Development and usability testing of an electronic patient-reported outcome (ePRO) solution for patients with inflammatory diseases in an Advanced Therapy Medicinal Product (ATMP) basket trial.

BACKGROUND: Electronic patient-reported outcome (ePRO) systems are increasingly used in clinical trials to provide evidence of efficacy and tolerability of treatment from the patient perspective. The aim of this study is twofold: (1) to describe how we developed an electronic platform for patients to report their symptoms, and (2) to develop and undertake usability testing of an ePRO solution for use in a study of cell therapy seeking to provide early evidence of efficacy and tolerability of treatment and test the feasibility of the system for use in later phase studies. METHODS: An ePRO system was designed to be used in a single arm, multi-centre, phase II basket trial investigating the safety and activity of the use of ORBCEL-C™ in the treatment of patients with inflammatory conditions. ORBCEL-C™ is an enriched Mesenchymal Stromal Cells product isolated from human umbilical cord tissue using CD362+ cell selection. Usability testing sessions were conducted using cognitive interviews and the 'Think Aloud' method with patient advisory group members and Research Nurses to assess the usability of the system. RESULTS: Nine patient partners and seven research nurses took part in one usability testing session. Measures of fatigue and health-related quality of life, the PRO-CTCAE™ and FACT-GP5 global tolerability question were included in the ePRO system. Alert notifications to the clinical team were triggered by PRO-CTCAE™ and FACT-GP5 scores. Patient participants liked the simplicity and responsiveness of the patient-facing app. Two patients were unable to complete the testing session, due to technical issues. Research Nurses suggested minor modifications to improve functionality and the layout of the clinician dashboard and the training materials. CONCLUSION: By testing the effectiveness, efficiency, and satisfaction of our novel ePRO system (PROmicsR), we learnt that most people with an inflammatory condition found it easy to report their symptoms using an app on their own device. Their experiences using the PROmicsR ePRO system within a trial environment will be further explored in our upcoming feasibility testing. Research nurses were also positive and found the clinical dashboard easy-to-use. Using ePROs in early phase trials is important in order to provide evidence of therapeutic responses and tolerability, increase the evidence based, and inform methodology development. TRIAL REGISTRATION: ISRCTN, ISRCTN80103507. Registered 01 April 2022, https://www.isrctn.com/ISRCTN80103507.

More and more patients tell clinicians how they feel by completing questionnaires electronically. Therefore, it is important to assess how easy it is for patients to do this. In this study, we describe how we developed an electronic platform for patients to report their symptoms and how we tested the usability of this platform with patient partners and research nurses. Once the electronic platform was developed, quality of life and symptoms questionnaires were programmed onto it. Alerts were sent to the clinical team if specific scores were obtained on the symptoms questionnaires. Although two patient partners were not able to finish the testing session because of technical issues, the ones who completed the session liked its simplicity and responsiveness. The research nurses also liked the system and only suggested minor modifications. Following this testing, we refined the electronic platform to test it further in a larger study which investigates the safety and use of a drug. We hope that thanks to this electronic platform, we will obtain useful information on the safety and efficacy of treatment.

Global use of electronic patient-reported outcome systems in nephrology: a mixed methods study.

OBJECTIVES: The use of electronic patient-reported outcome (ePRO) systems to support the management of patients with chronic kidney disease is increasing. This mixed-methods study aimed to comprehensively identify existing and developing ePRO systems, used in nephrology settings globally, ascertaining key characteristics and factors for successful implementation. STUDY DESIGN: ePRO systems and developers were identified through a scoping review of the literature and contact with field experts. Developers were invited to participate in a structured survey, to summarise key system characteristics including: (1) system objectives, (2) population, (3) PRO measures used, (4) level of automation, (5) reporting, (6) integration into workflow and (7) links to electronic health records/national registries. Subsequent semistructured interviews were conducted to explore responses. SETTING AND PARTICIPANTS: Eligible systems included those being developed or used in nephrology settings to assess ePROs and summarise results to care providers. System developers included those with a key responsibility for aspects of the design, development or implementation of an eligible system. ANALYTICAL APPROACH: Structured survey data were summarised using descriptive statistics. Interview transcripts were analysed using Codebook Thematic Analysis using domains from the Consolidated Framework for Implementation Research. RESULTS: Fifteen unique ePRO systems were identified across seven countries; 10 system developers completed the structured survey and 7 participated in semistructured interviews. Despite system heterogeneity, reported features required for effective implementation included early and sustained patient involvement, clinician champions and expanding existing electronic platforms to integrate ePROs. Systems demonstrated several common features, with the majority being implemented within research settings, thereby affecting system implementation readiness for real-world application. CONCLUSIONS: There has been considerable research investment in ePRO systems. The findings of this study outline key system features and factors to support the successful implementation of ePROs in routine kidney care.Cite Now.

