Log in or register to see all Alerts
New HTA Decisions in England
June 2020
Drug name
BRINEURA® (cerliponase alfa)
Company
BioMarin
Decision date
25/10/2019
Therapeutic area
Neurological conditions
Therapeutic sub area
Decision
Recommended
Indication
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.
Decision Detail
Only if the conditions in the managed access agreement are followed.
Summary
The main clinical evidence submitted by the company came from 3 studies (190-201, 190-202 and 190-901). Study 190-201 was a single-arm open-label study including 23 children aged 3 years to 16 years with late-infantile CLN2 treated with cerliponase alfa. After the completion of 190-201, patients were enrolled in an extension study (190-202) for long-term follow up.Study 190-901 was a natural history study that retrospectively evaluated disease progression in patients with untreated CLN2 (included in the DEM-CHILD database). The committee agreed that CLN2 scores showed that cerliponase alfa was effective in slowing disease progression in 2 key functional domains (motor and language). 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. The company considers all results of the economic analysis incorporating this arrangement commercial in confidence, so ICERs cannot be reported. In the absence of persuasive long-term evidence, the committee considered that the company’s original assumptions around disease stabilisation, mortality and starting distribution were unrealistic. Taking into account its preferred assumptions, the committee agreed that cerliponase alfa was associated with substantial incremental QALY gains and met the criteria for a QALY weight of 3.0. When applying a QALY weight of 3.0, the committee noted that, while highly uncertain, it was plausible that the ICER was within the range normally considered an effective use of NHS resources for highly specialised technologies. Also, the committee noted that some benefits associated with cerliponase alfa had not been captured in the economic analysis. 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.