Current trials in erythropoietic protoporphyria: are placebo controls ethical?
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...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
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BMC
2023-10-01
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| Series: | Orphanet Journal of Rare Diseases |
| Online Access: | https://doi.org/10.1186/s13023-023-02941-w |
| _version_ | 1827708417466171392 |
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| author | Jasmin Barman-Aksözen Mattia Andreoletti Alessandro Blasimme |
| author_facet | Jasmin Barman-Aksözen Mattia Andreoletti Alessandro Blasimme |
| author_sort | Jasmin Barman-Aksözen |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-03-10T17:03:21Z |
| format | Article |
| id | doaj.art-de6b9cb2c3a649a19af49339ecefe6d4 |
| institution | Directory Open Access Journal |
| issn | 1750-1172 |
| language | English |
| last_indexed | 2024-03-10T17:03:21Z |
| publishDate | 2023-10-01 |
| publisher | BMC |
| record_format | Article |
| series | Orphanet Journal of Rare Diseases |
| spelling | doaj.art-de6b9cb2c3a649a19af49339ecefe6d42023-11-20T10:54:13ZengBMCOrphanet Journal of Rare Diseases1750-11722023-10-011811310.1186/s13023-023-02941-wCurrent trials in erythropoietic protoporphyria: are placebo controls ethical?Jasmin Barman-Aksözen0Mattia Andreoletti1Alessandro Blasimme2International Porphyria Patient NetworkDepartment of Health Sciences and Technology, ETH ZurichDepartment of Health Sciences and Technology, ETH ZurichAbstract 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.https://doi.org/10.1186/s13023-023-02941-w |
| spellingShingle | Jasmin Barman-Aksözen Mattia Andreoletti Alessandro Blasimme Current trials in erythropoietic protoporphyria: are placebo controls ethical? Orphanet Journal of Rare Diseases |
| 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 |
| url | https://doi.org/10.1186/s13023-023-02941-w |
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