The conversation centered on the special requirements for caring for people with rare diseases, especially the question of how highly specialized expertise can be secured and better connected in the long term. Many of these diseases require treatment in specialized university medical centers, where clinical care, research, and diagnostics work closely and expertise is maintained for the future.
Rare diseases need specialized structures – Coordination is key to progress
Against this backdrop, it became clear how vital stable framework conditions are for such care structures. Progress happens where clinical care, research, and data use are closely interlinked. Advances in genomic medicine and personalized therapy approaches already show the potential of this interaction.
Health policy framework conditions also play a major role. The discussion addressed how the evaluation of new therapies under the Pharmaceutical Market Reorganization Act (AMNOG) can better take into account the particularities of rare and highly specialized indications in the future.
Another aspect concerned possibilities for earlier diagnostics. Advances in genetic diagnostics are opening new perspectives for expanded newborn genetic screening. To carefully examine the opportunities and challenges of such approaches, an interdisciplinary task force could help systematically analyze possible next steps.
A further focus of the conversation was on the role of overarching coordination structures. The National Action Alliance for People with Rare Diseases (NAMSE) has brought together key stakeholders from policy, care, research, and patient advocacy for many years, providing an important foundation for coordinated development and safeguarding achievements.
Especially for complex conditions that span multiple medical specialties and areas of care, such coordinated collaboration is vital to combine knowledge, leverage synergies, and achieve sustainable improvements.
Dialogue between Politics and Civil Society
Our heartfelt thanks go to Federal Health Minister Nina Warken for this open exchange. The conversation once again demonstrated how essential ongoing dialogue between politics, science, care, and civil society is to sustainably improve the situation of people with rare diseases. We look forward to continuing this dialogue.
