FDA proposes tighter norms to grant orphan status for children's treatments

Dec 19 (Reuters) - The U.S. Food and Drug Administration on Tuesday sought to tighten the norms for granting orphan drug status to treatments for children in a bid to clamp down on companies trying to use the special status to bypass pediatric drug trials.

The FDA said orphan status would only be granted if the disease in the pediatric population includes an 'orphan' subset and the drug meets all the other criteria for orphan designation. (

The companies would need to show the disease in children is different from that in adults for the treatment to get the designation, the regulator said in the draft guidance issued on Tuesday, as part of its Orphan Drug Modernization Plan.

If these conditions are not met, the FDA said it would not grant orphan status even if the treatment is being developed to treat a disease affecting fewer than 200,000 children.

The U.S. regulator encourages drugmakers to develop treatments for rare diseases that affect 200,000 patients or fewer, by granting drugs “orphan” status.

The designation provides drug developers with special development and market exclusivity incentives. (Reporting by Tamara Mathias in Bengaluru; Editing by Arun Koyyur)