Posted by Michael Wonder on 03 Jul 2017
EMA and FDA encourage use of innovative approaches in the development of medicines for Gaucher disease
3 July 2017 - Approach aims to facilitate development of medicines for rare paediatric diseases in general.
The EMA) and the United States FDA have developed a joint proposal to promote the use of innovative approaches in the development of medicines for Gaucher disease, which can apply to rare diseases in children in general.
This strategic collaborative approach from EMA and the FDA discusses possible ways to enhance the efficiency of medicine development in Gaucher disease, a rare lysosomal storage disorder, which is used as a model to reflect on recent progress made in the area of data extrapolation.
