Department of Health Services Research and Policy, Faculty of Public Health and Policy, London School of Hygiene and Tropical Medicine, London, UK.
Expert Rev Pharmacoecon Outcomes Res. 2023 Jun;23(5):469-482. doi: 10.1080/14737167.2023.2193690. Epub 2023 Apr 5.
The National Institute for Health and Care Excellence (NICE) in England has appraised three treatments for spinal muscular atrophy (SMA), namely, nusinersen, onasemnogene abeparvovec, and risdiplam. As rare disease treatments (RDTs) commonly face challenges in health technology assessment (HTA) processes due to their clinical and economic uncertainties, an in-depth review of these appraisals is useful to enable a deeper understanding of economic modeling considerations for SMA.
This review is a detailed analysis of NICE appraisals for SMA and aims to compare the economic modeling evidence from the three RDTs. This is done by examining differences and similarities and by discussing critical outstanding issues across the economic evaluations of the appraisals.
This article aims to contribute to the development of evidence that can be used as guidance to inform resource allocation decisions for RDTs for SMA, but also to be a resource about approaches for the generation, analysis and interpretation of economic modeling evidence for RDTs more broadly.
英国国家卫生与保健优化研究所（NICE）对脊髓性肌萎缩症（SMA）的三种治疗方法进行了评估，即nusinersen、onasemnogene abeparvovec 和 risdiplam。由于罕见病治疗（RDT）在临床和经济方面存在不确定性，通常在卫生技术评估（HTA）过程中面临挑战，因此深入审查这些评估结果有助于更好地了解 SMA 经济建模的考虑因素。
本综述对 NICE 对 SMA 的评估进行了详细分析，旨在比较三种 RDT 的经济建模证据。通过检查差异和相似之处，并讨论评估中经济评估的关键未决问题，来实现这一目标。
本文旨在为制定证据做出贡献，这些证据可作为指导，为 SMA 的 RDT 资源分配决策提供信息，同时也为更广泛地生成、分析和解释 RDT 经济建模证据的方法提供资源。
