Frontiers in Medicine (Aug 2021)
Defining Satisfactory Methods of Treatment in Rare Diseases When Evaluating Significant Benefit–The EU Regulator's Perspective
- Maria E. Sheean,
- Frauke Naumann-Winter,
- Frauke Naumann-Winter,
- Giuseppe Capovilla,
- Giuseppe Capovilla,
- Giuseppe Capovilla,
- Maria Elisabeth Kalland,
- Maria Elisabeth Kalland,
- Eva Malikova,
- Eva Malikova,
- Eva Malikova,
- Segundo Mariz,
- Darius Matusevicius,
- Darius Matusevicius,
- Robert Nistico,
- Robert Nistico,
- Brigitte Schwarzer-Daum,
- Brigitte Schwarzer-Daum,
- Stelios Tsigkos,
- Kyriaki Tzogani,
- Kristina Larsson,
- Armando Magrelli,
- Armando Magrelli,
- Violeta Stoyanova-Beninska,
- Violeta Stoyanova-Beninska
Affiliations
- Maria E. Sheean
- European Medicines Agency, Amsterdam, Netherlands
- Frauke Naumann-Winter
- Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, Netherlands
- Frauke Naumann-Winter
- Bundesinstitut für Arzneimittel und Medizinprodukte, Bonn, Germany
- Giuseppe Capovilla
- Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, Netherlands
- Giuseppe Capovilla
- Carlo Poma Hospital, Mantova, Italy
- Giuseppe Capovilla
- Fondazione Poliambulanza, Brescia, Italy
- Maria Elisabeth Kalland
- Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, Netherlands
- Maria Elisabeth Kalland
- Statens Legemiddelverk, Oslo, Norway
- Eva Malikova
- Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, Netherlands
- Eva Malikova
- State Institute for Drug Control, Bratislava, Slovakia
- Eva Malikova
- Department of Pharmacology and Toxicology, Comenius University, Bratislava, Slovakia
- Segundo Mariz
- European Medicines Agency, Amsterdam, Netherlands
- Darius Matusevicius
- Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, Netherlands
- Darius Matusevicius
- Läkemedelsverket, Uppsala, Sweden
- Robert Nistico
- Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, Netherlands
- Robert Nistico
- 0Malta Medicines Authority, San Gwann, Malta
- Brigitte Schwarzer-Daum
- Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, Netherlands
- Brigitte Schwarzer-Daum
- 1Medical University of Vienna, Vienna, Austria
- Stelios Tsigkos
- European Medicines Agency, Amsterdam, Netherlands
- Kyriaki Tzogani
- European Medicines Agency, Amsterdam, Netherlands
- Kristina Larsson
- European Medicines Agency, Amsterdam, Netherlands
- Armando Magrelli
- Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, Netherlands
- Armando Magrelli
- 2National Center for Drug Research and Evaluation, Istituto Superiore di Sanità, Rome, Italy
- Violeta Stoyanova-Beninska
- Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, Netherlands
- Violeta Stoyanova-Beninska
- 3College ter Beoordeling van Geneesmiddelen, Utrecht, Netherlands
- DOI
- https://doi.org/10.3389/fmed.2021.744625
- Journal volume & issue
-
Vol. 8
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
- orphan designation
- satisfactory methods of treatment
- significant benefit
- orphan regulation
- committee for orphan medicinal products
