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Including Patient-Reported Outcomesand Patient-Reported Resource-Use
Questionnaires in Studies
Brian W. Bresnahan, PhD, Sean D. Rundell, DPT, PhDAc
FrStCoW20thdebr
ªht
Rationale: More efficient and better informed healthcare systems are expected to have improved knowledge of the impact of interventions
on patient outcomes and resources used by patients and providers in specific health conditions.
Objectives: To describe trends related to putting patients at the center of healthcare decision making, regulatory trends and best practicerecommendations for developing high-quality patient-reported outcomes (PROs), and strategic issues related to including PROs in studies.
Materials and Methods: We summarize PRO concepts, definitions, and broadly-accepted scientific standards for developing, assessing,
and interpreting PROs. Three conceptual models are presented as examples for assessing PROs in relation to other outcomes. We discussdifferent perspectives for stakeholders, including regulatory issuespertaining to formal guidance forPROdevelopment and for use in trials.We
provide examples of PROs used in studies for assessing health outcomes in oncology and resource-use outcomes in low back pain patients.
Results: Psychometric scientists working closely withmulti-disciplinary teams and regulatory authorities have greatly improved the science of
collecting, assessing, and understanding patient-reported outcomes in clinical trials. A simplified framework is presented for strategic consid-erations for including PROs in studies, such as the appropriate timing for PROendpoints. Askingpatients about their health status and/or use of
resources improvesour understanding of how interventions andcareprocessesmay impact their lives and their budgets.Weprovide examples
from a back pain trial of patient-reported resource-use questionnaires for medicines taken and other services or products used by patients.
Conclusions: Healthcare stakeholders are placing increased emphasis on resource use and the impact of interventions on patients,
including effects associated with diagnostic tests. Patient-reported outcomes are being used in clinical practice and in clinical research,
supported by formal best-practice guidelines. Radiology has a role as an engaged stakeholder in the design, conduct, and interpretation of
patient-based evidence, and in its relevance to health policy implementation.
Key Words: Patient-reported outcomes; clinical studies; radiology; endpoints; health outcomes.
ªAUR, 2014
Patient-reported outcomes (PROs) provide valuable
health information. Patients, providers, and health
systems are being encouraged and incentivized to
involve patients and their families more directly in health
care decision making. Examples of support for additional ev-
idence development related to patient outcomes in the United
States include the creation of the Patient-Centered Outcomes
Research Institute (PCORI) (1), published priorities in
comparative effectiveness research (CER) (2–4), and the
Agency for Healthcare Research and Quality’s (AHRQ’s)
emphasis on patient-centered outcome research (5). ‘‘Pa-
tient-centered outcomes research (PCOR) helps people and
ad Radiol 2014; 21:1129–1137
om the Department of Radiology, University of Washington, 1959 NE Pacificreet, Seattle, WA 98195 (B.W.B., S.D.R.) and Comparative Effectiveness,st and Outcomes Research Center, University of Washington, Seattle,ashington (B.W.B., S.D.R.) Received April 1, 2014; accepted May 27,14. Funding: No external support was provided for the development ofis manuscript. Conflicts of Interest: No direct conflicts of interest areclared by the authors. Address correspondence to: B.W.B. e-mail:[email protected]
AUR, 2014tp://dx.doi.org/10.1016/j.acra.2014.05.008
their caregivers communicate and make informed health
care decisions, allowing their voices to be heard in assessing
the value of health care options’’ (1). Patient input is being
sought in developing research priorities, informing shared
decision-making models, selecting meaningful outcomes for
clinical research, and disseminating information to patients
and providers. Consequently, PROs and other relevant
patient-reported data have become more ingrained in clinical
research, observational research, clinical care, and quality
improvement initiatives.
In addition to engaging patients in research and in their health
care decision making, PROs are also valuable to more compre-
hensively measure treatment benefits and harms in clinical
research and CER. Manufacturers may choose to include
PROs in clinical trials to assess the impact of interventions on
patients, which has potential use in marketing their products.
Clinicians, health outcomes researchers, payers, or health policy
makers may desire PRO data to identify optimal strategies to
improve outcomes,whether related to increasingpositive health
effects in patients or reducing harms and/or negative effects.
