Last month, the Food and Drug Administration (FDA) announced a new Patient-Focused Drug Development Initiative to gather more information from patients about their individual disease burden and perspectives on treatment benefits and risks. FDA plans to use this information in its regulation of drug products. In this initiative, FDA will hold 20 disease-specific public meetings to discuss the range of severity for the disease, themeasures of benefit that matter most to patients and the adequacy of the existing treatment options. Bleeding disorders were included in the preliminary list of diseases. And NHF, joined by HFA and COTT, submitted written comments asking that FDA include bleeding disorders on the final list of diseases. The letter is available here.
NHF will continue to monitor the progress of this initiative, and will update the community as more information becomes available. For more information on the initiative, click here.
