Patient-reported outcomes (PRO) represent an increasingly important mechanism for incorporating patients' experiences and perspectives into their care to enhance cancer care delivery and outcomes. In this article, we discuss the importance of integrating PROs into both the clinical and research settings in oncology and highlight potential challenges. Clin Cancer Res; 24(8); 1777–9. ©2018 AACR.
See related article by Kim et al., p. 1780
In this issue of Clinical Cancer Research, Kim and colleagues sought to address the use of patient-reported outcome (PRO) measures in cancer clinical trials (1). Notably, there is a growing interest to incorporate PROs into the care of patients with cancer. PROs represent an opportunity to harness patients' voices by allowing them to self-report issues related to their physical and psychologic well-being. Specifically, PROs often include an assessment of patients' symptom burden and enable them to report various treatment side effects. In addition, patient-reported physical functioning can provide valuable information about patients' physical fitness and tolerance to treatment. Furthermore, quality-of-life (QOL) measures often assess patients' general well-being by asking them about various physical, social, emotional, and functional domains. Importantly, clinicians and researchers should strive to utilize rigorously developed and tested PRO measures that reliably assess clinically meaningful outcomes and respond to change as expected.
Numerous PRO measures exist for assessing patients' QOL, physical functioning, and symptom burden. For example, some commonly utilized QOL assessment tools include the Functional Assessment of Cancer Therapy-General (FACT-G), the European Organization for Research and Treatment of Cancer QOL Questionnaire (EORTC QLQ), and the Patient-Reported Outcomes Measurement Information System (PROMIS) measures. To assess physical functioning, clinicians and researchers may use tools such as the Medical Outcomes Study (MOS) 36-Item Short Form Health Survey (SF-36) and the Multidimensional Functional Assessment Questionnaire (MFAQ): Older American Resources and Services (OARS). Some of the more common tools to assess patients' symptom burden include the Edmonton Symptom Assessment System (ESAS), the Memorial Symptom Assessment Scale (MSAS), the Rotterdam Symptom Checklist, the MD Anderson Symptom Inventory (MDASI), and the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE). Thus, a broad range of widely utilized and validated tools exist to help clinicians and researchers incorporate PROs into both the clinical and research settings for patients with cancer.
Importantly, research has demonstrated that integrating PROs into the care of patients with cancer has the potential to improve their care delivery and outcomes. Specifically, evidence suggests that PROs are associated with important clinical outcomes, such as treatment tolerability, hospitalizations, and even survival (2, 3). In addition, PROs represent a potential solution to help clinicians recognize and address their patients’ physical and psychologic symptoms. Despite the high symptom burden of patients with cancer, clinicians often fail to reliably detect their patients’ symptoms and underestimate their severity, resulting in poor symptom management (4). Notably, patients’ symptoms represent a modifiable risk factor that, if properly managed, can prompt earlier initiation of supportive care measures and may result in better symptom management, decreased health care utilization, and potentially improved survival (3, 5). In fact, growing evidence supports the use of PRO-monitoring interventions to allow patients to communicate problematic symptoms and concerns with their clinicians, and thereby enhance patient outcomes.
When attempting to integrate PROs into the care of patients with cancer, both clinicians and researchers may experience several challenges. First, we must identify how we plan to utilize the PRO data. Specifically, we need to determine whether we are simply collecting the data for research purposes, whether we plan to utilize the data to impact patient care, or both (Fig. 1). If we intend to collect the PROs to inform clinical practice, we must outline a plan for reporting the PROs to the clinical team. Moreover, even when collecting the PRO data solely for research purposes, the study team should have a plan in place for monitoring the data and whether to alert patients’ clinical teams for certain survey responses (e.g., risk for self-harm). Furthermore, clinicians and researchers collecting PRO data need to explain clearly to patients how and when they should report any concerning issues to their clinical team, beyond simply reporting the issues in the surveys. In addition, the clinical and/or research team will need to determine who will collect the data, the mechanism for collecting the data (e.g., electronically, paper surveys, phone calls), how often to collect the data, and what method to deliver the data to the clinical team (e.g., medical record documentation, e-mail, phone calls) that works best for both patient care and practice workflow. Importantly, both clinicians and researchers will need to strategize ways to minimize missing data and how to handle the missing data. Thus, when planning to incorporate PROs into the care of patients with cancer, clinicians and researchers must think proactively about the various challenges involved and work to address these issues prior to data collection.
As detailed in this issue of Clinical Cancer Research, Kim and colleagues help to address some of the challenges inherent to the use of PROs in cancer clinical trials (1). Notably, they provide clarity regarding the use of PROs to guide adverse event monitoring. In addition, they highlight that researchers should expect discrepancies to exist between clinician and patient-reported adverse events, and thus, PROs should serve as an adjunct to help with adverse event reporting, not a replacement. Although PRO measures may assist in the overall understanding of a therapy's safety and tolerability, Kim and colleagues clarify that they do not consider PRO data to be safety data. Therefore, clinicians and/or researchers are not required to report PRO data directly to the FDA as safety data in clinical trials. Moreover, Kim and colleagues describe how clinicians and researchers may utilize PRO data to trigger a clinical assessment, which can inform CTCAE grade and attribution for reporting to the FDA. Importantly, they underscore the need for the study team to inform and remind patients about whether their PRO assessments are being shared with their clinical team, and if so, when and how their information is being delivered to their team. Furthermore, they outline that researchers must clearly describe how they plan to assess and report PROs within the study protocol and explain this to patients as part of informed consent. Collectively, the article by Kim and colleagues addresses many important concerns to help guide the appropriate use of PROs within cancer clinical trials.
In summary, as the use of PROs within routine cancer care and oncology research continues to rise, we anticipate that recommendations and guidance for their use will progressively evolve. In this issue of Clinical Cancer Research, Kim and colleagues provide important insights regarding the use of PRO measures in cancer clinical trials (1). Their recommendations highlight that clinicians and researchers must inform their patients and regularly remind them how their PRO data will be used. In addition, clinicians and researchers should urge patients to contact their clinical team for any questions or concerns as they normally would. The authors also underscore the fact that PROs and CTCAE safety data represent different yet complementary pieces of information to help inform the safety and tolerability of cancer treatment. Importantly, PROs provide a feasible and reliable mechanism for integrating the patient voice into cancer care to enhance care delivery and outcomes for patients with cancer, and we look forward to seeing exponential use of PROs in practice and research in the coming years.
Disclosure of Potential Conflicts of Interest
No potential conflicts of interest were disclosed.
Conception and design: R.D. Nipp, J.S. Temel
Development of methodology: R.D. Nipp, J.S. Temel
Writing, review, and/or revision of the manuscript: R.D. Nipp, J.S. Temel
Study supervision: R.D. Nipp, J.S. Temel
This work was supported by NCIK24 CA181253 (to J.S. Temel).