1. Pink Sheet

6,000, 7,000, 10,000? Why Counting Rare Diseases Is So Difficult

 
• By Derrick Gingery

FDA, NIH, sponsors and others use different figures, but some want everyone will settle on 10,000 for policy discussions, advocacy and research.

consensus
Advocates also want to verify the number of US patients with a rare disease and the number of rare diseases with pediatric onset. • Source: Shutterstock

Everyone in the pharmaceutical ecosystem agrees that developing treatments for rare diseases is important and challenging. There's a lot less consensus about how many rare diseases there are actually are, however. The US Food and Drug Administration website says there are more than 7,000 rare diseases affecting patients in the US. The National Institutes of Health said 7,000 last year, but now says 10,000.

Rare disease sponsors also disagree: Takeda Pharmaceutical Co. Ltd. says on its website that there are 6,000 known rare...

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Pink Sheet for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required
Start Free Trial

Already a subscriber?

More from Rare Diseases

More from Pink Sheet

EMA Explores Supporting Drug Development For High Unmet Need Disorder Scleroderma

 
• By Neena Brizmohun

While treatments for scleroderma are in development, none are approved for the condition that is associated with high morbidity and mortality. The European Medicines Agency says guidance on how to design development programs for the disorder is needed.

EMA Nod For First MASH Drug Rezdiffra, Cell Therapy Zemcelpro And 11 Others

 
• By Eliza Slawther

Madrigal Pharmaceuticals’ Rezdiffra is on track to become the first medicine approved in the EU for non-cirrhotic metabolic dysfunction-associated steatohepatitis after the European Medicines Agency recommended that it be granted conditional marketing authorization.

BIO Notebook: Policy Uncertainty Constraining Dealmaking

 
• By Mandy Jackson and Joseph Haas

Highlights from Day Four of the BIO International Convention include policy concerns helping constrain dealmaking, Novartis discussing its approach to partnering, and Generate looking for funding to move into Phase III.