Grienenberger Aurelie
Regulatory Affairs and Drug Development, Voisin Consulting, Paris, France.
J Biolaw Bus. 2004;7(3):58-61.
Whereas orphan regulations exist in almost all ICH regions, they may correspond to significantly different types of reality in terms of principles and regulatory requirements. The apparent similarity in regulations may lead to misunderstanding and cultural gaps between sponsors and regulatory authorities. Based on the European and the US experience, we decipher the main points stumbling blocks that US sponsor may strike when applying for an orphan designation in the EU. An update of the orphan drugs designations is provided as well as the upcoming challenges that orphan regulations will meet with the development of pharmaco-genomics and personalized medicines.
虽然几乎所有国际人用药品注册技术协调会（ICH）地区都存在孤儿药法规，但在原则和监管要求方面，它们可能对应着截然不同的实际情况。法规表面上的相似性可能导致申办方与监管机构之间产生误解和文化差异。基于欧洲和美国的经验，我们解读了美国申办方在欧盟申请孤儿药认定时可能遇到的主要障碍。同时还提供了孤儿药认定的最新情况，以及随着药物基因组学和个性化药物的发展，孤儿药法规即将面临的挑战。
