Moderated by Christian Dierks, the panel discussed with Christiane Mockenhaupt, Chairwoman of the self-help organisation for rare complement-mediated diseases MPGN and aHUS e.V., Prof. Dr. Barbara Sickmüller, President of the German Society for Regulatory Affairs and Prof. Dr. Thomas O.F. Wagner, Head of the Reference Centre for Rare Diseases Frankfurt, how the EU pharmaceutical package can be designed to secure and improve access to urgently needed therapies for people with rare and ultra-rare disease
It became clear that the reform of the package is particularly important for the ‘orphans of medicine’. It offers the opportunity to improve access to innovative therapies – but also to lose it. The reform process must therefore be closely and carefully monitored. Strong networking, reliable data through registries and innovative funding instruments such as public-private partnerships are essential to close the translational gap in Europe, which is still far too large.
Many thanks to all participants for their valuable contributions and to the organisers for choosing this highly relevant topic.
The recording of the discussion (in German) is available here: ROUNDTABLE