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0:00
Hello, I'm Allan Hackshaw,
and I'm going to give
a short session about
assessing efficacy in the
context of clinical trials.
0:10
In this short session,
we're going to cover how
efficacy is defined,
what makes an efficacy
measure 'good',
how it's evaluated,
and I'll end with a
very simple example
of evaluating efficacy.
0:25
Efficacy is a measure of
clinical or public
health benefit.
It's usually quite well defined,
often very obvious such as
death or the development
of a disorder
such as heart disease,
stroke or cancer,
and it can be
measured in many ways
depending on the
disorder of interest.
It can be used to reflect
chronic symptoms and
other disorders that are
similar that last a long time.
Benefit is a very general
word that means do we reduce
the risk of something
bad happening such as
a clinically relevant
disorder or death,
or do we improve or even
stabilize symptoms?
1:03
Measures of efficacy
often depend
on the different disease
types and states.
Two extreme ones
are acute disorders
such as advanced
cancers, heart attack,
strokes where people
often die from
that disorder or there
are chronic disorders
such as asthma,
psoriasis, depression
that people have for
most of their lives.
Individuals with acute or
chronic disorders may want
different impacts on them
from different interventions.
When we measure
efficacy it's not
just the point of view of
the health professional,
but also what
matters to patients.
The expectations of
benefit also differ
between different
types of disorders and
different types of people,
such as healthy people,
are we looking at
new interventions to
prevent something bad
happening later on,
or are we looking
to treat people
who already have the
disorder of interest?
Their expectations or
what they want out of
efficacy and interventions may
be quite different
to each other.
There's no simple
single rule in terms
