Gaucher disease drug development urged by EU and US regulators

An approach that aims to facilitate development of rare pediatric diseases in general, and as a starting point Gaucher disease in particular, has been outlined by regulatory bodies in the European Union and the USA.

The European Medicines Agency and the US Food and Drug Administration 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 the 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. The strategy document encourages medicine developers to make better use of: