MecaserminIncrelex
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).
Decisions on record
- Meeting Mar 2022 Recommended Primary insulin-like growth factor 1 deficiency
- Meeting Nov 2021 Not recommended Primary insulin-like growth factor 1 deficiency (Primary IGFD)
Access path
- TGA registered · Increlex
TGA label narrower than the PBS population
- Nov 2021Not recommended
vs no treatment
- ↻ resubmittedMar 2022Recommended · restricted
- PBS listing · Authority Required
From the public summary
5.1 The PBAC recommended the listing of mecasermin on the basis that it be available on a Section 100 Growth Hormone (GH) Program listing for the 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).PSD · Mar 2022
5.2 The PBAC considered that mecasermin would address an unmet clinical need, as there are no other reimbursed or TGA registered options for this patient population.PSD · Mar 2022
4.5 In November 2021, the PBAC considered that the ICER was unacceptably high and uncertain, and that a price reduction would be requiredPSD · Mar 2022
4.6 The resubmission requested the same price as the November 2021 submission and stated that a price reduction was not feasible. 7PSD · Mar 2022
Source: Table 1-2, p17 and Section 2, p90 of the submission.PSD · Nov 2021
The submission described mecasermin as superior in terms of effectiveness compared with no treatment, based on changes in height over time and near-adult height.PSD · Nov 2021
The PBAC noted and welcomed the input from a health care professional. The input described a range of benefits of long term treatment with mecasermin including improved growth and potentially improved neurodevelopmental function including motor as well as social function.PSD · Nov 2021
4.11 The resubmission (p19) proposed annual caps on PBS expenditure and stated that the sponsor would rebate % of the overall expenditure above the capped amount. The PBAC considered that adjustment to the estimates was required (see paragraph 4.9). For more detail on PBAC’s view, see section 5 PBAC outcome.PSD · Mar 2022
Cost-effectiveness
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.
In November 2021, the PBAC considered that the ICER was unacceptably high and uncertain, and that a price reduction would be required PBAC · 2022
Decision context
PopulationChildren 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.
Risk sharingRisk sharing arrangement proposed whereby a percentage rebate would apply above a cap threshold to provide certainty of overall PBS expenditure.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2022 | Recommended · restricted | no treatment | — | RCT · Height |
| Nov 2021 | Not recommended | no treatment | — | RCT · Height velocity |
Consumer voice
No consumer comments were received for this item.
The PBAC noted that no consumer comments were received for this item. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC adds specific biochemical, auxological, and bone age criteria not stated in the TGA label.