BelimumabBenlysta
Add-on therapy for active, autoantibody-positive systemic lupus erythematosus (SLE) with a high degree of disease activity despite standard therapy.
Decisions on record
- Meeting Jul 2020 Not recommended Belimumab is indicated as add-on therapy for reducing disease activity in adult patients with active, autoantibody- positive systemic lupus erythematosus (SLE) with a high degree of disease activity despite standard therapy.
- Meeting Nov 2019 Not recommended Systemic lupus erythematosus (SLE)
Access path
- TGA registered · Benlysta
TGA label narrower than the PBS population
- Nov 2019Not recommended
vs standard of care (SOC)
- Jul 2020Not recommended
Comparator changed: standard of care (SOC) → placebo
From the public summary
7.1 The PBAC did not recommend belimumab for the treatment of patients with active auto-antibody positive SLE with a high degree of disease activity despite ongoing standard therapy. The PBAC maintained its view from November 2019 that the evidence demonstrated a modest clinical benefit in a subset of patients. The PBAC 40PSD · Jul 2020
7.2 The PBAC acknowledged the clinical need for effective treatments for SLE, particularly for the group of patients who are not responding to current therapies. The PBAC noted the consumer comments describing the desire for a specific treatment option in this setting.PSD · Jul 2020
Model The model did not differentiate between responders and The resubmission restructured the SOC arm to structure non-responders in the SOC arms. split the responders and non-responders.PSD · Jul 2020
SDI The model assumed that treatment with belimumab The resubmission continued to assume that progression reduced the rate of SDI progression and consequently belimumab would reduce the rate of SDI reduced mortality. The PBAC considered that the progression. submission had not adequately demonstrated an The resubmission maintained that SLE disease improvement in SDI progression (para 6.23, Nov 2019 activity and damage is overall associated with PSD).PSD · Jul 2020
Inferior safety Abbreviations: BILAG, British Isles Lupus Assessment Group; FACIT, Functional Assessment of Chronic Illness Therapy; HRQoL, health- related quality of life; PGA, Physician Global Assessment; SC, subcutaneous; SDI, Systemic Lupus International Collaborating Clinics (SLICC)/ACR Damage Index; SELENA-SLEDAI, Safety of Estrogens in Lupus Erythematosus National Assessment version of the Systemic Lupus Erythematosus Disease Activity Index.PSD · Jul 2020
6.38 The resubmission described belimumab as superior in terms of effectiveness and inferior in terms of safety compared with placebo when used in combination with SOC.PSD · Jul 2020
6.3 Four of the 7 comments from individuals were from people who are living with SLE; they stated that access and affordability are important for pain relief and improvements in quality of life.PSD · Jul 2020
6.4 Three HCPs working in the SLE therapeutic area supported the PBS listing of belimumab. One stated that there is good evidence that the drug has utility in SLE, and there are many patients who have suboptimal control who would benefit from this medication.PSD · Jul 2020
Cost-effectiveness
ICER redacted; document states economic model was presented but values are not disclosed in public summary
Decision context
PopulationAdult patients with active SLE with a high degree of disease activity despite standard therapy, confirmed by elevated anti-dsDNA titre or low complement levels, SLEDAI-2K score ≥10, currently receiving hydroxychloroquine, immunosuppressant medication, and prednisolone ≥7.5 mg/day
Risk sharingRisk Sharing Arrangements (RSA) mentioned in context of potential leakage in severe lupus nephritis due to increased use for renal patients
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Jul 2020 | Not recommended | placebo | — | RCT · SRI (Systemic Lupus Erythematosus Responder Index) |
| Nov 2019 | Not recommended | standard of care (SOC) | — | RCT · SRI |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| BLISS-SC | Ph 3 | 839 | Percentage of Par. Achieving a SLE Responder Index (SRI) Response Rate at Week 52 | completed |
| EMBRACE | Ph 4 | 503 | Percentage of Participants Achieving a Systemic Lupus Erythematosus Responder Index (SRI) … | completed |
| BLISS-LN | Ph 3 | 448 | Double-blind Period: Percentage of Participants With Primary Efficacy Renal Response (PERR… | completed |
| BLISS-52 | Ph 3 | 865 | SLE Responder Index (SRI) Response Rate at Week 52 | completed |
| BLISS-76 | Ph 3 | 819 | SLE Responder Index (SRI) Response Rate at Week 52 | completed |
Consumer voice
Consumer input from individuals with SLE, healthcare professionals, and organisations emphasised the importance of access and affordability for pain relief and quality of life improvements. Healthcare professionals highlighted belimumab's utility for patients with suboptimal disease control, noting its potential to reduce steroid toxicity, while organisations stressed the disproportionate burden o
access and affordability are important for pain relief and improvements in quality of life Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to specific biomarker criteria (anti-dsDNA or low complement), SLEDAI-2K ≥10, and concurrent standard therapies; TGA label lacks these specifications.