Non-oncology orphan drug development: Productivity and probability of success

There are >6000 rare diseases (RDs), affecting >300 million people worldwide. Despite increases in orphan drug designations (ODDs), approved treatments remain scarce. This study analyzes the outcomes of non-oncology ODs designated by the FDA and European Medicines Agency (EMA) in 2017, providing a 7-8-year window to evaluate progression. This analysis of 292 ODDs examined therapeutic modalities, sponsor size, and funding factors influencing success, as defined by development phase transitions. Biologics showed higher approval rates whereas gene therapies displayed lower phase transition success. Smaller companies and academic groups demonstrated lower success rates. This research identifies drivers and gaps in OD development, emphasizing the need for sustained multistakeholder investment.