Measures of Effect in Clinical Studies

When conducting clinical studies, we often want to know the effect of treatments of exposures on health outcomes. Measures of effect are used in randomized controlled trials (RCTs) and include the odds ratio (of), risk ratio (RR), risk difference (RD), and number needed to treat (NNT). Dichotomous (binary) outcome data are common in clinical trials, where the outcome for each participant is one of two possibilities, such as dead of alive, of clinical improvement of no improvement.

To understand the difference between of and RR, it’s important to know the difference between risks and odds. Risk is a proportion that describes the probability of a health outcome occurring, while odds is a ratio that compares the probability of an event occurring to the probability of it not occurring. Absolute risk is the basic risk, while risk difference is the difference between the absolute risk of an event in the intervention group and the absolute risk in the control group. Relative risk is the ratio of risk in the intervention group to the risk in the control group.

The number needed to treat (NNT) is the number of patients who need to be treated for one to benefit. Odds are calculated by dividing the number of times an event happens by the number of times it does not happen. The odds ratio is the odds of an outcome given a particular exposure versus the odds of an outcome in the absence of the exposure. It is commonly used in case-control studies and can also be used in cross-sectional and cohort study designs. An odds ratio of 1 indicates no difference in risk between the two groups, while an odds ratio >1 indicates an increased risk and an odds ratio <1 indicates a reduced risk.

The acronym ICER stands for incremental cost-effectiveness ratio.

Methods of Economic Evaluation

There are four main methods of economic evaluation: cost-effectiveness analysis (CEA), cost-benefit analysis (CBA), cost-utility analysis (CUA), and cost-minimisation analysis (CMA). While all four methods capture costs, they differ in how they assess health effects.

Cost-effectiveness analysis (CEA) compares interventions by relating costs to a single clinical measure of effectiveness, such as symptom reduction of improvement in activities of daily living. The cost-effectiveness ratio is calculated as total cost divided by units of effectiveness. CEA is typically used when CBA cannot be performed due to the inability to monetise benefits.

Cost-benefit analysis (CBA) measures all costs and benefits of an intervention in monetary terms to establish which alternative has the greatest net benefit. CBA requires that all consequences of an intervention, such as life-years saved, treatment side-effects, symptom relief, disability, pain, and discomfort, are allocated a monetary value. CBA is rarely used in mental health service evaluation due to the difficulty in converting benefits from mental health programmes into monetary values.

Cost-utility analysis (CUA) is a special form of CEA in which health benefits/outcomes are measured in broader, more generic ways, enabling comparisons between treatments for different diseases and conditions. Multidimensional health outcomes are measured by a single preference- of utility-based index such as the Quality-Adjusted-Life-Years (QALY). QALYs are a composite measure of gains in life expectancy and health-related quality of life. CUA allows for comparisons across treatments for different conditions.

Cost-minimisation analysis (CMA) is an economic evaluation in which the consequences of competing interventions are the same, and only inputs, i.e. costs, are taken into consideration. The aim is to decide the least costly way of achieving the same outcome.

Costs in Economic Evaluation Studies

There are three main types of costs in economic evaluation studies: direct, indirect, and intangible. Direct costs are associated directly with the healthcare intervention, such as staff time, medical supplies, cost of travel for the patient, childcare costs for the patient, and costs falling on other social sectors such as domestic help from social services. Indirect costs are incurred by the reduced productivity of the patient, such as time off work, reduced work productivity, and time spent caring for the patient by relatives. Intangible costs are difficult to measure, such as pain of suffering on the part of the patient.

Methods of Economic Evaluation

There are four main methods of economic evaluation: cost-effectiveness analysis (CEA), cost-benefit analysis (CBA), cost-utility analysis (CUA), and cost-minimisation analysis (CMA). While all four methods capture costs, they differ in how they assess health effects.

Cost-effectiveness analysis (CEA) compares interventions by relating costs to a single clinical measure of effectiveness, such as symptom reduction of improvement in activities of daily living. The cost-effectiveness ratio is calculated as total cost divided by units of effectiveness. CEA is typically used when CBA cannot be performed due to the inability to monetise benefits.

Cost-benefit analysis (CBA) measures all costs and benefits of an intervention in monetary terms to establish which alternative has the greatest net benefit. CBA requires that all consequences of an intervention, such as life-years saved, treatment side-effects, symptom relief, disability, pain, and discomfort, are allocated a monetary value. CBA is rarely used in mental health service evaluation due to the difficulty in converting benefits from mental health programmes into monetary values.

Cost-utility analysis (CUA) is a special form of CEA in which health benefits/outcomes are measured in broader, more generic ways, enabling comparisons between treatments for different diseases and conditions. Multidimensional health outcomes are measured by a single preference- of utility-based index such as the Quality-Adjusted-Life-Years (QALY). QALYs are a composite measure of gains in life expectancy and health-related quality of life. CUA allows for comparisons across treatments for different conditions.

Cost-minimisation analysis (CMA) is an economic evaluation in which the consequences of competing interventions are the same, and only inputs, i.e. costs, are taken into consideration. The aim is to decide the least costly way of achieving the same outcome.

Costs in Economic Evaluation Studies

There are three main types of costs in economic evaluation studies: direct, indirect, and intangible. Direct costs are associated directly with the healthcare intervention, such as staff time, medical supplies, cost of travel for the patient, childcare costs for the patient, and costs falling on other social sectors such as domestic help from social services. Indirect costs are incurred by the reduced productivity of the patient, such as time off work, reduced work productivity, and time spent caring for the patient by relatives. Intangible costs are difficult to measure, such as pain of suffering on the part of the patient.

Measures of Effect in Clinical Studies

When conducting clinical studies, we often want to know the effect of treatments of exposures on health outcomes. Measures of effect are used in randomized controlled trials (RCTs) and include the odds ratio (of), risk ratio (RR), risk difference (RD), and number needed to treat (NNT). Dichotomous (binary) outcome data are common in clinical trials, where the outcome for each participant is one of two possibilities, such as dead of alive, of clinical improvement of no improvement.

To understand the difference between of and RR, it’s important to know the difference between risks and odds. Risk is a proportion that describes the probability of a health outcome occurring, while odds is a ratio that compares the probability of an event occurring to the probability of it not occurring. Absolute risk is the basic risk, while risk difference is the difference between the absolute risk of an event in the intervention group and the absolute risk in the control group. Relative risk is the ratio of risk in the intervention group to the risk in the control group.

The number needed to treat (NNT) is the number of patients who need to be treated for one to benefit. Odds are calculated by dividing the number of times an event happens by the number of times it does not happen. The odds ratio is the odds of an outcome given a particular exposure versus the odds of an outcome in the absence of the exposure. It is commonly used in case-control studies and can also be used in cross-sectional and cohort study designs. An odds ratio of 1 indicates no difference in risk between the two groups, while an odds ratio >1 indicates an increased risk and an odds ratio <1 indicates a reduced risk.

A study’s ability to correctly detect a true effect of difference may be calculated as Power = 1 – Type II error rate. In the given scenario, the power can be calculated as Power = 1 – 0.2 = 0.8. Type I error refers to a false positive, while Type II error refers to a false negative.

The new drug reduces the annual incidence of stroke by 0.5% and costs £100 more than conventional therapy. This means that for every 200 patients treated, one stroke would be prevented with the new drug compared to conventional therapy. The Number Needed to Treat (NNT) is 200 per year to prevent one stroke. Therefore, the annual cost of this treatment to prevent one stroke would be £20,000, which is the cost of treating 200 patients with the new drug (£100 x 200).

Measures of Effect in Clinical Studies

When conducting clinical studies, we often want to know the effect of treatments of exposures on health outcomes. Measures of effect are used in randomized controlled trials (RCTs) and include the odds ratio (of), risk ratio (RR), risk difference (RD), and number needed to treat (NNT). Dichotomous (binary) outcome data are common in clinical trials, where the outcome for each participant is one of two possibilities, such as dead of alive, of clinical improvement of no improvement.

To understand the difference between of and RR, it’s important to know the difference between risks and odds. Risk is a proportion that describes the probability of a health outcome occurring, while odds is a ratio that compares the probability of an event occurring to the probability of it not occurring. Absolute risk is the basic risk, while risk difference is the difference between the absolute risk of an event in the intervention group and the absolute risk in the control group. Relative risk is the ratio of risk in the intervention group to the risk in the control group.

The number needed to treat (NNT) is the number of patients who need to be treated for one to benefit. Odds are calculated by dividing the number of times an event happens by the number of times it does not happen. The odds ratio is the odds of an outcome given a particular exposure versus the odds of an outcome in the absence of the exposure. It is commonly used in case-control studies and can also be used in cross-sectional and cohort study designs. An odds ratio of 1 indicates no difference in risk between the two groups, while an odds ratio >1 indicates an increased risk and an odds ratio <1 indicates a reduced risk.

When dealing with a data set that is quantitative and measured on a ratio scale, the mean is typically the preferred measure of central tendency. However, if the data is skewed, the median may be a better choice as it is less affected by the skewness of the data.

Measures of Central Tendency

Measures of central tendency are used in descriptive statistics to summarize the middle of typical value of a data set. There are three common measures of central tendency: the mean, median, and mode.

The median is the middle value in a data set that has been arranged in numerical order. It is not affected by outliers and is used for ordinal data. The mode is the most frequent value in a data set and is used for categorical data. The mean is calculated by adding all the values in a data set and dividing by the number of values. It is sensitive to outliers and is used for interval and ratio data.

The appropriate measure of central tendency depends on the measurement scale of the data. For nominal and categorical data, the mode is used. For ordinal data, the median of mode is used. For interval data with a normal distribution, the mean is preferable, but the median of mode can also be used. For interval data with skewed distribution, the median is used. For ratio data, the mean is preferable, but the median of mode can also be used for skewed data.

In addition to measures of central tendency, the range is also used to describe the spread of a data set. It is calculated by subtracting the smallest value from the largest value.

The characteristics of precision, sensitivity, accuracy, and specificity are not influenced by the prevalence of the condition and remain stable. However, the positive predictive value is affected by the prevalence of the condition, particularly in cases where the prevalence is low. This is because a decrease in the prevalence of the condition leads to a decrease in the number of true positives, which in turn reduces the numerator of the PPV equation, resulting in a lower PPV. The formula for PPV is TP/(TP+FP).

Clinical tests are used to determine the presence of absence of a disease of condition. To interpret test results, it is important to have a working knowledge of statistics used to describe them. Two by two tables are commonly used to calculate test statistics such as sensitivity and specificity. Sensitivity refers to the proportion of people with a condition that the test correctly identifies, while specificity refers to the proportion of people without a condition that the test correctly identifies. Accuracy tells us how closely a test measures to its true value, while predictive values help us understand the likelihood of having a disease based on a positive of negative test result. Likelihood ratios combine sensitivity and specificity into a single figure that can refine our estimation of the probability of a disease being present. Pre and post-test odds and probabilities can also be calculated to better understand the likelihood of having a disease before and after a test is carried out. Fagan’s nomogram is a useful tool for calculating post-test probabilities.

Sampling Methods in Statistics

When collecting data from a population, it is often impractical and unnecessary to gather information from every single member. Instead, taking a sample is preferred. However, it is crucial that the sample accurately represents the population from which it is drawn. There are two main types of sampling methods: probability (random) sampling and non-probability (non-random) sampling.

Non-probability sampling methods, also known as judgement samples, are based on human choice rather than random selection. These samples are convenient and cheaper than probability sampling methods. Examples of non-probability sampling methods include voluntary sampling, convenience sampling, snowball sampling, and quota sampling.

Probability sampling methods give a more representative sample of the population than non-probability sampling. In each probability sampling technique, each population element has a known (non-zero) chance of being selected for the sample. Examples of probability sampling methods include simple random sampling, systematic sampling, cluster sampling, stratified sampling, and multistage sampling.

