The briefing paper explores the regulatory framework and the setting up of the Committee for Orphan Medicinal Products, and raises awareness around the latest developments in this field
The BIA has launched a new briefing paper on orphan medicinal products that looks into the 15 year application of the Orphan Regulation in Europe. The launch of the paper coincides with the European Conference on Rare Diseases and Orphan Products, which is being held in the UK for the first time and brings together stakeholders from across the rare disease community.
The briefing paper explores the regulatory framework and the setting up of the Committee for Orphan Medicinal Products within the European Medicines Agency, and raises awareness around the latest 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.
BIA Chief Executive Steve Bates said: '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,' he added.
Orphan designation allows companies to benefit from a number of incentives, including scientific advice on development and authorisation, access to the EU market under the centralised marketing authorisation procedure, 10 years of market exclusivity and SMEs can receive further assistance from the EMA’s SME Office.
To qualify for orphan designation, a medicine must meet certain criteria. Orphan medicines must be intended for the diagnosis, prevention or treatment of life-threatening or very serious conditions that affect no more than five in 10,000 people in the EU.
To date, the European Commission has authorised 124 orphan medicines for the benefit of patients suffering from rare diseases, more than 80% of which consist of new active substances. Some 25 orphan marketing authorisations are from SMEs as of January 2016. Furthermore, the European Commission has designated 1300 products as orphan medicinal products as of May 2016.
To download the briefing paper go to the BIA website. For the latest developments from the European Commission on Orphan Medicinal Products go to the European Commission website.