34 - Expanded access: analysis of the current regulatory landscape and ethical challenges in China

Background:  In recent years, the utilization of expanded access in China has increased significantly and is gradually progressing toward standardization and institutionalization. However, there are still regulatory gaps to mitigate, require more legally binding regulations to safeguard the best interest of the patients through high-quality ethical review and responsible use.

Methods:  This study explored the historical evolution and current application status of expanded access in China through literature review and case analysis. At the same time, international experiences are also analyzed from a comparative perspective. Finally, a regulatory landscape analysis was developed to identify particular challenges and concerns in the Chinese context.

Conclusion:  It has been observed that China has made efforts to promote expanded access in recent years, encouraging institutions to comply with regulations and striving to establish a more robust framework. However, more efforts are needed to streamline the process and the stakeholder engagement. Regarding the ethical challenges identified, whether the patient's best interest is guaranteed was always the first concern in expanded access, while there are no other alternative choices. Secondly, responsible use and mechanisms are essential to ensure the appropriate jurisdiction. Thirdly, fees, compensation, and fair access to all patients in need should also be taken seriously. Furthermore, reasonable, voluntary informed consent is one of the most critical ethical issues.

Limitations: Due to the variations among different countries and the fast evolution of the ethics review regulatory framework in China, more tailored research is needed to explore the perceptions and voices of different stakeholders regarding expanded access in the Chinese context. A more bottom-up strategy should be developed.

Discussion:  A more strategic effort should be made to engage various stakeholders in expanded access. It is significant to ensure that all the stakeholder voices are heard and a tailored participatory governance mechanism is developed. Among these, legal responsibilities and ethical imperatives should be identified as working priorities to promote responsible expanded access in China.