SelumetinibKoselugo
Treatment of symptomatic, inoperable plexiform neurofibroma(s) in paediatric patients aged 2 years and over with neurofibromatosis type 1 (NF1).
Decisions on record
- Meeting Mar 2024 Recommended Neurofibromatosis type 1 (NF1)
- Meeting Nov 2022 Not recommended Symptomatic, inoperable plexiform neurofibroma(s) in paediatric patients with neurofibromatosis type 1
Access path
- TGA registered · Koselugo
TGA label narrower than the PBS population
- Nov 2022Not recommended
vs supportive care
- ↻ resubmittedMar 2024Recommended · restricted
Comparator changed: supportive care → best supportive care
- PBS listing · Authority Required
From the public summary
The PBAC recommended the General Schedule PBS listing of selumetinib for the treatment of symptomatic inoperable plexiform neurofibroma(s) (PN) in paediatric patients with neurofibromatosis type 1 (NF1). The PBAC noted the high clinical need for treatments for this condition and considered that selumetinib provided a clinical benefit for patients compared to best supportive care (BSC).PSD · Mar 2024
The PBAC acknowledged the consumer comments relating to this submission which described the quality of life challenges associated with the disease and highlighted the high unmet clinical need for effective treatments. The PBAC noted the significant impact PN associated with NF1 can have on children, including pain and disfigurement.PSD · Mar 2024
The resubmission again presented a cost-utility analysis (CUA) of selumetinib compared to BSC.PSD · Mar 2024
In November 2022, the PBAC considered that there were a number of issues with the economic model that reduced its reliability for decision-making (paragraph 7.11, selumetinib PSD, November 2022 PBAC meeting). The ESC had considered that the 17PSD · Mar 2024
Selumetinib has an acceptable safety and tolerability profile for chronic treatment in children. Source: Table 1.1 of the resubmission.PSD · Mar 2024
Strikethrough text indicates change to the requested population from the July 2022 resubmission.PSD · Mar 2024
The PBAC noted and welcomed the input from individuals (22), health care professionals (2) and organisations (2) via the Consumer Comments facility on the PBS website. The comments from individuals described the physical and psychological burden of the disease and the high unmet clinical need for effective therapies for NF1 patients with PN.PSD · Mar 2024
Input from the Children’s Tumour Foundation described the lack of available therapies for patients with NF1 and the clinical need for affordable treatment options. The input also described the debilitating physical and psychological effects of tumours on NF1 patients and the complications associated with surgery.PSD · Mar 2024
Cost-effectiveness
ICER value redacted; document states 'remained high' after price reduction but does not publish the numeric value
Decision context
PopulationChildren aged 2 to 18 years at initiation of treatment with NF1 and symptomatic or likely-to-be-symptomatic, inoperable plexiform neurofibroma(s), with Karnofsky or Lansky Performance Score ≥70%, able to swallow capsules, treated by or in consultation with a specialist in neurofibromatosis
Risk sharingRisk Sharing Arrangement (RSA) with a rebate of % above the overall expenditure cap; also a fixed maximum cost per patient based on BSA of m²
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2024 | Recommended · restricted | best supportive care | — | RCT · ORR |
| Nov 2022 | Not recommended | supportive care | — | RCT · ORR |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| NCT02407405 | Ph 2 | 36 | Determine objective response rate | active not recruiting |
Consumer voice
Individuals, health care professionals, and organisations provided input describing the physical and psychological burden of NF1 with PN, strong unmet clinical need, and benefits of selumetinib including tumour stabilisation, pain reduction, and improved quality of life. Health professionals noted dramatic effects on quality of life and tolerable safety profile.
The comments from individuals described the physical and psychological burden of the disease and the high unmet clinical need for effective therapies for NF1 patients with PN. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to ages ≤18 years at initiation, requires capsule-swallowing ability, and mandates performance score ≥70%.