神経難病と療養支援の現状と今後の課題

抄録

In Japan, support for intractable diseases has entered a new era after legislation of the Act on Medical Care for Patients with Intractable Diseases in 2015 following the formulation of the Guidelines for Measures against Intractable Diseases. The challenges faced in treatment of intractable neurological diseases lie in the great diversity, complexity and individuality of symptoms, as no two people are alike. Furthermore, there are practical challenges associated with utilizing social resources, improving the support system. It is no exaggeration to say that the patient's quality of life (QOL) is determined by whether various social resources can be utilized or not. Therefore, when considering treatment support, it is critical to have a perspective of the illness course and to grasp the whole picture rather than being informed of only a certain treatment stage. The illness course begins with the “onset phase”, which is the period spanning the development of symptoms to the diagnosis of the disease. This is followed by the “progressive phase”, during which mild health problems and difficulties in daily activities become severe, followed by the “transitory phase”, which is when the patient makes various decisions about where they will receive care, which medical procedures they will undergo, and makes “links” based on these decisions ; this is then followed by the “sustenance and stable phase”. Since the disease is progressive, the patient repeatedly undergoes these four phases. However, even in the “sustenance and stable phase”, the patient experiences the “changing phase”, wherein the patient suffers from the development of novel and hither to unknown symptoms (complications or associated symptoms), but moves in time to the “terminal phase”. One characteristic of intractable neurological diseases is that this “terminal phase” cannot be clearly demarcated from other phases. This article provids phase–by–phase points to improve support for intractable diseases and serves an overview of experts' efforts and the challenges they face in working towards the development of support for such diseases.