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.
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.