Bioscience Evidence 2024, Vol.14, No.1, 16-23 http://bioscipublisher.com/index.php/be 21 Despite the challenges in technology and data, significant progress can be expected in the coming years with the rapid development of fields such as artificial intelligence, machine learning, and data science. By integrating multiple technologies and data resources, it is expected to overcome technological and data challenges and bring new breakthroughs to the discovery of rare disease drugs. 4.2 Cooperation and policy environment Promoting the discovery of rare disease drugs requires establishing a good cooperation and policy environment. Firstly, collaboration is a key factor in promoting research on rare diseases and drug discovery. Due to the limited number of patients with rare diseases, it is difficult for a single institution to obtain sufficient samples and data for research (Groft et al., 2021). Therefore, establishing an international cooperation network, including medical institutions, research institutions, pharmaceutical companies, and patient organizations, is key to promoting the discovery of rare disease drugs. This cooperation can promote data sharing, resource sharing, and knowledge exchange, accelerate research progress and drug development. The policy environment also plays an important role in promoting the discovery of rare disease drugs, and governments and regulatory agencies need to formulate corresponding policies and regulations to promote the research and marketing of rare disease drugs (Wen et al., 2018). This may include providing research and development funding, optimizing evaluation processes, implementing incentive measures, and reducing market entry barriers. The government should also strengthen the protection of patients with rare diseases, ensuring that they have access to appropriate medical resources and medication treatment. With the support of cooperation and policy environment, more progress can be expected in the discovery of rare disease drugs. International cooperation will strengthen research capabilities, promote knowledge sharing and technological exchange. Meanwhile, a favorable policy environment will provide more impetus and support for research institutions and pharmaceutical companies to promote the research and marketing of rare disease drugs. 4.3 Future development trends In the future, the discovery of rare disease drugs will face some key development trends, and personalized healthcare will become an important direction for rare disease drug discovery. With the rapid development of genomics and biotechnology, it is possible to better understand the genetic mechanisms of rare diseases and provide personalized treatment plans for patients. Through gene sequencing and analysis, it is possible to identify mutated genes in patients and develop targeted drugs based on their individual characteristics. Personalized healthcare will bring more precise and effective treatment methods to patients with rare diseases (Miller et al., 2021). Drug repositioning and drug combination therapy will also become future development trends. Drug repositioning refers to the application of existing drugs to new pathological targets, which can accelerate the drug development process and reduce research and development costs. The drug combination therapy principle utilizes the synergistic effect of multiple drugs to improve treatment effectiveness. These methods have great potential in the discovery of rare disease drugs, expanding the range of treatment options and improving treatment success rates. The discovery of rare disease drugs in the future will benefit from development trends such as personalized healthcare, artificial intelligence technology, drug repositioning, and combination therapy (Goetz and Schork, 2018). These trends will drive innovation and progress in the treatment of rare diseases, providing patients with better medical choices. With the continuous development of technology and cooperation, there is reason to be confident in the future of rare disease drug discovery. References Bendowska A., and Baum E., 2023, The significance of cooperation in interdisciplinary health care teams as perceived by polish medical students, International Journal of Environmental Research and Public Health, 20(2): 954. https://doi.org/10.3390/ijerph20020954 Boycott K., and Ardigò D., 2017, Addressing challenges in the diagnosis and treatment of rare genetic diseases, Nature Reviews Drug Discovery, 17: 151-152. https://doi.org/10.1038/nrd.2017.246
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