1. Pink Sheet
  2. >>Rare Diseases

How To Conduct A Multi-Company Trial For Rare Pediatric Diseases

 
• By Brenda Sandburg

US FDA draft guidance suggests efficacy endpoints for testing multiple drug products in the same clinical trials; advances an FDA-EMA joint proposal promoting innovative approaches for development of Gaucher disease medicines.

children's soft toy teddy bear with pills takes the temperature of a mercury glass thermometer. child' disease.

The US FDA is casting a spotlight on a new approach to the development of drugs for rare pediatric diseases in which companies would test multiple product candidates in the same clinical trial.

FDA and the European Medicines Agency suggested this course of action in a joint proposal to promote the use of...

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

Indegene Exec On DTP Distribution As A ‘Tactical Solution’, Feasibility Of MFN Model

 
• By Anju Ghangurde

Senior Indegene executive William Lobb talks about complexities of the direct-to-patient distribution model in the US, including potentially introducing new intermediaries into the system. Questions on MFN's feasibility and why it may be “catastrophic” to biopharma were also part of the discussion

Best Laid Plans: A Chronology Of Post-Pandemic COVID-19 Vaccines

 
• By Bridget Silverman

Quick responses to late-breaking changes to US FDA COVID-19 vaccine policy kept Novavax's Nuvaxovid and Moderna's mNexspike close to expected approval timelines despite revised indications and new post-marketing trial commitments.

Last Minute Course Shifts, Focus On Unknowns: What Sponsors Should Learn From Prasad’s Interventions

 
• By Sarah Karlin-Smith

The once and current CBER director’s justification for his COVID-19 decision stands as a warning of the uncertainty sponsors, particularly those in the vaccine space, may now face at FDA.