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US FDA proposes guidance to speed up drug development for rare disorders

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US FDA proposes guidance to speed up drug development for rare disorders

The U.S. Food and Drug Administration issued a draft guidance to simplify drug developing procedure for rare pediatric disorders.

The regulator is hoping the proposal would help speed up drug development in clinical trials by reducing the number of patients required on placebo and allowing companies to collaborate and test multiple drug products in a single study.

The proposal was developed in collaboration with the European Medicines Agency using Gaucher's as a disease model and could be extended to other rare pediatric disorders.

Gaucher's disease is a genetic disorder, which is caused by a deficiency of an enzyme required to break down certain fats, leading to enlarged liver and spleen.

The draft guidance is available for comment for about two months, after which the health regulator will release its final determinations.