Development and Content Validation of Novel Patient-Reported Outcome Measures to Assess Disease Severity and Change in Patients with Erythropoietic Protoporphyria: The EPP Impact Questionnaire (EPIQ).
Susan D MathiasLaurie BurkeHilary H ColwellGeorge MensingWill SavageHetanshi NaikPublished in: Patient related outcome measures (2024)
PRO measures are needed to assess important aspects of HRQoL and evaluate therapeutic response. These PRO measures are unique in assessing overall severity and change in EPP.