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 inimproving the lives of cancer patients by raising awareness andproviding valuable tools and services. However, as theyincreasingly seek to influence the focus of oncology research, thedesign of clinical trials and the generation of RWD, their role isevolving. In the era of patient-centricity, pharma must understandhow best to partner with these groups to generate, access andleverage RWe that can be used to improve patient lives.

Hammit mistry, PHD Principal, RWE Solutions, IMS [email protected]

oncology advocates: evolving areas of interest

The traditional goals of patient advocates in oncology hasbeen to raise awareness, educate patients and support thoseimpacted by cancer. Many of the most prominent advocacygroups have also invested in healthcare advancement andpolicy shaping. Collectively, these advocates have animpressive history of success which has seen them evolveinto crucial stakeholders for pharma and healthcaredecision makers.

The evolution of oncology advocacy has also been driven bygreater professionalism, more accessible information andadvancements in technology that drives connectivity. At thesame time, the mindset of donors is changing, with greaterexpectations for social return on investment for their charitablecontributions.1 And finally, the ever-growing focus onpatient-centricity increases the importance of includingpatient input at each stage of their journey. Clinicians,regulators and pharmaceutical companies now regularlywork alongside oncology advocates to better serve patientneeds, supporting broader collaborations than typicallyseen in the past.

While these changes are not limited to the oncology space,the life altering impact of a cancer diagnosis and lack ofeffective 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-centricityIn recent years, pharma has taken a more holistic viewof its role in improving patient care. The days of simplymanufacturing and marketing a drug have beenreplaced by a patient-centric focus with emphasis onproviding holistic support. This stems from a morecomplete understanding of how a disease impactspatients and their caregivers, fully appreciating theirunmet needs and what they value, even if it cannot bemet by drug intervention. Companies are increasinglykeen to deliver innovative solutions (drug + servicecombinations) and personalized care that improvespatient outcomes at a cost which is acceptable toregulators and decision makers. The belief is that trulyimpactful solutions will be valued by patients, cliniciansand payers, as well as benefiting the companies thatoffer them.

increased patient input. For other tumors, advancements intargeted therapies and improved cancer survivorship areimportant catalysts. Overall, cancer patients want to bemore involved in the treatment and management of theirdisease, and share their experiences. They seek betterinformation, hand pick clinicians and centers based onreputation, 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 oncologyadvocacy groups are shifting focus to serve their members inbroader ways. As they do so, they enter a domain wherepharma companies, academics, researchers and economistsare increasingly active: RWD and RWE. Access to first-hand,anonymized patient medical records, genomics data and self-reported outcomes allows these groups to drive RWDcollection, influence RWE generation and develop credibleviewpoints that shape the way cancer is managed. This isapparent in four key areas

1. Actively influencing clinical trial design and drivingtrial recruitment Particularly in oncology, there is a growing advocacymovement built on concerns that firstly, patients aretreated as guinea pigs in today’s clinical trial processes;and secondly, that many trials do not offer benefit to thepatients involved.2 Examples include trials of drugs thatoffer limited potential improvement over standard ofcare or blinded studies where only some patients receivethe newer, potentially more effective treatments – acommon problem in rare cancers where currenttreatments are unsatisfactory.



Patient advocates are therefore seeking opportunities toprovide earlier input to study protocol design, shapingtrial endpoints to reflect what really matters to patientsand caregivers. The outcome is that better designed trialswhich show benefit to the entire study cohort are morerelevant for patients and this in turn facilitatesrecruitment. The challenge for clinicians and pharma is

to ensure that patient demands are realistic and withinthe boundaries of modern science. One way to managequality control is to work collaboratively with patients togather, evaluate and prioritize research questions thatare truly meaningful for them (Figure 2).

2. Moderating online communities and managing accessto patient networks A number of technology providers have built intelligentplatforms to host patient communities, often partneringwith patient advocacy groups to manage content, improve the quality of information and drive valuabledialogue amongst forum members. Two such providers,HealthUnlocked and Inspire, follow a similar model,inviting a well respected advocacy group to moderatecontent for their specific disease area (Figure 3).

The primary aim of these forums is to provideinformation, education and social support to members.However, the significance of an easily reachable patientcommunity is not lost on researchers and pharma, whosee great value in patient surveys, PROs and thepossibility to accelerate clinical trial recruitment.Importantly, the advocacy group moderators play a keyrole in managing access to their communities, ensuringthat any requests or push-messages are in the bestinterests of patients. These communities have seenmassive growth in the last five years and with this trendlikely to continue, the volume and richness of self-reported RWD available will also increase further.

3. Establishing prominent thought leaders who shapehealthcare priorities The call for patient input into healthcare policy andregulatory 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 refl ect 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-identifi ed 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 qualifi ed 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 topicsrecommending patient input to decision making – a rolethat leading oncology patient advocacy groups oftenassume.8 It is becoming increasingly common to seeprominent patient advocates addressing medical andpolicy congresses and taking up a seat on theirgovernance committees/boards.

The desire to listen has allowed many to broach topicssuch as access to medicines, funding for research anddrug risk/benefit evaluations. This has led to theirinvolvement in generating powerful evidence fromreliable RWD sources that expresses the patient point ofview (Figure 4). Partnering with academics andregulators to design, conduct and publish evidence-basedresearch is a trend that is likely to continue.

Some may argue that the evolving patient advocateagenda takes them away from their traditional focus.Politically active groups often hold a larger share ofvoice, enjoy greater publicity and capture a greaterportion of donations versus their more socially consciouscounterparts. Does this detract from the core philosophyof patient support and wellbeing? There is certainly roomfor both types of oncology patient advocate but finding away to work in harmony and place patient interests atthe forefront of activities is vital to ensure positiveadvocacy impact.

4. Building rich patient datasets that drive advancementin scientific researchStratifying patients by genetic mutation, biomarkers andhistology is becoming more mainstream in cancer, fuelingthe momentum to develop targeted therapies andimmunotherapies. A precursor to these clinical efforts is

enabling the collection and dissemination of rich,anonymous patient-level data which can be interrogatedby data scientists. For some of the main cancer types,clinicians and scientists (often sponsored by pharma)have successfully established registries that havesupported the development and launch of targetedtherapies. This is not an easy task in any disease area butis especially challenging in rare or niche cancers given thelow incidence, lack of physician experts and geographicspread of patients. It is here where many cancer patientadvocacy groups are becoming more active, acting as acentral body to collect and share clinical, genomic andPRO data.12

The value of this is already being recognized by

• The clinical community, given the complex nature ofrare cancers and the lack of published research, commonendpoints and stratification tools. Registries can provideinsight into baseline patient care pathways, which canact as comparative endpoints in trial design and removethe need for control groups. They also serve to supportpost-marketing authorization evidence generation,particularly in rarer cancers or niche patient subsetswhere drugs may reach the market with relatively fewpatients included in trials.

• Regulators, who increasingly expect robust data onpatient populations and subgroups. The FDA, forexample, recently issued guidance urging manufacturersto compile more comprehensive natural histories of raredisease populations.13 The objective was to understanddisease manifestations, patient subtypes and clinicalmarkers and to observe the complete disease journey.



As advocacy groups connect disease communities andcollate increasingly more sophisticated patient data(Figure 5), there are clear reasons for pharma to supportregistry building and incorporate related intelligencewithin their drug submissions.

In most cases, oncology advocates collaborate withinterested KOLs and clinicians to build registries,ensuring they hold robust clinical information to sitalongside any personal history provided by the patients.Without such rigor there is a danger that registries lackthe structure, completeness and accuracy to be of valueto the scientific community. As yet, there is no commonstandard for registry design, structure or management,making it harder for researchers when exploringpotential sources and evaluating data robustness.

Partnerships: The way forward

As pharma continues to embrace the value of partneringwith advocacy groups, there are emerging challenges thatmust be carefully navigated, particularly in relation to RWDand evidence generation. Most companies already profileand segment these groups to identify those with sharedgoals, solid reputations and collaborative attitudes toworking with clinical and industry partners. However, thereare some common areas of misalignment and related goodpractices that they should keep in mind.

• Striking a balance around RWD IP and ownershipWhile pharma may see patient data as an asset withcommercial value or IP that provides competitiveadvantage, advocacy groups take the opposite view.Believing that knowledge drives advancement, many feelthis anonymized data should be shared in the public (orat least scientific) domain to accelerate real-worldunderstanding and R&D productivity.13,18

Striking a balance is critical to any partnership. One option isto drive towards hybrid registries where the bulk of dataremains in the public domain but certain measures or datapoints are reserved for sponsoring companies.

• Safeguarding advocacy independencePart of the power of advocacy groups lies in theirindependence. As companies seek out areas forcollaboration they must be careful to maintain their ownpatient-centric reputation without damaging theintegrity of advocacy partners.

