Dzhafer Nigyar, Papathanasiou Jannis V
Medical University of Sofia, Sofia, Bulgaria.
Medical University of Sofia, Plovdiv, Bulgaria.
Folia Med (Plovdiv). 2020 Sep 30;62(3):592-596. doi: 10.3897/folmed.62.e53742.
Despite clinical trials, there are still no approved specific therapies or any vaccine against COVID-19. The only option available is using investigational drugs for compassionate use. The update of the existing regulation regarding compassionate use is to ensure the effective and sustainable development of health policies and technologies over the COVID-19 pandemic and beyond.
The present short communication aimed to highlight the need for early and expanded access to investigational drugs for compassionate use as well as a call for an update of the existing regulation in Bulgaria concerning compassionate use in the era of COVID-19.
In EU and Bulgaria as well, the legal framework for compassionate use was introduced by Article 83 (1) of Regulation (EC) No 726/2004 of the European Parliament and of the Council; in principle, Regulations of the European Parliament and of the Council are mandatory for all Member States. Remdesivir appears to have a favorable clinical and safety profile, as reported in a case involving patients with severe COVID-19 through a compassionate use programme.
The overall probability of clinical improvement observed in 36 of 53 COVID-19 patients received intravenous remdesivir as part of a compassionate use programme was 68% (95% CI 40% to 80%). Thirty two patients (60%) demonstrated at least one adverse event, twelve 12 patients (23%) experienced serious adverse events and seven patients (13%) died.
The global pandemic mandates Bulgarian Drug Agency for a reasonable update of the existing national regulation concerning compassionate use and off-label therapies. In the era of COVID-19, it is important for Bulgarian patients to have early and expanded access to investigational drugs for compassionate use.
尽管进行了临床试验，但针对新冠病毒病（COVID-19）仍没有获批的特效疗法或任何疫苗。唯一可用的选择是使用试验性药物用于同情用药。更新现有的同情用药法规，是为了确保在应对COVID-19大流行及以后，卫生政策和技术能够有效且可持续地发展。
本简短通讯旨在强调有必要尽早并扩大试验性药物用于同情用药的获取途径，并呼吁保加利亚更新其在COVID-19时代关于同情用药的现有法规。
在欧盟以及保加利亚，同情用药的法律框架由欧洲议会和理事会第726/2004号（EC）条例第83(1)条引入；原则上，欧洲议会和理事会的条例对所有成员国均具有强制性。据一项通过同情用药项目涉及重症COVID-19患者的病例报告显示，瑞德西韦似乎具有良好的临床和安全性。
在一项同情用药项目中，53例接受静脉注射瑞德西韦的COVID-19患者中有36例临床症状得到改善，总体改善概率为68%（95%置信区间40%至80%）。32例患者（60%）出现至少一种不良事件，12例患者（23%）经历严重不良事件，7例患者（13%）死亡。
全球大流行要求保加利亚药品管理局合理更新其关于同情用药和超说明书用药疗法的现有国家法规。在COVID-19时代，让保加利亚患者尽早并扩大获取试验性药物用于同情用药非常重要。
