FDA simplifies trials for rare paediatric disorders

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The US Food and Drug Administration has issued a draft guidance to simplify the procedure of developing drugs for rare paediatric disorders, such as Gaucher’s disease, by eliminating the need for certain trials and minimising patient enrolment.

Reuters Health reports that the health regulator said the new approach reduces the number of patients on placebo by allowing companies to collaborate and test multiple drug products in the same clinical trial.

The FDA said the draft guidance was developed in collaboration with the European Medicines Agency, using Gaucher’s as a disease model. It added that the proposal could be extended to other rare paediatric 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 report says the guidance is available for comment for about two months, after which the FDA will release its final determinations.

Reuters Health report
Draft guidance

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