BACKGROUND AND RATIONALE
When treating cancer patients, their functioning and well-being is of utmost importance. How cancer patients feel and function are best measured using patient-reported outcomes (PROs), specifically, patients’ direct reports on outcomes such as symptoms, functioning, and health-related quality of life.1,2 Traditionally, PROs have been measured in clinical trials and other research studies to compare the impact of different treatment options from the patient’s perspective.3–7 More recently, there has been interest in using PROs to monitor the progress of individual patients and inform their management.8–13 This approach of using PROs involves having a patient complete a standardized questionnaire, providing that patient’s results to his/her clinical team, and using the PRO data—along with other clinical information (laboratory values, imaging studies)—to manage that patient’s care.
The use of PROs in clinical practice has demonstrated benefits, including promoting patient-clinician communication,14–17 assisting with problem detection,11–13,17 influencing management,16 and improving outcomes, such as symptom control, health-related quality of life, and functioning.14,18–20 Recent studies have shown a survival benefit associated with the intervention.21,22 PRO-cision Medicine23 is the concept of using patients’ own reports of their functioning and well-being to personalize their care.
Although evidence supports the effectiveness of PRO-cision Medicine, there are barriers to broad implementation. To promote the use of PROs, a panel of experts at a recent meeting prioritized: (1) helping patients and clinicians interpret the PRO scores, and (2) helping patients and clinicians act on the PRO results.24 This supplement addresses those key issues.
Issue #1: Interpreting PRO Scores
The issues associated with interpreting PRO scores have been well documented. Problems with interpretation stem from the multitude of PRO instruments and lack of standardization in how these PRO instruments are scored and scaled. On some PRO instruments, higher scores represent better outcomes; on other PRO instruments, higher scores indicate worse outcomes; and on still other PRO instruments, higher scores indicate “more” of the outcome (such that higher scores are better for function domains but worse for symptoms). Beyond the directionality of scoring, PRO instrument scaling also differs, further complicating interpretation. For example, some instruments’ scores are normed to a general population average of 50, whereas others are linearly transformed to a 0–100 scale, and others are simply summed. Both patients and clinicians have reported that this variability is confusing, with quotes such as, “Of course I have no idea if this is a good score or a bad score,” “Until you address the scaling issues it isn’t very useful…,” “A score of say, 50, meant one thing on one graph and something different on another one, which I thought was strange,” and at the most basic level, “I don’t know what the numbers mean.”25
To address the issue of score interpretation, we previously conducted a 3-part research study to identify the formats for displaying PRO data that were most accurately interpreted and rated as clearest.26–28 Following the completion of this research project, our Stakeholder Advisory Board suggested that we had created an evidence base sufficient to inform development of best-practice recommendations for the display of PRO data to promote understanding and use. We thus undertook a modified-Delphi consensus process to develop stakeholder-driven, evidence-based standards for presenting PROs in practice.29
The recommendations that emerged from the Delphi consensus panel include the value of: (1) providing descriptive y-axis labels (eg, none, mild, moderate, severe); (2) indicating scores that are possibly concerning; and (3) providing scores for reference populations. However, the Delphi panel also noted that the information needed to implement these recommendations is not available for many PRO instruments. For example, while the descriptive labels along the y-axis help add meaning to the numeric scores, the score ranges that would be associated with each category are unknown for most PRO instruments. Similarly, many PRO instruments do not have established threshold values to indicate which scores may be concerning. There is little comparison data from reference populations, and questions remain with regard to the appropriate comparators.
Issue #2: Acting on PRO Results
In addition to the challenges associated with understanding what scores mean, there is also the question of how issues identified by PRO measures should be addressed. That is, once a score has been identified as possibly concerning, what should clinicians (and patients) do to respond? As noted above, PRO measures may assess a number of domains ranging from symptoms, functional status, well-being, and health-related quality of life. Although clinicians are trained to manage specific somatic symptoms, based in part on established evidence for effective interventions, issues such as social function (ability to participate in work and hobbies) receive less attention. Clinicians may be reluctant to use PRO assessments if they do not feel comfortable responding to the results. In addition, with the increasing focus on patient empowerment and self-management, patients may also want guidance to self-manage issues identified by PRO measures.
