Objectives

Nusinersen (Spinraza®, Biogen) and onasemnogene abeparvosec (Zolgensma®, Novartis) are novel gene-based therapies for the orphan disease Spinal Muscular Atrophy. Onasemnogene abeparvosec has been allocated an acquisition cost of up to US$5 million per patient. We undertook a rapid inquiry to evaluate if onasemnogene abeparvosec is likely to be cost-effective for the UK NHS.

Methods

We used publicly available cost-effectiveness data and recommended methodology to perform cost-utility evaluation of onasemnogene abeparvosec versus best supportive care and nusinersen.

Results

Our evaluations highlight wide variations in cost and benefit estimates of nusinersen and indicate that onasemnogene abeparvosec is unlikely to represent value for money according to current standards of reimbursement. Results are discussed in the context of reimbursement decisions for orphan diseases.

Conclusion

Commonly implemented commercial confidentiality practices combined with uncertain data obscure scrutiny and justification of past and present reimbursement decisions for orphan drugs. Future cutting edge expensive therapies will be numerous, they will entail very substantial economic strains. We conclude that there is an urgent and increasing need for the development of improved procedures that can lead to equitable, consistent, and transparent decision-making.

中文翻译：

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价值500万美元的onasemnogene abeparvosec治疗脊髓性肌萎缩症是否代表NHS的“物有所值”？快速查询可能具有成本效益的建议。

目标

Nusinersen（Spinraza®，Biogen）和onasemnogene abeparvosec（Zolgensma®，Novartis）是基于基因的新型疗法，用于治疗孤儿疾病脊髓性肌萎缩症。已为每位患者分配了Onasemnogene abeparvosec的收购成本，最高可达500万美元。我们进行了快速调查，以评估onasemnogene abeparvosec对于英国NHS是否可能具有成本效益。

方法

我们使用可公开获得的成本效益数据和推荐的方法，对阿贝帕伏松致癌性基因与最佳支持治疗和新生儿治疗进行成本效用评估。

结果

我们的评估突显了努森人的成本和收益估计值的巨大差异，并表明根据当前的报销标准，致癌基因abeparvosec不太可能代表物有所值。在对孤儿疾病的报销决策中讨论了结果。

结论

普遍采用的商业机密做法加上不确定的数据，使对孤儿药过去和现在的报销决策的审查和理由难以理解。未来尖端的昂贵疗法将是无数种，它们将带来非常巨大的经济压力。我们得出结论，迫切且越来越需要开发改进的程序，以实现公平，一致和透明的决策。