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Introduction Patient‐reported outcomes (PROs) are direct reports from patients about the status of their health condition without amendment or interpretation by others. Patient‐reported outcome measures (PROMs) are the tools used to measure PROs; they are usually validated questionnaires patients complete by self‐assessing their health status. Whilst the benefits of using PROs and PROMs to guide real‐time patient care are well established, they have not been adopted by many oncology institutions worldwide. This literature review aimed to examine the barriers associated with using PROs and PROMs in routine oncology care. Methods A literature search was conducted across EMBASE, Medline and CINAHL databases. Studies detailing barriers to routine PRO use for real‐time patient care were included; those focusing on PRO collection in the research setting were excluded. Results Of 1165 records captured, 14 studies informed this review. At the patient level, patient time, incapacity and difficulty using electronic devices to complete PROMs were prominent barriers. At the health professional level, major barriers included health professionals’ lack of time and knowledge to meaningfully interpret and integrate PRO data into their clinical practice and the inability for PRO data to be acted upon. Prominent barriers at the service level included difficulties integrating PROs and PROMs into clinical workflows and inadequate information technology (IT) infrastructures for easy PRO collection. Conclusion This review has outlined potential barriers to routine PRO use in the oncology setting. Such barriers should be considered when implementing PROs into routine clinical practice. Patient‐reported outcomes (PROs) are direct reports from patients about the status of their health condition without amendment or interpretation by others. This review article outlines potential barriers to routine PRO use in the oncology setting. Such barriers should be considered when implementing PROs into routine clinical practice.

There is a growing interest in the collection and use of patient reported outcomes because they not only provide clinicians with crucial information, but can also be used for economic evaluation and enable public health decisions. During the collection phase of PROMs, there are several factors that can potentially bias the analysis of PROM data. It is crucial that the collected data are reliable and comparable. The aim of this paper was to analyze the type of bias that have already been taken into consideration in the literature. A literature review was conducted by the authors searching on PubMed database, after the selection process, 24 studies were included in this review, mostly regarding orthopedics. Seven types of bias were identified: Non-response bias, collection method related bias, fatigue bias, timing bias, language bias, proxy response bias, and recall bias. Regarding fatigue bias and timing bias, only one study was found; for non-response bias, collection mode related bias, and recall bias, no agreement was found between studies. For these reasons, further research on this subject is needed in order to assess each bias type in relation to each medical specialty, and therefore find correction methods for reliable and comparable data for analysis.

PurposeIn current cancer care, there is a growing debate about the value of using patient-reported outcome measures (PROMs) in daily clinical follow-up. A systematic review of the literature was conducted to assess the evidence of the effectiveness of the routine use of PROMs in daily cancer care in terms of patient outcomes, patient experiences and process indicators and to identify the effect of giving feedback about PROM findings to patients and/or health care professionals (HCPs).MethodsA systematic search was performed. Studies were eligible for inclusion when they (1) used a PROM as an intervention, with or without feedback to patients and/or HCPs, compared with not using a PROM, and (2) used a PROM as an intervention with feedback to patients and/or HCPs, compared with using a PROM without giving feedback to patients and/or HCPs.ResultsAfter screening of 8341 references, 22 original studies met the inclusion criteria. Most studies found a positive effect on survival, symptoms, HRQoL and patient satisfaction. In general, using feedback to patient and/or HCPs about the PROM results led to better symptom control, HRQoL, patient satisfaction and patient-doctor communication. The majority of included studies had insufficient power to detect significant differences in the outcomes assessed.ConclusionThis review shows that predominantly positive findings were found in the use of a PROM in daily cancer care. Additionally, more positive effects were seen when feedback is provided to patient and/or health care professionals, and it is thus highly recommended that this is always done.

