England: CSL Undeterred By Draft NICE Rejection Of Its £2.6m Hemophilia B Gene Therapy

 
• By Eliza Slawther
  
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CSL Behring said it remains confident that a market access agreement for its hemophilia B gene therapy Hemgenix can be reached after England’s HTA body NICE rejected the product because of uncertainties over its long-term benefit and cost-effectiveness.

Genetic Medicine with dna isolated on white.
• Source: Alamy

In draft guidance on 2 August, England’s health technology assessment body NICE recommended against National Health Service funding for CSL Behring’s Hemgenix (etranacogene dezaparvovec) to treat adults with moderately severe or severe hemophilia B without a history of factor IX inhibitors, citing uncertainties in both clinical evidence and its cost-effectiveness.

According to NICE, the clinical data uncertainties mean that cost-effectiveness estimates for the gene therapy are also uncertain. Regardless of...

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