Combining Orphan Drug and Breakthrough Designations: Opportunities and Challenges
Combining Orphan Drug and Breakthrough Designations: Opportunities and Challenges Maximizing Rare Disease Drug Development with Orphan and Breakthrough Designations Introduction: Strategic Use of Dual Designations Rare disease therapies face considerable scientific and logistical challenges due to limited patient populations, fragmented natural history data, and evolving regulatory expectations. Fortunately, the FDA and other global regulators offer…
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