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.
Keywords: Generalized myasthenia gravis with acetylcholine receptor antibody, gMG AChR+, Multi-criteria decision analysis, MCDA, Value assessment, Drugs evaluation, Rare disease, Orphan drug, HTA, Health technology assessment
Key Summary Points
| Efgartigimod alfa (Vyvgart®) is the first human IgG1 Fc fragment that inhibits the neonatal Fc receptor (FcRn), lowering IgG levels while preserving other immunoglobulins or albumin |
| This is the first study to apply multi-criteria decision analysis (MCDA) to assess the value contribution of a treatment in generalized myasthenia gravis (gMG) that includes all the most recent therapeutic alternatives |
| MCDA is a decision-support tool that provides a structured, reproducible, and transparent approach that allows the value of a drug to be evaluated holistically. Beyond the classical criteria of efficacy, safety, and costs, it addresses disease-related, treatment-specific, and contextual dimensions |
| This article shows the overall value contribution of efgartigimod compared with the three selected comparators was positive, providing nearly twice the value versus ravulizumab than for the other alternatives |
Introduction
Myasthenia gravis (MG) is a rare, chronic, autoimmune IgG-mediated disorder. The pathogenic IgG autoantibodies target critical proteins located on the postsynaptic membrane at the neuromuscular junction, leading to impaired neuromuscular transmission and subsequent muscle weakness [1–11]. MG is a heterogeneous condition with a fluctuating course and unpredictable progression, potentially leading to life-threatening myasthenic crises [12–14]. In addition to its physical impact, MG is a highly debilitating disease [15] and exerts a substantial psychological impact [16], placing a significant burden on patients, their families, and caregivers [10].
Although most patients initially present with ocular symptoms, > 80% progress to generalized myasthenia gravis (gMG) within 2 years [7, 12, 17]. Notably, 85% of gMG cases are associated with autoantibodies against acetylcholine receptors (anti-AChR), defining the gMG AChR+ subtype [2–5, 9, 10, 14, 18, 19]. The anti-acetylcholine receptor (AChR) autoantibodies exert their effects by blocking the acetylcholine binding on the AChR, inducing crosslinking and internalisation of AChR and activating the complement via these autoantibodies [20].
Currently, there is no curative treatment for gMG. The primary therapeutic goal is to achieve remission or minimal symptom expression (MSE). Management focuses on symptomatic relief using acetylcholinesterase inhibitors (AChEIs), chronic immunosuppressive therapy (corticosteroids and non-steroidal immunosuppressants), surgical options (thymectomy), and, for refractory patients, other options such as rituximab (not indicated for gMG) and eculizumab (indicated for the treatment of gMG but not reimbursed in Spain) may be used. Treatment is individualised, and in cases of severe weakness, intravenous immunoglobulins [IVIg] and therapeutic plasma exchange [PLEX]) are recommended [6, 11, 21]. However, 10–15% of patients remain refractory to available treatment [3, 4, 14, 17, 22, 23].
Efgartigimod alfa (Vyvgart®) is the first human IgG1 Fc fragment that inhibits the neonatal Fc receptor (FcRn), lowering IgG levels while preserving other immunoglobulins or albumin [24]. In Spain, it is indicated as an add-on to standard therapy for the treatment of adult patients with gMG AChR+ [25].
Multi-criteria decision analysis (MCDA) is a decision-support tool that provides a structured, reproducible, and transparent approach that allows the value of a drug to be evaluated holistically. Beyond the classical criteria of efficacy, safety, and costs, it addresses disease-related, treatment-specific, and contextual dimensions [26–31].
Recognised by healthcare decision-makers, MCDA has been increasingly used in the evaluation of new orphan drugs (ODs) and is considered a versatile method for informing value-based decisions [32–36]. The challenges in health technology assessments for ODs make traditional evaluation difficult [28, 37], but MCDA offers a more comprehensive approach by including multiple criteria and stakeholder perspectives.
The objective of this study was to assess the value of efgartigimod for the treatment of gMG AChR+ compared with ravulizumab, zilucoplan, and rozanolixizumab using MCDA methodology from the perspective of a Spanish multidisciplinary panel.
Methods
Study Design and MCDA Methodology
The study was conducted from January to April 2025 and adhered to the best practices in MCDA methodology [38]. It included a literature review, development of evidence matrices, criteria scoring, value contribution analysis, and discussion of the results.
