PBAC's latest decision on Asfotase alfa rch: Noted (2018). Considered for Treatment of juvenile-onset hypophosphatasia (HPP; onset between 6 months and 17 years of age). Asfotase alfa rch is used in combination with best supportive care.
PBAC outcome
Noted
Authority Required
ICER (AUD/QALY)
Redacted
commercial-in-confidence
Submissions
2
first 2017
Submissions
2
2017 → 2018
Eligible population
Patients with juvenile-onset hypophosphatasia (onset between 6 months and 17 years of age) with confirmed diagnosis by low alkaline phosphatase activity and HPP-related bone disease on skeletal imaging, and presenting with specified HPP-related morbidities (respiratory compromise, failure to thrive, vitamin B6-dependent seizures, developmental delay, or history of fractures) plus severe functional impairment and chronic musculoskeletal pain
Therapy area
Rare disease
Line of therapy
First-line
Evidence base
RCT
Primary endpoint
6MWT
Pivotal trial size
19 patients
Key trials
ENB-009-10, ENB-006-09, ENB-008-10, ALX-HPP-502
Comparator
best supportive care (BSC)
Economic model
CUA
ICER note
ICER values are redacted (indicated by '''''''' marks in pricing section); economic model exists but numeric ICER not published in public summary
Risk sharing
Risk-sharing arrangement in place — Annual expenditure cap per patient per year (value redacted) and overall financial cap proposed to year 6 of subsidy
Why PBAC said no
Reasons cited in the latest PSD: Heterogeneity of eligible population introduces significant uncertainty in magnitude of benefit; reliance on secondary outcomes with non-significant changes in primary outcome (6MWT distance and quality of life); uncertain utilities based on clinical expert opinion; uncertain costs due to self-administration assumptions; complex proposed restriction requiring further modification; insufficient evidence of superiority in juvenile-onset population aged 6-18 months; small sample size in juvenile-onset subgroup (n=8)
Submission history
Jul 2017: Recommended with restriction · Authority Required