EMA and FDA encourage innovative drug development for Gaucher disease

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The European Medicines Agency (EMA) and the United States Food and Drug Administration (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 approach 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 approach set out by this joint proposal aims to reduce the number of patients needed for clinical trials, meaning overall less burden on children and their families, while maintaining high quality standards for medicine development.

Medicine developers who wish to apply these innovative approaches in their development plan are advised to seek scientific advice. They can approach EMA or the FDA separately, or request parallel scientific advice from the two regulatory authorities if they wish.

In addition, EMA is finalising a reflection paper which outlines a systematic approach to scientifically sound and reliable extrapolation of data to support medicine authorisation. The paper, which is expected to be published the fourth quarter of 2017, will complement the approach published today.

The strategic collaborative approach from EMA and the FDA published today is the result of extensive collaborative work with various groups of stakeholders, including patients and healthcare professionals.