FT3 is unique in having patients as a fundamental and essential partner, says project member Durhane Wong-Rieger, President & CEO of the Canadian Organization for Rare Disorders
Tell us more about yourself.
Durhane Wong-Rieger – CORD
I am President & CEO of the Canadian Organization for Rare Disorders (CORD). As head of a global alliance of rare disease patient organizations in Canada, we are committed to improving the lives of all Canadian patients and families affected by rare diseases. CORD leads initiatives to change and implement policy, collaborate to improve healthcare infrastructure, services, and access; advocate on behalf of patients for interventions, including innovative medicines, devices, and supportive services and foster research. We also increase the capacity of the patient organizations to take an active role in advocacy and support through a variety of training programs and collaborative projects.
I am a psychologist by training and taught for about 20 years as a university professor before moving to full-time patient advocacy. I have two children born with rare conditions and a spouse with genetic heart disease and Parkinson’s Disease.
What does personalized medicine mean to you?
Personalized medicine is an “umbrella” concept that is inherent to rare diseases, whereby patients receive “targeted” care based on their individual diagnosis (disease, genetic/genomic profile, and lifestyle factors). The “rarer” the condition, the more challenging to get to a diagnosis but also the more critical to get it “precise” so the term “precision” healthcare is probably more applicable. It is more than just medicines and it may not be so much person-specific as targeted. Challenges exist on many levels, from system capacity, healthcare professional readiness, and patient awareness.
Why do you believe this topic is important as a patient advocate?
Raising awareness and educating patients about personalized medicine is essential to accessing what is available and advocating for what could and should be available. Obviously, the patient is key in terms of seeking access and increasing capacity and willingness (that is, resources).
Why is your organization interested in participating in this multi-stakeholder collaboration?
CORD, along with the international rare disease community, has been advancing models and centers for diagnosis, care, and treatment but also the development of innovative therapies (genetically defined and targeted). Our organization supports at the patient level development of patient registries to have patient populations available for clinical trials and to develop natural history studies as well as clinical trial design appropriate for “small, targeted” patient populations. Our organization has also been engaged in helping to define and collect patient outcome evidence, setting up access programs including new financing models for access, and promoting collaboration across all stakeholders so that innovations could be available as soon as possible but also cognizant of needs for sustainability (e.g., managed access, risk-sharing models, amortized financing).
What makes this collaboration unique?
FT3 is the first international collaboration where patients are not just equal partners but indeed an essential, fundamental partner. The patient voice and patient perspective are not just referenced but have primary status in terms of principles but also in decisions on how to proceed.
What are your ultimate expectations from the project?
The most important short-term expectation is that all stakeholders remain committed to the collaboration and the process; we must learn how to work together effectively. Other short-term expectations are the articulation of consensual vision, principles, desired outcomes and from this platform the definition of a working process for the collaborations that translate into meaningful actions. This is all very high level so more concretely, I expect we will identify some existing best practices and transformative initiatives that can be built upon and adapted. That means we need to continue to foster an open sharing environment and avoid competition and “me-first” thinking. I think we are on the right path to get there.
Ralph Riley is a Global Co Dx Market Access Leader with Janssen. As an FT3 member, he believes that the increased adoption of personalized medicine will create value across the entire health ecosystem
Tell us more about yourself.
Ralph Riley – Janssen
I am the Global Co Dx Market Access Leader where I focus on evidence generation activities to help shape policy and payment to enable patient access to testing across all Janssen’s therapeutic areas. The Co Dx Commercial Strategy Group in Janssen supports our pharmaceutical brand initiatives intended to improve patient outcomes by leveraging a variety of precision medicine tools to screen, diagnose, monitor, or select treatments for patients. I have more than 15 years at J&J – including 6 in our device sector in our former clinical diagnostics unit with the remaining time within R&D and Global Commercial Strategy at Janssen.
What does personalized medicine mean to you?
It’s the next generation of healthcare – where the patient gets treated and not just the disease. Personalized medicine leverages technologies to use validated information by a clinician and a patient to jointly make informed and timely choices about potential decisions regarding the patient’s health and well-being. It helps to more precisely answer questions regarding whether a patient is at risk for a disease, which disease a patient may have, how serious their particular disease may be, which treatment may best serve them and how they are doing once on treatment.
Why do you believe this topic is important?
I believe there is no more profound way to create value for the system than to dramatically increase our success in improving health, and prevention plays a major role in more precise treatment of diseases too.
Why is your organization interested in participating in this multi-stakeholder collaboration?
A variety of stakeholders – especially patients – but including providers, industry (pharmaceutical, device, diagnostic, laboratory) and payers are all important. Given the structure of the marketplace, the way to align discussion and demonstration of value for each stakeholder is to leverage a collaboration to listen and work with a common purpose. This means working with trade groups and patient organizations to find common ways to achieve better patient outcomes. These approaches are not new but are invaluable when large, strategic change is in the making.
What makes this collaboration unique?
I think in how it began- and will ultimately finish. With the collaborators directly listening to patients and their desires to give direction to our work. In a way that mirrors what I think personalized medicine means to me…with the patient at the start and finish of our intentions.
