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Drug Literature Eval
| Question | Answer |
|---|---|
| Quantitative (Study Design) | Numbers used to represent data |
| Qualitative (Study Design) | Words used to represent data |
| Interventional (Quantitative study design) | Allocation to forced intervention groups (Phase 0 - 4) |
| Observation (Quantitative study design) | Allocation to non-forced study groups |
| Research Evidence Pyramid | Increasing strength of evidence: In vitro (test-tube) research Animal research Case reports Case series Ecological Cross-sectional Case-control Cohort High quality systematic review with peer reviews Meta Analyses Interventional/RCT |
| Population | All individuals making up a common group; from which a sample (smaller set) can be obtained |
| Sample | A subset or portion of the full, complete population |
| Prospective (study design) | Outcome is not yet known at the start of the study |
| Retrospective (study design) | Outcome is already known at the start of the study |
| Null hypothesis (H0) | A research perspective which states there will be no true difference between the groups being compared |
| Alternative hypothesis (H1) | A research perspective which states there will be a true difference between the groups being compared |
| Case-control design | Useful when studying a rare disease Commonly generates an odds of exposure for each, then an odds ratio (OR) as the measure of association Customarily retrospective |
| Cohort design | Useful when studying a true rates (RR) Commonly generates the risk of disease/outcome for each, then a risk ratio/relative risk (RR) as measure of association Conducted in a retrospective or prospective fashion |
| Pre-clinical studies | Prior-to human investigation 'Bench' or animal research |
| Phase 0 | Exploratory; investigational new drug Assess PK, first in human use (healthy volunteers), small sample size for a short duration |
| Phase 1 | Investigational new drug Assess safety, tolerance, PK in healthy or diseased volunteers with small N, short duration |
| Phase 2 | Investigational new drug Assess effectiveness in diseased volunteers with narrow inclusion criteria, larger N, short to medium duration |
| Phase 3 | Investigation new drug; indication/population Assess effectiveness in diseased volunteers with inclusion criteria and various statistical perspectives used Larger N, longer duration |
| Phase 4 | Post FDA-approval called postmarketing Assess risk/benefits in expanded use criteria in population N for a longer duration |
| Internal validity | Purpose: to reduce investigator-bias by allowing their hand in group allocation, and to strive to make study groups as equal as possible based on known confounders and group size. |
| Forms of randomization | Simple: equal probability for allocation into one of the study groups Blocked: ensures balance within each intervention group (ie want equal group size) Stratified: ensures balance within known confounding variables |
| Single-blind | Study subjects are not informed which intervention group they are in, but investigators are permitted to know |
| Double-blind | Neither investigators nor study subjects are informed which intervention treatment group subjects are in |
| Open-label/unmasked/unblinded | Study subjects & researchers know what intervention is being received |
| Placebo (dummy therapy) | Inert treatment made to look identical in all aspects to active treatment |
| Placebo-effect | Improvement in condition by power of suggestion of being 'treated' |
| Hawthorne-effect | Study subjects change their behavior solely due to awareness of being studied/observed |
| Outcomes | Primary: the outcome variable that is the major variable or outcome Secondary: other variables of interest but not the primary outcome |
| Endopoints | Composite: combined endpoints Surrogate: physical sign or lab value used in place of clinical assessment |
| Internal validity: Intent to Treat (ITT) | Once randomized, always analyzed Use last known assessment for remaining study evaluations Preserves randomization and statistical power |
| Internal validity: Per Protocol | Must meet pre-established level of compliance/participate to be included Biases estimates of effects Can limit generalizability |
| Internal validity: run-in/lead-in | Subjects can be given placebo for initial, pre-study time frame to determine a new baseline of disease |
| Confounders | An extra variable that you didn't account for that may ruin experiment, giving you useless results, suggest there is a correlation, and even introduce bias |
| Descriptive statistics | Summarize results (ie mean, mode, SD) |
| Inferential statistics | Allow testing of our hypothesis and draw conclusion about a population using a sample from it |
| Random variables | A variable whose observed values may be considered as outcomes of an experiment and who values cannot be anticipated without certainty before the experiment is conducted |
| Random variables: discrete (counting) | 2 types Nominal: classified into groups in no particular order and no indication of relative severity Ordinal: ranked in a specific order, but with no consistent level of magnitude between ranks |
| Random variables: continuous (measuring) | 2 types Interval: data are ranked in a specific order with a consistent change in magnitude between units Ratio: like "interval" but with an absolute zero |
| Type I Error | Probability of making this error is a priori cut off (commonly 0.05) Reject the null when the null is true |
| Type II Error | Likelihood of making this error is beta Concluding that no difference exists in the sample when one truly does |
| Power | (1-beta) The probability of correctly rejecting the null when a difference exists in the population sample Dependent on sample size, beta, a priori |
| Nonparametric | No assumptions made Chi Square Categorical data |
| Parametric | 2 assumptions and BOTH must be met: Sample size >30 or normal dist Variable has to be interval level data Comparison of means t-test: 2 means paired t-test: 2 paired means ANOVA: more than 2 means repeated-measures ANOVA: more than 2 paired means |
| Relative Risk Reduction (RRR) | An estimate of the percentage of baseline risk removed as a result of therapy (efficacy) Does not consider magnitude or changes in baseline risk --> overinflates results |
| Absolute Risk Reduction | Difference in risk between experimental and control group (diff in event rates or risk diff) The number of patients who are spared as a result of treatment Changes along with baseline risk |
| Number Needed to Treat (NNT) | ONLY calculated when statistically significant results The number of patients who must undergo treatment in order for one patient to gain benefit Inverse with baseline risk =100/ARR (if %) or 1/ARR |
| Sensitivity | Proportion of time a test is positive in patients who have the disease (%true positives) = pts with disease/all pts with the disease = TP/(TP + FN) |
| Specificity | Proportion of time a test is negative in patients who do NOT have the disease (%true negatives) = pts w/o disease/all pts w/o disease = TN/(FP + TN) |
| Prevalence | All cases (new and existing) with the disease during a period of time or at a particular date in time Meaningful when reported as the number of cases as a fraction of the total population at risk. |
| Incidence | Rate of new cases of the disease occurring w/in a period of time More meaningful when reported as a fraction of the population at risk of developing the disease |
Created by:
hmariner1
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