23.

Randomised controlled trial

Dept. Epidemiology
School of Public Health
Jining Medical College
12 May 2009
Classification of mothod in epidemiology

 Descriptive epidemiology

 Analytical epidemiology

 Experimental studies or clinical trial

(Randomised controlled trial,RCT)

 Destination

 To compare the effectiveness of a specific

treatment

 Preventional (or prophylactic) interventions

 Principle of RCT

Example:

Group A (new anti-cancer drug) Length of survival

Side-effects
Group B (current treat) Quality of life

Some trials use three or more groups

 Placebo: non- treatment called a placebo. A placebo is
effectively a dummy treatment which appears to be real.

 Blind:The patients do not know whether they have been

allocated to the group receiving a new treatment, or an
old one, or a placebo.

 Double-blind RCT: Neither the patients nor the medical

staff know which intervention had been allocated.
 Cross-over trial
 Subjects receive one intervention for a certain period,

and then be changed over to another intervention.

 Multi-centre trials
 Studying subjects at more than one site

 Increase the sample size

 Meta-analysis :
 To summarize the results of several trials
 To “pool together” the results of trials
 Overall picture of an intervention’s efficacy

Drawbacks:
(1)publication bias: postive outcome more likely to be
published.
(2)quality of studies, quality of life and cost-
effectiveness are not normally evaluated

Systematic reviews: gold standard of clinical evidence

Study design

 Eligibility criteria:
patients who are most likely to benefit
from the treatment
they will not represent the whole population
of patients

 End of the trial

 changes in subjects’ condition, death or
other physical status
Follow up:until the end-point is reached.

Sample: calculated using formula.

 Ethical issues :
Subjects should not be exposed to known
or potential hazards
Unacceptable to withhold treatment from
subjects in a control group

Treaty of Helsinke
IRB approval
Informed consent
 Subjects

 The sample of subjects should be

representative of the population as a whole.

Randomly allocated to a particular treatment arm.

To ensure that their personal characteristics

have no influence over the treatment arm

 Data collection

Agreed points throughout the trial.

To check patient compliance with any treatments given,
including placebos.
Information about many factors,
including any side-effects of treatment.
 Data analysis

For continuous data: hypothesis tests,

confidence intervals.

For categorical data: Chi-squared test,

relative risk and

number needed to treat.

For other outcome variables

 Some advantages and disadvantages of
randomized controlled trials
Advantages
Allows effectiveness of a new
treatment to be evaluated.
Provides strong evidence of
effectiveness.
Less prone to confounding than
other study designs.
Disadvantages
Expensive and complicated to
perform.
Patients may refuse treatment –
non-compliance can affect
results.
A large sample size is needed.
Careful attention to ethical issues
is needed.
Informed patient consent is
essential.