Abstract：
OBJECTIVE: to assess the variability in expenditure compared to 2022 assuming different rates of shifting of therapy days from current active ingredients used for the treatment of haemophilia B to nonacog beta pegolDesign: descriptive cross-sectional study.
METHODS: consumption in the year 2022 (data source: Medicines Utilisation Monitoring Centre, Italian Medicines Agency) of all medicinal products available in Italy containing coagulation factor IX.
METHODS: for each active ingredient, the total number of therapy days and the variability in expenditure compared to 2022 were estimated on the basis of a switch of therapy days, between 5% and 20%, to nonacog beta pegol.
RESULTS: on the basis of considered scenarios, the analysis shows that the total annual expenditure for clotting factors used in the treatment of haemophilia B could remain at most unchanged or reduced. Particularly, the extent of the reduction in spending could vary from 0.11% to 2.26%. This trend would be in contrast to the stable increase seen in recent years, particularly in 2022.
CONCLUSIONS: this predictive spending assessment may be useful in evaluating the economic impact from new treatment options, such as etranacogene dezaparvovec gene therapy already approved by the European Medicines Agency and the Food and Drug Administration, and to improve pharmaceutical governance.
METHODS: consumption in the year 2022 (data source: Medicines Utilisation Monitoring Centre, Italian Medicines Agency) of all medicinal products available in Italy containing coagulation factor IX.
METHODS: for each active ingredient, the total number of therapy days and the variability in expenditure compared to 2022 were estimated on the basis of a switch of therapy days, between 5% and 20%, to nonacog beta pegol.
RESULTS: on the basis of considered scenarios, the analysis shows that the total annual expenditure for clotting factors used in the treatment of haemophilia B could remain at most unchanged or reduced. Particularly, the extent of the reduction in spending could vary from 0.11% to 2.26%. This trend would be in contrast to the stable increase seen in recent years, particularly in 2022.
CONCLUSIONS: this predictive spending assessment may be useful in evaluating the economic impact from new treatment options, such as etranacogene dezaparvovec gene therapy already approved by the European Medicines Agency and the Food and Drug Administration, and to improve pharmaceutical governance.
摘要：
目的：评估与2022年相比的支出变异性，假设治疗天数从目前用于治疗B型血友病的活性成分转移到nonacogβpegolDesign：描述性横断面研究。
方法：2022年的消费量（数据来源：药品利用监测中心，意大利药品管理局）在意大利提供的所有含有凝血因子IX的药品。
方法：对于每种活性成分，与2022年相比，治疗天数的总数和支出的变异性是根据治疗天数的转换估算的，在5%到20%之间，去nonacogbetapegol.
结果：根据所考虑的方案，分析表明，用于治疗乙型血友病的凝血因子的年度总支出最多可以保持不变或减少。特别是,支出减少的幅度可能从0.11%到2.26%不等。这一趋势将与近年来的稳定增长形成鲜明对比，尤其是2022年。
结论：这种预测性支出评估可能有助于评估新治疗方案的经济影响，如欧洲药品管理局和食品和药物管理局已经批准的etracogenedezaparvovec基因治疗，改善药品管理。
方法：2022年的消费量（数据来源：药品利用监测中心，意大利药品管理局）在意大利提供的所有含有凝血因子IX的药品。
方法：对于每种活性成分，与2022年相比，治疗天数的总数和支出的变异性是根据治疗天数的转换估算的，在5%到20%之间，去nonacogbetapegol.
结果：根据所考虑的方案，分析表明，用于治疗乙型血友病的凝血因子的年度总支出最多可以保持不变或减少。特别是,支出减少的幅度可能从0.11%到2.26%不等。这一趋势将与近年来的稳定增长形成鲜明对比，尤其是2022年。
结论：这种预测性支出评估可能有助于评估新治疗方案的经济影响，如欧洲药品管理局和食品和药物管理局已经批准的etracogenedezaparvovec基因治疗，改善药品管理。
