Abstract
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.
Highlights
Since the implementation of the European Union (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
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
Significant benefit is a unique European criterion which needs to be evaluated by the Committee for Orphan Medicinal Products (COMP) at initial orphan designation (OD) and at time of marketing authorisation (MA)
Summary
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. 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. The eligibility of a candidate medicine to orphan designation (OD) is assessed by a dedicated Committee for Orphan Medicinal Products (COMP) at the European Medicines Agency (EMA). To provide further information, in this article we aim to explain what constitutes a satisfactory method of treatment This naturally depends on the specific rare disease the medicinal product intends to diagnose, prevent, or treat. The COMP considers the standard of care in identifying appropriate comparators and the target patient population suitable for the analysis of significant benefit
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