Rare Pediatric Disease Priority Review Vouchers - Guidance for Industry

FDA has published its draft guidance on rare pediatric disease priority review.

This guidance is intended to assist developers of rare pediatric disease products in assessing whether their product may be eligible for rare pediatric disease designation and a rare pediatric disease priority review voucher. It also clarifies the process for requesting such designations and vouchers, sponsor responsibilities upon approval of a rare pediatric disease product application, and the parameters for using and transferring a rare pediatric disease priority review voucher.

Regulations on vouchers are quite urgently needed, even though they give an incentive to companies developing urgently needed drugs in niche indications. But they also do raise the question of the right incentive from an ethical perspective.

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