RARE Daily

ICER Finds BioMarin Gene Therapy Could Justify $2.5 Million Price Tag

August 31, 2020

Rare Daily Staff

The Institute for Clinical and Economic Review (ICER), an independent non-profit research institute that analyzes the value of therapies, released a draft evidence report on BioMarin Pharmaceutical’s hemophilia A gene therapy saying Roctavian would be cost effective at $2.5 million based on several scenarios it considered.

The report, though, is considered preliminary because of the U.S. Food and Drug Administration’s refusal to approve the therapy with the data BioMarin submitted. ICER said the preliminary draft marks the midpoint of its eight-month process of assessing hemophilia A treatments. It is seeking public comments on its report until 5 p.m. ET on September 23, 2020.

People living with hemophilia A lack enough factor VIII protein to help their blood clot and are at risk for painful and potentially life-threatening bleeds from even modest injuries. In cases where the condition is severe, people with hemophilia A experience painful, spontaneous bleeds into their muscles or joints. Even with the use of replacement factor VIII therapy, many people continue to experience bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life.

BioMarin’s experimental gene therapy Roctavian is an AAV-factor VIII gene therapy designed to restore adequate levels of factor VIII for normal clotting. The therapy is being developed as a one-time treatment for adults with severe hemophilia A and could eliminate the need for ongoing factor VIII treatments. The standard of care for most hemophilia A patients who are severely affected today is a regimen of intravenous infusions three times per week.

Because BioMarin has said it expected to price the gene therapy at between $2 million and $3 million, ICER used a price of $2.5 million for its analysis.

ICER said it found the therapy not cost effective for adult patients with hemophilia A without inhibitors at standard cost-effectiveness thresholds when using doses of factor VIII consistent with the underlying trial used in a meta-analysis. This finding, however, varied in the sensitivity analyses and importantly was completely reversed when the model incorporated doses of factor VIII that are more representative of current use in the United States.

Because Roctavian meets ICER’s criteria to be considered a high-impact single and short-term therapy, it performed two additional scenario analyses. In one of these scenarios, 50 percent of the modeled cost savings from treatment are “retained” by the health system instead of being ascribed to the therapy; in the other, cost savings from treatment beyond $150,000 are retained by the health system.

In both of those alternative scenarios, Roctavian at a price of $2.5 million was found to have extremely high incremental cost-effectiveness ratios, reflecting the high current costs of treating people with hemophilia A with factor VIII.

ICER said it used these alternative scenarios to provide empirical findings that may stimulate public dialogue on whether and how extremely large cost offsets should be incorporated in judgments of reasonable pricing for novel therapies that are delivered as single or short-term interventions.

“Overall, the findings illustrate that factor VIII is such an extremely costly treatment, especially at currently used dosages in the U.S., that new treatments are capable of generating large cost savings in comparison,” ICER said. “If prices of factor VIII were to come down from effective competition or other measures, the appropriate pricing of new treatments, as suggested by cost-effectiveness thresholds, would come down significantly as well.”

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