MA Jing, GAO Jinghong, LIU Chunguang. Issues Related to Expanded Access to Investigational Drugs for Compassionate Use[J]. Chinese Journal of Pharmacovigilance, 2021, 18(1): 47-51.
[1] FDA.Expanded access to investigational drugs for treatment use-policy[EB/OL].(2017-12-11)[2020-04-05].https://www.fda.gov/news-events/public-health-focus/expanded-access. [2] FDA.Investigational New Drug (IND) application[EB/OL].(2009-11-05)[2020-01-22].https://www.fda.gov/drugs/types-applications/investigational-new-drug-ind-application [3] FDA.FDA determines that vast majority of all expanded access requests may proceed[EB/OL].(2019-05-20)[2020-04-10].https://www.fda.gov/news-events/expanded-access/expanded-access-information-physicians. [4] FDA.How to submit a request (forms)[EB/OL].(2019-09-17)[2020-03-22].https://www.fda.gov/news-events/expanded-access/expanded-access-how-submit-request-forms [5] FDA.What are the roles and responsibilities?[EB/OL].(2019-05-09)[2020-04-10].https://www.fda.gov/news-events/expanded-access/expanded-access-information-industry#RolesResponsabilites [6] Jonathan P,Jarow MD.Expanded access to investigational drugs:the experience of the center of drug evaluation and researchover a 10-year period[J].Ther Innov Regul Sci,2016 ,11,50(6):705-709. [7] Gilead Sciences Initiates.Two phase 3 studies of investigational antiviral remdesivir for the treatment of COVID-19[EB/OL].(2020-02-26)[2020-04-10].https://www.gilead.com/news-and-press/press-room/press-releases/2020/2/gilead-sciences-initiates-two-phase-3-studies-of-investigational-antiviral-remdesivir-for-the-treatment-of-covid-19. [8] FDA.Coronavirus (COVID-19) update:FDA continues to facilitate development of treatments[EB/OL].(2020-03-19)[2020-04-11].https://www.fda.gov/news-events/press-announcements/coronavirus-covid-19-update-fda-continues-facilitate-development-treatments. [9] EMA.Guideline on compassionate use of medicinal products[EB/OL].(2007-07-19)[2020-04-10].https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/guideline-compassionate-use-medicinal-products-pursuant-article-83-regulation-ec-no-726/2004_en.pdf [10] EMA.Conditions of use,conditions for distribution and patients targeted and conditions for safety monitoring addressed??? to member states[EB/OL].(2020-03-03)[2020-04-05].https://www.ema.europa.eu/en/documents/other/conditions-use-conditions-distri-bution-patients-targeted-conditions-safety-monitoring-adressed_en-2.pdf. [11] EMA.Questions and answers on the compassionate use of medicines in the European Union[EB/OL].(2010-01-21)[2020-03-15].https://www.ema.europa.eu/en/documents/other/questions-answers-compassionate-use-medicines-european-union_en-0.pdf [12] EMA.How to request an opinion[EB/OL].(2010-01-21)[2020-04-15].https://ec.europa.eu/health//sites/health/files/files/eudralex/vol-1/reg_2004_726/reg_2004_726_en.pdf. [13] EMA.Compassionate use programmes in Europe[EB/OL].(2017-09-20)[2020-04-10].https://www.ema.europa.eu/en/documents/presentation/presentation-compassionate-use-programmes-europe-role-ema-member-states-cbouygues-ema_en.pdf. [14] CFDA.The general office of the General Administration of the People's Republic of China openly solicits opinions on the administrative measures for the use of drugs for clinical trials with extended sympathy (Draft for Soliciting Opinions)[EB/OL].(2017-12-15)[2020-04-13].http://www.nmpa.gov.cn/WS04/CL2095/229372.html. [15] NMPA.Law of the People's Republic of China on Pharmaceutical Administration[EB/OL].(2019-08-27)[2020-02-13].http://www.nmpa.gov.cn/WS04/CL2076/357712.html. [16] WHO.Ebola virus disease[EB/OL].(2019-12-01)[2020-04-18]https://www.who.int/news-room/fact-sheets/detail. [17] NHC.Special examination and approval procedures for drugs of State Food and Drug Administration[EB/OL].(2005-11-18)[2020-02-15].http://www.nhc.gov.cn/wjw/bmgz/201105/5c7bd2102c3749af968bb2816efc770e.shtml. [18] Sun YX,Wei FF,Feng XC,et al.Evolution and development of extended use system of clinical trial drugs in the United States[J].Chinese Journal of New Drugs(中国新药杂志),2017 (16):1880-1886. [19] Zou LM,Qi Y,Li TL.The enlightenment of E2E guidelines on post marketing risk management of innovative drugs in China[J].Chinese Journal of Pharmacovigilance(中国药物警戒),2019,16(11):670-674.