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Statement on U.S. FDA Report Highlighting Gaps in Pediatric Research

Treatment Action Group (TAG) welcomes a report released last week by the U.S. Food and Drug Administration (FDA) that highlights urgent gaps in pediatric research. This report confirms that we are missing critical information necessary to treat children affected by orphan diseases. Orphan diseases affect fewer than 200,000 people per year in the United States, and children are highly vulnerable to many of them. These knowledge gaps are a result of specific provisions within the Orphan Drug Act that exempt product developers from conducting pediatric studies otherwise required under the Pediatric Research Equity Act (PREA).

A policy brief jointly developed by TAG and the Elizabeth Glaser Pediatric AIDS Foundation (EGPAF), Ensuring Treatment for Children with Orphan Diseases: Ending Exemptions from the Pediatric Research Equity Act (PREA), describes how the pediatric research exemption for orphan drugs affects children with tuberculosis (TB). The brief proposes policy solutions for ensuring that the benefits of research and development extend to children in need of treatment for neglected or orphan diseases. We look forward to engaging with the FDA and other partners to advance solutions and appropriate action to address the exemption, effectively close gaps in pediatric research, and ensure the availability of current and future therapeutics for children to treat deadly diseases like TB.

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