In her presentation, Prof. Dr. Grüters-Kieslich highlighted the most pressing issues: the often still inadequate care and the lengthy, sometimes inadequately implemented structural measures in dealing with rare diseases. She emphasized that the vast majority of patients – and their families – are still in a precarious situation, both in terms of medical care and quality of life. “Rare diseases affect not only patients, but entire families. If you consider that with every diagnosis, family members are also deeply involved in the care and support process, this affects far more than just four million people in Germany,” Prof. Grüters-Kieslich emphasized.
The event provided an opportunity for an intensive exchange between experts from politics, industry, science and patient organizations. In addition to Prof. Grüters-Kieslich, Prof. Sylvia Thun, Director of the Core Unit eHealth and Interoperability at the Berlin Institute for Health (BIH), and Stefanie N. Falkenburg, Director Business Unit Rare Chiesi GmbH, provided valuable input. Moderated by Ulrich Tilly, Linda Heitmann, Member of the German Bundestag for Bündnis 90/Die Grünen, then discussed perspectives and solutions.
Key topics were the urgent need to further expand specialized centers for rare diseases, the establishment of a national registry for rare diseases, and the targeted promotion of research and therapy development. Prof. Grüters-Kieslich emphasized that the expansion of centers and the establishment of registries are key prerequisites for sustainable improvements in care. “It is not enough to drive research forward – we must also ensure that this research is translated into clinical practice and benefits patients,” she said.
During the discussion, it became clear that cross-sector collaboration is essential to achieve the necessary structural changes. “We agree that the challenges are enormous. We must act now. The patients cannot wait any longer,” said Annette-Grüters-Kieslich. The political agenda for rare diseases must be consistently driven forward so that the patients can benefit promptly and comprehensively from the progress in medical research.
Many thanks to Chiesi Deutschland for organizing the event and to all participants who contributed to the discussion with their expertise and commitment. Platforms like this are needed to continue the dialogue and set the course for a better future.
Picture: Reimann Fotografie