What are your ultimate expectations from the project?
Enablement of the dialog between patient and provider to make the best choices for the patient across their journey… with data and evidence to support those decisions…
A recent virtual workshop sought to determine the needs of patients when it comes to personalized or precision medicine. Danielle Barron reports
What is personalized medicine, and what does it mean for the everyday reality of patients?
A uniquely designed workshop at the recent Patient Engagement Open Forum sought to answer these questions by seeking to understand the landscape of information and support needs of the patient community (and others) in order to make informed decisions around personalized (or precision) medicine at various points in time.
Hosted by PARADIGM, PFMD and EUPATI, the Patient Engagement Open forum is a series of virtual events where a broad range of stakeholders aim to work together to turn patient engagement into reality. Although it migrated online this year, a large and eager audience still participated in each of the interactive and dynamic workshops.
“From diagnosis to treatment and beyond: personalized medicine – what’s in it for patients and understanding patient needs to make it a reality?” was the workshop led by the “From Testing to Treatment Program” (FT3). The program aims to accelerate the benefits of Personalized Medicine and Precision Medicine (PM) by generating better evidence, creating awareness and building the conditions for better access to targeted medicine in oncology and beyond.
FT3 is a global, open and collaborative multistakeholder program that aims to build synergies and accelerate pragmatic efforts to make PM an accessible reality for all those who could benefit from it, starting with oncology and testing.
During this PE Open Forum workshop, attendees participated in an interactive exercise aimed at understanding the landscape of information and support needs of the patient community (and others) in order to make informed decisions around PM at various points in time. The insights gathered will inform the co-creating of PM support and information materials for the patient community, supplementing existing resources and tailoring the content to best address the unmet needs of patients.
Each patient experience is different, but the workshop aimed to highlight distinct ‘moments’ that are relevant from a patient perspective in terms of personalized and precision medicine. These were: Risk; Prognosis; Diagnosis; Treatment; and Monitoring. Patients were asked to identify their needs as they related to each of these distinct aspects of their experience so that a picture could be painted of changing requirements at these different and discrete moments.
In total, a staggering 220 support and information needs were identified during the workshop. Grouping these and assimilating similar suggestions led to a “top 10” list of needs, which helped to paint a picture of the myriad questions patients understandably have about all aspects of their diagnosis, prognosis, treatment and outcomes. What emerged was that patients are missing or don’t know where to find information crucial to each aspect of their patient experience, from initial diagnosis to treatment to living with the condition.
Although these are simply a sample of the hundreds if needs identified during the workshop, they were those that were shared by the largest number of patients who participated. These insights will now be considered as the FT3 begins its co-creation initiative with patients.
Leslie Manot was a patient representative who spoke powerfully about her personal experience with rare lung cancer during the session. She praised the “brainstorming” format utilized within the workshop, saying it compared favorably to other similar webinars or web conferences.
“The format was very clear and well prepared,” said Manot. “The instructions were easy to understand and the results quickly readable.” She added that this event was hugely important, as it was a great opportunity for patients to pool their knowledge and strengths in order to improve their care at an international level.
Manot noted that it was a learning curve for patients too, helping them to understand that the struggles or difficulties they may face, other patients around the world face them too. “The surprise for me was to understand that most of the participants have the same issue as in France for test availability and drug market access.” She added she hopes the results will form the basis for discussions with the relevant authorities on the wide disparities in innovation access.
Why is it so important to capture patient insights? Manot said: “The patients are becoming experts in their disease, as due to internet utilization they have access to medical information, and they can share their experiences with other patients. Some of them even have a diploma to show they are a ‘patient-expert’. The professionals have knowledge but patients have experience.”
FT3 member Ivica Belina, President of the Coalition of Associations in Healthcare, says a multistakeholder approach is essential in order to foster a culture of personalized medicine in healthcare
Tell me about yourself?
Ivica Belina – KUZ
I’m the president of Coalition of Association in Healthcare, a national umbrella patient organization in Croatia, which gathers over 30 patient organizations in different disease areas. The organization was founded in 2002 by a group of enthusiasts who wanted to unify and amplify the voice of patients in Croatia. The main activities of the organization are the protection and promotion of patient’s rights, but also raising awareness of patient’s obligations in the healthcare system, promotion of the active role of patients in the healthcare system and in their own treatment, and public policy in healthcare.
In the past four years we have also established a few informal platforms of patient organizations in the field of rare diseases, patients treated with biologic therapy and oncology. My own background is related to work in different civil organizations, mainly focused on people with disabilities, and by education I’m an educational rehabilitator (special needs teacher).
What does personalized medicine mean to you?
I’m guided by the idea that we should treat the person, and not the disease. By that I mean that when a person becomes sick or afflicted with the disease or condition, the whole life of that person is influenced by this, and the entire ecosystem of that person is influenced to the same extent. So I believe that we should approach the patient as a whole, and provide all the necessary levels of support. Of course, I deeply understand the individual differences in the disease in different patients and the way they cope with the disease, and the only way to approach this is through personalized medicine.