Simple random sampling is a sample in which every member of the population has an equal chance of being chosen. Systematic sampling involves selecting every kth member of the population. Cluster sampling involves dividing a population into separate groups (called clusters) and selecting a random sample of clusters. Stratified sampling involves dividing a population into groups (strata) and taking a random sample from each strata. Multistage sampling is a more complex method that involves several stages and combines two of more sampling methods.

Overall, probability sampling methods give a more representative sample of the population, but non-probability sampling methods are often more convenient and cheaper. It is important to choose the appropriate sampling method based on the research question and available resources.

The Delphi Method: A Widely Used Technique for Achieving Convergence of Opinion

The Delphi method is a well-established technique for soliciting expert opinions on real-world knowledge within specific topic areas. The process involves multiple rounds of questionnaires, with each round building on the previous one to achieve convergence of opinion among the participants. However, there are potential issues with the Delphi method, such as the time-consuming nature of the process, low response rates, and the potential for investigators to influence the opinions of the participants. Despite these challenges, the Delphi method remains a valuable tool for generating consensus among experts in various fields.

Understanding Hypothesis Testing in Statistics

In statistics, it is not feasible to investigate hypotheses on entire populations. Therefore, researchers take samples and use them to make estimates about the population they are drawn from. However, this leads to uncertainty as there is no guarantee that the sample taken will be truly representative of the population, resulting in potential errors. Statistical hypothesis testing is the process used to determine if claims from samples to populations can be made and with what certainty.

The null hypothesis (Ho) is the claim that there is no real difference between two groups, while the alternative hypothesis (H1 of Ha) suggests that any difference is due to some non-random chance. The alternative hypothesis can be one-tailed of two-tailed, depending on whether it seeks to establish a difference of a change in one direction.

Two types of errors may occur when testing the null hypothesis: Type I and Type II errors. Type I error occurs when the null hypothesis is rejected when it is true, while Type II error occurs when the null hypothesis is accepted when it is false. The power of a study is the probability of correctly rejecting the null hypothesis when it is false, and it can be increased by increasing the sample size.

P-values provide information on statistical significance and help researchers decide if study results have occurred due to chance. The p-value is the probability of obtaining a result that is as large of larger when in reality there is no difference between two groups. The cutoff for the p-value is called the significance level (alpha level), typically set at 0.05. If the p-value is less than the cutoff, the null hypothesis is rejected, and if it is greater or equal to the cut off, the null hypothesis is not rejected. However, the p-value does not indicate clinical significance, which may be too small to be meaningful.

Stats Confounding

A confounding factor is a factor that can obscure the relationship between an exposure and an outcome in a study. This factor is associated with both the exposure and the disease. For example, in a study that finds a link between coffee consumption and heart disease, smoking could be a confounding factor because it is associated with both drinking coffee and heart disease. Confounding occurs when there is a non-random distribution of risk factors in the population, such as age, sex, and social class.

To control for confounding in the design stage of an experiment, researchers can use randomization, restriction, of matching. Randomization aims to produce an even distribution of potential risk factors in two populations. Restriction involves limiting the study population to a specific group to ensure similar age distributions. Matching involves finding and enrolling participants who are similar in terms of potential confounding factors.

In the analysis stage of an experiment, researchers can control for confounding by using stratification of multivariate models such as logistic regression, linear regression, of analysis of covariance (ANCOVA). Stratification involves creating categories of strata in which the confounding variable does not vary of varies minimally.

Overall, controlling for confounding is important in ensuring that the relationship between an exposure and an outcome is accurately assessed in a study.

The Hawthorne effect is a phenomenon where individuals may alter their actions of responses when they are aware that they are being monitored of studied. Out of the given choices, the only one that pertains to a change in behavior is the adherence to medication. The remaining options related to outcomes that are not under conscious control.

Types of Bias in Statistics

Bias is a systematic error that can lead to incorrect conclusions. Confounding factors are variables that are associated with both the outcome and the exposure but have no causative role. Confounding can be addressed in the design and analysis stage of a study. The main method of controlling confounding in the analysis phase is stratification analysis. The main methods used in the design stage are matching, randomization, and restriction of participants.

There are two main types of bias: selection bias and information bias. Selection bias occurs when the selected sample is not a representative sample of the reference population. Disease spectrum bias, self-selection bias, participation bias, incidence-prevalence bias, exclusion bias, publication of dissemination bias, citation bias, and Berkson’s bias are all subtypes of selection bias. Information bias occurs when gathered information about exposure, outcome, of both is not correct and there was an error in measurement. Detection bias, recall bias, lead time bias, interviewer/observer bias, verification and work-up bias, Hawthorne effect, and ecological fallacy are all subtypes of information bias.

You don’t need to concern yourself with the specifics of the various means. Simply keep in mind that the arithmetic mean is the one utilized in fundamental biostatistics.

Measures of Central Tendency

Measures of central tendency are used in descriptive statistics to summarize the middle of typical value of a data set. There are three common measures of central tendency: the mean, median, and mode.

The median is the middle value in a data set that has been arranged in numerical order. It is not affected by outliers and is used for ordinal data. The mode is the most frequent value in a data set and is used for categorical data. The mean is calculated by adding all the values in a data set and dividing by the number of values. It is sensitive to outliers and is used for interval and ratio data.

The appropriate measure of central tendency depends on the measurement scale of the data. For nominal and categorical data, the mode is used. For ordinal data, the median of mode is used. For interval data with a normal distribution, the mean is preferable, but the median of mode can also be used. For interval data with skewed distribution, the median is used. For ratio data, the mean is preferable, but the median of mode can also be used for skewed data.

In addition to measures of central tendency, the range is also used to describe the spread of a data set. It is calculated by subtracting the smallest value from the largest value.

Disease Rates and Their Interpretation

Disease rates are a measure of the occurrence of a disease in a population. They are used to establish causation, monitor interventions, and measure the impact of exposure on disease rates. The attributable risk is the difference in the rate of disease between the exposed and unexposed groups. It tells us what proportion of deaths in the exposed group were due to the exposure. The relative risk is the risk of an event relative to exposure. It is calculated by dividing the rate of disease in the exposed group by the rate of disease in the unexposed group. A relative risk of 1 means there is no difference between the two groups. A relative risk of <1 means that the event is less likely to occur in the exposed group, while a relative risk of >1 means that the event is more likely to occur in the exposed group. The population attributable risk is the reduction in incidence that would be observed if the population were entirely unexposed. It can be calculated by multiplying the attributable risk by the prevalence of exposure in the population. The attributable proportion is the proportion of the disease that would be eliminated in a population if its disease rate were reduced to that of the unexposed group.

The receiver operating characteristic (ROC) curve is a useful tool for evaluating the diagnostic accuracy of a test in distinguishing between healthy and diseased individuals. It helps to identify the optimal cut-off point between sensitivity and specificity.

Other methods, such as visual inspection of forest plots and Cochran’s Q test, can be used to assess heterogeneity in meta-analysis. Visual inspection of forest plots is a quick and easy method, while Cochran’s Q test is a more formal and widely accepted approach.

For more information on heterogeneity in meta-analysis, further reading is recommended.

The US-UK Diagnostic Project found that the higher rates of schizophrenia in New York were due to diagnostic bias, as US psychiatrists used broader diagnostic criteria. However, the use of standardised clinical interviews and operationalised diagnostic criteria greatly reduced the variability of both incidence and prevalence rates of schizophrenia. This was demonstrated in a study by Sartorius et al. (1986) which examined early manifestations and first-contact incidence of schizophrenia in different cultures.

The mean weight is 18kg with a standard deviation of 3kg. Three standard deviations below the mean is 9kg and three standard deviations above the mean is 27kg.

Standard Deviation and Standard Error of the Mean

Standard deviation (SD) and standard error of the mean (SEM) are two important statistical measures used to describe data. SD is a measure of how much the data varies, while SEM is a measure of how precisely we know the true mean of the population. The normal distribution, also known as the Gaussian distribution, is a symmetrical bell-shaped curve that describes the spread of many biological and clinical measurements.

68.3% of the data lies within 1 SD of the mean, 95.4% of the data lies within 2 SD of the mean, and 99.7% of the data lies within 3 SD of the mean. The SD is calculated by taking the square root of the variance and is expressed in the same units as the data set. A low SD indicates that data points tend to be very close to the mean.

On the other hand, SEM is an inferential statistic that quantifies the precision of the mean. It is expressed in the same units as the data and is calculated by dividing the SD of the sample mean by the square root of the sample size. The SEM gets smaller as the sample size increases, and it takes into account both the value of the SD and the sample size.

Both SD and SEM are important measures in statistical analysis, and they are used to calculate confidence intervals and test hypotheses. While SD quantifies scatter, SEM quantifies precision, and both are essential in understanding and interpreting data.

Clinical tests are used to determine the presence of absence of a disease of condition. To interpret test results, it is important to have a working knowledge of statistics used to describe them. Two by two tables are commonly used to calculate test statistics such as sensitivity and specificity. Sensitivity refers to the proportion of people with a condition that the test correctly identifies, while specificity refers to the proportion of people without a condition that the test correctly identifies. Accuracy tells us how closely a test measures to its true value, while predictive values help us understand the likelihood of having a disease based on a positive of negative test result. Likelihood ratios combine sensitivity and specificity into a single figure that can refine our estimation of the probability of a disease being present. Pre and post-test odds and probabilities can also be calculated to better understand the likelihood of having a disease before and after a test is carried out. Fagan’s nomogram is a useful tool for calculating post-test probabilities.

When conducting a literature review, it is important to broaden your search beyond traditional academic sources. This means including grey literature, such as reports, conference proceedings, and government documents. Additionally, it is crucial to consider both primary and secondary sources of evidence, as they can provide different perspectives and insights on your research topic. To ensure a comprehensive review, it is recommended to use multiple databases and search engines, rather than relying on a single source.

Evidence-based medicine involves four basic steps: developing a focused clinical question, searching for the best evidence, critically appraising the evidence, and applying the evidence and evaluating the outcome. When developing a question, it is important to understand the difference between background and foreground questions. Background questions are general questions about conditions, illnesses, syndromes, and pathophysiology, while foreground questions are more often about issues of care. The PICO system is often used to define the components of a foreground question: patient group of interest, intervention of interest, comparison, and primary outcome.

When searching for evidence, it is important to have a basic understanding of the types of evidence and sources of information. Scientific literature is divided into two basic categories: primary (empirical research) and secondary (interpretation and analysis of primary sources). Unfiltered sources are large databases of articles that have not been pre-screened for quality, while filtered resources summarize and appraise evidence from several studies.

There are several databases and search engines that can be used to search for evidence, including Medline and PubMed, Embase, the Cochrane Library, PsycINFO, CINAHL, and OpenGrey. Boolean logic can be used to combine search terms in PubMed, and phrase searching and truncation can also be used. Medical Subject Headings (MeSH) are used by indexers to describe articles for MEDLINE records, and the MeSH Database is like a thesaurus that enables exploration of this vocabulary.

Measures of Disease Frequency: Incidence and Prevalence

Incidence and prevalence are two important measures of disease frequency. Incidence measures the speed at which new cases of a disease are emerging, while prevalence measures the burden of disease within a population. Cumulative incidence and incidence rate are two types of incidence measures, while point prevalence and period prevalence are two types of prevalence measures.

Cumulative incidence is the average risk of getting a disease over a certain period of time, while incidence rate is a measure of the speed at which new cases are emerging. Prevalence is a proportion and is a measure of the burden of disease within a population. Point prevalence measures the number of cases in a defined population at a specific point in time, while period prevalence measures the number of identified cases during a specified period of time.

It is important to note that prevalence is equal to incidence multiplied by the duration of the condition. In chronic diseases, the prevalence is much greater than the incidence. The incidence rate is stated in units of person-time, while cumulative incidence is always a proportion. When describing cumulative incidence, it is necessary to give the follow-up period over which the risk is estimated. In acute diseases, the prevalence and incidence may be similar, while for conditions such as the common cold, the incidence may be greater than the prevalence.

Incidence is a useful measure to study disease etiology and risk factors, while prevalence is useful for health resource planning. Understanding these measures of disease frequency is important for public health professionals and researchers in order to effectively monitor and address the burden of disease within populations.