Typical good practice today is to work with a network ofpartners (other pharma industry sponsors, KOLs,academics/researchers, multiple advocacy groups) who share acommon goal. Here, with the resources, capabilities andknow-how to operate across all stakeholder groups, pharmatypically plays a key role in managing agreements, RFPs andworking 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 o� ered complete control in terms of how that information is shared

• PCAN sta� 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 profi ling

• They are also establishing a broader patient registry to enable further understanding of patient profi les, clinical trends and unmet needs

Scientifi c 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

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The richness of cancer RWD being generated and accessed with advocacysupport demands more open collaborative work to ultimately improve clinicaland experience outcomes

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• Ensuring trial endpoints of value to patientsWith the trend for stronger capture of patientperspectives during trial design, the likelihood ofmisalignment between clinical and quality of life (QoL)endpoints is very real.2,8

While clinically proving extension of life may be verymeaningful for manufacturers, patients may place moreemphasis on being able to carry out daily activities oravoid side-effects that impact their routine. A furtherchallenge for pharma is if patients see a trial as beingnot “fair”, for example if a particular cohort misses outon a high-potential investigational drug in order toprovide a control arm.2

Some general principles for pharma are to seek advocate inputearly in the trial design, test potential endpoints with a cross-section of patients and generate a patient-centric mix ofclinical and QoL measures to prove effectiveness. Patientadvocates can be a great sounding board for getting trials right.

• Addressing data quality/completeness issuesOn the surface, patient registries and patient-reportedinformation sources may seem the perfect solution tounderstanding a disease, especially when they report onthe number of patients involved/captured in datacollection. In reality, data quality and completeness arehighly variable, even when physicians are inputting datainto medical records. Missing data fields, incompletepatient histories and lack of real outcomes data arehugely detrimental for analysis. However, for advocacygroups this may be superfluous versus covering a largenumber of patient lives.

To help evaluate, cleanse and navigate the data chaos, it is worthemploying data scientists and research partners with experiencein these data types who can work closely with data owners toidentify solutions and help improve future data feeds.

• Being transparent on commercial interestsIn general, pharma/advocate partnerships run fairlysmoothly. However, there are three areas where viewsare likely to differ

1. Price of oncology drugs. This can be a major area ofdisagreement, even if both parties share the goal ofimproving patient access to new medicines. Onepotential route is to lower drug prices which couldobviously cause conflicts in the relationship.

2. Fair weather partners. There is an impression thatpharma is happy to partner with and sponsor advocateactivity while drugs are making money, but that theseactivities are the first to be cut if financials worsen ordrugs are at the end of patent exclusivity.

3. Survivorship care. This is a growing area of concernfor cancer advocacy groups and healthcare fundersgiven the improving life expectancy of many cancerpatients. The perception is that pharma will supportpatients while on their drug but forsake longer-termsurvivors who may need support as part of theirlifestyle and continue to drive healthcare resourceutilization.

Finding ways to partner with advocacy groups withoutjeopardizing the corporate position is an area forimprovement. One tried and tested approach is building trustthrough transparency and authenticity; being honest aboutwhere interests align with advocacy groups, where theypotentially misalign and what it is reasonable to expect froman industry partner.

Conclusion

A new paradigm is emerging where oncology advocacygroups with strong patient backing and fundraisingcapabilities are poised to transform into organizations thatsponsor drug research, engage pharma partners as peersand take control of RWD collection and evidence generation.

For pharma, this creates a need to better understand howoncology patient advocacy is evolving and how best topartner with groups on mutually beneficial projects.Organizationally, changes are already underway with“patient” roles becoming more common in the C-Suite andpatient input being gathered more routinely at key decisionpoints in the cancer drug lifecycle. However, more can bedone to embed good ways of working across the industryand to support oncology advocates in their game-changingactivities. The richness of cancer RWD being generated andaccessed with advocacy support demands more opencollaborative work with these groups to ultimately improveclinical and experience outcomes.


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For pharma, this creates a need to better understand how oncology patientadvocacy is evolving and how best to partner with groups on mutually beneficial projects

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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 advocateswithin 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 andspinal cord tumours at the top of the clinical research agenda, LauraMacdonald on behalf of the JLA Neuro-Oncology Group; InternationalBrain 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, 2015http://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 NetworkEurope, Bettina Ryll & Kristin Bryon. http://www.melanomapatientnetworkeu.org/press-release-mpne-2015.html

10 European Atlas of Access to Myeloma Treatment: a new approach toadvocacy; Ananda Plate.http://oncologypro.esmo.org/content/download/76233/1395454/file/2016-Patient-Advocate-Workshop-PLATE.pdf

11 European Atlas on Access to Myeloma Treatment, Myeloma PatientEurope. 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 CEOMultiple 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 AdvocacyGroups, Heather Gartman. https://globalgenes.org/raredaily/rarecast-tensions-brew-between-pharma-and-patient-advocacy-groups/

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