Real-World Evidence in Regulatory Submissions for Rare Diseases
Real-World Evidence in Regulatory Submissions for Rare Diseases Leveraging Real-World Evidence in Rare Disease Regulatory Submissions Introduction: Why Real-World Evidence Matters in Rare Disease Approval Traditional randomized controlled trials (RCTs) are often impractical in rare disease drug development due to small patient populations, genetic heterogeneity, and ethical constraints. In such contexts, real-world evidence (RWE)—clinical data…
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