FampridineFampyra
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).
Decisions on record
- Meeting Mar 2014 Not recommended Fampyra® Biogen Idec Australia Pty Ltd Indicated for the symptomatic improvement of walking ability in adult patients with multiple sclerosis (MS). Not currently PBS listed Listing Requested: Authority Required listing for the treatment for the symptomatic improvement of walking ability of an ambula
- Meeting Nov 2012 Not recommended Multiple sclerosis
Access path
- TGA registered · Fampyra
TGA label narrower than the PBS population
- 2012Not recommended
Modest absolute treatment effect of uncertain clinical relevance, questionable clinical significance of walking speed…
- Nov 2012Not recommended
Modest and uncertain absolute treatment effect with questionable clinical relevance; proposed T25FW restriction would…
- Mar 2014Not recommended
insufficient evidence of clinically relevant absolute treatment effect, high placebo response rate (50.8% in placebo vs…
Cost-effectiveness
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.
Listing was sought on a cost-utility basis with fampridine compared to placebo. Utility was measured in quality adjusted life years (QALYs) gained. PSD · 2014
Decision context
PopulationAmbulatory patients with clinically definite multiple sclerosis with EDSS score 4 to <7, confirmed by MRI, with baseline MSWS-12 score recorded.
Why it was knocked back
- 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)
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2014 | Not recommended | placebo (as a proxy for best supportive care) | — | RCT · Walking ability (timed 25-foot walk test, MSWS-12) |
| Nov 2012 | Not recommended | placebo | $15k–45k | RCT |
| 2012 | Not recommended | placebo | $15k–45k | RCT · any improvement in walking speed in at least three out of four measurements during the trial |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| MS-F203 | Ph 3 | 269 | Summary of Treatment Emergent Adverse Events (TEAE). | completed |
| MS-F204 | Ph 3 | 214 | Summary of Treatment Emergent Adverse Events (TEAE). | completed |
| ENABLE | Ph 4 | 901 | Change From Baseline in the Physical Component Scale (PCS) of the Short Form 36 Health Sta… | completed |
Consumer voice
The PBAC received input from 115 individuals, 6 health care professionals, and 4 organisations describing benefits of fampridine treatment including improved mobility, quality of life, reduced fatigue, and improved access to treatment. Support was noted from MS Australia, neurologist associations, and MS research organisations.
The PBAC noted and welcomed the input from individuals (115), health care professionals (6) and organisations (4) via the Consumer Comments facility on the PBS website. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricted to ambulatory patients with EDSS 4-7, MRI confirmation, and baseline MSWS-12 documented; TGA label covers all adult MS patients.