First licensed gene therapy for haemophilia B not recommended for use in the NHS
Written by Jeff Courtney, August 2, 2023
The first attempt to get a gene therapy for haemophilia B commissioned in the UK has been rejected by NICE, the government body tasked with reviewing the clinical and cost-effectiveness of new treatments for the NHS in England.
At a NICE committee meeting in July all evidence about CSL Behring’s product Hemgenix (Etranacogene Dezaparvovec) was considered, alongside submissions from the Haemophilia Society and our members. In addition, Ross Bennett and John Curley, who have haemophilia B, gave their views and explained what it is like to live with severe haemophilia B.
However, NICE decided that, at present, the treatment should not be prescribed on the NHS. This is likely to be for reasons of cost-effectiveness, compared to other treatments currently used. Also, while the treatment is expected to be effective for most people in preventing bleeds there is uncertainty over how long the treatment can be expected to last for.
The evidence will be reconsidered at a meeting in September which may lead to a different decision, particularly if there is a change in the price of the product or more evidence to evaluate. NICE is running a consultation, which closes on 23 August, for the public and stakeholders to consider the decision and the evidence and share their views.
Clive Smith, Chair of the Haemophilia Society said: “Gene therapies for haemophilia B can vastly reduce the burden of treatment, improve joint health and potentially eliminate painful bleeds. We are disappointed with this initial decision and hope that NICE further considers these benefits carefully and helps eligible patients to access these innovative, potentially long-lasting treatments as soon as possible.”
Professor Pratima Chowdary, Professor of Haematology and Haemostasis, University College London and Chair of the UK Haemophilia Centres Doctors’ Association praised the work of the UK scientific and clinical community who pioneered the initial science behind haemophilia B gene therapy and said it was ‘unfortunate’ that it could not yet be made available to UK patients.
She said: “The negative recommendations from NICE are disappointing, considering the strength of the recently published international HOPE-B study, which clearly demonstrates the potential for long-term clinical benefits of a single infusion of etranacogene dezaparvovec.
“NICE’s review of the evidence submitted for the first gene therapy for haemophilia B suggests limited flexibility in the process to appreciate the potential for multi-decade durability of gene therapies. I am hopeful that through continued collaboration with NICE, we can enable access to this important treatment option.”