The prevalence of the disease is 20% as there are currently 200 cases out of a total population of 1000.

Cost utility analysis is a method used in health economics to determine the cost-effectiveness of a health intervention by comparing the cost of the intervention to the benefit it provides in terms of the number of years lived in full health. The cost is measured in monetary units, while the benefit is quantified using a measure that assigns values to different health states, including those that are less desirable than full health. In health technology assessments, this measure is typically expressed as quality-adjusted life years (QALYs).

It is important to note that confidence intervals are derived from standard errors, not standard deviation, despite the common misconception. It is crucial to avoid mixing up these two terms.

Measures of dispersion are used to indicate the variation of spread of a data set, often in conjunction with a measure of central tendency such as the mean of median. The range, which is the difference between the largest and smallest value, is the simplest measure of dispersion. The interquartile range, which is the difference between the 3rd and 1st quartiles, is another useful measure. Quartiles divide a data set into quarters, and the interquartile range can provide additional information about the spread of the data. However, to get a more representative idea of spread, measures such as the variance and standard deviation are needed. The variance gives an indication of how much the items in the data set vary from the mean, while the standard deviation reflects the distribution of individual scores around their mean. The standard deviation is expressed in the same units as the data set and can be used to indicate how confident we are that data points lie within a particular range. The standard error of the mean is an inferential statistic used to estimate the population mean and is a measure of the spread expected for the mean of the observations. Confidence intervals are often presented alongside sample results such as the mean value, indicating a range that is likely to contain the true value.

When a study has good external validity, its findings can be applied to other populations with confidence.

Validity in statistics refers to how accurately something measures what it claims to measure. There are two main types of validity: internal and external. Internal validity refers to the confidence we have in the cause and effect relationship in a study, while external validity refers to the degree to which the conclusions of a study can be applied to other people, places, and times. There are various threats to both internal and external validity, such as sampling, measurement instrument obtrusiveness, and reactive effects of setting. Additionally, there are several subtypes of validity, including face validity, content validity, criterion validity, and construct validity. Each subtype has its own specific focus and methods for testing validity.

Clinical tests are used to determine the presence of absence of a disease of condition. To interpret test results, it is important to have a working knowledge of statistics used to describe them. Two by two tables are commonly used to calculate test statistics such as sensitivity and specificity. Sensitivity refers to the proportion of people with a condition that the test correctly identifies, while specificity refers to the proportion of people without a condition that the test correctly identifies. Accuracy tells us how closely a test measures to its true value, while predictive values help us understand the likelihood of having a disease based on a positive of negative test result. Likelihood ratios combine sensitivity and specificity into a single figure that can refine our estimation of the probability of a disease being present. Pre and post-test odds and probabilities can also be calculated to better understand the likelihood of having a disease before and after a test is carried out. Fagan’s nomogram is a useful tool for calculating post-test probabilities.

Measures of Central Tendency

Measures of central tendency are used in descriptive statistics to summarize the middle of typical value of a data set. There are three common measures of central tendency: the mean, median, and mode.

The median is the middle value in a data set that has been arranged in numerical order. It is not affected by outliers and is used for ordinal data. The mode is the most frequent value in a data set and is used for categorical data. The mean is calculated by adding all the values in a data set and dividing by the number of values. It is sensitive to outliers and is used for interval and ratio data.

The appropriate measure of central tendency depends on the measurement scale of the data. For nominal and categorical data, the mode is used. For ordinal data, the median of mode is used. For interval data with a normal distribution, the mean is preferable, but the median of mode can also be used. For interval data with skewed distribution, the median is used. For ratio data, the mean is preferable, but the median of mode can also be used for skewed data.

In addition to measures of central tendency, the range is also used to describe the spread of a data set. It is calculated by subtracting the smallest value from the largest value.

A t-test is a statistical method used to determine if there is a significant difference between the means of two groups. It is a type of statistical inference.

Types of Statistics: Descriptive and Inferential

Statistics can be divided into two categories: descriptive and inferential. Descriptive statistics are used to describe and summarize data without making any generalizations beyond the data at hand. On the other hand, inferential statistics are used to make inferences about a population based on sample data.

Descriptive statistics are useful for identifying patterns and trends in data. Common measures used to describe a data set include measures of central tendency (such as the mean, median, and mode) and measures of variability of dispersion (such as the standard deviation of variance).

Inferential statistics, on the other hand, are used to make predictions of draw conclusions about a population based on sample data. These statistics are also used to determine the probability that observed differences between groups are reliable and not due to chance.

Overall, both descriptive and inferential statistics play important roles in analyzing and interpreting data. Descriptive statistics help us understand the characteristics of a data set, while inferential statistics allow us to make predictions and draw conclusions about larger populations.

An asymmetric funnel plot may indicate the presence of publication bias, although this is not a definitive confirmation. The x-axis typically represents a measure of effect, such as the risk ratio of odds ratio, although other measures may also be used.

Stats Publication Bias

Publication bias refers to the tendency for studies with positive findings to be published more than studies with negative findings, leading to incomplete data sets in meta-analyses and erroneous conclusions. Graphical methods such as funnel plots, Galbraith plots, ordered forest plots, and normal quantile plots can be used to detect publication bias. Funnel plots are the most commonly used and offer an easy visual way to ensure that published literature is evenly weighted. The x-axis represents the effect size, and the y-axis represents the study size. A symmetrical, inverted funnel shape indicates that publication bias is unlikely, while an asymmetrical funnel indicates a relationship between treatment effect and study size, indicating either publication bias of small study effects.

Measures of Disease Frequency: Incidence and Prevalence

Incidence and prevalence are two important measures of disease frequency. Incidence measures the speed at which new cases of a disease are emerging, while prevalence measures the burden of disease within a population. Cumulative incidence and incidence rate are two types of incidence measures, while point prevalence and period prevalence are two types of prevalence measures.

Cumulative incidence is the average risk of getting a disease over a certain period of time, while incidence rate is a measure of the speed at which new cases are emerging. Prevalence is a proportion and is a measure of the burden of disease within a population. Point prevalence measures the number of cases in a defined population at a specific point in time, while period prevalence measures the number of identified cases during a specified period of time.

It is important to note that prevalence is equal to incidence multiplied by the duration of the condition. In chronic diseases, the prevalence is much greater than the incidence. The incidence rate is stated in units of person-time, while cumulative incidence is always a proportion. When describing cumulative incidence, it is necessary to give the follow-up period over which the risk is estimated. In acute diseases, the prevalence and incidence may be similar, while for conditions such as the common cold, the incidence may be greater than the prevalence.

Incidence is a useful measure to study disease etiology and risk factors, while prevalence is useful for health resource planning. Understanding these measures of disease frequency is important for public health professionals and researchers in order to effectively monitor and address the burden of disease within populations.

Common Biases and Errors in Research

Reverse causality occurs when a risk factor appears to cause an illness, but in reality, it is a consequence of the illness. Information bias is a type of error that can occur in research. Two examples of information bias are observer bias and recall bias. Observer bias happens when the experimenter’s biases affect the study’s findings. Recall bias occurs when participants in the case and control groups have different levels of accuracy in their recollections.

There are two types of errors in research: Type I and Type II. A Type I error is when a true null hypothesis is incorrectly rejected, resulting in a false positive. A Type II error is when a false null hypothesis is not rejected, resulting in a false negative. It is essential to be aware of these biases and errors to ensure accurate and reliable research findings.

To calculate the event rates for the medication and control groups, we divide the number of events by the total number of subjects in each group. For the medication group, the event rate is 0.2 (30/150), and for the control group, it is 0.4 (80/200).

We can also calculate the absolute risk reduction (ARR) by subtracting the event rate in the medication group from the event rate in the control group: ARR = CER – EER = 0.4 – 0.2 = 0.2.

Finally, we can use the ARR to calculate the number needed to treat (NNT), which represents the number of patients who need to be treated with the medication to prevent one additional event compared to the control group. NNT = 1/ARR = 1/0.2 = 5.

The odds ratio is used in case-control studies to measure the association between exposure and outcome, while the relative risk is used in cohort studies to measure the risk of developing an outcome in the exposed group compared to the unexposed group. To convert the odds ratio to a relative risk, one can use the formula: relative risk = odds ratio / (1 – incidence in the unexposed group x odds ratio).

Types of Primary Research Studies and Their Advantages and Disadvantages

Primary research studies can be categorized into six types based on the research question they aim to address. The best type of study for each question type is listed in the table below. There are two main types of study design: experimental and observational. Experimental studies involve an intervention, while observational studies do not. The advantages and disadvantages of each study type are summarized in the table below.

Type of Question Best Type of Study

Therapy Randomized controlled trial (RCT), cohort, case control, case series
Diagnosis Cohort studies with comparison to gold standard test
Prognosis Cohort studies, case control, case series
Etiology/Harm RCT, cohort studies, case control, case series
Prevention RCT, cohort studies, case control, case series
Cost Economic analysis

Study Type Advantages Disadvantages

Randomized Controlled Trial – Unbiased distribution of confounders – Blinding more likely – Randomization facilitates statistical analysis – Expensive – Time-consuming – Volunteer bias – Ethically problematic at times
Cohort Study – Ethically safe – Subjects can be matched – Can establish timing and directionality of events – Eligibility criteria and outcome assessments can be standardized – Administratively easier and cheaper than RCT – Controls may be difficult to identify – Exposure may be linked to a hidden confounder – Blinding is difficult – Randomization not present – For rare disease, large sample sizes of long follow-up necessary
Case-Control Study – Quick and cheap – Only feasible method for very rare disorders of those with long lag between exposure and outcome – Fewer subjects needed than cross-sectional studies – Reliance on recall of records to determine exposure status – Confounders – Selection of control groups is difficult – Potential bias: recall, selection
Cross-Sectional Survey – Cheap and simple – Ethically safe – Establishes association at most, not causality – Recall bias susceptibility – Confounders may be unequally distributed – Neyman bias – Group sizes may be unequal
Ecological Study – Cheap and simple – Ethically safe – Ecological fallacy (when relationships which exist for groups are assumed to also be true for individuals)

In conclusion, the choice of study type depends on the research question being addressed. Each study type has its own advantages and disadvantages, and researchers should carefully consider these when designing their studies.

Measures of dispersion are used to indicate the variation of spread of a data set, often in conjunction with a measure of central tendency such as the mean of median. The range, which is the difference between the largest and smallest value, is the simplest measure of dispersion. The interquartile range, which is the difference between the 3rd and 1st quartiles, is another useful measure. Quartiles divide a data set into quarters, and the interquartile range can provide additional information about the spread of the data. However, to get a more representative idea of spread, measures such as the variance and standard deviation are needed. The variance gives an indication of how much the items in the data set vary from the mean, while the standard deviation reflects the distribution of individual scores around their mean. The standard deviation is expressed in the same units as the data set and can be used to indicate how confident we are that data points lie within a particular range. The standard error of the mean is an inferential statistic used to estimate the population mean and is a measure of the spread expected for the mean of the observations. Confidence intervals are often presented alongside sample results such as the mean value, indicating a range that is likely to contain the true value.

The data gathered will be measured on an ordinal scale, where each answer option is ranked. For instance, 2 is considered lower than 4, and 4 is lower than 5. In an ordinal scale, it is not necessary for the difference between 4 (satisfactory) and 2 (unsatisfactory) to be the same as the difference between 5 (extremely satisfactory) and 3 (neutral). This is because the numbers are not assigned for quantitative measurement but are used for labeling purposes only.

Scales of Measurement in Statistics

In the 1940s, Stanley Smith Stevens introduced four scales of measurement to categorize data variables. Knowing the scale of measurement for a variable is crucial in selecting the appropriate statistical analysis. The four scales of measurement are ratio, interval, ordinal, and nominal.

Ratio scales are similar to interval scales, but they have true zero points. Examples of ratio scales include weight, time, and length. Interval scales measure the difference between two values, and one unit on the scale represents the same magnitude on the trait of characteristic being measured across the whole range of the scale. The Fahrenheit scale for temperature is an example of an interval scale.

