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Opinion

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.