Consideration on " Patient-Centered" Drug Development for Rare Diseases

The unmet clinical needs of patients with rare diseases persist. Many rare diseases lack effective treatments, and drug development for rare diseases faces greater challenges than that for common multiple diseases. In recent years, the concept of " patient-centered" drug development has been widely adopted. The Center for Drug Evaluation(CDE) of the National Medical Products Administration has successively issued a series of relevant guiding principles, such as the Technical Guidelines for the Implementation of Patient-Centered Clinical Trials, to promote a " patient-centered" drug development model. The implementation of the " patient-centered" approach in rare disease drug research and development, with a focus on patient perspectives and active engagement, can effectively facilitate a comprehensive understanding of rare diseases and patient needs among drug research and development enterprises, researchers, and regulatory agencies. This approach also enhances the accuracy and efficiency of rare disease drug research and development. The CDE will continue to prioritize the integration of the " patient-centered" concept into rare disease drug research and development, effectively enhance the involvement of rare patients in the drug research and development process, and leverage the guiding role of patients′ perspectives on drug research and development.