Listening to the Patient Voice Adds Value to Cancer Clinical Trials.

Randomized clinical trials are critical for evaluating the safety and efficacy of interventions in oncology and informing regulatory decisions, practice guidelines, and health policy. Patient-reported outcomes (PROs) are increasingly used in randomized trials to reflect the impact of receiving cancer therapies from the patient perspective and can inform evaluations of interventions by providing evidence that cannot be obtained or deduced from clinicians' reports or from other biomedical measures. This commentary focuses on how PROs add value to clinical trials by representing the patient voice. We employed 2 previously published descriptive frameworks (addressing how PROs are used in clinical trials and how PROs have an impact, respectively) and selected 9 clinical trial publications that illustrate the value of PROs according to the framework categories. These include 3 trials where PROs were a primary trial endpoint, 3 trials where PROs as secondary endpoints supported the primary endpoint, and 3 trials where PROs as secondary endpoints contrast the primary endpoint findings in clinically important ways. The 9 examples illustrate that PROs add valuable data to the care and treatment context by informing future patients about how they may feel and function on different treatments and by providing clinicians with evidence to support changes to clinical practice and shared decision making. Beyond the patient and clinician, PROs can enable administrators to consider the cost-effectiveness of implementing new interventions and contribute vital information to policy makers, health technology assessors, and regulators. These examples provide a strong case for the wider implementation of PROs in cancer trials.

Symptom burden and health-related quality of life in chronic kidney disease: A global systematic review and meta-analysis.

BACKGROUND: The importance of patient-reported outcome measurement in chronic kidney disease (CKD) populations has been established. However, there remains a lack of research that has synthesised data around CKD-specific symptom and health-related quality of life (HRQOL) burden globally, to inform focused measurement of the most relevant patient-important information in a way that minimises patient burden. The aim of this review was to synthesise symptom prevalence/severity and HRQOL data across the following CKD clinical groups globally: (1) stage 1-5 and not on renal replacement therapy (RRT), (2) receiving dialysis, or (3) in receipt of a kidney transplant. METHODS AND FINDINGS: MEDLINE, PsycINFO, and CINAHL were searched for English-language cross-sectional/longitudinal studies reporting prevalence and/or severity of symptoms and/or HRQOL in CKD, published between January 2000 and September 2021, including adult patients with CKD, and measuring symptom prevalence/severity and/or HRQOL using a patient-reported outcome measure (PROM). Random effects meta-analyses were used to pool data, stratified by CKD group: not on RRT, receiving dialysis, or in receipt of a kidney transplant. Methodological quality of included studies was assessed using the Joanna Briggs Institute Critical Appraisal Checklist for Studies Reporting Prevalence Data, and an exploration of publication bias performed. The search identified 1,529 studies, of which 449, with 199,147 participants from 62 countries, were included in the analysis. Studies used 67 different symptom and HRQOL outcome measures, which provided data on 68 reported symptoms. Random effects meta-analyses highlighted the considerable symptom and HRQOL burden associated with CKD, with fatigue particularly prevalent, both in patients not on RRT (14 studies, 4,139 participants: 70%, 95% CI 60%-79%) and those receiving dialysis (21 studies, 2,943 participants: 70%, 95% CI 64%-76%). A number of symptoms were significantly (p < 0.05 after adjustment for multiple testing) less prevalent and/or less severe within the post-transplantation population, which may suggest attribution to CKD (fatigue, depression, itching, poor mobility, poor sleep, and dry mouth). Quality of life was commonly lower in patients on dialysis (36-Item Short Form Health Survey [SF-36] Mental Component Summary [MCS] 45.7 [95% CI 45.5-45.8]; SF-36 Physical Component Summary [PCS] 35.5 [95% CI 35.3-35.6]; 91 studies, 32,105 participants for MCS and PCS) than in other CKD populations (patients not on RRT: SF-36 MCS 66.6 [95% CI 66.5-66.6], p = 0.002; PCS 66.3 [95% CI 66.2-66.4], p = 0.002; 39 studies, 24,600 participants; transplant: MCS 50.0 [95% CI 49.9-50.1], p = 0.002; PCS 48.0 [95% CI 47.9-48.1], p = 0.002; 39 studies, 9,664 participants). Limitations of the analysis are the relatively few studies contributing to symptom severity estimates and inconsistent use of PROMs (different measures and time points) across the included literature, which hindered interpretation. CONCLUSIONS: The main findings highlight the considerable symptom and HRQOL burden associated with CKD. The synthesis provides a detailed overview of the symptom/HRQOL profile across clinical groups, which may support healthcare professionals when discussing, measuring, and managing the potential treatment burden associated with CKD. PROTOCOL REGISTRATION: PROSPERO CRD42020164737.

