UK health watchdog rejects gene therapy for haemophilia B


In a draft guidance consultation, the UK National Institute for Health and Care Excellence (NICE) has decided not to recommend the use of Hemgenix (etranacogene dezaparvovec) on the NHS for treating haemophilia B (congenital factor IX deficiency).

Although the gene therapy was granted a marketing authorisation by the UK Medicines and Healthcare products Regulatory Agency (MHRA) earlier this year, the NICE committee did not feel it represented “an acceptable use of NHS resources”.

The Institute acknowledged that clinical trial evidence suggests that etranacogene dezaparvovec reduces the number of bleeding episodes a person has each year, but felt that there is not enough evidence on how well it works in the long term.

The draft guidance continued: “An indirect comparison of etranacogene dezaparvovec with FIX prophylaxis treatments suggests that it improves bleeding outcomes. But there are problems with this evidence, such as differences between studies in the methods used, and the definition and measurement of bleeding outcomes. So, the indirect comparison results are highly uncertain.”

The list price per treatment for a single dose of etranacogene dezaparvovec is £2,600,000. NICE argues that the cost-effectiveness estimates are uncertain because of uncertainties in the long-term clinical evidence and some of the assumptions used to estimate cost effectiveness, adding: “They are also above what NICE considers an acceptable use of NHS resources. So etranacogene dezaparvovec is not recommended.”

Guidance is subject to consultation

In response to the draft guidance, CSL Behring’s General Manager for the UK and Ireland, Eduardo Cabas said: “NICE has expressed its commitment to a more progressive approach when assessing highly innovative medicines, and CSL Behring asks NICE to honour this commitment when assessing etranacogene dezaparvovec and other gene therapies.”

Hemgenix was approved by the US Food and Drug Administration (FDA) in November 2022 and granted a conditional marketing authorisation the European Commission in February 2023.

NICE invites comments on the draft guidance from the stakeholders for this evaluation and the public and states that the recommendations could change before final publication.

Edited by: Diana Spencer, Senior Digital Content Editor, Drug Discovery World

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