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BIO Comments on FDA Draft Guidance on Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases

February 16, 2018

BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases.

The Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) provide new pediatric information in labeling for over 600 products.

BIO appreciates the FDA’s efforts to clarify and provide notice of a policy change intended to enable the FDA to apply the Orphan Drug Act (ODA) and PREA to non-rare adult indications corresponding to orphan-designated pediatric subpopulations. However, BIO’s comments detail general concerns with the guidance and specific line edits to clarify and improve the guidance.

 

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BIO Letter Clarification Of Orphan Designation Of Drugs And Biologics For Pedaitric Subpopulations Of Common Diseases FDA-2017-D-6380
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