W7 Evidence based medicine Flashcards

1
Q

What is the definition of Evidence-Based Medicine?

A

Evidence based medicine is the * use of current best evidence in making decisions about the care of individual patients.
*(conscientious, explicit, and judicious)

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2
Q

Why do we need evidence-based medicines?

A
  • To ensure medicines sold are of high quality, safety and efficacy
    *They are the basis for the recommendations we make to patients and other health professionals through pharmacy services
  • It is essential that pharmacists are available to retrieve, appraise and apply research evidence as the basis for clinical decision making
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3
Q

What is an example of observational study?
What is an example of experimental study?

A

-Cross-sectional study/ Cohort study/ Case-control study
-Animal testing/ RCTs

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4
Q

What are the two types of Experimental Study design? (2)

A

Basically uses experiments
1. Animal and laboratory studies. Involves animals e.g. guinea pig, rat. Prior to human trials.

  1. Randomised controlled trial (RCT) – Involves humans (and considered the Gold standard)
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5
Q

Animal and laboratory study:
What is tested?
What are the Disadvantages?

A

*Animal research uses animals to test potential pharmaceuticals prior to human trials
* Its application is limited considering the difference between human and animal physiology, not ethical
* Experiments are undertaken in a highly controlled environment

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6
Q

What is case-control study?

A

-Retrospective in nature
- Well-designed
- Cases have outcome of interest whereas control lacks the outcome of interest
- Determines which factors contribute to an outcome
e.g. Why some patients need longer intensive therapy than others for tuberculosis

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7
Q

What are the pros and cons of case-control study design

A

Advantages:
* Less expensive
* Easier to do and take less time
* Useful when obtaining follow-up data that is difficult to obtain due to the nature of population being studied
* More efficient if the disease is rare
* This design may be the only ethical way to evaluate
something

Disadvantages:
* Potential recall bias
* Subject to selection bias
* Generally do not allow investigators to calculate an incidence or absolute risk

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8
Q

What are Cross-sectional studies?

A
  • An observational study design where outcomes and exposures are measured concurrently (at the same time)
  • Participants are selected based on set inclusion and exclusion criteria.
    *Mainly used in Population based research
  • There is a target population and a small study sample is taken from this

e.g. Testing if there is correlation between smoking and COPD, both are tested at same time
Exposed and outcome present = Has both
Exposed and no outcome = Smokes but no COPD
Unexposed and outcome= X smoke has COPD etc

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9
Q

What is a Randomised Controlled Trial (RCT)?

A

-Prospective study*
- Randomly assigned subject to receive control (treatment/placebo)/intervention

*A research study that follows over time groups of individuals who are alike in many ways but differ by a certain characteristic

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10
Q

What are the Advantages and Disadvantage of Randomized Controlled Trials (RCT) ?

A

Advantages
* Considered the gold standard
* This design allows for washout of most population bias
* Reduced influence by confounders
* Reduced variability in the outcome(s)
* Easier to blind patients than observational studies

Disadvantages
* More time consuming
* More expensive

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11
Q

What is a Systematic Review?

A
  • Identify relevant research studies (A literature review) done by researchers

-To locate, appraise and synthesise the best available evidence(relating to a specific research question)
- To provide informative and evidence-based answers.
- Combine with professional judgment to make decisions about how to deliver interventions or to make changes to policy.

Highest level of evidence, secondary evidence
Follows rigorous protocol
Collects all known research

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12
Q

Systematic review Vs Meta Analysis:

A

Systematic- Only to identify relevant research
Meta- Gather information from research then carry out a statistical test

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13
Q

What is a Meta-Analysis?
What is the adv and disadv?

A

The use of statistical methods to summarise the results of independent studies. By combining information from all relevant studies, meta-analysis can provide more precise estimates of the effects of health care than those derived from the individual studies included within a review.

Advantage
* Objective evaluation of research findings

Disadvantage
* Not all topics have sufficient research evidence to allow a meta-analysis to be conducted

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14
Q

What are some advantages of Guidelines (e.g., NICE guidelines)

A
  • Are on topic of relevance to population (usually determined by NHS England or the Department of Health and Social Care).
  • Thorough and transparent development process to ensure fairness.
  • Representative stakeholders involved in the process.
  • Guidelines are developed using thorough literature reviews of many RCTs and other forms of evidence.
  • Directly applicable to patients.
  • Often take into account a huge number of potential treatments and drug classes, e.g. type 2 diabetes guidelines.
  • Complex issues simplified.
  • Regularly reviewed.
  • Take into account cost considerations.
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15
Q

What are the Disadv of Guidelines (e.g., NICE guidelines)?

A
  • Many resources so a lot of literature review to do
  • Conflict of interest sometimes
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16
Q

Sources of evidence:
What is at the top? (most reliable)
Bottom?

A

Levels
1- Clinical Practice Guidelines, Meta-Analysis Systematic reviews , RCT’s
2. Cohort studies
3. Case control studies
4. Case report or Case series
5. Narrative reviews, expert opinions, editorials
Animal and Lab studies

Top= Clinical Practice Guidelines, Meta-Analysis Systematic reviews , RCT’s
Bottom= Expert opinion, animal and lab studies Narrative reviews, editorials

17
Q

What are endpoints?

A

An endpoint is an event or outcome that can be objectively measured in a study.
* Endpoints should be pre-defined and, ideally, sufficiently powered (sample size is representative of population)

18
Q

What is meant by a Primary endpoint ?

