FingolimodAKM Fingolimod
Treatment of relapsing-remitting multiple sclerosis (RRMS) in paediatric and adolescent patients weighing 40 kg or less.
Decisions on record
- Meeting Jul 2019 Recommended Relapsing-remitting multiple sclerosis (RRMS)
- Meeting Mar 2011 Recommended Multiple sclerosis
Access path
- TGA registered · AKM Fingolimod
TGA label narrower than the PBS population
- Mar 2011Recommended
vs intramuscular interferon beta-1a (Avonex)
- Jul 2019Recommended · restricted
Comparator changed: intramuscular interferon beta-1a (Avonex) → fingolimod 500 mcg
- PBS listing · Authority Required
From the public summary
6.1 The PBAC recommended the Authority Required (telephone) listing of fingolimod, in the form 250 microgram capsules, on the general schedule for treatment of relapsing- remitting multiple sclerosis (RRMS) in patients weighing 40kg or less. In making this 7PSD · Jul 2019
6.2 The PBAC accepted that fingolimod 500 mcg taken once daily by patients weighing 40kg or less was likely to be replaced by fingolimod 250 mcg, but noted that in addition to this population, patients of any weight currently being treated with fingolimod 500 mcg every second day would also be likely to be prescribed fingolimod 250 mcg.PSD · Jul 2019
5.13 The submission proposed the same published and effective prices for fingolimod 250 mcg capsules as the current fingolimod 500 mcg capsules, based on equivalent efficacy, safety and systemic exposure of fingolimod 250 mcg in patients weighing ≤40kg with fingolimod 500 mcg in heavier patients. For more detail on PBAC’s view, see section 6 PBAC outcome.PSD · Jul 2019
5.10 The submission claimed that fingolimod 250 mcg in paediatric patients weighing 40 kg or less was non-inferior in comparative effectiveness and non-inferior in comparative safety to fingolimod 500 mcg in adults and children weighing over 40 kg.PSD · Jul 2019
5.11 The PBAC considered that the claim of non-inferior comparative effectiveness was reasonable.PSD · Jul 2019
5.2 The PBAC noted and welcomed the input from two organisations via the Consumer Comments facility on the PBS website. The comments described the need for a greater number of treatments for RRMS in paediatrics and low weight patients, and how relapses can result in short and long term disabilities, which significantly affect the lives of families who care for these patients.PSD · Jul 2019
Cost-effectiveness
Cost-minimisation analysis; no ICER calculated as fingolimod 250 mcg proposed same price as fingolimod 500 mcg
The PBAC noted that its recommendation was on a cost-minimisation basis and advised that, because fingolimod 250 mcg is not expected to provide a substantial and clinically relevant improvement in efficacy, or reduction of toxicity over fingolimod 500 mcg, or not expected to address a high and urgent unmet clinical need over fingolimod 500 mcg, the criteria prescribed by the National Health (Pharmaceuticals and Vaccines – Cost Recovery) Regulations 2009 for Pricing Pathway A were not met. PBAC · 2019
Decision context
PopulationPaediatric and adolescent patients aged 10 to less than 18 years with relapsing-remitting multiple sclerosis weighing 40 kg or less
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Jul 2019 | Recommended · restricted | fingolimod 500 mcg | — | RCT · ARR |
| Mar 2011 | Recommended | intramuscular interferon beta-1a (Avonex) | $45k–75k | RCT · OS | PFS | DFS | ORR | QoL | Surrogate |
Consumer voice
Two consumer organisations highlighted the need for more treatment options for RRMS in paediatric and low-weight patients, and described how relapses cause short and long-term disabilities that significantly impact families.
The comments described the need for a greater number of treatments for RRMS in paediatrics and low weight patients Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to paediatric/adolescent patients aged 10–18 years weighing ≤40 kg with RRMS, excluding adults in the TGA label.