1-Clincial Trials Flashcards

(26 cards)

1
Q

Define clinical trial

A

A planned experiment involving patients and designed to explain the most appropriate treatment for future patients with a given medical condition

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2
Q

What is the purpose of a clinical trial?

A

Provide reliable evidence of treatment efficacy and safety

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3
Q

Define efficacy

A

Ability of a health care intervention to improve the health of a defined group under specific conditions

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4
Q

Define safety

A

Ability of a health care intervention not to harm a defined group under specific conditions

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5
Q

What are the disadvantages of comparison with historical controls?

A

Less information about potential bias
Unable to control for confounders
Selection less rigorous

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6
Q

What needs to be defined before a trial starts?

A
Disease of interest 
Treatments to be compared 
Outcomes to be measured 
Possible bias and confounders
Eligible patients 
Patients who need to be excluded
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7
Q

What are the steps of conducting a trial?

A
Identify source of eligible patients 
Invite them to trial 
Consent willing patients 
Allocate patients to treatment 
Follow up patients identically 
Minimise losses to follow up 
Maximise compliance
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8
Q

What questions are asked when comparing results of a trial?

A

Is there an observable difference between treatment?
Could the difference have arisen by chance - statically significant?
How big is the difference between treatments - clinically important?
Is the observed difference attributable to the treatment?

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9
Q

What are the types of outcome in a trial?

A
Patho-physiological (tumour size/ECG change)
Clinically defined (mortality/morbidity) 
Patient focused (quality of life/satisfaction)
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10
Q

When are outcomes measured in a trial?

A

Baseline - for inadvertent differences in groups
During trial - side effects, is one group being disadvantaged
Final - effects of treatment

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11
Q

What does random allocation lead to?

A

Minimal allocation bias - each patient has equal chance of being allocated to each treatment
Minimal confounding - groups likely to be similar in size and characteristic due to chance

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12
Q

What are the potential consequences of open label treatment?

A

Behavioural effects - patient
Non treatment effects - clinician
Measurement bias - investigator

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13
Q

Define the placebo effect

A

Patients attitude/illness improves as they feel something is being done

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14
Q

Define a placebo

A

Inert substance appearing identical in every way to active form it is being compared to, aiming to cancel out placebo effect

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15
Q

How can compliance be maximised?

A
Simplify instructions 
Ask about side effects 
Measure compliance (urine/blood levels, tablet counts)
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16
Q

Define as treated analysis

A

Only including patients who have complied in any analysis

17
Q

What are the disadvantages of as treated analysis?

A
Loses randomisation (and therefore minimal allocation bias) 
Won’t address if there is a reason for low compliance such as negative side effects
18
Q

What is intention to treat analysis?

A

Analysis according to original allocation of treatment groups regardless of whether they complied or completed follow up

19
Q

What are the advantages of intention to treat analysis?

A

Compares likely effect of using treatment in routine clinical practice
Preserves effects of randomisation - minimal selection bias and confoundings

20
Q

What is the collective ethic surrounding human clinical trials?

A

Patients should have treatments properly tested for safety and efficacy

21
Q

What are the individual ethics in relation to human clinical trials?

A

Beneficence
Non maleficence
Autonomy
Justice

22
Q

What issues must be considered for a trial to be considered ethical?

A
Clinical equipoise 
Scientifically robust 
Ethical recruitment 
Valid consent 
Voluntariness
23
Q

What governs the ethics of clinical trials in the uk?

A

NHS trust and PCT R and D office

24
Q

What are the roles of clinical trials?

A

Testing the safety of drug
Phase I - proof of principle
Phase II - Proof of concept and dose range finding
Phase III - pharmoeconomics

25
Describe the regulatory process of clinical trials
MHRA give approval for human clinical trials Phase III data given to MHRA to be granted a product licence Wording of packaging and marketing regulated by MHRA, can remove product from market
26
What are the differences in phases I, II and III?
Phase I - first into man study, single/multiple ascending dose on small number of healthy volunteers to evaluate safety Phase II - Patients with disease but no co-morbidities to determine dose for therapeutic action Phase III - Large number of patients with disease and co-morbidities to determine efficacy against standard treatment/placebo