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

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In draft guidance on 2 August, England’s health technology assessment body NICE recommended against National Health Service funding for CSL Behring’s Hemgenix (etranacogene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.

More from Europe

Trump’s Tariffs Will Lead To ‘Instability’ And ‘Less Investment’ In UK

 

While the pharma industry appears to be exempt from US tariffs imposed by President Trump, a member of the UK House of Lords says the details are unknown and warned that uncertainty “leads to less investment” in business as a whole.

Industry Benefits As EU CTIS Hits Global Data Sharing Milestone

 

The EU Clinical Trials Information System has achieved primary registry designation in the International Clinical Trials Registry Platform in a move that is expected to reduce regulatory burden for companies and help them lower compliance costs by aligning with publication requirements in medical journals.

UK Health Secretary Admits VPAG ‘More Expensive’ Than Expected & Needs To Be ‘Resolved’

 

The UK government has listened to industry concerns about high clawback rates under the voluntary scheme and will review it in June in a bid to resolve the issue and “move on to bigger and more important things,” health secretary Wes Streeting says.

AI Could Be Used ‘In The Deliberation’ Of HTA Reviews In England, Says NICE

 

England’s health technology assessment institute, NICE, is looking to “reimagine” its evaluation process with the help of AI, rather than just using this technology to speed up its existing processes.

More from Geography

Pink Sheet Podcast: Understanding The US FDA Reduction-In-Force And Its Long-Term Impact

Pink Sheet reporter and editors discuss how the massive layoffs will reshape the FDA and its impact on the drug and biologics industries.

Trump’s Tariffs Will Lead To ‘Instability’ And ‘Less Investment’ In UK

 

While the pharma industry appears to be exempt from US tariffs imposed by President Trump, a member of the UK House of Lords says the details are unknown and warned that uncertainty “leads to less investment” in business as a whole.

Industry Benefits As EU CTIS Hits Global Data Sharing Milestone

 

The EU Clinical Trials Information System has achieved primary registry designation in the International Clinical Trials Registry Platform in a move that is expected to reduce regulatory burden for companies and help them lower compliance costs by aligning with publication requirements in medical journals.