The Evolving Market Access Landscape for Orphan Drugs

The historical access environment for orphan drugs was based on a few critical factors, namely that these products addressed very small, high unmet need populations that led to low overall budgetary impact despite high per-capita treatment costs. However, recent developments in the U.S. and Europe suggest payers are becoming more aggressive in scrutinizing price and implementing restrictions to patient access that have traditionally been reserved for competitive, high budget impact drug classes.