Why do you believe this topic is important as a member of a patient organization?
The meaning of personalized medicine is important to me, because if I was ever to become a patient, I could get much more actively involved in the treatment and management of my disease, get much more information on my disease, codecide on my treatment and care programs, achieve better quality of life, help other patients with my data, information and experience, achieve better and closer relationship with my treatment and care providers, get involved in clinical trials… I believe that this is important to all patients.
Why is your organization interested in participating in this multi-stakeholder collaboration? What has your organization been doing in this space?
My organization is always involved in the dialogues between different stakeholders in healthcare, we strive to have different perspectives on problems in healthcare, and raise topics that are not present in the public space. We see the personalized approach as the only way to improve treatment outcomes and quality of life of patients in the existing healthcare environment, but also better working conditions for the people who are professionally involved in healthcare. To achieve this, we need to transcend medicine and the individual roles of the different healthcare stakeholders, and make the leap forward together. Actually, I don’t believe that any positive change is possible without a multistakeholder approach.
What makes this collaboration unique?
The topic of personalized medicine transcends the disease-specific approach, positions of different stakeholders in healthcare and local/national/regional interests. Since this is such a new area, we all need to find a way to bring it to life, so it would become standard in healthcare. The special thing about this collaboration is that everyone involved is there from the start, so no one feels like he/she has been handed with already developed solutions, we are creating them together. Along the way, we are also detecting some problems and issues, but also the ways to solve them. For me, the uniqueness and strength of this collaboration is in so many different individualities working together, challenging themselves and everybody else in an attempt to make personalized medicine our everyday reality.
Tanya Knott, Director, SJK Foundation, spoke to Danielle Barron about her vision of personalized medicine and the personal tragedy that drives her to make it a reality.
Tanya Knott – Sarah Jennifer Knott Foundation
Tell us more about you
I am the founder and director of the Sarah Jennifer Knott Foundation. I established the foundation with family and friends in 2016 following the death of my sister Sarah aged 31 from cancer of unknown primary also known as CUP. The aim of the foundation is to raise awareness, education and research into CUP. This is an unusual type of cancer where the patient presents with a secondary cancer but the origin of the cancer is unknown. If the primary is unknown, the treatment is unknown. In Ireland, cancer of unknown primary affects up to 400 patients annually. Approximately 3-5% of all cancers are cancer of unknown primary, making CUP the fourth most common cause of death from cancer worldwide.
My background is in healthcare, I am a registered nurse and worked for many years in the pharmaceutical industry. Throughout Sarah’s illness I realized the huge gap in care for patients like Sarah with CUP and armed with this knowledge I set up the foundation in an effort to address the unmet needs for patients diagnosed with this devastating illness.
What does personalized medicine mean to you?
In the future, where the cancer began will not be the treatment priority, and instead treatment will be tailored to the unique genetic make up found by genomic profiling. Enabling targeted treatment options specific to the individual patients needs. For patients with cancer of unknown primary and unusual cancers, genetic profiling offers a vital opportunity for targeted treatment. Studies have shown that relevant mutations are observed in 30-85% of patients with cancer of unknown primary. Genomic profiling is opening up access to clinical trials and access to revolutionary new treatments; unfortunately, despite these benefits, the transition to personalized medicine is slow.
Why do you believe this topic is important?
I believe access to personalized medicine is vital as it has the potential to transform patient’s lives, especially for patients with limited treatment options. Personalized medicine is a rapidly evolving field, with more and more targeted treatments available, but without the diagnostic profiling some of these therapies are not being considered for patients where they could be of huge benefit.
So much has changed since Sarah was ill, that if she was alive today there is a high chance that through genomic profiling an actionable mutation may have been found and she could have lived longer with controlled disease and many more treatment options.
Why is your organization interested in participating in this multi-stakeholder collaboration?
SJK are honored to be founding members of this multi-stakeholder collaboration. One of our main objectives at SJK is to advocate for access to genomic profiling for all those who need it. Our goal is completely aligned with this global project. I quickly realized that to ensure patient access to genomic profiling I would need to be a part of something bigger! The barriers are so great and diverse it needs a multi-stakeholder collaboration to understand and overcome all the many obstacles. Through the multi-stakeholder approach we have the solutions to the barriers, we know where to focus for change.
The patient benefit is that they are given the option. Personalized medicine may not be an option for all patients, but to have the opportunity to be fully informed and to have the opportunity to discuss and understand the option with their HCP is a great benefit. Patients can also benefit from more accurate diagnoses, targeted treatments, with greater survival than perhaps their current treatment and with potentially less side effects. Patients can benefit from access to clinical trials and patients and their families can benefit from preventing hereditary cancers.
What makes this collaboration unique?
This is a unique collaboration as it is bringing together all the multi-stakeholders required to make personalized medicine a reality. As a global initiative, we can learn from best practices in countries and share these learnings with other countries. All stakeholders come with their expertise, the drive to overcome the obstacles, identify the solutions and implement the changes needed.