In this article, we provide a general overview of PROs and
published recommendations for their inclusion in clinical
1129
BRESNAHAN AND RUNDELL Academic Radiology, Vol 21, No 9, September 2014
trials. We review definitions specific to PROs and identify US-
based and international guidance related to regulatory processes
and standards. Specifically, we outline recommendations associ-
atedwith evidence generation using PROs in studies to support
labeling claims (6–8). We use simplified graphics to depict
strategic issues, provide examples of patient-reported diaries
and PRO questions, and list practical aspects of including
PROs and resource-use questionnaires in research.
SCIENTIFIC STANDARDS FOR PROS
General Guidance on Including Patient-BasedOutcomes in Studies
The development of and appropriate use of high-quality
PROs require scientific expertise, time, and substantial
expense associated with questionnaire (instrument) develop-
ment, testing, validation, and refinement. Multiple disciplines
have collaborated in developing strong guidance on health
outcomes measurement, developing PROs and resource-use
instruments, and incorporating PROs in clinical research.
Extensive texts by experts in PRO development, analysis,
and interpretation provide guidance on designing instru-
ments, selecting instruments, and assessing responses (9,10).
These multidisciplinary works have focused on
� Defining health and health domains,
� Measuring physical, social, and psychological well-being,
� Unique issues in assessing mental status, pain, and general
health status and quality of life (QOL),
� Including QOL or other PRO scales in clinical trials,
� Scales, tests, and measures,
� Selecting and administering instruments,
� Analyzing, interpreting, and presenting PRO data,
� Distinct perspectives, including cultural and language is-
sues, and
� Issues and implications in health policy and health economics.
AUS Food andDrugAdministration (FDA)Web site descrip-
tively and graphically outlines their Clinical Outcomes Assess-
ment (COA) Qualification Program, including discussion of
four types of outcomes: PROs, clinician-reported outcomes,
observer-reported outcomes, and performance outcome mea-
sures (11). The educational information outlines the US FDA’s
COA-focused ‘‘wheel’’representing the components of PRO in-
strument development processes. It contains five spokes outlining
steps to follow in evaluating a ‘‘concept of interest for a claim:’’
� Identify content of use and concept of interest,
� Draft instrument and evaluate content validity,
� Cross-sectional evaluation of other measurement properties,
� Longitudinal evaluation of measurement properties/inter-
pretation methods, and
� Modify instrument.
Other resources are available toprovide scientific guidanceon
PRO measurement (12), recommendations for documenting
1130
PRO-based data via PROEvidence Dossiers, eliciting concepts
for PROs, and assessing respondent understanding (13–15).
Definitions and Desired Properties of PROs
More psychometric research has been conducted in the assess-
ment of patient-reported health outcomes, such as in symptom
severity or symptom impact scales and health-related quality-
of-life (HRQOL) measures, compared to research on the
best approaches to collecting resource-use data or financial
end points, such as patient expenditures or their willingness
to pay for specific interventions. Important data may not be
captured in electronic records or may be difficult to assess via
patient observation or physical examination. In the absence
of more objective tests or mechanisms (eg, blood pressure
monitor), we can ask patients how they feel, their levels of
symptoms, and how their symptoms impact their lives, as
well as their general ability to function in their personal lives
or in social settings. For patient-reported data, scientific princi-
ples have centered on using appropriate language, clarity, and
structure for asking questions, the scoring of and evaluation
of responses in instruments, analyzing PROdata in comparative
studies, determining clinical meaning and meaning to the pa-
tient for changes in outcomes, and assessing how representative
or generalizable findings may be for health systems.
Desired properties of patient-reported instruments are
rooted in methods of survey-based research and cognitive psy-
chology. The following are standard definitions for standard
characteristics of well-tested health outcomes measures (8):
� Measurement (psychometric) properties: All the attributes rele-
vant to the application of a PRO instrument including the
content validity, construct validity, reliability, and ability to
detect change (responsiveness).
� Content validity: Evidence from qualitative research demon-
strating that the instrument measures the concept of inter-
est including evidence that the items and domains of an
instrument are appropriate and comprehensive relative to
its intended measurement concept, population, and use.
