Core Viewpoint - The article highlights the severe shortage of rare disease medications, emphasizing the challenges in research and development for "orphan drugs" due to high costs, long timelines, and limited market returns [1][2][6]. Group 1: Current Situation of Rare Disease Medications - There are over 7,000 known rare diseases globally, affecting more than 300 million patients, yet less than 10% have approved treatments [1]. - In China, the number of rare disease patients exceeds 20 million, with over 200,000 new cases each year [1]. - The lack of effective treatments leads patients to rely on expensive imported drugs, as domestic drug development is slow [4][10]. Group 2: Challenges in R&D for Orphan Drugs - The development of innovative drugs typically requires over $1 billion and takes 10-15 years, with orphan drug development facing even greater difficulties [4][7]. - Domestic pharmaceutical companies show low enthusiasm for orphan drug development due to high costs, long timelines, and uncertain commercial returns [7][11]. - A weak foundation in basic research and a lack of systematic patient registration hinder the progress of rare disease studies [5][13]. Group 3: Innovations and Approaches in Drug Development - Some companies are adopting a "global first" strategy, seeking FDA approval for orphan drug status before introducing products to the domestic market [8]. - The introduction of generic drugs, such as domestically produced chlorobazam, significantly reduces treatment costs and improves accessibility for patients [9][10]. - Innovative drugs, like the one developed by Wuhan New Force Biotechnology, are nearing approval, showcasing the potential for breakthroughs in rare disease treatment [8]. Group 4: Policy and Support for Rare Disease Drug Development - Recent years have seen an increase in policy support for rare disease medications in China, with 43 rare disease treatments approved from 2018 to 2022 [10][11]. - The article suggests that a multi-faceted approach, including tax incentives and improved approval processes, is necessary to enhance R&D motivation for orphan drugs [12][13]. - Collaboration among government, pharmaceutical companies, and research institutions is essential to establish a supportive ecosystem for rare disease drug development [12][13].