Results of a pilot feasibility randomised controlled trial exploring the use of an electronic patient-reported outcome measure in the management of UK patients with advanced chronic kidney disease.

OBJECTIVES: The use of routine remote follow-up of patients with chronic kidney disease (CKD) is increasing exponentially. It has been suggested that online electronic patient-reported outcome measures (ePROMs) could be used in parallel, to facilitate real-time symptom monitoring aimed at improving outcomes. We tested the feasibility of this approach in a pilot trial of ePROM symptom monitoring versus usual care in patients with advanced CKD not on dialysis. DESIGN: A 12-month, parallel, pilot randomised controlled trial (RCT) and qualitative substudy. SETTING AND PARTICIPANTS: Queen Elizabeth Hospital Birmingham, UK. Adult patients with advanced CKD (estimated glomerular filtration rate ≥6 and ≤15 mL/min/1.73 m2, or a projected risk of progression to kidney failure within 2 years ≥20%). INTERVENTION: Monthly online ePROM symptom reporting, including automated feedback of tailored self-management advice and triggered clinical notifications in the advent of severe symptoms. Real-time ePROM data were made available to the clinical team via the electronic medical record. OUTCOMES: Feasibility (recruitment and retention rates, and acceptability/adherence to the ePROM intervention). Health-related quality of life, clinical data (eg, measures of kidney function, kidney failure, hospitalisation, death) and healthcare utilisation. RESULTS: 52 patients were randomised (31% of approached). Case report form returns were high (99.5%), as was retention (96%). Overall, 73% of expected ePROM questionnaires were received. Intervention adherence was high beyond 90 days (74%) and 180 days (65%); but dropped beyond 270 days (46%). Qualitative interviews supported proof of concept and intervention acceptability, but highlighted necessary changes aimed at enhancing overall functionality/scalability of the ePROM system. LIMITATIONS: Small sample size. CONCLUSIONS: This pilot trial demonstrates that patients are willing to be randomised to a trial assessing ePROM symptom monitoring. The intervention was considered acceptable; though measures to improve longer-term engagement are needed. A full-scale RCT is considered feasible. TRIAL REGISTRATION NUMBER: ISRCTN12669006 and the UK NIHR Portfolio (CPMS ID: 36497).

Use of the kidney failure risk equation to inform clinical care of patients with chronic kidney disease: a mixed-methods systematic review.

RATIONALE AND OBJECTIVE: The Kidney Failure Risk Equation (KFRE) predicts the risk of end-stage kidney disease in patients with chronic kidney disease (CKD). This study aimed to evaluate the impact of the utility of KFRE in clinical practice. STUDY DESIGN: Systematic review. SETTING AND STUDY POPULATIONS: Adult patients with CKD but not receiving renal replacement therapy enrolled in studies where KFRE was used in clinical care pathways. SELECTION CRITERIA FOR STUDIES: All studies published from April 2011 to October 2021 identified from Medline, Cumulative Index to Nursing and Allied Health Literature, Embase and reference and citation searches of included studies. DATA EXTRACTION: Relevant data were extracted, and two reviewers independently assessed study quality using appropriate appraisal tools. ANALYTICAL APPROACH: Findings reported as a narrative synthesis due to heterogeneity of the included studies. RESULTS: Of 1635 studies identified, 440 duplicates were removed. The remaining 1195 titles and abstracts were screened. All five studies for full-text review were included in the analysis. Three uses of KFRE were assessed: (1) primary to specialty care interface; (2) general nephrology to multidisciplinary care transition; and (3) treatment planning. Evidence of impact on number of patient referrals into nephrology care was conflicting. However, wait times improved in one study. Although KFRE identified high-risk patients for increased multidisciplinary support, there was concern patients stepped down, no longer meeting eligibility criteria, may lack access to services. CONCLUSIONS: This is the first systematic review of studies that have assessed the actual impact of KFRE in clinical practice with five studies of varying quality reported to date. Trials are in progress assessing the impact on clinical outcomes of using KFRE in clinical practice, and KFRE is being incorporated into guidelines for CKD management. Further studies are needed to assess the impact of KFRE on clinical care. TRIAL REGISTRATION NUMBER: Protocol registered on PROSPERO before initiation of the study (Ref: CRD42020219926).

