The rise and rise of rare disease competition

For years, rare diseases were seen as a safe harbor for biotech and pharma: small patient populations, little competition, and robust pricing that payers accepted. That era is ending. The orphan drug market is becoming more crowded, contested, and expensive, as payers and regulators demand more evidence and large pharma reshapes the economics.

The shift could be profound. In 2024, more than half of all new medicines approved in the USA carried orphan designation. Launch prices are higher than ever, with a median of around $300,000 per year, yet the backdrop has changed.

Far from monopolies, companies now face rivals in the same indications, tougher pricing negotiations, and new policy frameworks that blunt the once-automatic advantages of orphan status.