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Treatment

Nendyah Roestijawati
Ideas and evidence
Studies of treatment effects

Observational and experimental


studies
Experimental study cohort study
which conditions of study are
specified for making unbiased
comparisons
Selection treatment group
Nature of interventions
Management follow up
Measuring outcomes
Randomized Controlled Trials

Standard of excellence of scientific


study of the effects of treatment
Same as cohort study except that
treatment is assigned by
randomization rather than
physician and patient
Structure of RCT
Ethics

No compelling reason to believe that either


randomly allocated treatment is better than other
equipoise
Experimental intervention might be better than
control
Primary outcomes must be benefit cannot
randomly one is more harmful for other
Patients must fully understand the consequences of
participating the study
Trial must be stopped if there is effectiveness, harm,
futility in continuing
Sampling

To increase homogeneity, strengthen


internal validity, distinguish signal
and noise
Inclusion criteria : patients really do
have the condition being studied
strict diagnostic criteria
Sampling

Exclusion criteria :
Comorbidity
Not expected live long enough
Contraindications of one treatments
cannot randomized
Refuse to participate
Do not cooperated
Intervention

Generalizability can be easily


described and reproduced in other
setting
Complexity : single weak effects,
multifaceted more effective and
amenable
Strength : different from alternative
managements and reasonable to
expect that the outcome will be
affected
Comparison groups

No intervention
Being part of study Hawthorne
effect
Usual care
Placebo treatment placebo
effect
Another intervention current use
Changes of outcomes
Allocating treatment

Random allocation/randomization
free of confounding
Baseline characteristics
comparing frequency in treat and
control group
Stratified randomization patients
gathered into groups with similar
levels of prognostic factors
Baseline characteristic

Characteristics Liberal Restricted


Age 81.8 81.5
Male 24.8 23.7
Any cardiovascular disease 63.3 62.5
Tobacco use < 600 mg/d 11.6 11.3
Anaesthesiology risk score 3.0 2.9
General anaesthesia 54.0 56.2
Lived in nursing house 10.3 10.9
Stratified randomization
Differences arising after randomization

Patients may not have disease


Compliance
Cross-over
Co-interventions
Blinding

To minimize change behaviour


because they aware which
treatment received
Single blind and double blind
ambiguous
Open label/trial : no attempt to
blind
Assessment of outcomes

Composite outcome

Performance status Definition


0 Asymptomatic
1 Symptomatic, fully ambulatory
2 Symptomatic, in bed <50% of
the day
3 Symptomatic, in bed >50% of
the day
4 Bedridden
5 Dead
Assessment of outcomes

Treatment effects

Measure Definition
Relative Risk Control event rate - treatment event rate
Reduction Control event rate
Absolute Rate Control event rate treatment event rate
Reduction
Number needed 1
to treat or --------------------------------------------------------
number needed Control event rate treatment event rate
to harm
Efficacy and effectiveness

Efficacy trials (ideal) : patient offered


interventions, follow instruction, get best
possible care, do not care other disease
randomized
Effectiveness trials (ordinary): patient not
take the assigned treatment, doctor
and facility not the best
Implementation gap : difference
between efficacy and effectiveness
Result analysis

Best choice
treatment

Experiment
treatment is
better
Superiority and non inferiority trials

Superiority trials : one treatment better


than another
Non-inferiority trials : new treatment
unlikely to be less effective than current
treatment but in other studies to be more
effective than placebo inferiority
margin
Variations on randomized trials

Cluster randomized trials doctors,


hospitals, clinics, communities
Cross over trials control for
differences response not related
treatment effect
Tailoring results to individual patients

Subgroups
Effectiveness in individual patients
efficacious, effective, working,
benefits worth discomfort and risk
Trials N=1 rarely done and
publish
Limitations randomized trials

Does not settle specific question


Expensive
Phases of clinical trials

I. Identify a dose range that


tolerated and safe
II. Efficacious and relationship
between dose and efficacy
III. Randomized trials : efficacy and
rates of common side effects

Post marketing surveillance


Terimakasih

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