1. Pink Sheet

Unituxin Appeal Win Suggests NICE STA Process Is Not Appropriate For Pediatric Orphan Drugs

 
• By Francesca Bruce

The UK charity Solving Kids’ Cancer has won an appeal against a NICE decision declining to recommend United Therapeutics Unituxin (dinutuximab) for treating high-risk neuroblastoma in pediatric populations. The case raises questions over whether NICE's standard technology appraisals are appropriate for medicines for rare conditions in children.

Gavel_1200x675

The appeal panel of NICE, the health technology appraisal institute for England and Wales, has upheld an appeal by the charity Solving Kids’ Cancer against NICE’s decision not to recommend United Therapeutics Corp.'s Unituxin (dinutuximab) for use in a small number of children with high-risk neuroblastoma.

The panel agreed with the appellant that NICE had exceeded its powers and that its recommendation was unreasonable in the light of the evidence submitted to it.

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 Market Access

More from Pink Sheet

User Fee-Funded Staff Would Drop In FY 2026 US FDA Budget

 
• By Derrick Gingery

The reductions across medical product programs in the agency’s budget request include hundreds of positions funded by user fees, which may foreshadow its upcoming user fee restructuring strategy.

Canada Cuts Red Tape With Single Ethics Review For Trials

 
• By Neena Brizmohun

A new research ethics review system for multi-site clinical trials in Canada is expected to help improve study start-up times, avoid unnecessary duplication and boost the country’s competitiveness on a global stage.

EMA Official Clarifies Benefits And Limitations Of Innovation Pathways

 
• By Eliza Slawther

A European Medicines Agency official provided an overview and update of the support the EMA offers developers of innovative products and discussed, among other things, its Innovation Task Force, Quality Innovation Group and a scientific advice pilot for clinical trials.