Rare diseases, orphan drugs, and their regulation in Asia: Current status and future perspectives

Worldwide collaboration for orphan drug designation

“…The MHLW/PMDA require similar considerations to those requested by the EMA when describ ing the orphan condition in an application 3 . • Financial subsidies for up to 50% of expenses for clinical and non-clinical research • Subsidies through the NIBIO to reduce the financial burden of product development • User fee waivers, 15% tax credits, up to 20% corporate tax reduction and a 30% reduction in marketing application fees…”

Towards government-funded special biomedical research programs to combat rare diseases in China

“…Given this reality, the government should assume the responsibility of taking on the important task of promoting the sustained development of a system of medical care for and research into rare diseases. In addition to efforts to specifically define and classify rare diseases, specific legislation has been drafted to encourage discovery and development of orphan drugs, and the health insurance system for rare diseases has been improved in many countries and regions, such as the USA, EU, Australia, Japan, and South Korea (8). Government-funded special biomedical research programs to enhance basic and applied research on rare diseases should also receive full attention.…”

“…In Western countries, many research centers or projects have been established to support special biomedical research programs on rare diseases and development of orphan drugs, such as the Office of Rare Diseases Research (ORDR) established in the USA in 1993 within the National Institutes of Health (NIH) and the Rare Disease Task Force (RDTF) established in the EU in 2004 within the European Commission Public Health Directorate (8). In Asian countries, biomedical research on rare diseases has made great advances in Japan due to the system known as the Specified Disease Treatment Research Program established in 1972 with the support of the Ministry of Health, Labor, and Welfare (MHLW) (12).…”

Defining rare diseases in China

“…At a seminar conducted by the Genetics Branch of the Chinese Medical Association on May 17, 2010, experts mainly in the field of medical genetics suggested that rare diseases in China be defined as "disorders with a prevalence less than 1/500,000 or with an incidence less than 1/10,000 among newborns" (4). This definition sets a threshold lower than all currently established definitions in use worldwide, thus excluding most recognized rare disorders.…”