Patient-reported outcome measures for clinical decision-making in outpatient follow-up: validity and reliability of a renal disease questionnaire.

BACKGROUND: Patient-reported outcome measures are increasingly used by clinicians to support communication in telephone- or face-to-face consultations with patients. A renal disease questionnaire has been developed, but not sufficiently evaluated through clinimetrics in clinical setting. Hence, we aimed to evaluate the content validity, construct validity and the test-retest reliability of a renal disease questionnaire to be used for clinical decision-making. METHODS: A content, construct validity and test-retest reliability study was conducted in 3 nephrology outpatient clinics in Central Denmark Region, Denmark. Content validity (face validity, comprehensibility and relevance) was assessed among 8 patients and 6 clinicians. Reliability was assessed by asking outpatients with chronic kidney disease to complete the questionnaire twice. Reliability was assessed by kappa statistics and agreement by percentage. Construct validity was determined using 4 a priori defined hypotheses and comparing 2 known groups. RESULTS: Five new domains emerged, 6 items were rephrased and 3 items were removed following the content validity test. A total of 160 patients completed the questionnaire with median 8 days (IQR 2 days) between assessments. The test-retest reliability parameters of the single items in the questionnaire were substantial to almost perfect as all the observed weighted kappa values ranged from 0.61 to 0.91, 95% CI (0.34 to 0.95). In total, 61% of the single items showed almost perfect agreement. In total, 3 of the 4 hypotheses were accepted and 44% of the items showed satisfying known-group discriminative validity. CONCLUSION: A renal disease questionnaire used for clinical decision-making in outpatient follow-up showed acceptable content validity and substantial to almost perfect reliability. Sufficient construct validity was not established. Incorporating the questionnaire into routine clinical practice may improve the evaluation of disease burden in patients with chronic kidney disease. We ask patients with chronic kidney disease (CKD) in Central Region Denmark to complete a questionnaire before each outpatient visit. The answers they provide are used to support communication with their health care provider. A questionnaire requires testing to ensure it can accurately capture important information about patient's symptoms and quality of life. When questionnaires are used to support communication between patients and health care professionals, they need to have good measurement properties. This means they need to be: (1) trustworthy, (2) relevant to a patient's health condition, (3) consistent and produce stable results every time. We explored the measurement properties of a questionnaire designed to be used in the face-to face outpatient visits for patients with CKD. We found that the questionnaire captured consistent and stable results. Using this questionnaire may help health care professionals to assess the patients´ burden of symptoms with a more patient-centered approach. Potentially, the use of the questionnaire will increase the patients´ ability to cope with their symptoms and strengthen patients´ involvement in the clinical decisions concerning their treatment.

Using patient-reported outcome measures during the management of patients with end-stage kidney disease requiring treatment with haemodialysis (PROM-HD): a qualitative study.