With the rise of digital health technologies and telemedicine, the need for evidence-based evaluation is growing. Patient-reported outcome measures (PROMs) and patient-reported experience measures (PREMs) are recommended as an essential part of the evaluation of telemedicine. For the first time, a systematic review has been conducted to investigate the use of PROMs and PREMs in the evaluation studies of telemedicine covering all application types and medical purposes. This study investigates the following research questions: in which scenarios are PROMs and PREMs collected for evaluation purposes, which PROM and PREM outcome domains have been covered and how often, which outcome measurement instruments have been used and how often, does the selection and quantity of PROMs and PREMs differ between study types and application types, and has the use of PROMs and PREMs changed over time. We conducted a systematic literature search of the MEDLINE and Embase databases and included studies published from inception until April 2, 2020. We included studies evaluating telemedicine with patients as the main users; these studies reported PROMs and PREMs within randomized controlled trials, controlled trials, noncontrolled trials, and feasibility trials in English and German. Of the identified 2671 studies, 303 (11.34%) were included; of the 303 studies, 67 (22.1%) were feasibility studies, 70 (23.1%) were noncontrolled trials, 20 (6.6%) were controlled trials, and 146 (48.2%) were randomized controlled trials. Health-related quality of life (n=310; mean 1.02, SD 1.05), emotional function (n=244; mean 0.81, SD 1.18), and adherence (n=103; mean 0.34, SD 0.53) were the most frequently assessed outcome domains. Self-developed PROMs were used in 21.4% (65/303) of the studies, and self-developed PREMs were used in 22.3% (68/303). PROMs (n=884) were assessed more frequently than PREMs (n=234). As the evidence level of the studies increased, the number of PROMs also increased (τ=-0.45), and the number of PREMs decreased (τ=0.35). Since 2000, not only has the number of studies using PROMs and PREMs increased, but the level of evidence and the number of outcome measurement instruments used have also increased, with the number of PREMs permanently remaining at a lower level. There have been increasingly more studies, particularly high-evidence studies, which use PROMs and PREMs to evaluate telemedicine. PROMs have been used more frequently than PREMs. With the increasing maturity stage of telemedicine applications and higher evidence level, the use of PROMs increased in line with the recommendations of evaluation guidelines. Health-related quality of life and emotional function were measured in almost all the studies. Simultaneously, health literacy as a precondition for using the application adequately, alongside proper training and guidance, has rarely been reported. Further efforts should be pursued to standardize PROM and PREM collection in evaluation studies of telemedicine.

Background In the Phase 3 MycarinG study (NCT03971422), rozanolixizumab improved myasthenia gravis (MG)‐specific outcomes versus placebo in patients with generalised MG, including those measured by the five independent MG Symptoms patient‐reported outcome (PRO) scales: Muscle Weakness Fatigability (MWF), Physical Fatigue (PF) and Bulbar Muscle Weakness (BMW) as secondary endpoints and Ocular Muscle Weakness and Respiratory Muscle Weakness (exploratory endpoints). This research aimed to provide further insights into these improvements. Methods Post hoc analyses evaluated correlation (Pearson coefficient) between MG Symptoms PRO and subdomain scores of MG Activities of Daily Living (MG‐ADL) and Quantitative MG (QMG) at baseline. Proportions of responders reaching clinically meaningful thresholds and analyses at the item level (observed mean change and Rasch modelling of predicted change from baseline) are reported for MWF, PF, and BMW with rozanolixizumab versus placebo at Day 43. Results Correlation coefficients between MG Symptoms PRO and MG‐ADL were strong (≥ 0.7) for ocular and bulbar scores and moderate (0.5 to < 0.7) for other scores. Correlations with clinician‐assessed QMG scores were generally weak (< 0.5). For MWF, PF, and BMW, greater proportions of responders were observed with rozanolixizumab 7 mg/kg (46.9%, 31.3% and 26.6%, respectively) or 10 mg/kg (56.5%, 48.4% and 32.3%) versus placebo (28.1%, 26.6% and 10.9%). Item‐level analyses demonstrated rozanolixizumab benefit at a symptom‐specific level. Discussion MG Symptoms PRO scales correlate well with concepts in MG‐ADL while assessing additional concepts, such as PF and MWF. Results from the MG Symptoms PRO in MycarinG reflected improvements from baseline in patient‐relevant symptoms, including fatigue, with rozanolixizumab.

