Determining value in the treatment of activated PI3Kδ syndrome in Spain: a multicriteria decision analysis from the perspective of key stakeholders.
Maria R Abad SazatornilCarmen AleranyLaia AlsinaEduardo López GranadosOlaf NethJose Luis PovedaJacques G RivièreCarlos Rodríguez-GallegoJoanne B Tutein NoltheniusRaquel FigueiredoSilvia Subías LabazuyAlicia GilPublished in: Global & regional health technology assessment (2024)
APDS is considered a severe disease, with limited understanding by key stakeholders of how treatment success is assessed in clinical practice, the serious impact that has on patients and the associated high economic burden. This study brings to light how MCDA methodology could represent a useful tool to complement current clinical and decision-making methods used by APDS experts and evaluators.