VosoritideVOXZOGO
Treatment of patients with achondroplasia whose epiphyses are not closed.
Decisions on record
- Meeting Nov 2022 Recommended Achondroplasia with open epiphyses no PSD
- Meeting Sep 2022 Deferred Achondroplasia in patients whose epiphyses are not closed no PSD
- Meeting Jul 2022 Not recommended Achondroplasia in patients whose epiphyses are not closed
Access path
- TGA registered · VOXZOGO
TGA label narrower than the PBS population
- Jul 2022Deferred
vs Best supportive care (BSC)
- Nov 2022Not recommended
unacceptably high ICER at proposed price, uncertain uptake rates, safety of vosoritide inferior to placebo, quality use…
From the public summary
The PBAC did not recommended vosoritide for the treatment of patients with achondroplasia whose epiphyses are not closed. The PBAC recognised that there are no treatments on the PBS available specifically for this condition, and it considered that the addition of vosoritide offered high added therapeutic value.PSD · Jul 2022
The PBAC noted the input from individuals and health care professionals highlighting the impact of an increase in final height in terms of accessing environment, improved functionality and exercise tolerance along with the potential for reduced pain and medical complications. The PBAC acknowledged there are currently no treatment options available to patients.PSD · Jul 2022
The submission presented a stepped economic evaluation including a modelled cost- utility analysis (CUA) comparing vosoritide with BSC based on a direct randomised trial (study 111-301). This is appropriate given the claim of superior efficacy of vosoritide against placebo.PSD · Jul 2022
The submission presented a cohort simulation model that included two health states (alive and dead) capturing patient mortality and complications via risks applied each cycle. Health-related QoL was incorporated in the model via the application of continuous health specific utility values (based on patient’s height relative to the age- and sex-matched general population) to patients remaining alive each cycle and utility decrements to patients …PSD · Jul 2022
The submission described vosoritide as superior in terms of effectiveness compared with placebo. The claim is appropriate and well-supported in 5 to < 18 year old children, but not well-supported in children under 5 years old. The key issues were:PSD · Jul 2022
Data from the one trial to provide data on children under 5 years of age (Study 111-206) included only eight sentinel intervention patients with no controlled comparison. The PSCR provided additional data from the randomised treatment arms for children 0-5 years from Study 111-206 (see paragraph 6.15).PSD · Jul 2022
The comments from individuals who would like access to the medicine to treat their own health condition described the experience of living with achondroplasia.PSD · Jul 2022
Experiences included difficulties in reaching objects, with manoeuvring and with coordination. The individuals also described suffering from joint and back pain along with other comorbidities and the medical treatment required to address these issues.PSD · Jul 2022
Cost-effectiveness
ICER stated as 'unacceptably high at the proposed price' but the numeric value is redacted/not published in the public document.
The PBAC considered the incremental cost effectiveness ratio (ICER) in this setting was unacceptably high at the proposed price, even in the context of relatively small patient numbers and high added therapeutic value. PBAC · 2022
Decision context
PopulationPatients with achondroplasia whose epiphyses are not closed (age-agnostic, though evidence strongest in children 5 to <18 years).
Risk sharingThe sponsor indicated amenability to discussing appropriate risk-sharing arrangements to manage uncertainties associated with estimated uptake.
Why it was knocked back
- unacceptably high ICER at proposed price, uncertain uptake rates, safety of vosoritide inferior to placebo, quality use of medicines concerns regarding training for prescribers and carers
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2022 | Not recommended | best supportive care (BSC) | — | RCT · AGV |
| Jul 2022 | Deferred | Best supportive care (BSC) | — | RCT · Annual growth velocity |
Consumer voice
Individuals with achondroplasia and their families described difficulties with daily activities, mobility, and pain, and anticipated benefits from height gains including increased independence, participation in school activities, and improved access to environments. Healthcare professionals noted observed improvements in functionality, exercise tolerance, quality of life, and potential reduction i
Experiences included difficulties in reaching objects, with manoeuvring and with coordination. The individuals also described suffering from joint and back pain along with other comorbidities and the medical treatment required to address these issues. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to first-line use, while TGA label contains no line-of-therapy restriction.