Testing other measurement properties will not replace or
rectify problems with content validity.
� Construct validity: Evidence that relationships among items,
domains, and concepts conform to a priori hypotheses con-
cerning logical relationships that should exist with other
measures or characteristics of patients and patient groups.
� Reliability: The ability of a PRO instrument to yield consis-
tent reproducible estimates of true treatment effect.
� Ability to detect change (responsiveness): Evidence that a PRO
instrument can identify differences in scores over time in
individuals or groups who have changed with respect to
the measurement concept.
Conceptual Models Providing Context for PROs
We provide three examples of models for conceptualizing
how patient outcomes and PROs may be framed in relation
to other measures/outcomes.
Figure 1. Modified example of a conceptual framework for head
and neck cancer. Adapted from Wilson and Cleary, Journal of the
American Medical Association (1995).
Academic Radiology, Vol 21, No 9, September 2014 INCLUDING PROs IN STUDIES
1) Wilson and Cleary (1995) reported an oft-cited conceptual
model depicting the association of alternative factors or
concepts in influencing health status. They outlined a rela-
tion among biological and physiological factors, symptoms,
function, general health perceptions, and overall QOL (22).
2) WorldHealthOrganization researchers developed the Inter-
nationalClassification of Functioning,Disability andHealth
(ICF), a classification of health and health-related domains.
The ICF tool allows for the explicit influence of environ-
mental factors on health. The ICF conceptual framework
maps the interaction of a health condition (disease or disor-
der); body functions and structures, activities, and participa-
tion; and environmental and personal factors (23).
3) Calvert et al. (2013) developed a Consolidated Standards of
Reporting Trials PRO extension aimed to improve the
quality of PRO reporting in clinical trials and in informing
clinical practice and health policy. Their model outlines
how PROs and epidemiology data from population studies
and clinical trials can be used to inform guideline develop-
ment. More transparent PRO reporting may influence
clinical trial designs andmay allow stakeholders to assess ev-
idence of intervention effects more consistently (24).
Conceptual frameworks can be helpful in studying the rela-
tion among various outcomes (eg, genetic, biologic, clinical,
or diagnostic) and patient symptoms, patient function, and
HRQOL. Figure 1 presents a conceptual scenario for assessing
PROs and outcomes in head and neck cancer, which represents
a simplified adaptation of the model of Wilson and Cleary. For
example, we can study the impact on daily functioning from
head and neck cancer and treatment interventions in relation
to clinician-reported outcomes, physiologic outcomes, fam-
ily/caregiver outcomes, and the impact on PROs.
Multidisciplinary Study Teams
Designing and conducting clinical and observational studies,
including those using PROs, should incorporate input from
multiple stakeholders. It is recommended that effective study
teams be comprised interdisciplinary researchers, as well as
patients, their advocates, and payers or health technology
assessment experts when appropriate. Study teams, particu-
larly those within medical product companies, often include
statisticians, clinicians, regulatory experts, health services
researchers, clinical research coordinators, international
health systems experts for global trials, data coordinators, pro-
grammers, marketing representatives, and other relevant
personnel. Academic teams would likely include the same sci-
entific members but may not include marketing or regulatory
representatives if not studying a product for review and mar-
keting approval. Public relations and communications experts
are helpful when engaging in promotional activities or media
communication related to health research.
Regulatory and Research Guidance for Including PROsin Trials
For more than 20 years, teams of cognitive psychologists, (bio)
statisticians, psychometricians, health outcome researchers, ep-
idemiologists, health service researchers, clinicians, economists,
regulatory agencies, and other stakeholders have partnered to
recommend the best practices for designing patient-based
instruments and incorporatingPROs in clinical research (eg, in-
strument psychometrics, collection, analysis, and interpreta-
tion) (6–10). In 2009, the US FDA finalized a guidance
document for industry on studying PROs in clinical trials to
provide a framework for developing end point models related
to the treatment of disease and the impact on symptoms, with
recommendations for primary and secondary end points. The
report covers evaluations of PRO instruments (ie, properties),
clinical trial designs and using PROs, and recommended data
analysis approaches. A major investment in PROs by the
US National Institutes of Health led to the innovative
Patient-Reported Outcomes Measurement Information
Systems (PROMIS) tools for assessing health status. Advanced
measurement science approaches andmultidisciplinary collabo-
ration led to extensive research on instruments, measurement,
interpretation, along with tools, recommendations, and
available instruments for assessing physical, mental, and social
well-being in studies (16,17).
