Congress has already granted the FDA flexibility in evaluating therapies for rare diseases, including the use of real-world evidence and natural history data when traditional large-scale trials are not feasible. The question before the FDA now is not if those tools can be applied – they can – but if the agency has the courage to use them before more patients lose their autonomy, and ultimately, their lives, to rare disease.
The post The Lethal Cost of Regulatory Perfection in Rare Disease appeared first on MedCity News.
Ransomware incident responder gave info to BlackCat cybercriminals during negotiations, DOJ alleges
It’s not like threat actors weren’t telling some of us about rogue negotiators. They were. Now I wonder how many other journalists also disbelieved the
