The European Medicines Agency (EMA)'s Committee for Orphan Medicinal Products (COMP) says it will integrate the views of patients, expand international cooperation and collaborate with health technology assessment (HTA) bodies this year, aiming for a better understanding of orphan drug designation.

The European Union (EU) orphan medicinal product regulation was introduced 12 years ago to incentivise the development of medicines for rare diseases. The number of designations has been increasing steadily, with 107 in 2011 and 148 last year, and more than 150 are expected this year, says EMA. 

"In line with this trend, in 2012, 19 applications for marketing authorisation concerned designated orphan medicines, compared with 14 in 2011," it says.

Discussing plans to expand its already-active international cooperation this year, COMP says that, following the collaboration begun with the US Food and Drug Administration (FDA) in 2008, last year 62% of applications for orphan drug designations were submitted in parallel in the EU and US. COMP also began to collaborate with the Japanese regulatory authorities in 2012, and is anticipating a closer collaboration with Health Canada this year.

EMA has started working closely with EUnetHTA (an organisation that focuses on scientific cooperation in HTA in Europe) towards a better understanding on orphan designation, marketing authorisation of orphan drugs and initiatives by EU national authorities on availability of designated orphan products.  The dialogue “will explore ways of sharing information for the common benefit of patients affected by rare diseases and the financial sustainability of healthcare systems," it says.

Also, to better serve and address patients' needs, COMP says it will "proactively" request contributions from expert patients in areas where their advice is key, such as contribution to patient care, improvement to quality of life, assessment of significant benefit and the development of medicines in specific indications.

COMP plans to work to increase patient organisations' involvement and integrate their views in areas such as treatment modality and new formulations and, in particular, to strengthen its interactions with disease-specific patient groups and the European Organisation for Rare Diseases (EURORDIS), also developing new interactions with international patient organisations.

- EU orphan designation can be granted at any stage of the development process to medicines intended for diagnosis, prevention or treatment of life-threatening or very serious conditions that affect no more than five in 10,000 people in the EU or for which development costs would not be covered by the marketing return without incentives. If there are already methods of treatment, prevention or diagnosis for the rare disease, orphan designation is possible only if the medicine provides patients with significant benefit.

Sponsors of orphan medicines benefit from a range of incentives, including 10 years' market exclusivity, fee reductions for some EMA services, protocol assistance and access to the EU centralised authorisation procedure.