Ordinal scales categorize observed values into set categories that can be ordered, but the intervals between each value are uncertain. Examples of ordinal scales include social class, education level, and income level. Nominal scales categorize observed values into set categories that have no particular order of hierarchy. Examples of nominal scales include genotype, blood type, and political party.

Data can also be categorized as quantitative of qualitative. Quantitative variables take on numeric values and can be further classified into discrete and continuous types. Qualitative variables do not take on numerical values and are usually names. Some qualitative variables have an inherent order in their categories and are described as ordinal. Qualitative variables are also called categorical of nominal variables. When a qualitative variable has only two categories, it is called a binary variable.

Measures of Disease Frequency: Incidence and Prevalence

Incidence and prevalence are two important measures of disease frequency. Incidence measures the speed at which new cases of a disease are emerging, while prevalence measures the burden of disease within a population. Cumulative incidence and incidence rate are two types of incidence measures, while point prevalence and period prevalence are two types of prevalence measures.

Cumulative incidence is the average risk of getting a disease over a certain period of time, while incidence rate is a measure of the speed at which new cases are emerging. Prevalence is a proportion and is a measure of the burden of disease within a population. Point prevalence measures the number of cases in a defined population at a specific point in time, while period prevalence measures the number of identified cases during a specified period of time.

It is important to note that prevalence is equal to incidence multiplied by the duration of the condition. In chronic diseases, the prevalence is much greater than the incidence. The incidence rate is stated in units of person-time, while cumulative incidence is always a proportion. When describing cumulative incidence, it is necessary to give the follow-up period over which the risk is estimated. In acute diseases, the prevalence and incidence may be similar, while for conditions such as the common cold, the incidence may be greater than the prevalence.

Incidence is a useful measure to study disease etiology and risk factors, while prevalence is useful for health resource planning. Understanding these measures of disease frequency is important for public health professionals and researchers in order to effectively monitor and address the burden of disease within populations.

The median value is equivalent to Q2 (the second quartile).

Measures of dispersion are used to indicate the variation of spread of a data set, often in conjunction with a measure of central tendency such as the mean of median. The range, which is the difference between the largest and smallest value, is the simplest measure of dispersion. The interquartile range, which is the difference between the 3rd and 1st quartiles, is another useful measure. Quartiles divide a data set into quarters, and the interquartile range can provide additional information about the spread of the data. However, to get a more representative idea of spread, measures such as the variance and standard deviation are needed. The variance gives an indication of how much the items in the data set vary from the mean, while the standard deviation reflects the distribution of individual scores around their mean. The standard deviation is expressed in the same units as the data set and can be used to indicate how confident we are that data points lie within a particular range. The standard error of the mean is an inferential statistic used to estimate the population mean and is a measure of the spread expected for the mean of the observations. Confidence intervals are often presented alongside sample results such as the mean value, indicating a range that is likely to contain the true value.

Cohort studies involve following a group of individuals over a period of time to investigate whether exposure to a particular factor affects disease incidence. Although they are costly and time-consuming, they offer several benefits. For instance, they can examine rare exposure factors and are less prone to recall bias than case-control studies. Additionally, they can measure disease incidence and risk. Results are typically presented as the relative risk of developing the disease due to exposure to the factor.

Negative predictive value = 172 / 177

Clinical tests are used to determine the presence of absence of a disease of condition. To interpret test results, it is important to have a working knowledge of statistics used to describe them. Two by two tables are commonly used to calculate test statistics such as sensitivity and specificity. Sensitivity refers to the proportion of people with a condition that the test correctly identifies, while specificity refers to the proportion of people without a condition that the test correctly identifies. Accuracy tells us how closely a test measures to its true value, while predictive values help us understand the likelihood of having a disease based on a positive of negative test result. Likelihood ratios combine sensitivity and specificity into a single figure that can refine our estimation of the probability of a disease being present. Pre and post-test odds and probabilities can also be calculated to better understand the likelihood of having a disease before and after a test is carried out. Fagan’s nomogram is a useful tool for calculating post-test probabilities.

When investigating prognosis, the hierarchy of study designs starts with a systematic review of cohort studies, followed by a cohort study, follow-up of untreated patients from randomized controlled trials, case series, and expert opinion. The strength of evidence provided by a study depends on its ability to minimize bias and maximize attribution. The Agency for Healthcare Policy and Research hierarchy of study types is widely accepted as reliable, with systematic reviews and meta-analyses of randomized controlled trials at the top, followed by randomized controlled trials, non-randomized intervention studies, observational studies, and non-experimental studies.

Understanding Stats Variables

Variables are characteristics, numbers, of quantities that can be measured of counted. They are also known as data items. Examples of variables include age, sex, business income and expenses, country of birth, capital expenditure, class grades, eye colour, and vehicle type. The value of a variable may vary between data units in a population. In a typical study, there are three main variables: independent, dependent, and controlled variables.

The independent variable is something that the researcher purposely changes during the investigation. The dependent variable is the one that is observed and changes in response to the independent variable. Controlled variables are those that are not changed during the experiment. Dependent variables are affected by independent variables but not by controlled variables, as these do not vary throughout the study.

For instance, a researcher wants to test the effectiveness of a new weight loss medication. Participants are divided into three groups, with the first group receiving a placebo (0mg dosage), the second group a 10 mg dose, and the third group a 40 mg dose. After six months, the participants’ weights are measured. In this case, the independent variable is the dosage of the medication, as that is what is being manipulated. The dependent variable is the weight, as that is what is being measured.

Cost minimisation analysis is employed to compare net costs when the observed effects of health care interventions are similar. To conduct this analysis, it is necessary to have clinical evidence that demonstrates the differences in health effects between alternatives are negligible of insignificant. This approach is commonly used by institutions like the National Institute for Health and Care Excellence (NICE).

In order to make assertions about causation, strong internal validity is necessary.

Validity in statistics refers to how accurately something measures what it claims to measure. There are two main types of validity: internal and external. Internal validity refers to the confidence we have in the cause and effect relationship in a study, while external validity refers to the degree to which the conclusions of a study can be applied to other people, places, and times. There are various threats to both internal and external validity, such as sampling, measurement instrument obtrusiveness, and reactive effects of setting. Additionally, there are several subtypes of validity, including face validity, content validity, criterion validity, and construct validity. Each subtype has its own specific focus and methods for testing validity.

A test that seems to measure what it is intended to measure has strong face validity.

Validity in statistics refers to how accurately something measures what it claims to measure. There are two main types of validity: internal and external. Internal validity refers to the confidence we have in the cause and effect relationship in a study, while external validity refers to the degree to which the conclusions of a study can be applied to other people, places, and times. There are various threats to both internal and external validity, such as sampling, measurement instrument obtrusiveness, and reactive effects of setting. Additionally, there are several subtypes of validity, including face validity, content validity, criterion validity, and construct validity. Each subtype has its own specific focus and methods for testing validity.

Understanding Hypothesis Testing in Statistics

In statistics, it is not feasible to investigate hypotheses on entire populations. Therefore, researchers take samples and use them to make estimates about the population they are drawn from. However, this leads to uncertainty as there is no guarantee that the sample taken will be truly representative of the population, resulting in potential errors. Statistical hypothesis testing is the process used to determine if claims from samples to populations can be made and with what certainty.

The null hypothesis (Ho) is the claim that there is no real difference between two groups, while the alternative hypothesis (H1 of Ha) suggests that any difference is due to some non-random chance. The alternative hypothesis can be one-tailed of two-tailed, depending on whether it seeks to establish a difference of a change in one direction.

Two types of errors may occur when testing the null hypothesis: Type I and Type II errors. Type I error occurs when the null hypothesis is rejected when it is true, while Type II error occurs when the null hypothesis is accepted when it is false. The power of a study is the probability of correctly rejecting the null hypothesis when it is false, and it can be increased by increasing the sample size.

P-values provide information on statistical significance and help researchers decide if study results have occurred due to chance. The p-value is the probability of obtaining a result that is as large of larger when in reality there is no difference between two groups. The cutoff for the p-value is called the significance level (alpha level), typically set at 0.05. If the p-value is less than the cutoff, the null hypothesis is rejected, and if it is greater or equal to the cut off, the null hypothesis is not rejected. However, the p-value does not indicate clinical significance, which may be too small to be meaningful.

Kappa is used to assess the consistency of reliability between different raters.

Understanding the Kappa Statistic for Measuring Interobserver Variation

The kappa statistic, also known as Cohen’s kappa coefficient, is a useful tool for quantifying the level of agreement between independent observers. This measure can be applied in any situation where multiple observers are evaluating the same thing, such as in medical diagnoses of research studies. The kappa coefficient ranges from 0 to 1, with 0 indicating complete disagreement and 1 indicating perfect agreement. By using the kappa statistic, researchers and practitioners can gain insight into the level of interobserver variation present in their data, which can help to improve the accuracy and reliability of their findings. Overall, the kappa statistic is a valuable tool for understanding and measuring interobserver variation in a variety of contexts.

Evidence-based medicine involves four basic steps: developing a focused clinical question, searching for the best evidence, critically appraising the evidence, and applying the evidence and evaluating the outcome. When developing a question, it is important to understand the difference between background and foreground questions. Background questions are general questions about conditions, illnesses, syndromes, and pathophysiology, while foreground questions are more often about issues of care. The PICO system is often used to define the components of a foreground question: patient group of interest, intervention of interest, comparison, and primary outcome.

When searching for evidence, it is important to have a basic understanding of the types of evidence and sources of information. Scientific literature is divided into two basic categories: primary (empirical research) and secondary (interpretation and analysis of primary sources). Unfiltered sources are large databases of articles that have not been pre-screened for quality, while filtered resources summarize and appraise evidence from several studies.

There are several databases and search engines that can be used to search for evidence, including Medline and PubMed, Embase, the Cochrane Library, PsycINFO, CINAHL, and OpenGrey. Boolean logic can be used to combine search terms in PubMed, and phrase searching and truncation can also be used. Medical Subject Headings (MeSH) are used by indexers to describe articles for MEDLINE records, and the MeSH Database is like a thesaurus that enables exploration of this vocabulary.

To address the issue of drop-outs in a study, an intention to treat (ITT) analysis can be employed. Drop-outs can lead to attrition bias, which creates systematic differences in attrition across treatment groups. In an ITT analysis, all patients are included in the groups they were initially assigned to through random allocation. To handle missing data, two common methods are last observation carried forward and worst case scenario analysis.

A one-tailed hypothesis indicates a specific direction of association between groups. The researcher not only declares that there will be a distinction between the groups but also defines the direction in which the difference will occur.

Validity in statistics refers to how accurately something measures what it claims to measure. There are two main types of validity: internal and external. Internal validity refers to the confidence we have in the cause and effect relationship in a study, while external validity refers to the degree to which the conclusions of a study can be applied to other people, places, and times. There are various threats to both internal and external validity, such as sampling, measurement instrument obtrusiveness, and reactive effects of setting. Additionally, there are several subtypes of validity, including face validity, content validity, criterion validity, and construct validity. Each subtype has its own specific focus and methods for testing validity.

Types of Primary Research Studies and Their Advantages and Disadvantages

Primary research studies can be categorized into six types based on the research question they aim to address. The best type of study for each question type is listed in the table below. There are two main types of study design: experimental and observational. Experimental studies involve an intervention, while observational studies do not. The advantages and disadvantages of each study type are summarized in the table below.