Resource-Use questionnaires completed by patients. Patients’resource-use data are a key component of comprehensive
cost-effectiveness assessment (CEA). A 1993 US Panel on
Cost-Effectiveness in Medicine (18) provided recommenda-
tions related to conducting health economic evaluations,
followed by an International Society of Pharmaceutical Out-
comesResearch task force report on ‘‘good research practices’’
for conducting CEA in conjunction with clinical studies (19).
Cost-effectiveness data may or may not use data collected
directly from patients, but specific health conditions may war-
rant taking steps to collect patient-reported resource use.
The prospective use of cost-utility assessment (CUA)
generally requires PRO data collection and/or preference
elicitation. Responses to health outcome measures coordinate
1131
BRESNAHAN AND RUNDELL Academic Radiology, Vol 21, No 9, September 2014
with ‘‘utility weights’’ corresponding to alternative health
states related to a condition or its treatments (eg, improved
function or health detriment). Retrospective or prospective
CEAs/CUAs may use published utility weights that were pre-
viously collected on a specific target group of patients, derived
using patient-reported (or community-reported) approaches.
Generalizing or applying weights to specific settings or pe-
riods may be a challenge. Several textbooks discuss methods
and standards for economic evaluation and CEA to improve
health care and decision making in medicine (20,21).
Use or cost data may be obtained from IT systems (eg, elec-
tronic medical record and/or claims data) of participating
study sites or health systems. Specific types of resource use,
however, may require patient-reported input. Examples
include time spent on care or attending provider visits, per-
sonal expenditures on condition-related products for the
home, filled prescriptions, purchase of over-the-counter
(OTC) medications, or actual prescription and OTCmedica-
tions taken, including dose. Copayment or coinsurance infor-
mation may be substantial and worth collecting for distinct
medical interventions, such as oncology medications/thera-
pies, arthritis medications, magnetic resonance imaging or
molecular imaging procedures, or surgeries.
STRATEGIC DEVELOPMENT OF PATIENT-REPORTED EVIDENCE
Considering PRO Impact Early in Product Life Cyclesfor Medicines and Procedures
Gondek et al. provide examples of how PROs may be consid-
ered for inclusion in oncology trials during a product’s life cy-
cle, focusing on industry-sponsored trials (25). Many
principles of strategic product development have relevance
to medical products in general. For medicines or procedures,
phase 1 studies may assess estimated ranges of ‘‘target product
profiles’’ and potential PRO opportunities. Target product
profiles include expected positive and negative effects and po-
tential ranges of magnitudes of effects for interventions (eg, ef-
ficacy, safety, administration). Target profiles can be used to
estimate risk–benefit balance or other clinical or economic
metrics (26). These profiles can subsequently be refined and
assessed in phase 2 clinical studies, which are increasingly
testing PRO instruments (eg, symptoms, HRQOL, other)
in association with alternative doses, regimens, or procedural
techniques, and in correlation with other study end points.
Larger phase 3 studies should include the final instruments
and cultural/language translations based on the regulatory or
market strategy. Organizations are using portfolio planning ap-
proaches to improve decisionmaking for multiproduct research
and development pipelines. This process includes developing
strong evidence profiles for products with good differentiation
opportunities and terminating development earlier for products
with lower probabilities of technical success or market success.
PRO collection in clinical development programs may inform
1132
the ‘‘evidence upside’’ for products targeting competitive mar-
ketplaces (ie, supporting a differentiation strategy) and inform
the ‘‘evidence downside’’ for products with poor safety profiles,
dose uncertainty, or a lack of effect on PROs that are a compet-
itive standard for specific conditions. Evidence standards for
assessing quantitative benefit–risk balance for medical products
are likely to increasingly become linked to patient-reported
general health status measures, condition-specific PROs, and
patient satisfaction metrics.
