Rare diseases are not a niche topic: In Germany alone, over four million people live with one of more than 8,000 known rare conditions. Patients and their families suffer from long diagnostic journeys, lack of therapy options, and fragmented structures. Their care is not just a medical concern, but also a political and societal responsibility.
At the same time, rare diseases are a driving force for progress and a blueprint for the personalized medicine of the future: Research in this field focuses on precision therapies, new care models, and digital solutions—innovations that go far beyond rare diseases themselves. Investing in this area not only strengthens care for individuals, but also lays the foundation for improvements in many common diseases.
To ensure this engine of innovation remains sustainable, politics and society must act decisively now: We need the National Action Alliance for People with Rare Diseases to be firmly established with a dedicated office to ensure dialogue among all relevant stakeholders. Centers for Rare Diseases, which offer guidance and perspectives to those affected, must receive structural and permanent funding. Research depends on targeted support, reliable frameworks for orphan drugs, and less bureaucracy for clinical trials. Equally important are building registries—both nationally and internationally—using electronic patient records, improving early detection through genetic newborn screening, and raising awareness of the topic in medical education.
Rare diseases are not only a test for the effectiveness of our healthcare system. They are an opportunity to make it innovative and future-proof.
This article by Prof. Dr. Annette Grüters-Kieslich was published on October 16, 2025, in the health magazine “Future Medicine,” included in a special edition of the weekly newspaper DIE ZEIT. The magazine is available as an e-paper for download.
