ResearchPUBMEDMay 1
Analysis of Real-World Data Utilization in the Orphan Drug Approval Process: Focusing on New Drug Marketing Applications Submitted to the FDA.
Researchers looked at how the FDA approves medicines for rare diseases from 2020 to 2024. They found that 129 rare disease drugs were approved during this time, and 25 of them (about 19%) used real-world data—information collected from patients outside of traditional clinical trials. Real-world data is becoming more important for rare diseases because it's often impossible to run large traditional studies when so few people have a disease.
WHY IT MATTERSIf you have a rare disease, this research shows the FDA is increasingly accepting real-world evidence from patient experiences and medical records to approve new treatments, which could mean faster access to medicines when traditional large-scale trials aren't feasible.
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