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Orphan Drugs Expert Group launched Open PDF
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European Expert Group of Orphan Drug Incentives

The multidisciplinary and cross-functional expert group brings together representatives of the broad rare disease community, including researchers, academia, patient representatives, members of the investor community, rare disease companies, and trade associations. The Group aspires to become a reliable source of information for policy-makers and an established space for the discussion of consensual policy proposals, in a constant effort to bring together the knowledge and needs of the whole rare disease ecosystem.

This group aimed to become the source of ground-breaking ideas and potential solutions that will provide input to the Orphan Medicinal Products (OMP) Regulation Evaluation. The experts have worked together from 2020-2021 and developed fourteen policy proposals throughout the entire OMP lifecycle. The policy report was presented to the public on 11 June 2021 during a dedicated launch event.

The Group continued the intense work by developing its policy proposals for the revision of the OMP Regulation. In 2023, the OD Expert Group presented its proposals during a workshop in the European Parliament, including ideas for the modulation of market exclusivity, the definition of significant benefit and the improvements to regulatory pathways.

Through a series of workshops, consultations, and as a result of a collaborative effort, the OD Expert Group has developed through the years its proposals for a European HTA fit for Rare Diseases, capable of addressing the key challenges linked to medicinal products for rare and ultra-rare condition. In 2023, during our event, the OD Expert Group officially launched its reports on HTA.


Read our 14 Recommendations

Read our Full Report “Modulating Incentives for OMP Development: Modulation framework and policy proposals

Read our Report: AN EU HTA FIT FOR RARE DISEASES – Part 1: Clinical evidence in joint clinical assessments

Read our Report: AN EU HTA FIT FOR RARE DISEASES – Part 2: Stakeholder involvement in joint clinical assessments

Our Policy Proposals