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

Autor:	Sheean ME; European Medicines Agency, Amsterdam, Netherlands., Naumann-Winter F; Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, Netherlands.; Bundesinstitut für Arzneimittel und Medizinprodukte, Bonn, Germany., Capovilla G; Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, Netherlands.; Carlo Poma Hospital, Mantova, Italy.; Fondazione Poliambulanza, Brescia, Italy., Kalland ME; Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, Netherlands.; Statens Legemiddelverk, Oslo, Norway., Malikova E; Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, Netherlands.; State Institute for Drug Control, Bratislava, Slovakia.; Department of Pharmacology and Toxicology, Comenius University, Bratislava, Slovakia., Mariz S; European Medicines Agency, Amsterdam, Netherlands., Matusevicius D; Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, Netherlands.; Läkemedelsverket, Uppsala, Sweden., Nistico R; Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, Netherlands.; Malta Medicines Authority, San Gwann, Malta., Schwarzer-Daum B; Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, Netherlands.; Medical University of Vienna, Vienna, Austria., Tsigkos S; European Medicines Agency, Amsterdam, Netherlands., Tzogani K; European Medicines Agency, Amsterdam, Netherlands., Larsson K; European Medicines Agency, Amsterdam, Netherlands., Magrelli A; Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, Netherlands.; National Center for Drug Research and Evaluation, Istituto Superiore di Sanità, Rome, Italy., Stoyanova-Beninska V; Committee of Orphan Medicinal Products, European Medicines Agency, Amsterdam, Netherlands.; College ter Beoordeling van Geneesmiddelen, Utrecht, Netherlands.
Jazyk:	angličtina
Zdroj:	Frontiers in medicine [Front Med (Lausanne)] 2021 Aug 27; Vol. 8, pp. 744625. Date of Electronic Publication: 2021 Aug 27 (Print Publication: 2021).
DOI:	10.3389/fmed.2021.744625
Abstrakt:	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. Competing Interests: 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. (Copyright © 2021 Sheean, Naumann-Winter, Capovilla, Kalland, Malikova, Mariz, Matusevicius, Nistico, Schwarzer-Daum, Tsigkos, Tzogani, Larsson, Magrelli and Stoyanova-Beninska.)
Databáze:	MEDLINE
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