Haemophilia treatment first to be recommended by NICE as part of new drug pricing agreement 

Etranacogene dezaparovovec (Hemgenix; CSL Behring) is also the first medicinal product to enter the Innovative Medicines Fund.
Hand showing haemophilia in hand

The National Institute for Health and Care Excellence (NICE) has approved the first medicinal product under a new payment model introduced as part of the 2024 Voluntary Scheme for Branded Medicines Pricing, Access and Growth (VPAG).

On 27 June 2024, manufacturer CSL Behring announced that etranacogene dezaparovovec (Hemgenix), for treatment of haemophilia, was the first “advanced therapy medicinal product to use an innovative outcomes-based payment model” as part of the 2024 VPAG.

The VPAG is a new voluntary agreement between the UK government, NHS England and the Association of the British Pharmaceutical Industry (ABPI). The scheme replaced the previous ‘Voluntary pricing and access scheme’ (VPAS) on 31 December 2023 and is effective until 31 December 2028.

Etranacogene dezaparvovec addresses the underlying genetic cause of haemophilia B by introducing a copy of the human Factor IX coding DNA sequence into hepatocytes. A single infusion can reduce or eliminate the need for routine infusions, currently administered every 3–21 days.

It was recommended by NICE in final draft guidance published on 20 June 2024, making it the first and only gene therapy approved for treatment of moderately severe or severe haemophilia B in the UK.

The approved treatment is also the first medicinal product to enter the Innovative Medicines Fund (IMF), which will allow the manufacturer immediate reimbursement by NHS England, through a managed access agreement.

David Watson, executive director of patient access at Association of the British Pharmaceutical Industry (ABPI), commented: “The approval of this treatment is the first of what we hope will be many more examples of the UK using innovative payment models to ensure patients can benefit from new advanced therapies.

“Looking at the new medicines pipeline, these payment models are likely to become much more important in the years ahead.

“It also marks the first time a medicine has been granted managed access through the IMF since it was launched in 2022. We hope we will now see more new medicines reaching patients through this pathway as was intended when it was set up two years ago,” he added.

It is the first of two innovative payment model pilots to explore the practicalities of outcomes-based agreements for advanced therapy medicinal products introduced by the VPAG.

Commenting on the approval, Clive Smith, chair of the Haemophilia Society, said: “The availability of gene therapy through the NHS for people living with severe haemophilia B marks a major milestone for our community.

Haemophilia B is a rare genetic bleeding disorder in which a gene mutation causes coagulant factor IX, an important clotting protein, to be deficient or missing.

“At its most effective, gene therapy has the potential to transform lives by eliminating painful bleeds and removing the need for regular, invasive treatment.”

“There are currently about 2,000 people in the UK who live with haemophilia B. NICE estimates around 250 of those will be eligible for the new treatment in England,” said Smith.

“If gene therapy is successful, it should eliminate the need for day-to-day treatment for at least three years and potentially beyond a decade.”

Last updated
The Pharmaceutical Journal, PJ, July 2024, Vol 313, No 7987;313(7987)::DOI:10.1211/PJ.2024.1.323363

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