PBAC's latest decision on Alglucosidase alfa: Not recommended (2013). Considered for 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.
PBAC outcome
Not recommended
Not applicable
ICER (AUD/QALY)
$500k/QALY
$500k/QALY–— · across 4 submissions
Submissions
6
first 2008
Submissions
6
2008 → 2013
Eligible population
Patients 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.
Therapy area
Metabolic
Line of therapy
Not applicable
Evidence base
Single-arm
Primary endpoint
OS
Pivotal trial size
283 patients
Key trials
LOTS, Güngör et al. (EMC/IPA Pompe survey)
Comparator
standard (palliative) therapy including intensive respiratory support, cardiac care, dietary therapy and rehabilitative services
Economic model
Not modelled
Budget impact
$10M
ICER note
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.
ICER basis
Range aggregates 4 submissions (2008–2013) — not a single base case.
Risk sharing
Risk-sharing arrangement in place — PBAC noted that dose escalation circumstances could be managed through a risk-sharing arrangement.
Why PBAC said no
Reasons cited in the latest PSD: 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
Jul 2008: Recommended with restriction — ICER $75k/QALY
Mar 2009: Not recommended — ICER $45k/QALY
Nov 2010: Not recommended — ICER $45k/QALY
Jul 2011: Deferred
Nov 2012: Not recommended
Mar 2013: Not recommended · Not applicable — ICER $500k/QALY