Plasma Protein Therapeutics Association, Annapolis, MD 21401, USA.
Haemophilia. 2013 Jul;19(4):e228-38. doi: 10.1111/hae.12121. Epub 2013 Mar 28.
Prophylaxis has been established as the treatment of choice in children with haemophilia and its continuation into the adult years has been shown to decrease morbidity throughout life. The cost of factor therapy has made the option questionable in cost-effectiveness studies. The role of prophylaxis in pharmacokinetic dosage and tolerization against inhibitor formation were used to model the cost utility of prophylaxis vs. on-demand (OD) therapy over a lifetime horizon in severe haemophilia A. The model was applied to a single provider national health system exemplified by the United Kingdom's National Health Service and a third party provider in the United States. The incremental cost-effectiveness ratio (ICER) was estimated and compared to threshold values used by payer agencies to guide reimbursement decisions. A cost per quality-adjusted life year (QALY) was also estimated for Sweden. Prophylaxis was dominant over OD treatment in the UK. The model resulted in an ICER - $68 000 - within the range of treatments reimbursed in the USA. In Sweden, a cost/QALY of SEK 1.1 million was also within the range of reimbursed treatments in that country. Dosage- and treatment-induced inhibitor incidence were the most important variables in the model. Subject to continuing clinical evidence of the effectiveness of pharmacokinetic dosage and the role of prophylaxis in decreasing inhibitor incidence, treatment for life with prophylaxis is a cost-effective therapy, using current criteria for the reimbursement of health care technologies in a number of countries.
预防治疗已被确立为儿童血友病的首选治疗方法，其在成年期的持续应用已被证明可降低终生发病率。由于因子治疗的费用较高，在成本效益研究中，这种治疗方案的选择受到了质疑。本研究旨在通过对药代动力学剂量和抑制物形成的耐受作用进行建模，比较终身接受预防治疗与按需治疗（OD）对严重 A 型血友病患者的成本效用。该模型应用于单一提供者的国家卫生系统，如英国国民保健制度（NHS）和美国的第三方提供者。并估计增量成本效益比（ICER），并与支付机构用于指导报销决策的阈值进行比较。还对瑞典的每质量调整生命年（QALY）成本进行了估计。预防治疗在英国优于 OD 治疗。该模型得出的 ICER 为 68000 美元，处于美国可报销治疗范围之内。在瑞典，每 QALY 的成本为 110 万瑞典克朗，也在该国可报销治疗范围之内。剂量和治疗诱导的抑制物发生率是模型中最重要的变量。如果继续有临床证据表明药代动力学剂量的有效性和预防治疗在降低抑制物发生率方面的作用，那么终生接受预防治疗是一种具有成本效益的治疗方法，符合目前一些国家对医疗技术报销的标准。
