On November 25, 2025, the change4RARE initiative brought together experts from science, society, politics, business, and self-help at Berlin’s HELIX HUB. In a moderated dialogue, participants exchanged ideas on how access to therapies for people living with rare diseases can be improved, and the role AMNOG plays in this. With keynote speeches, Nora Seitz, Member of the German Bundestag, and Prof. Dr. Annette Grüters-Kieslich opened the event, setting important impulses for the following contributions and discussions.
A brief look at the background is useful to better understand the significance of AMNOG in the context of rare diseases: The Medicines Market Reorganization Act regulates how the additional benefit of new medicines is assessed before they are reimbursed by statutory health insurance. For rare diseases, however, this process often leads to difficulties—such as small patient numbers, limited study data, and evaluation criteria that do not fully reflect the particularities of small cohorts.
What Does an AMNOG Need to Truly Reach Rare Diseases?
This is where the input from Prof. Dr. Annette Grüters-Kieslich came in. She emphasized that rare diseases are often underestimated—both in their complexity and in their significance for the overall healthcare system—and highlighted the importance of a process that reflects real-world conditions: “With a rare disease, there are often only a few patients—but the need is no less. If we evaluate innovations with criteria developed for large patient groups, we lose valuable opportunities. An adapted AMNOG could help ensure therapies reach where they are truly needed.”
In the ensuing discussion, participants delved deeper into these perspectives and identified the current limits of AMNOG procedures regarding rare diseases. Several approaches were named to improve access to innovative therapies in the future, including:
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Adjusting evaluation criteria for small patient cohorts: Traditional evidence requirements cannot often be met with very small groups, necessitating more specific and flexible evaluation logic.
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Broader interpretation of “medical need”: In addition to clinical endpoints, non-medical needs such as individual burdens, complicated supply pathways, or structural bottlenecks should be more strongly considered.
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Thinking in patient pathways: A patient-centered assessment of benefits should map the entire course of care, from diagnosis through therapy to aftercare.
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Greater involvement of scientific expertise: Knowledge from research, specialized clinics, and care facilities is essential to allow realistic and quality-assured evaluations.
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Consistent use of reliable real-world data: High-quality health services research and robust registries can help close evidence gaps and better contextualize additional benefit.
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Stronger cooperation across sector boundaries: Interdisciplinary and cross-sector collaboration is key to creating timely and effective care solutions for rare diseases.
Outlook: What Matters Now
The expert dialogue made it clear that people with rare diseases still face considerable hurdles—from diagnosis and ongoing care to accessing innovative therapies. At the same time, it’s evident that the willingness to tackle these challenges together is growing, transcending disciplinary and sectoral boundaries.
With the new federal government’s commitment to strengthening care for rare diseases, a political window is opening for long-overdue reforms. Further development of AMNOG can become a central lever—provided the specific requirements of rare diseases are consistently taken into account.
The Eva Luise and Horst Köhler Foundation will actively accompany this process and advocate that the perspectives of those affected, as well as scientific and clinical expertise in the field of rare diseases, are reliably incorporated into political decision-making.
Impressions from the Event
About change4RARE
change4RARE is an interdisciplinary initiative by Alexion Pharma Germany, bringing together experts from medicine, research, healthcare, politics, and self-help to improve the situation for people with rare diseases. The focus is on better use of reliable data, earlier and more precise diagnosis, connected care structures, and fair access to innovative therapies. The goal is to develop practical solutions and drive political and structural change to provide tangible benefits for those affected.
Photos: Dierks+Company