Patient-reported outcomes (PROs) are valuable for shared decision making and research. Patient-reported outcome measures (PROMs) are questionnaires used to measure PROs, such as health-related quality of life (HRQL). Although core outcome sets for trials and clinical practice have been developed separately, they, as well as other initiatives, recommend different PROs and PROMs. In research and clinical practice, different PROMs are used (some generic, some disease-specific), which measure many different things. This is a threat to the validity of research and clinical findings in the field of diabetes. In this narrative review, we aim to provide recommendations for the selection of relevant PROs and psychometrically sound PROMs for people with diabetes for use in clinical practice and research. Based on a general conceptual framework of PROs, we suggest that relevant PROs to measure in people with diabetes are: disease-specific symptoms (e.g. worries about hypoglycaemia and diabetes distress), general symptoms (e.g. fatigue and depression), functional status, general health perceptions and overall quality of life. Generic PROMs such as the 36-Item Short Form Health Survey (SF-36), WHO Disability Assessment Schedule (WHODAS 2.0), or Patient-Reported Outcomes Measurement Information System (PROMIS) measures could be considered to measure commonly relevant PROs, supplemented with disease-specific PROMs where needed. However, none of the existing diabetes-specific PROM scales has been sufficiently validated, although the Diabetes Symptom Self-Care Inventory (DSSCI) for measuring diabetes-specific symptoms and the Diabetes Distress Scale (DDS) and Problem Areas in Diabetes (PAID) for measuring distress showed sufficient content validity. Standardisation and use of relevant PROs and psychometrically sound PROMs can help inform people with diabetes about the expected course of disease and treatment, for shared decision making, to monitor outcomes and to improve healthcare. We recommend further validation studies of diabetes-specific PROMs that have sufficient content validity for measuring disease-specific symptoms and consider generic item banks developed based on item response theory for measuring commonly relevant PROs. Graphical Abstract

PurposePatient-reported outcomes (PROs) are an increasingly popular tool to optimize care and bridge the gap between patient experience and clinician understanding. The aim of this review was to identify mechanisms through which PROs facilitate patient-clinician communication in the adult oncology population.MethodsWe conducted a systematic review of the published literature using the following data sources: MEDLINE, EMBASE, CINAHL, PsycINFO, Cab Direct, and CDSR. Studies included in this review reported on the outcomes of PRO use, used PROs as an intervention and not as a study outcome measurement tool, included cancer patients or survivors as study participants, and analyzed patient-clinician communication.ResultsWe identified 610 unique records, of which 43 publications met the inclusion and exclusion criteria. Synthesis of the reviewed studies provided evidence of the usefulness of PROs in facilitating patient-clinician communication on a variety of topics. We identified mechanisms though which PROs influenced patient-clinician communication to include increasing symptom awareness, prompting discussion, streamlining consultations, and facilitating inter-professional communication. Barriers to PRO use in communication improvement include technical problems impeding its administration and completion, compliance issues due to lack of incentive or forgetfulness, and use of PROs that do not appropriately assess issues relevant to the patient. Facilitators include increased education on PRO use, using PRO tools that patients find more acceptable, and providing patient data summaries in an easily accessible format for clinicians.ConclusionsOur review suggests that PROs facilitate patient-clinician communication through various mechanisms that could perhaps contribute to improvements in symptom management and survival. The impact of PROs on clinical outcomes, however, remains poorly studied.

Changing to burosumab, a monoclonal antibody targeting fibroblast growth factor 23, significantly improved phosphorus homeostasis, rickets, lower-extremity deformities, mobility, and growth versus continuing oral phosphate and active vitamin D (conventional therapy) in a randomized, open-label, phase 3 trial involving children aged 1–12 years with X-linked hypophosphatemia. Patients were randomized (1:1) to subcutaneous burosumab or to continue conventional therapy. We present patient-reported outcomes (PROs) from this trial for children aged ≥ 5 years at screening (n = 35), using a Patient-Reported Outcomes Measurement Information System (PROMIS) questionnaire and SF-10 Health Survey for Children. PROMIS pain interference, physical function mobility, and fatigue scores improved from baseline with burosumab at weeks 40 and 64, but changed little with continued conventional therapy. Pain interference scores differed significantly between groups at week 40 (− 5.02, 95% CI − 9.29 to − 0.75; p = 0.0212) but not at week 64. Between-group differences were not significant at either week for physical function mobility or fatigue. Reductions in PROMIS pain interference and fatigue scores from baseline were clinically meaningful with burosumab at weeks 40 and 64 but not with conventional therapy. SF-10 physical health scores (PHS-10) improved significantly with burosumab at week 40 (least-squares mean [standard error] + 5.98 [1.79]; p = 0.0008) and week 64 (+ 5.93 [1.88]; p = 0.0016) but not with conventional therapy (between-treatment differences were nonsignificant). In conclusion, changing to burosumab improved PRO measures, with statistically significant differences in PROMIS pain interference at week 40 versus continuing with conventional therapy and in PHS-10 at weeks 40 and 64 versus baseline.Trial registration: ClinicalTrials.gov NCT02915705

