Ten years have passed since OrphanPacific was established. The environment surrounding rare diseases has been gradually changing over the past 10 years. Many pharmaceutical companies have focused on rare diseases as their target therapeutic area for research and development activities and it has become one of several key strategic areas for companies. Indeed, awareness of rare diseases is also gradually improving thanks to awareness-raising activities such as Rare Disease Day, which is a great pleasure to see.
On the other hand, there are reports that about half of the orphan drugs approved in Europe and the U.S. are not approved in Japan, and about one third do not have information on development in Japan (possibly development has not commenced), so we have to say that access to therapeutic drugs is still limited. Because of the small number of patients with many rare diseases and the lack of epidemiological data on the clinical courses of each disease, it is difficult to develop a drug and would take very long time. In addition, the low awareness of diseases and the difficulty in obtaining accurate information often causes patients and their families to feel alone, and make it difficult for physicians to diagnosis and treat those patients.
In response to these challenges surrounding rare diseases, OrphanPacific aims to develop and launch orphan drugs with a new business model called the Innovative Pharma Model, and to deliver remedies to patients with rare diseases, making the system sustainable. Our challenge to bring smiles and happiness to as many patients with rare diseases and their families as possible, has just begun.