A Review of Quantitative Risk-Benefit Methodologies for Assessing Drug Safety and Efficacy-Report of the ISPOR Risk-Benefit Management Working Group
A Review of Quantitative Risk-Benefit Methodologies for Assessing Drug Safety and Efficacy-Report of the ISPOR Risk-Benefit Management Working Group
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DOI：
10.1111/j.1524-4733.2010.00725.x
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发表时间：
2010-07-01
期刊：
影响因子：
4.5
通讯作者：
Raisch, Dennis W.
中科院分区：
文献类型：
--
作者：
Guo, Jeff J.;Pandey, Swapnil;Raisch, Dennis W.
目的：尽管监管机构在新药批准过程中评估了任何新药治疗的风险和获益，但通常不会进行定量风险-获益评估（RBA），也不会在使用时以一致和综合的框架呈现。我们的目的是确定和描述出版的定量RBA方法为pharmacists.Methods：使用MEDLINE和其他基于互联网的搜索引擎，进行了系统的文献综述，以确定定量方法RBA。这些不同的RBA方法进行了总结，以突出其差异对制药行业和监管机构的影响。对文献中确定的12种定量RBA方法的理论模型、参数和关键特征进行了综述和比较，包括风险和获益评估的定量框架、获益-风险分析、无症状和毒性的质量调整时间、需要治疗的数量（NNT）和需要伤害的数量及其相对值调整版本、最低临床疗效、增量净健康获益、风险-获益平面（RBP），概率模拟法、多准则决策分析法（MCDA）、风险收益等值线法（RBC）和陈述偏好法（SPM）。而一些方法（例如，NNT）依赖于主观加权方案或非统计评估，其他方法（例如，RBP，MCDA，RBC和SPM）评估联合分布的好处和risk.Conclusions：几个定量RBA方法可用于帮助减轻对主观药物评估的关注，并帮助指导当局更客观和透明的决策。在评价一种新的药物治疗时，我们建议在不同的治疗适应症和治疗人群中使用多种RBA方法，以限制风险-获益特征。
Objective:Although regulatory authorities evaluate the risks and benefits of any new drug therapy during the new drug-approval process, quantitative risk-benefit assessment (RBA) is not typically performed, nor is it presented in a consistent and integrated framework when it is used. Our purpose is to identify and describe published quantitative RBA methods for pharmaceuticals.Methods:Using MEDLINE and other Internet-based search engines, a systematic literature review was performed to identify quantitative methodologies for RBA. These distinct RBA approaches were summarized to highlight the implications of their differences for the pharmaceutical industry and regulatory agencies.Results:Theoretical models, parameters, and key features were reviewed and compared for the 12 quantitative RBA methods identified in the literature, including the Quantitative Framework for Risk and Benefit Assessment, benefit-less-risk analysis, the quality-adjusted time without symptoms and toxicity, number needed to treat (NNT), and number needed to harm and their relative-value-adjusted versions, minimum clinical efficacy, incremental net health benefit, the risk-benefit plane (RBP), the probabilistic simulation method, multicriteria decision analysis (MCDA), the risk-benefit contour (RBC), and the stated preference method (SPM). Whereas some approaches (e.g., NNT) rely on subjective weighting schemes or nonstatistical assessments, other methods (e.g., RBP, MCDA, RBC, and SPM) assess joint distributions of benefit and risk.Conclusions:Several quantitative RBA methods are available that could be used to help lessen concern over subjective drug assessments and to help guide authorities toward more objective and transparent decision-making. When evaluating a new drug therapy, we recommend the use of multiple RBA approaches across different therapeutic indications and treatment populations in order to bound the risk-benefit profile.