Background Many cancer patients experience high symptom burden. Healthcare in the USA is reactive, not proactive, and doctor-patient communication is often suboptimal. As a result, symptomatic patients may suffer between clinic visits. In research settings, systematic assessment of electronic patient-reported outcomes (ePROs), coupled with clinical responses to severe symptoms, has eased this symptom burden, improved health-related quality of life, reduced acute care needs, and extended survival. Implementing ePRO-based symptom management programs in routine care is challenging. To study methods to overcome the implementation gap and improve symptom control for cancer patients, the National Cancer Institute created the Cancer-Moonshot funded I mproving the M anagement of sym P toms during A nd following C ancer T reatment (IMPACT) Consortium. Methods S ymptom Management IM plementation of P atient R eported Outcomes in O ncology (SIMPRO) is one of three research centers that make up the IMPACT Consortium. SIMPRO, a multi-disciplinary team of investigators from six US health systems, seeks to develop, test, and integrate an e lectronic sy mptom m anagement program (eSyM) for medical oncology and surgery patients into the Epic electronic health record (EHR) system and associated patient portal. eSyM supports real-time symptom tracking for patients, automated clinician alerts for severe symptoms, and specialized reports to facilitate population management. To rigorously evaluate its impact, eSyM is deployed through a pragmatic stepped wedge cluster-randomized trial. The primary study outcome is the occurrence of an emergency department treat-and-release event within 30 days of starting chemotherapy or being discharged following surgery. Secondary outcomes include hospitalization rates, chemotherapy use (time to initiation and duration of therapy), and patient quality of life and satisfaction. As a type II hybrid effectiveness-implementation study, facilitators and barriers to implementation are assessed throughout the project. Discussion Creating and deploying eSyM requires collaboration between dozens of staff across diverse health systems, dedicated engagement of patient advocates, and robust support from Epic. This trial will evaluate eSyM in routine care settings across academic and community-based healthcare systems serving patients in rural and metropolitan locations. This trial’s pragmatic design will promote generalizable results about the uptake, acceptability, and impact of an EHR-integrated, ePRO-based symptom management program. Trial registration ClinicalTrials.gov NCT03850912 . Registered on February 22, 2019. Last updated on November 9, 2021.

The psychometric process used to establish a relationship between the scores of two (or more) instruments is generically referred to as linking . When two instruments with the same content and statistical test specifications are linked, these instruments are said to be equated. Linking and equating procedures have long been used for practical benefit in educational testing. In recent years, health outcome researchers have increasingly applied linking techniques to patient-reported outcome (PRO) data. However, these applications have some noteworthy purposes and associated methodological questions. Purposes for linking health outcomes include the harmonization of data across studies or settings (enabling increased power in hypothesis testing), the aggregation of summed score data by means of score crosswalk tables, and score conversion in clinical settings where new instruments are introduced, but an interpretable connection to historical data is needed. When two PRO instruments are linked, assumptions for equating are typically not met and the extent to which those assumptions are violated becomes a decision point around how (and whether) to proceed with linking. We demonstrate multiple linking procedures—equipercentile, unidimensional IRT calibration, and calibrated projection—with the Patient-Reported Outcomes Measurement Information System Depression bank and the Patient Health Questionnaire-9. We validate this link across two samples and simulate different instrument correlation levels to provide guidance around which linking method is preferred. Finally, we discuss some remaining issues and directions for psychometric research in linking PRO instruments.