Path to transform Rare Disease Care within our complex health system
Anthea Cherednichenko
Vice President, Franchise Head in US Business Unit and Global Program Lead. Global commercial leader. Championing for rare diseases. Striving for empowered teams where innovation, and our people, thrive.
Once again, Takeda Austria hosted its annual roundtable on rare diseases at the Schafalm talks in the historical location of Alpbach, gathering expert stakeholders from within the healthcare system to address the challenges faced by individuals living with rare diseases. As the host of this event for the third consecutive year, I have been consistently inspired by the passion and engagement of diverse stakeholders who share a common goal: to create a better future for these patients. While there may be differences of opinion on how we get there, the consensus remains that change is necessary.?Listening to the session this year with my Public Health hat firmly on, I was inspired to explore deeply the challenges faced by rare disease patients and caretakers in Austria including the existing inefficiencies in the healthcare system and consider how collaborative approaches are necessary to overcoming these obstacles.
Challenges in Rare Disease Care
One of the critical challenges in rare disease care is the often high cost of treatment, coupled with limited data, making it difficult to assess the value and financial risks associated with these life-saving or changing therapies. Moreover, the fragmented funding system in Austria often results in individual patient costs burdening or surpassing the budget of a small region. The introduction of Advanced Therapy Medicinal Products (ATMP), such as gene and cell therapies, is expected to further amplify these challenges.?Furthermore, it puts to the test how we prioritise public health spending and how we equitably distribute healthcare across a population.
Identifying System Inefficiencies
Whilst there is no utopian way to allocate health resources, there are tools that can be used to maximize the health outcomes.?In the real world, all health systems have inefficiencies.?To address these inefficiencies, it can help to adopt a holistic approach to patient costs and benefits, or outcomes. ?In Austria, there is a lack of emphasis on considering the cost-effectiveness and health benefits of various treatment settings, such as home care, which can often be both more efficient and effective for patients. ?Additionally, patients and caretakers face difficulties navigating through the system and accessing lower-cost diagnosis pathways, such as primary care centres.
Improving Patient Outcomes
While price cutting on pharmaceuticals may offer short-term cost savings, it does not provide a sustainable solution. In Austria, only 12.9 % of total healthcare spend is allocated to drugs, with the majority of 37.2% being allocated to hospitals. To enhance efficiency, the healthcare system must explore strategies to triage patients and provide outpatient care effectively. By better aligning funding mechanisms, we can further ensure a more connected and resource conscious approach to patient treatment.
Path to Transformation
To achieve a more effective and patient-centric healthcare system for rare diseases, a collaborative effort is required.?Stakeholders from all sectors must set aside politics and work together towards the shared vision I heard at our roundtable.?Starting dedicated funding pots for rare diseases can be established, providing much-needed shared resources and risk reduction.?Furthermore, pharmaceutical companies can contribute by introducing models and risk-sharing approaches to prioritize patient outcomes, ensuring mutual commitment and accountability.
Addressing the challenges in rare disease care requires a united front from all stakeholders involved. By fostering bold collaboration and piloting innovative models, I believe we can pave the way for improved patient outcomes in Austria, even beyond rare diseases.?Let us seize this opportunity and the energy I observe in our stakeholders and shape a healthcare system that not only manages costs but also delivers holistic value, providing patients with the care they deserve. Together, we can make a meaningful difference in the lives of those living with rare diseases.
Head of Patient Value & Access
1 年Thanks a lot for sharing your thoughts and highlighting so many important aspects, Anthea! “WHY does the industry host a stakeholder round table like in Alpbach?" The answer to it seems to come as a surprise to some: Because we have a significant interest in promoting a healthy and sustainable healthcare system. We witness patients getting lost in the system, particularly during the transition from pediatric to adult care. We also see cases where patients do not receive the legally mandated adequate and appropriate treatment, due to various reasons. A healthy, sustainable healthcare system sets us all up for future success: as a society as well as an industry. Thanks to everyone involved, for your passion and your engagement to change things to the better, I truly enjoyed it. Thank you Anthea Cherednichenko for role modeling! ?? ?? ?
Senior Partner & Lead Facilitator Integral Leadership Dynamics. Inspire | Lead | Deliver Global Executive Learning Experiences & Team Development Inspiring leaders, managers, teams to be and perform at their best.
1 年Sounds like a wonderful opportunity to challenge the existing perceived “status quo”, which in their own ways, all sectors and organisations face and either cannot see or choose to avoid. A wonderful initiative Anthea Cherednichenko to create and leave a legacy where what we first discovered, will be left in a better position than we found it. And too, when we pass the “torch” on to others who will follow, with a similar purpose and desire to create better opportunities for those who need it most….. ????