In the European Union demonstration of ‘significant benefit’ is mandatory if satisfactory methods exist for a disease targeted by a new orphan medicinal product. Significant benefit is required at the time of orphan designation, when it can be supported by preclinical studies, and at the time of marketing authorization, when clinical data are needed. For the first time, our work has identified, defined and organized the scientific grounds on which significant benefit is granted in the European Union, based on a review of the orphan medicinal products authorized in the years 2000–2015, and on the working experience of the Committee of Orphan Medicinal Products. The resulting conceptual framework is a tool for medicine developers to reflect on potential areas of advantage of their candidate products, and for a broad range of stakeholders to stimulate the discussion on the added value of orphan medicines across the whole development lifecycle.

在欧洲联盟，如果存在针对新的孤儿药物产品针对的疾病的令人满意的方法，则必须证明“具有重大益处”。在指定孤儿时（如果可以得到临床前研究的支持），以及在需要上市数据时（需要临床数据），都需要显着的益处。我们的工作首次基于对2000-2015年授权的孤儿药产品的审查，并根据2000年至2015年的工作经验，确定，定义和组织了可在欧盟中获得重大利益的科学依据。孤儿药品委员会。由此产生的概念框架是药物开发人员反思其候选产品的潜在优势领域的工具，