Products in competitive markets can use high-quality PRO
evidence to enhance value propositions. PRO strategies may
differentiate devices or medicines or they may be needed to
meet marketplace evidence standards. Needs for testing the
impact of medical devices on patient outcomes will continue
to increase, particularly for devices impacting patients’ symp-
toms and ability to function. Knee replacement and hip
replacement products, for example, are being advertised
directly to consumers. They will need to demonstrate value
to payers and patients through evidence of impact on PROs,
including productivity, time to return towork, ability to func-
tion, and HRQOL.
Timing of PRO Inclusion in Studies
PROs should be assessed in association with other study end
points and demographic characteristics. With appropriate po-
wer, subgroups, health system characteristics, and/or clinical
care metrics may also be evaluated in relation to PROs. Link-
ing the expected timing of clinical effects of interventions and
how they may impact symptoms, function, or HRQOL will
help to determine a PRO inclusion strategy.
Researchers want to select the best available measure and
determine when to administer PROs during a study. Psychol-
ogists, biostatisticians, and health services researchers are
essential participants in making these decisions. The first
step is to determine an appropriate research question, and
how the research objectives align with a study’s general out-
comes assessment plan and the PRO collection strategy. Hol-
lingworth and Jarvik (27) describe and depict how standard
scientific methods (ask question, design study, obtain data,
assess, modify) are associated with evidence-based radiology
and the health technology assessment decision hierarchy,
including levels assessing cost effectiveness and patient
outcomes.
Aligning the timing and responsiveness of a PRO to the
timing of clinical outcomes based on a treatment, procedure,
test, or device is challenging. Preliminary (eg, phase 2) data
are helpful in developing a comprehensive PRO strategy. In-
terventions may have shorter term effects on patients,
requiring short-term PRO collection, while others may
have a delayed impact subsequent to a treatment or procedure,
thus requiring delayed timing for PRO end points. Figure 2
presents a simplified high-level perspective on connecting ex-
pected clinical effects of interventions in trials with PROmea-
surement qualities.
Figure 2. Simplified example of patient-
reported outcomes (PRO) timing in relationto timing of clinical effect from intervention.
Academic Radiology, Vol 21, No 9, September 2014 INCLUDING PROs IN STUDIES
Additional Practical Issues Related to Including PROsin Trials
Researchers should
� Link records with electronic medical records and other IT
system (ie, evolve from paper-based systems) to evaluate
clinical measures, demographics, PROs, resource-use
questionnaires, claims-based resource use, systems data.
� Use well-tested, psychometrically appropriate instruments,
particularly when desiring to publish the evidence and/or
use evidence for marketing purposes.
� Include an instructions page for respondents, outlining ex-
pectations for them in completing the forms.
� Pilot test instruments and batteries of multiple instruments
to determine the expected completion time needed and to
identify challenges patients may have in completing
questionnaires.
� Include PRO and resource-use questionnaire in the overall
clinical trial or observational study schedule of events (eg,
baseline and other time points).
� Include PRO training for investigators, clinical research
coordinators, and data coordinators as part of clinical trial
investigator meetings. PRO data quality standards are no
different than those for other clinical endpoints, including
a goal to minimize missing data.
B Many PROs ask about symptoms or function within
the past week or month, or in relation to the day of
completion or receiving an intervention. There is usu-
ally a finite window after events in which patients are
more likely to have more accurate recall.
B More intensive patient-reported diaries, consisting of
resource-use questions that might not be available
except for direct patient completion, may ask patients
to complete daily information (eg, medication dose)
or several times a week for a specified short period.
� Use prestudies or smaller studies to test PRO and utiliza-
tion questionnaires in the target population (eg, elderly)
and expect an iterative process until the research team is
comfortable with the forms/platform and the strategy to
prioritize, collect, and assess PROs.
EXAMPLES FROM CLINICAL STUDIES
Numerous cases of using PROs and resource-use diaries are
available in published literature. We provide examples of
PROs used in back pain and oncology studies. Out-of-
pocket expenses, time spent on care, and utilization not
captured by electronic health records are important outcomes
that need to be collected directly from the patient. Low back
pain and leg pain in older adults can produce significant
disability and a decreased quality of life, which places a large
burden on patients, providers, payers, and health systems.
