The first drug to treat an incurable disease called "distal myopathy," which is said to affect only about 400 patients in Japan, has been approved by the government, and on the 28th, a patient group held a press conference to discuss the need for a drug to treat a serious rare disease for which there is no cure. appealed for prompt approval.
The press conference was held by a group of patients suffering from ``distal myopathy,'' an incurable disease that affects approximately 400 patients in Japan and causes gradual muscle weakness.
Until now, there was no cure for this incurable disease, but after 15 years of research and development, on the 26th, the first drug to slow the progression of symptoms was approved, ahead of the rest of the world.
Yuriko Oda, a representative of a patient group who attended the press conference, said, ``I am extremely happy and grateful for the new drug's approval.However, some people have died while receiving the drug, and I myself have experienced symptoms that have progressed.'' I wanted to take medicine before I did that."
Currently, Japan has a system for early approval of therapeutic drugs for rare diseases for which there is no cure, subject to certain conditions. Although safety had been confirmed, efficacy had not been confirmed in overseas clinical trials, so approval under this system was not achieved and additional data had to be submitted.
Oda said, ``For rare diseases for which there are no treatments, if a certain level of safety is recognized, I would like to see a system in which drugs are approved early, in order to promote the development of new drugs for rare diseases.'' I appealed.
The patient group plans to submit a written request to the government soon.
A website that connects rare disease patients
One of the challenges faced by rare diseases is the difficulty in recruiting participants when conducting clinical trials for therapeutic drugs, and it has been pointed out that this is due to the extremely small number of patients, making it difficult to create networks such as patient associations. I am.
In an effort to support interaction and collaboration among patients with these serious rare diseases, the ``Japan Council for Intractable Diseases and Illness Groups,'' created by patient associations for intractable and chronic diseases, has released a website that introduces information on patient associations. I am.
This site, commonly known as ``Nan Community,'' has registered 67 incurable diseases that are thought to affect less than 10,000 people.By entering the name of the disease, you can see SNS posted by patients facing the same disease. This means that you can search for contact information, etc.
Kunio Tsuji, executive director of the Japan Association for Intractable Diseases and Diseases, who is himself a patient with an intractable neurological disease, said, ``Recently, information on clinical trials is increasingly being shared between patient groups and pharmaceutical companies. "Collaboration between patients and medical care has become increasingly important. Through this site, we would like to provide patients with access to information that can help them."