{"title":"Fidanacogene Elaparvovec (Beqvez)","authors":"Cadth","doi":"10.51731/cjht.2024.851","DOIUrl":null,"url":null,"abstract":"\nCADTH recommends that Beqvez be reimbursed by public drug plans for the treatment of adults with moderately severe to severe hemophilia B (congenital factor IX deficiency) who are negative for neutralizing antibodies to variant adeno-associated virus (AAV) serotype Rh74 , if certain conditions are met. \nBeqvez should only be covered to treat adult patients (aged 18 years or older) with circulating coagulation factor IX (FIX:C) activity of 2% or less and bleeding that requires ongoing prophylactic treatment. Beqvez should not be covered if the patient has FIX inhibitors or has previously received gene therapy for hemophilia B. \nBeqvez should only be reimbursed if prescribed by specialists who have expertise in treating hemophilia B and if the cost of Beqvez is reduced. Beqvez is a 1-time therapy. \n","PeriodicalId":9437,"journal":{"name":"Canadian Journal of Health Technologies","volume":"26 10","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2024-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Fidanacogene Elaparvovec (Beqvez)\",\"authors\":\"Cadth\",\"doi\":\"10.51731/cjht.2024.851\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"\\nCADTH recommends that Beqvez be reimbursed by public drug plans for the treatment of adults with moderately severe to severe hemophilia B (congenital factor IX deficiency) who are negative for neutralizing antibodies to variant adeno-associated virus (AAV) serotype Rh74 , if certain conditions are met. \\nBeqvez should only be covered to treat adult patients (aged 18 years or older) with circulating coagulation factor IX (FIX:C) activity of 2% or less and bleeding that requires ongoing prophylactic treatment. Beqvez should not be covered if the patient has FIX inhibitors or has previously received gene therapy for hemophilia B. \\nBeqvez should only be reimbursed if prescribed by specialists who have expertise in treating hemophilia B and if the cost of Beqvez is reduced. Beqvez is a 1-time therapy. \\n\",\"PeriodicalId\":9437,\"journal\":{\"name\":\"Canadian Journal of Health Technologies\",\"volume\":\"26 10\",\"pages\":\"\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2024-03-06\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Canadian Journal of Health Technologies\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.51731/cjht.2024.851\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Canadian Journal of Health Technologies","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.51731/cjht.2024.851","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
引用次数: 0
摘要
CADTH 建议,Beqvez 用于治疗中重度至重度血友病 B(先天性 IX 因子缺乏症)成人患者,且变异腺相关病毒 (AAV) 血清型 Rh74 中和抗体阴性,但需满足特定条件。Beqvez 只能用于治疗循环凝血因子 IX (FIX:C) 活性为 2% 或更低且出血需要持续预防性治疗的成年患者(18 岁或以上)。如果患者患有 FIX 抑制剂或曾接受过 B 型血友病基因治疗,则 Beqvez 不在承保范围内。Beqvez 只有在由具有治疗 B 型血友病专业知识的专科医生处方且 Beqvez 的费用降低的情况下才能获得报销。Beqvez 为一次性疗法。
CADTH recommends that Beqvez be reimbursed by public drug plans for the treatment of adults with moderately severe to severe hemophilia B (congenital factor IX deficiency) who are negative for neutralizing antibodies to variant adeno-associated virus (AAV) serotype Rh74 , if certain conditions are met.
Beqvez should only be covered to treat adult patients (aged 18 years or older) with circulating coagulation factor IX (FIX:C) activity of 2% or less and bleeding that requires ongoing prophylactic treatment. Beqvez should not be covered if the patient has FIX inhibitors or has previously received gene therapy for hemophilia B.
Beqvez should only be reimbursed if prescribed by specialists who have expertise in treating hemophilia B and if the cost of Beqvez is reduced. Beqvez is a 1-time therapy.