RuxolitinibJakavi
Recommended HaematologyRestricted 💬 consumer voice
Addition of nurse practitioners as prescriber type for existing PBS-listed ruxolitinib for treatment of myelofibrosis, polycythemia vera, acute graft versus host disease, and chronic graft versus host disease.
9
Submissions
4 resub
2013–26
On the record
$45k–75k
ICER range
1 sourced ICER · 2013
Not modelled
Cost basis
Decisions on record
- Meeting Mar 2026 Recommended Myelofibrosis, graft versus host disease, polycythemia vera
- Meeting Mar 2025 Recommended Polycythemia vera (PV), resistant to or intolerant of hydroxycarbamide
- Meeting Nov 2022 Recommended Moderate to severe chronic graft versus host disease refractory to or dependent on corticosteroids no PSD
- Meeting Sep 2022 Not recommended Moderate to severe chronic graft versus host disease refractory to, dependent on or intolerant of corticosteroids no PSD
- Meeting Jul 2022 Recommended Grade II to IV acute graft versus host disease, moderate to severe chronic graft versus host disease
- Meeting Mar 2021 Noted Myelofibrosis no PSD
- Meeting Nov 2019 Not recommended Polycythemia vera (PV)
- Meeting Jul 2016 Recommended Myelofibrosis
3 earlier decisions
- Mar 2015 Recommended Myelofibrosis
- Jul 2014 Deferred Novartis Pharmaceuticals Australia Pty Ltd New listing (Major submission) Myelofibrosis Authority required listing for the treatment of disease related symptoms or splenomegaly in patients with intermediate to high risk primary (idiopathic) myelofibrosis (MF), post-polycythemia MF and post-essential
- Jul 2013 Not recommended Bone marrow disorder
Access path
- TGA registered · Jakavi
TGA label narrower than the PBS population
- Jul 2013Not recommended
high and unacceptable ICER, unreliable ICER based on modelling issues and sensitivity to assumed survival and utility…
- Jul 2014Deferred
Comparator changed: Best Available Therapy (BAT) → placebo
- Mar 2015Recommended
Comparator changed: placebo → placebo or best supportive care
- Jul 2016Recommended
Evidence: RCT → Cost-minimisation
- Nov 2019Not recommended
Evidence: Cost-minimisation → RCT
- ↻ resubmittedSep 2022Recommended · restricted
Comparator changed: best available therapy (50% hydroxycarbamide and 50% peginterferon) →…
- Nov 2022Recommended · restricted
- Mar 2025Recommended
Listing: Restricted → Authority Required
- Mar 2026Recommended · restricted
Evidence: RCT → Other
- PBS listing · Restricted
RecommendedDeferredNot recommended
From the public summary
PBAC outcome
7.1 The PBAC recommended the listing of listing for ruxolitinib for the treatment of adult patients with polycythaemia vera (PV) who are resistant to or intolerant of hydroxycarbamide (hydroxyurea) (HC/HU). The PBAC noted the resubmission provided updated data which reported significant improvements in major event-free survival (EFS), reductions in venesections and improvements in duration of response that were clinically relevant.PSD · Mar 2025
The PBAC noted that while no statistically significant differences in progression-free survival (PFS) or overall survival (OS) were reported in the clinical trial evidence, differences in these outcomes were assumed in the economic model and maintained throughout the 20 year time horizon.PSD · Mar 2025
Economic analysis
6.58 The resubmission presented a stepped economic evaluation of ruxolitinib compared to BAT in adults with PV who are resistant or intolerant to HC/HU. The economic evaluation was based on direct randomised trials (MAJIC, RESPONSE and RESPONSE-2 trials) and implemented a modelled evaluation. The economic evaluation was presented as cost-utility/cost-effectiveness analysis.PSD · Mar 2025
6.59 Compared to the July 2019 submission, the key changes in the resubmission model were:PSD · Mar 2025
Clinical claim
Source: Table 1.1 Key components of the clinical issue addressed by the resubmission, p13 of the submission.PSD · Mar 2025
