Autor: |
Jasmin Barman-Aksözen, Mattia Andreoletti, Alessandro Blasimme |
Jazyk: |
angličtina |
Rok vydání: |
2023 |
Předmět: |
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Zdroj: |
Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-3 (2023) |
Druh dokumentu: |
article |
ISSN: |
1750-1172 |
DOI: |
10.1186/s13023-023-02941-w |
Popis: |
Abstract 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. |
Databáze: |
Directory of Open Access Journals |
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