FDA doubles down on patient engagement to support rare disease research

Clinical Trials Arena March 4, 2024
Justine Ra

For Rare Disease Day, several panels organised by the FDA discussed the importance overcoming accessibility barriers during drug development.

The US Food and Drug Administration (FDA) has outlined patient empowerment and engagement as key objectives in its drive to promote rare disease drug development.

To mark this year’s Rare Disease Day, the agency hosted a webinar inviting FDA officials, patients, and advocacy leaders for a day of panels and discussions to delve into the key challenges in rare disease drug development.

The resultant discussions underscored the importance of overcoming the barrier of accessibility and engagement for patients living with rare diseases and discussed how the agency is prioritising rare disease drug development to address real-world patient needs.

Agency action

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