Value contribution of leniolisib in the Treatment of Activated PI3Kδ syndrome (APDS) in Spain using Multi-Criteria Decision Analysis (MCDA)
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 AP...
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| Language: | English |
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2025-01-01
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| Series: | Global & Regional Health Technology Assessment |
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| Online Access: | https://journals.aboutscience.eu/index.php/grhta/article/view/3199 |
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| author | María Reyes Abad Carmen Alerany Luis Ignacio Gonzalez Olaf Neth Concepción Payares-Herrera Carlos Rodríguez-Gallego Jose Luis Trillo Kirsten H. Herrmann Raquel Figueiredo Alicia Gil |
| author_facet | María Reyes Abad Carmen Alerany Luis Ignacio Gonzalez Olaf Neth Concepción Payares-Herrera Carlos Rodríguez-Gallego Jose Luis Trillo Kirsten H. Herrmann Raquel Figueiredo Alicia Gil |
| author_sort | María Reyes Abad |
| collection | DOAJ |
| description | 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.
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| format | Article |
| id | doaj-art-e27430184e10473fa3d551348664652e |
| institution | Kabale University |
| issn | 2284-2403 2283-5733 |
| language | English |
| publishDate | 2025-01-01 |
| publisher | AboutScience Srl |
| record_format | Article |
| series | Global & Regional Health Technology Assessment |
| spelling | doaj-art-e27430184e10473fa3d551348664652e2025-01-28T14:04:08ZengAboutScience SrlGlobal & Regional Health Technology Assessment2284-24032283-57332025-01-0112110.33393/grhta.2025.3199Value contribution of leniolisib in the Treatment of Activated PI3Kδ syndrome (APDS) in Spain using Multi-Criteria Decision Analysis (MCDA)María Reyes Abad0Carmen Alerany1Luis Ignacio Gonzalez2Olaf Neth3Concepción Payares-Herrera4Carlos Rodríguez-Gallego5Jose Luis Trillo6Kirsten H. Herrmann7Raquel Figueiredo8Alicia Gil9Pharmacy Department, H.U. Miguel Servet, Zaragoza - SpainPharmacy Department, H.U. Vall d’ Hebron, Barcelona - SpainImmunodeficiencies Unit H.U. 12 de Octubre, Madrid - SpainPaediatric Infectious Diseases, Rheumatology and Immunology Unit H.U. Virgen del Rocío, Seville - SpainClinical Pharmacology Department H.U. Puerta del Hierro, Majadahonda (Madrid) -SpainDepartment 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 - SpainClinical Department of Malvarrosa, Valencia - SpainPharming Technologies B.V., Leiden - The NetherlandsPharming Technologies B.V., Leiden - The NetherlandsOmakase Consulting S.L., Barcelona - SpainBackground: 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. https://journals.aboutscience.eu/index.php/grhta/article/view/3199Activated phosphoinositide 3-kinase (PI3K) δ Syndrome (APDS)Decision-makingMulti-criteria Decision Analysis (MCDA)Ultra-rare disease, leniolisib |
| spellingShingle | María Reyes Abad Carmen Alerany Luis Ignacio Gonzalez Olaf Neth Concepción Payares-Herrera Carlos Rodríguez-Gallego Jose Luis Trillo Kirsten H. Herrmann Raquel Figueiredo Alicia Gil Value contribution of leniolisib in the Treatment of Activated PI3Kδ syndrome (APDS) in Spain using Multi-Criteria Decision Analysis (MCDA) Global & Regional Health Technology Assessment Activated phosphoinositide 3-kinase (PI3K) δ Syndrome (APDS) Decision-making Multi-criteria Decision Analysis (MCDA) Ultra-rare disease, leniolisib |
| title | Value contribution of leniolisib in the Treatment of Activated PI3Kδ syndrome (APDS) in Spain using Multi-Criteria Decision Analysis (MCDA) |
| title_full | Value contribution of leniolisib in the Treatment of Activated PI3Kδ syndrome (APDS) in Spain using Multi-Criteria Decision Analysis (MCDA) |
| title_fullStr | Value contribution of leniolisib in the Treatment of Activated PI3Kδ syndrome (APDS) in Spain using Multi-Criteria Decision Analysis (MCDA) |
| title_full_unstemmed | Value contribution of leniolisib in the Treatment of Activated PI3Kδ syndrome (APDS) in Spain using Multi-Criteria Decision Analysis (MCDA) |
| title_short | Value contribution of leniolisib in the Treatment of Activated PI3Kδ syndrome (APDS) in Spain using Multi-Criteria Decision Analysis (MCDA) |
| title_sort | value contribution of leniolisib in the treatment of activated pi3kδ syndrome apds in spain using multi criteria decision analysis mcda |
| topic | Activated phosphoinositide 3-kinase (PI3K) δ Syndrome (APDS) Decision-making Multi-criteria Decision Analysis (MCDA) Ultra-rare disease, leniolisib |
| url | https://journals.aboutscience.eu/index.php/grhta/article/view/3199 |
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