PBAC's latest decision on Mecasermin: Deferred (2022). Considered for Long-term treatment of growth failure in children and adolescents from 2 to 18 years with severe primary insulin-like growth factor 1 deficiency (Primary IGFD).
PBAC outcome
Deferred
Authority Required
ICER (AUD/QALY)
Redacted
commercial-in-confidence
Submissions
2
first 2021
Submissions
2
2021 → 2022
Eligible population
Children and adolescents from 2 to 18 years (up to 19th birthday) with severe primary insulin-like growth factor 1 deficiency (IGFD), defined as basal IGF-1 level below the 2.5th percentile adjusted for age and gender; with short stature at least 3 standard deviations below norm; with slow growth (baseline height velocity less than 25th percentile for bone age); without growth hormone deficiency; with bone age less than 13.5 years in girls or 15.5 years in boys; without secondary causes of IGFD; and without epiphyseal closure.
Therapy area
Endocrinology
Line of therapy
Initial treatment
Evidence base
RCT
Primary endpoint
Height
Key trials
MS301, Study 1419, EU-IGFD Registry, Petriczko et al. 2019
Comparator
no treatment
Economic model
CUA
Budget impact
$15M
Eligible patients/year
80
ICER note
ICER not stated in resubmission. At November 2021 meeting, PBAC considered ICER unacceptably high and uncertain; price reduction was required but resubmission stated price reduction was not feasible.
Risk sharing
Risk-sharing arrangement in place — Risk sharing arrangement proposed whereby a percentage rebate would apply above a cap threshold to provide certainty of overall PBS expenditure.
Submission history
Nov 2021: Recommended with restriction · Authority Required