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. 2021 Aug 27:8:744625.
doi: 10.3389/fmed.2021.744625. eCollection 2021.

Defining Satisfactory Methods of Treatment in Rare Diseases When Evaluating Significant Benefit-The EU Regulator's Perspective

Maria E Sheean  1 , Frauke Naumann-Winter  2   3 , Giuseppe Capovilla  2   4   5 , Maria Elisabeth Kalland  2   6 , Eva Malikova  2   7   8 , Segundo Mariz  1 , Darius Matusevicius  2   9 , Robert Nistico  2   10 , Brigitte Schwarzer-Daum  2   11 , Stelios Tsigkos  1 , Kyriaki Tzogani  1 , Kristina Larsson  1 , Armando Magrelli  2   12 , Violeta Stoyanova-Beninska  2   13
Affiliations

Defining Satisfactory Methods of Treatment in Rare Diseases When Evaluating Significant Benefit-The EU Regulator's Perspective

Maria E Sheean et al. Front Med (Lausanne). .

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.

Keywords: committee for orphan medicinal products; orphan designation; orphan regulation; satisfactory methods of treatment; significant benefit.

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Conflict of interest statement

The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

References

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