AmifampridineRUZURGI
Recommended NeurologyAuthority RequiredNot applicable line 💬 consumer voice
Treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults and children aged 6 years and above.
2
Submissions
1 resub
2021–22
On the record
$75k–95k
ICER range
1 sourced ICER · 2022
ICER stated
Cost basis
Decisions on record
- Meeting Mar 2022 Recommended Lambert-Eaton myasthenic syndrome
- Meeting Nov 2021 Not recommended Lambert-Eaton myasthenic syndrome
Access path
- TGA registered · RUZURGI
TGA label equal than the PBS population
- Nov 2021Not recommended
vs placebo
- ↻ resubmittedMar 2022Recommended · restricted
- PBS listing · Authority Required
RecommendedDeferredNot recommended
From the public summary
PBAC outcome
5.1 The PBAC recommended the listing of amifampridine with an Authority Required listing (Telephone/Online) for the treatment of Lambert-Eaton myasthenic syndrome (LEMS). The resubmission provided a revised economic model and financial estimates in response to previous concerns raised by the PBAC.PSD · Mar 2022
5.2 The PBAC was satisfied that amifampridine provides, for some patients, a significant improvement in efficacy over placebo. The PBAC recalled from its November 2021 consideration that a claim of superior comparative effectiveness was reasonable for amifampridine compared with placebo, however the magnitude of the effect was uncertain due to the limitations of the available clinical evidence.PSD · Mar 2022
Economic analysis
4.4 As an early re-entry resubmission, the economic analysis has not been independently evaluated.PSD · Mar 2022
4.5 A summary of the key matters to be addressed by the resubmission is presented in Table 1. 4PSD · Mar 2022
Clinical claim
The submission described amifampridine as superior in terms of effectiveness compared with placebo. The evaluation and ESC considered this claim was partially supported, but the magnitude of the effect was likely overestimated and uncertain.PSD · Nov 2021
The key issues were: Small patient numbers (N=32 for DAPPER; N=26 for DUKE) and short duration of evaluation (up to 3.5 days for DAPPER; 6 days for DUKE). The evaluation noted it is challenging to conduct larger trials due to LEMS being a rare condition, and the ethical concerns associated with placebo trials when there is a known treatment. Inadequate justification for the clinical significance of thresholds related to 3TUG and QMG.PSD · Nov 2021
Consumer comments
The HCP noted the rarity of LEMS but stated that diagnosis can usually be reached with certainty. The HCP described the positive impact that amifampridine has on muscle strength and quality of life for patients receiving treatment, and how they rely on it for their daily function. The HCP stated that amifampridine is generally well tolerated and appears to be without major long-term side effects or challenges in its prescribing.PSD · Nov 2021
The PBAC noted the advice received from Myasthenia Alliance Australia (MAA) clarifying the likely use of amifampridine in clinical practice. The MAA stated that feedback from people suffering with LEMS indicated that amifampridine is effective for a range of users and the free base form appears well tolerated. The MAA also shared concerns that not having access to amifampridine would be detrimental to health outcomes and quality of life.PSD · Nov 2021
Cost-effectiveness
The PBAC noted that the pre-PBAC response for the resubmission provided a price reduction resulting in an ICER of $75,000 to <$95,000 /QALY, consistent with PBAC advice from its November 2021 consideration that the cost effectiveness of amifampridine would be considered acceptable if the pre-PBAC response assumptions were used and the ICER was less than $85,000/QALY gained. PBAC · 2022
ICER uncertainEconomic model disputedPrice cut / RSA neededCost-effectiveness accepted
Decision context
PopulationAdults and children aged 6 years and above with Lambert-Eaton myasthenic syndrome (LEMS), including both paraneoplastic LEMS (pLEMS) and autoimmune LEMS (aLEMS).
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2022 | Recommended · restricted | placebo | $75k–95k | RCT · QoL |
| Nov 2021 | Not recommended | placebo | — | RCT · Surrogate |
Consumer voice
No consumer comments were received for the resubmission. Previously, the PBAC received input from 9 individuals, 1 healthcare professional, and 1 organisation regarding amifampridine consideration at the November 2021 meeting.
The PBAC noted that no consumer comments were received for this item in relation to the resubmission. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label equal than PBS population — Both TGA and PBAC cover identical population: adults and children aged 6+ with LEMS, with PBAC explicitly clarifying both pLEMS and aLEMS subtypes.