EMA Reports Steady Increase in Orphan Drug Designations

The European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) have confirmed that they will incorporate the views of patients, increase international cooperation and collaborate with health technology assessment (HTA) bodies, with the aim of gaining a better understanding for orphan drug designation.

The European Union (EU) orphan medicinal product regulation was started 12 years ago to encourage the development of treatments for rare diseases.  Since then, the number has been growing steadily, with 107 in 2011 and 148 last year, and more than 150 expected in 2013.

“In line with this trend, in 2012, 19 applications for marketing authorisation concerned designated orphan medicines, compared with 14 in 2011,” EMA confirmed.

Discussing plans to increase their already-active international cooperation, the committee commented that, following the collaboration with the US Food and Drug Administration (FDA) in 2008, last year 62% of applications for orphan drug designations were submitted in parallel in both the EU and US.

The Committee for Orphan Medicinal Products also started to work in partnership with the Japanese regulatory authorities in 2012, and the COMP are expecting a closer partnership with Health Canada this year.

EMA has also started working closely with the EUnetHTA (an organisation that concentrates on scientific cooperation in HTA in Europe) towards an improved understanding on orphan designation, marketing authorisation of orphan drugs and initiatives by EU national authorities on availability of designated orphan products.   The co-operation “will explore ways of sharing information for the common benefit of patients affected by rare diseases and the financial sustainability of healthcare systems,” they said.

Also, to serve and address patients’ needs better, COMP have revealed that they will “proactively” request contributions from expert patients in capacities 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.

The committee plans to work to increase patient organisations’ involvement and incorporate their views in areas such as treatment modality and new formulations and to improve the interactions with disease-specific patient groups and the European Organisation for Rare Diseases.

Sponsors of orphan medicines benefit from a variety of incentives, including 10 years’ market exclusivity, fee reductions for some of the European Medicines Agency’s services, protocol assistance and access to the European Union’s centralised authorisation procedure.


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