2026-04-17T04:22:43Zhttps://recercat.cat/oai/request

oai:recercat.cat:11351/129922025-10-24T10:28:05Zcom_2072_378070com_2072_378040col_2072_378092

00925njm 22002777a 4500 dc Abad, Reyes author Alerany Pardo, Carme author Gonzalez-Granado, Luis Ignacio author Neth, Olaf author PAYARES HERRERA, MARIA CONCEPCION author Rodríguez-Gallego, Carlos author 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. http://hdl.handle.net/11351/12992 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 Value contribution of leniolisib in the Treatment of Activated PI3Kδ syndrome (APDS) in Spain using Multi-Criteria Decision Analysis (MCDA)