Orphan drugs: A problem of public health and an economic stake

Orphan drugs (OD) qualify as drugs scientifically viable but not viable from an economic point of view either because the number of patients who might benefit is too small or because the populations concerned are too poor to afford the drugs. Sick people who could be treated by OD and their families have been fighting for years to induce authorities in charge of health to set up a programme to stimulate research into new treatment for rare diseases. The United States, initiated work in order to confer status on OD leading to the Orphan Drug Act in 1983. It defined the conditions for attribution of the OD status and also made attractive proposals to pharmaceutic industries in order to improve their development. More recently, in 1993,Japan took similar decisions, while the European Union and France are also on the way, as recent meetings of European ministers of health show. In contrast, developing countries are still excluded from medical res earch as very few tropical dis eases have treatment. Legislation for OD, first planned to make up for the high costs of research and development, proved its efficiency. But ODs must move to a new status as some are now becoming the object of important economic stakes.