Assessing the value contribution of bimekizumab for the treatment of moderate-to-severe psoriasis using a multidisciplinary reflective multi-criteria decision analysis

Zozaya, Néboa; Abdalla, Fernando; Zamora, Santiago Alfonso; Filgueiras, Jesús Balea; Carrascosa, José Manuel; Sánchez, Olga Delgado; Sinisterra, Francisco Dolz; García-Ruiz, Antonio; Pinto, Pedro Herranz; Manfredi, Antonio; Olmos, José Martínez; Luna, Paloma Morales de los Ríos; Puig, L.; Ros, S.; Vega-Hidalgo, Álvaro

doi:10.1080/14737167.2022.2063842

Cited by 8 publications

(7 citation statements)

References 39 publications

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“…Several studies have been published regarding the use of reflective MCDA to assess the VC of drugs in the management of hematological and non-hematological diseases 18,20,21,37,38 . Olipudase alfa, a new drug for the management of the non-CNS manifestations of ASMD, has one of the highest VC s found in the literature.…”

Section: Discussionmentioning

confidence: 99%

Value contribution of olipudase alfa therapy for the management of the non-CNS manifestations of acid sphingomyelinase deficiency by multi-criteria decision analysis

Villarubia,

Reyes-Abad,

González-Meneses

et al. 2023

SANGREE

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Introduction:Olipudase alfa, an enzyme replacement therapy, is the first treatment approved for acid sphingomyelinase deficiency (ASMD), a rare lysosomal storage disease. Objective: To determine the value contribution of olipudase alfa to treat non-central nervous system (CNS) manifestations of ASMD using MCDA methodology. Methods: A literature review was conducted to populate an adapted MCDA framework for orphan-drug evaluation. A multidisciplinary panel of 8 experts were trained in MCDA methodology and scored the evidence matrix. Scoring consistency was explored through a test-retest reliability analysis. Results: ASMD was considered a severe disease (mean, 4.1 ± 0.6) with significant unmet needs (4.9 ± 0.4). Experts perceived that olipudase alfa may improve efficacy/effectiveness (4.4 ± 0.7), and has a favorable safety profile (3.0 ± 2.6). Additionally, its therapeutic benefit was considered relevant (4.3 ± 0.7) and patient-reported outcomes (PROs) (2.4 ± 1.8) were rated as moderate. Lower medical costs (2.6 ± 1.3) and indirect cost savings (2.9 ± 1.1) would be expected. Evidence presented was considered adequate (3.9 ± 1.0). The test-retest analysis indicated consistency in the scoring of almost all criteria. Lower correlation scores were observed in criteria with more dispersion as expert's scoring perception was addressed during the discussion session. Conclusion: Olipudase alfa may provide a high-value intervention to treat non-CNS manifestations of ASMD.

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Section: Discussionmentioning

confidence: 99%

Value contribution of olipudase alfa therapy for the management of the non-CNS manifestations of acid sphingomyelinase deficiency by multi-criteria decision analysis

Villarubia,

Reyes-Abad,

González-Meneses

et al. 2023

SANGREE

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“…However, the MCDA methodology can be particularly useful as a complement to this approach, as it consists of a structured (stepwise validated methodology), multidimensional (participation of experts from different professional fields), transparent (criteria, weights and scores are explicit) and systematic (replicable) approach, which incorporates several criteria and their individual value contribution to the decision or resource allocation problem [36]. Its popularity has been evident in the healthcare field in recent years, both nationally and internationally [36][37][38][39], in various areas, such as oncology [40][41][42], rare diseases [43][44][45], dermatology [46][47][48], and in the field of decisions related to the implementation of vaccines [19,20,22,[49][50][51][52].…”

Section: Discussionmentioning

confidence: 99%

Evaluation of Antibody-based Preventive Alternatives for Respiratory Syncytial Virus: A Novel Multi-Criteria Decision Analysis Framework and Assessment of Nirsevimab in Spain

