
MMRF ANNUAL REPORT 2014, Walter Capone President and CEO
Multiple Myeloma Research Foundation. http://www.themmrf.org/wp-
content/uploads/MMRF_D_Annual-Report-2014_FINAL.pdf
16
About MMRF. http://www.themmrf.org/about-mmrf
17
The MMRF CoMMpass study. http://www.themmrf.org/research-
partners/mmrf-data-bank/commpass-study/
18
RARECast: Tensions Brew Between Pharma and Patient Advocacy
Groups, Heather Gartman. https://globalgenes.org/raredaily/rarecast-
tensions-brew-between-pharma-and-patient-advocacy-groups/
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Advocacy and the cancer patient viewpoint

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