These research projects are conducted on diseases designated under the Act on Medical Care for Patients with Intractable Diseases (Intractable Diseases Law). The designation of intractable disease has to satisfy the following four conditions: 1. Unknown etiology 2. Lack of effective treatment, 3. Rarity, and 4. Necessity of long-term treatment. These designated Intractable Diseases together with Specific Pediatric Chronic Diseases are the subjects of the research projects, which aim to contribute to raising the standards of healthcare received by all patients and to improving their QOL.
The project groups are expected to coordinate with academic societies and national centers concerned, due to the project’s mission to promote measures against intractable diseases and pediatric chronic diseases and to contribute to the promotion of R&D relating to the disease that individual projects are engaged in. An additional and significant role that has to be played by the project groups is the promotion of research using the databases on patients with designated intractable diseases and specific pediatric chronic diseases, together with liaising and coordinating with other relevant databases.
The diseases and syndromes that the project program is tackling are very wide-ranging. The research and measures that need to be promoted are manifoldly diverse, including work on the number of patients and the stages of information diffusion. Therefore, the research projects should not limit their scope to producing and revising clinical practice guidelines but proceed with flexibility of concept, adjusting to the particular characteristics or issues that each disease poses.