PBAC's latest decision on Miglustat: Not recommended (2011). Considered for Treatment of progressive neurological manifestations in adults and paediatric patients with Niemann-Pick disease Type C (NP-C) disease.
PBAC outcome
Not recommended
2011
ICER (AUD/QALY)
Not modelled
no economic evaluation
Submissions
3
first 2008
Submissions
3
2008 → 2011
Eligible population
adults and paediatric patients with Niemann-Pick disease Type C (NP-C) disease
No updated modelled economic evaluation was presented. At July 2010 PBAC meeting, PBAC accepted that miglustat is unlikely to be cost-effective, but no specific ICER value stated in this re-submission.
ICER (historical)
$10.0M/QALY in an earlier submission (2010) — the latest submission carried no numeric base case. No single PSD states this combined range; see source PSDs.
Why PBAC said no
Reasons cited in the latest PSD: Clinical evidence did not demonstrate miglustat superior to placebo in effectiveness; videofluoroscopy swallowing studies lacked blinded outcome assessment and control groups with no consistent improvement shown; case series data based on small patient numbers with large variability and unvalidated scales; survival analysis comparison between treated and untreated cohorts had unmatched baseline characteristics and unclear comparability, confounding cannot be excluded; chain of argument from dysphagia treatment effect to lifespan extension via aspiration pneumonia reduction not sufficiently established; applicability of dysphagia-pneumonia associations to NP-C patients uncertain; failed to meet LSDP eligibility criteria 4 and 5