1. In Vivo
  2. >>Market Intelligence

Fresh Recommendations For Rare Disease Trial Sponsors

 
• By Daniel Chancellor

Best practices to help sponsors design patient-centric studies, build trial awareness among important physician and health care professional groups, leverage advocacy groups to speed up patient recruitment, and ultimately gain a competitive edge in rare disease trial strategies.  

Rare disease awareness
Balloons released for rare disease day • Source: Alamy

Approved drugs are available for just 5% of the approximately 7,000 rare diseases identified, many of which are life threatening or limiting. The pharmaceutical industry is making inroads through R&D, despite the unique challenges facing clinical trials for rare diseases, not to mention often navigating without regulatory precedents and an uncertain commercial outlook. Progress can therefore feel slow despite the priority that many drug companies place on these patients and their unmet needs.

Rare diseases are so-called because of the low number of prevalent patients, with long-standing definitions being fewer than 200,000 people...

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

Podcast: Brain+ CEO Discusses “Groundbreaking” Potential Of CST Assistant For Dementia Patients

 
• By Anabel Costa-Ferreira

Devika Wood, CEO of Brain+, explains the importance of developing health tech solutions for dementia and the growing need to both raise awareness and improve overall access to nondrug interventions like CST.

Behind The Buyout: Dispatches From The Dealmaking Table

 
• By Jo Shorthouse

In a challenging funding environment for biopharma, strategic dealmaking has become a critical growth engine. In Vivo explores what it truly takes to navigate high-stakes acquisitions and partnerships, drawing on insights from seasoned industry leaders.

Crisis Or Opportunity? US MFN Policy Could Test Japan’s Appetite For Reforms

 
• By Lisa Takagi

While the adoption of most favored nation drug pricing in the US stands to affect Japanese biopharma firms now heavily reliant on this market, it might also present an opportunity for pricing and policy reforms at home.