Oncology conditions impact physical, mental, and emotional
well-being and can have substantial financial implications on
patients. Patient-reported resource-use data can complement
other health record information to provide a more compre-
hensive perspective for economic analyses.
PROs are the primary method to assess the concept of in-
terest in back pain studies, that is, pain in patients (levels, types,
and impact on function) (28). Two widely used back pain–
specific instruments are the Roland Morris Disability Ques-
tionnaire (RMDQ) (29,30) and the Oswestry Disability
Index (31). Visual analog scales (VAS) and numeric rating
scales (NRS) are also commonly used in pain studies,
including those studying back pain.
Graves et al. (32) used the RMDQ and other PROs to
examine associations in receiving early magnetic resonance
11
33Figure 3. Simplified example of patient-reported medication resource-use questionnaires.
BRESNAHAN AND RUNDELL Academic Radiology, Vol 21, No 9, September 2014
imaging and low back pain outcomes inWashington State La-
bor and Industry (WA L&I) claimants. They used a
population-based prospective cohort design to assess health
outcomes and disability status 1 year after an injury. In the
Lumbar Epidural Steroid Injections for Spinal Stenosis
(LESS) double-blind, randomized, controlled trial, patients
with spinal stenosis were randomized to receive an epidural
steroid injection with a local anesthetic or a local anesthetic
alone. Patients received up to two injections during the first
6 weeks of their randomized intervention and subsequently
had the option to crossover to the other intervention arm.
Outcomes included the RMDQ, back and leg pain NRS
measures, and other PROs, collected at baseline, 3 weeks, 6
weeks, 3 months, 6 months, and 12 months after enrollment
(33). In LESS, the PRO collection strategy aligned with the
expected timing of clinical effects from interventions. More
intensive PRO and resource-use data collection was used in
the first 6 weeks, consistent with the primary end point
timing. In addition to ‘‘in-system’’ claims-based resource use
captured in IT systems of participating sites, the LESS study
captured ‘‘out-of-system’’ use and medication use directly
from patient-completed resource-use questionnaires. Exam-
ples of these resource-use questionnaires are displayed below
for medication use and for provider visits (Figs 3 and 4).
1134
PROs are alsowidely used in oncology studies and extensive
research has produced guidance on collecting, analyzing, and
interpreting PROs. Researchers have evaluated PROs used
in oncology trials, provided recommendations for improve-
ments, and highlighted challenges and issues with integrating
PROs in National Cancer Institute–sponsored oncology trials
conducted in clinical trial networks (34–36). Differences in
general approach at sites or in systems for PRO integration
and variance in standards for PRO data quality assurance
were identified as challenges to overcome. Osoba (37) con-
ducted a review of HRQOL and PROs used in cancer clinical
trials from 2000 to 2010, assessing 118 studies in breast, lung,
prostate, colorectal, and other cancers for the use of PROs,
findings, methodological issues, predictive/prognostic poten-
tial, and integration with biomedical end points. An extensive
1999 report by theUnited Kingdom’sNationalHealth Service
Health Technology Assessment (HTA) Research and Devel-
opment Programme reviewed and evaluated methods for the
assessment of HRQOL and survival data from studies (38).
The HTA report highlighted methods and challenges for
assessing survival time and PROs, given incomplete data or
missing data due to death, protocol violations, or study
dropout due to other reasons is a major issue in oncology
studies. In 2012, the Center for Medical Technology Policy
Figure 4. Simplified example of patient-
reported treatment and service resource-use questionnaires.
Figure 5. Oncology patient-reported out-
comes example: Functional Assessment of
Cancer Therapy–General 7 (version 4) ques-tionnaire (40,41); FACT-G7 (version 4) re-
printed with permission from developers at
FACIT.org.
Academic Radiology, Vol 21, No 9, September 2014 INCLUDING PROs IN STUDIES
released an Effectiveness Guidance Document for incorpo-
rating PROs in CER in adult oncology (39). Figures 5 and 6
present simplified examples of cancer-related PRO scales
used in clinical trials and/or clinical care.
