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DHP Research 2012 To discuss any points raised in this paper contact: info@dhpresearch.com
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DHP Research 2012 To discuss any points raised in this paper contact: info@dhpresearch.com
www.dhpresearch.com
A PROM should be designed to provide information around a given concept which must be
made explicit by the instruments authors.
Common concepts include, health status,
health-related quality of life (HRQoL), QoL,
well-being, treatment satisfaction, symptoms
and functioning.
Health related quality of life (HRQoL) Often referred to as the degree to which the treatment
and the disease as perceived by the individual to impact
on those aspects of their life - in addition to health
which are considered important5.
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Developing a measurement
strategy
An effective way to establish the link between the measured outcome such as the patients well-being following
an intervention programme is to develop a measurement
strategy, which requires a clear understanding of the
disease and the outcomes relevant to the disease area
and patient (Figure 1).
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Conceptual model
Reduce recurrent
hypoglycaemia
Secondary endpoint
Improves QoL assessed by
PROM
Figure 2. Endpoint model showing relationship between treatment effects and outcomes
DHP Research 2012 To discuss any points raised in this paper contact: info@dhpresearch.com
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DHP Research 2012 To discuss any points raised in this paper contact: info@dhpresearch.com
www.dhpresearch.com
A cautionary note:
Reliability tells how consistent our results are, whilst validity tell us whether
we are measuring what we think we are.
Reliability
Because PROMs are often used in clinical trials to measure change over time, the adequacy of a PROM for a variety of clinical applications depends on its reliability or
ability to provide consistent and reproducible estimates
of true treatment effects. For this we need to look for
evidence of good test-retest reliability.
Test-retest: Can be established by the administration of the PROM at different time points at a time
interval which is long enough with stable patients to
minimize memory effects. This is usually obtained
using correlation analysis of the two sets of scores
where the coefficient should not only be statistically
significant but, also high e.g. >0.80.
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PROMs can be applied to obtain data from the patients perspective in a range of areas and applications.
PROMs play an increasingly significant role in clinical research such as in randomized control Phase III trials as
secondary endpoints to provide added value to the primary biomedical outcomes.
Commercially, data derived from PROMs can for example,
be used to support product differentiation, provide intelligence for the targeting of drug development and marketing and in some cases support labelling claims.
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data
Interpretation of data derived from a PROM
can be challenging, particularly with regard to
understanding the meaning of what a change
or difference in a score means clinically.
Interpretation of PROM data is linked with both the objectives of the study and the constructs measured by the
PROM and as a consequence should be a key factor in the
development of any measurement strategy.
Understandably, optimising the patients health is of primary importance for the clinician and therefore, is more
likely to look for improvements in health status. However, what might be of specific interest to the clinician in
terms of outcome may not always correspond to what is
of relevance to the patient.
Remembering that PROMs are assessing outcomes from
the perspective of the patient, any change in the score
may or may not be related to a clinical outcome. As an
example, patients with diabetes who have changed to a
different insulin treatment considered more convenient
but, in all other respects is the same are unlikely to
report any improvement in clinical outcomes but, may
well report that their QoL has been enhanced as a consequence of the greater flexibility in their lives.
Despite the many thousands of PROMs developed and
their application, there is often lacking an understanding
as to what a PROM score represents and what is a meaningful change in score11. Should the differences be big or
small and what are the implications for clinical practice
and health policy?
When large samples (macro level) of patients are studied, differences in PROM scores between patient groups
might be numerically small but, highly statistically significant. However, data obtained at the macro level are difficult to apply at the individual patient (micro) level12.
Interpretability is also something that cannot be established from a one-off study but, is based on a body of
evidence developed over time through a variety of studies, and perspectives11. Nevertheless, there are a number
of approaches that that can aid the interpretation of
PROM data and which are summarised below11.
Statistical significance The statistical significance of the probability of treatment (A) is better than treatment (B).
Effect size A way of quantifying the difference between two groups of patients that has
many advantages over the use of statistical significance alone and emphasises the size of the
difference rather than confounding this with
sample size.
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3.
This will combine a number of factors including development of the measurement strategy and a detailed and comprehensive review of the literature to
define exactly what the measured outcomes are.
This is the point at which selection must be based on
the purpose of using a PROM, its scientific and
measurement qualities and whether its possible to
easily interpret the scores as to really what they
mean. Selection must not be based on the name of
the PROM or because it has been used in other studies.
Ultimately, we are only scratching the surface here when
it comes to the implantation of a PROM. The key comes
down to this: You have to plan for measurement. It is
almost impossible to measure patient reported outcomes
without a clear measurement strategy defining exactly
the inputs and desired outcomes.
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References
1.
Zigmond AS, Snaith RP (1981) The hospital anxiety and depression scale. Acta Psychiatr Scand
67: 361-370
2.
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number of scientific journals. He also lectures at undergraduate and post graduate level at the Universities of
Brighton, London and City.
Between 2003 and 2008 Keith was Associate Director of
the North East London Consortium For Research & Development (NELCRAD), where he was responsible for strategy development, capacity building and increasing primary care research across east London.
Keith is author of the Diabetes Health Profile (DHP), a
diabetes-specific questionnaire designed to identify the
psychological and behavioural impact of living with diabetes.
Literature reviews and evaluation of existing patient reported outcome (PRO) measures
Qualitative interviews to derive appropriate content for PROs
Write academic scripts
Training in PROs and research methodologies
Development of patient reported outcomes
(PRO)measures
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