Registration opens for FDA/EMA orphan product designation and grant workshop

News 02/09/2013

Registration is open for the joint United States Food and Drug Administration (FDA) / European Medicines Agency (EMA) orphan product designation and grant workshop. The event will be held on Friday 4 October 2013 at the FDA White Oak Campus in Silver Spring, Maryland, USA.

The aim of the workshop is to provide information on the EMA and FDA programmes for orphan medicinal product designation as well as on the FDA programmes for orphan product grants and humanitarian use device designation to pharmaceutical, biotechnology and device companies, as well as to academics.

The information session will be held in the morning and will be webcast on the EMA and FDA websites.

The afternoon session will be dedicated to one-to-one meetings with both EMA (by teleconference) and FDA staff during which participants will have the opportunity to present to the two Agencies a draft application based on a medicine under development that holds promise for the treatment of a rare disease.

These one-to-one sessions are intended to help participants improve their applications to ensure that they fulfil the requirements of both the EMA and the FDA procedures and increase their chance of obtaining an orphan designation for their medicine in both the EU and the USA.

There is no registration fee for attending the workshop; however registration is required. Participants can register for both morning and afternoon sessions on the FDA website (LINK).

Registration is limited to 240 participants for the morning session; up to 50 teams (formed of one to three participants) may register for the one-to-one sessions.

The FDA/EMA orphan product designation and grant workshop is conducted in partnership with the European Organisation for Rare Disease (EURORDIS), Genetic Alliance, and the National Organization for Rare Disorders (NORD).

International cooperation for orphan medicines

This is the third year that this workshop has been held. This event is organised as part of a broader initiative to expand active international cooperation in the area of orphan medicines.

The EMA and the FDA started collaboration in 2008, allowing applications for orphan medicine designations to be submitted in parallel to the two agencies. The parallel submission process helps rationalise the development of orphan medicines by facilitating access to parallel scientific advice (protocol assistance) from the two regulatory authorities.

Based on the success of this collaboration, which led to a parallel submission for 62% of the dossiers submitted to the EMA in 2012, last year the EMA began to collaborate with the Japanese regulatory authorities. On 19September 2013, the EMA will organise a meeting at the Embassy of Japan in London to explain the European Union orphan-medicinal-product regulation to Japanese companies based in Europe.

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