OBJECTIVES: Patients undergoing haemodialysis report elevated symptoms and reduced health-related quality of life, and often prioritise improvements in psychosocial well-being over long-term survival. Systematic collection and use of patient-reported outcomes (PROs) may help support tailored healthcare and improve outcomes. This study investigates the methodological basis for routine PRO assessment, particularly using electronic formats (ePROs), to maximise the potential of PRO use, through exploration of the experiences, views and perceptions of patients and healthcare professionals (HCPs) on implementation and use of PROs in haemodialysis settings. STUDY DESIGN: Qualitative study. SETTING AND PARTICIPANTS: Semistructured interviews with 22 patients undergoing haemodialysis, and 17 HCPs in the UK. ANALYTICAL APPROACH: Transcripts were analysed deductively using the Consolidated Framework for Implementation Research (CFIR) and inductively using thematic analysis. RESULTS: For effective implementation, the potential value of PROs needs to be demonstrated empirically to stakeholders. Any intervention must remain flexible enough for individual and aggregate use, measuring outcomes that matter to patients and clinicians, while maintaining operational simplicity. Any implementation must sit within a wider framework of education and support for both patients and clinicians who demonstrate varying previous experience of using PROs and often confuse related concepts. Implementation plans must recognise the multidimensionality of end-stage kidney disease and treatment by haemodialysis, while acknowledging the associated challenges of delivering care in a highly specialised environment. To support implementation, careful consideration needs to be given to barriers and facilitators including effective leadership, the role of champions, effective launch and ongoing evaluation. CONCLUSIONS: Using the CFIR to explore the experiences, views and perceptions of key stakeholders, this study identified key factors at organisational and individual levels which could assist effective implementation of ePROs in haemodialysis settings. Further research will be required to evaluate subsequent ePRO interventions to demonstrate the impact and benefit to the dialysis community.

Impact of Using Risk-Based Stratification on Referral of Patients With Chronic Kidney Disease From Primary Care to Specialist Care in the United Kingdom.

INTRODUCTION: The externally validated Kidney Failure Risk Equation (KFRE) for predicting risk of end-stage renal disease (ESRD) has been developed, but its potential impact in a population on referrals for patients with chronic kidney disease (CKD) from primary to specialty nephrology care is not known. METHODS: A cross-sectional population-based study of individuals in United Kingdom primary care registered in The Health Improvement Network database was conducted. National Institute of Health and Care Excellence (NICE) 2014 CKD guidelines versus the 4-variable KFRE set at a >3% risk of ESRD at 5 years were applied to patients identified with CKD stage 3-5 between January 1, 2016, and March 31, 2017. RESULTS: In all, 39,476 (36.6%) of 107,962 adults with CKD stage 3-5 had a urine albumin:creatinine ratio (ACR) available and entered into the primary analysis. Of that, 7566 (19.2%) patients fulfilled NICE criteria for referral, 2386 (31.5%) of whom had a ≤3% 5-year risk of ESRD. Also 8663 (21.9%) patients had a >3% 5-year risk of ESRD, 3483 (40.2%) of whom did not fulfill NICE criteria; this represents 8.8% of the primary population. By using the KFRE threshold rather than NICE criteria for referral, 5869 patients (14.9% of the primary analysis population) would have been reallocated between primary and specialist care. Imputational analysis was used for missing ACR measurements and showed similar results. CONCLUSIONS: A risk-based referral approach would lead to a substantial reallocation of patients between primary care and specialist nephrology care with only a small increase in numbers eligible, ensuring those at higher risk of progression are identified.

Developing a core outcome set for traumatic brachial plexus injuries: a systematic review of outcomes.

OBJECTIVE: To identify what outcomes have been assessed in traumatic brachial plexus injury (TBPI) research to inform the development of a core outcome set for TBPI. DESIGN: Systematic review. METHOD: Medline (OVID), EMBASE, CINAHL and AMED were systematically searched for studies evaluating the clinical effectiveness of interventions in adult TBPIs from January 2013 to September 2018 updated in May 2021. Two authors independently screened papers. Outcome reporting bias was assessed. All outcomes were extracted verbatim from studies. Patient-reported outcomes or performance outcome measures were extracted directly from the instrument. Variation in outcome reporting was determined by assessing the number of unique outcomes reported across all included studies. Outcomes were categorised into domains using a prespecified taxonomy. RESULTS: Verbatim outcomes (n=1491) were extracted from 138 studies including 32 questionnaires. Unique outcomes (n=157) were structured into 4 core areas and 11 domains. Outcomes within the musculoskeletal domain were measured in 86% of studies, physical functioning in 25%, emotional functioning in 25% and adverse events in 33%. We identified 63 different methods for measuring muscle strength, 16 studies for range of movement and 63 studies did not define how they measured movement. More than two-thirds of the outcomes were incompletely reported in prospective studies. CONCLUSION: This review of outcome reporting in TBPI research demonstrated an impairment focus and heterogeneity. A core outcome set would ensure standardised and relevant outcomes are reported to facilitate future systematic review and meta-analysis. PROSPERO REGISTRATION NUMBER: CRD42018109843.

