Messori A, Fadda V, Trippoli S
Laboratorio di Farmacoeconomia, c/o University of Florence, Via Guimaraes 5-7, 59100 Prato, Italy.
J Chemother. 2011 Apr;23(2):67-70. doi: 10.1179/joc.2011.23.2.67.
National healthcare systems as well as local institutions generally reimburse numerous off-label uses of anticancer drugs, but an explicit framework for managing these payments is still lacking. As in the case of on-label uses, an optimal management of off-label uses should be aimed at a direct proportionality between cost and clinical benefit. Within this framework, assessing the incremental cost/effectiveness ratio becomes mandatory, and measuring the magnitude of the clinical benefit (e.g. gain in overall survival or progression-free survival) is essential.This paper discusses how the standard principles of cost-effectiveness and value-for-money can be applied to manage the reimbursement of off-label treatments in oncology. It also describes a detailed operational scheme to appropriately implement this aim. Two separate approaches are considered: a) a trial-based approach, which is designed for situations where enough information is available from clinical studies about the expected effectiveness of the off-label treatment; b) an individualized payment-by-results approach, which is designed for situations in which adequate information on effectiveness is lacking; this latter approach requires that each patient receiving off-label treatment is followed-up to determine individual outcomes and tailor the extent of payment to individual results.Some examples of application of both approaches are presented in detail, which have been extracted from a list of 184 off-label indications approved in 2010 by the Region of tuscany in italy. these examples support the feasibility of the two methods proposed.In conclusion, the scheme described in this paper represents an operational solution to an unsettled problem in the area of oncology drugs.
国家医疗保健系统以及地方机构通常会为抗癌药物的众多未标明用途报销费用，但仍缺乏管理这些报销的明确框架。与标明用途的情况一样，对未标明用途的最佳管理应以成本与临床效益之间的直接比例关系为目标。在此框架内，评估增量成本/效益比变得至关重要，而衡量临床效益的大小（例如总生存期或无进展生存期的增加）则必不可少。本文讨论了成本效益和性价比的标准原则如何应用于管理肿瘤学中未标明用途治疗的报销。它还描述了一个详细的操作方案以适当实现这一目标。考虑了两种不同的方法：a）基于试验的方法，该方法适用于从临床研究中可获得足够关于未标明用途治疗预期有效性信息的情况；b）个体化的按结果付费方法，该方法适用于缺乏有效性充分信息的情况；后一种方法要求对每一位接受未标明用途治疗的患者进行随访，以确定个体结果并根据个体结果调整报销范围。详细介绍了这两种方法的一些应用实例，这些实例摘自意大利托斯卡纳地区2010年批准的184种未标明用途适应症清单。这些实例支持了所提出的两种方法的可行性。总之，本文所述方案是解决肿瘤药物领域一个未解决问题的操作方案。
