在可测量范围之外的观测值存在的情况下,我们对均值和方差的估计是否正确?

IF 2.9 4区 医学 Q2 PHARMACOLOGY & PHARMACY
Markéta Janošová, Stanislav Katina, Jozef Hanes
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引用次数: 0

摘要

用于临床试验安全评估的实验室测量受所用实验室设备的限制。这些限制决定了设备可以精确测量的值范围。当观测值超出可测量范围时,这就产生了估计正态分布参数的问题。使用易于实现的估计方法可能很诱人,但是选择不正确的方法可能导致估计偏倚(估计过低或过高)并改变研究结果,例如,比较两个总体时关于均值的两样本检验结果不正确或回归线估计偏倚。在本文中,我们考虑使用四种方法:忽略未测量的观测值,用极限的倍数代替未测量的观测值,使用截断的正态分布,以及使用带截尾观测值的正态分布。为了比较这两种方法,我们设计了仿真研究,并利用相对误差μ μ - μ μ $$ \frac{\hat{\mu}-\mu }{\mu } $$、比值σ μ σ $$ \frac{\hat{\sigma}}{\sigma } $$和两种参数的均方误差测量了它们在几种不同情况下的精度。根据本次模拟研究的结果,如果在可测量范围外的观测量低于40%, we recommend using a normal distribution with censored observations in practice. These recommendations should be incorporated into guidelines for good statistical practice. If the amount of observations outside of measurable range exceeds 40%, we advise not to use the data for any statistical analysis. To illustrate how the choice of method can affect the estimates, we applied the methods to real-life laboratory data.
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Are We Estimating the Mean and Variance Correctly in the Presence of Observations Outside of Measurable Range?

Laboratory measurements used for safety assessments in clinical trials are subject to the limits of the used laboratory equipment. These limits determine the range of values which the equipment can accurately measure. When observations fall outside the measurable range, this creates a problem in estimating parameters of the normal distribution. It may be tempting to use methods of estimation that are easy to implement, however selecting an incorrect method may lead to biased estimates (under- or overestimation) and change the research outcomes, for example, incorrect result of two-sample test about means when comparing two populations or biased estimation of regression line. In this article, we consider the use of four methods: ignoring unmeasured observations, replacing unmeasured observations with a multiple of the limit, using a truncated normal distribution, and using a normal distribution with censored observations. To compare these methods we designed a simulation study and measured their accuracy in several different situations using relative error μ ̂ - μ μ $$ \frac{\hat{\mu}-\mu }{\mu } $$ , ratio σ ̂ σ $$ \frac{\hat{\sigma}}{\sigma } $$ , and mean square errors of both parameters. Based on the results of this simulation study, if the amount of observations outside of measurable range is below 40%, we recommend using a normal distribution with censored observations in practice. These recommendations should be incorporated into guidelines for good statistical practice. If the amount of observations outside of measurable range exceeds 40%, we advise not to use the data for any statistical analysis. To illustrate how the choice of method can affect the estimates, we applied the methods to real-life laboratory data.

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来源期刊
Pharmacology Research & Perspectives
Pharmacology Research & Perspectives Pharmacology, Toxicology and Pharmaceutics-General Pharmacology, Toxicology and Pharmaceutics
CiteScore
5.30
自引率
3.80%
发文量
120
审稿时长
20 weeks
期刊介绍: PR&P is jointly published by the American Society for Pharmacology and Experimental Therapeutics (ASPET), the British Pharmacological Society (BPS), and Wiley. PR&P is a bi-monthly open access journal that publishes a range of article types, including: target validation (preclinical papers that show a hypothesis is incorrect or papers on drugs that have failed in early clinical development); drug discovery reviews (strategy, hypotheses, and data resulting in a successful therapeutic drug); frontiers in translational medicine (drug and target validation for an unmet therapeutic need); pharmacological hypotheses (reviews that are oriented to inform a novel hypothesis); and replication studies (work that refutes key findings [failed replication] and work that validates key findings). PR&P publishes papers submitted directly to the journal and those referred from the journals of ASPET and the BPS
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