dc.contributor
Institut Català de la Salut
dc.contributor
[Reyes Abad M] Pharmacy Department, H.U. Miguel Servet, Zaragoza, Spain. [Alerany C] Servei de Farmàcia, Vall d’Hebron Hospital Universitari, Barcelona, Spain. [González LI] Immunodeficiencies Unit H.U. 12 de Octubre, Madrid, Spain. [Neth O] Paediatric Infectious Diseases, Rheumatology and Immunology Unit H.U. Virgen del Rocío, Seville, Spain. [Payares-Herrera C] Clinical Pharmacology Department H.U. Puerta del Hierro, Majadahonda (Madrid) –Spain. [Rodríguez-Gallego C] Department of Immunology, University Hospital of Gran Canaria Dr Negrin, Las Palmas de Gran Canaria, Spain. Department of Medical and Surgical Sciences, School of Medicine, University of Las Palmas de Gran Canaria, Las Palmas de Gran Canaria, Spain. Department of Clinical Sciences, University Fernando Pessoa Canarias, Las Palmas de Gran Canaria, Spain
dc.contributor
Vall d'Hebron Barcelona Hospital Campus
dc.contributor.author
Abad, Reyes
dc.contributor.author
Alerany Pardo, Carme
dc.contributor.author
Gonzalez-Granado, Luis Ignacio
dc.contributor.author
Neth, Olaf
dc.contributor.author
PAYARES HERRERA, MARIA CONCEPCION
dc.contributor.author
Rodríguez-Gallego, Carlos
dc.date.accessioned
2025-10-24T10:28:05Z
dc.date.available
2025-10-24T10:28:05Z
dc.date.issued
2025-04-25T10:12:32Z
dc.date.issued
2025-04-25T10:12:32Z
dc.identifier
Reyes Abad M, Alerany C, Gonzalez LI, Neth O, Payares-Herrera C, Rodríguez-Gallego C, et al. Value contribution of leniolisib in the Treatment of Activated PI3Kδ syndrome (APDS) in Spain using Multi-Criteria Decision Analysis (MCDA). Glob Reg Heal Technol Assess. 2025;12(1):9-15.
dc.identifier
http://hdl.handle.net/11351/12992
dc.identifier
10.33393/grhta.2025.3199
dc.identifier
001412560800001
dc.identifier.uri
https://hdl.handle.net/11351/12992
dc.description.abstract
Activated phosphoinositide 3-kinase delta syndrome; Decision-making; Ultra-rare disease
dc.description.abstract
Síndrome de fosfoinositida 3-quinasa delta activada; Toma de decisiones; Enfermedad ultra rara
dc.description.abstract
Síndrome de fosfoinosítida 3-quinasa delta activada; Presa de decisions; Malaltia ultra rara
dc.description.abstract
Background: Activated phosphoinositide 3-kinase (PI3K) δ Syndrome (APDS) is an ultra-rare, potentially life-threatening disease that lacks approved treatments in Spain. This study aimed to apply Multi-Criteria Decision Analysis (MCDA) to assess the value of the first pharmacological treatment for APDS in Spain.
Methods: A multidisciplinary group of 8 experts evaluated the selective PI3Kδ inhibitor leniolisib against Standard of Care (SoC). An MCDA framework tailored for Orphan Drugs (ODs), consisting of 5 comparative and 2 quantitative non-comparative criteria, was used. Re-scoring followed a group discussion.
Results: Leniolisib scored higher than SoC in all criteria, including efficacy and safety. It was deemed highly valuable as the first disease-modifying treatment, with a positive therapeutic impact and potential to improve patients’ quality of life. Additionally, leniolisib may lead to cost savings. The supporting data was considered of high quality.
Conclusion: Based on MCDA methodology and stakeholder experience in APDS management, leniolisib is seen as a value-added treatment option compared to SoC in Spain.
dc.description.abstract
This study was sponsored by Pharming Technologies B.V., Leiden, The Netherlands.
dc.format
application/pdf
dc.publisher
AboutScienceSrl
dc.relation
Global & Regional Health Technology Assessment;12(1)
dc.relation
https://doi.org/10.33393/grhta.2025.3199
dc.rights
Attribution-NonCommercial 4.0 International
dc.rights
http://creativecommons.org/licenses/by-nc/4.0/
dc.rights
info:eu-repo/semantics/openAccess
dc.subject
Avaluació de resultats (Assistència sanitària)
dc.subject
Síndromes de deficiència immunitària - Tractament
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Inhibidors enzimàtics - Ús terapèutic
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CHEMICALS AND DRUGS::Enzymes and Coenzymes::Enzymes::Transferases::Phosphotransferases::Phosphotransferases (Alcohol Group Acceptor)::Phosphatidylinositol 3-Kinases::Phosphatidylinositol 3-Kinase
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Other subheadings::Other subheadings::Other subheadings::/antagonists & inhibitors
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DISEASES::Immune System Diseases::Immunologic Deficiency Syndromes
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Other subheadings::Other subheadings::Other subheadings::/drug therapy
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ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Diagnosis::Prognosis::Treatment Outcome
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COMPUESTOS QUÍMICOS Y DROGAS::enzimas y coenzimas::enzimas::transferasas::fosfotransferasas::fosfotransferasas (grupo alcohol aceptor)::fosfatidil inositol 3 cinasas::fosfatidilinositol 3-cinasa
dc.subject
Otros calificadores::Otros calificadores::Otros calificadores::/antagonistas & inhibidores
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ENFERMEDADES::enfermedades del sistema inmune::síndromes de inmunodeficiencia
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Otros calificadores::Otros calificadores::Otros calificadores::/farmacoterapia
dc.subject
TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::diagnóstico::pronóstico::resultado del tratamiento
dc.title
Value contribution of leniolisib in the Treatment of Activated PI3Kδ syndrome (APDS) in Spain using Multi-Criteria Decision Analysis (MCDA)
dc.type
info:eu-repo/semantics/article
dc.type
info:eu-repo/semantics/publishedVersion
