Alglucosidase AlfaMyozyme
Treatment of late-onset Pompe disease in patients with confirmed diagnosis based on enzymology or mutational analysis and clinical features of juvenile or late-onset disease.
Decisions on record
- Meeting Nov 2012 Not recommended Long-term treatment of patients with a confirmed diagnosis of Pompe disease (acid alfa-glucosidase deficiency).
- Meeting Jul 2011 Deferred Pompe disease
- Meeting Nov 2010 Not recommended Long-term treatment of patients with a confirmed diagnosis of Pompe disease (acid alfa-glucosidase deficiency).
- Meeting Nov 2009 Not recommended Long-term treatment of patients with a confirmed diagnosis of Pompe disease (acid alfa-glucosidase deficiency).
- Meeting Mar 2009 Not recommended Long-term treatment of patients with a confirmed diagnosis of Pompe disease (acid alfa-glucosidase deficiency).
- Meeting Jul 2008 Not recommended Pompe disease
Access path
- TGA registered · Myozyme
TGA label narrower than the PBS population
- Jul 2008Not recommended
Not rejected for PBS Highly Specialised Drug listing; rejected on basis of unacceptably high cost effectiveness.…
- Mar 2009Not recommended
unacceptably high cost effectiveness, uncertainty in extrapolating short-term surrogate outcomes (6MWT, FVC) to…
- ↻ resubmittedNov 2009Recommended
- Nov 2010Not recommended
Highly uncertain link between short-term surrogate outcomes (FVC, 6MWT) and patient survival; non-significant…
- Jul 2011Deferred
Evidence: RCT, observational studies → RCT | Other (case series, observational study)
- Nov 2012Recommended
Evidence: RCT | Other (case series, observational study) → Registry
- Nov 2012Recommended
Evidence: RCT | Other (case series, observational study) → Registry
- Mar 2013Recommended
Evidence: Registry → Single-arm
- PBS listing · Not applicable
From the public summary
For PBAC’s views, see Recommendations and Reasons. 10. Economic Analysis The submission presented a trial based economic evaluation in the form of a cost- effectiveness analysis, which was considered valid. The analysis only included Pompe disease in patients less than 26 weeks of age and no economic data for late-onset Pompe disease were presented. Quality of life was not considered.PSD · Jul 2008
The analysis used a time horizon of 52 weeks (date of birth to 52 weeks) i.e 52 weeks of treatment from the first infusion.PSD · Jul 2008
Cost-effectiveness
PBAC calculated that at the price proposed, the incremental cost per one life-year gained assuming one year of life extension for four years of treatment was substantially greater than $500,000; if one year gained for eight years of treatment, the incremental cost more than doubled. No formal economic evaluation was presented.
At the price proposed in the submission, based on EMC/IPA survey data, the incremental cost per one life-year gained assuming one year of life extension is gained for four years of treatment was substantially greater than $500, 000. PSD · 2013
Decision context
PopulationPatients with a confirmed diagnosis of late-onset Pompe disease based on enzymology or mutational analysis meeting treatment initiation criteria including impaired respiratory function or significant muscular weakness, excluding those who are invasive ventilator dependent, current smokers, or with other severe life-threatening diseases.
Risk sharingPBAC noted that dose escalation circumstances could be managed through a risk-sharing arrangement.
Why it was knocked back
- unacceptably high incremental cost-effectiveness ratio, insufficient certainty that drug substantially extends lifespan in late-onset Pompe disease, non-randomised observational data subject to residual confounding and unmeasured confounders, heterogeneous late-onset population, modest survival differences compared to infantile-onset disease, price unjustifiably high relative to outcomes
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2013 | Recommended | standard (palliative) therapy including intensive respiratory support, cardiac care, dietary therapy and rehabilitative | $500k | Single-arm · OS |
| Nov 2012 | Recommended | standard (palliative) therapy including intensive respiratory support, cardiac care, dietary therapy and rehabilitative | — | Registry · OS |
| Nov 2012 | Recommended | standard (palliative) therapy including intensive respiratory support, cardiac care, dietary therapy and rehabilitative | — | Registry · OS |
| Jul 2011 | Deferred | standard (palliative) therapy including intensive respiratory support, cardiac care, dietary therapy and rehabilitative | — | RCT | Other (case series, observational study) · Other (survival benefit based on FVC and 6MWT improvement, survival data from non-randomised comparison of treated vs untreated patients in the EMC/IPA survey population) |
| Nov 2010 | Not recommended | standard (palliative) therapy including intensive respiratory support, cardiac care, dietary therapy and rehabilitative | $15k–45k | RCT, observational studies · OS | PFS | DFS | ORR | QoL | Surrogate |
| Nov 2009 | Recommended | standard (palliative) therapy including intensive respiratory support, cardiac care, dietary therapy and rehabilitative | $15k–45k | RCT |
| Mar 2009 | Not recommended | standard (palliative) therapy including intensive respiratory support, cardiac care, dietary therapy and rehabilitative | $15k–45k | RCT · 6-minute walk test, percent predicted forced vital capacity |
| Jul 2008 | Not recommended | standard (palliative) care | $45k–75k | RCT, historical control · OS, invasive ventilator-free survival |
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricted to late-onset disease with specific treatment initiation criteria; TGA label includes all Pompe disease without phenotype or clinical severity restrictions.