Current trials in erythropoietic protoporphyria: are placebo controls ethical?

Jasmin Barman-Aksözen; Mattia Andreoletti; Alessandro Blasimme

doi:10.1186/s13023-023-02941-w

Current trials in erythropoietic protoporphyria: are placebo controls ethical?

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

Authors

Jasmin Barman-Aksözen¹, Mattia Andreoletti², Alessandro Blasimme³

Affiliations

¹ International Porphyria Patient Network, Hegarstrasse 3, 8032, Zurich, Switzerland.
² Department of Health Sciences and Technology, ETH Zurich, Hottingerstrasse 10, 8092, Zurich, Switzerland.
³ Department of Health Sciences and Technology, ETH Zurich, Hottingerstrasse 10, 8092, Zurich, Switzerland. alessandro.blasimme@hest.ethz.ch.

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.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Europe
Humans
Protoporphyria, Erythropoietic* / drug therapy
Randomized Controlled Trials as Topic* / ethics
United States