Evaluating Patient-Based Outcome Measures - NIHR Health ...

The rapid expansion of efforts to assess
outcomes of health care from the patient’s
perspective has resulted in hundreds of instruments
that have in common that they purport
to provide standardised assessments of matters
of importance to patients such as functional
status, subjective health and broader aspects of
health-related QoL. Seven major types of instrument
can be distinguished: disease-specific, site
or region-specific, dimension-specific, generic,
global or summary, individualised, and utility.
These distinctions between types should not
be viewed as rigid since instruments can have
properties associated with more than one kind.
Given that the vast majority of such instruments
are candidates for inclusion in trials, investigators
facing the need to select an instrument or
instruments to include for any specific trial
have quite a daunting decision.
There are substantial areas of uncertainty and
dispute regarding outcome measurement. Over a
number of issues, gaps and limitations of concepts
and measurement have been acknowledged in the
literature. This review has built on and attempted
to integrate previous efforts to identify desirable
properties of patient-based outcome measures.
Chapter 4
Conclusions
Health Technology Assessment 1998; Vol. 2: No. 14
It is very encouraging that authors from three
disciplines of social science, economics and
statistics can agree to this document; this is itself
an important step in progress to define the field.
Broad assent to the principles of the review was
also obtained from a wide range of disciplines and
expertise relevant to health technology assessment
and health services research: comments on a draft
were sought from those with expertise in clinical
medicine and clinical trials, health economics,
health service research, psychology, sociology,
statistics. Every effort was made to respond to
and integrate expert advisors’ suggestions. We
feel that the resulting document presents views
based on substantial consensus about issues.
Despite clear limitations in the evidence available
to date, it is possible to conclude that there are
eight criteria that can provide an explicit framework
for decisions about selection of patient-based
outcome measures in trials. In determining how
best to assess outcomes from the patient’s perspective
in the context of a clinical trial, investigators
need to consider candidate patient-based outcome
measures in terms of appropriateness, reliability,
validity, responsiveness, precision, interpretability,
acceptability and feasibility.
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