The Food and Drug Administration (FDA, the Agency, or we) is announcing a public meeting and an opportunity for public comment on “Patient-Focused Drug Development for Hereditary Angioedema.” Patient-Focused Drug Development is part of FDA's performance commitment under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V). The public meeting is intended to allow FDA to obtain patients' perspectives on the impact of hereditary angioedema (HAE) on daily life. FDA also is seeking patients' views on treatment approaches for HAE. For each topic, a brief patient/caregiver panel discussion will begin the dialogue. This will be followed by a facilitated discussion, inviting comments from other patient and caregiver participants. In addition to input generated through this public meeting, FDA is interested in receiving patient input addressing these questions through electronic or written comments.
HAE is a rare genetic disorder that affects less than 200,000 individuals in the United States. It is associated with episodic recurrent attacks of swelling of the body caused by abnormalities in a protein called C1-Esterase Inhibitor. Most cases occur because there is either not enough of the protein or because the protein does not work normally to help prevent swelling of the body.
More information, including suggested questions for comment, is available via the Federal Register notice.