Gaucher disease: a strategic collaborative approach from the European Medicines Agency and Food and Drug Administration - Scientific guideline
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