People with rare diseases and disabilities face common challenges in the Nordic region. Small populations in each individual country make it tricky to set up treatment protocols and gather data, for example, and Nordic cooperation is being highlighted as a possible answer to many of the issues.
It is the rare that unites, and only another rare truly understands the difficulties. We have found this to be truer than many institutions or governments realize. With this in mind, SBONN, a Nordic network for people living with rare diseases, has been a channel for exchanging information and valuable best practices for over a decade. We have influenced, learned, and shared knowledge with each other for the betterment of all people with a rare disease or disability.
Getting national healthcare and social welfare systems to acknowledge the complex challenges that people living with rare diseases and disabilities face in navigating the systems is slow progress. It is therefore vitally important that the Nordics, with similar views on human rights, ethical healthcare, and social welfare, come together to discuss the problems each country faces individually, but with a view to solving them together.
National strategy development in all Nordic countries is one such achievement. Denmark and Finland are leading the way for Norway, Sweden, and Iceland, and SBONN is an active participant, freely sharing our patient perspectives on what has worked – or not worked. For a decade, our focus has been on the differences and challenges in the Nordics. This work has included setting up the knowledge/hospital infrastructure in the European Reference Networks (ERN), hospital networks, or competence centers.
The holistic approach of multi-disciplinary teams, not just looking after the person with the rare disease, but considering all aspects of their lives, is something patients are asking for. And while the supportive voices from professionals agree, the holistic approach rarely fits into the current national healthcare and social welfare systems.”
We have also been gathering data, where we can, but we are slowly understanding just how little we actually know. Many rare diseases are multi-systemic, and as such, difficult to treat. The holistic approach of multi-disciplinary teams, not just looking after the person with the rare disease, but considering all aspects of their lives, is something patients are asking for. And while the supportive voices from professionals agree, the holistic approach rarely fits into the current national healthcare and social welfare systems.
Nordic cooperation is needed to manage the purchasing of orphan drugs, to develop treatment plans and guidelines, and just for the good old exchange of knowledge! Working together has so many benefits for tackling rare diseases. We need to connect patients via organizations, fund research, and find economic and efficient ways to get access to innovations. Ignoring support networks in treatment plans is an oversight, sadly seen too many times in real life.
Research is also very important. Not only into new drugs and treatments, but also to understand us, the people living with rare diseases. This is sorely lacking in the Nordics. We are expensive to treat and keep in hospitals, and healthcare systems look for diagnoses for us for years, even decades.
We are expensive to treat and keep in hospitals, and healthcare systems look for diagnoses for us for years, even decades.”
More emphasis on gathering data on physical and occupational therapies could become key to finding better ways to manage rare diseases and disabilities, as there are only a few hundred rare-disease drugs available on the market globally. Granted, the numbers are rising but so is the number of rare diseases, currently at around 8,000. And the costs increasingly accumulate, as rare diseases are left in the statistical foot notes of government budgets, usually due to a lack of knowledge on the issues’ economic impact. Rare disease treatments often get overlooked with words like “there is no evidence of the effectiveness for disease x”, even if the rare patient has been visibly benefitting from the treatment. More case studies are therefore needed. In rare diseases even the experience of a sole case has value.
The Nordic countries could be global leaders in rare diseases, but it takes work, knowledge, and understanding the fundamental issues of people living with rare diseases and disabilities.”
Rare disease communities in social media thrive, as it is the only access to information on “day-to-day life hacks” that people with rare diseases have. We turn to each other, as many feel unsupported by the medical field. Patient organizations want more involvement and to get invited to the tables where policies and plans are made for us. We want to get our voice heard as a community and get recognition as a group dealing with severe adversities in current society. The Nordic countries could be global leaders in rare diseases, but it takes work, knowledge, and understanding the fundamental issues of people living with rare diseases and disabilities.
Rare diseases are not rare, if you look at the whole picture.
About the Author
This column was originally written by Katri Asikainen, CEO, HARSO, The Finnish Alliance of Rare Diseases and Disabilities Organizations – Secretary for SBONN (The Nordic Network of Patient Associations in Rare Diseases) 2023-2024
