Baumevieille Marie, Daveluy Amélie, Aulois-Griot Marine, Haramburu Françoise
Laboratoire Droit et Economie de la Santé, Université Victor Segalen Bordeaux 2, Bordeaux, France.
Therapie. 2007 Jan-Feb;62(1):9-16. doi: 10.2515/therapie:2007005. Epub 2007 Mar 21.
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.).
2000年，欧盟通过了两项法规，以提高孤儿药在欧盟的可及性。为描述法国当前的孤儿药市场，五年后进行了一项横断面分析，考虑了包括指定、注册、报销等公开可用信息。这些信息大多可在互联网上获取。2005年11月，法国有18种孤儿药上市，其处方和配药条件受限。其中4种在注册前可用于同情用药。8种将很快重新评估，卫生当局要求提供补充数据。关于孤儿药的信息分散在不同的法国或欧洲网站上，这给卫生专业人员获取信息带来了困难。评估药物效用的“透明度委员会”的意见为每种孤儿药提供了一些详细信息（疾病患病率、治疗替代方案等）。
