Joint European Medicines Agency (EMA), US Food and Drug Administration (FDA), and Japanese Ministry of Health, Labour and Welfare (MHLW) and Pharmaceuticals and Medical Devices Agency (PMDA) orphan medicinal product workshop
The aim of the workshop is to provide information to companies, as well as to academics, on the EMA, FDA and MHLW-PMDA systems for orphan medicinal product designation as well as on the grant programmes for development of orphan medicinal products that are available. These programmes aim to promote the development of new medicines for the treatment of rare diseases. This workshop is now fully booked. However, the plenary session will be publicly broadcasted on the EMA website (prior registration not required) - please click on the 'multimedia' tab to watch live on the day. Following the workshop, the recording and presentation slides will also be made available.
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Agenda - Joint EMA-FDA-MHLW-PMDA orphan medicinal product workshop (PDF/124.02 KB)
First published: 19/12/2013
Last updated: 19/12/2013 -
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Presentation - Orphan designation system in Japan (Ministry of Health, Labour and Welfare) (PDF/255.29 KB)
First published: 28/03/2014
Last updated: 28/03/2014 -
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Presentation - FDA Orphan Drug Designation 101 (James H. Reese) (PDF/2.24 MB)
First published: 28/03/2014
Last updated: 28/03/2014 -
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Presentation - Orphan drug designation in the European Union (Stiina Aarum) (PDF/1.47 MB)
First published: 28/03/2014
Last updated: 28/03/2014 -
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Presentation - Grant frameworks: Japan (Hirofumi Kusunoki) (PDF/253.41 KB)
First published: 28/03/2014
Last updated: 28/03/2014 -
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Presentation - FDA orphan products grants program overview (Erica K McNeilly) (PDF/357.4 KB)
First published: 28/03/2014
Last updated: 28/03/2014 -
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Presentation - IRDiRC: EU Research funding for rare disease research (Irene Norstedt) (PDF/1.89 MB)
First published: 28/03/2014
Last updated: 28/03/2014 -
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Presentation - Incentives and regulatory considerations: Situation in Europe (Segundo Mariz) (PDF/153.17 KB)
First published: 28/03/2014
Last updated: 28/03/2014 -
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Presentation - FDA incentives (John D. Milto) (PDF/225.73 KB)
First published: 28/03/2014
Last updated: 28/03/2014 -
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Presentation - Incentives and regulatory considerations in orphan drug/medical device development - Situation in Japan (Hiroshi Takeda) (PDF/543.11 KB)
First published: 28/03/2014
Last updated: 28/03/2014 -
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Presentation - European Orphan Designation: Legal basis (Agnes Mathieu) (PDF/114 KB)
First published: 28/03/2014
Last updated: 28/03/2014