At her confirmation hearing, Dr. Heidi Overton, President Donald Trump’s nominee to lead the Food and Drug Administration, called rare-disease treatments a “key priority.” The agency is also shaping PDUFA VIII, a five-year agreement that will determine FDA funding and its approach to reviewing new drugs. This is a rare opportunity and a serious responsibility.

To make that priority a reality, Overton must strengthen FDA capacity and provide clear, efficient regulatory pathways that can help save lives.

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