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The main goal of many medical studies is to evaluate the effect of a treatment or the risk of disease under given conditions. This entry introduces and discusses measures of effect and/or risk when the factors or variables of interest are categorical (i.e., nominal) in nature. The discussion focuses on the case of dichotomous variables (factors), that is, those that take only two values that often indicate the presence or absence of a characteristic (disease, treatment, exposure, etc.). The data consist of information on n individuals who have been categorized according to the presence or absence of two factors. The information is presented in a 2 × 2 contingency table like the one in Table 1.

The numbers in the table denote the frequency of each cell. For example, a is the number of individuals for whom both factors were present, and b those for whom A was present and B was absent. Thus, the total number of patients is n = a + b + c + d. Common types of factors are exposure, treatment, and disease. The statistical significance of the association between the two factors is tested using the chisquare test (or the likelihood test). The result of the test is a p value, which measures the chance of the observed relationship under the assumption that there is none. Accordingly, a small p value (e.g., < .05) leads to a “significant” result and the rejection of the assumption of no association. The size of the p value is determined, in a critical way, by the sample size and can't be used to assess the strength of the association. When the association (or effect, when one factor is the “cause” of the other) is significant, the measure of its strength is critical. Below are the most common measures of association.

Definition 1. The relative risk (RR) is defined as

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where Pr[ ] indicates probability.

Definition 2. The risk difference (RD), also called attributable risk, is defined as

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Definition 3. The odds ratio (OR) is defined as

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Table 1 The 2 × 2 contingency table

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Comparing Measures

As mentioned, the significance of the association is tested using the chi-square test (or the likelihood test). However, when it comes to measuring the strength of the association, there is not only a lack of consensus but also considerable confusion. The three measures defined above assess the strength of the relationship but do so in different ways. The confusion in the interpretation of these measures is caused by the fact that each one is based on ratios (relative frequencies or odds). So, in the case of RR and OR, the reader is faced with the daunting task of interpreting ratios of ratios. It is therefore crucial to understand the differences and, consequently, the correct way to interpret these measures.

The RR is a relative measure interpreted as a percentage. It is important to note that both RR and 1/RR are relative risks and assess the strength of an association from a different point of view. The choice of denominator, which is not always obvious, determines the value and the interpretation. For example, RR = 1.30 describes a risk increase of 30%, whereas 1/RR = .77 is interpreted as a 23% decrease in risk. The key is to realize that RR describes a 30% risk increase of exposure (i.e., A present) relative to nonexposure (i.e., A absent), whereas 1/RR represents a 23% risk decrease in nonexposure relative to exposure. Without careful description, the interpretation of the value will depend on whether RR or 1/RR is used, when, in fact, they are measuring the same association. In general, relative measures such as RR and OR are not easy to interpret and can be misleading. The first two columns of Table 2 contain combinations of risks that yield the same value of RR, but represent very different situations.

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