Exam2 Flashcards
Advantages to randomization
1) Assignment of a patient to a study group is determined by chance
2) Unpredictability
3) Rules out self-selection of subjects
4) Provides control over confounding, even by factors that are hard to measure or unknown to the investigator
5) Distributes potential confounders similarly across comparison groups (exposed/non-exposed)
6) Randomization does not guarantee the comparability of groups – therefore stratified matching
Effects of non-compliance
Drop-outs do not take the treatment, either knowingly or unknowingly when they shouldn’t while drop-ins either knowingly or unknowingly the treatment when they shouldnt. The effect is that there is less difference seen in treatment effect, the groups will be less different than they should have been.
Intention to treat analysis
Analytical method for randomized trials. primary type of analysis done, all individuals randomly allocated to treatment are analyzed regardless of whether they received treatment or not.
Efficacy analysis
Analytical method for randomized trials. Used for reduction of risk. Determines the treatment effects under the ideal conditions in those who take the full treatment as directed (with compliance) but excludes those without compliance. excluding non-compliers usually over-estimates the effectiveness of a therapy
Matching in case control
individual matching
frequency matching - case and control groups
DISADVANTAGES
cannot study the effect of the matching factor
may reduce statistical power
matching factor must be included in the analysis
may be difficult and time consuming to match
Advantages of intention to treat
- It preserves the benefits of randomization
- Maintains the statistical power of the original study
- Helps ensure that the study results are unbiased
- Gives info on the effectiveness of a treatment under everyday practice condition
- Excluding non-compliers would over-estimate the success of intervention (conservative approach)
Number needed to treat
number needed to treat = number of patients who need to be treated in order to prevent one additional bad outcome
NNT = 1/(rate in untreated – rate in treated)
Rate = mortality, incidence (?)
Efficacy
used to analyze the extent of the reduction in outcomes by treatment - vaccines
(Rate in controls – rate in treated)/(rate in controls)
AKA reduction in risk
type I errors
The treatments do not differ but we conclude that they do (alpha)
type II errors
The treatments differ but we conclude that they do not (beta)
alpha
probability of making a type I error, concluding that treatments differ when they don’t
the level of statistical significance
beta
the probability of making a type II error, concluding that the treatments do not differ when they do differ
Strengths of randomized trials
1 - Control over the assignment of the treatment
2 - Randomization ensures the treatment and control groups are balanced, reducing bias and confounding
3 - Blinding minimizes bias
4 - Prospective design allows for temporality and causal relations
5 - Provides firm basis for statistical hypothesis testing - gold standard
Weaknesses of randomized trials
1 - generalizability - testing is done on a small number of motivated volunteers
2 - close monitoring may not be the case in a community setting
3 - expensive and labor intensive
When does the odds ratio approximate the risk ratio?
when the incidence is low, when the disease is rare
cohort effect
the influence of membership in a particular cohort - shared temporal experience or common life experience
Advantages of cohort studies
temporality
Direct determination of risk
Can design the study to follow the exposures you specifically want
Size of the cohort under control by study investigators
Can study rare exposures
Disadvantages of cohort studies
Takes a long time Expensive Need a lot of justification and supporting scientific data Not great for studying rare outcomes Subjects lost to follow up
Biases for cohort studies
Information bias on exposure and related factors
Bias in assessing outcomes
Bias in analysis and reporting
Bias from non-response and loss to follow up
Advantages of retrospective cohort
Faster – you don’t have to wait for cases to accrue
Less expensive than cohort study
You have a lot of exposure data to choose from
Advantages for case control studies
Rare diseases
Disease with long induction/latency periods (cancers)
Lower cost than cohort studies
Faster than cohort, no follow-up time needed
Less expensive than cohort studies
Can be exploratory (little known about disease)
Can assess multiple exposures – good for diseases about which little is known
Tend to use smaller sample sizes than cohort studies
Good for dynamic populations
Good for when exposure data are expensive or difficult to obtain
Disadvantages of retrospective cohort study
relies on available exposure info only, may not be in info that you want/need
Disadvantages of case control studies
No incidence or temporality
Information on previous exposures may not be available or accurate
Difficult to obtain appropriate controls
Representativeness of cases and controls is often unknown – selection bias
case-control biases
Information bias – interview cases more than controls
Recall bias: controls don’t remember as well
Misclassification bias (differential vs. non-differential)
Response bias: some groups of people may respond better than others
Selection bias -
Control selection – controls should be cases if they had been exposed
Controls should be from the same place as cases, or else there will be bias
Why try to choose incident cases for case control?
incident cases have less chance of change of exposure
difficult to assess temporality with prevalent cases
survivorship bias for prevalent cases - tend to be longterm survivors
how to choose controls - case-control
controls should be comparable to cases except with no disease and exposure experience
potential for exposure should be the same
controls should come from the same population as cases
controls should represent those who would have been cases if they were exposed
hospital based controls - pros and cons
easy to ID, more willing to participate, if from the same source population, minimal bias
However, not population based, may be from different source populations, exposure of interest may be associated with other diseases that are used as controls
Cross-sectional advantages
Estimate the magnitude and distribution of a health problem
Good for hypothesis generation
Useful for planning interventions
Repeated cross-sectional studies can show changes in trends in disease and risk factors
Low cost and generalizable
Cross-sectional disadvantages
No incidence data
No temporality
Healthy worker survivor effect – long term prevalence – long term survivors favored
Not good for disease with low prevalence (rare or short duration)