Contribute Try STAT+ Today

As regulators approve a growing number of orphan drugs, prices for these medicines, which treat small groups of patients with rare diseases, have been climbing ever higher. And a new study finds that, while the drugs may offer larger health gains than other medications, they are not nearly as cost-effective.

To wit, the researchers found that orphan drugs were five times more likely to offer a health benefit than other medicines approved by the Food and Drug Administration. But the median costs were substantially higher — $47,650 versus $2,870. Moreover, payers would have to spend 2.7 times more money on an orphan drug than another medicine in order for a patient to gain an extra year of perfect health.

Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!

GET STARTED

What is it?

STAT+ is STAT's premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond.

What's included?

  • Daily reporting and analysis
  • The most comprehensive industry coverage from a powerhouse team of reporters
  • Subscriber-only newsletters
  • Daily newsletters to brief you on the most important industry news of the day
  • STAT+ Conversations
  • Weekly opportunities to engage with our reporters and leading industry experts in live video conversations
  • Exclusive industry events
  • Premium access to subscriber-only networking events around the country
  • The best reporters in the industry
  • The most trusted and well-connected newsroom in the health care industry
  • And much more
  • Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr.