Magrelli Federica M, Merra Alessia, Pellegrini Graziella
Holostem Terapie Avanzate S.r.l., Modena, Italy.
Stefano Ferrari Regenerative Medicine Center, University of Modena and Reggio Emilia, Modena, Italy.
Front Bioeng Biotechnol. 2020 Jun 10;8:440. doi: 10.3389/fbioe.2020.00440. eCollection 2020.
Advanced therapy medicinal products (ATMPs) are the new frontier of medicine. Advanced therapy medicinal products are set out to satisfy unmet medical needs and provide new innovative, cutting-edge therapies for serious or life-threatening diseases, thus providing new therapeutic options for people with few or no possibility of treatment. They are divided into four groups including gene therapy medicinal products, cell-based therapy medicinal products, tissue-engineered products, and combined ATMPs, which in Europe refer to products that incorporate one or more medical devices with any of the previously mentioned ATMPs as part of the advanced medicine product (AIFA, 2017; Ten Ham et al., 2018). Advanced therapy medicinal products can potentially have long-term benefits, thus bringing a long-lasting positive impact on patient health. Advanced therapy medicinal product therapies are often administered just once or twice, which gives patients the possibility to heal quickly compared to traditional therapies. They also provide a long-term saving opportunity, both in terms of costs of treatments and procedures that are no longer necessary and in terms of quality of life and productivity. The resolution of the patient's illness has a monetary impact on the patient, the patient's caretakers, and especially on the society (Alliance for Regenerative Medicine, 2019). The aim of this paper was to provide an overview on the use of ATMPs approved in Europe, with a focus on blindness and visual impairment and the related economic burden. In this case study, the effective cost of a blind patient in different European countries was compared after treatment with ATMPs or traditional therapies, focusing on visual impairment caused by corneal opacity. Our evaluation includes an overview of the global economic impact of the two types of therapies on the society. We estimated direct healthcare costs, direct non-healthcare costs, and labor productivity losses, to include costs on healthcare, services, patients, their families and for the society in general. We could conclude that the costs of the two therapeutic approaches are comparable.
先进治疗医药产品(ATMPs)是医学的新前沿。先进治疗医药产品旨在满足未被满足的医疗需求,为严重或危及生命的疾病提供新的创新前沿疗法,从而为几乎没有或没有治疗可能性的人提供新的治疗选择。它们分为四类,包括基因治疗医药产品、细胞治疗医药产品、组织工程产品和联合ATMPs,在欧洲,联合ATMPs指的是将一个或多个医疗设备与任何前述ATMPs结合在一起的产品,作为先进医药产品的一部分(意大利药品管理局,2017;滕·哈姆等人,2018)。先进治疗医药产品可能具有长期益处,从而对患者健康产生持久的积极影响。先进治疗医药产品疗法通常只需给药一两次,与传统疗法相比,这使患者有可能更快痊愈。它们还提供了长期的节省机会,无论是在不再需要的治疗和程序成本方面,还是在生活质量和生产力方面。患者疾病的解决对患者、患者的护理人员,尤其是对社会都有金钱上的影响(再生医学联盟,2019)。本文的目的是概述在欧洲获批的先进治疗医药产品的使用情况,重点关注失明和视力损害以及相关的经济负担。在本案例研究中,比较了不同欧洲国家的失明患者在接受先进治疗医药产品或传统疗法治疗后的有效成本,重点关注角膜混浊引起的视力损害。我们的评估包括这两种疗法对社会的全球经济影响概述。我们估计了直接医疗成本、直接非医疗成本和劳动生产率损失,以涵盖医疗保健、服务、患者、其家庭以及整个社会的成本。我们可以得出结论,两种治疗方法的成本相当。