Evidence suggests that providing guidance on how to respond to PRO-identified issues can contribute to the successful use of PROs in clinical practice. One of the earliest randomized controlled trials evaluating a PRO intervention provided problem-specific resource and management suggestions and found significant improvements in identifying problems, better management of them, and improved patient outcomes in the intervention group.30 Later studies suggested that providing such recommendations for managing PRO issues is an important component of the intervention’s success.31 A number of groups have undertaken projects to develop suggestions for responding to PRO issues.32,33 However, each of these projects has used different methods, and guidance is needed for others seeking to implement PROs in routine patient care.
To address the issues with interpreting PRO scores and acting on PRO results, we invited a series of papers from experts with experience developing methods for interpreting and/or acting on PROs in clinical practice. This resulting supplement can serve as a “toolkit” of different methods to which researchers and clinicians can refer when implementing PRO use in routine care.
To develop the supplement, the principal investigators (PIs: C.S., M.B., A.W.) first convened a Steering Group with expertise in methods for interpreting and/or acting on PRO scores (see the Acknowledgments section). During a conference call and follow-up emails, the PIs and Steering Group identified additional experts who could also contribute papers to the supplement. Experts on the final list were invited to participate and contribute a paper to the series. An honorarium was provided to each Steering Group member and to each author team, with the author team determining the appropriate division of the funds.
To ensure coordination across papers in the supplement, the PIs held a kick-off conference call with the paper authors in December 2017. Subsequently, each author team submitted a draft abstract in January 2018, and then circulated its draft paper in May 2018. In June 2018, the PIs, Steering Group, and at least one representative from each author team met in person in Baltimore, MD. During this meeting, each paper was presented and discussed by the group as whole. Revised papers were reviewed by the PIs before being submitted to Medical Care for external peer review.
Notably, this supplement is designed to be a “methods toolkit,” with the individual papers describing different approaches to aid PRO score interpretation and/or develop guidance for acting on PRO results. Because of this focus on methods, many of the papers do not include data or results, although in some cases previous publications are referenced that have applied the methods. Although the supplement focuses on cancer, some papers also describe methods used in other conditions that would be applicable to cancer. Similarly, many of the methods applied in the cancer context could also be applied in other disease areas. Finally, the supplement focuses on PRO data in individual patient care, with the goal to describe methods that can help with interpretation/action at the individual patient level. Below, we provide an overview of the papers in the supplement.
A PRO-cision MEDICINE METHODS TOOLKIT
The supplement includes 14 papers: 6 describe different methods for interpreting PROs, and 8 describe how different PRO systems have addressed issues related to interpreting PRO scores and/or acting on the PRO results.
The 6 papers describing methods to aid interpretation cover a range of approaches. The first paper, by Shi et al,34 describes quantitative methods for identifying cutpoints on questionnaires, while the second paper, by Cook et al,35 describes a qualitative approach, “bookmarking,” for setting cutpoints. Another innovative approach for PRO score interpretation, as described by Browne et al,36 is using modern psychometric methods, in this case the Rasch model, to benchmark the performance associated with different scores on a scale—and how this information could be used both to inform patient’s treatment decision-making, as well as to monitor their progress. As such, these 3 papers explain different methods, or “tools,” that can be used to determine what score ranges represent different levels, such as none, mild, moderate, or severe. The fourth paper, by Jensen et al,37 describes how reference values can aid interpretation of score meaning—and reviews methods for collecting and applying the data for these reference values. Another approach for aiding interpretation is discussion between patients and providers, what the Oliver et al38 paper refers to as “feedforward” of the patient’s perspective to his/her clinical team to inform that particular patient’s management. They also describe “feedback” population analytics based on data aggregated across patients to inform treatment decision-making, predictive modeling, and patient-centered care. Three case studies describe PRO systems at different levels of maturity in implementing feedforward and feedback. Finally, the paper by King et al39 provides a thoughtful review of different metrics commonly used for group-level PRO interpretation [eg, minimal important difference, definitions of responders (ie, improved, stable, worsened)], and the extent to which they can aid in the interpretation of individual patient PRO scores.
