MiglustatOPFOLDA

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.

The PBAC considered that the evidence provided was not sufficient to support the validity and reliability of the four scales used in the studies. Additionally, given the lack of blinded outcome assessment there is potential for investigator/assessor bias in the assessment of the subjective outcome of swallowing function. PBAC · 2011

Populationadults and paediatric patients with Niemann-Pick disease Type C (NP-C) disease

• 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

Label narrower than PBS population — PBAC restricts to patients who cannot tolerate or failed ERT; TGA label includes those for whom ERT is simply not an option.