Issues Related to Expanded Access to Investigational Drugs for Compassionate Use

doi:10.19803/j.1672-8629.2021.01.09

Abstract

Abstract: Objective To study the laws and regulations and application approval by some foreign drug regulatory agencies concerning expanded access to investigational drugs for treatment use so as to provide reference for the design of related guidelines in China.Methods By referring to the laws and regulations,technical guidelines,annual reports and relevant literature issued by drug regulatory agencies of the United States and the European Union,the relevant concepts,categories,application requirements,risk and benefit assessment of drugs used in overseas extended clinical trials were analyzed in depth.Results and Conclusion Compassionate use of clinical trial drugs is a potential way in which patients obtain research drugs when they are suffering from serious diseases or life-threatening diseases that cannot be effectively treated with marketed drugs.As research drugs are not approved into the market,evidence about their safety and efficacy is lacking.In order to ensure the safety of subjects,ethical committees need to perform their duties by conducting strict examination of treatments.

Key words: expanded access, compassionate use, clinical trials

CLC Number:

R994.11

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.

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