The Evidence and Value: Impact on DEcisionMaking (EVIDEM) framework, specifically adapted for the evaluation of ODs [30, 38–41], was used, which included 12 criteria grouped into 3 dimensions (Table 1) [40].
Table 1.
Multicriteria decision analysis framework adapted to evaluate ODs from the EVIDEM framework [40]
| Quantitative criteria |
|---|
| Disease-related criteria |
| Disease severity |
| Unmet needs |
| Treatment-related criteria |
| Efficacy/effectiveness |
| Safety/tolerability |
| Patient-reported outcomes (PROs) |
| Therapeutic impact |
| Other medical costs |
| Non-medical/indirect costs |
| Quality of evidence or level of evidence and level of recommendation |
| Contextual criteria |
|---|
| Population access priorities |
| Common goals and specific interests |
| System capacity and appropriate use of the intervention |
Comparators
The value contribution of efgartigimod in treating gMG AChR+ was assessed against three recently approved and reimbursed therapeutic alternatives in Europe and Spain for the treatment of gMG AChR+: ravulizumab (IgG2/4k monoclonal antibody, C5 inhibitor), zilucoplan (macrocyclic peptide, C5 inhibitor), and rozanolixizumab (IgG4 monoclonal antibody, FcRn inhibitor).
Literature Review and Evidence Matrices Development
A comprehensive literature review was conducted in January 2025 following a predefined protocol to gather relevant evidence on the disease, its management, and efgartigimod and selected comparator treatments. The literature review was aligned with the criteria included in the MCDA EVIDEM framework adapted for ODs [40].
The review encompassed biomedical data sources and grey literature, such as PubMed/Medline, Google Scholar, the European Medicines Agency website, websites of relevant scientific societies, patient association websites, and international clinical trials registries (clinicaltrials.gov). The search was limited to articles published within the last 10 years.
Articles were initially screened by title and abstract, excluding those that did not align with the research objective or eligibility criteria. Duplicates, articles in languages other than Spanish or English, and animal research studies were also excluded. Eligible articles underwent full-text assessment. Data collected from the literature review were used to develop three evidence matrices [40].
Multidisciplinary Expert Panel Design and Training
A multidisciplinary panel of eight experts was established, including four neurologists experienced in patients with gMG patient care and treatment, three hospital pharmacists, and one regional former evaluator representing six regions in Spain (Andalusia, Aragon, Catalonia, Galicia, Madrid, and Valencia).
All experts participated in a 1-h training session on MCDA methodology conducted in February 2025. Following the training, experts scored the matrices offline and included the rationale behind their scores.
Criteria Weighting
The relative weights of the quantitative criteria were derived from a previous national study involving 98 stakeholders in drug evaluation committees at national and regional levels across Spain [42].
Criteria Scoring
According to the adapted MCDA framework [40], non-comparative criteria were scored from 0 to + 5, and comparative criteria from – 5 to + 5, with + 5 indicating the most favourable outcome in both cases. Contextual criteria were assessed qualitatively as having a positive, neutral, or negative impact on healthcare systems [31, 39].
Data Collection and Analysis
Data from the evidence matrices were compiled into a centralised database and analysed using Microsoft Excel, specifically customised for MCDA [34].
Quantitative criteria scores were analysed using descriptive statistics, including mean, median, range (minimum–maximum), and standard deviations (SD). In the quantitative comparative criteria—efficacy/effectiveness, safety/tolerability, patient-reported outcomes (PROs), medical costs, and indirect costs—efgartigimod was evaluated against the three previously mentioned therapeutic alternatives.
For each criterion, the value contribution (VCx) was calculated as the product of its weight (Wx) and standardised scores (Sx). The overall value contribution, ranging from – 1 to 1, was obtained by summing the individual value contributions of all quantitative criteria. Scores > 0 reflect a favourable perception relative to the comparator [43]:
Contextual criteria were assessed by experts as having a positive, neutral, or negative impact and subsequently converted into scores of 1, 0, and − 1, respectively. These scores were then expressed as percentages reflecting the proportion of experts favouring each option.
Reflective Discussion
An online MCDA workshop was held in March 2025, following the scoring of the three matrices, to facilitate reflective discussion of each criterion. The 3-h session included an overview of the study’s objectives and methodology, followed by a criterion-by-criterion presentation of the results. This facilitated in-depth analysis and discussion of the rationale behind the experts’ evaluations. Experts were given the opportunity to revise their scores based on insights gained during the discussion.
