PBAC's latest decision on Canakinumab: Recommended with restriction (2017). Considered for Treatment of adults and children with moderate to severe cryopyrin-associated periodic syndromes (CAPS), including familial cold autoinflammatory syndrome, Muckle-Wells syndrome, and neonatal-onset multisystem inflammatory disease.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Cost-min
cost-minimisation analysis
Submissions
2
first 2015
Submissions
2
2015 → 2017
Eligible population
Adults and children with moderate to severe cryopyrin-associated periodic syndromes (CAPS), including familial cold autoinflammatory syndrome, Muckle-Wells syndrome, and neonatal-onset multisystem inflammatory disease.
Therapy area
Rare disease
Line of therapy
First-line
Evidence base
Meta-analysis
Primary endpoint
Disease flare
Pivotal trial size
31 patients
Key trials
D2304, D2306, D2307, D2307E1, D2201, D2401, D2308
Comparator
anakinra
Economic model
Cost-minimisation
ICER note
Cost-minimisation analysis; no ICER calculated as submission claimed equivalent effectiveness and safety to comparator.
Risk sharing
Risk-sharing arrangement in place — The ESC considered that consistent with anakinra, it would be more appropriate to manage any risk of use beyond the restriction through risk sharing arrangements.
Submission history
Mar 2015: Not recommended · Authority Required
Nov 2017: Recommended with restriction · Authority Required