SPIRIT-PRO Extension explanation and elaboration: guidelines for inclusion of patient-reported outcomes in protocols of clinical trials.

Patient-reported outcomes (PROs) are used in clinical trials to provide valuable evidence on the impact of disease and treatment on patients' symptoms, function and quality of life. High-quality PRO data from trials can inform shared decision-making, regulatory and economic analyses and health policy. Recent evidence suggests the PRO content of past trial protocols was often incomplete or unclear, leading to research waste. To address this issue, international, consensus-based, PRO-specific guidelines were developed: the Standard Protocol Items: Recommendations for Interventional Trials (SPIRIT)-PRO Extension. The SPIRIT-PRO Extension is a 16-item checklist which aims to improve the content and quality of aspects of clinical trial protocols relating to PRO data collection to minimise research waste, and ultimately better inform patient-centred care. This SPIRIT-PRO explanation and elaboration (E&E) paper provides information to promote understanding and facilitate uptake of the recommended checklist items, including a comprehensive protocol template. For each SPIRIT-PRO item, we provide a detailed description, one or more examples from existing trial protocols and supporting empirical evidence of the item's importance. We recommend this paper and protocol template be used alongside the SPIRIT 2013 and SPIRIT-PRO Extension paper to optimise the transparent development and review of trial protocols with PROs.

International perspectives on suboptimal patient-reported outcome trial design and reporting in cancer clinical trials: A qualitative study.

PURPOSE: Evidence suggests that the patient-reported outcome (PRO) content of cancer trial protocols is frequently inadequate and non-reporting of PRO findings is widespread. This qualitative study examined the factors influencing suboptimal PRO protocol content, implementation, and reporting, and use of PRO data during clinical interactions. METHODS: Semi-structured interviews were conducted with four stakeholder groups: (1) trialists and chief investigators; (2) people with lived experience of cancer; (3) international experts in PRO cancer trial design; (4) journal editors, funding panelists, and regulatory agencies. Data were analyzed using directed thematic analysis with an iterative coding frame. RESULTS: Forty-four interviews were undertaken. Several factors were identified that could influenced effective integration of PROs into trials and subsequent findings. Participants described (1) late inclusion of PROs in trial design; (2) PROs being considered a lower priority outcome compared to survival; (3) trialists' reluctance to collect or report PROs due to participant burden, missing data, and perceived reticence of journals to publish; (4) lack of staff training. Strategies to address these included training research personnel and improved communication with site staff and patients regarding the value of PROs. Examples of good practice were identified. CONCLUSION: Misconceptions relating to PRO methodology and its use may undermine their planning, collection, and reporting. There is a role for funding, regulatory, methodological, and journalistic institutions to address perceptions around the value of PROs, their position within the trial outcomes hierarchy, that PRO training and guidance is available, signposted, and readily accessible, with accompanying measures to ensure compliance with international best practice guidelines.

Past and Current Practice of Patient-Reported Outcome Measurement in Randomized Cancer Clinical Trials: A Systematic Review.

OBJECTIVES: In our systematic review, we assessed past and current practice of patient-reported outcome (PRO) measurement in cancer randomized, controlled trials (RCTs). METHODS: We included RCTs with PRO endpoints evaluating conventional medical treatments, conducted in patients with the most prevalent solid tumor types (breast, lung, colorectal, prostate, bladder, and gynecological cancers) and either published in 2004 to 2018 or registered on clinicaltrials.gov and initiated in 2014 to 2019. Frequency of use of individual PRO measures was assessed overall, over time, and by cancer site. RESULTS: Screening of 42 095 database records and 3425 registered trials identified 480 published and 537 registered trials meeting inclusion criteria. Among published trials, the European Organisation for Research and Treatment of Cancer (EORTC) measures were used most often (54.8% of trials), followed by the Functional Assessment of Chronic Illness Therapy (FACIT) measures (35.8%), the EQ-5D (10.2%), the SF-36 (7.3%), and the MD Anderson Symptom Inventory (MDASI; 2.5%). Among registered trials, the EORTC measures were used in 66.1% of the trials, followed by the FACIT measures (25.9%), the EQ-5D (23.1%), the SF-36 (4.8%), the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE; 2.2%), the Patient-Reported Outcomes Measurement Information System (PROMIS) measures (1.7%), and the MDASI measures (1.1%). CONCLUSION: The PRO measures most frequently used in RCTs identified in our review differ substantially in terms of content and domains, reflecting the ongoing debate among the scientific community, healthcare providers, and regulators on the type of PRO to be measured. Current findings may contribute to better informing the development of an internationally agreed core outcome set for future cancer trials.