Ethics Statement
The authors declare that no experiments involving humans or animals were conducted for this research, and no patient data were included in this article. Full consent to participate in the study and to publish the findings was obtained from all contributors.
Results
Quantitative Criteria
Disease-Related Criteria
gMG AChR+ was perceived as a severe, highly disabling, and heterogeneous condition with a fluctuating nature, associated with significant morbidity and potentially life-threatening exacerbations, such as myasthenic crises (mean ± SD: 3.9 ± 0.8). Some experts did not consider this disease to be currently fatal, owing to available therapeutic options, and found that mortality rates are higher among patients > 70 years old and in cases involving delayed diagnosis.
Although experts considered symptomatic treatments to be often effective, there are still considerable “unmet needs” (mean ± SD: 4.6 ± 0.5). Participants highlighted the adverse effects (AEs) associated with current therapies and noted that a substantial proportion of patients do not achieve MSE (Fig. 1).
Fig. 1.
Quantitative comparative criteria scores of efgartigimod compared with ravulizumab in the treatment of gMG AChR+
Treatment-Related Criteria
Experts agreed that ravulizumab, zilucoplan, and rozanolixizumab provide added value in the treatment of gMG AChR+, with efgartigimod achieving higher average positive scores in all comparative criteria versus ravulizumab, zilucoplan, and ravulizumab (Figs. 1, 2, 3).
Fig. 2.
Quantitative comparative criteria scores of efgartigimod compared with zilucoplan in the treatment of gMG AChR+
Fig. 3.
Quantitative comparative criteria scores of efgartigimod compared with rozanolixizumab in the treatment of gMG AChR+
Efficacy/Effectiveness
Compared with ravulizumab (mean ± SD: 2.3 ± 1.3), efgartigimod was perceived to achieve more consistent and clinically significant results in both scales, Myasthenia Gravis Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG), within a shorter timeframe—4 compared with 26 (mean ± SD: 2.3 ± 1.3) weeks. Additionally, efgartigimod met the study’s objectives, achieving a > 35% treatment difference in patient’s AChR+. In contrast, ravulizumab did not meet the objective (− 1.9) with a least squares mean change in total MG-ADL of − 1.6 after 26 weeks.
Although zilucoplan demonstrated significant improvements in the MG-ADL and QMG scales, in general, experts considered the efficacy profile of efgartigimod to be better (mean ± SD: 1.9 ± 1.2) as a higher proportion of patients treated with efgartigimod achieved MSE (40% versus 14%) and a smaller proportion required rescue therapy (1.2% versus 5%). Furthermore, the response to efgartigimod was assessed at week 4, whereas the evaluation of zilucoplan was conducted at week 12, leading experts to conclude that efgartigimod may have a more rapid onset of action.
Compared with rozanolixizumab (mean ± SD: 1.1 ± 1.0), experts noted that the treatments share the same mechanism of action, suggesting significant differences in outcomes are unlikely. Available evidence showed similar responder rates in the general population, 67–71% for efgartigimod (week 4) and 72–69% for rozanolixizumab (day 43). However, experts highlighted that rozanolixizumab did not demonstrate the same rapid onset or symptom improvement as efgartigimod.
Safety/Tolerability
Efgartigimod’s safety profile was perceived as positive compared with the three alternatives (Figs. 1, 2, 3).
Compared with ravulizumab (mean ± SD: 2.1 ± 1.0), experts emphasised that clinical trials of efgartigimod reported a lower incidence of AEs, with no reported fatalities and no need for meningococcal vaccination. Nonetheless, they noted that long-term safety data are limited for both treatments.
In the comparative versus zilucoplan (mean ± SD: 1.5 ± 1.3), experts reported that AEs were less frequent in the efgartigimod treatment group than in the placebo group, with most being mild. Conversely, the zilucoplan study indicated a higher incidence of AEs in the treatment group, with some cases of exacerbation of MG and with 12% experiencing severe adverse events (SAEs). Experts also highlighted that patients treated with zilucoplan require prior meningococcal vaccine, whereas this is not required for efgartigimod. On the other hand, experts noted that mild-to-moderate infections were more common among patients receiving efgartigimod, with an incidence rate of up to 46%, compared with 37% in the placebo group. Furthermore, in the efgartigimod and zilucoplan Open-Label Extension (OLE) studies, discontinuation rates were similar, at 8.3% for efgartigimod and 9% for zilucoplan.
