Evaluation of EU legislation on Orphan Medicinal Products (OMPs) and Paediatric Medicines

Brussels  11 August 2020 – 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 evaluation of EU legislation on Orphan Medicinal Products (OMPs) and Paediatric Medicines. Focusing on the impact of the incentives introduced for research, development and marketing for these specific products, the evaluation is complemented by a recently published study, specifically on the EU Regulation on OMPs.

The 20th anniversary evaluation of the OMP Regulation is an opportunity to celebrate the great achievements of the EU in fostering research and treatment of rare diseases.

There is a time before and after the Regulation.  Before the Regulation came into force there was hardly any focus on rare diseases.  Since its inception, many orphan medicines have been developed and rare disease patients in need gained access to therapies.  With the introduction of incentives attached to the granting of an orphan designation – including an up to 10-year market exclusivity, or protocol assistance targeted at SMEs – the Regulation allowed to drive investments in the research and development of OMPs.

As noted by the evaluation, there are a number key elements improving the life of rare disease patients that go beyond the remit of the Regulation. These include rare diseases and pharmaceutical policies at the national level, access to timely diagnosis and availability of medical expertise and rare diseases centers of excellence across Europe. With several rare diseases still without treatment, a holistic approach, taking into account the complexity of the ecosystem for the development of rare disease therapies, is paramount.

Commenting on the need for a holistic approach Alexander Natz, EUCOPE Secretary General said:

The ecosystem for the development of rare disease therapies rests on three essential foundations: the R&D capability of the public and private sector; the European incentives structure; and the market environment.  If you take away one, the whole structure is endangered.

In line with the key takeaways from the evaluation, an event recently organised by EUCOPE served as a starting point to discuss how Europe can remain at the forefront of rare diseases innovation building on a coherent and coordinate EU policy approach to rare diseases. This should encompass: the use of real-world evidence to reduce evidential uncertainty across the lifecycle of rare diseases therapies; harnessing the untapped potential of early stakeholder dialogue for the success of orphan medicines and industrial policies fostering both European home-grown innovation and global investments.

Further stressing that the OMP and Paediatric Regulations do not exist in a void, we call for any actions following the evaluation to also address the challenges for the EU pharmaceutical innovation ecosystem identified in the Pharmaceutical Strategy Roadmap and build on the lessons learnt from the COVID-19 crisis on cooperation and regulatory simplification.

About EUCOPE

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.