The committee noted an ongoing extension study, PEDFIC2, which uses odevixibat at a dose of 120 micrograms/kg/day and includes people with progressive familial intrahepatic cholestasis (PFIC) types 1, 2, 3 and 6. It also recalled that further data was expected from the company's planned indirect treatment comparison with partial external biliary diversion (PEBD) and the global registry study. These could resolve some of the uncertainty around odevixibat's treatment effect.
The committee noted that the following data would be useful at the time of the next guidance review:
the ongoing effect of odevixibat on serum bile acid levels and pruritus, survival outcomes, liver transplant rates and alternative utility values for people having high-dose odevixibat in PEDFIC2
clinical effectiveness by PFIC subtypes from PEDFIC2, particularly types 3 and 6
the clinical effectiveness of odevixibat compared with PEBD from the company's indirect treatment comparison
UK-specific data on starting age and stopping rates for odevixibat
alternative utility decrements for carers of people with PFIC and for having a stoma bag.