Saiyarsarai Parisa, Khorasani Elahe, Photogeraphy Hasti, Ghaffari Darab Mohsen, Seyedifar Meysam
Department of Pharmacoeconomics and Pharmaceutical Administration, Faculty of Pharmacy, Tehran University of Medical Sciences, Tehran, Iran.
Evidence-Based evaluation of cost-effectiveness and clinical outcomes, the Institute of Pharmaceutical Sciences (TIPS), Tehran University of Medical Sciences, Tehran.
Medicine (Baltimore). 2020 Jul 10;99(28):e20949. doi: 10.1097/MD.0000000000020949.
Thalassemia is a hereditary disease, which caused economic burden in developing countries. This study evaluated the cost utility of new formulation of deferasirox (Jadenu) vs deferoxamine (Desferal) among B-Thalassemia-major patients from payer perspective in Iran.
An economic-evaluation through Markov model was performed. A systematic review was conducted in order to evaluate the clinical effectiveness of comparators. Because of chelating therapy is weight-dependent, patients were assumed to be 2 years-old at initiation in first and 18 years-old in second scenario, and model was estimated lifetime costs and utilities. Costs were calculated to the Iran healthcare system through payer perspective and measured effectiveness using quality-adjusted life years (QALYs). One-way sensitivity analysis and budget impact analysis was also employed.
The 381 studies were retrieved from systematic searching through databases. After eliminating duplicate and irrelevant studies, 2 studies selected for evaluating the effectiveness. Jadenu was associated with an incremental cost-effectiveness ratio (ICER) of 1470.6 and 2544.7 US$ vs Desferal in first and second scenario respectively. The estimated ICER for Jadenu compared to generic deferoxamine was 2837.0 and 6924.1 US$ for first and second scenario respectively. For all scenarios Jadenu is presumed as cost-effective option based on calculated ICER which was lower than 1 gross domestic product per capita in Iran. Sensitivity analysis showed that different parameters except discount rate and indirect cost did not have impact on results. Based on budget impact analysis the estimated cost for patients using Desferal (based on the market share of brand) was 44,021,478 US$ in 3 years vs 42,452,606 US$ in replacing 33% of brand market share with Jadenu. This replacement corresponded to the cost saving of almost 1,568,872 US$ for the payers in 3 years. The calculated cost of using generic deferoxamine in all patients was 68,948,392 US$. The increase in the cost of using Jadenu for 10% of all patients in this scenario would be 934,427 US$ (1.36%) US$ at the first year.
Based on this analysis, film-coated deferasirox appeared to be cost-effective treatment in comparison with Desferal for managing child and adult chronic iron overload in B-thalassemia major patients of Iran.
地中海贫血是一种遗传性疾病，在发展中国家造成经济负担。本研究从伊朗医保支付方的角度，评估了新型去铁斯若（Jadenu）与去铁胺（Desferal）在重型β地中海贫血患者中的成本效益。
通过马尔可夫模型进行经济评估。开展系统评价以评估对照药物的临床有效性。由于螯合疗法依赖体重，在第一种情况中假设患者起始治疗时为2岁，在第二种情况中为18岁，并对模型估算终身成本和效用。从医保支付方角度计算伊朗医疗保健系统的成本，并使用质量调整生命年（QALY）衡量有效性。还进行了单向敏感性分析和预算影响分析。
通过数据库系统检索共获得381项研究。在剔除重复和不相关研究后，选择2项研究评估有效性。在第一种和第二种情况中，与Desferal相比，Jadenu的增量成本效益比（ICER）分别为1470.6美元和2544.7美元。与普通去铁胺相比，Jadenu在第一种和第二种情况中的估计ICER分别为2837.0美元和6924.1美元。基于计算得出的ICER低于伊朗人均国内生产总值，在所有情况中Jadenu都被认为是具有成本效益的选择。敏感性分析表明，除贴现率和间接成本外，不同参数对结果无影响。基于预算影响分析，使用Desferal的患者（基于品牌市场份额）在3年内的估计成本为44,021,478美元，而用Jadenu替代33%的品牌市场份额时为42,452,606美元。这种替代在3年内为医保支付方节省了近1,568,872美元。在所有患者中使用普通去铁胺的计算成本为68,948,392美元。在这种情况下，所有患者中有10%使用Jadenu，成本在第一年将增加934,427美元（1.36%）。
基于该分析，对于伊朗重型β地中海贫血患者的儿童和成人慢性铁过载管理，与Desferal相比，薄膜包衣去铁斯若似乎是具有成本效益的治疗方法。
