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05
Efficacy Parameters
Major efficacy parameters for most
disease are well established
but
Definite protocol should select those
tests and parameters within the
limits of resources available to the
clinical trial e.g.

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 Choosing efficacy
parameters
1. Objective measurements that
have been validated and widely
accepted
e.g.
- B.P. reading
- Insulin serum
- Glycosolated haemoglobin
- Lipid ratios

- Direct visualization and

measure of lesion
e.g. Ulcer – endoscopy
arterial blockage - angio
tumor size – X Ray or PET
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1. Reduction in the progress or even
reversal of the disease
e..g reduction in the severity of lesions
reduction in manifestation of the disease
reduction in duration of the disease
• Improved symptomatology of the
patient
• Improvement of biochemical and lab
parameters
• Improved “Quality of Life”
• Clinical global evaluation by the
Doctor and patient
• Reduced mortality in those trials
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where this parameter has relevance.

Note :

The +ve and –ve points of each

efficacy parameter incorporated in
the protocol must be evaluated
against the increase, it will cause to
–
d. Time requirements
e. Personal efforts
f. Financial costs
g. Additional complexity especially to
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the statistical evaluation of the final

What are the best
parameters?
Which parameter to select in a
protocol ?
C. Combination of parameters that are practical
and make sense to measure should be the best.
Parameters of different targets
• Objective – Clinical
• Subjective – symptomatic
• Biochemical
• Pathological
• Pharmacological
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What are the parameter that determine
better efficacy over standard
treatment?
DIRECT
a. Time of onset
d. Magnitude of effect
e. Duration of effect
f. Decreased rate of relapse
g. Improved quality of life
INDIRECT PATIENT COMPLIANCE
i. Decreased number of tab/day or dose / day
j. Better taste
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k. Lower cost
 Factors affecting efficacy

a. Physical position
b. Relax or exercise
c. After cold or warm bath
d. Patient empty their bladder
e. Prior to meal time
f. Tests at various time of the day
(Circardian rhythm)

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 Example disease where
parameter to measure is clear
Hypertension - B.P.
reading
Here these are many
subparameters -
d. Fall in B.P. of 10 mm or more
e. Fall in B.P. to desired level –
Normal
f. Fall in B.P. over x no. of week
g. Maintenance of B.P. over y no.
of months
h. Maintenance of normal level BP
for z no. of months
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 Example of disease when
parameter to meet is more
complicated
e.g. post myocardial infarction – treated to
prevent re-infarction and sudden death
- Follow up for reinfarction – angiography
- No of deaths after 1 year – 2 year

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 Efficacy Parameters in
Preventive Therapy
• Prevention of disease : e.g. Vaccine
• Prevention of episodes of disease
i.e. number, duration or severity of
episode is decreased e.g. asthma,
epilepsy.
C. Prevention of progress of underlying
disease.
R1 to prevent more serious sequelae
of this disease.
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 Preventive Therapy
A. Vaccine – Efficacy Parameter

a. Prospective Epidermiology Protocol

b. Prospective & Retrospective protocol
c. Protective level

G. Preventive of Episode of disease during last 1

week / 1 month how many episode has the
patient experienced

0
1
2-5
6-10 1 YR DCR 2.4.05
 Other Efficacy Parameters
A. “Escape medication” e.g. gastric ulcers,
asthma, knowingly incorporated into the
protocol. Use Antacid sos or ventoline
sos evaluate the frequency of use of
escape medication under treatment with
the trial during as compared to the
baseline or in case of crossover design
the placebo period.
• Decrease in dose of supportive therapy
• 5 years survival in case of cancer
therapy 1 YR DCR 2.4.05
 Criteria to measure efficacy

1. All or none – Presence of absence

criteria
2. Graded criteria applied to
a. Subjective

as well as
b. Objective symptoms and signs

5. Global criteria
a. Dr. evaluation
b. Patients evaluation

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 Grading Systems for efficacy
parameters

• 0 Not at all B. 0 Absent

1 Mild 1 Slightly
present
2 Moderate 2 Clearly
noticeable
3 Markedly 3 Definitely
present
4 Severe 4 Continuous &
gross 1 YR DCR 2.4.05
0 Absent
1 Rarely present (occurs less
than _____times per___)
2 Occasionally present (occurs less
than __ times)
3 Often present (occurs __ to __
times per ___)
4 Almost always present (occurs
more than __ times per __)
e.g. asthma attack

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 Extended parameters e.g. asthma

No. of attacks per ____

frequency
How severe was the attack
severity
How long did last attack last
duration

(each of these parameter can also be

graded)

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Type of End points used to
measure efficacy
1. Time for a parameter to improve by
a fixed percent (50% or 75% or
100%)
2. Magnitude of improvement at a
fixed time i.e. (after 1 week, 1
month)
3. Duration of improvement while on
maintenance therapy
4. Time to recurrence of symptoms
after treatment is stopped
5. Degree of recurrence of symptoms
after treatment is stopped
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 Methods of Evaluating efficacy
parameters
Compared to baseline or placebo
C. Active drug
E. Baseline or placebo or comparative
drug
• Ratio :A
B
9. Incremental change : A minus
B
• Proportionate change : A-B
B
4. Percent change 1 YR DCR 2.4.05
: A-B x
 Efficacy parameter with
drawing
Eg. Gastric ulcer
End Point
100% Drug A = Clinical Symptom
0% of patients Drug B
With healed = Endoscopy
Ulcers
0
Time
100% Drug A
0% of patients
With ulcer Drug B = Endoscopy
Recurrence
0

Time 1 YR DCR 2.4.05

 VISUAL ANALOG SCALES
Provide quantitative measures to subjective rating e.g.
stress, anxiety, alertness, depression, hunger, itch
Pain parameter has been proved to be reliable
measured by these scales.
Date Time of assessment
Score
0 10
No pain Very
severe
pain

0% 25 % 50 %
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75% 100%
 Patient Diaries
Date from Patient Diaries give important
efficacy information in outpatient
evaluation e.g. antiepileptic treatment
evaluation anti diarhoea treatment.

- The diaries should be designed to make it

user friendly.
- Instruction for filling the data should be
discussed with the patient and printed on
the diary.
- The diary should be kept with the patient all
time i.e.
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 Quality of Life
There are a few formal definitions of
this term
The term “Quality of Life” is used
loosely by many
We shall only discuss health related
Quality of Life

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Schipper et al (1990)
“Quality of life represents the functional
effect of an illness and its consequent
therapy upon a patient as perceived by
the patient”

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 Four areas contribute
to the overall effect
1. Physical and
occupational
function
2. Psychologic state
3. Social interaction
4. Somatic sensations
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 Components of Quality of life Tests
I Physical and occupational
b. Capabilities
c. Dysfunction
d. Amount of time at works
e. Work status
VI. Psychological
g. Neuropsychologic function (eg.
Perceiving, thinking and remembering)
h. Emotional state (eg. Anxiety, anger,
depression)
i. Personal productivity
j. Intimacy and sexual function
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I. Socia l
b. Number of contacts per week / month
c. Quality of social contacts at home, school,
job
d. Quality and quantity of social contact with
friends
e. Interaction at work
f. Recreational activities
VII. Somatic s ensations
h) Sleep
i) Pain
j) Bowel movements etc.
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Is an End point an End point?
e.g. Blood pressure measurement

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