Redefining Statistical Significance For Rare Disease Research

 
• By Christian Schneider

Biopharma Excellence recently convened three experts in the world of rare disease in a roundtable to discuss the emerging approaches to data collection when patient populations are very small. Their discussion has been summarized for In Vivo.    

Pencil
• Source: Shutterstock

A recent panel discussion hosted by Biopharma Excellence featured experts in biotech and the evolving regulatory environment to talk about the challenge of achieving statistical significance with novel and highly targeted therapies, especially for rare/ultra-rare diseases.

Daniel O’Connor of the UK’s MHRA noted that the challenge of achieving statistical significance when target populations are tiny is...

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on In Vivo 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 Market Intelligence

The Goldilocks Isotope: Perspective Therapeutics’ ‘Just Right’ Alpha Radiotherapeutic

 
• By David Wild

Thijs Spoor's bet on lead-212 is paying off as Perspective Therapeutics advances three clinical programs with promising early efficacy signals and a comprehensive manufacturing strategy.

Pharma’s Dance With Trump Risks Distracting From Longer-Term Threats

 
• By Melanie Senior

Industry is trying to side-step President Trump’s tariff- and price-curbing policy proposals. It may do better facing up to enduring R&D productivity and drug affordability challenges.

Tackling Fraud In The UK: Pharma, Are You Ready?

 
• By Trevor Francis

UK pharma faces a critical turning point as the Economic Crime and Corporate Transparency Act introduces the "Failure to Prevent Fraud" offense in September 2025, requiring companies to transform their approach to fraud prevention or face severe consequences.

Finding The Fire: AltruBio’s Novel Approach To Autoimmune Disease

 
• By David Wild

CEO Judy Chou brings big pharma experience to tackle chronic inflammation with the company's first-in-class PSGL-1 targeting therapy.

More from In Vivo

Deals Shaping The Industry, May 2025

 
• By In Vivo Team

An interactive look at pharma, medtech and diagnostics deals made during May 2025. Data courtesy of Biomedtracker.

Podcast: Flagship’s Vision To Predict And Prevent Illness, With Raj Panjabi

 
• By David Wild

Flagship Pioneering senior partner Raj Panjabi discusses shifting health care from reactive treatment to AI-powered prediction and prevention of disease before symptoms emerge.

Rising Leaders 2025: Namrata Saroj On The Importance Of Authenticity In Ophthalmology

 
• By Sushmita Panda

Namrata Saroj, chief business officer of Ocular Therapeutix, is highly respected in the retina community for her contributions to drug development. She talked to In Vivo about her journey in ophthalmology, leadership philosophy and the importance of authenticity in a close-knit specialty.