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    • Has all of the relevant evidence been taken into account?

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    Are the summaries of clinical and cost effectiveness reasonable interpretations of the evidence?

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Draft guidance consultation

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1 Recommendations

1.1

Cerliponase alfa is not recommended, within its marketing authorisation, for treating neuronal ceroid lipofuscinosis type 2 (CLN2) also known as tripeptidyl peptidase 1 deficiency.

1.2

This recommendation is not intended to affect treatment with cerliponase alfa that was funded with managed access before final guidance was published. People already having cerliponase alfa for treating CLN2, or who start cerliponase alfa before the end of the managed access period (December 2025), can continue with treatment until they and their NHS healthcare professional consider it appropriate to stop. This decision should be made jointly by the healthcare professional, the child or young person, and their parents or carers.

Why the committee made these recommendations

This evaluation reviews the evidence for cerliponase alfa for CLN2 (NICE highly specialised technologies guidance 12). It also reviews new data collected as part of the managed access agreement (MAA). CLN2 is a type of Batten disease, which is a group of rare genetic disorders. It progresses rapidly, causing seizures and dementia, and gradual loss of speech, mobility and vision. It leads to greatly reduced quality of life and a shortened life expectancy. Cerliponase alfa has been available through the MAA but is not routinely available in the NHS. Standard care without cerliponase alfa is supportive, aiming to relieve symptoms and maintain function and quality of life.

The new evidence includes data from clinical trials and from children having treatment in the NHS in England. Clinical trial evidence suggests that cerliponase alfa slows progression of CLN2. Patient experts and clinical experts have also explained that cerliponase alfa is a transformative treatment. But there is not much evidence about how well it works long term.

The committee took into account the condition's rarity, severity and the effect of cerliponase alfa on quality and length of life. But using the proposed price of the medicine, the most likely cost-effectiveness estimate is not within what NICE considers an acceptable use of NHS resources. So, cerliponase alfa is not recommended.