Odevixibat

7.1 The PBAC did not recommend odevixibat for the treatment of cholestatic pruritus in Alagille syndrome (ALGS) in patients aged six months and older. The PBAC considered that there is high unmet clinical need for this rare genetic condition, which has severe impacts on quality of life, including sleep, comfort, and wellbeing.PSD · Mar 2026

The PBAC considered the financial estimates need to be revised. The PBAC considered that a risk sharing arrangement (RSA) would be required.PSD · Mar 2026

6.51 The type of economic evaluation presented was a cost-utility analysis, using quality- adjusted life years (QALYs) as the final health outcome. The key components of the 6.52PSD · Mar 2026

6.53 Table 13. The model structure was similar to the model submitted for the PBAC July- 2025 resubmission of odevixibat in PFIC, which the ESC previously considered unreliable for decision making because of both implausible structural assumptions and continuing uncertainty in data inputs (see Odevixibat PFIC PBAC July-2025 PSD, paragraphs 6.63 and 13.3).PSD · Mar 2026

6.46 The submission described odevixibat as superior in terms of effectiveness compared to SoC. The evaluation considered that this claim was partially supported by direct evidence from ASSERT, i.e.:PSD · Mar 2026

• Odevixibat treatment led to a statistically significant decrease in the ObsRO pruritus scratching score over 24 weeks compared with placebo (LS mean difference - 0.88 [95% CI: -1.44, -0.33] p=0.0024).PSD · Mar 2026

6.2 The PBAC noted and welcomed the input from individuals (1), health care professionals (4) and organisations (2) via the Consumer Comments facility on the PBS website associated with this resubmission. The PBAC also recalled that the July 2024 submission received input from individuals (35), health care professionals (4) and organisations (3). The comments described a range of benefits of treatment with odevixibat.PSD · Mar 2025

6.3 The PBAC noted the advice received from PFIC Network, PFIC and Related Disorders Australia, and Liver Foundation stating that PFIC often results in a “horrendous itch” that, in some cases may be incessant and lead to continuous scratching and bleeding, lack of sleep, anxiety, depression, post-traumatic stress disorder, and ultimately poor quality of life for patients, as well as their families.PSD · Mar 2025