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Questions and Answers
What is a primary reason for conducting sample size calculations in research?
What is a primary reason for conducting sample size calculations in research?
Which of the following best defines a Type I error?
Which of the following best defines a Type I error?
Which factor does NOT impact the determination of sample size in a study?
Which factor does NOT impact the determination of sample size in a study?
What is a result of conducting a small study?
What is a result of conducting a small study?
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What happens when a study is too small in sample size?
What happens when a study is too small in sample size?
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How does trial size impact the credibility of results?
How does trial size impact the credibility of results?
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Why is it important to adjust sample sizes for loss to follow-up?
Why is it important to adjust sample sizes for loss to follow-up?
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In the context of sample size, what does 'risk ratio' indicate?
In the context of sample size, what does 'risk ratio' indicate?
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What was the primary outcome measured in the clinical trial investigating CBT for reducing depression in adults with cancer?
What was the primary outcome measured in the clinical trial investigating CBT for reducing depression in adults with cancer?
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What difference in BDI-II score did investigators consider necessary to recommend CBT for clinical practice?
What difference in BDI-II score did investigators consider necessary to recommend CBT for clinical practice?
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Which factor does NOT contribute to the power of a study?
Which factor does NOT contribute to the power of a study?
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What is one way to increase the power of a clinical trial?
What is one way to increase the power of a clinical trial?
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In the context of the CBT study, what represents the expected treatment effect associated with CBT?
In the context of the CBT study, what represents the expected treatment effect associated with CBT?
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What is the primary endpoint of the clinical trial investigating the effectiveness of cognitive behavioural therapy?
What is the primary endpoint of the clinical trial investigating the effectiveness of cognitive behavioural therapy?
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What is the expected mean BDI-II score in the control group following treatment?
What is the expected mean BDI-II score in the control group following treatment?
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What treatment difference must be detected to implement CBT in clinical practice based on the study's criteria?
What treatment difference must be detected to implement CBT in clinical practice based on the study's criteria?
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What level of statistical significance is set for this trial?
What level of statistical significance is set for this trial?
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What does a sample size calculation help determine in this clinical trial?
What does a sample size calculation help determine in this clinical trial?
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With what degree of power is the trial designed to detect the treatment effect?
With what degree of power is the trial designed to detect the treatment effect?
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What variability is indicated by the standard deviation of the BDI-II scores in cancer patients?
What variability is indicated by the standard deviation of the BDI-II scores in cancer patients?
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How many individual CBT sessions will the intervention group receive in addition to treatment as usual?
How many individual CBT sessions will the intervention group receive in addition to treatment as usual?
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What is the remaining percentage of participants after a 20% attrition from an initial 120 participants?
What is the remaining percentage of participants after a 20% attrition from an initial 120 participants?
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How is the adjusted sample size calculated when the attrition rate is 25%?
How is the adjusted sample size calculated when the attrition rate is 25%?
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What does a p-value greater than 0.05 indicate in the context of sample size calculations?
What does a p-value greater than 0.05 indicate in the context of sample size calculations?
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Why should post hoc power calculations be avoided according to the available guidelines?
Why should post hoc power calculations be avoided according to the available guidelines?
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What does the term 'Q' represent when calculating the adjustment for loss to follow-up?
What does the term 'Q' represent when calculating the adjustment for loss to follow-up?
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Which of the following is NOT a reason for obtaining a p-value greater than 0.05?
Which of the following is NOT a reason for obtaining a p-value greater than 0.05?
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What is an advisable method to report findings beyond post hoc power calculations?
What is an advisable method to report findings beyond post hoc power calculations?
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Which statement is true about the relation between sample size and Type I and Type II errors?
Which statement is true about the relation between sample size and Type I and Type II errors?
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What is meant by the term 'clinically important effect' in sample size calculations?
What is meant by the term 'clinically important effect' in sample size calculations?
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Which factor is typically assumed to be the same between groups when determining sample size?
Which factor is typically assumed to be the same between groups when determining sample size?
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What statistical power is commonly specified in sample size calculations?
What statistical power is commonly specified in sample size calculations?
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In the context of binary outcomes, what does the variable p1
represent?
In the context of binary outcomes, what does the variable p1
represent?
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Which component is NOT part of the required sample size formula for comparing two means?
Which component is NOT part of the required sample size formula for comparing two means?
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How is the required sample size for means and proportions generally related?
How is the required sample size for means and proportions generally related?
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What does the significance level typically represent in sample size calculations?
What does the significance level typically represent in sample size calculations?
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Which of the following can be affected by the standard deviation in sample size calculations?
Which of the following can be affected by the standard deviation in sample size calculations?
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What is the primary effect of larger sample sizes in hypothesis testing?
What is the primary effect of larger sample sizes in hypothesis testing?
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How does an increase in variability of outcomes between groups affect sample size requirements?
How does an increase in variability of outcomes between groups affect sample size requirements?
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What is the significance of 'power' in sample size calculations?
What is the significance of 'power' in sample size calculations?
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What is the impact of a smaller significance level on sample size requirements?
What is the impact of a smaller significance level on sample size requirements?
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In the context of anticipating loss to follow-up, what should researchers do?
In the context of anticipating loss to follow-up, what should researchers do?
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What method is typically used to estimate required sample size for continuous outcomes adjusting for baseline?
What method is typically used to estimate required sample size for continuous outcomes adjusting for baseline?
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Which factor is likely to increase sample size requirements when conducting a hierarchical study?
Which factor is likely to increase sample size requirements when conducting a hierarchical study?
