EUCOPE Resources / Events & Meetings - October 3, 2022

Event Report: The revision of the EU legal framework for orphan drugs and its impact on Germany

On September 5, 2022, the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) and Bundesverband der Pharmazeutischen Industrie (BPI) organised an online event on the revision of the EU regulatory framework for orphan drugs and its impact on Germany. The upcoming revision was discussed together with patient representatives, policy makers, regulators, physicians and representatives of the industry and counted on the participation of more than 200 attendees.

The discussion focused on the opportunities and risks of the expected changes to the Orphan Medicinal Product Regulation (Regulation 141/2000) and the interplay of this reform with the discussed changes to the orphan regime in Germany.

In the case of medicinal products for rare diseases, two political strands are currently running in parallel: the EU is revising the regulatory framework (advantages of approval and market exclusivity) and the Bundestag is discussing an AMNOG reform.

The German AMNOG treats orphan drugs according to EU approval criteria. The two strands of regulation are indirectly related. Without careful consideration of the impact and implications of the discussed provisions we risk that the combined effect of these two reforms produce a double damage.

To further advance a European strategy on rare diseases as well as the discussions on areas of unmet needs, an open exchange with all stakeholders should continue to take place in order to avoid hampering the achievements of the current system.

 Review our Event Report – The revision of the EU legal framework for orphan drugs and its impact on Germany




  • Dr Alexander Natz, European Confederation of Pharmaceutical Entrepreneurs (EUCOPE)


  • Dr. Andrew Ullmann, Member of the German Bundestag


  • Miriam Schlangen, head of the National Action Alliance for People with Rare Diseases (NAMSE) and Cystic Fibrosis Association (Mukoviszidose e.V.)
  • Martin Lack, Head of Pharmaceuticals Department, National Association of Statutory Health Insurance Physicians (Kassenärztliche Bundesvereinigung)
  • Matthias Heck, Senior Director International TA Policy Strategy/ Government Affairs & Policy (EU/Germany)


  • Matthias Wilken, Bundesverband der Pharmazeutischen Industrie (BPI)