Summary
Reviewed
definitions
epidemiology
study of the distribution and determinants of health-related states or events
(including disease), and the application of this study to the control of diseases
and other health problems
clinical epidemiology
study of the patterns, causes, and effects of health and disease in patient
populations and the relationships between exposures or treatments and
health outcomes in order to guide evidence based clinical decision making
= biological science + clinical sciences + epidem + health services
research
data sources = protocol driven (specific to q eg. prospective cohort),
routine practice driven (eg. electronic health records, routine admin data),
combination (eg. patient cohorts, clinical registries)
design = randomised control trial (RCT) gold standard, also observational
where treatment given and observed as part of normal care
hypothesis
supposition, arrived at from observation or reflection, that leads to
refutable predictions - any conjecture cast in a form that will allow it to be
tested and refuted
alternative = that exposure is associated with outcome, only adopted if null
is proved implausible
null = that groups do not differ ie. exposure not associated with outcome
exposure
Summary 1
, exposure, risk factor, or other characteristic being observed or measured that
is hypothesised to influence an event or manifestation eg. smoking, age
outcome
the measured result/effect of an action/event eg. death, disease progression,
morbidity
clinical outcome assessments
definition = measurement of patients health status/treatment response
based on own perceptions > used to evaluate impact of intervention
on symptoms, functioning, health-related quality of life (QOL), well-
being, feeling
should be measured by specific condition eg. breast cancer (not
specialty/intervention) and include full overall cycle of care inc follow
up rather than single visit
1. clinician reported (ClinROMs)
completed by healthcare practitioner (HCP) based on clinical
judgement using standardised tools/guidelines for consistency, may be
objective (eg. BP) or subjective (eg. perceived mood)
eg. karnofsky performance status scale, hamilton rating scale for
depression
cons = subjective measures often reported inaccurately (observer
bias), eg. Stephens observing lung cancer care found that clinicians
underestimated symptom severity vs patient 15% time, with inc
disagreement between between patient and clinician ratings with inc
severity
2. observer reported (obsROMs)
completed by someone who regularly observes/interacts with patient
(eg. family) who can provide third party insight that the individual may
be unaware of, may be objective (eg. frequency of behaviour) or
subjective (eg. perceived mood)
eg. behavioural assessment scale for children
Summary 2
, cons = symptoms may be misinterpreted or inflated esp by caregivers
3. performance (PerfOMs)
direct objective assessment of patient’s ability to complete specific
task related to health status/function, measured by
HCP/caregiver/trained assessor with standardised tools/scoring
eg. timed up and go, grip strength
4. patient reported (PROMs)
patient’s experiences/preferences/treatment responses directly
reported by patient without interpretation/modification (digital tools,
standardised qs) = allows objective measurement of health related
QOL
(distinct from PREM (patient reported experience measure) which
looks at environment eg. how clean the hospital was, how nicely they
were treated)
types
generic = perceptions of general wellbeing relating to
physical/social functioning, pain, anxiety, depression eg. Hospital
Anxiety & Depression Scale, EQ-5D (global QOL, used in
population studies), SF-36, Brief Pain Inventory, Female Sexual
Function Index
disease specific = perceptions of health related to disease with
qs/scales sensitive to small changes eg. EORTC (european
organisation for research and treatment of cancer): QLQ-C30 (core
module for cancer with specific modules)/QLQ-OV28/QLQ-CX24 +
FACT-G/FACT-O (functional assessment of cancer therapy)
currently only used in research to monitor effects of experimental
treatment to see if benefit>side effects (SEs), but significant evidence
to support routine use eg. morris survey of oncologists found 80%
believed hrQOL should be routinely collected:
at individual level, help patients highlight symptoms they may not
have thought to raise, with comparison of baseline/follow up qs
Summary 3
, useful to track disease/treatment progression > also evidence of
utility as prognostic tool in conditions from depression to cancer
eg. study on 282 cancer patients found strong prognostic
association between PROMs of physical function, fatigue,
depression, anxiety, pain with both overall and hosp-free survival,
with sys review also highlighting global QOL, emotional/social role,
constipation, anorexia, nausea and cog function as prognosticators
at a broader level, compiled data can be used to inform other
patients about benefits/SEs of treatments to support shared
decision making eg. 2022 cochrane review found patient-
perceived HCP communication to sig increase by ave 36% when
PROMs used = enhance patient engagement/adherence and
satisfaction
systemically, allows benchmarking/auditing to identify and improve
poor clinical performance to cost effectively improve outcomes =
key focus for NHS for value based healthcare (VBHC)
to support this implementation into routine care, several areas of
focus:
training and education, inc education materials for patients,
training for HCPs, and admin support to facilitate integration to
EHRs/workflow (avoid increasing clinician and patient burden)
national consensus on which PROMs to collect, when, and what
the trigger scores are that flag for further clinical management,
with focus on equal accessibility throughout (language barriers?)
