Rare Diseases: US FDA Gives More Guidance On Premarket Safety Evaluation, Historical Controls
Agency specifies several approaches to augment safety assessment in a new draft guidance, which also encourages participation by patients and their advocates in the drug development process.
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FDA wants another trial in ultra-orphan Barth syndrome, but could not identify a feasible design – so Stealth submitted the NDA anyways, based on a retrospective natural history control trial
Consolidated Appropriations Act gives FDA $5.58bn overall, 8.7% more than allocated in FY 2018. FDA received $268.8m in additional budget authority and $178.3m in additional user fee funds. FY 2019 funding would have lapsed Feb. 16 and another government shutdown would have started if President Trump didn't sign the bill on Feb. 15.
Agency would receive more than $268m in additional dollars for medical product and food safety as part of substantial FY 2019 increase.