Value contribution of leniolisib in the Treatment of Activated PI3Kδ syndrome (APDS) in Spain using Multi-Criteria Decision Analysis (MCDA)

Abstract

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.