Mestre-Ferrándiz,

Rivero,

Orrico-Sánchez

et al. 2023

Preprint

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Background Respiratory syncytial virus (RSV) is a highly infectious disease that poses a significant clinical and medical burden, as well as social disruption and economic costs, recognized by the World Health Organization as a public health issue. After several failed attempts to find preventive candidates (compounds, products, including vaccines), new alternatives might be available, one being nirsevimab, the first and only option approved for RSV prevention in neonates and infants during their first RSV season. The objective of this study was to develop a novel multi-criteria decision analysis (MCDA) framework for RSV antibody-based preventive alternatives and to use it to assess the value of nirsevimab vs. placebo as a systematic immunization approach to prevent RSV in neonates and infants during their first RSV season in Spain. Methods Based on a pre-established model called Vaccinex, an ad-hoc MCDA framework was created to reflect relevant attributes for the assessment of current and future antibody-based preventive measures for RSV. The estimated value of nirsevimab was obtained by means of an additive linear model combining weights and scores assigned by a multidisciplinary committee of 9 experts. A Re-test and three sensitivity analyses were conducted. Results Nirsevimab was evaluated through a novel framework with 26 criteria by the committee as a measure that adds value (positive final estimated value: 0.56 ± 0.11) to the current RSV scenario in Spain, by providing a high efficacy for prevention of neonates and infants. In addition, its implementation might generate cost savings in hospitalizations and to the healthcare system and increase the level of public health awareness among the general population, while reducing health inequities. Conclusions Under a methodology with increasing use in the health field, nirsevimab has been evaluated as a measure which adds value for RSV prevention in neonates and infants during their first RSV season in Spain.

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“…Son varios los artículos publicados sobre el uso del MCDA reflexivo para valorar la contribución de valor de los fármacos en el manejo de enfermedades hematológicas y no hematológicas 18,20,21,37,38 . La olipudasa alfa, un nuevo fármaco para el tratamiento de las manifestaciones no relacionadas con el SNC del ASMD ha obtenido una de las contribución de valor más altas en la literatura médica.…”

Section: Discussionunclassified

Contribución de valor de olipudase alfa en el tratamiento de las manifestaciones no relacionadas con el SNC del déficit de esfingomielinasa ácida mediante análisis de decisión multicriterio

Villarubia,

Reyes-Abad,

González-Meneses

et al. 2023

SANGRE

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Introducción: Olipudasa alfa es el primer tratamiento indicado para el déficit de esfingomielinasa ácida (ASMD), una enfermedad rara lisosomal. Objetivo: Determinar el valor de olipudasa alfa en el tratamiento de las manifestaciones no relacionadas con el sistema nervioso central (SNC) del ASMD utilizando la metodología MCDA. Metodología: Se realiza una revisión de la literatura para poblar un marco MCDA adaptado para medicamentos huérfanos. Un panel multidisciplinario de 8 expertos puntuó la matriz de evidencia. Se exploró la consistencia de la puntuación mediante un análisis test-retest. Resultados: El ASMD se consideró una enfermedad grave (media de 4.1 ± 0.6), con importantes necesidades no cubiertas (4.9 ± 0.4). Los expertos percibieron una mejora en eficacia/efectividad (4.4 ± 0.7) con un perfil de seguridad favorable (3.0 ± 2.6). Además, su beneficio terapéutico se consideró relevante (4.3 ± 0.7) y los resultados reportados por los pacientes (PROs) (2.4 ± 1.8) se calificaron como moderados. Se consideró que olipudasa alfa produciría un ahorro de costes médicos (2.6 ± 1.3) y de costes indirectos (2.9 ± 1.1). La evidencia presentada se consideró adecuada (3.9 ± 1.0). El análisis test-retest indicó consistencia en la puntuación en casi todos los criterios, con correlaciones más bajas en los criterios con mayor dispersión. Conclusión: Olipudasa alfa proporciona un alto valor al tratamiento de las manifestaciones no relacionadas con el SNC del ASMD.Palabras clave: Evaluación de medicamentos. Déficit de Esfingomielinasa ácida. Olipudasa alfa. Análisis de decisión multicriterio (MCDA). Enfermedades raras.

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Assessing the value contribution of bimekizumab for the treatment of moderate-to-severe psoriasis using a multidisciplinary reflective multi-criteria decision analysis

Cited by 8 publications

References 39 publications

Value contribution of olipudase alfa therapy for the management of the non-CNS manifestations of acid sphingomyelinase deficiency by multi-criteria decision analysis

Value contribution of olipudase alfa therapy for the management of the non-CNS manifestations of acid sphingomyelinase deficiency by multi-criteria decision analysis

Evaluation of Antibody-based Preventive Alternatives for Respiratory Syncytial Virus: A Novel Multi-Criteria Decision Analysis Framework and Assessment of Nirsevimab in Spain

Contribución de valor de olipudase alfa en el tratamiento de las manifestaciones no relacionadas con el SNC del déficit de esfingomielinasa ácida mediante análisis de decisión multicriterio

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