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LAURA E. STRONG
The Past, Present, and Future of Patient-Reported Outcomes
in Oncology
Laura E. Strong, PhD
OVERVIEW
Patient-reported outcomes capture a unique and important perspective of oncology therapy. Surveys to properly capture patientreported
outcome measures have been under development for more than 2 decades. More recent efforts to understand the clinical
significance of patient-reported outcomes, called performance measures, are underway. Patient-reported outcomes can be used in a
variety of ways, including therapy decisions for an individual patient, payment for treatment, research into disease progression, or new
drug development. Technology has already enabled electronic systems to capture and search patient-reported outcomes and in the
future will assist in capturing everyday activities, which, in combination with improved informatics to sort the meaningful and actionable
information, will reduce the time commitment for both patients and providers.
Although the conversation about patient-centered medicine
gained traction in the United States with the formation
of the nonprofıt Patient-Centered Outcomes
Research Institute as part of the Affordable Care Act in 2010,
the discussion was started within several organizations 2 decades
ago. 1-4 These, and other, organizations have facilitated
processes that allow patients to contribute their voice.
A critical step in involving patients in their care is to capture
their experience, but historically this information has
not been captured in a format that can be broadly useful. The
U.S. Food and Drug Administration (FDA) has described
four ways in which a patient’s condition can be assessed:
• Patient-reported outcome (PRO)
• Clinician-reported outcome (ClinRO)
• Observer-reported outcome (ObsRO)
• Performance outcome (PerfO; e.g., timed walk test)
These assessments focus on the physical, mental, and social
health of the patient and can be used to evaluate disease
symptoms or adverse events caused by intervention such as
drugs or surgery. The major distinction of the PRO is the direct
involvement of the patient rather than indirect reporting
by others. Although often thought of as symptoms or functional
status, PROs can also include satisfaction with care and
treatment adherence. The signifıcance of the patient’s voice
in oncology has been demonstrated in studies comparing reporting
of symptoms by patients and clinicians. Patient reporting
of Common Terminology Criteria for Adverse
Events (CTCAE) is more strongly associated with measures
of daily health status than that of clinicians. 5,6 The direct potential
benefıts of PROs for patients include improved management
of symptoms and side effects that would likely result
in a higher quality of life and greater satisfaction with care
providers.
Two common settings for PROs are in general clinical
practice or in the FDA approval of new interventions, typically
drugs. In both cases, the questionnaires used to collect
PROs are generally referred to as PRO measures
(PROMs). A variety of PROMs are available, from the National
Institutes of Health’s PRO Measurement Information
System (PROMIS) to the European Organization for
Research and Treatment of Cancer’s QLQ-C30, which is focused
on patients with cancer and has disease-specifıc
PROMs. 7,8 The National Cancer Institute has pushed for the
development of a PRO version of the CTCAE called PRO-
CTCAE. 9 PROMs need to be reliable (get the same answers
repeatedly), valid (measure what they are designed to measure),
and sensitive/responsive (show differences between
patients or within patients over time). In addition, the PROM
needs to fıt the specifıc purpose, meaning that some tools
may need to be tailored to specifıc disease symptoms and/or
stage of disease. 10 Although the FDA has provided guidance
on the use of PROs to support approval, the agency has
also described the disadvantages of using PROs as an endpoint
in cancer drug development, which include uncertainty
about the clinical signifıcance of small changes in
PROs. 11,12
Recently, a new concept has emerged that could address
questions of clinical signifıcance: the PRO-based performance
measure (PRO-PM). With funding from the Centers
for Medicare & Medicaid Services, the National Quality Fo-
From Quintessence Biosciences, Inc, School of Medicine and Public Health, University of Wisconsin-Madison, Madison, WI.
Disclosures of potential conflicts of interest are found at the end of this article.
Corresponding author: Laura E. Strong, PhD, Quintessence Biosciences Inc., 3300 Commercial Ave., Madison, WI 53714; email: lstrong@wisc.edu.
© 2015 by American Society of Clinical Oncology.
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