Patients expectation for drug discovery development targeting rare cancers

Abstract

In Japan, each year 1 million people are diagnosed as cancer patients, Of which one in five are rare cancer patients, but there are close to 200 different cancer types under this rare cancer category. There are very few patients with each rare cancer and research has not progressed, and the development of drugs is delayed. For that reason, various organizations such as the Cancer Drug Development Forum (CDDF) are active in Europe. In this CDDF, people are working on improving the development efficiency of cancer drugs through collaboration with academic societies, regulatory authorities, policy makers, industry, patient organizations, health technology assessment committee officials, and others. There are very few specialists in rare cancers, and there is also an issue that patients do not know where specialized rare cancer hospitals are. In order to remedy such situation, the neuroendocrine society (ENETS) in Europe launched a specialized hospital system certified by ENETS, and is promoting the consolidation of specialists. In addition, ENETS has formulated clinical practice guidelines, and standard medical treatments are sent to NET medical clinics worldwide. In Japan, efforts to develop new drugs through participation in Western-style multistakeholder forum has not yet developed. From now on, what kind of drug development system is needed to relieve rare cancer patients in Japan? I would like to think about the solution in Japan with academic societies, regulators, pharmaceutical companies, and patient support groups.