Type of Question Best Type of Study

Therapy Randomized controlled trial (RCT), cohort, case control, case series
Diagnosis Cohort studies with comparison to gold standard test
Prognosis Cohort studies, case control, case series
Etiology/Harm RCT, cohort studies, case control, case series
Prevention RCT, cohort studies, case control, case series
Cost Economic analysis

Study Type Advantages Disadvantages

Randomized Controlled Trial – Unbiased distribution of confounders – Blinding more likely – Randomization facilitates statistical analysis – Expensive – Time-consuming – Volunteer bias – Ethically problematic at times
Cohort Study – Ethically safe – Subjects can be matched – Can establish timing and directionality of events – Eligibility criteria and outcome assessments can be standardized – Administratively easier and cheaper than RCT – Controls may be difficult to identify – Exposure may be linked to a hidden confounder – Blinding is difficult – Randomization not present – For rare disease, large sample sizes of long follow-up necessary
Case-Control Study – Quick and cheap – Only feasible method for very rare disorders of those with long lag between exposure and outcome – Fewer subjects needed than cross-sectional studies – Reliance on recall of records to determine exposure status – Confounders – Selection of control groups is difficult – Potential bias: recall, selection
Cross-Sectional Survey – Cheap and simple – Ethically safe – Establishes association at most, not causality – Recall bias susceptibility – Confounders may be unequally distributed – Neyman bias – Group sizes may be unequal
Ecological Study – Cheap and simple – Ethically safe – Ecological fallacy (when relationships which exist for groups are assumed to also be true for individuals)

In conclusion, the choice of study type depends on the research question being addressed. Each study type has its own advantages and disadvantages, and researchers should carefully consider these when designing their studies.

Types of Bias in Statistics

Bias is a systematic error that can lead to incorrect conclusions. Confounding factors are variables that are associated with both the outcome and the exposure but have no causative role. Confounding can be addressed in the design and analysis stage of a study. The main method of controlling confounding in the analysis phase is stratification analysis. The main methods used in the design stage are matching, randomization, and restriction of participants.

There are two main types of bias: selection bias and information bias. Selection bias occurs when the selected sample is not a representative sample of the reference population. Disease spectrum bias, self-selection bias, participation bias, incidence-prevalence bias, exclusion bias, publication of dissemination bias, citation bias, and Berkson’s bias are all subtypes of selection bias. Information bias occurs when gathered information about exposure, outcome, of both is not correct and there was an error in measurement. Detection bias, recall bias, lead time bias, interviewer/observer bias, verification and work-up bias, Hawthorne effect, and ecological fallacy are all subtypes of information bias.

To ensure validity in qualitative studies, a technique called member checking of respondent validation is used. This involves interviewing a subset of the participants (typically around 11) to confirm that their perspectives align with the study’s findings.

Qualitative research is a method of inquiry that seeks to understand the meaning and experience dimensions of human lives and social worlds. There are different approaches to qualitative research, such as ethnography, phenomenology, and grounded theory, each with its own purpose, role of the researcher, stages of research, and method of data analysis. The most common methods used in healthcare research are interviews and focus groups. Sampling techniques include convenience sampling, purposive sampling, quota sampling, snowball sampling, and case study sampling. Sample size can be determined by data saturation, which occurs when new categories, themes, of explanations stop emerging from the data. Validity can be assessed through triangulation, respondent validation, bracketing, and reflexivity. Analytical approaches include content analysis and constant comparison.

The chi-squared test is a statistical test that does not rely on any assumptions about the underlying distribution of the data, making it a non-parametric test.

Choosing the right statistical test can be challenging, but understanding the basic principles can help. Different tests have different assumptions, and using the wrong one can lead to inaccurate results. To identify the appropriate test, a flow chart can be used based on three main factors: the type of dependent variable, the type of data, and whether the groups/samples are independent of dependent. It is important to know which tests are parametric and non-parametric, as well as their alternatives. For example, the chi-squared test is used to assess differences in categorical variables and is non-parametric, while Pearson’s correlation coefficient measures linear correlation between two variables and is parametric. T-tests are used to compare means between two groups, and ANOVA is used to compare means between more than two groups. Non-parametric equivalents to ANOVA include the Kruskal-Wallis analysis of ranks, the Median test, Friedman’s two-way analysis of variance, and Cochran Q test. Understanding these tests and their assumptions can help researchers choose the appropriate statistical test for their data.

Measures of dispersion are used to indicate the variation of spread of a data set, often in conjunction with a measure of central tendency such as the mean of median. The range, which is the difference between the largest and smallest value, is the simplest measure of dispersion. The interquartile range, which is the difference between the 3rd and 1st quartiles, is another useful measure. Quartiles divide a data set into quarters, and the interquartile range can provide additional information about the spread of the data. However, to get a more representative idea of spread, measures such as the variance and standard deviation are needed. The variance gives an indication of how much the items in the data set vary from the mean, while the standard deviation reflects the distribution of individual scores around their mean. The standard deviation is expressed in the same units as the data set and can be used to indicate how confident we are that data points lie within a particular range. The standard error of the mean is an inferential statistic used to estimate the population mean and is a measure of the spread expected for the mean of the observations. Confidence intervals are often presented alongside sample results such as the mean value, indicating a range that is likely to contain the true value.

If the p-value is lower than the predetermined alpha level of 0.05, the outcome is considered significant.

Understanding Hypothesis Testing in Statistics

In statistics, it is not feasible to investigate hypotheses on entire populations. Therefore, researchers take samples and use them to make estimates about the population they are drawn from. However, this leads to uncertainty as there is no guarantee that the sample taken will be truly representative of the population, resulting in potential errors. Statistical hypothesis testing is the process used to determine if claims from samples to populations can be made and with what certainty.

The null hypothesis (Ho) is the claim that there is no real difference between two groups, while the alternative hypothesis (H1 of Ha) suggests that any difference is due to some non-random chance. The alternative hypothesis can be one-tailed of two-tailed, depending on whether it seeks to establish a difference of a change in one direction.

Two types of errors may occur when testing the null hypothesis: Type I and Type II errors. Type I error occurs when the null hypothesis is rejected when it is true, while Type II error occurs when the null hypothesis is accepted when it is false. The power of a study is the probability of correctly rejecting the null hypothesis when it is false, and it can be increased by increasing the sample size.

P-values provide information on statistical significance and help researchers decide if study results have occurred due to chance. The p-value is the probability of obtaining a result that is as large of larger when in reality there is no difference between two groups. The cutoff for the p-value is called the significance level (alpha level), typically set at 0.05. If the p-value is less than the cutoff, the null hypothesis is rejected, and if it is greater or equal to the cut off, the null hypothesis is not rejected. However, the p-value does not indicate clinical significance, which may be too small to be meaningful.

Qualitative research is a method of inquiry that seeks to understand the meaning and experience dimensions of human lives and social worlds. There are different approaches to qualitative research, such as ethnography, phenomenology, and grounded theory, each with its own purpose, role of the researcher, stages of research, and method of data analysis. The most common methods used in healthcare research are interviews and focus groups. Sampling techniques include convenience sampling, purposive sampling, quota sampling, snowball sampling, and case study sampling. Sample size can be determined by data saturation, which occurs when new categories, themes, of explanations stop emerging from the data. Validity can be assessed through triangulation, respondent validation, bracketing, and reflexivity. Analytical approaches include content analysis and constant comparison.

SIGLE is a database that specializes in collecting and indexing grey literature.

Evidence-based medicine involves four basic steps: developing a focused clinical question, searching for the best evidence, critically appraising the evidence, and applying the evidence and evaluating the outcome. When developing a question, it is important to understand the difference between background and foreground questions. Background questions are general questions about conditions, illnesses, syndromes, and pathophysiology, while foreground questions are more often about issues of care. The PICO system is often used to define the components of a foreground question: patient group of interest, intervention of interest, comparison, and primary outcome.

When searching for evidence, it is important to have a basic understanding of the types of evidence and sources of information. Scientific literature is divided into two basic categories: primary (empirical research) and secondary (interpretation and analysis of primary sources). Unfiltered sources are large databases of articles that have not been pre-screened for quality, while filtered resources summarize and appraise evidence from several studies.

There are several databases and search engines that can be used to search for evidence, including Medline and PubMed, Embase, the Cochrane Library, PsycINFO, CINAHL, and OpenGrey. Boolean logic can be used to combine search terms in PubMed, and phrase searching and truncation can also be used. Medical Subject Headings (MeSH) are used by indexers to describe articles for MEDLINE records, and the MeSH Database is like a thesaurus that enables exploration of this vocabulary.

Measures of Central Tendency

Measures of central tendency are used in descriptive statistics to summarize the middle of typical value of a data set. There are three common measures of central tendency: the mean, median, and mode.

The median is the middle value in a data set that has been arranged in numerical order. It is not affected by outliers and is used for ordinal data. The mode is the most frequent value in a data set and is used for categorical data. The mean is calculated by adding all the values in a data set and dividing by the number of values. It is sensitive to outliers and is used for interval and ratio data.

The appropriate measure of central tendency depends on the measurement scale of the data. For nominal and categorical data, the mode is used. For ordinal data, the median of mode is used. For interval data with a normal distribution, the mean is preferable, but the median of mode can also be used. For interval data with skewed distribution, the median is used. For ratio data, the mean is preferable, but the median of mode can also be used for skewed data.

In addition to measures of central tendency, the range is also used to describe the spread of a data set. It is calculated by subtracting the smallest value from the largest value.

T-tests are appropriate for parametric data, which means that the data should conform to a normal distribution.

Choosing the right statistical test can be challenging, but understanding the basic principles can help. Different tests have different assumptions, and using the wrong one can lead to inaccurate results. To identify the appropriate test, a flow chart can be used based on three main factors: the type of dependent variable, the type of data, and whether the groups/samples are independent of dependent. It is important to know which tests are parametric and non-parametric, as well as their alternatives. For example, the chi-squared test is used to assess differences in categorical variables and is non-parametric, while Pearson’s correlation coefficient measures linear correlation between two variables and is parametric. T-tests are used to compare means between two groups, and ANOVA is used to compare means between more than two groups. Non-parametric equivalents to ANOVA include the Kruskal-Wallis analysis of ranks, the Median test, Friedman’s two-way analysis of variance, and Cochran Q test. Understanding these tests and their assumptions can help researchers choose the appropriate statistical test for their data.

Measures of Effect in Clinical Studies

When conducting clinical studies, we often want to know the effect of treatments of exposures on health outcomes. Measures of effect are used in randomized controlled trials (RCTs) and include the odds ratio (of), risk ratio (RR), risk difference (RD), and number needed to treat (NNT). Dichotomous (binary) outcome data are common in clinical trials, where the outcome for each participant is one of two possibilities, such as dead of alive, of clinical improvement of no improvement.

To understand the difference between of and RR, it’s important to know the difference between risks and odds. Risk is a proportion that describes the probability of a health outcome occurring, while odds is a ratio that compares the probability of an event occurring to the probability of it not occurring. Absolute risk is the basic risk, while risk difference is the difference between the absolute risk of an event in the intervention group and the absolute risk in the control group. Relative risk is the ratio of risk in the intervention group to the risk in the control group.

The number needed to treat (NNT) is the number of patients who need to be treated for one to benefit. Odds are calculated by dividing the number of times an event happens by the number of times it does not happen. The odds ratio is the odds of an outcome given a particular exposure versus the odds of an outcome in the absence of the exposure. It is commonly used in case-control studies and can also be used in cross-sectional and cohort study designs. An odds ratio of 1 indicates no difference in risk between the two groups, while an odds ratio >1 indicates an increased risk and an odds ratio <1 indicates a reduced risk.

Methods of Economic Evaluation

There are four main methods of economic evaluation: cost-effectiveness analysis (CEA), cost-benefit analysis (CBA), cost-utility analysis (CUA), and cost-minimisation analysis (CMA). While all four methods capture costs, they differ in how they assess health effects.

Cost-effectiveness analysis (CEA) compares interventions by relating costs to a single clinical measure of effectiveness, such as symptom reduction of improvement in activities of daily living. The cost-effectiveness ratio is calculated as total cost divided by units of effectiveness. CEA is typically used when CBA cannot be performed due to the inability to monetise benefits.

Cost-benefit analysis (CBA) measures all costs and benefits of an intervention in monetary terms to establish which alternative has the greatest net benefit. CBA requires that all consequences of an intervention, such as life-years saved, treatment side-effects, symptom relief, disability, pain, and discomfort, are allocated a monetary value. CBA is rarely used in mental health service evaluation due to the difficulty in converting benefits from mental health programmes into monetary values.

