FDA Launches Rare Disease Framework Initiative to Systematize Regulatory Flexibility

FDA has launched a rare disease framework initiative, led by the Rare Disease Innovation Hub together with the Office of the Commissioner, CBER and CDER, with a public meeting scheduled for October 28 to gather stakeholder input. The initiative aims to promote more consistent, certain and innovative approaches to rare disease drug development while maintaining rigorous standards. Only about 5% of rare diseases currently have FDA-approved treatments.