Compared with rozanolixizumab (mean ± SD: 1.3 ± 1.5), experts noted that the proportion of patients developing anti-drug and neutralising antibodies was lower in the efgartigimod trial. Although both treatments share the same mechanism of action, do not require meningococcal vaccination, and present similar AEs, experts noted that infections—particularly respiratory and urinary—were more frequent in patients treated with efgartigimod. However, headaches and diarrhoea were more common in patients treated with rozanolixizumab.
As with the efficacy criteria, some experts highlighted the limitation of comparing treatments that differ in molecular characteristics and are evaluated across diverse patient populations.
Patient-Reported Outcomes (PROs)
Regarding PROs, experts considered that efgartigimod provided a more favourable profile than ravulizumab (mean ± SD: 2.0 ± 1.3) (Fig. 1), primarily due to its versatility, having two available routes of administration—intravenous (IV) and subcutaneous (SC)—whereas ravulizumab is only available for IV administration. However, some participants valued the ravulizumab fixed dosing schedule positively over the efgartigimod cyclical regimen.
Compared with zilucoplan, experts generally perceived both treatments as similar in terms of PROs (mean ± SD: 0.9 ± 1.4) (Fig. 2), although with a slight preference for efgartigimod due to its availability in both IV and SC formulations, whereas zilucoplan is limited to SC administration and requires daily dosing. Nevertheless, some experts favoured the fixed dosing regimen of zilucoplan over the cyclical regimen of efgartigimod.
Compared with rozanolixizumab (mean ± SD: 0.9 ± 1.5) (Fig. 3), differences were minimal, as both drugs share a similar mechanism of action and a cyclical dosing regimen. However, experts highlighted efgartigimod’s advantage in route-of-administration versatility, since rozanolixizumab can only be administered subcutaneously.
Other Medical and Non-medical/Indirect Costs
Although efgartigimod’s long-term direct and indirect cost savings was perceived as positive compared with ravulizumab (mean ± SD: 2.5 ± 1.7 and 1.4 ± 1.8, respectively), expert opinions tended to align more closely when comparing it with zilucoplan (mean ± SD: 1.6 ± 1.8, and 0.5 ± 1.1, respectively) and rozanolixizumab (mean ± SD: 1.5 ± 1.9 and 0.4 ± 1.1, respectively), as shown in Figs. 1, 2, and 3. Expert’s scoring was supported by efgartigimod’s favourable efficacy and safety profile, together with its potential to reduce hospitalizations, intensive care unit (ICU) admissions, and the need for concomitant treatments, such as corticosteroids, IVIg, or PLEX—features that some considered suggestive of a possible disease-modifying effect. However, some experts cautioned that current evidence remains insufficient to confirm this assumption.
Therapeutic Impact
Regarding the “therapeutic impact” criterion (mean ± SD: 3.9 ± 0.6), participants highlighted efgartigimod’s novel mechanism of action, rapid onset of action, maintained effect, consistent improvement across assessment scales, potential to reduce concomitant treatments, positive safety profile, and the availability of both IV and SC administration routes.
Quality of Evidence
The “quality of evidence” criterion was rated as highly relevant (mean ± SD: 4.3 ± 0.9), with experts acknowledging the well-designed and balanced Phase III trials, although they noted the short-term and surrogate nature of the endpoints analysed. Additionally, they emphasised that the OLE studies were solely concentrated on safety and highlighted the necessity for long-term data.
Value Contribution of Efgartigimod
The value contribution analysis showed that efgartigimod obtained a notable positive value contribution in the treatment of gMG and across all three comparisons: 0.22 versus ravulizumab, 0.14 versus zilucoplan, and 0.13 versus rozanolixizumab, with the criteria of “Efficacy/effectiveness”, “Safety/tolerability”, and “Other medical costs” contributing the most (Fig. 4).
Fig. 4.
By-criteria value contribution of efgartigimod compared with ravulizumab, zilucoplan, and rozanolixizumab
Contextual Criteria
The expert panel’s evaluation of contextual criteria was predominantly positive (Fig. 5). Eighty-eight percent of participants considered that efgartigimod would positively impact the criterion “population access priorities”, positioning it as an additional therapeutic option that could improve care and outcomes for patients. However, some experts argued that current national strategies remain largely theoretical and do not reflect the need for personalised, patient-centred care.
Fig. 5.
Value contribution of the qualitative/contextual criteria of efgartigimod in the treatment of gMG AChR+
All participants agreed on the beneficial influence of efgartigimod on “common goals and specific interests”, reflecting consensus among scientific societies, patient associations, and other stakeholders regarding the incorporation as a new treatment option for the treatment of gMG.
