[Orphan drugs in France (2001-2005): access to information and characteristics]

In year 2000, two european regulations have been adopted in order to increase the availability of orphan drugs in the European Union. To describe the current orphan drug market in France, a cross sectional analysis was conducted, five years later, taking account of publicly available informations, including designation, registration, reimbursement. These informations are mostly available on the Internet. Eighteen orphan drugs were marketed in France in November 2005, with restricted prescription and dispensing conditions. Four of them were available before registration for compassional use. Eight will be reassessed soon, with complementary data requested by health authorities. Informations about orphan drugs are scattered on different French or European websites, which complicate access for health professionals. The opinions of "Commission de Transparence", which assess utility of drugs, give some precisions for each orphan drug (prevalence of the disease, therapeutic alternative, etc.).