Sheean Maria E, Naumann-Winter Frauke, Capovilla Giuseppe, Kalland Maria Elisabeth, Malikova Eva, Mariz Segundo, Matusevicius Darius, Nistico Robert, Schwarzer-Daum Brigitte, Tsigkos Stelios, Tzogani Kyriaki, Larsson Kristina, Magrelli Armando, Stoyanova-Beninska Violeta
European Medicines Agency, Amsterdam, Netherlands.
Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, Netherlands.
Front Med (Lausanne). 2021 Aug 27;8:744625. doi: 10.3389/fmed.2021.744625. eCollection 2021.
Since the implementation of the EU Orphan Regulation in 2000, the Committee for Orphan Medicinal Products at the European Medicines Agency has been evaluating the benefits of proposed orphan medicines vs. satisfactory treatment methods. This type of evaluation is foreseen in the Orphan Regulation as the orphan designation criterion called the "significant benefit." In this article, based on 20 years of experience, we provide a commentary explaining what is considered a satisfactory method of treatment in the context of the EU Orphan Regulation and for the purpose of the assessment of significant benefit. We discuss the challenges posed by continuously changing clinical practise, which is associated with the increasing number of treatment options, evolving nature of medicinal therapeutic indications and our understanding of them.
自2000年欧盟孤儿药法规实施以来，欧洲药品管理局的孤儿药品委员会一直在评估拟议的孤儿药相对于令人满意的治疗方法的益处。孤儿药法规将这种评估作为称为“显著益处”的孤儿药认定标准。在本文中，基于20年的经验，我们提供一篇评论，解释在欧盟孤儿药法规背景下以及为评估显著益处的目的，什么被视为令人满意的治疗方法。我们讨论了不断变化的临床实践所带来的挑战，这与治疗选择的增加、药物治疗适应症的不断演变以及我们对它们的理解有关。
