Intractable and rare diseases are an important public health issue and a challenge to medical care. In recent years, much progress has been made in the United States (US), the European Union (EU), and some parts of Asia including Japan, South Korea, and Taiwan, involving specific legislation to encourage discovery and development of orphan drugs, patients’ advocacy organizations to provide vast information on intractable and rare diseases and improve patients’ access to healthcare, special research programs to strengthen basic and applied research on intractable and rare diseases, and so on. While China is also actively promoting regulation of intractable and rare diseases, but still lags far behind the US, EU, Japan, and other countries and regions with orphan drug legislation. Based on systematic analysis of the current status and future perspectives for intractable and rare diseases in Asia, we recommend that three important aspects of support from government, patients’ advocacy organizations and rare disease registry networks, special research programs and global information exchange platform, should be given great attention in promoting the development of intractable and rare diseases research in Asian countries.
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