突发公共卫生事件中同情用药制度的理性审视

Rational Review of Compassionate Drug Use System in Public Health Emergencies

同情用药制度旨在通过制药企业为危重患者提供尚未批准上市的试验用药,以争取潜在有益的医疗救治机会。该制度蕴含着深刻的人文主义关怀,能一定程度拓宽药品可及性,有助制药企业获取试验药品的安全性和有效性数据,被视为应对新冠肺炎疫情的利剑,但其与超说明书用药、药品专利强制许可制度存在明显的制度差异,此外还存在一定的制度供给局限:一则适用范围有限而难以应对突发公共卫生事件的广泛传播,再则所提供试验药物的安全性和有效性不确定而难以契合突发公共卫生事件群体性的特点。故该制度难以应对突发公共卫生事件。

Compassionate drug use system aims to provide critically ill patients with investigational medicine that have not yet been approved,and strive for potentially beneficial treatment.The system contains profound hu⁃manistic care,which can broaden the accessibility of medicines to a certain extent,and help pharmaceutical enter⁃prises to obtain the data of safety and effectiveness of investigational medicine.And it is regarded as a sharp sword in the fight against COVID-19.However,there are obvious institutional differences between it and off-label drug use and compulsory drug patent licensing system,and there are still some institutional supply limitations:on one hand,the limitation of the application scope of compassionate use does not match the wide spread of public health emergencies;on the other hand,the uncertainty about the safety and effectiveness of experimental drugs provided by compassionate use does not fit the group characteristics of public health emergencies.Therefore,it is difficult for compassionate drug use system to respond to public health emergencies.