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Statistical Power, the Belmont Report, and the Ethics of Clinical Trials

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Abstract

Achieving a good clinical trial design increases the likelihood that a trial will take place as planned, including that data will be obtained from a sufficient number of participants, and the total number of participants will be the minimal required to gain the knowledge sought. A good trial design also increases the likelihood that the knowledge sought by the experiment will be forthcoming. Achieving such a design is more than good sense—it is ethically required in experiments when participants are at risk of harm. This paper argues that doing a power analysis effectively contributes to ensuring that a trial design is good. The ethical importance of good trial design has long been recognized for trials in which there is risk of serious harm to participants. However, whether the quality of a trial design, when the risk to participants is only minimal, is an ethical issue is rarely discussed. This paper argues that even in cases when the risk is minimal, the quality of the trial design is an ethical issue, and that this is reflected in the emphasis the Belmont Report places on the importance of the benefit of knowledge gained by society. The paper also argues that good trial design is required for true informed consent.

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Notes

  1. Generally, the power to detect a difference is a function of four parameters: (1) the significance level required to declare a difference, which is generally set to an α = 0.05 level, (2) the true difference from the null hypothesis, (3) the sample size within study groups, and (4) the variance of the data within treatment groups (the within group standard deviation or other measure of “noise” in the measurement). In this paper, “power” refers to the power to detect a “clinically significant difference” (the difference that is large enough to affect treatments and so of importance in study design) at a fixed α and measure of variance; hence, this paper considers the relationship between sample size and power at a fixed level of the other three parameters.

  2. The first cutoff referred to concerns thresholds to declare efficacy (i.e., the p-value); the second kind of cutoff, a consideration of power, is more of a “design consideration” than a “reporting consideration.” That is, if a study is done that is underpowered, but still is significant, then power is no longer a consideration. Power has more to do with whether it is reasonable to conduct the research rather than whether it is reasonable to report the research.

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Correspondence to Sara H. Vollmer.

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Vollmer, S.H., Howard, G. Statistical Power, the Belmont Report, and the Ethics of Clinical Trials. Sci Eng Ethics 16, 675–691 (2010). https://doi.org/10.1007/s11948-010-9244-0

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  • DOI: https://doi.org/10.1007/s11948-010-9244-0

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