European Medicines Agency, 30 Churchill Place, Canary Wharf, London E14 5EU, UK.
European Medicines Agency, 30 Churchill Place, Canary Wharf, London E14 5EU, UK.
Drug Discov Today. 2018 Oct;23(10):1801-1805. doi: 10.1016/j.drudis.2018.06.018. Epub 2018 Jun 25.
Small and medium-sized enterprises (SMEs) are an important source of innovative medicines. Compared with their larger counterparts, they experience challenges as a result of insufficient human and financial resources that can hamper drug development and regulatory compliance. This analysis reviews the profile of major objections raised in marketing authorisation applications for medicines for human use submitted by SMEs to the European Medicines Agency (EMA) between 2011 and 2015 and their impact on the outcome of applications. It showed that SMEs experience challenges in the quality (e.g. manufacturing process validation and control and/or characterisation data of drug substance or drug product) and clinical sections of marketing authorisation applications (e.g. analysis or robustness of pivotal data or selection of submitted studies, study design issues and marginal or no clinical relevant efficacy), with deficiencies in demonstrating clinical efficacy representing the major eventual hurdles to authorisation.
中小企业（SMEs）是创新药物的重要来源。与大型企业相比，中小企业由于人力资源和财力资源不足，在药物开发和法规遵从方面面临挑战。本分析审查了 2011 年至 2015 年间中小企业向欧洲药品管理局（EMA）提交的人用药品上市许可申请中提出的主要反对意见的概况及其对申请结果的影响。结果表明，中小企业在药品上市许可申请的质量（如生产工艺验证和控制以及/或原料药或药品的特性数据）和临床部分（如关键数据的分析或稳健性或提交研究的选择、研究设计问题和边际或无临床相关疗效）方面面临挑战，在证明临床疗效方面的不足是获得批准的主要最终障碍。
