Exploration of a new way of drug accessibility for patients with rare diseases in China: the system of compassionate use under the epidemic situation

ABSTRACT

Under the outbreak of COVID-19, the unapproved experimental drug remdesivir was used by the US in order to treat the first diagnosed COVID-19 patient, following the principle of compassionate use. The application of compassionate use also provides a new way of drug accessibility for patients with rare diseases. By analyzing the relevant official websites, laws and regulations, guidelines and domestic and foreign literature of the US, the European Union, France and the UK, this paper focused on the analysis of the use of compassionate use system in the US, the UK and France, so as to provide reference for the application of compassionate use of drugs under clinical trials for patients with rare diseases in China. Both the Expanded Access (EA) and the Right To Try system in the US provide patients with access to experimental drugs in advance. French Autorisation Temporaire D'utilisation (ATU) shortens the waiting time of patients to obtain drugs and accelerates the treatment of more than 20 000 patients (rare and non-rare diseases) within three years. The UK's Early Access to Medicines Scheme (EAMS) combined with subsequent drug marketing and reimbursement, shortens the corresponding approval time for drugs. It is suggested that when implementing the compassionate use system for rare diseases in line with the national conditions of China, such important aspects be fully considered as simple and flexible methods, procedures for quick approval, reasonable cost-sharing mechanism, and system to ensure drug safety. Meanwhile, ensuring patients' right to know about drug use, opening and sharing information platform, effective incentive policy and other important aspects should be noted.