Pioneering SMA therapies for all types: survival gains, cost dynamics, and performance-based agreements

Pioneering SMA therapies for all types: survival gains, cost dynamics, and performance-based agreements

Abstract Background The purpose of this study was to assess the impact of survival improvements and performance-based managed entry agreements (PBMEAs) on the cost implications of introducing innovative spinal muscular atrophy (SMA) treatments, nusinersen, onasemnogene abeparvovec, and risdiplam, fo...

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Main Authors: Ahmed Al-jedai, Hajer Al-Mudaiheem, AlJohara AlSakran, Fahad A. Bashiri, Fouad Ghamdi, Mohammad A. Almuhaizea, Abdulaziz AlSamman, Nancy Awad, Rita Ojeil
Format: Article
Language:English
Published: BMC 2025-08-01
Series:Cost Effectiveness and Resource Allocation
Subjects:
Spinal muscular atrophy (SMA)
Budget impact model (BIM)
Health technology assessment (HTA)
Kingdom of saudi arabia (KSA)
Saudi ministry of health (MoH)
Performance-based managed entry agreement (PBMEA)
Online Access:https://doi.org/10.1186/s12962-025-00647-3
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https://doi.org/10.1186/s12962-025-00647-3

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