Shetty Yashashri C, Brahma Smita, Manjesh P S
Department of Pharmacology and Therapeutics, Seth Gordhandas Sundardas Medical College and King Edward Memorial Hospital, Mumbai, Maharashtra, India.
Department of Pharmacology and Therapeutics, LTMMC and Sion Hospital, Mumbai, Maharashtra, India.
Perspect Clin Res. 2020 Jan-Mar;11(1):3-7. doi: 10.4103/picr.PICR_119_18. Epub 2019 Apr 26.
The World Health Organization defines compassionate use (CU) as a "program that is intended to provide potentially life-saving experimental treatments to patients suffering from a disease for which no satisfactory authorized therapy exists and/or who cannot enter a clinical trial. For many patients, these programs represent their last hope." Over the years, an increasing number of requests and isolated cases have paved the way for more robust CU programs by pharmaceutical companies and guidelines by eminent regulatory bodies globally. In India, although there is no formal mention of the term "Compassionate Use" by the Central Drugs Standard Control Organization, there are provisions in the Drugs and Cosmetics Act 1940 and Rules 1945 to allow drugs to be imported as and when necessary. Such applications can be submitted to the Drug Controller General of India by a hospital, patient, or a pharmaceutical company. The evidence of such use of drugs is underlined by the availability of bedaquiline and delamanid for extensively drug-resistant tuberculosis (TB) and multidrug-resistant TB patients, respectively. CU is in its nascent stage in India owing to the lack of policies and laws needed to govern it. There is a need for regulatory bodies and pharmaceutical companies to work together to extend the spectrum of CU of drugs for the betterment of needy patients.
世界卫生组织将同情用药(CU)定义为“一项旨在为患有某种疾病且没有令人满意的获批治疗方法和/或无法参加临床试验的患者提供可能挽救生命的试验性治疗的计划。对许多患者而言,这些计划是他们最后的希望。”多年来,越来越多的请求和个别案例为制药公司更完善的同情用药计划以及全球知名监管机构制定的指南铺平了道路。在印度,尽管中央药品标准控制组织没有正式提及“同情用药”一词,但1940年《药品和化妆品法案》及1945年《规则》中有允许在必要时进口药品的条款。此类申请可由医院、患者或制药公司提交给印度药品总监。分别为广泛耐药结核病(TB)和耐多药结核病患者提供贝达喹啉和德拉马尼,就突出了此类药品使用的证据。由于缺乏管理同情用药所需的政策和法律,印度的同情用药尚处于起步阶段。监管机构和制药公司需要共同努力,扩大药品同情用药的范围,以改善贫困患者的状况。