Closed due to overcrowding… was the registration list for the political expert discussion “Rare Diseases in the German Bundestag” on 10 October, initiated by Erich Irlstorfer, Member of the German Bundestag. The level of interest in the political sphere is impressive – and shows the urgency of the issue.
“People with rare diseases need decisions and, above all, action”
This urgency was also emphasised by Professor Annette Grüters-Kieslich in her keynote speech: People with rare diseases and their families don’t have time for long discussions, they need decisions and above all action! The professor of paediatrics pointed out that every working day, a new rare disease is described – so they are anything but rare. However, the care situation is particularly challenging, as small case numbers and often late diagnosis – in children after four years, in adults after an average of eight years – make targeted therapies difficult. The key is targeted research, which in the field of rare diseases must always be inextricably linked to care.
The Chairwoman of the Eva Luise and Horst Köhler Foundation would like to see better framework conditions for research, structured access to diagnostics, innovative therapies and drugs, as well as reliable social and financial support for patients. It is an important success that rare diseases are explicitly mentioned in the draft of the Hospital Reform Act. The reform must now be designed in such a way as to ensure that care is tailored to the needs of patients. Grüters-Kieslich stressed that positive developments such as the National Action Plan for People with Rare Diseases (NAMSE) and the establishment of the National Register for Rare Diseases (NARSE) must now be made permanent. Reliable funding for rare disease centres is also essential. We don’t have an awareness problem, we have an implementation problem,” says Grüters-Kieslich. Let’s tackle it!
Empowering self-help and spreading knowledge
Geske Wehr, Chairwoman of ACHSE e.V., focused on the concerns of patients in the subsequent discussion moderated by Erich Irlstorfer. She emphasised: “The German healthcare system is only made for healthy people! People with rare diseases still do not have equal access to appropriate care. Self-help as the fourth pillar of the healthcare system is not sufficiently supported. Wehr called for the expertise of rare disease centres to be disseminated more widely. We can’t have patients still carrying CDs with pictures from A to B,” she explained, citing telemedicine as a possible approach to shortening care paths: “Knowledge has to travel!
Policy in motion: perseverance for structural change
Erich Irlstorfer, the driving force behind a recent motion by the CDU/CSU parliamentary group in the German Bundestag calling for more support for patients and self-help, as well as improvements in research and care, summarised: “Together we face the task of finally giving people with rare diseases the attention and support they deserve and need. The issue has been underestimated for too long. The health politician urged stakeholders to continue to push the issue: “The rare disease package is in place. Now it is up to us to keep it going,” he stressed. A milestone has been reached with the white paper on the Bavarian campaign for rare diseases. It now needs to be disseminated and taken to the European level. We need perseverance in order to anchor structural improvements,” Erich Irlstorfer stressed. More than four million people and their families are counting on us.
Our thanks go to Erich Irlstorfer and his team for organising this valuable event and to all participants and contributors for their important input. We look forward to continuing the discussion!
