ICER Publishes Evidence Report on Treatments for Myasthenia Gravis

While eculizumab and efgartigimod each provide important health benefits, both would need to be priced more than 95% lower than eculizumab's current US list price to reach common thresholds for cost-effectiveness

At September 24 virtual meeting, ICER's independent appraisal 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, September 10, 2021 - The Institute for Clinical and Economic Review (ICER) today released its revised Evidence Report assessing the comparative clinical effectiveness and value of eculizumab (Soliris®; sponsor: Alexion Pharmaceuticals) and efgartigimod (sponsors: argenx, Halozyme Therapeutics, and Zai Lab) for the treatment of generalized myasthenia gravis (gMG). 

"Myasthenia gravis is a serious lifelong disease with life-threatening manifestations, and conventional therapy with high-dose corticosteroids remains inadequate for most patients," said Foluso Agboola, MBBS, MPH, ICER's Vice President of Research. "While our review of available evidence suggests that both eculizumab and efgartigimod appear to significantly improve function and quality of life for these patients, there are uncertainties about longer-term outcomes for efgartigimod and how it will be dosed in real-world settings. Efgartigimod's price is not yet known, but our analysis suggests that the current list price for eculizumab is far higher than the usual thresholds for cost-effectiveness."

This Evidence Report will be reviewed at a virtual public meeting of the New England Comparative Effectiveness Public Advisory Council (New England CEPAC) on September 24, 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

For adults with gMG who are positive for anti-AChR antibodies and refractory to conventional therapy, the evidence provides moderate certainty that eculizumab delivers a small net benefit over conventional therapy alone, with the possibility of a substantial net benefit ("B+"). For efgartigimod, balancing the short-term benefits demonstrated in the clinical trials with uncertainties about dosing and lack of long-term outcomes, ICER determined that the evidence provides moderate certainty that this new treatment is at least comparable to conventional therapy alone, with the possibility of delivering a substantial net benefit ("C++") in adults with gMG who are positive for anti-AChR antibodies. The current evidence is insufficient ("I") to compare eculizumab and efgartigimod to each other, or to either rituximab or intravenous immune globulin.  

For adults with gMG who are negative for anti-AChR antibodies, the evidence is insufficient ("I") to compare efgartigimod to conventional therapy alone.

Key Cost-Effectiveness Findings

ICER's health-benefit price benchmark (HBPB) for eculizumab is $13,200-$19,400 per year, a range that would require at least a 97% discount off the treatment's current US list price of $470,200. ICER's HBPB for efgartigimod, which does not yet have a US price, is $18,300-$28,400.

ICER's 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.

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