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Rare Disease Pharmaceuticals in Japan to See New Demand for Skilled Human Resources

Philip Carrigan

According to recent statistics on the prevalence rate of rare diseases, per 10,000 individuals, the United States has 7.5, Japan 4, and Australia 1.2 patients with rare diseases. The most important challenge drug developers and pharmaceutical companies face with any rare disease is the scarcity of literature and guidelines. It is difficult for developers to decide on an endpoint to the examination process and to anticipate any potential effects of treatment. Without information on the natural history of the disease, designing a clinical trial is tricky. Rare diseases represent both a challenge and an opportunity for drug developers and regulators ...