This annual report provides an update on FDA's work to support medical countermeasure-related public health preparedness and response efforts.
“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.
Medable Inc., the leading technology provider for modern clinical trials, today announced that the Partnership for Advancing Clinical Trials (PACT) consortium in conjunction with the Tufts Center for the Study of Drug Development (CSDD) has produced compelling results from a new study.
Soon there could be nutrition labels on the front of food packages at the grocery store — not just on the back or side. The Food and Drug Administration said the goal of potentially implementing this new label on prepackaged goods is to “help ensure that consumers in the U.S. have greater access to nutrition information they can use to identify healthier food choices.”
The Food and Drug Administration is expected to propose a change to prepackaged food sold in America: a requirement that the front of the packages display key nutrient information in addition to the nutrition label that’s already on the back.
Regular engagement with patients participating in registries is key to ensuring they continue to participate in long-term follow-up about a disease’s progression or the effectiveness of a treatment, leaders of patient advocate groups who have created effective registries said this week. The advice comes as FDA officials struggle with how to keep patients in registries and are looking at possible incentives.
FDA’s total request of $7.2bn for FY 2025, an increase of $341m from the FY 2024 numbers, “will allow the agency to continue to leverage new and emerging technologies,” and unite the Human Foods Program under “a single line of authority,” FDA Commissioner Robert Califf said during the Agriculture, Rural Development, Food and Drug Administration subcommittee budget request hearing on April 18.
This news that Reagan-Udall Foundation for the FDA and the ASH Research Collaborative have established a new Real-World-Evidence Consortium for Sickle Cell Disease which aims to "develop consensus recommendations on clinical outcomes important to treating people with SCD and apply those standards to real-world data sets."
