Block 2 lect 1 Flashcards by Betsy Guillen

Why do we conduct research ?

To answer the descriptive
[who, what, when, where]

analytical questions
[why, how]

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What is a drug ?

-a product intended for use in DX, CURE, TX, PREVENTION of a disease

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the drug discovery process :

-new insight into a disease state

-new tech
-testing compounds
-unanticipated effects
-eventually a product arises

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6 major sources of drug products:

-semi synthetic (penicillin)
-recombinant DNA tech
-Plant sources (digoxin)
-animal sources (premarin)
-minerals/earth sources
microbiological sources

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Stage 1: Drug Product Discovery

-focuses on target tissues and compounds

-LAB SCREENING (3-6 yrs)

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Stage 2: Pre-clinical Studies

-determine TOXICITY/ SAFETY

in vitro and in vivo

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In Vivo testing consists of:

test drugs ON LIVING ANIMALS : 2 mammals and 2 administrations routes

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In Vitro:

performed outside of normal environment of cells

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Investigational new drug application:

-drug sponsor/developer submits IND application BEFORE starting clinical research.

-request approval to give drug to humans
-FDA has 30 days to review the application

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Stage 3- PHASE 1, Clinical Trails

-healthy volunteers or
-with dx/ condition who failed conventional tx

-no control group
-focused on safety, tolerability, toxicity

-70% move on to phase 2

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stage 3, Phase II Clinical Trials

-ppl w/ disease of interest
-placebo group
-focuses on efficacy
-short term side effects

[ideal and controlled conditions]

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efficacy

-power to produce a desired result or effect

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Stage 3, Phase III Clinical Trials

-ppl w/ disease if interest
-focuses on efficacy AND effectiveness
-most time consuming phase

(superiority over inferiority)

-drug achieves health result under “real world” conditions

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effectiveness

-producing a result that is wanted and have an intended effect

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Vaccine Efficacy:

vaccine will reduce risk of infection by irrespective of transmission setting

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New Drug Application Process:

-gets through phase III clinical trials
-all trial are complete
-must demonstrate benefits» risks

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Stage 3, Phase IV Clinical Trials

-long-term data from post marketing surveillance

-MONITOR safety and efficacy
(look into rare side effects)

-pharmacovigilance

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Clinical Trial Phase 1

-toxicity/ tolerability
-healthy volunteers with disease or failure of tx
-unblinded, uncontrolled
<100

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Clinical Trial Phase 2

-efficacy and safety
-subjects with target disease
RCT
1-2 yrs
>100

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Clinical Trial phase III

-efficacy, effectiveness, and safety
-subjects with target disease
RCT
-several years

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Clinical Trail phase IV

-long term data
-target disease and additional disease

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What is research design?

-plan that allows researchers to gather answers to study questions

-provide valid results: Internal vs external

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Internal Validity:

degree which results of a study are correct to members od the source pop. (free from bias)

[quality of design]

-STATISTICALLy SIG?

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External validity:

results are true to members outside source population (generalize)
[applicability to practice]

CLINICALLY SIG?

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The 9 causation considerations:

-temporarily -strength -biological gradient -consistency -specificity -plausibility -coherence -analogy -experiment

temporality:

cause must occur before the effect

strength

plausibility inc. strength of relationship btw. variables

biological gradient

linear relationship btw tx and clinical outcome

consistency

cause and effect relationship observed across different settings

specificity

A single cause for an effect

plausibility

possibility for relationship btw casue and effect

coherence

cause and effect consistent w existing knowledge

analogy

phenomenon may show causation

experiment

-cause can be manipulated, leading to changes in effect

Experimental Designs:

researchers CONTROL one variable to see the other variable impact [RCT]

Observational Designs:

researcher OBSERVES two variables (case control)

Prospective

-data collection after study -determine variable and objective [RCT, prosp. cohort}

Retrospective

-evaluates past or existing data -minimal resources -rely in previous data [case control, retrospective cohort]

What are the two types of data classification:

-primary: data collected to answer a research question -secondary: using data from previous collection

Primary Data collection: [Self reports]

-collects thoughts, perceptions, attitudes, behaviors

Primary Data collection: [observational]

-gather info activities, characteristics, comm., interaction Obtrusive: participant aware is present Unobtrusive: unaware, researcher hidden/ in disguise

Primary Data collection: [biological measure't ]

-objective markers -clinical lab testing types: biophysical (BP) , biochemical (chemical) , microbiological

Measurement error- -systemic error:

-measurement / observation is always off target in same direction [BIASED]

Measurement error: -Random error

-due to chance (statistical adjustments)

Systematic Error: aka bias

-changing variables that can alter true values. -assessment bias -threat to internal validity

Types of Bias: Selection Bias

-error method from researcher where they select subj. for study participation Direct vs. indirect

Types of Bias: Information bias

-error occurs due to : wrong info collect from pt, or not the same effort put into both groups -different sources , data from previous records,

Types of information bias: recall reporting

-recall: defects in memory -reporting: one group reports more than the other (occurs in personal or sensitive)

Types of Bias: Confounding

-error that occurs when outcome of interest is influenced by outside factor -mask assoc. or falsely demonstrate assoc.

two ways to reduce systemic error:

-blind-masking -randomization

blind masking:

- those involved in research are unaware of participant allocation open label, single blind, double blind, triple blind

open label

-no blinding; least objective -all involved are aware

single blind

-one of those involved is unaware tx -participant is blinded

double blind

-subj. and investigators are unaware of tx allocation -phase 3 trials -

triple blind

-MOST OBJ. participants, investigators, and those resp. for monitoring data are blinded

randomization:

-all participants have equal chance to be assigned to intervention or control simple, block, stratified, adaptive

Randomization: Simple

-single sequence of random assignments easy for larger sizes

Randomization: Block

-put subjects to test -equal in size

Randomization: stratified

predefined strata based on what researchers find important

Randomization: adaptive

-modification of randomization