NICE draft guidance recommends new treatment for rare liver condition

pharmafile | January 17, 2022 | News story | Sales and Marketing  

On Friday 14 January, NICE published a final draft Highly Specialised Technologies guidance, recommending odevixibat in line with its marketing authorisation, as an option for patients aged 6 months and over, with all types of progressive familial intrahepatic cholestasis (PFIC).

The positive recommendation follows an improved commercial offer in the form of a discount to the price of the drug from the company, alongside the Appraisal Committee’s view that some of the company’s assumptions in its economic model underestimated the cost effectiveness of odevixibat.

PFIC is a group of very rare and serious disorders. In many cases, patients diagnosed with PFIC experience end-stage liver disease by 10 years old. PFIC is a very rare and serious genetic condition that reduces or stops the flow of bile acids from the liver. This causes bile to collect in the liver (cholestasis) which can lead to 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.

The Appraisal Committee also concluded that there were benefits not captured in the company’s economic model, including the innovative nature of odevixibat, and the invasive nature of other treatments for the condition. Taking all this into consideration the committee agreed that odevixibat is a cost-effective use of NHS resources within the context of a highly specialised service.

Current treatment for PFIC includes medicines not licensed for this condition, surgery such as an operation called partial external biliary diversion, or a liver transplant. Odevixibat is the first licensed treatment for the condition.

The prevalence of PFIC in England is unknown, though worldwide estimates range between 1 per 50,000 to 1 per 100,000 live births. The symptoms of the most common types of PFIC usually begin in the first months of life. Symptoms progress at varying rates depending on the type, but usually develop into cirrhosis within the first decade of life. PFIC is fatal if untreated.

Ana Ovey

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