— For belimumab and voclosporin, independent appraisal committee votes highlight the importance of several potential benefits of both treatments for patients and families beyond health improvement —

— The majority of the committee votes that the long-term value for money at current pricing is high for belimumab and intermediate for voclosporin —

— At policy roundtable, experts emphasize the need for steps to reduce inequity in access to new therapies, including reductions in insurance barriers, avoidance of extending exclusivity periods through patented dosing algorithms, and by ensuring broader representation in clinical studies of racial and ethnic communities that bear a disproportionate burden from lupus nephritis —

BOSTON, April 16, 2021 – The Institute for Clinical and Economic Review (ICER) today released a Final 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 disproportionately 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 remaining uncertainty about both treatments’ longer-term outcomes, their estimated prices appear to be aligned with their anticipated clinical benefits.  More research is needed to confirm these benefits, but for patients and clinicians to have responsibly priced options specifically indicated for lupus nephritis is a major win for patients and the entire health system.”

“One policy element that was discussed at the public meeting, however, is worth attention. Aurinia sought and achieved a late-stage patent on voclosporin’s dosing protocol. This unusual patent will likely deny patients access to a more affordable generic version of the treatment until 2037 – a full 10 years beyond what was previously expected.  If extended patents through dosing algorithms becomes a new trend, we could see future budget headroom shrink for other innovative products, while patients and the entire health system struggle even more with affordability.”

ICER’s report on these therapies was reviewed at the March 2021 public meeting of the New England Comparative Effectiveness Public Advisory Council (New England CEPAC), one of ICER’s three independent evidence appraisal committees.

Voting on Clinical Effectiveness and Contextual Considerations

During the public meeting, the New England CEPAC panelists voted unanimously (12-0) that the evidence is adequate to demonstrate that both belimumab and voclosporin provide a net health benefit over standard induction therapy alone.

During their deliberations, panel members also weighed the therapies’ other potential benefits, disadvantages, and contextual considerations.  For both treatments, voting highlighted the following as particularly important for payers and other policymakers to note:

  • The magnitude of the lifetime impact on individual patients of the condition being treated.
  • The positive impact of treatment on patients’ ability to achieve major life goals related to education, work, or family life.
  • The positive impact of treatment on caregivers’ quality of life and/or ability to achieve major life goals related to education, work, or family life.
  • The impact of treatment on helping advance society’s goal of reducing health inequities.
  • The impact of treatment on improving the chances for patients to become pregnant and bear children safely.

Voting on Long-Term Value for Money

After reviewing the clinical evidence and considering the treatments’ other potential benefits, disadvantages, and contextual considerations noted above, the New England CEPAC evaluated the long-term value for these treatments.

A majority of panelists found that belimumab, at its current pricing, represents “high” long-term value for money.  The estimated annual price for belimumab is $43,000 for patients who remain on treatment, lower than ICER’s recommended health-benefit price benchmark (HBPB) range of approximately $45,000-$61,000 per year.

A majority of panelists found that voclosporin, at its current pricing, represents “intermediate” long-term value for money. The estimated annual price for voclosporin is $92,000, towards the higher end of ICER’s recommended HBPB of approximately $72,000-$101,000 per year.

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.

Access and Affordability Alert

ICER is issuing an Access and Affordability Alert for both belimumab and voclosporin. At the current price for belimumab, 80% of the eligible patients could be treated in a given year before crossing the ICER budget impact threshold of $819 million per year. At the price for voclosporin, 35% of the eligible patients could be treated before crossing this threshold. Discussions with clinical experts suggested that ideal clinical use would exceed these levels.

The purpose of an ICER Access and Affordability Alert is to signal to stakeholders and policy makers that the amount of added health care costs associated with a new service may be difficult for the health system to absorb over the short term without displacing other needed services or contributing to rapid growth in health care insurance costs that threaten sustainable access to high-value care for all patients.

Key Policy Recommendations  

Following the voting session, a policy roundtable of experts — including clinical experts, patient advocates, and representatives from US payers and relevant pharmaceutical companies — convened to discuss the implications of the evidence for policy and practice. Key recommendations stemming from the roundtable discussion include:

  • All stakeholders have a responsibility and an important role to play in ensuring that effective new treatment options for patients with lupus nephritis are introduced in a way that will help reduce health inequities.
  • Both belimumab and voclosporin are judged to be priced in reasonable alignment with estimates of their benefits for patients, and this consideration should guide payers to design coverage criteria that do not narrow coverage from the FDA label, although coverage criteria may define terms left indeterminate in the FDA label to assure appropriate use. 
  • Manufacturers should commit to expanding their research, both before and after regulatory approval, to include adequate representation of patients with lupus nephritis from Black and other non-white populations.
  • Manufacturers should not seek to use common sense dosing algorithms as a tool to gain prolongation of their patents, thereby adding to future health care affordability concerns and reducing the headroom for future innovative therapies.

ICER’s detailed set of policy recommendations, including considerations for establishing prior authorization criteria, is available in the Final Evidence Report and in the standalone Policy Recommendations document.

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.