— The evidence suggests belimumab and voclosporin represent important new treatment options for lupus nephritis; there is substantial uncertainty regarding long-term use and outcomes, but at estimated net prices, both drugs appear priced in alignment with ICER’s recommended health-benefit price benchmark ranges —

— At March 26 virtual meeting, the New England Comparative Effectiveness Public Advisory Committee will review the evidence, hear further testimony from stakeholders, and deliberate over these treatments’ comparative clinical effectiveness, other potential benefits, and long-term value for money —

BOSTON, March 12, 2021 – The Institute for Clinical and Economic Review (ICER) today released an Evidence Report assessing the comparative clinical effectiveness and value of belimumab (Benlysta®, GlaxoSmithKline) and voclosporin (Lupkynis™, Aurinia) for the treatment of lupus nephritis (LN). The FDA approved belimumab on December 17, 2020 and voclosporin on January 22, 2021.

“Lupus nephritis is a serious chronic disease, one that has a proportionately large impact on Black, Hispanic, and other communities of color in the US,” said Steven D. Pearson, MD, MSc, ICER’s President.  “Both belimumab and voclosporin are important new treatment options. Despite substantial uncertainty that remains about longer-term use and outcomes with both treatments, their estimated net price in the market is aligned with their anticipated clinical benefits for patients. At our public meeting, we will engage patient representatives, clinical experts, payers, and the drug makers to discuss how the evidence can best support appropriate care and coverage. We will also tackle how to make changes so that new treatments like these are studied in more diverse patient populations to ensure that patients and clinicians have the information they need to tailor care for each individual.” 

This Evidence Report will be reviewed at a virtual public meeting of the New England Comparative Effectiveness Public Advisory Council (New England CEPAC) on March 26, 2021. The New England CEPAC is one of ICER’s three independent evidence appraisal committees comprising medical evidence experts, practicing clinicians, methodologists, and leaders in patient engagement and advocacy.

A draft version of this report was previously open for a four-week public comment period. The updated Evidence Report and voting questions reflect changes made based on comments received from patient groups, clinicians, drug manufacturers, and other stakeholders. Detailed responses to public comments can be found here.

Key Clinical Findings

In the clinical trials, belimumab increased the complete renal response and primary efficacy renal response at two years compared with standard therapy alone, with benefits seen after the first year appearing stable at year two. There were no significant increases in adverse events or discontinuations compared with standard induction therapy for LN.

Voclosporin nearly doubled the complete response and markedly increased the partial response at one year compared with standard therapy alone. Adverse events were comparable to standard induction therapy for LN, but the FDA added a black box warning consistent with that of cyclosporin for possible serious infections and malignancies.

Based on this evidence, ICER determined that there is high certainty that both belimumab and voclosporin provide at least a small net health benefit compared to usual care, with the possibility of a substantial benefit (“B+”).

Key Cost-Effectiveness Findings

ICER’s recommended health-benefit price benchmark (HBPB) range for belimumab is between $45,000-$61,000 per year, while ICER estimates that belimumab’s actual annual net US price is approximately $43,000 for patients remaining on treatment. ICER’s HBPB for voclosporin is between $72,000-$101,000 per year, while ICER estimates that voclosporin’s actual annual net US price is $92,000 for patients remaining on treatment.    

The HBPB is a price range suggesting the highest US price a manufacturer should charge for a treatment, based on the amount of improvement in overall health patients receive from that treatment, when a higher price would cause disproportionately greater losses in health among other patients in the health system due to rising overall costs of health care and health insurance. In short, it is the top price range at which a health system can reward innovation and better health for patients without doing more harm than good.

About ICER

The Institute for Clinical and Economic Review (ICER) is an independent non-profit research institute that produces reports analyzing the evidence on the effectiveness and value of drugs and other medical services. ICER’s reports include evidence-based calculations of prices for new drugs that accurately reflect the degree of improvement expected in long-term patient outcomes, while also highlighting price levels that might contribute to unaffordable short-term cost growth for the overall health care system.

ICER’s reports incorporate extensive input from all stakeholders and are the subject of public hearings through three core programs: the California Technology Assessment Forum (CTAF), the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), and the New England Comparative Effectiveness Public Advisory Council (New England CEPAC). These independent panels review ICER’s reports at public meetings to deliberate on the evidence and develop recommendations for how patients, clinicians, insurers, and policymakers can improve the quality and value of health care. For more information about ICER, please visit ICER’s website.