Press Release

OD Expert Group kicks off Phase 2

The multi-stakeholder European Expert Group on Orphan Drug Incentives (OD Expert Group) has officially announced the kick off of Phase 2 of its work on 29 March, during the DIA Europe session on “The European Expert Group on Orphan Drug Incentives: a Blueprint for Health and Pharmaceutical Innovation”.  On the same day, the Ministers of Health of the 27 European Members States met in the Employment, Social Policy, Health and Consumer Affairs Council (EPSCO) to discuss a European response to rare diseases, as part of the work of the French Presidency of the EU, that made this topic one of its priorities.

The OD Expert Group decided it was timely and important to contribute to the political debate by focussing the Phase 2 of its work on two topics, the Orphan Medicinal Products (OMP) Regulation and the new EU Health Technology Assessment (HTA) Regulation, creating content based on some of the recommendations the Group published last year:

  • The first topic covers the upcoming revision of the OMP Regulation and how to address unmet medical needs. It will build on the OD Expert Group’s proposals on modulation of market exclusivity, on strengthening EMA’s role in advising OMP developers through the OMP pathway, and on increasing legal certainty around the concept of Significant Benefit.
  • The second topic will focus on methodologies for orphan drugs under the new EU HTA Regulation, starting from the proposal on creating a common EU value assessment for OMPs.

The session at DIA Europe 2022 “The European Expert Group on Orphan Drug Incentives: a Blueprint for Health and Pharmaceutical Innovation” has been the opportunity to reflect on the achievements and next steps of the multi-stakeholder OD Expert Group with Yann Le Cam, CEO of EURORDIS, and Alexander Natz, Secretary General at EUCOPE.  The Experts discussed how the recommendations of the Group and its upcoming work in Phase 2 aims to contribute to the revision of the OMP Regulation and the implementation of the EU HTA Regulation.

The event gave the opportunity to discuss, with key EU decision-makers, the Experts’ past and upcoming work and its contribution to the evolving rare disease landscape in Europe. The debate also featured Lina Nordquist, Member of Swedish Parliament and health policy spokesperson, and Violeta Stoyanova, Chair of the Committee for Orphan Medicinal Products at the EMA.

Posted on 29. March 2022