Odevixibat is recommended, within its marketing authorisation, as an option for treating progressive familial intrahepatic cholestasis (PFIC) in people 6 months and older. It is recommended only if the company provides odevixibat according to the commercial arrangement.
Why the committee made these recommendations
PFIC is a rare and serious genetic condition that reduces or stops the flow of bile acids from the liver. This can cause severe pruritus (itching), poor growth and liver damage. PFIC severely affects the quality of life of people with the condition, and of their families and carers. It is fatal if untreated. Current treatment includes medicines not licensed for this condition (off label), then surgery such as an operation called partial external biliary diversion (PEBD) and, finally, a liver transplant.
Results from clinical trials suggest that, in people with the PFIC types 1 and 2, odevixibat reduces bile acid levels in the blood and pruritus compared with placebo (with or without off-label medicines). There is limited data for other types of PFIC. The clinical effectiveness of odevixibat when using the dose escalation schedule that would be used in NHS practice compared with PEBD is also uncertain.
The company's cost-effectiveness estimates are above what NICE usually considers acceptable for highly specialised technologies. However, several assumptions in the company's economic model are uncertain and possibly conservative, including:
the percentage of people having odevixibat also having PEBD
the average age at which treatment is started
the reduction in quality of life from having a stoma bag
death after a liver transplant.
When taking all these assumptions into account, the cost effectiveness of odevixibat is likely to be lower than the company's estimate. Also, the model does not capture:
health-related benefits from delaying or stopping lifelong immunosuppression after a liver transplant
the effect on quality of life for carers of people with PFIC
the invasive nature of other treatments
the young age at which PFIC can develop
the innovative nature of odevixibat.
After taking all this into account, odevixibat is recommended for use in the NHS for PFIC.