News / Partnerships - March 29, 2022

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”.

An intense year of discussion and exchanges between the Experts, as well as with representatives of the EU Institutions and the broad rare disease community, led the OD Expert Group to decide to build further on its work. Phase 2 will focus on a more granular approach to the Orphan Medicinal Products (OMP) Regulation and to the EU Health Technology Assessment (HTA).

Phase 2 builds on the OMP Regulation and EU HTA Regulation

Phase 2 of the OD Expert Group will therefore cluster on two topics, creating content based on some of the previous recommendations.

  • The first topic covers the upcoming revision of the OMP Regulation and how to address unmet medical needs; it will build on the 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 discuss a methodology for orphan drugs under the new EU HTA Regulation, starting from the proposal on creating a common EU value assessment for OMPs.

Phase 2 will carry on through 2022. The OD Expert Group has already starting discussing the recommendations around the OMP Regulation, while the work on the EU HTA will begin in the second half of the year. The Expert Group will publish its new recommendations during the course of 2022, and host a launch event to publicly share its work.

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.

You can find the original press release here.