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What must researchers consider when conducting time to event analysis?
What must researchers consider when conducting time to event analysis?
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What effect does a significant loss to follow up have on a study’s power?
What effect does a significant loss to follow up have on a study’s power?
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Flashcards
Sample Size
Sample Size
The number of participants required to obtain reliable evidence of a treatment effect, while also achieving precise estimates of the effect.
Type I Error
Type I Error
Incorrectly rejecting a true null hypothesis. This means concluding there is an effect when there is not.
Type II Error
Type II Error
Incorrectly failing to reject a false null hypothesis. This means failing to detect an effect that actually exists.
Alpha level (α)
Alpha level (α)
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Power (1 - β)
Power (1 - β)
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Limitations of small studies
Limitations of small studies
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Publication Bias
Publication Bias
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Appropriate Sample Size
Appropriate Sample Size
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Power
Power
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Effect size
Effect size
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Standard deviation (SD)
Standard deviation (SD)
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Clinically Important Effect
Clinically Important Effect
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Significance Level (Alpha)
Significance Level (Alpha)
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Sample Size Formula (Two Means)
Sample Size Formula (Two Means)
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Sample Size Formula (Two Proportions)
Sample Size Formula (Two Proportions)
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Sample Size Relationship
Sample Size Relationship
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Primary Outcome Measure
Primary Outcome Measure
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What is the primary outcome measure?
What is the primary outcome measure?
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How will the data be analysed?
How will the data be analysed?
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What results are expected in the control group?
What results are expected in the control group?
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How small a treatment difference needs to be detected?
How small a treatment difference needs to be detected?
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With what degree of certainty?
With what degree of certainty?
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What is the standard deviation of BDI-II scores?
What is the standard deviation of BDI-II scores?
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What is the statistical significance level?
What is the statistical significance level?
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What formula is used for sample size calculation?
What formula is used for sample size calculation?
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Attrition Adjustment
Attrition Adjustment
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Post Hoc Power Calculation
Post Hoc Power Calculation
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Standard Deviation
Standard Deviation
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Statistical Power
Statistical Power
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Alpha (α)
Alpha (α)
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Statistical Power (1- β)
Statistical Power (1- β)
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Detectable Difference
Detectable Difference
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Sample Size Per Group
Sample Size Per Group
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Variability in the Outcome
Variability in the Outcome
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Loss to Follow-Up
Loss to Follow-Up
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Hierarchical Data Structures
Hierarchical Data Structures
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Correlation Between Baseline and Follow-Up
Correlation Between Baseline and Follow-Up
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Intraclass Correlation (ICC)
Intraclass Correlation (ICC)
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Study Notes
Power and Sample Size in Mental Health Research
- Sample size calculations are crucial for mental health research.
- Understanding Type I and Type II errors is vital.
- Key factors determining sample size include:
- The magnitude of the expected effect.
- The variability in the data.
- The desired level of statistical power.
- The significance level (alpha).
- Calculating sample size for continuous and binary outcomes is important in the study design.
- Loss to follow-up must be considered in adjusting sample size.
- Sample sizes should be large enough to detect clinically important effects, but not so large that resources are wasted.
Approaches to Sample Size Determination
- Statistical/Scientific Approach: Determine the number of patients needed to reliably detect a treatment effect (if one exists).
- Economic/Pragmatic Approach: Assess the availability of patients, recruitment time, and cost. Consider how many patients are available, how long recruitment will take and what the cost of the trial will be.
- Ethical Considerations: Establish how soon a trial should be stopped given an inferior treatment.
- Credibility Considerations: Ensure the size of the trial is sufficient to create reliable results.
- Important to consider ethical implications and limitations of the available resources, such as patient numbers.
Small Studies
- Small studies often fail to detect clinically important and realistically sized treatment effects.
- They may show a clinically significant but not statistically significant effect.
- Small studies frequently yield imprecise estimates.
- Wide confidence intervals for effect estimates are common in small studies.
- Findings from small studies are less likely to be representative of the population as a whole.
- Publication bias favours small studies with statistically significant (p<0.05) results over those that are not. This can lead to misleading conclusions. Therefore, small studies with statistically significant results are more often published compared to small studies with non-significant results.
- Unethical when participants' time is spent in a study with a low chance of producing useful outcomes.
Results from Trials of Different Sizes
- Examples of trials (1st Australian and ISIS-2) illustrate how different sample sizes can lead to varying results.
- Analysis of trials of the drug streptokinase showed varying results depending on sample size, with the larger trial yielding a more reliable result.
Sample Size: Too Small vs. Too Large
- Too Small:
- Lack of precision in results.
- Difficulty distinguishing real improvements from chance variation.
- Ethical issues may arise if the study is too small to detect a true effect, wasting resources and participants' time.
- Too Large:
- Waste of resources (patients, funding, time).
- Ethical issues, especially for trials that involve treatments that may be inferior, wasting resources and participants' time.
Drawing the Wrong Conclusions
- Type I Error: False positive; concluding an effect exists when none exists in reality. This is a false positive result.
- Type II Error: False negative; concluding no effect exists when one does. This is a false negative result.
- Alpha (α)—Probability of a Type I error.
- Beta (β)—Probability of a Type II error.
- Power (1-β)—Probability of detecting an effect when one exists.
- Important to consider Type I and Type II errors in the context of sample size decisions.
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Description
This quiz explores the critical aspects of sample size calculations in mental health research. It covers essential topics such as Type I and Type II errors, statistical power, and factors influencing sample size determination. Gain insight into both statistical and pragmatic approaches to ensure effective study designs.