Cost-utility analysis (CUA) is a special form of CEA in which health benefits/outcomes are measured in broader, more generic ways, enabling comparisons between treatments for different diseases and conditions. Multidimensional health outcomes are measured by a single preference- of utility-based index such as the Quality-Adjusted-Life-Years (QALY). QALYs are a composite measure of gains in life expectancy and health-related quality of life. CUA allows for comparisons across treatments for different conditions.

Cost-minimisation analysis (CMA) is an economic evaluation in which the consequences of competing interventions are the same, and only inputs, i.e. costs, are taken into consideration. The aim is to decide the least costly way of achieving the same outcome.

Costs in Economic Evaluation Studies

There are three main types of costs in economic evaluation studies: direct, indirect, and intangible. Direct costs are associated directly with the healthcare intervention, such as staff time, medical supplies, cost of travel for the patient, childcare costs for the patient, and costs falling on other social sectors such as domestic help from social services. Indirect costs are incurred by the reduced productivity of the patient, such as time off work, reduced work productivity, and time spent caring for the patient by relatives. Intangible costs are difficult to measure, such as pain of suffering on the part of the patient.

Scientific literature can be classified into two main types: primary and secondary sources. Primary sources are original research studies that present data and analysis without any external evaluation of interpretation. Examples of primary sources include randomized controlled trials, cohort studies, case-control studies, case-series, and conference papers. Secondary sources, on the other hand, provide an interpretation and analysis of primary sources. These sources are typically removed by one of more steps from the original event. Examples of secondary sources include evidence-based guidelines and textbooks, meta-analyses, and systematic reviews.

Evidence-based medicine involves four basic steps: developing a focused clinical question, searching for the best evidence, critically appraising the evidence, and applying the evidence and evaluating the outcome. When developing a question, it is important to understand the difference between background and foreground questions. Background questions are general questions about conditions, illnesses, syndromes, and pathophysiology, while foreground questions are more often about issues of care. The PICO system is often used to define the components of a foreground question: patient group of interest, intervention of interest, comparison, and primary outcome.

When searching for evidence, it is important to have a basic understanding of the types of evidence and sources of information. Scientific literature is divided into two basic categories: primary (empirical research) and secondary (interpretation and analysis of primary sources). Unfiltered sources are large databases of articles that have not been pre-screened for quality, while filtered resources summarize and appraise evidence from several studies.

There are several databases and search engines that can be used to search for evidence, including Medline and PubMed, Embase, the Cochrane Library, PsycINFO, CINAHL, and OpenGrey. Boolean logic can be used to combine search terms in PubMed, and phrase searching and truncation can also be used. Medical Subject Headings (MeSH) are used by indexers to describe articles for MEDLINE records, and the MeSH Database is like a thesaurus that enables exploration of this vocabulary.

Clinical tests are used to determine the presence of absence of a disease of condition. To interpret test results, it is important to have a working knowledge of statistics used to describe them. Two by two tables are commonly used to calculate test statistics such as sensitivity and specificity. Sensitivity refers to the proportion of people with a condition that the test correctly identifies, while specificity refers to the proportion of people without a condition that the test correctly identifies. Accuracy tells us how closely a test measures to its true value, while predictive values help us understand the likelihood of having a disease based on a positive of negative test result. Likelihood ratios combine sensitivity and specificity into a single figure that can refine our estimation of the probability of a disease being present. Pre and post-test odds and probabilities can also be calculated to better understand the likelihood of having a disease before and after a test is carried out. Fagan’s nomogram is a useful tool for calculating post-test probabilities.

Measures of Disease Frequency: Incidence and Prevalence

Incidence and prevalence are two important measures of disease frequency. Incidence measures the speed at which new cases of a disease are emerging, while prevalence measures the burden of disease within a population. Cumulative incidence and incidence rate are two types of incidence measures, while point prevalence and period prevalence are two types of prevalence measures.

Cumulative incidence is the average risk of getting a disease over a certain period of time, while incidence rate is a measure of the speed at which new cases are emerging. Prevalence is a proportion and is a measure of the burden of disease within a population. Point prevalence measures the number of cases in a defined population at a specific point in time, while period prevalence measures the number of identified cases during a specified period of time.

It is important to note that prevalence is equal to incidence multiplied by the duration of the condition. In chronic diseases, the prevalence is much greater than the incidence. The incidence rate is stated in units of person-time, while cumulative incidence is always a proportion. When describing cumulative incidence, it is necessary to give the follow-up period over which the risk is estimated. In acute diseases, the prevalence and incidence may be similar, while for conditions such as the common cold, the incidence may be greater than the prevalence.

Incidence is a useful measure to study disease etiology and risk factors, while prevalence is useful for health resource planning. Understanding these measures of disease frequency is important for public health professionals and researchers in order to effectively monitor and address the burden of disease within populations.

The Kaplan-Meier plot is the most effective graphical representation of survival probability. It presents the overall likelihood of an individual’s survival over time from a baseline, and the comparison of two lines on the plot can indicate whether there is a survival advantage. To determine if the distinction between the two groups is significant, a log rank test can be employed.

– Treating 1000 patients with a new statin for five years prevented 50 MIs.
– The number needed to treat (NNT) to prevent one MI is 20 (1000/50).
– NNT provides information on treatment efficacy beyond statistical significance.
– Based on these data, treating as few as 20 patients over five years may prevent an infarct.
– Cost economic data can be calculated by factoring in drug costs and costs of treating and rehabilitating a patient with an MI.

The most effective way to eliminate of manage potential confounding factors is to randomize a large enough sample size. This approach addresses all potential confounders, regardless of whether they were measured in the study design. Matching involves pairing individuals who received a treatment of intervention with non-treated individuals who have similar observable characteristics. Post-hoc methods, such as stratification, regression analysis, and analysis of variance, can be used to evaluate the impact of known or suspected confounders.

Clinical tests are used to determine the presence of absence of a disease of condition. To interpret test results, it is important to have a working knowledge of statistics used to describe them. Two by two tables are commonly used to calculate test statistics such as sensitivity and specificity. Sensitivity refers to the proportion of people with a condition that the test correctly identifies, while specificity refers to the proportion of people without a condition that the test correctly identifies. Accuracy tells us how closely a test measures to its true value, while predictive values help us understand the likelihood of having a disease based on a positive of negative test result. Likelihood ratios combine sensitivity and specificity into a single figure that can refine our estimation of the probability of a disease being present. Pre and post-test odds and probabilities can also be calculated to better understand the likelihood of having a disease before and after a test is carried out. Fagan’s nomogram is a useful tool for calculating post-test probabilities.

An NNT of 1 indicates that every patient who receives the treatment experiences a positive outcome, while no patient in the control group experiences the same outcome. This represents an ideal outcome.

Measures of Effect in Clinical Studies

When conducting clinical studies, we often want to know the effect of treatments of exposures on health outcomes. Measures of effect are used in randomized controlled trials (RCTs) and include the odds ratio (of), risk ratio (RR), risk difference (RD), and number needed to treat (NNT). Dichotomous (binary) outcome data are common in clinical trials, where the outcome for each participant is one of two possibilities, such as dead of alive, of clinical improvement of no improvement.

To understand the difference between of and RR, it’s important to know the difference between risks and odds. Risk is a proportion that describes the probability of a health outcome occurring, while odds is a ratio that compares the probability of an event occurring to the probability of it not occurring. Absolute risk is the basic risk, while risk difference is the difference between the absolute risk of an event in the intervention group and the absolute risk in the control group. Relative risk is the ratio of risk in the intervention group to the risk in the control group.

The number needed to treat (NNT) is the number of patients who need to be treated for one to benefit. Odds are calculated by dividing the number of times an event happens by the number of times it does not happen. The odds ratio is the odds of an outcome given a particular exposure versus the odds of an outcome in the absence of the exposure. It is commonly used in case-control studies and can also be used in cross-sectional and cohort study designs. An odds ratio of 1 indicates no difference in risk between the two groups, while an odds ratio >1 indicates an increased risk and an odds ratio <1 indicates a reduced risk.

Measures of Effect in Clinical Studies

When conducting clinical studies, we often want to know the effect of treatments of exposures on health outcomes. Measures of effect are used in randomized controlled trials (RCTs) and include the odds ratio (of), risk ratio (RR), risk difference (RD), and number needed to treat (NNT). Dichotomous (binary) outcome data are common in clinical trials, where the outcome for each participant is one of two possibilities, such as dead of alive, of clinical improvement of no improvement.

To understand the difference between of and RR, it’s important to know the difference between risks and odds. Risk is a proportion that describes the probability of a health outcome occurring, while odds is a ratio that compares the probability of an event occurring to the probability of it not occurring. Absolute risk is the basic risk, while risk difference is the difference between the absolute risk of an event in the intervention group and the absolute risk in the control group. Relative risk is the ratio of risk in the intervention group to the risk in the control group.

The number needed to treat (NNT) is the number of patients who need to be treated for one to benefit. Odds are calculated by dividing the number of times an event happens by the number of times it does not happen. The odds ratio is the odds of an outcome given a particular exposure versus the odds of an outcome in the absence of the exposure. It is commonly used in case-control studies and can also be used in cross-sectional and cohort study designs. An odds ratio of 1 indicates no difference in risk between the two groups, while an odds ratio >1 indicates an increased risk and an odds ratio <1 indicates a reduced risk.

The prevalence refers to the percentage of individuals in a population who currently have a particular condition, while the incidence is the frequency at which new cases of the condition arise within a specific timeframe.

Clinical tests are used to determine the presence of absence of a disease of condition. To interpret test results, it is important to have a working knowledge of statistics used to describe them. Two by two tables are commonly used to calculate test statistics such as sensitivity and specificity. Sensitivity refers to the proportion of people with a condition that the test correctly identifies, while specificity refers to the proportion of people without a condition that the test correctly identifies. Accuracy tells us how closely a test measures to its true value, while predictive values help us understand the likelihood of having a disease based on a positive of negative test result. Likelihood ratios combine sensitivity and specificity into a single figure that can refine our estimation of the probability of a disease being present. Pre and post-test odds and probabilities can also be calculated to better understand the likelihood of having a disease before and after a test is carried out. Fagan’s nomogram is a useful tool for calculating post-test probabilities.

Age is a type of measurement that follows a ratio scale, which means that the values can be compared as multiples of each other. For instance, if someone is 20 years old, they are twice as old as someone who is 10 years old.

Scales of Measurement in Statistics

In the 1940s, Stanley Smith Stevens introduced four scales of measurement to categorize data variables. Knowing the scale of measurement for a variable is crucial in selecting the appropriate statistical analysis. The four scales of measurement are ratio, interval, ordinal, and nominal.

Ratio scales are similar to interval scales, but they have true zero points. Examples of ratio scales include weight, time, and length. Interval scales measure the difference between two values, and one unit on the scale represents the same magnitude on the trait of characteristic being measured across the whole range of the scale. The Fahrenheit scale for temperature is an example of an interval scale.

Ordinal scales categorize observed values into set categories that can be ordered, but the intervals between each value are uncertain. Examples of ordinal scales include social class, education level, and income level. Nominal scales categorize observed values into set categories that have no particular order of hierarchy. Examples of nominal scales include genotype, blood type, and political party.

Data can also be categorized as quantitative of qualitative. Quantitative variables take on numeric values and can be further classified into discrete and continuous types. Qualitative variables do not take on numerical values and are usually names. Some qualitative variables have an inherent order in their categories and are described as ordinal. Qualitative variables are also called categorical of nominal variables. When a qualitative variable has only two categories, it is called a binary variable.

The α value, also known as the type I error, is the predetermined probability that is considered acceptable for making an error. If the P value is lower than the predetermined α value, then the null hypothesis (Ho) is rejected, and it is concluded that the observed difference, association, of correlation is statistically significant.

Understanding Hypothesis Testing in Statistics

In statistics, it is not feasible to investigate hypotheses on entire populations. Therefore, researchers take samples and use them to make estimates about the population they are drawn from. However, this leads to uncertainty as there is no guarantee that the sample taken will be truly representative of the population, resulting in potential errors. Statistical hypothesis testing is the process used to determine if claims from samples to populations can be made and with what certainty.

