Behavioral Science Flashcards
Cross-sectional study
Collects data from a group of people to assess frequency of disease (and related risk factors) at a particular point in time
What is happening?
Disease prevalence
Can show risk factor association with disease, but does not establish causality
Case-control study
“Retrospective”
Compares a group of people with disease to a group without disease
Looks for prior exposure or risk factor
What happened?
Odds Ratio
“Patients with COPD had higher odds of a Hx of smoking than those without COPD”
Cohort study
“Retrospective or Prospective”
Compares a group with a given exposure or risk factor to a group without such exposure
Looks to see if exposure increases the likelihood of disease
Can be prospective (asks, “Who will develop disease?”) or retrospective (asks, “Who developed the disease [exposed vs nonexposed]?”)
Relative Risk
“Smokers had a higher risk of developing COPD than nonsmokers”
Twin concordance study
Compares the frequency with which both monozygotic twins or both dizygotic twins develop the same disease
Measures heritability and influence of environmental factors (nature vs nurture)
Adoption study
Compares siblings raised by biological vs adoptive parents
Measures heritability and influence of environmental factors
Clinical Trial
Experimental study involving humans. Compares therapeutic benefits of 2 or more treatments or of treatment and placebo. Study quality improves when study is randomized, controlled, and double-blinded (neither patient nor doctor knows whether the patient is in the treatment or control group).
Triple blind refers to the additional blinding of the researchers analyzing the data
Phase 1 - small number of healthy volunteers. “Is it safe?” assesses safety, toxicity, pharmacokinetics, and pharmacodynamics.
Phase 2 - Small number of patients with disease of interest. “Does it work?” Assesses treatment efficacy, optimal dosing, and adverse effects.
Phase 3 - Large number of patients randomly assigned either to the treatment under investigation or to the best available treatment (or placebo). “Is it good or better?” Compares the new treatment to the current standard of care
Phase 4 - Postmarketing surveillance of patients after treatment is approved. “Can it stay?” Detects rare or long-term adverse effects. Can result in treatment being withdrawn from market.
Sensitivity
“True positive rate”
Proportion of all people with disease who test positive, or the probability that a test detects disease when disease is present.
Value approaching 100% is desirable for RULING OUT disease and indicates a LOW FALSE NEG rate. High sensitivity test used for screening in diseases with low prevalence.
= TP/ (TP + FN)
= 1 - FN rate
SN-N-OUT = highly SeNsitive test, when Negative, rules OUT disease.
If sensitivity is 100%, TP/(TP - FN) = 1, FN = 0, and all negatives must be TNs
Specificity
“True negative rate”
Proportion of all people without disease who test negative, or the probability that a test indicates no disease when disease is absent.
Value approaching 100% is desirable for RULING IN disease and indicates a LOW FALSE POSITIVE rate. High specificity tests used for confirmation after a positive screening test.
= TN/ (TN + FP)
= 1 - false positive rate
SP-P-IN - highly specific test when positive rules in a disease.
Positive predictive value (PPV)
Proportion of positive test results that are true positive.
Probability that a person actually has the disease given a positive test result
= TP/ (TP + FP)
PPV varies directly with prevalence or pretest probability; high pretest probability leads to high PPV
Negative predictive value (NPV)
Proportion of negative test results that are true negative.
Probability that person actually is disease free given a negative test result
= TN/ (TN + FN)
NPV varies inversely with prevalence or pretest probability; high pretest probability leads to low NPV
Incidence vs prevalence
Incidence rate = # of new cases/# of people at risk (during a time period)
Prevalence = # of existing cases/# of people at risk (at a point in time)
Prevalence = incidence for short duration disease (common cold)
Incidence looks at new cases (incidents)
Prevalence looks at ALL current cases
Prevalence = pretest probability
Quantifying risk
Definitions and formulas are based on the classic 2x2 or contingency table.
Disease (+) (-)
Risk factor/ (+) a b
intervention (-) c d
Odds Ratio (OR)
Typically used in case-control studies. Odds that the group with the disease (cases) was exposed to a risk factor (a/c) divided by the odds that the group without the disease (controls) was exposed (b/d)
OR = (a/c) / (b/d)
OR = ad/bc
Relative Risk (RR)
Typically used in cohort studies.
Risk of developing disease in the exposed group divided by risk in the unexposed group (if 21% of smokers develop lung cancer vs 1% of nonsmokers, then RR = 21). If prevalence is low, OR = RR
RR =[a/(a+b)] / [c/(c+d)]
Attributable risk (AR)
The difference in risk between exposed and unexposed groups, or the proportion of diseases occurrences that are attributable to the exposure (if risk of lung cancer in smokers is 21% and risk in nonsmokers is 1%, then 20% of the lung cancer risk in smokers is attributable to smoking)
AR = [a/(a+b)] - [c/(c+d)]
Relative risk reduction (RRR)
The proportion of risk reduction attributable to the intervention as compares to a control (if 2% of patients who receive a flu shot develop the flu, while 8% of unvaccinated patients develop the flu, then RR = 2/8 = 0.25, and RRR = 0.75)
RRR = 1 - RR
Absolute risk reduction (ARR)
The difference in risk (not the proportion) attributable to the intervention as compared to a control (if 8% of people who receive a placebo vaccine develop the flu vs 2% of people who receive a flu vaccine, then ARR = 8% - 2% = 6% = 0.06)
ARR = [c/(c+d)] - [a/(a+b)]
Number needed to treat (NNT)
Number of patients who need to be treated for 1 patient to benefit
NNT = 1/ARR
Number needed to harm (NNH)
Number of patients who need to be exposed to a risk factor for 1 patient to be harmed
NNH = 1/AR
Precision
The consistency and reproducibility of a test (reliability)
The absence of random variation in a test
Random error reduces precision in a test
Increased precision leads to lower standard deviation
Increased precision leads to higher statistical power (1-Beta)