Orphan Drug Development and Challenges in Japan

This document is a technical report that discusses the complexities surrounding rare diseases and the development of orphan drugs in Japan. It outlines the definition of rare diseases, which affect a small number of patients, and the criteria for designating orphan drugs, including the necessity for high medical need and the absence of alternative treatments. The report details the establishment of OrphanPacific by CMIC Group in 2012, aimed at addressing the challenges of drug development for rare diseases. It highlights the societal impact of these diseases, affecting approximately 350 million patients worldwide, and the lack of accessible information for the public. The document also describes initiatives taken by OrphanPacific to engage with patients and their families to better understand their needs, and the company's efforts to create sustainable business models that support the delivery of medications. Furthermore, it discusses the 'drug loss/drug lag' issue in Japan and how CMIC Group's Pharmaceutical Value Creator model assists foreign companies in launching orphan drugs in the Japanese market.