The Lethal Cost of Regulatory Perfection in Rare Disease

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.

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