The following positions are supported by Aspire4Rare – an initiative of UCB Pharma GmbH, change4RARE – an initiative of Alexion Pharma Germany GmbH, Ipsen Pharma GmbH as well as the Eva Luise und Horst Köhler Stiftung.
Key Message
In our health care system based on solidarity, more needs to be done to ensure that people with rare diseases, in particular, can better participate in medical progress. To this end, targeted measures to improve care and promote research and innovation must be taken now.
Key demands:
Strengthen Coordination of Measures
The National Action Alliance for People with Rare Diseases must institutionalise a regular, multi-stakeholder dialogue on the coordination of care and research. This requires a sustainable organisation with an office and political contacts.
Securing Care Structures
Structured care in specialised centres must be guaranteed by secure funding. This is essential to ensure efficient and high-quality diagnostics and treatment.
Ensure favourableFramework Conditions and Incentives for Research and Development
The European funding system for translational research in rare diseases must be strengthened. EU market exclusivity for orphan drugs should be extended. The conduct of clinical trials in Europe must also be accelerated and made less bureaucratic to speed up the development of new therapies.
Securing Patient Access to Orphan Drugs in the Long Term
In order to ensure rapid and comprehensive access to orphan drugs, it is necessary to maintain the orphan drug regulation in the national benefit assessment procedure. The evidence requirements for medicines for specific therapeutic situations, such as congenital, often ultra-rare diseases, also need to be adapted.
Utilising Digital Transformation
The potential of digital data collection must be harnessed for care, research and innovation. This includes the creation of a national rare disease patient registry and the use of electronic patient records.
Expand Early Detection
The early detection of treatable diseases must be improved. This includes extended newborn screening and increased awareness of rare diseases throughout the healthcare system.
Strengthen Patient Participation and Self-Help
Patients must be actively involved in research and care. Their participation should be strengthened and recognised as a valuable part of medical progress.
