Registration opens for joint EMA/FDA/MHLW-PMDA orphan medicinal product workshop

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19/12/2013

Registration opens for joint EMA/FDA/MHLW-PMDA orphan medicinal product workshop

Registration is open for the third workshop on orphan product designation and grants jointly organised by the European Medicines Agency (EMA), the US Food and Drug Administration (FDA) and, for the first time, the Japanese Ministry of Health, Labour and Welfare (MHLW) and Pharmaceuticals and Medical Devices Agency (PMDA).

The event will be held on Monday 10 March 2014 at the EMA, London, United Kingdom.

The aim of the workshop is to provide information to companies as well as academics on the EMA, FDA and MHLW-PMDA systems for orphan medicine designation and on the grant programmes available for the development of orphan medicines. These programmes aim to promote the development of new medicines for the treatment of rare diseases.

The workshop’s morning session will provide information on these different systems and will be broadcast live on the EMA website.

The afternoon session will be dedicated to one-to-one meetings with EMA, FDA and MHLW-PMDA staff, during which participants will have the opportunity to present a draft application for orphan designation and discuss issues with the three agencies concerning 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 the structure and content of their application to meet the EMA, FDA and MHLW-PMDA application requirements.

There is no registration fee for attending the workshop. Participants need to register online for both morning and afternoon sessions. Registration is limited to 150 participants for the morning session.

The workshop is being conducted in partnership with the European Organisation for Rare Diseases (EURORDIS), Genetic Alliance and the National Organization for Rare Disorders (NORD).

International cooperation for orphan medicines

This is the third time that this workshop has been held and the first time that MHLW-PMDA have been involved as organisers. The previous workshops were held in London in November 2011 and in Bethesda, Maryland, USA, in October 2012. The event is organised to stimulate active international cooperation in the area of orphan medicines.

The EMA and the FDA started working together in 2008 to encourage applications for orphan designation to be submitted in parallel to the two agencies. The parallel submission of orphan designation applications has been very successful, with 62% of dossiers submitted in parallel to the EMA and the FDA in 2012.

The two agencies also provide parallel scientific advice to sponsors during the development phase of their products.

Collaboration between the EMA and the MHLW-PMDA in the field of orphan medicines was initiated in 2012. The agencies currently exchange information on the legal grounds, regulatory systems and operational aspects of orphan medicine development.

Related information

Contact point:

Contact at EMA: orphandrugs@ema.europa.eu

Contact at FDA: Eleanor Dixon-Terry at Eleanor.Dixon-Terry@fda.hhs.gov

Contact at MHLW/PMDA: Akiko Nitta at nitta-akiko@pmda.go.jp