Press Releases - August 11, 2020

Press Release: Study on Orphan Regulation

EUCOPE, the European association for small to medium-sized companies in the field of pharmaceuticals, biotechnologies – many of which are researching and developing rare disease treatments – notes the publication of the study on the EU Regulation 141/2000 on Orphan Medicinal Products (OMPs) (here below). The study aims to inform the upcoming European Commission evaluation of EU OMPs and Paediatric Medicines Regulations.

The study acknowledges the achievements of the Regulation in fostering research and treatment of rare diseases and the clear added value of an EU-level action in this field. However, with 95% of rare conditions still without treatment, strengthening the regulatory, scientific and incentives environment for orphan medicine development remains critical for people with rare diseases.  The need for a strong orphan regulatory framework is clear. Incentives coupled with increasing scientific knowledge make it possible to develop new technologies to treat more rare diseases more effectively as well as to successfully translate the experiences gathered to more common diseases.

Many companies, mostly small to medium sized EUCOPE’s members, were able to pursue scientific advance and deliver new therapies to patients with rare diseases thanks to the Regulation.  As highlighted by a study EUCOPE commissioned to the UK Office of Health Economics and recently presented at the 10th European Conference on Rare Diseases (ECRD), more than a third (35%) of granted marketing authorisations for OMPs are held by OMP-focused developers.

Commenting on the publication of the study, Alexander Natz, EUCOPE Secretary General said:

We are at a crossroad. EU institutions, Member States, HTA bodies, payers, pharmaceutical industry and the scientific and rare disease community all share a responsibility to build on the achievements of the OMP Regulation. EUCOPE stands ready to contribute to this effort in cooperation with all relevant stakeholders.

Awaiting the publication of the OMP and Paediatric Regulations evaluation, we call on the European Commission to fully consider in any future policy proposals the impact and interplay of the OMP Regulation with the broader pharmaceutical innovation ecosystem and its role in supporting EU’s strategic objective of improving rare diseases patient access to diagnosis, information and care.


EUCOPE is Europe’s trade body for small to medium-sized innovative companies working in the field of pharmaceuticals and medical technologies. EUCOPE gives voice to more than 900 research-oriented innovative companies and associations active in research, development of pharmaceuticals, biotechnologies and medical devices. Many of our members are developing therapeutic solutions for persons living with a rare disease, who had little to no treatment available just a few years ago.