In May the BIA launched a briefing paper on Orphan Medicinal Products that looked into the 15-year application of the Orphan Regulation in Europe.

The paper coincided with the European Conference on Rare Diseases & Orphan Products, which was held in the UK for the first time and brought together stakeholders from across the rare disease community.

The briefing paper explores the EU regulatory framework and the setting up of the Committee for Orphan Medicinal Products within the European Medicines Agency (EMA) and raises awareness around the latest regulatory policy developments in this field. The European Commission is currently in the process of considering a review of its Communication (2003/C 178/02) to streamline and adapt the existing guidance to technical progress. This revision is of great interest to BIA members as a large number of biotech medicines are authorised with an orphan indication.

To download the briefing paper go to the BIA website.

omp table

The BIA continues to play an active role at EU level in influencing the regulations that have an impact on how UK bioscience tackles rare diseases. We have seen that the incentives of the Orphan Regulation have been essential in stimulating research and development of orphan medicines for rare diseases.

It is therefore important that the requirements to demonstrate significant benefit of orphan medicines are appropriate, given the complexities of dealing with small patient populations and rare conditions to be treated.

To date, the European Commission has authorised 121 orphan medicines for the benefit of patients suffering from rare diseases. Some 25 orphan marketing authorisations are from SMEs as of January 2016. Furthermore, the European Commission has designated 1,349 products as orphan medicinal products.

The European Commission has launched a public consultation on the concept of ‘similar medicinal product’ in the context of the orphan legislation until 4 November 2016. If members wish to comment on the proposed changes to Article 3 of Commission Regulation (EC) 847/2000 please contact Christiane Abouzeid.

For more information on the latest policy and regulatory affairs work at the BIA, download our quarterly report covering April to July 2016.