endpoint
outcome that can be measured objectively to determine whether the
intervention is beneficial
clinical (eg. overall survival/mortality) vs surrogate (event implying
outcome eg. cease of med records)
singular or multiple/composite markers
patient (eg. symptoms) vs disease (eg. biomarkers)
Summary 4
Reviewed
definitions
epidemiology
study of the distribution and determinants of health-related states or events
(including disease), and the application of this study to the control of diseases
and other health problems
clinical epidemiology
study of the patterns, causes, and effects of health and disease in patient
populations and the relationships between exposures or treatments and
health outcomes in order to guide evidence based clinical decision making
= biological science + clinical sciences + epidem + health services
research
data sources = protocol driven (specific to q eg. prospective cohort),
routine practice driven (eg. electronic health records, routine admin data),
combination (eg. patient cohorts, clinical registries)
design = randomised control trial (RCT) gold standard, also observational
where treatment given and observed as part of normal care
hypothesis
supposition, arrived at from observation or reflection, that leads to
refutable predictions - any conjecture cast in a form that will allow it to be
tested and refuted
alternative = that exposure is associated with outcome, only adopted if null
is proved implausible
null = that groups do not differ ie. exposure not associated with outcome
exposure
Summary 1
, exposure, risk factor, or other characteristic being observed or measured that
is hypothesised to influence an event or manifestation eg. smoking, age
outcome
the measured result/effect of an action/event eg. death, disease progression,
morbidity
clinical outcome assessments
definition = measurement of patients health status/treatment response
based on own perceptions > used to evaluate impact of intervention
on symptoms, functioning, health-related quality of life (QOL), well-
being, feeling
should be measured by specific condition eg. breast cancer (not
specialty/intervention) and include full overall cycle of care inc follow
up rather than single visit
1. clinician reported (ClinROMs)
completed by healthcare practitioner (HCP) based on clinical
judgement using standardised tools/guidelines for consistency, may be
objective (eg. BP) or subjective (eg. perceived mood)
eg. karnofsky performance status scale, hamilton rating scale for
depression
cons = subjective measures often reported inaccurately (observer
bias), eg. Stephens observing lung cancer care found that clinicians
underestimated symptom severity vs patient 15% time, with inc
disagreement between between patient and clinician ratings with inc
severity
2. observer reported (obsROMs)
completed by someone who regularly observes/interacts with patient
(eg. family) who can provide third party insight that the individual may
be unaware of, may be objective (eg. frequency of behaviour) or
subjective (eg. perceived mood)
eg. behavioural assessment scale for children
Summary 2
, cons = symptoms may be misinterpreted or inflated esp by caregivers
3. performance (PerfOMs)
direct objective assessment of patient’s ability to complete specific
task related to health status/function, measured by
HCP/caregiver/trained assessor with standardised tools/scoring
eg. timed up and go, grip strength
4. patient reported (PROMs)
patient’s experiences/preferences/treatment responses directly
reported by patient without interpretation/modification (digital tools,
standardised qs) = allows objective measurement of health related
QOL
(distinct from PREM (patient reported experience measure) which
looks at environment eg. how clean the hospital was, how nicely they
were treated)
types
generic = perceptions of general wellbeing relating to
physical/social functioning, pain, anxiety, depression eg. Hospital
Anxiety & Depression Scale, EQ-5D (global QOL, used in
population studies), SF-36, Brief Pain Inventory, Female Sexual
Function Index
disease specific = perceptions of health related to disease with
qs/scales sensitive to small changes eg. EORTC (european
organisation for research and treatment of cancer): QLQ-C30 (core
module for cancer with specific modules)/QLQ-OV28/QLQ-CX24 +
FACT-G/FACT-O (functional assessment of cancer therapy)
currently only used in research to monitor effects of experimental
treatment to see if benefit>side effects (SEs), but significant evidence
to support routine use eg. morris survey of oncologists found 80%
believed hrQOL should be routinely collected:
at individual level, help patients highlight symptoms they may not
have thought to raise, with comparison of baseline/follow up qs
Summary 3
, useful to track disease/treatment progression > also evidence of
utility as prognostic tool in conditions from depression to cancer
eg. study on 282 cancer patients found strong prognostic
association between PROMs of physical function, fatigue,
depression, anxiety, pain with both overall and hosp-free survival,
with sys review also highlighting global QOL, emotional/social role,
constipation, anorexia, nausea and cog function as prognosticators
at a broader level, compiled data can be used to inform other
patients about benefits/SEs of treatments to support shared
decision making eg. 2022 cochrane review found patient-
perceived HCP communication to sig increase by ave 36% when
PROMs used = enhance patient engagement/adherence and
satisfaction
systemically, allows benchmarking/auditing to identify and improve
poor clinical performance to cost effectively improve outcomes =
key focus for NHS for value based healthcare (VBHC)
to support this implementation into routine care, several areas of
focus:
training and education, inc education materials for patients,
training for HCPs, and admin support to facilitate integration to
EHRs/workflow (avoid increasing clinician and patient burden)
national consensus on which PROMs to collect, when, and what
the trigger scores are that flag for further clinical management,
with focus on equal accessibility throughout (language barriers?)
endpoint
outcome that can be measured objectively to determine whether the
intervention is beneficial
clinical (eg. overall survival/mortality) vs surrogate (event implying
outcome eg. cease of med records)
singular or multiple/composite markers
patient (eg. symptoms) vs disease (eg. biomarkers)
Summary 4
