Elosulfase AlfaVIMIZIM
Treatment of patients with mucopolysaccharidosis type IVA (MPS IVA; Morquio A Syndrome).
Decisions on record
- Meeting Mar 2016 Not recommended The treatment of mucopolysaccharido sis type IVA (MPS IVA; Morquio A syndrome).
- Meeting Nov 2014 Not recommended 5 mg/5 mL injection, 5 mL vial Vimizim® Biomarin Pharmaceuticals Australia Pty Ltd New listing (Major submission) Mucopolysaccharidosis type IVA Section 100 (Highly Specialised Drugs) listing for the treatment of mucopolysaccharidosis type IVA in patients who meet certain criteria. On the basis of d
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- TGA registered · VIMIZIM
TGA label narrower than the PBS population
- Nov 2014Not recommended
vs placebo in combination with standard medical management
- Mar 2016Not recommended
unclear clinically significant clinical benefit compared to placebo, unreliable and unacceptably high ICER (more than…
From the public summary
7.1 The PBAC did not recommend the Authority Listing Section 100 PBS listing for elosulfase alfa for the treatment of patients with mucopolysaccharidosis (MPS) IVA (Morquio A Syndrome). The PBAC considered that while there was sufficient basis to conclude that treatment with elosulfase alfa results in a small but meaningful clinically improvement, the cost effectiveness of the drug was highly uncertain and unacceptable.PSD · Mar 2016
7.2 In the context of of the discussion of the impacts of Morquio A syndrome at the consumer hearing the PBAC considered that the benefits captured by the 6MWT are small but likely to be clinically meaningful. In addition the analysis of the adjusted MOR-004/005 and MorCAP analysis suggests that this response is likely to be sustained. The clinical effect of elosulfase alfa treatment in patients under 5 years of age was less certain.PSD · Mar 2016
6.28 The re-submission presented a cost-utility analysis over a 50-year time horizon, compared to a cost-effectiveness model with a 24-week time horizon in the previous submission. A summary of the model structure and rationale is presented in Table 7.PSD · Mar 2016
The ESC considered that the time horizon was appropriate in the context of a life- long disease, but that there was considerable uncertainty associated with extrapolation from the 24 week trial data out to 50 years. 13PSD · Mar 2016
Slightly greater incidence of adverse events PBAC Comment: Superior efficacy not accepted, inferior safety accepted 3PSD · Mar 2016
ICER: more than PBAC Comment: Economic evaluation resulted in $200,000/QALY unacceptably high ICER and was unreliable: • Applicability trial population • Duration economic evaluation • Cost of AEs excludedPSD · Mar 2016
6.8 The PBAC noted and welcomed the input from individuals (444), health care professionals (5) and organisations (1) via the Consumer Comments facility on the PBS website. The comments described a range of benefits of treatment with Elosulfase Alfa including an increase in mobility, stamina and strength, and reduced need for surgery.PSD · Mar 2016
6.9 The PBAC noted the advice received from Mucopolysaccharide & Related Diseases Society Australia (MPS Australia) clarifying the likely use of elsulfase alfa in clinical practice. The PBAC specifically noted the advice that the use of elsulfase alfa may reduce frequency and length of hospital stays and improve the quality of life of patients.PSD · Mar 2016
Cost-effectiveness
ICER stated as 'more than $200,000/QALY' but exact bounds not specified; reported as redacted/commercially sensitive in the cost table.
Decision context
PopulationPatients with mucopolysaccharidosis type IVA (Morquio A Syndrome) aged 5 years and over with baseline 6-minute walk test distance of 30–325 metres, as per the MOR-004 trial population; the re-submission proposed inclusion of all children under age 5 years without continuation criteria until age 5.
Risk sharingPay-for-performance arrangements proposal with a cap at redacted vials per injection.
Why it was knocked back
- unclear clinically significant clinical benefit compared to placebo, unreliable and unacceptably high ICER (more than $200,000/QALY), uncertain clinical trial population applicability, concerns about duration of economic evaluation, cost of adverse events excluded from analysis, unreliable utility values
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2016 | Not recommended | placebo in combination with standard medical management | — | RCT · 6MWT |
| Nov 2014 | Not recommended | placebo in combination with standard medical management | — | RCT · Other |
Consumer voice
Consumers reported multiple benefits of Elosulfase Alfa treatment including increased mobility, stamina and strength, reduced need for surgery, and potential reduction in hospital stays with improved quality of life.
The comments described a range of benefits of treatment with Elosulfase Alfa including an increase in mobility, stamina and strength, and reduced need for surgery. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricted to age ≥5 years with baseline 6MWT 30–325 metres, versus TGA label covering all MPS IVA patients.