ICER Publishes Evidence Report on Therapies for Hemophilia A

Today the Institute for Clinical and Economic Review (ICER) released a report concluding that compared to common current dosing levels of prophylactic factor VIII, Emicizumab provided comparable or better clinical benefits. The report also found Emicizumab to be cost-saving due to the extremely high VIII prices brought about by lack of competition. In a review of valoctocogene roxaparvovec, preliminary analyses suggest that at a price of $2.5 million, the drug would also be cost-saving compared to prophylactic factor VIII for the same reasons as Emicizumab .

“Treatments to reduce the risk of bleeds for patients with hemophilia have profoundly improved the lives of patients and caregivers,” said David Rind, MD, ICER’s Chief Medical Officer. Rind went on to say, “Recently, exciting new strategies for prophylaxis like emicizumab have become available, and gene therapies have even raised the possibility of a cure on the not-too-distant horizon. Our analysis of the evidence suggests that emicizumab is comparable to or better than factor VIII prophylaxis and does save money overall. But our report notes that this is because the prices for factor VIII, despite recombinant technologies and apparent competition in the marketplace, have not decreased, making prophylaxis with factor VIII an extremely expensive treatment required for a lifetime.”

Read more here.