Sistema de Salut de Catalunya
Institut Català de la Salut
[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
Vall d'Hebron Barcelona Hospital Campus
2025-04-25T10:12:32Z
2025-04-25T10:12:32Z
2025
Activated phosphoinositide 3-kinase delta syndrome; Decision-making; Ultra-rare disease
Síndrome de fosfoinositida 3-quinasa delta activada; Toma de decisiones; Enfermedad ultra rara
Síndrome de fosfoinosítida 3-quinasa delta activada; Presa de decisions; Malaltia ultra rara
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.
This study was sponsored by Pharming Technologies B.V., Leiden, The Netherlands.
Article
Published version
English
Avaluació de resultats (Assistència sanitària); Síndromes de deficiència immunitària - Tractament; Inhibidors enzimàtics - Ús terapèutic; CHEMICALS AND DRUGS::Enzymes and Coenzymes::Enzymes::Transferases::Phosphotransferases::Phosphotransferases (Alcohol Group Acceptor)::Phosphatidylinositol 3-Kinases::Phosphatidylinositol 3-Kinase; Other subheadings::Other subheadings::Other subheadings::/antagonists & inhibitors; DISEASES::Immune System Diseases::Immunologic Deficiency Syndromes; Other subheadings::Other subheadings::Other subheadings::/drug therapy; ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Diagnosis::Prognosis::Treatment Outcome; COMPUESTOS QUÍMICOS Y DROGAS::enzimas y coenzimas::enzimas::transferasas::fosfotransferasas::fosfotransferasas (grupo alcohol aceptor)::fosfatidil inositol 3 cinasas::fosfatidilinositol 3-cinasa; Otros calificadores::Otros calificadores::Otros calificadores::/antagonistas & inhibidores; ENFERMEDADES::enfermedades del sistema inmune::síndromes de inmunodeficiencia; Otros calificadores::Otros calificadores::Otros calificadores::/farmacoterapia; TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::diagnóstico::pronóstico::resultado del tratamiento
AboutScienceSrl
Global & Regional Health Technology Assessment;12(1)
https://doi.org/10.33393/grhta.2025.3199
Attribution-NonCommercial 4.0 International
http://creativecommons.org/licenses/by-nc/4.0/
Articles científics - HVH [3439]
