<p>“Patient-focused drug development” emphasizes multi-stakeholder collaboration and advocates for in-depth listening to patients’ opinions to facilitate all stages of drug development. In the field of rare disease drug development, implementing the concept of “patient-centered” drug development, focusing on patients’ perspectives, and heeding patients’ voices will serve as an effective approach for pharmaceutical companies, researchers, and regulatory authorities to gain a deeper understanding of rare diseases and patients’ needs, thereby enhancing the precision and efficiency of rare disease drug development. The “Care Program” is a pilot program launched by the Center for Drug Evaluation, NMPA focusing on rare diseases. Its aim is to guide pharmaceutical companies to incorporate the perspectives of rare disease patients throughout the entire drug development process and improve the scientific rigor and standardization of integrating patients’ voices into the clinical development of rare disease drugs. The launch of the “Care Program” is expected to accumulate valuable experience for future regulatory work and drug development efforts.</p>