Full event summary report can be found below:
APARDO is proud to have helped organise the first-ever EU-China Rare Disease Policy and Access Summit, bringing together experts from Europe and China in Dublin, Ireland in October 2024 to make a difference for everyone living with rare diseases. Whether we are in Brussels or Beijing, there are many shared challenges we face as a rare disease community. That is why we joined forces to come up with a vision for the future of rare disease in both geographies, based on five recommendations.
First, including patients in shared decision making must become a reality and not just a promise on paper. Health systems have a duty to recognise patients’ expertise and to integrate it into policymaking and service delivery. To this end, governments should provide financial and training support for such organisations. Additionally, global exchanges between patient organisations would allow them to benefit from each other’s knowledge and experience.
Second, we must seek to optimise and improve early diagnosis through technology uptake, in addition to training for current and future healthcare professionals. Expanding access to newborn screening could go some way to shortening the diagnostic odyssey, as could harnessing the potential of AI to detect patterns in health records that may point towards a rare disease. Education and training on rare diseases and genetic medicine is also crucial to make sure health systems have access to the skills needed to provide these services.
Third, pooling and harmonisation of data can boost research and support value assessment of new health technologies. All too often, though, hospitals and medical institutions use systems that are not compatible with each other. This limits health system capacity to generate large datasets for research purposes, as well as posing difficulties for patients who move home (whether domestically or internationally). Establishing or improving data quality of rare disease registries is an important step forward, which allows researchers and medicine developers to apply to access the data for their studies.
Fourth, European and Chinese people living with rare diseases would benefit from care networks to help them access multidisciplinary care no matter where in their country they live. To make this a success, the care networks need to be integrated into health systems so that local doctors can refer patients to them and the networks can facilitate research and expertise-sharing across the country.
Fifth, funding for rare disease treatments and innovation represents a real barrier to access. Out-of-pocket costs still hit people living with rare diseases hard, and there is an urgent need to close the rare disease funding gap in reimbursement rates by encouraging health systems to implement innovative payment mechanisms. This should be part of a cohesive system of incentives and regulations that promotes development of new medicines and gets them to the people who need them most.
We look forward to continuing to exchange information and experiences with our partners in Europe and China, and hope this platform will provide an opportunity for ongoing cooperation between people living with rare diseases.
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