Roctavian is assessed to provide substantial cost savings per hemophilia A patient in a preliminary independent report

In the Draft Evidence Report, ICER finds Roctavian is a dominant treatment at an assumed place holder price of $2.5 million, providing substantial cost savings and projected gains in quality adjusted life years.

These findings were robust to numerous sensitivity analyses and scenario analyses. Marking an important milestone in ICER's eight-month process of assessing this treatment, the Draft Evidence Report is now open for public comment, with a final report expected near the end of the year.

In the Draft Evidence Report, ICER calculated the lifetime cost of managing hemophilia A among clinically eligible patients using one-time administration with Roctavian versus emicizumab prophylaxis. Total costs in the model include treatment, treatment-related adverse events, treatment for bleeding episodes, arthropathy, surgery, and non-drug costs. ICER assumed annual cost of emicizumab to be $640K per year and one-time Roctavian price to be $2.5M. ICER modelled the effect of Roctavian to last 12 years (before patients were assumed to switch back to prophylaxis) for the entire cohort post infusion. ICER arrived at greater than $4M cost saving per patient over a lifetime with projected improvement in quality of life. Importantly, ICER incorporated an outcomes-based warranty agreement in its base-case economic model, an innovative approach that BioMarin plans to offer that will allow effective risk sharing for the period of four years and have it ready to implement with payers at launch. While Roctavian is approved in the European Union, it is still an investigational therapy in the U.S. and therefore does not have a price.