Regarding “system capacity and appropriate use of the intervention”, all participants rated the introduction of efgartigimod into their healthcare system as neutral or positive. While many agreed that the NHS has the infrastructure and trained professionals to support its use, other experts emphasised the need for specific training due to its cyclical regimen.
Discussion
To our knowledge, this is the first study to apply MCDA to assess the value contribution of a treatment in gMG that includes all the most recent therapeutic alternatives. Using the adapted EVIDEM framework [40] for OD evaluation, a multidisciplinary panel of experts assessed the value of efgartigimod compared with the three therapeutic alternatives—ravulizumab, zilucoplan, and rozanolixizumab—recently approved in Europe for the treatment of gMG AChR+ [44–46].
The overall value contribution of efgartigimod compared with the three selected comparators was positive, providing nearly twice the value versus ravulizumab than for the other alternatives. Importantly, across all the criteria considered to estimate the value contribution, efgartigimod consistently demonstrated a higher value than all treatment alternatives, underscoring its strong positioning within the treatment landscape.
After the MCDA methodology training session, participants individually scored each criterion of three evidence matrices, providing the rationale behind their scores. The reflective group discussion then enabled deeper analysis and refinement of perspectives, resulting in a more comprehensive evaluation of efgartigimod’s value in the treatment of gMG AChR+.
gMG AChR+ was considered a severe and heterogeneous condition with considerable morbidity and risk of life-threatening exacerbations. Conventional treatments can improve health outcomes but lack curative options, leaving important unmet needs, particularly concerning AEs and the limited proportion of patients achieving MSE. Efgartigimod was deemed to provide valuable benefits due to its innovative mechanism of action, rapid onset, maintained effects, safety profile, and flexible administration, supported by robust Phase III trial evidence, albeit with limitations related to surrogate short-term endpoints and insufficient long-term data, as per any product recently approved and commercialised.
Conversely, the comparative criteria—“efficacy/effectiveness”, “safety/tolerability”, “PROs”, “other medical costs”, and “non-medical/indirect costs”—contributed less to the overall scores. Experts agreed that all the alternatives offered benefits in managing the disease, with efgartigimod achieving moderately higher scores than the three alternatives, particularly compared with ravulizumab.
Experts assigned positive scores to efgartigimod’s efficacy, highlighting its rapid onset of action, the higher proportion of patients achieving MSE, and its more consistent clinical improvements on MG-ADL and QMG scales compared with its alternatives—particularly ravulizumab. While zilucoplan and rozanolixizumab were considered effective in terms of MG-ADL and QMG scores, efgartigimod’s MSE and ability to elicit meaningful responses by week 4 were highlighted as key differentiators.
Regarding safety, efgartigimod was associated with fewer SAEs than ravulizumab and zilucoplan; however, higher rates of infections were observed, which would require monitoring. Rozanolixizumab and efgartigimod were perceived to have similar safety profiles.
Efgartigimod was rated positively in terms of PROs, largely due to its dual administration route (IV and SC), which offers greater flexibility and convenience for the patients. While some experts valued the fixed dosing regimens of ravulizumab and zilucoplan positively for their predictability, efgartigimod’s versatility was seen as a meaningful advantage, particularly from a patient-centred perspective, for some experts.
Despite promising findings, experts highlighted certain limitations in the comparative analyses—such as the absence of direct head-to-head trials and the heterogeneity in study populations and outcome measures—which make it difficult to reach definitive conclusions. This partly accounts for the observed SD in expert ratings across all comparative criteria.
Additionally, participants anticipated potential long-term savings with efgartigimod treatment—particularly in direct medical costs and primarily when compared with ravulizumab—driven by improved disease control and reduced reliance on hospital-based care. Further research is needed to confirm this potential.
Experts reported no major barriers to the introduction of efgartigimod into the NHS.
MCDA is increasingly being used in the evaluation of ODs across various levels in Spain, including the national [34, 47], regional [39, 48] and hospital levels [40, 49]. Its application is being actively encouraged within the Orphar-SEFH Strategic Plan 2024–2027 [50].
One of the notable strengths of this study is the involvement of a multidisciplinary panel of experts, which substantially enhances the richness of the findings and the conclusions derived and resembles real-life settings from evaluation committees in Spain. Another strength is the use of relative weights for the study criteria, derived from a previous national study involving 98 stakeholders across Spain [42]; they were previously used in other published MCDA studies [51, 52], ensuring that quantitative criteria reflect the perspectives of a broad and representative sample of experts actively engaged in drug evaluation.
