EMA and FDA encourage use of innovative approaches in the development of medicines for Gaucher disease

Approach aims to facilitate development of medicines for rare paediatric diseases in general

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 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. The strategy document encourages medicine developers to make better use of:

• extrapolation of available clinical data, including through appropriate modelling and simulation techniques, to predict how a medicine may work in children and adolescents on the basis of studies conducted in adults or other paediatric populations;
• the possibility to test the safety and efficacy of medicines developed by different companies in one single trial, so-called multi-arm, multi-company clinical trials. As the same control arm is used to compare more than one medicine under evaluation, this approach facilitates the clinical testing of medicines while reducing the total number of children included in trials.

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.

The FDA will be publishing this strategy paper in a different format in the next few months.

Source: EMA

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