A qualitative study exploring the parceived impact of race on leprosy-affected persons' experiences of diagnosis and treatment of leprosy in Southeast Brazil

Leprosy is a chronic, yet curable, infectious disease that causes severe morbidity in Brazil with an annual incidence of 33,000 new cases. Recent studies in Brazil showed a correlation between leprosy and the percentage of non-whites affected. Racial discrimination has been documented as a barrier to accessing healthcare. This study seeks to explore whether patients perceive race to have impacted on their leprosy care. A qualitative study was undertaken. 20 semi-structured interviews were conducted with patients' at Instituto Lauro de Souza Lima (ILSL), Brazil. Thematic analysis was used to analyse the data. 4 main themes were generated: racism is part of Brazilian culture, difficulties associated with the diagnosis of Leprosy, barriers to accessing treatment, and a lack of health education. Participants felt that racism infiltrates all aspects of Brazilian society, with isolated incidents in healthcare settings. Racism is known to be associated with socioeconomic inequalities. Leprosy control continues to be hindered by various social determinants of health. This, in turn, affects patients' access to leprosy treatment and care. This study may inform the continuing development of equitable leprosy control strategies in Brazil.

Development of an electronic patient-reported outcome measure (ePROM) system to aid the management of patients with advanced chronic kidney disease.

BACKGROUND: Effective management of patients with chronic kidney disease (CKD) relies on timely detection of clinical deterioration towards end stage kidney failure. We aimed to design an electronic Patient-Reported Outcome Measure (ePROM) system, which would allow patients with advanced CKD (pre-dialysis) to: (i) remotely self-report their symptoms using a simple and secure online platform; (ii) share the data with the clinical team in real-time via the electronic patient record to help optimise care. We adopted a staged development process which included: a systematic review of PROMs used in CKD; formation of a co-design team; prototype system design/development, user acceptance testing and refinement; finalisation of the system for testing in a pilot/feasibility trial. RESULTS: A co-design team was convened, including patients with lived experience of CKD; clinical team members; IT/Informatics experts; academics; and Birmingham Clinical Trials Unit representatives. A prototype system was developed and iterative changes made before finalisation during a series of operational meetings. The system allows patients to remotely self-report their symptoms; provides tailored self-management advice; allows monitoring of real-time patient ePROM data; sends automated notifications to the patient/clinical team in the advent of a severe symptom report; and incorporates longitudinal ePROM symptom data into the electronic patient record. Feasibility of the system will be evaluated as part of the National Institute for Health Research funded RePROM (Renal electronic Patient-Reported Outcome Measure) pilot trial (ISRCTN12669006). CONCLUSIONS: Routine ePROM collection with real-time feedback has the potential to improve outcomes and reduce health service costs. We have successfully developed a trial-ready ePROM system for advanced CKD, the feasibility of which is currently being explored in a pilot trial. Assuming feasibility is demonstrated, formal evaluation of efficacy will take place in a future multi-centre randomised controlled trial.

The impact of patient-reported outcome data from clinical trials: perspectives from international stakeholders.

BACKGROUND: Patient-reported outcomes (PROs) are increasingly collected in clinical trials as they provide unique information on the physical, functional and psychological impact of a treatment from the patient's perspective. Recent research suggests that PRO trial data have the potential to inform shared decision-making, support pharmaceutical labelling claims and influence healthcare policy and practice. However, there remains limited evidence regarding the actual impact associated with PRO trial data and how to maximise PRO impact to benefit patients and society. Thus, our objective was to qualitatively explore international stakeholders' perspectives surrounding: a) the impact of PRO trial data, b) impact measurement metrics, and c) barriers and facilitators to effectively maximise the impact of PRO trial data upon patients and society. METHODS: Semi-structured interviews with 24 international stakeholders were conducted between May and October 2018. Data were coded and analysed using reflexive thematic analysis. RESULTS: International stakeholders emphasised the impact of PRO trial data to benefit patients and society. Influence on policy-impact, including changes to clinical healthcare practice and guidelines, drug approval and promotional labelling claims were common types of PRO impact reported by interviewees. Interviewees suggested impact measurement metrics including: number of pharmaceutical labelling claims and interviews with healthcare practitioners to determine whether PRO data were incorporated in clinical decision-making. Key facilitators to PRO impact highlighted by stakeholders included: standardisation of PRO tools; consideration of health utilities when selecting PRO measures; adequate funding to support PRO research; improved reporting and dissemination of PRO trial data by key opinion leaders and patients; and development of legal enforcement of the collection of PRO data. CONCLUSIONS: Determining the impact of PRO trial data is essential to better allocate funds, minimise research waste and to help maximise the impact of these data for patients and society. However, measuring the impact of PRO trial data through metrics is a challenging task, as current measures do not capture the total impact of PRO research. Broader international multi-stakeholder engagement and collaboration is needed to standardise PRO assessment and maximise the impact of PRO trial data to benefit patients and society.

