针对脂蛋白的RNA疗法的早期健康经济评估的观点(a)。

IF 2.7 3区 医学 Q3 ENDOCRINOLOGY & METABOLISM Current Opinion in Endocrinology & Diabetes and Obesity Pub Date : 2025-04-01 Epub Date: 2025-02-12 DOI:10.1097/MED.0000000000000904
Angela Burvill, Gerald F Watts, Zanfina Ademi
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引用次数: 0

摘要

审查目的:对新药物进行早期卫生经济评价是有用的,因为它们考虑到对卫生服务的影响。我们回顾了最近关于降低血浆脂蛋白升高(a) [Lp(a)]在二级预防中的预期临床结果的文献,这是经济评估有效性的重要信息。我们回顾了最近针对Lp(a)的RNA疗法的早期经济评估。最近的研究发现:基于rna的治疗,如果获得批准,可能最初用于患有动脉粥样硬化性心血管疾病(ASCVD)和非常高Lp(a)的成人。成人ASCVD患者复发事件的绝对风险高,升高的Lp(a)是一个风险增强因素。二级预防中Lp(a)的有效降低可能与冠心病或ASCVD事件的显著相对风险降低相关;这需要在目前正在进行和未来的临床试验中得到证实。一项经济评估估计了olpasiran和pelacarsen的价值,在不同的支付意愿阈值下,与标准护理二级预防相比。摘要:早期经济评估评估与新药相关的长期临床效益和成本后果。在等待主要心血管结局试验结果的同时,现有的Lp(a)和心血管疾病的偶然证据可作为现有的最佳证据用于早期经济评估。
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Perspectives on early health economic evaluations of RNA therapies targeted at lipoprotein(a).

Purpose of review: Early health economic evaluations of new medications are useful, as they consider the implications for health services.We reviewed recent literature on expected clinical outcomes of lowering of elevated plasma lipoprotein(a) [Lp(a)] in secondary prevention, which is essential information on effectiveness for economic evaluations.We reviewed a recent early economic evaluation of RNA therapies targeted at Lp(a).

Recent findings: RNA-based therapies, if approved, would likely be used initially in adults with established atherosclerotic cardiovascular disease (ASCVD) and very high Lp(a). Adults with ASCVD have high absolute risk of recurrent events and elevated Lp(a) serves as a risk-enhancing factor.Potent lowering of Lp(a) in secondary prevention may be associated with significant relative risk reductions of coronary heart disease or ASCVD events; this needs confirmation in currently ongoing and future clinical trials.One economic evaluation has estimated the value of olpasiran and pelacarsen, at various willingness-to-pay thresholds, compared with standard-of-care secondary prevention.

Summary: Early economic evaluations estimate longer-term clinical benefits and cost consequences associated with new medications.Existing casual evidence of Lp(a) and cardiovascular disease can be used in early economic evaluations as best available evidence, while awaiting results from major cardiovascular outcomes trials.

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来源期刊
CiteScore
5.80
自引率
3.10%
发文量
128
审稿时长
6-12 weeks
期刊介绍: ​​​​​​​​Current Opinion in Endocrinology, Diabetes and Obesity delivers a broad-based perspective on the most recent and exciting developments in the field from across the world. Published bimonthly and featuring twelve key topics – including androgens, gastrointestinal hormones, diabetes and the endocrine pancreas, and neuroendocrinology – the journal’s renowned team of guest editors ensure a balanced, expert assessment of the recently published literature in each respective field with insightful editorials and on-the-mark invited reviews.
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