Schiff Leora
Altius Strategy Consulting, Somerville, Massachusetts.
Clin Ther. 2015 May 1;37(5):1092-112. doi: 10.1016/j.clinthera.2015.02.009. Epub 2015 Apr 4.
Public discourse regarding the hepatitis C virus (HCV) drug Sovaldi® (sofosbuvir) has become inflamed, generating much heat but little light concerning the clinical, health economic, and quality-of-life merits of Sovaldi®. The purpose of this article is to provide a factual basis for evaluating the claims regarding the benefits of Sovaldi® relative to its costs.
A comprehensive review was conducted of news stories highlighted in the daily updates of the electronic newsletters BIO SmartBrief, FiercePharma, FierceBiotech and BioCentury Extra published from November 1, 2013, through December 31, 2014, on the topics of the HCV market, Sovaldi®, and other HCV therapeutics. Also reviewed were recent practice guidelines on the management of HCV infections, prescribing information on all HCV drugs approved by the US Food and Drug Administration, and health technology assessments of Sovaldi® and Harvoni(TM) (sofosbuvir/ledipasvir).
Sovaldi® and Harvoni(TM) have provided significant improvements in the treatment of HCV, with all-oral regimens and cure rates exceeding 90% in some populations of patients with HCV. Sovaldi® prevents significant health care resource utilization in patients who would otherwise develop cirrhosis and require a liver transplant; however, only a small proportion of patients with HCV develop cirrhosis, and fewer require liver transplants. Because it is not possible to identify those patients whose HCV will progress to severe liver disease, it would be necessary to treat a large number of patients with HCV to prevent disease progression in this subpopulation, resulting in a considerable loss to health plans even over a 20-year horizon. The claim that treating all patients with HCV with Sovaldi® would cost nearly as much as the current total US expenditure on all prescription drugs, while factually correct, is not a realistic scenario. Many patients with HCV will continue to go undiagnosed. In addition, the medical expense for those who are treated will be spread out over many years. However, the unexpectedly large, up-front cost of covering these drugs has had a major impact on health plan budgets, resulting in losses for some plans.
Sovaldi® represents an enormous advance in the care of some populations of HCV-infected patients, but also a major cost burden to health plans. As the first of a number of anticipated, paradigm-changing drugs to treat medical conditions affecting large patient populations, Sovaldi® should act as a wake-up call for all health care stakeholders to engage in a meaningful, fact-based discussion about managing the cost of innovative new drugs to balance the needs of drug manufacturers, health plans, providers, and, above all, patients.
关于丙型肝炎病毒（HCV）药物索华迪®（索磷布韦）的公众讨论已变得激烈，虽引发了诸多热议，但对于索华迪®在临床、卫生经济及生活质量方面的优势却鲜少提供有价值的见解。本文旨在为评估有关索华迪®效益与成本的相关说法提供事实依据。
对2013年11月1日至2014年12月31日期间在电子通讯《生物科技简报》《医药前沿》《生物科技前沿》和《生物世纪特刊》每日更新中突出报道的有关HCV市场、索华迪®及其他HCV治疗药物的新闻报道进行了全面回顾。还查阅了近期关于HCV感染管理的实践指南、美国食品药品监督管理局批准的所有HCV药物的处方信息以及索华迪®和哈瓦尼™（索磷布韦/雷迪帕韦）的卫生技术评估。
索华迪®和哈瓦尼™在HCV治疗方面取得了显著进展，全口服治疗方案在部分HCV患者群体中的治愈率超过90%。索华迪®可避免那些否则会发展为肝硬化并需要进行肝移植的患者大量使用医疗资源；然而，只有一小部分HCV患者会发展为肝硬化，需要肝移植的患者更少。由于无法识别哪些HCV患者的病情会进展为严重肝病，因此有必要治疗大量HCV患者以预防该亚组患者的疾病进展，即使在20年的时间范围内，这也会给医保计划带来相当大的损失。声称用索华迪®治疗所有HCV患者的成本几乎与美国目前所有处方药的总支出相当这一说法，虽然事实上是正确的，但并非现实情况。许多HCV患者仍将未被诊断。此外，接受治疗患者的医疗费用将分摊在许多年。然而，这些药物意外高昂的前期成本对医保计划预算产生了重大影响，导致一些医保计划出现亏损。
索华迪®代表了在部分HCV感染患者护理方面的巨大进步，但也是医保计划的一项重大成本负担。作为众多预期的、改变范式的治疗影响大量患者群体疾病的药物中的首个药物，索华迪®应给所有医疗保健利益相关者敲响警钟，促使他们就管理创新新药成本以平衡药品制造商、医保计划、医疗服务提供者以及最重要的患者的需求展开有意义的、基于事实的讨论。
