Service de pharmacologie clinique et pharmacovigilance, INSERM, Inst Neurosci Syst, Aix-Marseille university, AP-HM, 13005 Marseille, France.
Service de pharmacologie clinique et pharmacovigilance, INSERM, Inst Neurosci Syst, Aix-Marseille university, AP-HM, 13005 Marseille, France; Orphandev-FCRIN, INSERM, 13005 Marseille, France.
Therapie. 2020 Apr;75(2):133-139. doi: 10.1016/j.therap.2020.02.003. Epub 2020 Feb 13.
The research and drug development process in rare diseases is challenging in addition to those for common diseases. To stimulate its development, the orphan drug designation (ODD) was introduced in in European Union in 2000. In the present paper, we describe the main characteristics of ODD in European Union in particular the requested criteria for ODD, the overview of the general procedure and the main incentives for Sponsors and finally the predicted factors related to successful development and marketing approval of orphan drugs after designation. In accordance with regulation, an application for ODD must be submitted to European Agency including a scientific part based on relevant scientific literature related to the condition and results on experimental studies with the specific product (and clinical studies if available). Three following criteria are a central position in this application: medical plausibility, rarity and medical significant benefit. The Committee for Orphan Medicinal Products (COMP) is the European Medicines Agency's (EMA) committee responsible for recommending orphan designation of medicines for rare diseases. Even if pre-submission meetings are not mandatory, EMA strongly encourages sponsors to request a pre-submission meeting with the Agency prior to filing an application. Experience has shown that they have a positive impact on the success rate of the applications. The full application should be submitted in English via secure online portal. ODD makes the sponsor eligible for a number of orphan incentives including the 10-year market exclusivity and the protocol assistance by COMP. Based on literature and on the experience accumulated by our team ORPHANDEV F-CRIN-labelled platform the successful translation of rare disease research into orphan drug discovery is dependent of a clearly justified medical significant benefit, the disease class, its prevalence and the disease-specific scientific output, previous experience of the sponsor with a previous successful orphan drug to the market increased.
除了常见疾病外，罕见病的研究和药物开发过程也极具挑战性。为了刺激其发展，2000 年欧盟引入了孤儿药认定（ODD）。在本文中，我们描述了欧盟孤儿药认定的主要特征，特别是 ODD 的要求标准、一般程序概述、赞助商的主要激励措施，以及最后与指定后成功开发和市场批准孤儿药相关的预测因素。根据规定，ODD 的申请必须提交给包括科学部分的欧洲机构，该科学部分基于与疾病相关的相关科学文献和特定产品的实验研究结果（如有临床研究）。在该申请中，以下三个标准处于中心位置：医学合理性、罕见性和医学重要性。孤儿药品委员会（COMP）是负责推荐罕见病药品孤儿认定的欧洲药品管理局（EMA）委员会。尽管预提交会议不是强制性的，但 EMA 强烈鼓励赞助商在提交申请前请求与该机构进行预提交会议。经验表明，它们对申请的成功率有积极影响。完整的申请应以英文通过安全在线门户提交。ODD 使赞助商有资格获得多项孤儿激励措施，包括 10 年的市场独占权和 COMP 的方案援助。基于文献和我们团队 ORPHANDEV F-CRIN 标记平台的经验，将罕见病研究成功转化为孤儿药发现取决于明确的医学重要性、疾病类别、其流行程度和疾病特异性科学产出、赞助商以前的经验以及以前成功推向市场的孤儿药。
