Mennini Francesco Saverio, Marcellusi Andrea, Gitto Lara, Iannone Florenzo
Economic Evaluation and HTA (EEHTA) CEIS, Faculty of Economics, University of Rome "Tor Vergata", Rome, Italy.
Institute for Leadership and Management in Health, Kingston University London, London, UK.
Clin Drug Investig. 2017 Apr;37(4):375-386. doi: 10.1007/s40261-016-0491-y.
Rheumatoid arthritis (RA) is an autoimmune disease with a substantial medical and economic burden. In Italy, it affects approximately 280,000 people, therefore representing the musculoskeletal disease with the highest economic impact in terms of costs for the National Health Service and the social security system.
The aim of this study was to estimate the annual economic burden of RA in Italy and determine the potential cost reduction considering the most effective biologic treatment for early rapidly progressing RA (ERPRA) patients.
The model developed considers both direct costs that are mainly due to the pharmacological treatments, and indirect costs, which also include the productivity lost because of the disease. A systematic literature review provided the epidemiological and economic data used to inform the model. A one-way probabilistic sensitivity analysis based on 5000 Monte Carlo simulations was performed. Furthermore, specific scenario analyses were developed for those patients presenting an ERPRA, with the aim of evaluating the effectiveness of different biologic treatments for this subgroup of patients and estimating potential cost reduction.
The total economic burden associated with RA was estimated to be €2.0 billion per year (95% confidence interval [CI] €1.8-2.3 billion). Forty-five percent of the expenditure was due to indirect costs (95% CI €0.8-1.0 billion); 45% depended on direct medical costs (95% CI €0.7-1.1 billion), and the residual 10% was determined by direct non-medical costs (95% CI €0.16-0.25 billion). In particular, the costs estimated for ERPRA patients totalled €76,171,181, of which approximately €18 million was associated with patients with a high level of anti-citrullinated protein antibodies (ACPA). The results of the analysis outline how it is possible to obtain a cost reduction for ERPRA patients of between €1 and €3 million by varying the number of patients with a high level of immunoglobulin G treated with the most effective biologic drug. In fact, the latter may determine higher efficacy outcomes, especially for poor prognostic ERPRA patients, ensuing higher levels of productivity.
This study presents a pioneering approach to estimate the direct and indirect costs of RA. The model developed is a useful tool for policy makers as it allows to understand the economic implications of RA treatment in Italy, identify the most effective allocation of resources, and select the most appropriate treatment for ERPRA patients.
类风湿关节炎(RA)是一种自身免疫性疾病,带来了巨大的医疗和经济负担。在意大利,约有28万人受其影响,因此就国民医疗服务体系和社会保障系统的成本而言,它是对经济影响最大的肌肉骨骼疾病。
本研究旨在估算意大利RA的年度经济负担,并考虑针对早期快速进展型RA(ERPRA)患者最有效的生物治疗方法,确定潜在的成本降低情况。
所开发的模型既考虑了主要由药物治疗导致的直接成本,也考虑了间接成本,间接成本还包括因疾病导致的生产力损失。系统的文献综述提供了用于为模型提供信息的流行病学和经济数据。基于5000次蒙特卡洛模拟进行了单向概率敏感性分析。此外,针对ERPRA患者开展了特定的情景分析,目的是评估不同生物治疗方法对该亚组患者的有效性,并估算潜在的成本降低情况。
与RA相关的总经济负担估计为每年20亿欧元(95%置信区间[CI]为18亿至23亿欧元)。45%的支出归因于间接成本(95%CI为8亿至10亿欧元);45%取决于直接医疗成本(95%CI为7亿至11亿欧元),其余10%由直接非医疗成本决定(95%CI为1.6亿至2.5亿欧元)。特别是,ERPRA患者的估计成本总计76171181欧元,其中约1800万欧元与抗瓜氨酸化蛋白抗体(ACPA)水平高的患者相关。分析结果表明,通过改变接受最有效生物药物治疗的高免疫球蛋白G水平患者的数量,ERPRA患者有可能实现100万至300万欧元的成本降低。事实上,后者可能带来更高的疗效结果,尤其是对于预后不良的ERPRA患者,从而提高生产力水平。
本研究提出了一种估算RA直接和间接成本的开创性方法。所开发的模型对政策制定者而言是一个有用的工具,因为它有助于了解意大利RA治疗的经济影响,确定最有效的资源分配方式,并为ERPRA患者选择最合适的治疗方法。