The null hypothesis (Ho) is the claim that there is no real difference between two groups, while the alternative hypothesis (H1 of Ha) suggests that any difference is due to some non-random chance. The alternative hypothesis can be one-tailed of two-tailed, depending on whether it seeks to establish a difference of a change in one direction.

Two types of errors may occur when testing the null hypothesis: Type I and Type II errors. Type I error occurs when the null hypothesis is rejected when it is true, while Type II error occurs when the null hypothesis is accepted when it is false. The power of a study is the probability of correctly rejecting the null hypothesis when it is false, and it can be increased by increasing the sample size.

P-values provide information on statistical significance and help researchers decide if study results have occurred due to chance. The p-value is the probability of obtaining a result that is as large of larger when in reality there is no difference between two groups. The cutoff for the p-value is called the significance level (alpha level), typically set at 0.05. If the p-value is less than the cutoff, the null hypothesis is rejected, and if it is greater or equal to the cut off, the null hypothesis is not rejected. However, the p-value does not indicate clinical significance, which may be too small to be meaningful.

To determine the number needed to treat (NNT), we first calculated the absolute risk reduction (ARR) using the formula ARR = CER – EER, where CER is the control event rate and EER is the experimental event rate. In this case, the ARR was 0.47, which is the reciprocal of the NNT. Therefore, the NNT was calculated as 2.12. This means that for every two patients treated with the active medication, at least one patient will have a better outcome compared to those treated with a placebo.

Measures of Effect in Clinical Studies

When conducting clinical studies, we often want to know the effect of treatments of exposures on health outcomes. Measures of effect are used in randomized controlled trials (RCTs) and include the odds ratio (of), risk ratio (RR), risk difference (RD), and number needed to treat (NNT). Dichotomous (binary) outcome data are common in clinical trials, where the outcome for each participant is one of two possibilities, such as dead of alive, of clinical improvement of no improvement.

To understand the difference between of and RR, it’s important to know the difference between risks and odds. Risk is a proportion that describes the probability of a health outcome occurring, while odds is a ratio that compares the probability of an event occurring to the probability of it not occurring. Absolute risk is the basic risk, while risk difference is the difference between the absolute risk of an event in the intervention group and the absolute risk in the control group. Relative risk is the ratio of risk in the intervention group to the risk in the control group.

The number needed to treat (NNT) is the number of patients who need to be treated for one to benefit. Odds are calculated by dividing the number of times an event happens by the number of times it does not happen. The odds ratio is the odds of an outcome given a particular exposure versus the odds of an outcome in the absence of the exposure. It is commonly used in case-control studies and can also be used in cross-sectional and cohort study designs. An odds ratio of 1 indicates no difference in risk between the two groups, while an odds ratio >1 indicates an increased risk and an odds ratio <1 indicates a reduced risk.

Measures of dispersion are used to indicate the variation of spread of a data set, often in conjunction with a measure of central tendency such as the mean of median. The range, which is the difference between the largest and smallest value, is the simplest measure of dispersion. The interquartile range, which is the difference between the 3rd and 1st quartiles, is another useful measure. Quartiles divide a data set into quarters, and the interquartile range can provide additional information about the spread of the data. However, to get a more representative idea of spread, measures such as the variance and standard deviation are needed. The variance gives an indication of how much the items in the data set vary from the mean, while the standard deviation reflects the distribution of individual scores around their mean. The standard deviation is expressed in the same units as the data set and can be used to indicate how confident we are that data points lie within a particular range. The standard error of the mean is an inferential statistic used to estimate the population mean and is a measure of the spread expected for the mean of the observations. Confidence intervals are often presented alongside sample results such as the mean value, indicating a range that is likely to contain the true value.

Measures of Effect in Clinical Studies

When conducting clinical studies, we often want to know the effect of treatments of exposures on health outcomes. Measures of effect are used in randomized controlled trials (RCTs) and include the odds ratio (of), risk ratio (RR), risk difference (RD), and number needed to treat (NNT). Dichotomous (binary) outcome data are common in clinical trials, where the outcome for each participant is one of two possibilities, such as dead of alive, of clinical improvement of no improvement.

To understand the difference between of and RR, it’s important to know the difference between risks and odds. Risk is a proportion that describes the probability of a health outcome occurring, while odds is a ratio that compares the probability of an event occurring to the probability of it not occurring. Absolute risk is the basic risk, while risk difference is the difference between the absolute risk of an event in the intervention group and the absolute risk in the control group. Relative risk is the ratio of risk in the intervention group to the risk in the control group.

The number needed to treat (NNT) is the number of patients who need to be treated for one to benefit. Odds are calculated by dividing the number of times an event happens by the number of times it does not happen. The odds ratio is the odds of an outcome given a particular exposure versus the odds of an outcome in the absence of the exposure. It is commonly used in case-control studies and can also be used in cross-sectional and cohort study designs. An odds ratio of 1 indicates no difference in risk between the two groups, while an odds ratio >1 indicates an increased risk and an odds ratio <1 indicates a reduced risk.

Disease Rates and Their Interpretation

Disease rates are a measure of the occurrence of a disease in a population. They are used to establish causation, monitor interventions, and measure the impact of exposure on disease rates. The attributable risk is the difference in the rate of disease between the exposed and unexposed groups. It tells us what proportion of deaths in the exposed group were due to the exposure. The relative risk is the risk of an event relative to exposure. It is calculated by dividing the rate of disease in the exposed group by the rate of disease in the unexposed group. A relative risk of 1 means there is no difference between the two groups. A relative risk of <1 means that the event is less likely to occur in the exposed group, while a relative risk of >1 means that the event is more likely to occur in the exposed group. The population attributable risk is the reduction in incidence that would be observed if the population were entirely unexposed. It can be calculated by multiplying the attributable risk by the prevalence of exposure in the population. The attributable proportion is the proportion of the disease that would be eliminated in a population if its disease rate were reduced to that of the unexposed group.

Stevens suggested that scales can be categorized into one of four types based on measurements.

Scales of Measurement in Statistics

In the 1940s, Stanley Smith Stevens introduced four scales of measurement to categorize data variables. Knowing the scale of measurement for a variable is crucial in selecting the appropriate statistical analysis. The four scales of measurement are ratio, interval, ordinal, and nominal.

Ratio scales are similar to interval scales, but they have true zero points. Examples of ratio scales include weight, time, and length. Interval scales measure the difference between two values, and one unit on the scale represents the same magnitude on the trait of characteristic being measured across the whole range of the scale. The Fahrenheit scale for temperature is an example of an interval scale.

Ordinal scales categorize observed values into set categories that can be ordered, but the intervals between each value are uncertain. Examples of ordinal scales include social class, education level, and income level. Nominal scales categorize observed values into set categories that have no particular order of hierarchy. Examples of nominal scales include genotype, blood type, and political party.

Data can also be categorized as quantitative of qualitative. Quantitative variables take on numeric values and can be further classified into discrete and continuous types. Qualitative variables do not take on numerical values and are usually names. Some qualitative variables have an inherent order in their categories and are described as ordinal. Qualitative variables are also called categorical of nominal variables. When a qualitative variable has only two categories, it is called a binary variable.

While quantitative methods are typically used for treatment evaluation, qualitative studies can also provide valuable insights by interpreting, qualifying, of illuminating findings. This is especially beneficial when examining unexpected results, as they can help to test the primary hypothesis.

Qualitative research is a method of inquiry that seeks to understand the meaning and experience dimensions of human lives and social worlds. There are different approaches to qualitative research, such as ethnography, phenomenology, and grounded theory, each with its own purpose, role of the researcher, stages of research, and method of data analysis. The most common methods used in healthcare research are interviews and focus groups. Sampling techniques include convenience sampling, purposive sampling, quota sampling, snowball sampling, and case study sampling. Sample size can be determined by data saturation, which occurs when new categories, themes, of explanations stop emerging from the data. Validity can be assessed through triangulation, respondent validation, bracketing, and reflexivity. Analytical approaches include content analysis and constant comparison.

Kappa is used to assess the consistency of agreement between different raters.

Understanding the Kappa Statistic for Measuring Interobserver Variation

The kappa statistic, also known as Cohen’s kappa coefficient, is a useful tool for quantifying the level of agreement between independent observers. This measure can be applied in any situation where multiple observers are evaluating the same thing, such as in medical diagnoses of research studies. The kappa coefficient ranges from 0 to 1, with 0 indicating complete disagreement and 1 indicating perfect agreement. By using the kappa statistic, researchers and practitioners can gain insight into the level of interobserver variation present in their data, which can help to improve the accuracy and reliability of their findings. Overall, the kappa statistic is a valuable tool for understanding and measuring interobserver variation in a variety of contexts.

Validity in statistics refers to how accurately something measures what it claims to measure. There are two main types of validity: internal and external. Internal validity refers to the confidence we have in the cause and effect relationship in a study, while external validity refers to the degree to which the conclusions of a study can be applied to other people, places, and times. There are various threats to both internal and external validity, such as sampling, measurement instrument obtrusiveness, and reactive effects of setting. Additionally, there are several subtypes of validity, including face validity, content validity, criterion validity, and construct validity. Each subtype has its own specific focus and methods for testing validity.

The key characteristic that sets ratio variables apart from interval variables is the presence of a meaningful zero point. On a ratio scale, this zero point signifies the absence of the measured attribute, while on an interval scale, the zero point is simply a point on the scale with no inherent significance.

Scales of Measurement in Statistics

In the 1940s, Stanley Smith Stevens introduced four scales of measurement to categorize data variables. Knowing the scale of measurement for a variable is crucial in selecting the appropriate statistical analysis. The four scales of measurement are ratio, interval, ordinal, and nominal.

Ratio scales are similar to interval scales, but they have true zero points. Examples of ratio scales include weight, time, and length. Interval scales measure the difference between two values, and one unit on the scale represents the same magnitude on the trait of characteristic being measured across the whole range of the scale. The Fahrenheit scale for temperature is an example of an interval scale.

Ordinal scales categorize observed values into set categories that can be ordered, but the intervals between each value are uncertain. Examples of ordinal scales include social class, education level, and income level. Nominal scales categorize observed values into set categories that have no particular order of hierarchy. Examples of nominal scales include genotype, blood type, and political party.

Data can also be categorized as quantitative of qualitative. Quantitative variables take on numeric values and can be further classified into discrete and continuous types. Qualitative variables do not take on numerical values and are usually names. Some qualitative variables have an inherent order in their categories and are described as ordinal. Qualitative variables are also called categorical of nominal variables. When a qualitative variable has only two categories, it is called a binary variable.

Likert scales are a type of ordinal scale used in surveys to measure attitudes of opinions. Respondents are presented with a series of statements of questions and asked to rate their level of agreement of frequency of occurrence on a scale of options. For instance, a Likert scale question might ask how often someone prays, with response options ranging from never to daily. While the responses are ordered in terms of frequency, the intervals between each option are not necessarily equal of quantifiable. Therefore, Likert scales are considered ordinal rather than interval scales.

Scales of Measurement in Statistics

In the 1940s, Stanley Smith Stevens introduced four scales of measurement to categorize data variables. Knowing the scale of measurement for a variable is crucial in selecting the appropriate statistical analysis. The four scales of measurement are ratio, interval, ordinal, and nominal.

Ratio scales are similar to interval scales, but they have true zero points. Examples of ratio scales include weight, time, and length. Interval scales measure the difference between two values, and one unit on the scale represents the same magnitude on the trait of characteristic being measured across the whole range of the scale. The Fahrenheit scale for temperature is an example of an interval scale.

Ordinal scales categorize observed values into set categories that can be ordered, but the intervals between each value are uncertain. Examples of ordinal scales include social class, education level, and income level. Nominal scales categorize observed values into set categories that have no particular order of hierarchy. Examples of nominal scales include genotype, blood type, and political party.

