Hopes for intractable and rare diseases research

Abstract

February 28, 2013 marks the sixth international "Rare Disease Day". On and around this day, hundreds of patient organizations from more than 60 countries and regions worldwide plan to host awareness campaigns in line with this year's theme, "Rare Disorders without Borders". Public awareness of intractable and rare diseases has heightened in recent decades. Much progress has also been made worldwide, such as specific legislation to encourage discovery and development of orphan drugs in the United States (US), the European Union (EU), and some parts of Asia. However, there are still many gaps in knowledge with regard to therapeutic tools and strategies. Intractable and rare diseases cause patients substantial physical suffering, psychological despair, and economic hardships due to bleak therapeutic outcomes and the lack of practical support in everyday life. The features of intractable and rare diseases and the increasing number of types of identified diseases make these diseases an important public health issue and a challenge to medical care worldwide. The following are specific aspects of research on intractable and rare diseases that need to be promptly promoted. ...