PBAC's latest decision on Fampridine: Not recommended (2014). Considered for Symptomatic improvement of walking ability in ambulatory patients with clinically definite multiple sclerosis who meet specified criteria (EDSS score 4-7, confirmed MS diagnosis by MRI, baseline MSWS-12 recorded).
PBAC outcome
Not recommended
Authority Required
ICER (AUD/QALY)
Not modelled
no economic evaluation
Submissions
2
first 2012
Submissions
2
2012 → 2014
Eligible population
Ambulatory patients with clinically definite multiple sclerosis with EDSS score 4 to <7, confirmed by MRI, with baseline MSWS-12 score recorded.
Therapy area
Neurology
Line of therapy
Any
Evidence base
RCT
Primary endpoint
Walking ability (timed 25-foot walk test, MSWS-12)
Pivotal trial size
638 patients
Key trials
MS-F202, MS-F203, MS-F204, DER-401, ENABLE
Comparator
placebo (as a proxy for best supportive care)
Economic model
CUA
ICER note
ICER not stated in document. Economic evaluation based on cost-utility analysis with QALYs, but no numeric ICER value is presented in the public summary.
ICER (historical)
$15k/QALY–$45k/QALY in an earlier submission (2012) — 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: insufficient evidence of clinically relevant absolute treatment effect, high placebo response rate (50.8% in placebo vs 55.4-57.0% in fampridine groups for 6-point MSWS-12 improvement) undermining responder identification, proposed PBS restriction would not adequately limit access to patients who truly respond, ENABLE study comparison inadequate (single-arm open-label, biased in favour of fampridine), lack of clinically meaningful outcome measures (fatigue, cognitive function not directly assessed)