Congress has granted the FDA flexibility in evaluating treatments for rare diseases, including using real-world evidence and natural history data when traditional large-scale trials are not feasible. The question now facing the FDA is not whether these tools can be applied — they can — but whether the agency has the courage to use them before more patients lose their autonomy and ultimately their lives due to rare diseases.
The post The Deadly Cost of Well-Regulated Rare Diseases appeared first on MedCity News.
