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EMA and FDA calls for innovative approaches for Gaucher disease medicines

The regulatory agencies also urge a similar method for all paediatric diseases

FDA EMARegulators in the US and Europe have outlined a series of proposals, including multi-arm clinical trials, in a bid to improve research into new, innovative treatments for the rare genetic disorder Gaucher disease. 

The joint proposal from the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) also urges the use of innovative methods in developing medicines for diseases in children in general, and encourages medicine developers to extend its clinical data through modelling and simulation techniques.

The agencies are also encouraging the use of multi-arm clinical trials to test the safety and efficacy of medicines developed by different companies in one single trial.

The 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,” the EMA said in a statement.

In addition, the EMA is finalising a reflection paper - expected to be published in the fourth quarter of 2017 - that outlines an efficient approach to allow reliable extrapolation of data to support medicine authorisation.

Article by
Gemma Jones

4th July 2017

From: Regulatory

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