1. In Vivo
  2. >>Market Intelligence

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

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.

Late-Stage GLP-1 Drug Trials Outside The Cardiometabolic Space

 
• By David Wild

A look at Novo Nordisk, Eli Lilly and other companies' late-stage clinical studies of GLP-1 drugs in indications ranging from neurodegeneration to oncology, and alcoholic liver disease to autoimmune conditions.

UK Bets On Regulatory Innovation As Competitive Advantage In Synthetic Biology

 
• By David Wild

The UK government has prioritized synthetic biology and created a "concierge service" for biotechs just as the US cuts science funding.

More from In Vivo

AI In Health Delivery: Patients Most Confident When HCPs Are In Charge

 
• By Ashley Yeo

Annual survey of patients and professionals shows how attitudes to health system transformation are evolving and what stakeholders are demanding as acceptance of AI tools accelerates.

Medtech Innovators Court London-Based Investors At BioWales 2025

 
• By Ashley Yeo

BioWales in London 2025 showcased the efforts healthtech innovators are making to meet investors on their own turf, illustrating changing attitudes and evolving needs.

Rising Leaders 2025: Metsera’s Whit Bernard’s Musical Path To Biotech Leadership

 
• By David Wild

Metsera CEO Whit Bernard applies an unconventional leadership philosophy to develop next-generation obesity therapeutics, including monthly GLP-1 injections and oral peptides.