This article explores the role of ChatGPT in improving the diagnosis and treatment of rare and complex diseases. Rare and complex diseases pose significant challenges due to their atypical symptoms and limited understanding. Artificial intelligence (AI) and natural language processing (NLP) technologies have shown promise in assisting medical professionals in diagnosing and managing these conditions. ChatGPT, an advanced AI model, is discussed as a tool that can contribute to better patient outcomes by analyzing symptoms, accessing medical literature, and aiding in the development of personalized treatment plans. The article highlights the potential of ChatGPT in diagnosing rare diseases by analyzing patient symptoms and generating diagnostic suggestions. It also discusses its role in disease prediction, early screening, and drug discovery. However, the effectiveness of ChatGPT is dependent on the quality and diversity of the data it is trained on, and there are ethical considerations related to algorithmic bias and patient data privacy. The article emphasizes the importance of collaboration between AI and healthcare professionals to ensure patient safety and the provision of quality care. It also notes that while ChatGPT can provide valuable assistance, it should not replace the expertise of medical professionals. The integration of AI into healthcare must be done responsibly, with a focus on ethical considerations and patient well-being. The study was supported by various funding sources, including the National Natural Science Foundation of China and the Sichuan Province Foundation. The authors declare no conflicts of interest. The article concludes that the role of AI in healthcare is likely to expand, offering innovative solutions for addressing the diagnostic and therapeutic challenges of rare and complex diseases.This article explores the role of ChatGPT in improving the diagnosis and treatment of rare and complex diseases. Rare and complex diseases pose significant challenges due to their atypical symptoms and limited understanding. Artificial intelligence (AI) and natural language processing (NLP) technologies have shown promise in assisting medical professionals in diagnosing and managing these conditions. ChatGPT, an advanced AI model, is discussed as a tool that can contribute to better patient outcomes by analyzing symptoms, accessing medical literature, and aiding in the development of personalized treatment plans. The article highlights the potential of ChatGPT in diagnosing rare diseases by analyzing patient symptoms and generating diagnostic suggestions. It also discusses its role in disease prediction, early screening, and drug discovery. However, the effectiveness of ChatGPT is dependent on the quality and diversity of the data it is trained on, and there are ethical considerations related to algorithmic bias and patient data privacy. The article emphasizes the importance of collaboration between AI and healthcare professionals to ensure patient safety and the provision of quality care. It also notes that while ChatGPT can provide valuable assistance, it should not replace the expertise of medical professionals. The integration of AI into healthcare must be done responsibly, with a focus on ethical considerations and patient well-being. The study was supported by various funding sources, including the National Natural Science Foundation of China and the Sichuan Province Foundation. The authors declare no conflicts of interest. The article concludes that the role of AI in healthcare is likely to expand, offering innovative solutions for addressing the diagnostic and therapeutic challenges of rare and complex diseases.