Key takeaways
- TA636 / TA1069 NICE technology appraisal numbers for eculizumab (terminated, 2020) and efgartigimod (not recommended, June 2025) in generalised myasthenia gravis
- 0 cost-effectiveness dossiers formally reviewed by NICE for a complement inhibitor in myasthenia gravis; eculizumab's appraisal closed before one was submitted
- GID-TA10986 / ID4003 NICE's technology appraisal and project identifiers for the efgartigimod submission that closed as TA1069
- 9 sheets in the HTA Strategy Model: authority landscape, PICO framework, comparator defence, value-dossier self-assessment, HEOR gap register, economic model and submission timeline, client alignment questions
NICE has never modelled a cost-per-QALY for a myasthenia gravis biologic: eculizumab's appraisal closed before submission, efgartigimod's closed on evidence gaps rather than a quantified ICER breach, leaving a new entrant with no comparator or price benchmark to build against.
NICE's two myasthenia gravis biologic appraisals failed in different ways, and the difference matters for anyone building a new submission. Eculizumab's appraisal (TA636) was terminated in June 2020 after AstraZeneca chose not to submit a cost-effectiveness dossier, so no evidence was ever formally reviewed and no cost-per-QALY figure exists for any complement inhibitor in this indication. Efgartigimod's appraisal (technology appraisal GID-TA10986, project ID4003) ran to a full committee review and closed with final guidance TA1069 on 4 June 2025, but the committee's own conclusion cited gaps and uncertainties in the cost-effectiveness evidence rather than a specific quantified ICER breach. Its ADAPT trial showed a 68% MG-ADL responder rate, so the clinical result did not fail; the value case at the submitted price did. NICE never specified whether the comparator, the utility values, or the economic model structure was the primary driver of that judgment.
That combination leaves a new entrant with no reusable comparator, no reusable ICER, and no diagnosed failure point to correct, a materially harder starting position than an indication where at least one full appraisal set a price benchmark. The practical response starts well before the dossier is drafted. Sponsor the Myasthenia Gravis Association UK's refractory-patient survey roughly 12 months ahead of submission to generate the disease-burden and unmet-need evidence NICE's committee has twice found wanting, and engage the Association of British Neurologists' MG guideline committee 18-24 months pre-submission so the clinical-pathway assumptions in the economic model are agreed before they are challenged. Our gap register treats both appraisals as separate failure modes requiring separate defences, not one generic access gap.
UK myasthenia gravis NICE appraisal precedent — two different failure modes, no reusable comparator or ICER
| Agent | NICE TA | Appraisal Outcome | Reusable Precedent for New Entrant |
|---|---|---|---|
| Eculizumab | TA636 | Terminated, June 2020, before dossier submission | None. No evidence was ever formally reviewed |
| Efgartigimod | TA1069 (GID-TA10986, ID4003) | Not recommended, June 2025 — evidence gaps cited, no quantified ICER breach | None. Committee did not specify which evidence input drove the rejection |
Sources: NICE TA636 (eculizumab), termination decision, 2020; NICE TA1069 (efgartigimod, appraisal GID-TA10986, project ID4003), final guidance, 4 June 2025.
What this model answers
Every section answers a named commercial question your team is asking, scoped to your asset.
