Elafibranor is recommended, within its marketing authorisation, as an option for treating primary biliary cholangitis in adults, when used:
with ursodeoxycholic acid (UDCA), if the primary biliary cholangitis has not responded well enough to UDCA, or
alone, if UDCA cannot be tolerated.
Elafibranor is only recommended if the company provides it according to the commercial arrangement.
Why the committee made this recommendation
Usual treatment for primary biliary cholangitis is UDCA. If UDCA does not work well enough, or cannot be tolerated, obeticholic acid is typically used, with or without UDCA. There is an unmet need for treatments other than obeticholic acid because it does not work well enough for everyone and can make itching worse in some people.
Clinical trial evidence shows that after 1 year, more people who have elafibranor have normal results for some liver function tests than people who have placebo. Elafibranor has not been directly compared with obeticholic acid. The results of an indirect comparison suggest that more people who have elafibranor have normal results for some liver function tests at 1 year than people who have obeticholic acid, but these are uncertain.
There are also some uncertainties in the economic model, such as:
how well elafibranor works compared with obeticholic acid
the relationship between some liver function tests and changes in primary biliary cholangitis, including in the longer term
the assumptions used in the economic model.
The most likely cost-effectiveness estimates are within the range that NICE considers an acceptable use of NHS resources, even when taking into account the uncertainties. So, elafibranor is recommended.