Patient and clinician opinions of patient reported outcome measures (PROMs) in the management of patients with rare diseases: a qualitative study.

BACKGROUND: Rare diseases may be life-threatening or chronically debilitating conditions. Patient care needs are often complex and challenging to coordinate and deliver effectively. Rare diseases and their clinical management may therefore substantially impact on patients' health-related quality of life (HRQOL). The use of patient-reported outcome measures (PROMs) may complement clinical assessments by elucidating patients' perspectives on their health status and care priorities. This study explored the opinions of patients and clinicians on the use of PROMs in the management of patients with rare diseases in routine clinical practice. METHODS: A total of 15 semi-structured one-to-one interviews were conducted with four patients with primary sclerosing cholangitis (PSC); five renal transplant recipients; and six PSC doctors from University Hospitals Birmingham (UHB) NHS Foundation Trust. A focus group session was also conducted with 10 clinical staff members (doctors, nurses and other allied health professionals from UHB). The suitability and acceptability of the Chronic Liver Disease Questionnaire (CLDQ) and the Short Form 12 (SF12) were assessed by patients with PSC and their doctors while the Paediatric quality of life inventory Transplant Module (PedsQL-TM) and the EuroQoL-5 dimensions (EQ. 5D) were evaluated by the renal transplant recipients and their doctors. The discussions were audio recorded and transcribed verbatim. Coding of the transcripts was done using the Nvivo 11 Plus software. Thematic analysis was conducted to identify the main themes and subthemes. RESULTS: Four themes were identified, namely: (i) potential benefits of PROMs in the management of rare diseases; (ii) views on selected questionnaires; (iii) practical considerations for implementation; and (iv) potential facilitators and barriers of implementation. Patients and clinicians suggested that the use of ePROMs may facilitate patient-centred care by promoting patient-clinician communication, highlighting aspects of HRQOL that are important to patients and encouraging patient involvement in their care. They also felt that the disease-specific CLDQ and PedsQL-TM were more relevant than the generic SF12 and EQ-5D. CONCLUSIONS: Patients with rare diseases often experience impaired HRQOL. The use of an ePROM system may enhance the routine management of patients with rare diseases.

Care providers' and patients' attitudes toward using electronic-patient reported outcomes to support patients with traumatic brain injury: a qualitative study (PRiORiTy).

OBJECTIVES: To (a) identify residual symptoms and deficits resulting from a traumatic brain injury (TBI) and impact on patients' and their families' quality of life; (b) explore views and experience of care providers, researchers, patients, and carers of using PROMs; and (c) explore their attitudes toward reporting symptoms and impacts on an electronic platform. Methods: Qualitative semi-structured interviews with people with TBI and their carers; health-care professionals, researchers, and third sector staff members working with people with TBI. Results: Symptoms and long-term impacts of TBI included cognitive problems, difficulties functioning, anxiety, and depression. PROMs were seen as improving knowledge of residual symptoms and their impact post-TBI but not always accurately reflecting patients' residual problems. Challenges to completing PROMs were cognitive impairment and lack of insight into condition. Perceived advantages of an electronic platform included easy data collection; flexibility; improving workflow; and the ability to send/ receive feedback and reminders easily. Suggested features of an electronic platform included simple layout, lay language, short questions, few items on the screen, and capability to send/receive feedback and additional information. Conclusion: There is a demand for reporting symptoms and their impact electronically, providing the layout is kept simple and feedback from clinicians is provided.