NICE does not support NHS funding of haemophilia gene therapy

The UK National Institute for Health and Care Excellence (NICE) has published draft guidance recommending against the use of a new gene therapy for haemophilia B on the NHS.

The recommendation is based on the uncertainty surrounding the long-term effectiveness of Hemgenix (etranacogene dezaparvovec), as well as concerns about its cost-effectiveness and whether it is 'an acceptable use of NHS resources'.

A statement from the committee that made the recommendation said: 'Evidence from a clinical trial suggests that etranacogene dezaparvovec reduces the number of bleeding episodes a person has each year. But there is not enough evidence on how well it works in the long term.'

Haemophilia B is a genetic bleeding disorder caused by low levels or deficiency in blood clotting Factor IX (FIX), a protein which helps form blood clots. Haemophilia B represents about 15 percent of patients with haemophilia, affecting around 1 in 40,000 people, mostly men.

Current treatments involve regular, life-long intravenous infusions to prevent bleeding episodes, while a one-off, single dose of Hemgenix could allow adults with moderately-severe and severe haemophilia B to produce their own FIX for up to two years.

Hemgenix was approved by the US Food and Drug Administration (FDA) in November 2022 (see BioNews 1169) and was administered to the first patient this June. In March 2023, it was given conditional marketing authorisation by the UK's Medicines and Healthcare Products Regulatory Agency (MHRA), meaning that it can be sold to, and used by healthcare providers in the UK.

Professor Pratima Chowdary, haematologist at University College London and chair of the UK Haemophilia Centres Doctors Association, 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.'

Hemgenix is marketed at a list price of £2.6 million (around $3.3 million) per dose, but has entered into a 'commercial arrangement', which would apply if Hemgenix is eventually recommended for use by the NHS. NICE has approved similarly expensive gene therapies for conditions such as metachromatic leukodystrophy (see BioNews 1179) and spinal muscular atrophy (BioNews 1098). Both are degenerative genetic conditions that are otherwise fatal in early childhood, and in both cases, current evidence suggests these gene therapies provide a long-term cure.

NICE is still accepting comments on its draft guidance, and a second appraisal committee meeting is scheduled to be held in September, with a final decision expected towards the end of November.