A

The main result/s that is measured at the end of a study to see if a given intervention was effective – needs to be clinically relevant and must always be powered.

e.g If a pt has quit smoking or not

19
Q

What is a Secondary endpoint?

A

These are additional events of interest, but which the study may not be specifically powered to assess.
e.g. Smoking cessation has improved QOL or Stamina when the main outcome is abstinence

20
Q

What are Patient-oriented endpoints? (POEs)

A

An ideal endpoint should be a valid and applicable measure of how a patient feels, functions or survives.

POEs measure this directly, e.g. number of fractures, strokes, myocardial infarction, deaths, caner recurrence, pain levels, number of migraines, etc.

21
Q

What are the Advantages of POEs? (3)

A

They are measures of true patient benefit
clinically meaningful
clinical certainty

22
Q

What are Disadvantages of Patient Oriented Endpoints?

A

Need large sample size
Longer trial duration
More expensive
Delay in potentially beneficial medicines coming to market

23
Q

What are DOEs?

A

Disease oriented endpoints (DOEs)

DOEs – also known as “surrogate” endpoints: do not directly measure how a person feels, functions or survives, but which should be so closely associated with a clinically meaningful endpoint that they are taken to be a reliable substitute for them

e.g. blood sugar, blood pressure, cholesterol levels, bone mineral density.

24
Q

What are examples of DOE’s?

What are the advantages and disadvantages of DOEs?

A

Blood sugar, blood pressure, cholesterol
levels, bone mineral density.

Advantages:
smaller sample size, shorter trial duration, less expensive, expediates medicines to market.
* Disadvantages: May not relate to clinically meaningful outcomes, may not change at all stages of disease, may predict that harmful treatments are
effective.

25
Q

Examples of Disease vs. Patient Oriented Endpoints

A
  • DOE: Beta-carotene and vitamin E are good antioxidants
  • POE: Neither vitamin prevents cancer or cardiovascular disease
  • DOE: Anti-arrythmic drug X decreases the incidence of premature ventricular contractions on ECGs
  • POE: Anti-arrythmic drug X is associated with an increase in mortality
26
Q

Define:
Absolute Risk Reduction (ARR) and
Relative Risk Reduction (RRR):

A
  • ARR: straightforward difference between event
    rates.
    In trial 1, 40% - 30% = 10%
    In trial 2, 10% - 7.5% = 2.5%
  • RRR: the difference in event rates expressed
    relative to what you started with.
    In trial 1, 10%/40% = 0.25 or 25%
    In trial 2, 2.5%/10% = 0.25 or 25%
27
Q

What is the Number needed to treat (NNT)?
How is it calculated

A
  • The NNT estimates the number of patients
    we need to treat for one patient to benefit.
  • NNT = 100/ARR(%). (Absolute Relative Risk)
  • In trial 1, NNT = 100/10 = 10
  • In trial 2, NNT = 100/2.5 = 40
28
Q

Describe THREE (3) strengths and THREE (3) limitations of randomised controlled trials for assessing medicine safety and efficacy.

A

Strengths:
1. Reduced variability in outcomes
2. Eliminates/ washes out population bias
3. Easier to blind patients than observational studies

Limitations:
1. More time consuming
2. More expensive
3.

29
Q
  • Of all people using ramipril, 5% will have a heart attack in the next 5 years. In people using ramipril + amlodipine, the percentage is 4.2%. Which of the following is correct?
    a. Number needed to treat is 1.25
    b. Number needed to treat is 125
    c. Number needed t treat is 20
    d. Number needed to treat is 23.8
A

ARR= 5-4.2= 0.8
NNT = 100/0.8 = 125
So, B is correct

30
Q

What is the Relative Risk Reduction? (RRR)

A

-The difference in event rates expressed relative to what you started with. (smaller/bigger)
-Ratio

The ARR/control group

31
Q

What is Absolute Risk Reduction? (ARR)

A

-Straightforward difference between event rates of Trial 1 and 2 (Control and Treatment)
- Subtract

32
Q

What is NNT?
How is it calculated?

A

-Numbers needed to treat- estimate the number of patients we need to treat for one patient to benefit.
-100/ARR (%)

33
Q

What is a cohort study?

A
  • Almost always prospective, but sometimes can be retrospective cohort studies
  • Some are exposed subjects and others are unexposed to a certain intervention to see if they develop an outcome. e.g give some patients a medication/counselling session
34
Q

What are the advantages of cohort studies? (3)

A
  1. Can more clearly show the time of exposure and development of the outcome because the subjects are without the disease at baseline.
  2. Allows for evaluation of more than one outcome as it relates to an exposure
  3. Allows for calculation

An example of a cohort study in medicine would be a study that follows a group of individuals who are all exposed to a particular risk factor, such as smoking, and compares their outcomes to those of a group of individuals who are not exposed to smoking.

35
Q

Disadvantages of a Cohort study?

A
  • Can be expensive and time consuming of needing to follow a large number of people
  • Loss of follow up can begin to introduce bias
  • May not be good for rare diseases
36
Q

Examples of Cohort Studies:

A

British Doctor’s Cohort Study
Framingham Heart Study
Nurses’ Health Study
Physicians Health Study
Women’s Health Initiative

37
Q

Systematic review Vs Meta Analysis:

A

Systematic- Only to identify relevant research
Meta- Gather information from research then carry out a statistical test