In recent years, the number of rare diseases with multiple approved therapies has surged from seven rare diseases with more than three approved therapies in 2015 to 54 in 2025.
Given that average launch prices for orphan drugs are seven times higher than those of non-orphan drugs, and with an estimated 1 in 10 Americans affected by a rare disease, US payers have increasingly started to differentially manage these rare categories to lower costs.
As presented at the 2026 Asembia Specialty Pharmacy Summit in Las Vegas, CRA Life Sciences consultant Joe Black provides an overview of the key drivers compelling payers to differentially manage competitive rare disease categories, while examining select rare disease analogs to identify strategies manufacturers can use to optimize access in these increasingly competitive markets.
