Real-World Evidence Informs Rare Disease Care, Guides New Rx

Originally published by our sister publication Specialty Pharmacy ContinuumBy Marcus A. BanksWith fewer than 10% of the world’s roughly 7,000 rare diseases having an approved treatment, the fastest path to new therapies may not run through traditional clinical trials at all—but through the real-world evidence (RWE) from patients already taking these drugs.RWE typically is derived from data collected outside controlled trials (e.g., routine care, registries, and insurance claims). Tif