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. 2023 Oct 16;18(1):325.
doi: 10.1186/s13023-023-02941-w.

Current trials in erythropoietic protoporphyria: are placebo controls ethical?

Jasmin Barman-Aksözen  1 , Mattia Andreoletti  2 , Alessandro Blasimme  3
Affiliations

Current trials in erythropoietic protoporphyria: are placebo controls ethical?

Jasmin Barman-Aksözen et al. Orphanet J Rare Dis. .

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.

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Conflict of interest statement

JB-A declares that the views presented in the manuscript are her own and that she has no competing interests to declare. AB and MA have no competing interests to declare.

References

    1. Todd DJ. Erythropoietic protoporphyria. Br J Dermatol. 1994;131(6):751–66. doi: 10.1111/j.1365-2133.1994.tb08577.x. - DOI - PubMed
    1. Langendonk JG, Balwani M, Anderson KE, et al. Afamelanotide for Erythropoietic Protoporphyria. N Engl J Med. 2015;373(1):48–59. doi: 10.1056/NEJMoa1411481. - DOI - PMC - PubMed
    1. European Medicinces Agency, Committee for Medicinal Products for Human Use (CHMP)23. (2014): European Public Assessment Report Scenesse ®,. October 2014. EMA/CHMP/709396/2014 Rev.1. https://www.ema.europa.eu/en/documents/assessment-report/scenesse-epar-p... (last accessed 16 July 2023).
    1. Evers AW, Colloca L, Blease C, Annoni M, Atlas LY, Benedetti F, Bingel U, Büchel C, Carvalho C, Colagiuri B, Crum AJ. Implications of placebo and nocebo effects for clinical practice: expert consensus. Psychother Psychosom. 2018;87(4):204–10. doi: 10.1159/000490354. - DOI - PMC - PubMed
    1. World Medical Association World Medical Association Declaration of Helsinki: ethical principles for medical research involving human subjects. JAMA. 2013;310(20):2191–4. doi: 10.1001/jama.2013.281053. - DOI - PubMed

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