1.1 Cerliponase alfa is recommended as an option for treating neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency, only if the conditions in the managed access agreement are followed.
1.2 This recommendation is not intended to affect treatment with cerliponase alfa that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop. For children or young people, this decision should be made jointly by the clinician and the child or young person, or the child's or young person's parents or carers.
Why the committee made these recommendations
CLN2 is a genetic disease that progresses rapidly, and leads to loss of speech, mobility and vision, progressive dementia and early death. Current treatment options are limited to symptomatic relief, and supportive and palliative care. Cerliponase alfa is expected to restore deficient TPP1 activity in the brain caused by the genetic mutation.
Clinical evidence shows that, in the short term, cerliponase alfa improves quality of life, and slows the deterioration of motor and language function. However, there is only short-term clinical evidence, so assumptions about long-term disease stabilisation and mortality are very uncertain.
The cost-effectiveness estimates meet the criteria for a quality-adjusted life year weight of 3.0 (that is, they show that cerliponase alfa provides substantial extra health and quality-of-life benefits), but are also very uncertain. However, they could plausibly be within the range that NICE normally considers an effective use of NHS resources for highly specialised technologies.
It is also recognised that CLN2 is a rare, devastating condition, that there is a substantial unmet need for an effective treatment, and that there are benefits beyond direct health benefits not captured in the economic analysis.
Taking all these factors into account, cerliponase alfa could provide value for money within the context of a highly specialised service. However, there is substantial clinical uncertainty and a high financial risk to the NHS. Therefore, a managed access agreement is needed to ensure the financial risk is addressed while allowing people expected to benefit most from cerliponase alfa access to it as further data are collected.