Gaucher disease: a strategic collaborative approach from the European Medicines Agency and Food and Drug Administration
Table of contents
This document aims to increase the chances of rapid and smooth agreement of the paediatric investigation plan (EMA)/pediatric study plan (FDA). It also discusses the possibility of a multi-arm, multi-company clinical trial for the treatment of Gaucher disease, as one approach to address the feasibility of developing multiple products for a rare disease in a limited timeframe.
Keywords: Gaucher disease, lysosomal storage disorder, enzyme replacement therapy, substrate-reduction therapy
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Gaucher disease: a strategic collaborative approach from the European Medicines Agency and Food and Drug Administration (PDF/278.83 KB)
Adopted
First published: 30/06/2017
Last updated: 30/06/2017
EMA/237265/2017 -
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Overview of comments received on 'Gaucher disease: a strategic collaborative approach from the European Medicines Agency and Food and Drug Administration' (EMA/44410/2014) (PDF/335.48 KB)
First published: 30/06/2017
Last updated: 30/06/2017
EMA/752905/2016 -
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Gaucher disease: A strategic collaborative approach from European Medicines Agency and Food and Drug Administration (PDF/142.25 KB)
First published: 14/05/2014
Last updated: 14/05/2014
EMA/44410/2014