PBAC's latest decision on Carglumic acid: Not recommended (2015). Considered for Treatment of acute hyperammonaemia due to organic acidaemias (isovaleric acidaemia, methylmalonic acidaemia, and propionic acidaemia).
PBAC outcome
Not recommended
Authority Required
ICER (AUD/QALY)
—
ICER not stated
Submissions
1
first 2015
Submissions
1
2015 → 2015
Eligible population
Neonates and young children with acute hyperammonaemia secondary to organic acidaemias (isovaleric acidaemia, methylmalonic acidaemia, or propionic acidaemia), typically in hospital or intensive care settings.
Therapy area
Metabolic
Line of therapy
Not applicable
Evidence base
Single-arm
Primary endpoint
Surrogate
Pivotal trial size
58 patients
Comparator
placebo or standard care without carglumic acid
Economic model
CEA
ICER note
Economic evaluation presented but ICER not explicitly stated in the public text; document indicates cost-effectiveness analysis was conducted but specific numeric ICER values are not disclosed in this PSD.
Why PBAC said no
Reasons cited in the latest PSD: unclear clinical need for PBS listing given uncertainty about use outside hospital admission, clinical claim not adequately supported by case report evidence lacking control group, surrogate endpoint (plasma ammonia reduction) relationship to patient-relevant outcomes unclear, small patient numbers with high risk of bias and confounding factors not accounted for