Don’t Call It A Center Of Excellence: Accelerating Rare Disease Cures Program Launched By CDER
‘Umbrella’ program is intended to ensure all rare disease and related programs at CDER are working together, which seems similar to the rare disease center of excellence concept that stakeholders want to coordinate activities across the FDA.
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Ultra-Rare Orphan Drugs: Advocates Begin Laying Groundwork For New Incentive, Special Pathway
Rare disease advocates push for National Academies of Sciences, Engineering and Medicine study on how or whether ultra-rare should be defined, which could create legislative pressure. In an interview, Ultragenyx CEO Emil Kakkis suggests patient thresholds, accelerated approval triggers.
Does The Orphan Drug Act Need A Tune-Up?
A better description of the US FDA’s regulatory flexibility may be necessary, but tinkering with the foundational law for rare disease drug development could open the door for more substantial and unwanted changes.
Product Differences Necessitate Some US FDA Inconsistency In Rare Diseases, Center Directors Say
Development standards for a one-time gene therapy should be different from a drug taken regularly, CBER Director Peter Marks says.