Is treatment heterogeneity an Achilles' heel for comparative effectiveness research?

Is treatment heterogeneity an Achilles' heel for comparative effectiveness research?
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治疗异质性是比较有效性研究的致命弱点吗?

DOI:
10.1002/phar.1131
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发表时间:
2012
期刊:
影响因子:
4.1
通讯作者:
Lambert,BruceL
Lambert,BruceL
中科院分区:
医学2区
文献类型:
--
作者:
Alexander,GCaleb;Lambert,BruceL

文献摘要

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《美国复苏与再投资法案》包括11亿美元用于支持比较有效性研究。此后,为扩大比较有效性研究进行了额外的投资,包括支持以患者为中心的结果研究所,这是一个非营利性研究所,其任务是优先考虑和资助比较有效性研究,并将这些努力的结果传播给各个利益相关者。通过比较有效性研究提高医疗保健价值的努力既受到热情的欢迎,也受到阻力。支持者认为,更多地了解不同治疗方法的相对有效性,将使决策者能够选择最安全、最有效的方案,最终增加医疗保健美元的价值。反对者声称,比较有效性研究迫使人们采取“一刀切”的方法。批评强调了治疗反应的个体差异,并警告不要过度依赖“平均效果”。例如,对具有里程碑意义的抗高血压和降脂治疗预防心脏病发作试验(ALLHAT)的经验教训的回顾得出结论,该试验证明了比较有效性研究的价值,但随后强调,基于人群平均风险的研究忽视了个体患者的需求。同一篇综述提醒读者,误用平均效应数据可能会使患者无法获得最佳护理。另一些人则认为,比较有效性研究是“针对新药的”,是“旨在消除个体差异的,而个体差异是下一代药物治疗的核心。
The American Recovery and Reinvestment Act included $1.1 billion to support comparative effectiveness research. Additional investments to expand comparative effectiveness research have since been made, including support for the Patient Centered Outcomes Research Institute, a nonprofit institute tasked with prioritizing and funding comparative effectiveness research and disseminating the results of these efforts to various stakeholders.Efforts to increase the value of health care through comparative effectiveness research have met with both enthusiasm and resistance. Proponents argue that knowing more about the comparative effectiveness of different treatments will empower decision makers to choose the safest and most effective options, ultimately increasing the value of the health care dollar. Opponents claim that comparative effectiveness research forces a “one-size-fits-all” approach. Criticism highlights individual differences in treatment response and warns against the perils of overreliance on “average effects.” For example, a review of lessons learned from the landmark Antihypertensive and Lipid-Lowering Treatment to Prevent Heart Attack Trial (ALLHAT) concluded that the trial demonstrated the value of comparative effectiveness research, but then emphasized that studies based on population averages risk overlooking the needs of individual patients. 1 The same review cautions readers that misapplication of average-effect data might prevent patients from receiving optimal care. Others have argued that comparative effectiveness research is “rigged against new drugs” and is “designed to eliminate the individual differences that are at the heart of the next generation of