Rare Daily Staff

The Institute for Clinical and Economic Review, in a newly released report on the comparative effectiveness and value of Alexion Pharmaceutical’s Soliris (eculizumab), found that its wholesale price is 97 to 98 percent too high relative to its value as a treatment for generalized myasthenia gravis, a rare and chronic autoimmune condition.

ICER, an independent non-profit research institute that analyzes the evidence on the effectiveness and value of drugs and other medical services, also reviewed Argenx’, Halozyme Therapeutics’, and Zai Lab’s efgartigimod as a treatment for generalized myasthenia gravis, but said its price is not yet known.

The organization said a majority of the members of its New England Comparative Effectiveness Public Advisory Council, one of ICER’s three independent evidence appraisal committees, reviewed its report, and found that Soliris represents “low” long-term value for money. ICER’s recommended health-benefit price benchmark range for Soliris is between $13,200-$19,400, pricing levels that would require a 97 to 98 percent discount off the treatment’s wholesale acquisition cost of $653,100.

All panelists found that efgartigimod represents “low” long-term value for money at the assumed price of $418,400. ICER’s recommended health-benefit price benchmark range for efgartigimod is $18,300-$28,400.

The health-benefit price benchmark is a price range ICER uses to suggest the highest U.S. price a manufacturer should charge for a treatment. It said the figure is the top price range at which a health system can reward innovation and better health for patients without doing more harm than good considering losses in health to other patients in the health system due to rising overall costs.

ICER’s report on these therapies was reviewed at the September 2021 public meeting of the New England Comparative Effectiveness Public Advisory Council.

“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,” said Foluso Agboola, ICER’s vice president of research. “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. Further, it’s important to monitor these treatments’ effectiveness in minority populations to develop a complete picture of their overall efficacy.”

ICER noted Soliris was first approved for paroxysmal nocturnal hemoglobinuria and hemolytic uremic syndrome, ultra-rare conditions with a cumulative prevalence of less than 4 per million. The population with generalized myasthenia gravis is 14-20 per 100,000. It said if only 15 percent of this population is considered to have refractory generalized myasthenia gravis, the population now eligible for treatment with Soliris, it is more than seven times as large as when the drug was first approved, yet the price has not come down.

ICER said there is no excuse for this level of pricing, and payers should not use it as a benchmark or standard for future therapies in this clinical area or others.

Photo: Foluso Agboola, ICER’s vice president of research

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