- Eculizumab's appraisal never reached a cost-effectiveness review and efgartigimod's was rejected on evidence gaps rather than a quantified ICER. Which precedent, if either, does a new submission actually inherit? • TA636's termination mechanics versus TA1069's evidence-gap rejection • why neither sets a reusable comparator or ICER benchmark • the comparator-defence approach this leaves for a new entrant
- What does NICE's finding of gaps and uncertainties in the cost-effectiveness evidence for efgartigimod actually mean for a new PICO framework and economic model? • The three candidate failure points (comparator choice, utility values, and economic model structure) NICE left undiagnosed • how the value-dossier self-assessment tests each before submission
- When should a sponsor engage patient groups and clinical societies ahead of a UK gMG submission, and what evidence does each generate? • MAGS UK refractory-patient survey timing (roughly 12 months pre-submission) and the Association of British Neurologists' guideline-committee engagement window (18-24 months pre-submission) • what each contributes to the dossier
What's inside
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1 · The Binding Constraint 2 pp
- Why the absence of any completed comparator or ICER precedent, not a specific evidence gap, is what a new submission must solve first
- Pressure-tested against the TA636 termination and TA1069 rejection before the rest of the model is built out
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2 · HTA Authority Landscape 3 pp
- NICE's technology appraisal process and how TA636 and TA1069 diverge in what each actually tested
- GID-TA10986/ID4003 appraisal mechanics and why termination and rejection require different defences
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3 · PICO Framework 3 pp
- Population, Intervention, Comparator, Outcomes built without a reusable within-indication comparator precedent
- Refractory and moderate-to-severe gMG population definitions drawn from the roughly 4,000-patient NICE-relevant pool
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4 · Comparator Defence 3 pp
- The 3-test comparator defence framework applied to a new complement-inhibitor or FcRn-class entrant
- Building the comparator case against the pyridostigmine plus immunosuppressant backbone with no prior NICE-accepted biologic comparator
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5 · Value Dossier Self-Assessment 3 pp
- 5-module, 15-check self-assessment against submission readiness
- Testing the dossier against the three undiagnosed failure points behind TA1069's rejection
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6 · HEOR Gap Register 3 pp
- Both appraisal failure modes scored separately by likelihood of being raised and impact if raised
- Submission-blocking versus manageable classification, and why an unsubmitted dossier and a cited evidence gap require different fixes
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7 · Economic Model & Submission Timeline 4 pp
- Economic model type selection and the IVIg cost-offset and subcutaneous-administration savings inputs
- Milestone timeline incorporating the MAGS UK survey (roughly 12 months pre-submission) and ABN engagement (18-24 months pre-submission)
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8 · Client Alignment Questions 2 pp
- The open HEOR and stakeholder-engagement questions your team must close before the dossier is finalised
Appendix and source ledger included · 45-minute analyst readout included with delivery
Custom model delivered in 72 hours.
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Commission this model
AXLRx delivers rare-disease HTA strategy models built for market access and HEOR teams navigating NICE precedent gaps and undiagnosed evidence failures. Custom model in 72 hours.
- 1. Submit your requestSpecify your indication, HTA bodies, and comparator scope.
- 2. Scoping callAXLRx analyst confirms comparator set, evidence-gap scope, and stakeholder-engagement timeline before building.
- 3. DeliveryResearch-verified HTA strategy model in 72 hours with optional analyst readout.
How AXLRx builds this model
Prepared by MoatRx analysts on the AXLRx commercial consulting engine.
Every AXLRx HTA strategy model is built from primary HTA-body sources: NICE technology appraisals and final guidance documents, not secondary summaries. Every comparator claim is pressure-tested through the 3-test defence framework before being accepted.
UK myasthenia gravis HTA sources: NICE TA636 (eculizumab, terminated 2020) and NICE TA1069 (efgartigimod, appraisal GID-TA10986, project ID4003, final guidance June 2025).
- NICE TA636 termination mechanics (no dossier submitted, no cost-effectiveness review conducted) verified against the live NICE guidance page
- NICE TA1069 final guidance, appraisal number GID-TA10986, and project ID4003 verified against the same NICE committee documents
- MAGS UK survey timing and Association of British Neurologists engagement window verified against AXLRx's UK myasthenia gravis launch-readiness research
Frequently asked questions
Deliverables What formats are included with every model?
Every commissioned HTA Strategy Model includes an editable 9-sheet Excel model (Cover, HTA Authority Landscape, PICO Framework, Comparator Defence, Value Dossier Self-Assessment, HEOR Gap Register, Economic Model & Submission Timeline, Client Alignment Questions, QC) and a PDF methodology brief. No PowerPoint deck, since an HTA strategy model is built to be worked in directly, not presented from. An optional 45-minute analyst readout call is included.
Sources How is the HTA evidence verified?
AXLRx builds from primary sources only: NICE technology appraisals and final guidance documents, not secondary summaries. Every appraisal outcome and evidence gap is independently verified before inclusion.
Customisation Can I scope this to a specific comparator set or evidence-generation plan?
Yes. The intake form captures your indication, target HTA bodies, and comparator set. A scoping call confirms scope, including pre-submission stakeholder-engagement timing, before research starts. Commission via the intake form to start.