ICER has released a draft report that indicates Roctavian, BioMarin’s new gene therapy for hemophilia A, will beat the current antibody-based therapeutic Hemlibri. Unlike Hemlibri, which is a prophylactic, Roctavian restores the gene lacking in hemophilia A, factor VIII. The organization predicts that the one-time price of $2.5 million for Roctavian will be substantially lower than that of 12 years worth of Hemlibra at $640,000 annually.
According to Steve Bryson, PhD, “Based on these assumptions, ICER estimated more than $4 million would be saved using Roctavian instead of Hemlibra per patient over a lifetime. Gene therapy also was associated with slightly fewer bleeds and higher quality-adjusted life years (QALY) — a measure of disease burden, including both the quality and the quantity of life lived; one QALY corresponds to one year of perfect health.”
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(Source: Hemophilia News Today, September 21st, 2022)