Wegscheider Karl, Drabik A, Bleich C, Schulz H
Institut für Medizinische Biometrie und Epidemiologie, Universitätsklinikum Hamburg-Eppendorf, Martinistraße 52 (Gebäude W34), 20246, Hamburg, Deutschland,
Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz. 2015 Mar;58(3):298-307. doi: 10.1007/s00103-014-2106-1.
The legal terms "benefit" and "added benefit" and the procedures related to their assessment, given the application of a new medical intervention for approval, should help to restrict the market to those products for which there is much evidence of their benefit or added benefit. The term implies the fiction of an overall benefit for all patients with the same disease. However, from the perspective of health services research and that of epidemiology the term inevitably has to be extended to cover the benefit to a variety of groups of patients or users of a broad spectrum of medical interventions in the real world, ranging from inpatient treatment to vaccination or screening programs. Thus, the assessment of benefit requires a comparison of the new product with all the alternatives currently available for routine care, explicitly taking into account user preferences. Hence, the assessment of benefit in health services research is not one-dimensional and requires new types of studies that go beyond the traditional phase III trials (efficacy trials). New approaches are mainly developed by comparative effectiveness research (CER). CER studies also use randomized designs, because they are currently the best available method for causal inferences. However, randomization in CER is extended to a much broader framework, including health-related databases, registers, epidemiological studies, feasibility studies, and post hoc analyses. CER has developed the necessary and appropriate designs and statistical methods. In addition, some of these methods allow an adaptive assessment of benefit, which can be used to monitor the implementation of new health care interventions or programs.
法律术语“益处”和“附加益处”以及与其评估相关的程序，鉴于新医疗干预措施申请获批的情况，应有助于将市场限制在那些有充分证据证明其益处或附加益处的产品上。该术语意味着对所有患有相同疾病的患者都有总体益处的虚构概念。然而，从卫生服务研究和流行病学的角度来看，该术语不可避免地必须扩展，以涵盖对现实世界中各种患者群体或广泛医疗干预措施使用者的益处，范围从住院治疗到疫苗接种或筛查项目。因此，对益处的评估需要将新产品与目前可用于常规护理的所有替代方案进行比较，明确考虑用户偏好。因此，卫生服务研究中的益处评估并非一维的，需要超越传统III期试验（疗效试验）的新型研究。新方法主要由比较效果研究（CER）开发。CER研究也采用随机设计，因为它们目前是进行因果推断的最佳可用方法。然而，CER中的随机化扩展到了更广泛的框架，包括与健康相关的数据库、登记册、流行病学研究、可行性研究和事后分析。CER已经开发出了必要且合适的设计和统计方法。此外，其中一些方法允许对益处进行适应性评估，可用于监测新医疗保健干预措施或项目的实施情况。
