The pharmaceutical industry is vital for safeguarding people’s lives and health. This study is based on a macro perspective and provides a comprehensive and in-depth comparison regarding the development trends of the pharmaceutical industry in China and abroad. It is pointed out that the global market size and strategic position of the pharmaceutical industry in various countries are constantly improving. Meanwhile, the development prospects of China’s pharmaceutical industry are broad, and the potential market space is large. On this basis, we further review and explore the driving factors behind the stable and rapid development of China’s pharmaceutical industry in recent years, which include demographic changes, global economic instability, and continuous investment from innovative entities such as governments and enterprises. Moreover, from the perspective of patients, we propose the challenges faced by the pharmaceutical industry in five aspects: precision, safety, timeliness, affordability, and policy guarantee. Furthermore, targeted policy recommendations are proposed to focus on building and maintaining a refined large-scale population cohort and data platform, sorting out China’s cutting-edge technology fields, promoting the industrialization of pharmaceutical research and development, improving multi-level medical security channels, and enhance the full chain of policy protection and the implementation efficiency, thus to provide a reference for subsequent investment, policy formulation, and development decisions for China’s pharmaceutical industry.