AnifrolumabSaphnelo
Treatment of patients with severe systemic lupus erythematosus (SLE) with high disease activity (SLEDAI-2K ≥10) despite standard of care (triple therapy comprising an antimalarial, immunosuppressant, and oral corticosteroid ≥7.5 mg/day).
Decisions on record
- Meeting Mar 2024 Recommended Systemic lupus erythematosus (SLE) with high disease activity despite standard therapy
- Meeting Mar 2023 Not recommended Systemic lupus erythematosus (SLE) with severe disease activity despite standard therapy
- Meeting Jul 2022 Not recommended Systemic lupus erythematosus with high disease activity despite standard therapy
Access path
- TGA registered · Saphnelo
TGA label narrower than the PBS population
- Jul 2022Not recommended
vs Standard of care alone (placebo)
- Mar 2023Not recommended
Comparator changed: Standard of care alone (placebo) → SOC alone (placebo)
- ↻ resubmittedMar 2024Recommended · restricted
Comparator changed: SOC alone (placebo) → Standard of care alone (placebo)
- PBS listing · Authority Required
From the public summary
7.1 The PBAC recommended a complex authority required (CAR) Section 100 Highly Specialised Drugs (S100 HSD) Authority Required (In Writing/HPOS) listing of anifrolumab for the treatment of patients with severe systemic lupus erythematosus (SLE) with high disease activity despite standard of care (SOC).PSD · Mar 2024
7.2 The PBAC noted that the impact of SLE is significant for patients and considered there is a clinical need for effective treatments in the requested population. The PBAC noted the strong consumer support for this item and considered that some of the impacts described are difficult to measure in clinical trials, such as quality of life impacts associated with fluctuating disease severity.PSD · Mar 2024
6.35 The resubmission presented a new stepped economic evaluation starting with a trial- based cost per responder analysis informed by TULIP 1 and 2 and then implementing a modelled cost-utility analysis using evidence from TULIP 1 and 2, TULIP LTE and the Australian cohort of the Asia-Pacific Lupus Collaboration (APLC).PSD · Mar 2024
6.36 The March 2023 resubmission presented an ICER of $75,000 to < $95,000 per additional QALY gained. The PBAC had previously expressed concern that the March 2023 model did not provide robust estimates and had not adequately addressed the concerns with the July 2022 model (paragraph 7.7, anifrolumab PSD, March 2023).PSD · Mar 2024
prednisone (or equivalent)), anifrolumab 300 mg IV Q4W added to SOC has superior effectiveness and inferior (manageable) safety compared to SOC alone. Source: Table 1-6, p40 of the resubmission.PSD · Mar 2024
AZA=azathioprine; BICLA=BILAG–Based Composite Lupus Assessment; BILAG=British Isles Lupus Assessment Group; CLASI=Cutaneous Lupus Erythematosus Disease Area and Severity Index; HRQoL=health related quality of life; LLDAS=low lupus disease activity state; MTX=methotrexate; OCS=oral corticosteroids; PGA=physician’s global assessment; SDI=SLICC/ACR (Systemic Lupus International Collaborating Clinics/American College of Rheumatology) Damage Index; …PSD · Mar 2024
6.3 The PBAC noted the consumer input was consistent with the previously considered comments. Health professionals supported the proposed listing and described an unmet clinical need for the group of patients who have refractory disease, despite other therapies. Individuals described the significant impact of SLE on patient quality of life and noted that anifrolumab treatment would be unavailable to most, in the absence of PBS listing due to cost.PSD · Mar 2024
6.4 The PBAC noted the advice received from the Australasian Society of Clinical Immunology and Allergy (ASCIA), the Australian Rheumatology Association (ARA), Lupus WA, CreakyJoints Australia (and parent organisation, Global Healthy Living Foundation Australia), and Dragon Claw Charity Ltd. All organisations supported the proposed listing.PSD · Mar 2024
Cost-effectiveness
ICER value redacted in public document; economic model presented but specific ICER figure not publicly stated
The PBAC agreed with the evaluation that the comparatively short trial follow-up (52 weeks vs 30-year time horizon) and the lack of direct evidence on organ damage and mortality results in an uncertain ICER. PBAC · 2022
Decision context
PopulationAdult patients (≥18 years) with severe systemic lupus erythematosus with persistent disease activity (SLEDAI-2K ≥10) despite standard of care triple therapy (hydroxychloroquine, immunosuppressant, and prednisolone ≥7.5 mg/day), excluding those with severe active lupus nephritis or severe active CNS lupus.
Risk sharing2-tier Risk Sharing Agreement (RSA) proposed to address uncertainty in patient population size and response rates in clinical practice
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2024 | Recommended · restricted | Standard of care alone (placebo) | — | RCT · LLDAS response |
| Mar 2023 | Not recommended | SOC alone (placebo) | — | RCT · SRI(4) / BICLA response |
| Jul 2022 | Not recommended | Standard of care alone (placebo) | — | RCT · Other |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| TULIP-LTE (NCT02794285) | Ph 3 | 559 | Exposure-adjusted Incidence Rates (EAIRs) of Adverse Events of Special Interest (AESIs) | completed |
Consumer voice
Health professionals and patient organisations supported PBS listing of anifrolumab for SLE, describing unmet clinical need for refractory disease and significant patient quality-of-life impacts. Individuals noted that anifrolumab would be unavailable to most patients without PBS listing due to cost.
Health professionals supported the proposed listing and described an unmet clinical need for the group of patients who have refractory disease, despite other therapies. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to severe SLE with SLEDAI-2K ≥10 despite triple therapy; TGA label includes moderate-severe SLE on standard therapy without these specifications.