Vanda Pinto, Author at Hemophilia News Today

Starting or switching to prophylactic, or preventive Hemlibra (emicizumab) may lead to a significant reduction in treatment-associated expenses for hemophilia A patients in the U.S., a real-world study reported. These findings highlight the cost-benefits of using Hemlibra — and may help future cost analysis studies in assessing new therapies for hemophilia, the investigators said. "Importantly," the researchers noted, their study did not factor in key secondary savings seen by children with hemophilia A taking preventive Hemlibra. "Consideration of only healthcare costs will also not likely capture all the economic benefits of initiating emicizumab [Hemlibra], such as the time saved by the increased convenience of simplified prophylaxis for people with haemophilia and their families," the researchers wrote. The study, “Real-world cost estimates of initiating emicizumab in US patients with haemophilia A,” was published in the journal Haemophilia. Hemlibra is an approved treatment for managing the symptoms of hemophilia A, an inherited bleeding disorder caused by the lack of a blood-clotting protein called factor VIII, also known as FVIII. Originally developed by Roche subsidiary Chugai Pharmaceutical, Hemlibra now is marketed in the U.S. by Genentech. Previous studies have shown that Hemlibra can effectively lower the number of bleeding episodes in patients with severe hemophilia A, both with and without FVIII inhibitors. These inhibitors are neutralizing antibodies produced by the body’s immune system that target FVIII and lower the effectiveness of replacement therapies — standard treatments for hemophilia that involve supplying the clotting protein patients are missing. Despite Hemlibra’s proven efficacy and its ability to improve the quality of life of patients compared with other treatments, “the monetary consequences for starting or switching to [Hemlibra] prophylaxis in a cohort of real-world patients have not been previously evaluated,” the researchers wrote. Now, researchers from several U.S. institutions reported data from a real-world study that aimed to estimate the cost of hemostatic therapies, or treatments to stop bleeding. The team evaluated the costs of treatment in a group of 92 hemophilia A patients, ages 4 to 14, six months before and after the initiation of preventive Hemlibra. Treatment costs between patients with and without inhibitors also were compared. Among the children enrolled, 18 (20%) had active inhibitors, and from these, 11 (61%) were on prophylaxis. In contrast, 64 patients (86%) without inhibitors were on regular treatment to prevent bleeds. The median age for all patients in the study was 8.7 years. In the six months prior to receiving preventive Hemlibra, the estimated total costs of hemostatic therapy — which included prophylaxis and on-demand treatment for bleeds — amounted to a median of $176,720 per patient. These costs dropped significantly following the start of Hemlibra, decreasing to a median of $128,099 per patient. That was a savings of $48,621 per patient — a drop of 27.5%. This reduction in median total costs was more evident in patients with inhibitors, in whom treatment costs dropped from $365,677 to $117,680. Although therapy expenses also decreased in patients without inhibitors — from a median of $158,609 to $130,452 — this difference was not statistically significant. “Notably, once patients were on [Hemlibra], there was not a significant difference in total costs of haemostatic therapy between patients with and without inhibitors,” the researchers wrote. The team then analyzed the costs of preventive treatment before and after Hemlibra. For all patients receiving prophylaxis before Hemlibra, the median cost of preventive treatment lowered significantly from $197,489 to $128,099. Specifically, for the 11 patients with inhibitors who were receiving prophylaxis prior to Hemlibra, the median estimated cost of prophylaxis dropped from $400,478 to $117,680. The 64 patients without inhibitors who were on prophylactic treatment experienced a smaller cost reduction — from $177,111 to $130,452 — after changing to Hemlibra. The total costs of treating bleeds also dropped significantly after the start of Hemlibra, decreasing from a mean of $24,441 to $2,542 per patient. For that measure, the researchers considered the type of hemostatic therapy being used, along with the dose, the number of doses, and the number of bleeds taking place during the six months of the study. “Starting or switching to prophylaxis with [Hemlibra] results in decreased costs for the treatment of patients with haemophilia A,” the researchers concluded. “This information should help inform on payer and healthcare system decisions about the use [of Hemlibra]. It may also help model future cost analysis studies evaluating other new therapies for haemophilia,” they wrote. The researchers noted that Hemlibra use "has been increasing, especially among paediatric patients," and said their findings were especially notable given that "haemophilia drugs are already some of the most expensive per patient." "This result has important implications for treatment payers as it demonstrates that switching prophylaxis to emicizumab is likely more convenient, decreases bleeds and decreases costs," the team wrote. Of note, the lead author of the study has received research support from Accugen, Spark Therapeutics, and uniQure.

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