Eight papers describe different systems that have been developed to collect PROs for patient monitoring and management and the methods they have used to aid interpretation of PRO scores and/or guide action on the PRO results. Notably, the different systems vary in terms of their characteristics, including their purposes for data collection; the types of patients who are targeted for PRO completion; which PRO questionnaires are used; how, when, and where the PROs are collected; and how, when, and where the PROs are reported. The characteristics of these PRO systems, as well as the 3 case studies described by Oliver et al,38 are shown in Table 1. This diversity across the PRO systems’ designs, as well as the different approaches the systems have used to aid interpretation of the PRO scores and/or act on the PRO results, suggest a range of options for addressing these issues.
First, Blackford et al40 describe how the PatientViewpoint web system determines which scores to highlight as possibly concerning, either in absolute terms or a significant worsening, as well as how guidance was developed for acting on these possibly concerning scores. In addition, the paper describes methodologic research the authors pursued to use needs assessments and patients’ reports of their most bothersome issues to identify possibly concerning scores. Haverman et al41 describe the KLIK system developed for children with chronic disease and their parents in the Netherlands, which is now also being used in adults. They highlight how KLIK scores can be reported in various formats, including literal representation of the individual items, sum scores, and different graphic displays. From the United Kingdom, Absolom et al42 describe the electronic patient self-Reporting of Adverse events: Patient Information and aDvice (eRAPID) system, which focuses specifically on chemotherapy adverse events and provides immediate severity tailored feedback for self-management or advice to contact the provider. The paper describes the methods they used to enhance patients’ and clinicians’ engagement with the symptom reports to promote their use in clinical practice. The “Symptom Care at Home” PRO system in the Mooney et al43 paper is unique in that it uses daily phone symptom collection. The interactive voice response system provides automated self-management coaching for patients and alerts to the oncology team about poorly controlled symptoms. In contrast to the other PRO systems, which primarily collect multi-item PROs, Zahrieh et al44 discuss their Beacon system’s focus on single item data collection to highlight the patient’s single biggest concern. In an example of population-wide PRO data collection, Barbera et al45 describe how Ontario has implemented routine PRO reporting across the province over the past decade. They report the various pragmatic methods and evolving approaches used to make the PRO system more useful in practice. In an example from Australia, Girgis et al46 describe the Patient-Reported Outcomes for Personalised Treatment and Care eHealth system (PROMPT-Care), which is fully integrated in hospital electronic oncology information systems. The PROMPT-Care developers emphasized selection of PRO measures that are brief and clinically actionable, and providing care pathways that fit in the clinical workflow to address identified issues. Finally, Stover et al47 describe different methods for alerting clinicians about concerning symptom questionnaire responses from different research studies evaluating the use of PROs in clinical practice. The percentage of PRO reports that trigger an alert varied widely, depending on the guidelines used to determine which scores would generate an alert, as well as due to the different contexts in which the research studies were conducted.
The use of PROs in clinical practice has the potential to promote patient-centered, personalized care. However, until issues related to interpreting PRO scores and acting on PRO results are addressed, the impact of PROs in practice will be limited. Together, the 14 papers in this supplement provide a range of options or “tools” that clinicians and researchers can apply to the use of PROs in clinical practice. The methodologic papers suggest alternative approaches, which can be used alone or in combination to aid PRO score interpretation. In addition, the descriptions of the different methods used by the various PRO systems illustrate the diversity of approaches that can be used, both for interpreting PRO scores and for acting on the PRO results. As such, this supplement “Methods Toolkit” can inform clinicians and researchers aiming to implement routine PRO reporting by providing methodological fundamentals and real-world examples of how to interpret PRO scores and act on PRO results.
Patients value routine PRO data collection more highly when their clinicians actually use the data to inform their care.25 Implementing systems that arm patients and clinicians with the tools to understand the PRO scores, and then act on them, promotes the effective use of PROs in clinical practice. Seeing their data used in their care will encourage patients to complete the questionnaires. More complete data has the potential to not only improve patients’ own care, but also provides more complete datasets for secondary analyses in patient-centered and comparative effectiveness research.48 In all these ways, we can promote personalized patient care based on patients’ reports of their functioning and well-being—“PRO-cision Medicine.”
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