The effectiveness and value of belimumab and voclosporin for lupus nephritis

Systemic lupus erythematosus (SLE) is an autoimmune disease that affects between 300,000 and 1.5 million Americans.1 It is more common in women (90% of diagnosed cases) and in non-Whites (4 times higher prevalence in Black patients; 2 times higher prevalence in Hispanic patients). Approximately half of patients with SLE will be diagnosed with lupus nephritis (LN), characterized by inflammation in the kidney, proteinuria, and progressive kidney damage that can lead to kidney failure.2,3 LN typically presents in patients who are aged 20-40 years,4,5 and it is the most common cause of death and disability in patients with SLE. Guidelines for the treatment of LN recommend induction therapy with high-dose corticosteroids combined with either mycophenolate mofetil (MMF) or cyclophosphamide, followed by maintenance therapy with MMF.6,7 Unfortunately, fewer than half of patients with LN respond to current combination therapy, so there is a large unmet need for new therapies. The US Food and Drug Administration (FDA) recently approved 2 new therapies for LN. Belimumab, a parenteral B-lymphocyte inhibitor already approved by the FDA for SLE, was approved for LN in December 2020. Voclosporin, an oral calcineurin inhibitor that is reported to be safer than other calcineurin inhibitors (less kidney damage), was approved in January 2021. The Institute for Clinical and Economic Review (ICER) conducted a systematic literature review and cost-effectiveness analysis to evaluate the health and economic outcomes of belimumab and voclosporin to treat LN. Complete details of ICER’s systematic literature search and protocol, as well as the methodology and model structure for the economic evaluation, are available on ICER’s website. Here, we present the summary of our findings and highlights of the policy discussion with key stakeholders held at a public meeting of the New England Comparative Effectiveness Public Advisory Council on March 26, 2021. The detailed report is available on the ICER website at https://icer.org/wp-content/uploads/2020/11/ICER_Lupus-Nephritis_Final-Evidence-Report_041621.pdf