Dersimelagon in Erythropoietic Protoporphyrias

To the Editor: The International Porphyria Patient Network welcomes additional treatments for the erythropoietic protoporphyrias, such as dersimelagon, which was described by Balwani et al. (April 13 issue). 1 New drugs could help to address the needs of underserved subgroups such as children and may provide patients and health care professionals with treatment choices. The risks and benefits associated with various treatments are best assessed by applying comparable outcome measures, such as the same validated quality-of-life instruments and efficacy end points. The phase 2 trial of dersimelagon conducted by Balwani et al. missed this opportunity because its outcome measures differed from . . .