Maria Grazia Ursino, Annalisa Milano, Filippo Viti De Angelis, Eva Alessi, Francesco Trotta
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Data were obtained from reimbursement and pricing dossiers, and negotiation assessments. Trends in the number of EoIs submitted and the additional discounts negotiated were analyzed, along with the relationship between the negotiated discount and subsequent drug expenditure. Determinants influencing the extent of the negotiated discount were assessed, including drug type, orphan drug designation, innovativeness status, number of EoIs, disease incidence and prevalence, estimated number of patients, revenue projections, availability of therapeutic alternatives, and efficacy outcomes. A Wilcoxon nonparametric test was used to evaluate the associations between determinants and the negotiated additional discount, with a significance level of 0.05.</p><p><strong>Results: </strong>The study identified nine medicines: five of these were used in onco-hematologic therapeutic areas, while the remaining four were immunosuppressants for dermatologic and/or rheumatologic conditions. These nine active substances accounted for 65 approved therapeutic indications, of which 50 were reimbursed by the Italian NHS, including the first indication; the analysis focused only on 40 reimbursed EoIs. The additional discount obtained for EoIs averages approximately 12.5% (95% CI 9.4-16.6%), with a median value of approximately 11%. This latter value was used as the threshold in the analysis of the determinants potentially impacting the negotiated discount amount. Discounts greater than 11% were significantly associated with EoI beyond the fifth and oncology drugs. The additional discount seemed small when compared with the increased spending.</p><p><strong>Conclusion: </strong>The study provides valuable insights into the negotiation outcomes for medicines with multiple therapeutic indications, particularly in onco-hematologic and immunosuppressive areas. The analysis revealed that additional discounts for EoIs averaged 12.5%, with a median of 11%, a value used to assess the impact of specific determinants. A discount higher than 11% was statistically correlated with drugs having more than five indications and oncology treatments, showing their influence in negotiations. However, the savings from discounts were modest relative to the increased drug spending as more indications were approved. 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引用次数: 0
摘要
背景:意大利国家卫生服务(NHS)已经报销的药物的新治疗适应症的授权是一个重要的问题。本研究旨在估计额外折扣归因于指标的延伸(eoi),以探索消费和协商折扣之间的潜在相关性,并找到影响折扣的具体因素(决定因素)。方法:研究重点是2003年至2017年在意大利批准的至少有四种治疗适应症的药物,包括首次批准和eoi,随访时间延长至2021年,以获取已完成谈判过程的所有信息。数据来自报销和定价档案以及谈判评估。分析了提交的eoi数量和谈判的额外折扣的趋势,以及谈判的折扣与随后的药品支出之间的关系。我们评估了影响协商折扣程度的决定因素,包括药物类型、孤儿药指定、创新状态、eoi数量、疾病发病率和流行率、估计患者数量、收入预测、治疗替代方案的可用性和疗效结果。使用Wilcoxon非参数检验来评估决定因素与协商额外折扣之间的关联,显著性水平为0.05。结果:该研究确定了9种药物:其中5种用于肿瘤血液治疗领域,而其余4种用于皮肤和/或风湿病条件的免疫抑制剂。这9种活性物质占65个批准的治疗适应症,其中50个由意大利NHS报销,包括第一个适应症;分析只关注40份已报销的意向书。eoi获得的额外折扣平均约为12.5% (95% CI 9.4-16.6%),中位数约为11%。后一个值被用作分析可能影响谈判折扣金额的决定因素的阈值。折扣大于11%与第五种和肿瘤药物以外的EoI显著相关。与增加的支出相比,额外的折扣似乎很小。结论:AML似乎会产生大量的直接经济费用。减少住院天数可以显著减轻欧盟AML的经济负担。此外,有必要进行研究,全面评价所涉经济问题,特别是总费用和间接费用。注册:该研究为具有多种治疗适应症的药物的谈判结果提供了有价值的见解,特别是在肿瘤血液学和免疫抑制领域。分析显示,eoi的额外折扣平均为12.5%,中位数为11%,该值用于评估特定决定因素的影响。在统计学上,超过11%的折扣与具有5种以上适应症的药物和肿瘤治疗相关,显示出它们在谈判中的影响力。然而,随着越来越多的适应症被批准,折扣带来的节省相对于增加的药物支出来说是适度的。这表明通过折扣实现的成本控制与由于扩大药物使用而增加的支出之间存在不平衡。
Pricing for Multi-Indication Drugs in the Italian Regulatory Context.
Background: The authorization of new therapeutic indications for drugs already reimbursed by the Italian National Health Service (NHS) represents a matter of importance. This study aims to estimate the additional discount attributed to the extension of indications (EoIs) to explore the potential correlation between spending and negotiated discounts and to find specific factors (determinants) that impact on discount.
Methods: The study focused on drugs approved in Italy between 2003 and 2017 with at least four therapeutic indications, including the first approved and EoIs, with follow-up extended until 2021 to acquire all the information on the negotiation process that has been completed. Data were obtained from reimbursement and pricing dossiers, and negotiation assessments. Trends in the number of EoIs submitted and the additional discounts negotiated were analyzed, along with the relationship between the negotiated discount and subsequent drug expenditure. Determinants influencing the extent of the negotiated discount were assessed, including drug type, orphan drug designation, innovativeness status, number of EoIs, disease incidence and prevalence, estimated number of patients, revenue projections, availability of therapeutic alternatives, and efficacy outcomes. A Wilcoxon nonparametric test was used to evaluate the associations between determinants and the negotiated additional discount, with a significance level of 0.05.
Results: The study identified nine medicines: five of these were used in onco-hematologic therapeutic areas, while the remaining four were immunosuppressants for dermatologic and/or rheumatologic conditions. These nine active substances accounted for 65 approved therapeutic indications, of which 50 were reimbursed by the Italian NHS, including the first indication; the analysis focused only on 40 reimbursed EoIs. The additional discount obtained for EoIs averages approximately 12.5% (95% CI 9.4-16.6%), with a median value of approximately 11%. This latter value was used as the threshold in the analysis of the determinants potentially impacting the negotiated discount amount. Discounts greater than 11% were significantly associated with EoI beyond the fifth and oncology drugs. The additional discount seemed small when compared with the increased spending.
Conclusion: The study provides valuable insights into the negotiation outcomes for medicines with multiple therapeutic indications, particularly in onco-hematologic and immunosuppressive areas. The analysis revealed that additional discounts for EoIs averaged 12.5%, with a median of 11%, a value used to assess the impact of specific determinants. A discount higher than 11% was statistically correlated with drugs having more than five indications and oncology treatments, showing their influence in negotiations. However, the savings from discounts were modest relative to the increased drug spending as more indications were approved. This suggests an imbalance between the cost control achieved through discounts and the rising expenditure due to expanded drug use.
期刊介绍:
PharmacoEconomics - Open focuses on applied research on the economic implications and health outcomes associated with drugs, devices and other healthcare interventions. The journal includes, but is not limited to, the following research areas:Economic analysis of healthcare interventionsHealth outcomes researchCost-of-illness studiesQuality-of-life studiesAdditional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in PharmacoEconomics -Open may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances.All manuscripts are subject to peer review by international experts. Letters to the Editor are welcomed and will be considered for publication.