Correale Jorge, Chiquete Erwin, Boyko Alexey, Beran Roy G, Strauch Jorge Barahona, Milojevic Snezana, Frider Nadina
Department of Neurology, Raúl Carrea Institute for Neurological Research, Foundation for the Fight against Infant Neurological Illnesses (FLENI), Buenos Aires, Argentina.
Department of Neurology and Psychiatry, Salvador Zubirán National Institute of Medical Sciences and Nutrition, Mexico City, Mexico.
Drug Des Devel Ther. 2016 Jun 30;10:2109-17. doi: 10.2147/DDDT.S106802. eCollection 2016.
Both proprietary and nonproprietary medicines are expected to undergo rigorous preapproval testing and both should meet stringent health authority regulatory requirements related to quality to obtain approval. Nonproprietary (also known as copy, or generic) medicines, which base their authorization and use on the proprietary documentation and label, are often viewed as a means to help lower the cost and, thus, increase patient access. If these medicines fail to meet quality standards, such as good manufacturing practice and bioequivalence (in humans), they are then defined as substandard copies and can pose serious risks to patients in terms of safety and efficacy. Potentially noncontrolled or different manufacturing process and excipients in nonproprietary medicines may result in poor batch-to-batch reproducibility (accurate and consistent quantity of each ingredient in each capsule/tablet) and lower quality. Substandard, nonproprietary copies of medicines that are immunomodulatory or immunosuppressive are of concern to patients due to their possible untoward safety and lack of efficacy events. This article reviews the potential risks associated with nonproprietary medicines that do not meet the regulatory requirements of the United States Food and Drug Administration, the European Medicines Agency, or the World Health Organization. The clinical implications for patients are described. This article focuses on nonproprietary medicines for multiple sclerosis, particularly fingolimod, that are not identical to proprietary versions and could thus fail to meet efficacy expectations or have different impact on the safety of patients with multiple sclerosis.
专利药和非专利药都需要经过严格的批准前测试,并且两者都应符合卫生当局与质量相关的严格监管要求才能获得批准。非专利药(也称为仿制药或通用名药),其授权和使用基于专利文件和标签,通常被视为一种有助于降低成本从而增加患者可及性的手段。如果这些药物不符合质量标准,如良好生产规范和(人体)生物等效性,那么它们就被定义为不合格仿制品,在安全性和有效性方面可能会给患者带来严重风险。非专利药中潜在的不受控或不同的生产工艺及辅料可能导致批次间重现性差(每个胶囊/片剂中每种成分的数量准确且一致)以及质量较低。免疫调节或免疫抑制类药物的不合格非专利仿制品因其可能的不良安全性和缺乏疗效事件而受到患者关注。本文综述了不符合美国食品药品监督管理局、欧洲药品管理局或世界卫生组织监管要求的非专利药相关的潜在风险。描述了对患者的临床影响。本文重点关注多发性硬化症的非专利药,尤其是芬戈莫德,这些非专利药与专利药版本不同,因此可能无法达到疗效预期或对多发性硬化症患者的安全性产生不同影响。