Mennini Francesco Saverio, Cicchetti Americo, Sciattella Paolo, Rumi Filippo, Zanuzzi Matteo, Carletto Angelica, Sammarco Annalisa, Romano Federica, Russo Pierluigi
Departments of Healthcare Planning, Medical Devices, Pharmaceuticals, and Policies in Support of the National Health Service, Italian Ministry of Health, Rome, Italy.
Faculty of Economics, Economic Evaluation and HTA, CEIS, University of Rome "Tor Vergata", Rome, Italy.
Drugs Real World Outcomes. 2025 Mar;12(1):25-33. doi: 10.1007/s40801-024-00463-w. Epub 2024 Dec 7.
The increasing demand for orphan drugs and the financial challenges associated with their reimbursement highlights the need to understand the dynamics between expected and actual pharmaceutical expenditures, particularly in the context of pricing and reimbursement negotiations.
The study aims to identify the potential determinants of the difference in expected pharmaceutical spending after negotiation and the actual expenditure incurred (ΔS), as well as the difference in expected pharmaceutical spending before and after the price and reimbursement negotiation process (ΔSn) for rare disease drugs in Italy.
The analysis focused on orphan drugs authorised by European Medicines Agency, with reimbursement applications to the Italian Medicines Agency from January 2013 to January 2019. Expected post-negotiation spending was estimated by applying negotiated discounts and financial-based market entry agreements to the expected pharmaceutical expenditure. The actual expenditure was approximated through company turnovers. Beta regression models were applied to identify potential determinants of ΔS and ΔSn.
The study analysed 52 reimbursed orphan drugs, with 41 (78.8%) with a single indication, 25 (48.1%) antineoplastics and immunomodulators and 18 (34.6%) conditionally/fully innovative. The median expenditure in the first 3 years post-commercialisation was 7.6% lower than expected post-negotiation. The reduction was less pronounced for innovative drugs (p = 0.011), drugs with a prices and reimbursement procedure in the subsequent 3 years (p = 0.007) and those with multiple indications (p = 0.021). Payment-by-result was the only significant variable associated with the expected spending ratio before and after negotiation (p = 0.002).
The actual expenditure for orphan drugs aligns with the expected post-negotiation. Yet, innovative orphan drugs exhibit a higher actual expenditure than estimated, suggesting the market values their added therapeutic value or the actual therapeutic need they meet, and configuring innovativeness status as the main determinant of the orphan drugs financial impact in the multiple regression analysis.
对孤儿药的需求不断增加以及与之相关的报销财务挑战凸显了理解预期和实际药品支出之间动态关系的必要性，特别是在定价和报销谈判的背景下。
本研究旨在确定意大利罕见病药物谈判后预期药品支出与实际发生支出之间差异（ΔS）的潜在决定因素，以及价格和报销谈判过程前后预期药品支出的差异（ΔSn）。
分析聚焦于欧洲药品管理局批准的孤儿药，这些孤儿药于2013年1月至2019年1月向意大利药品管理局提交了报销申请。通过将谈判折扣和基于财务的市场准入协议应用于预期药品支出，来估计谈判后的预期支出。实际支出通过公司营业额进行估算。应用贝塔回归模型来确定ΔS和ΔSn的潜在决定因素。
该研究分析了52种已报销的孤儿药，其中41种（78.8%）有单一适应症，25种（48.1%）为抗肿瘤药和免疫调节剂，18种（34.6%）为有条件/完全创新药。商业化后前3年的支出中位数比谈判后的预期低7.6%。对于创新药（p = 0.011）、在随后3年有价格和报销程序的药物（p = 0.007）以及有多种适应症的药物（p = 0.021），这种降低不太明显。按结果付费是与谈判前后预期支出比率相关的唯一显著变量（p = 0.002）。
孤儿药的实际支出与谈判后的预期相符。然而，创新孤儿药的实际支出高于估计值，这表明市场重视其增加的治疗价值或其满足的实际治疗需求，并将创新状态确定为多元回归分析中孤儿药财务影响的主要决定因素。