Data can also be categorized as quantitative of qualitative. Quantitative variables take on numeric values and can be further classified into discrete and continuous types. Qualitative variables do not take on numerical values and are usually names. Some qualitative variables have an inherent order in their categories and are described as ordinal. Qualitative variables are also called categorical of nominal variables. When a qualitative variable has only two categories, it is called a binary variable.

The threshold score that results in the lowest misclassification rate, achieved by minimizing both false positive and false negative rates, is known as the optimal threshold. Based on the findings of the previous study, the ideal cut off for identifying caseness on the MDQ is five, making it the optimal threshold.

The Normal distribution is a probability distribution that is continuous in nature.

Standard Deviation and Standard Error of the Mean

Standard deviation (SD) and standard error of the mean (SEM) are two important statistical measures used to describe data. SD is a measure of how much the data varies, while SEM is a measure of how precisely we know the true mean of the population. The normal distribution, also known as the Gaussian distribution, is a symmetrical bell-shaped curve that describes the spread of many biological and clinical measurements.

68.3% of the data lies within 1 SD of the mean, 95.4% of the data lies within 2 SD of the mean, and 99.7% of the data lies within 3 SD of the mean. The SD is calculated by taking the square root of the variance and is expressed in the same units as the data set. A low SD indicates that data points tend to be very close to the mean.

On the other hand, SEM is an inferential statistic that quantifies the precision of the mean. It is expressed in the same units as the data and is calculated by dividing the SD of the sample mean by the square root of the sample size. The SEM gets smaller as the sample size increases, and it takes into account both the value of the SD and the sample size.

Both SD and SEM are important measures in statistical analysis, and they are used to calculate confidence intervals and test hypotheses. While SD quantifies scatter, SEM quantifies precision, and both are essential in understanding and interpreting data.

Gender has traditionally been recorded as either male of female, creating a binary of dichotomous variable. Other categorical variables, such as eye color and ethnicity, can be grouped into two or more categories. Continuous variables, such as temperature, height, weight, and age, can be placed anywhere on a scale and have mathematical properties. Ordinal variables allow for ranking, but do not allow for direct mathematical comparisons between values.

Calculation of Standardised Mortality Ratio (SMR)

To calculate the SMR, age and sex-specific death rates in the standard population are obtained. An estimate for the number of people in each category for both the standard and study populations is needed. The number of expected deaths in each age-sex group of the study population is calculated by multiplying the age-sex-specific rates in the standard population by the number of people in each category of the study population. The sum of all age- and sex-specific expected deaths gives the expected number of deaths for the whole study population. The observed number of deaths is then divided by the expected number of deaths to obtain the SMR.

The SMR can be standardised using the direct of indirect method. The direct method is used when the age-sex-specific rates for the study population and the age-sex-structure of the standard population are known. The indirect method is used when the age-specific rates for the study population are unknown of not available. This method uses the observed number of deaths in the study population and compares it to the number of deaths that would be expected if the age distribution was the same as that of the standard population.

The SMR can be interpreted as follows: an SMR less than 1.0 indicates fewer than expected deaths in the study population, an SMR of 1.0 indicates the number of observed deaths equals the number of expected deaths in the study population, and an SMR greater than 1.0 indicates more than expected deaths in the study population (excess deaths). It is sometimes expressed after multiplying by 100.

A study that has a greater sample size is considered to have higher power, meaning it is capable of detecting a significant difference of effect that is clinically relevant.

The Importance of Power in Statistical Analysis

Power is a crucial concept in statistical analysis as it helps researchers determine the number of participants needed in a study to detect a clinically significant difference of effect. It represents the probability of correctly rejecting the null hypothesis when it is false, which means avoiding a Type II error. Power values range from 0 to 1, with 0 indicating 0% and 1 indicating 100%. A power of 0.80 is generally considered the minimum acceptable level.

Several factors influence the power of a study, including sample size, effect size, and significance level. Larger sample sizes lead to more precise parameter estimations and increase the study’s ability to detect a significant effect. Effect size, which is determined at the beginning of a study, refers to the size of the difference between two means that leads to rejecting the null hypothesis. Finally, the significance level, also known as the alpha level, represents the probability of a Type I error. By considering these factors, researchers can optimize the power of their studies and increase the likelihood of detecting meaningful effects.

SIGLE is a database that contains unpublished of ‘grey’ literature, while CINAHL is a database that focuses on healthcare and biomedical journal articles. The Cochrane Library is a collection of databases that includes the Cochrane Reviews, which are systematic reviews and meta-analyses of medical research. EMBASE is a pharmacological and biomedical database, and PsycINFO is a database of abstracts from psychological literature that is created by the American Psychological Association.

Clinical tests are used to determine the presence of absence of a disease of condition. To interpret test results, it is important to have a working knowledge of statistics used to describe them. Two by two tables are commonly used to calculate test statistics such as sensitivity and specificity. Sensitivity refers to the proportion of people with a condition that the test correctly identifies, while specificity refers to the proportion of people without a condition that the test correctly identifies. Accuracy tells us how closely a test measures to its true value, while predictive values help us understand the likelihood of having a disease based on a positive of negative test result. Likelihood ratios combine sensitivity and specificity into a single figure that can refine our estimation of the probability of a disease being present. Pre and post-test odds and probabilities can also be calculated to better understand the likelihood of having a disease before and after a test is carried out. Fagan’s nomogram is a useful tool for calculating post-test probabilities.

The use of allocation concealment is being implemented by the researchers to prevent interference from investigators of patients in the randomisation process. This is important as knowledge of group allocation can lead to patient refusal to participate of researchers manipulating the allocation process. By using distant call centres for allocation concealment, the risk of selection bias, which refers to systematic differences between comparison groups, is reduced.

Cost effectiveness analysis (CEA) is an economic evaluation method that compares the costs and outcomes of different courses of action. The outcomes of the interventions must be measurable using a single variable, such as life years gained, making it useful for comparing preventative treatments for fatal conditions.

Qualitative research is a method of inquiry that seeks to understand the meaning and experience dimensions of human lives and social worlds. There are different approaches to qualitative research, such as ethnography, phenomenology, and grounded theory, each with its own purpose, role of the researcher, stages of research, and method of data analysis. The most common methods used in healthcare research are interviews and focus groups. Sampling techniques include convenience sampling, purposive sampling, quota sampling, snowball sampling, and case study sampling. Sample size can be determined by data saturation, which occurs when new categories, themes, of explanations stop emerging from the data. Validity can be assessed through triangulation, respondent validation, bracketing, and reflexivity. Analytical approaches include content analysis and constant comparison.

The statistical significance of the result is questionable as the confidence interval encompasses values below 100. This implies that there is a possibility that the actual value could be lower than 100, which contradicts the observed value of 120 indicating a rise in mortality in this population.

Calculation of Standardised Mortality Ratio (SMR)

To calculate the SMR, age and sex-specific death rates in the standard population are obtained. An estimate for the number of people in each category for both the standard and study populations is needed. The number of expected deaths in each age-sex group of the study population is calculated by multiplying the age-sex-specific rates in the standard population by the number of people in each category of the study population. The sum of all age- and sex-specific expected deaths gives the expected number of deaths for the whole study population. The observed number of deaths is then divided by the expected number of deaths to obtain the SMR.

The SMR can be standardised using the direct of indirect method. The direct method is used when the age-sex-specific rates for the study population and the age-sex-structure of the standard population are known. The indirect method is used when the age-specific rates for the study population are unknown of not available. This method uses the observed number of deaths in the study population and compares it to the number of deaths that would be expected if the age distribution was the same as that of the standard population.

The SMR can be interpreted as follows: an SMR less than 1.0 indicates fewer than expected deaths in the study population, an SMR of 1.0 indicates the number of observed deaths equals the number of expected deaths in the study population, and an SMR greater than 1.0 indicates more than expected deaths in the study population (excess deaths). It is sometimes expressed after multiplying by 100.

Since a superiority trial involves comparing a new drug with an already existing treatment that can also reduce HbA1c levels, a substantial sample size is necessary to establish a significant distinction.

Study Designs for New Drugs: Options and Considerations

When launching a new drug, there are various study design options available. One common approach is a placebo-controlled trial, which can provide strong evidence but may be deemed unethical if established treatments are available. Additionally, it does not allow for a comparison with standard treatments. Therefore, statisticians must decide whether the trial aims to demonstrate superiority, equivalence, of non-inferiority to an existing treatment.

Superiority trials may seem like the obvious choice, but they require a large sample size to show a significant benefit over an existing treatment. Equivalence trials define an equivalence margin on a specified outcome, and if the confidence interval of the difference between the two drugs falls within this margin, the drugs are assumed to have a similar effect. Non-inferiority trials are similar to equivalence trials, but only the lower confidence interval needs to fall within the equivalence margin. These trials require smaller sample sizes, and once a drug has been shown to be non-inferior, larger studies may be conducted to demonstrate superiority.

It is important to note that drug companies may not necessarily aim to show superiority over an existing product. If they can demonstrate that their product is equivalent of even non-inferior, they may compete on price of convenience. Overall, the choice of study design depends on various factors, including ethical considerations, sample size, and the desired outcome.

Stats: Correlation and Regression

Correlation and regression are related but not interchangeable terms. Correlation is used to test for association between variables, while regression is used to predict values of dependent variables from independent variables. Correlation can be linear, non-linear, of non-existent, and can be strong, moderate, of weak. The strength of a linear relationship is measured by the correlation coefficient, which can be positive of negative and ranges from very weak to very strong. However, the interpretation of a correlation coefficient depends on the context and purposes. Correlation can suggest association but cannot prove of disprove causation. Linear regression, on the other hand, can be used to predict how much one variable changes when a second variable is changed. Scatter graphs are used in correlation and regression analyses to visually determine if variables are associated and to detect outliers. When constructing a scatter graph, the dependent variable is typically placed on the vertical axis and the independent variable on the horizontal axis.

Understanding Hypothesis Testing in Statistics

In statistics, it is not feasible to investigate hypotheses on entire populations. Therefore, researchers take samples and use them to make estimates about the population they are drawn from. However, this leads to uncertainty as there is no guarantee that the sample taken will be truly representative of the population, resulting in potential errors. Statistical hypothesis testing is the process used to determine if claims from samples to populations can be made and with what certainty.

The null hypothesis (Ho) is the claim that there is no real difference between two groups, while the alternative hypothesis (H1 of Ha) suggests that any difference is due to some non-random chance. The alternative hypothesis can be one-tailed of two-tailed, depending on whether it seeks to establish a difference of a change in one direction.

Two types of errors may occur when testing the null hypothesis: Type I and Type II errors. Type I error occurs when the null hypothesis is rejected when it is true, while Type II error occurs when the null hypothesis is accepted when it is false. The power of a study is the probability of correctly rejecting the null hypothesis when it is false, and it can be increased by increasing the sample size.

P-values provide information on statistical significance and help researchers decide if study results have occurred due to chance. The p-value is the probability of obtaining a result that is as large of larger when in reality there is no difference between two groups. The cutoff for the p-value is called the significance level (alpha level), typically set at 0.05. If the p-value is less than the cutoff, the null hypothesis is rejected, and if it is greater or equal to the cut off, the null hypothesis is not rejected. However, the p-value does not indicate clinical significance, which may be too small to be meaningful.

Choosing the right statistical test can be challenging, but understanding the basic principles can help. Different tests have different assumptions, and using the wrong one can lead to inaccurate results. To identify the appropriate test, a flow chart can be used based on three main factors: the type of dependent variable, the type of data, and whether the groups/samples are independent of dependent. It is important to know which tests are parametric and non-parametric, as well as their alternatives. For example, the chi-squared test is used to assess differences in categorical variables and is non-parametric, while Pearson’s correlation coefficient measures linear correlation between two variables and is parametric. T-tests are used to compare means between two groups, and ANOVA is used to compare means between more than two groups. Non-parametric equivalents to ANOVA include the Kruskal-Wallis analysis of ranks, the Median test, Friedman’s two-way analysis of variance, and Cochran Q test. Understanding these tests and their assumptions can help researchers choose the appropriate statistical test for their data.