CONCLUSIONS
We are in a new era of health care, with greater focus on pa-
tients, the outcomes they experience, and the resource and
financial burden on patients, families, payers, and health sys-
tems. However, patient-related terminology may not be
used in a consistent manner, and alternative stakeholders
may use similar terms quite differently. As discussed, PROs
used in clinical research often refer to specific, psychometri-
cally appropriate patient-reported measures collected at spe-
cific time points in a clinical trial to assess the impact of
experimental interventions on patients. Alternatively, in clin-
ical care settings, patient-centered concepts deemed impor-
tant to patients as part of their care processes, such as
symptom improvement, communication with providers, or
adequate access to interventions may not always be assessed
systematically, comparatively, or in a manner consistent with
trial-based approaches. In addition, the collection of PROs
in trials or in practice does not guarantee that the outcomes
reported are the most informative or useful to patients or
other stakeholders. Research organizations and/or health
1135
Figure 6. Adapted example of a cancer-
related patient-reported outcomes (PRO)
nausea scale used by the University of Ro-chester Cancer Center Research Base (34)
Printed with permission.
BRESNAHAN AND RUNDELL Academic Radiology, Vol 21, No 9, September 2014
policy setters, including AHRQ, PCORI, payers, manufac-
turers, or health systems, may have distinct needs or mandates
for patient-based information, and their objectives for
wanting patient-reported evidence may not be aligned.
Although not without similar challenges, patient-reported
resource-use questionnaires provide an excellent option to
collect additional patient-based outcomes in clinical research,
which can complement claims-based or systems-based
resource-use evaluation. For studies examining pain, other
debilitating conditions, or interventions impacting how pa-
tients feel and function, PROs may be the best source of
data or the only appropriate method of obtaining relevant
data on symptoms, HRQOL, or other outcomes important
to patients.
From a radiology perspective, as pointed out by Carlos et al.
(42), the imaging community should be actively engaged
stakeholders in designing, conducting, and disseminating
patient-centered research. Multidisciplinary collaborations
can be helpful in investigating and appropriately accentuating
those uses of imaging that add substantial value to patients and
health systems, as well as identifying imaging applications that
may be less appropriate in specific patient populations.
In this manuscript, we have given examples of studies on
patients with pain conditions that have complex interactions
among pain, function, medication use, HRQOL, and eco-
nomic outcomes. A single PRO measure likely will not cap-
ture a comprehensive view of all pain-related outcomes.
General health measures and condition-specific instruments
are needed. Likewise, patients with cancer may experience
adverse effects associated with treatments, and/or improve-
ments in energy or functional ability, along with other health
effects. PRO data should be assessed in conjunction with sur-
vival data, tumor response, or other biomarker information
for patient subgroups and alternative treatment strategies.
One limitation may be additional time and/or expense associ-
ated with collecting PRO data. From a practical perspective,
potentially needing patient consent (with documentation)
1136
for PRO collection adds challenges for lower cost pragmatic
trials. Institutional review boards may require that translations
be made available for non–English-speaking patients.
Patient-reported resource-use questionnaires provide more
comprehensive profiles of consequences associated with alter-
native interventions under study, including health impact and
financial implications. Electronic medical records and claims
data are powerful tools to assess clinical and economic
resource use, but they generally do not include patients’
out-of-pocket expenditures or the amount of time spent
engaging health systems or caring for patients or family mem-
bers. Patients, clinicians, researchers, and other stakeholders
can use outcome information from studies to make more
informed health care decisions, as well as to inform guidelines
and/or health policy.
ACKNOWLEDGMENTS
The authors would like to acknowledge Dr. David Cella for
providing permission to use the FACT-G7 (version 4)as an
oncology example of a patient-reported outcome (PRO),
Dr. Jeffrey Sloan for permission to adapt the nausea scale
developed and used by the University of Rochester Cancer
Center Research Base as an example of a PRO used in cancer
care and trials, Dr. Jarvik and Dr. Friedly of the Back pain
Outcomes using Longitudinal Data (BOLD) Project for
permission to use the back pain PRO examples, and Mr.
Daniel Willems and Mr. Marc Schiermeier for assistance
with the manuscript figures.
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