Nevertheless, the study is not without certain limitations: (1) The results depend somewhat on the composition of the expert panel, their value judgments, and their levels of experience. To mitigate potential bias arising from expertise, all participants received training in MCDA methodology prior to the individual scoring and discussion sessions. Additionally, the study participants included key decision-makers, and the group size was representative, often exceeding that of actual drug evaluation committees in Spain. This aligns with the sizes typically observed in other MCDA exercises [49, 51–54]. Data gaps were identified for some criteria, particularly direct costs for zilucoplan and rozanolixizumab and indirect costs for efgartigimod and the alternatives [3]. The criteria “pharmaceutical cost” and “opportunity cost and affordability” were excluded in accordance with ORPHAR-SEFH guidelines [40] because of the absence of pricing information, especially for rozanolixizumab, which was still undergoing the P&R process in Spain at the time of the study [4]. Differences in patient populations across trials limited direct comparability [5, 6]. Due to national compliance rules and limitations, patients’ perspectives were not included in the study, which may have limited the assessment of certain criteria, such as PROs.
Future studies should consider including patient representatives and expanding the expert panel to other hospitals and regions to capture a broader range of perspectives.
Conclusion
To the best of the authors’ knowledge, this study is the first to use MCDA methodology to assess the value contribution of a new treatment option for the treatment of gMG AChR+, including all the most novel therapeutic alternatives. A multidisciplinary panel of stakeholders in Spain agreed that the recently approved alternatives for the treatment of gMG AChR+ offer significant therapeutic benefits over traditional symptomatic treatments. In general, efgartigimod was perceived to provide a positive value contribution in all criteria compared with its alternatives in terms of efficacy, safety, PROs, and costs.
The use of reflective MCDA methodology not only facilitates a comprehensive understanding of the perceived value of a new treatment by considering a wide array of value attributes compared with existing treatment options but also supports informed decision-making regarding the selection of the most suitable therapy for these patients.
Author Contribution
Celia Martín Machín and Alicia Gil: conceptualization and formal analysis. Celia Martín Machín and Alicia Gil: data curation, methodology, supervision, writing–original draft. Elena Cortés-Vicente, Antonio Guerrero, Carmina Díaz, Eva Martínez, Francisco J. Toja-Camba, María R. Abad, José M. Serra, Jose L. Trillo, Celia Martín Machín and Alicia Gil: input of the content, writing–reviewing, editing and validation.
Funding
The author(s) disclosed receipt of the following financial support for the research, authorship, and/or publication of this article: This work and the journal’s Rapid Service fee have been funded by argenx.
Data Availability
The data that support the findings of this study are available from the corresponding author upon reasonable request.
Declarations
Conflict of Interest
María R. Abad: I declare that I have received honoraria for the development of this project from argenx and speakers’ fees from UCB Pharma, Johnson & Johnson, Vertex, Organon, Biogen, CINFA, Astra Zeneca, GSK, and Sanofi Aventis. Elena Cortés-Vicente: I declare that I have received honoraria for speaking engagements and consultancy activities from argenx, UCB, Alexion, and Janssen. Francisco J. Toja-Camba: I declare that I have received honoraria for the development of this project from argenx. Carmina Díaz: I declare that I have received honoraria for the development of this project from argenx. Included received speakers fees from ALEXION and UCB. Eva Martínez: I declare that I have received honoraria for the development of this project from argenx. José M. Serra: I declare that I have received honoraria for the development of this project from argenx. Jose L. Trillo: I declare that I have received honoraria for the development of this project from argenx. Antonio Guerrero: I declare that I have received honoraria for the development of this project from argenx and received speaker’s fees from ALEXION and UCB.
Ethical Approval
The authors declare that no experiments on humans or animals have been carried out for this research. The authors declare that no patient data appear in this article. Full consent to participate in the study and to publish the findings was obtained from all contributors.
Footnotes
This article was revised due to update in conflict of interest section.
Publisher's Note
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Change history
3/12/2026
A Correction to this paper has been published: 10.1007/s12325-026-03532-4
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Associated Data
This section collects any data citations, data availability statements, or supplementary materials included in this article.
Data Availability Statement
The data that support the findings of this study are available from the corresponding author upon reasonable request.





