FDA Proposes Drug Development Guidance for Rare Pediatric Diseases

December 6, 2017

By Reuters Staff

(Reuters) - The U.S. Food and Drug Administration issued a draft guidance on Wednesday to simplify the procedure of developing drugs for rare pediatric disorders, such as Gaucher's disease, by eliminating the need for certain trials and minimizing patient enrollment.

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. http://bit.ly/2iwQt3i

The FDA said the draft guidance was developed in collaboration with the European Medicines Agency, using Gaucher's as a disease model.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 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 guidance is available for comment for about two months, after which the FDA will release its final determinations.

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