NICE: Deal agreed for continued access to life-changing treatment for rare inherited disease
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NICE is consulting on draft guidance for cerliponase alfa for
treating neuronal ceroid lipofuscinosis type 2 (CLN2), a type of
Batten disease. The draft guidance does not, at
present, recommend the treatment's use for future patients with
this rare, life limiting disease due to its high price and the
limited evidence of long-term effectiveness. However,
with the support of NICE, NHS England (NHSE) and BioMarin have
agreed a deal to provide permanent...Request free trial
NICE is consulting on draft guidance for cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 (CLN2), a type of Batten disease. The draft guidance does not, at present, recommend the treatment's use for future patients with this rare, life limiting disease due to its high price and the limited evidence of long-term effectiveness. However, with the support of NICE, NHS England (NHSE) and BioMarin have agreed a deal to provide permanent access to cerliponase alfa (also called Brineura) within its licensed indication for people with CLN2 who have already been initiated onto treatment or will be started on treatment before the end of the newly-extended managed access period. This is due to close when NICE's evaluation concludes or by the end of 2025, whichever is sooner. This means that people will be able to stay on the treatment even if NICE is unable to recommend it for new patients beyond that point. NICE, NHSE and the company continue to work towards a solution to secure access to all future patients but at the moment the treatment is not considered cost effective. Helen Knight, director of medicines evaluation at NICE, said: “We're pleased that NICE has been able to support NHSE and the company in reaching an agreement to make access to cerliponase alfa permanent for everyone who has already started treatment and those who will start treatment before the managed access agreement ends in December. NICE, together with NHS England, remains committed to working with the company to try to reach a long-term deal that will give access to cerliponase alfa to all eligible people after that time.” The treatment has been available to NHS patients while further evidence has been collected on its use and benefits. That ‘managed access period' has been extended several times, although without the requirement for additional data collection, and will now end in December 2025 (or when NICE publishes its final recommendations if earlier). The additional evidence collected prior to the extension of the MAA is being evaluated by NICE to determine whether it can be used routinely on the NHS. After looking at the new evidence from children having treatment in the NHS in England, the independent NICE committee concluded that, although cerliponase alfa has substantial short-term benefits in slowing the rate at which CLN2 gets worse, there is not enough evidence to show what benefits it provides in the long term. In particular this relates to how a person's health state when they start treatment dictates how much they will benefit from treatment. The committee also took into account the rarity of CLN2, its severity and the effect of cerliponase alfa on length and quality of life. It recognised the significant benefits it provides by applying a cost-effectiveness threshold 1.5 times higher than that normally applied to treatments for ultra-rare conditions and more than 5 to 8 times higher than that normally applied to treatments being evaluated outside of the HST programme. But, because of the lack of long-term evidence of effectiveness, the price of the medicine proposed by the company is still far higher than NICE can consider an effective use of NHS resources. Helen Knight continued: “We know this is not entirely the news people in the Batten disease community were hoping for. However, this is not the end of the story. We will continue to work with all parties towards a solution.” With a list price of more than £500,000 per patient for each year's treatment, cerliponase alfa is an enzyme replacement therapy administered directly into the brain via a surgically implanted permanent access device. It is estimated that in the UK there are around 30 to 50 children living with CLN2 with around 3 to 6 children newly diagnosed each year. Managed Access NICE recommended cerliponase alfa under the temporary agreement (managed access agreement, or “MAA”) in 2019. At the time uncertainty about its clinical benefits and the high financial risk to the NHS meant that additional data needed to be collected. MAAs enable NHS patients to access promising and potentially cost-effective medicines for a fixed period, at a discount, while further evidence is gathered on how these medicines can be provided and how well they work outside clinical trials. MAAs help to address clinical uncertainties for the NHS.
About CLN2
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