Core Viewpoint - The current challenges in rare disease medication include low accessibility, high burden, insufficient diagnostic and treatment systems, regional disparities in support, conflicts between payment systems and high drug prices, and inadequate incentives for drug research and market development [1][2]. Group 1: Current Challenges - Rare disease medications face low accessibility and high financial burden on patients [1]. - There is a lack of a comprehensive diagnostic and treatment system, leading to insufficient support capabilities [1]. - Disparities in medication support exist across different regions [1]. - There are conflicts between the payment systems and the high prices of medications [1]. - The current drug research and market incentives are insufficient to address the needs of rare diseases [1]. Group 2: Opportunities and Recommendations - Increased attention to rare diseases in China has created opportunities for medication support [2]. - Proposed measures include optimizing the entry process for rare disease medications and conducting price negotiations through the medical insurance system [2]. - A multi-layered medical security and comprehensive assistance system led by the government is necessary to address the challenges [2]. - Recommendations include innovating the medical insurance entry model by adopting international risk-sharing agreements tailored to China's context [2][3]. - Optimizing outpatient insurance policies to create a collaborative multi-layered security system is essential [3]. - Establishing a special fund for rare disease medication with provincial-level coordination and independent accounting is recommended [3].