BioMarin’s ROCTAVIAN™ gene therapy for adults with severe hemophilia A: substantial cost saving per patient predicted in ICER analysis

ICER Draft Evidence Report propounds $4 million lifetime saving per adult with severe hemophilia for BioMarin’s ROCTAVIAN™ gene therapy vs conventional treatment cost.

The Institute for Clinical and Economic Review (ICER) has issued a Draft Evidence Report on BioMarin’s Roctavian gene therapy for adults with severe hemophilia A. The assessment purports that, as well as immensely reducing both disease and treatment burden for patients, Roctavian  gene therapy may also confer savings of $4 million per patient lifetime treatment costs in comparison with current emicizumab therapy.

ICER released their Draft Evidence Report in September 2022 to update their previous hemophilia A assessment of the comparative clinical effectiveness and value of ROCTAVIAN™.

People with hemophilia A have a mutation in the gene responsible for producing a protein called Factor VIII that is necessary for blood clotting, and Roctavian (valoctocogene roxaparvovec) gene therapy is a one-time infusion that delivers a functional gene designed to enable the body to produce its own Factor VIII. This can relieve patients of a significant treatment burden as individuals with severe hemophilia A are currently treated with a prophylactic regimen of intravenous Factor VIII infusions administered 2–3 times per week (100–150 infusions per year) or a bispecific monoclonal antibody that mimics the activity of Factor VIII administered 1–4 times per month (12–48 infusions per year).

Individuals with severe hemophilia, defined as <1% normal Factor VIII levels, make up ~50% of the hemophilia A population. Even with current best treatment regimens, many continue to experience painful, spontaneous bleeds into their muscles or joints, resulting in progressive and debilitating joint damage. This major impact on their quality of life is also alleviated by Roctavian gene therapy.

ICER calculated the lifetime cost of treating hemophilia A among clinically eligible patients with one-time administration of Roctavian versus ongoing management with monoclonal antibody emicizumab. They estimated the annual cost of emicizumab therapy to be $640k per year, incorporating costs for: treatment; treatment-related adverse events; treatment for bleeding episodes; arthropathy and surgery. ICER allocated a price of $2.5m to one-time Roctavian treatment and modelled its effects to last 12 years for the entire cohort, after which patients were assumed to switch back to standard prophylaxis. This cost comparison model arrived at >$4m cost saving over a lifetime per patient.

Jeff Ajer, Executive Vice President and Chief Commercial Officer at BioMarin said, “BioMarin is pleased that ICER recognizes the potentially transformative impact of Roctavian as possibly the first gene therapy treatment for severe hemophilia A, and potential to not only deliver profound patient benefit, but also potential long-term healthcare savings.”

ICER incorporated an outcomes-based warranty agreement in its economic model. BioMarin is planning to offer this innovative pricing approach to payers to ameliorate the high up-front cost of the one-time Roctavian treatment by effectively risk sharing for several years post-infusion; for example, with a 5-year outcomes-based agreement, if a patient returns to prophylaxis after 4 years, then 20% of the initial price would be refunded by BioMarin.

The Draft Evidence Report is now open for public comment and will next be evaluated by one of ICER’s independent evidence appraisal committees, with a final Evidence Report expected near the end of 2022. The European Commission granted conditional marketing authorization to Roctavian for severe hemophilia in August 2022, and October saw the FDA accept resubmission of the Biologics License Application for Roctavian. The Evidence Base will be interested to see how the debate around pricing for expensive gene therapies develops in Europe and the US.