Current trials in erythropoietic protoporphyria: are placebo controls ethical?

A new active substance called “dersimelagon” (MT-7117) is being tested as an alternative treatment option for Erythropoietic protoporphyria (EPP). At the moment, dersimelagon is being tested both in the US and in Europe in a phase III placebo-controlled RCT. However, given the availability of an alr...

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Autores principales: Barman-Aksözen, Jasmin, Andreoletti, Mattia, Blasimme, Alessandro
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Position Statement
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10580501/
https://www.ncbi.nlm.nih.gov/pubmed/37845740
http://dx.doi.org/10.1186/s13023-023-02941-w
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author Barman-Aksözen, Jasmin
Andreoletti, Mattia
Blasimme, Alessandro
author_facet Barman-Aksözen, Jasmin
Andreoletti, Mattia
Blasimme, Alessandro
author_sort Barman-Aksözen, Jasmin
collection PubMed
description A new active substance called “dersimelagon” (MT-7117) is being tested as an alternative treatment option for Erythropoietic protoporphyria (EPP). At the moment, dersimelagon is being tested both in the US and in Europe in a phase III placebo-controlled RCT. However, given the availability of an already approved treatment option for EPP the use of a placebo arm is questionable from an ethics point of view. We analyze the issue and suggest that a noninferiority active-control trial without placebo is an ethically and scientifically more valid design to test the efficacy of dersimelagon as well as other EPP treatments.
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spelling pubmed-105805012023-10-18 Current trials in erythropoietic protoporphyria: are placebo controls ethical? Barman-Aksözen, Jasmin Andreoletti, Mattia Blasimme, Alessandro Orphanet J Rare Dis Position Statement A new active substance called “dersimelagon” (MT-7117) is being tested as an alternative treatment option for Erythropoietic protoporphyria (EPP). At the moment, dersimelagon is being tested both in the US and in Europe in a phase III placebo-controlled RCT. However, given the availability of an already approved treatment option for EPP the use of a placebo arm is questionable from an ethics point of view. We analyze the issue and suggest that a noninferiority active-control trial without placebo is an ethically and scientifically more valid design to test the efficacy of dersimelagon as well as other EPP treatments. BioMed Central 2023-10-16 /pmc/articles/PMC10580501/ /pubmed/37845740 http://dx.doi.org/10.1186/s13023-023-02941-w Text en © The Author(s) 2023 https://creativecommons.org/licenses/by/4.0/Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) . The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/ (https://creativecommons.org/publicdomain/zero/1.0/) ) applies to the data made available in this article, unless otherwise stated in a credit line to the data.
spellingShingle Position Statement
Barman-Aksözen, Jasmin
Andreoletti, Mattia
Blasimme, Alessandro
Current trials in erythropoietic protoporphyria: are placebo controls ethical?
title Current trials in erythropoietic protoporphyria: are placebo controls ethical?
title_full Current trials in erythropoietic protoporphyria: are placebo controls ethical?
title_fullStr Current trials in erythropoietic protoporphyria: are placebo controls ethical?
title_full_unstemmed Current trials in erythropoietic protoporphyria: are placebo controls ethical?
title_short Current trials in erythropoietic protoporphyria: are placebo controls ethical?
title_sort current trials in erythropoietic protoporphyria: are placebo controls ethical?
topic Position Statement
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10580501/
https://www.ncbi.nlm.nih.gov/pubmed/37845740
http://dx.doi.org/10.1186/s13023-023-02941-w
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