This annual report provides an update on FDA's work to support medical countermeasure-related public health preparedness and response efforts.
Webinar
July 16, 2024 | 2-3pm (eastern)
The Awards will be presented in three categories:
“One of the things that we’ve noticed, not just in the pandemic, is this problem of missing race and ethnicity information,” explained Carla Rodriguez-Watson, Ph.D., MPH, Director of Research at the Reagan-Udall Foundation for the FDA. “If you do not have this information, where patients are being seen, where their information is being collected, then we have a limited view into what that population distribution of disease is.”
Ultragenyx Pharmaceutical's work to get the FDA to recognize a biomarker as a surrogate endpoint looks to have paid off. With the agency agreeing the biomarker is a reasonable surrogate endpoint, Ultragenyx is preparing to file for approval of its Sanfilippo syndrome gene therapy around the end of the year.
