PBAC's latest decision on Selumetinib: Recommended with restriction (2024). Considered for Treatment of symptomatic, inoperable plexiform neurofibroma(s) in paediatric patients aged 2 years and over with neurofibromatosis type 1 (NF1).
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Redacted
commercial-in-confidence
Submissions
2
first 2022
Submissions
2
2022 → 2024
Eligible population
Children aged ≥2 years and ≤18 years at treatment initiation with NF1 and symptomatic or likely-to-be-symptomatic, inoperable plexiform neurofibroma(s), able to swallow capsules, with Karnofsky or Lansky Performance Score ≥70%.
Therapy area
Rare disease
Line of therapy
First-line
Evidence base
RCT
Primary endpoint
ORR
Key trials
SPRINT trial Stratum I, NCT02407405
Comparator
best supportive care
Economic model
CUA
ICER note
ICER value is redacted/commercial-in-confidence; the document states 'the ICER remained high' after a price reduction but does not publish the numeric value.
Risk sharing
Risk-sharing arrangement in place — Risk Sharing Arrangement (RSA) with a rebate above the overall expenditure cap; fixed maximum cost per patient based on BSA with percentage rebate for use exceeding caps.
Submission history
Nov 2022: Recommended with restriction · Authority Required
Mar 2024: Recommended with restriction · Authority Required