Leveraging Real-World Evidence in Rare Disease Studies: Biostatistical Challenges and Solutions
In the realm of rare disease research, where patient populations often number in the mere hundreds globally, traditional randomized controlled trials (RCTs) face insurmountable hurdles like recruitment delays and ethical dilemmas in placebo use. Real-world evidence (RWE), derived from real-world data (RWD) sources such as electronic health records (EHRs), registries, and claims databases, offers a […]
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