Abstract
INTRODUCTION: Generalized myasthenia gravis (gMG) is a rare, chronic autoimmune neuromuscular disorder that currently lacks a curative treatment. Efgartigimod alfa is the first human IgG1 Fc fragment approved in Spain for the management of this condition. This study aims to evaluate the value of efgartigimod for treating gMG with anti-acetylcholine receptor (gMG AChR+) antibodies compared with ravulizumab, zilucoplan, and rozanolixizumab using a multi-criteria decision analysis (MCDA) framework. METHODS: A multidisciplinary group of eight experts evaluated the value of efgartigimod against ravulizumab, zilucoplan, and rozanolixizumab. The MCDA framework adapted for evaluating orphan drugs (ODs) in Spain, comprising nine quantitative and three qualitative criteria, was used. RESULTS: gMG AChR+ has been recognised as a severe and debilitating condition with considerable unmet needs. Efgartigimod achieved favourable average scores compared with ravulizumab, zilucoplan, and rozanolixizumab across all comparative parameters, including efficacy, safety, and patient-reported outcomes (PROs), potentially resulting in cost savings. Efgartigimod was deemed to have a significant therapeutic impact, with supporting data considered high quality. Efgartigimod alfa showed a higher overall value contribution than the three comparators, with the difference being most notable for ravulizumab. Furthermore, it was concluded that efgartigimod aligns well with the specific priorities, objectives, and capacities of the National Healthcare System (NHS). CONCLUSION: Efgartigimod has been recognised as a valuable option for gMG AChR+ treatment in Spain by a multidisciplinary panel of experts through the application of MCDA, receiving higher scores compared with ravulizumab, zilucoplan, and rozanolixizumab.