Abbreviations: mg, milligram. a BAT includes treatment with HC/HU, peginterferon α-2a or observation. b Response was defined as HCT <45% without venesection and/or all of the three items: platelet count ≤ 400 x 109/L, WBC < 10 x 109/L, and absence of splenomegaly on imaging c Event-free is reduction in the risk of patient relevant outcomes such as thrombosis, haemorrhage, and progression to MF or AML.PSD · Mar 2025
Consumer comments
6.2 The PBAC noted and welcomed the input from individuals (11), health care professionals (1) and organisations (3) via the Consumer Comments facility on the PBS website. The PBAC noted the comments from individuals who would like to access the medicine to treat their own health condition described the impact of PV on their quality of life (QoL) and the contribution of side-effects from currently availablePSD · Mar 2025
6.3 Input from the Leukaemia Foundation stated that current BAT is largely focused on prevention of thrombosis with more options needed that treat the underlying cause of the disease. The Leukaemia Foundation input noted that ruxolitinib has shown efficacy in reducing spleen volume, controlling haematocrit and improving symptoms of disease by directly targeting JAK1 and JAK2 signalling pathways.PSD · Mar 2025
Cost-effectiveness
Not applicable — this is a Category 4 submission regarding prescriber type eligibility only, not a clinical or economic evaluation of the drug itself.
The ESC considered this issue was partially addressed as while the PFS health states were now more objective, the trial did not demonstrate a significant difference in this outcome for patients treated with ruxolitinib. In addition, the ESC noted the updated results continued to demonstrate no OS benefit and considered the model reliance on this outcome remained inappropriate. PSD · 2025
ICER uncertainICER / price too highEconomic model disputed ×2Immature survival dataSurrogate endpointCost-effectiveness accepted
Decision context
PopulationNurse practitioners seeking to prescribe ruxolitinib for patients with intermediate-1, intermediate-2, and high-risk myelofibrosis; polycythemia vera; acute graft versus host disease (Grade II to IV); and chronic graft versus host disease (moderate to severe).
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2026 | Recommended · restricted | — | — | Other |
| Mar 2025 | Recommended | best available therapy (BAT) | — | RCT · Response rate |
| Nov 2022 | Recommended · restricted | best available therapy (BAT) | — | RCT · ORR |
| Sep 2022 | Recommended · restricted | Best available therapy (BAT) | — | RCT · ORR |
| Nov 2019 | Not recommended | best available therapy (50% hydroxycarbamide and 50% peginterferon) | — | RCT · Surrogate |
| Jul 2016 | Recommended | — | — | Cost-minimisation |
| Mar 2015 | Recommended | placebo or best supportive care | — | RCT · OS |
| Jul 2014 | Deferred | placebo | — | RCT · OS |
| Jul 2013 | Not recommended | Best Available Therapy (BAT) | $45k–75k | RCT · Spleen volume reduction |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| REACH3 | Ph 3 | 330 | Efficacy of Ruxolitinib Versus Investigator's Choice Best Available Therapy (BAT) in Parti… | completed |
| REACH2 | Ph 3 | 310 | Overall Response Rate (ORR) at Day 28 | completed |
| JUMP | Ph 3 | 2,233 | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) up to 5… | completed |
| RESPONSE-2 | Ph 3 | 149 | Number of Participants Achieving Hematocrit (Hct) Control at Week 28 | completed |
Consumer voice
Consumers with polycythemia vera described the significant impact of the disease on their quality of life and identified side effects from currently available treatments as a key concern.
The PBAC noted the comments from individuals who would like to access the medicine to treat their own health condition described the impact of PV on their quality of life (QoL) and the contribution of side-effects from currently available Consumer comments · PSD
quality of lifeside effectsunmet needtreatment burden
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to polycythaemia vera only; TGA label includes myelofibrosis variants and graft-versus-host disease.