Development Of Patient-reported Outcome Measures Research Articles

Patient-reported outcome measures (PROMs): A review of generic and condition-specific measures and a discussion of trends and issues.

BackgroundPatient‐reported outcome measures (PROMs) are questionnaires that collect health outcomes directly from the people who experience them. This review critically synthesizes information on generic and selected condition‐specific PROMs to describe trends and contemporary issues regarding their development, validation and application.MethodsWe reviewed academic and grey literature on validated PROMs by searching databases, prominent websites, Google Scholar and Google Search. The identification of condition‐specific PROMs was limited to common conditions and those with a high burden of disease (eg cancers, cardiovascular disorders). Trends and contemporary issues in the development, validation and application of PROMs were critically evaluated.ResultsThe search yielded 315 generic and condition‐specific PROMs. The largest numbers of measures were identified for generic PROMs, musculoskeletal conditions and cancers. The earliest published PROMs were in mental health‐related conditions. The number of PROMs grew substantially between 1980s and 2000s but slowed more recently. The number of publications discussing PROMs continues to increase. Issues identified include the use of computer‐adaptive testing and increasing concerns about the appropriateness of using PROMs developed and validated for specific purposes (eg research) for other reasons (eg clinical decision making).ConclusionsThe term PROM is a relatively new designation for a range of measures that have existed since at least the 1960s. Although literature on PROMs continues to expand, challenges remain in selecting reliable and valid tools that are fit‐for‐purpose from the many existing instruments.Patient or public contributionConsumers were not directly involved in this review; however, its outcome will be used in programmes that engage and partner with consumers.

Patient reported outcome measures in trigeminal neuralgia - A systematic review of psychometric performance.

Trigeminal neuralgia (TN) is a rare condition for which there are multiple treatment options available. To date, there has been difficulty in comparing the outcomes of treatment due to the variety of patient-reported outcome measures (PROMs) and their inadequate psychometric testing. The aim of this review was to assess the psychometric properties of PROMs used to date in TN and make recommendations for their use in future studies. Five electronic databases (MEDLINE, EMBASE, CINAHL Plus, PsycINFO, Health and Psychosocial Instruments) were searched for studies assessing the development of PROMs or their psychometric properties in TN studies. The records obtained were assessed independently by two reviewers for their methodological quality, following guidance from the Consensus-based Standards for the selection of Health Measurement Instruments (COSMIN). Six studies were identified and information on five PROMs (Brief Pain Inventory Facial (BPI-F), Visual Analogue Scale (VAS), Barrow Neurology Institute Pain Scale (BNI-PS), Penn Facial Pain Scale-Revised (Penn-FPS-R) and Trigeminal Neuralgia Quality of Life Score) were retrieved. The Penn-FPS-R demonstrated moderate quality evidence for sufficient content validity. The BPI-F showed moderate evidence for sufficient internal consistency and structural validity but low evidence for inconsistent content validity. The Trigeminal Neuralgia Quality of Life score showed very low-quality evidence for insufficient content validity, structure validity and responsiveness. No evidence was found on the assessment of any psychometric properties of the VAS and BNI-PS in TN. There is limited evidence of the psychometric performance of patient-reported outcomes for TN and recommendations for their inclusion in future studies cannot be made. The validation of PROMs in TN studies should be a priority in this field of research. This review highlights the knowledge gap in the field of psychometrics of patient reported outcomes measures in the field of TN. Given the unavailability of an objective outcome measure for pain or health related quality of life, psychometrically sound PROMs are essential for assessing medical and surgical treatment outcomes in TN.

Development of a New Quality of Life Measure for Duchenne Muscular Dystrophy Using Mixed Methods: The DMD-QoL.

ObjectiveBased on concerns about existing patient-reported outcome measures (PROMs) for assessing quality of life (QoL) in Duchenne muscular dystrophy (DMD), we describe the mixed methods development of a new QoL PROM for use in boys and men with DMD: the DMD-QoL.MethodsThe DMD-QoL was developed in 3 stages. First, draft items were generated from 18 semistructured qualitative interviews with boys and men with DMD, analyzed using framework analysis. Second, cognitive debriefing interviews with patients (n = 10), clinicians (n = 8), and patients' parents (n = 10) were undertaken, and a reduced item set was selected and refined. Third, psychometric data on the draft items from a cross-sectional online survey (n = 102) and stakeholder input from patients and patients' parents were used to produce the final questionnaire. Patient and public involvement and engagement was embedded throughout the process.ResultsFrom an initial draft of 47 items, a revised set of 27 items was produced at stage 2, and this set was further refined at stage 3 to generate the DMD-QoL, a 14-item QoL PROM. The DMD-QoL is designed for use from 7 years of age by proxy report and from 10 years of age by self-report or proxy report. The final measure showed good psychometric properties.ConclusionThe DMD-QoL is a new 14-item QoL PROM for boys and men with DMD, with demonstrable content and face validity.

The role of response domain and scale label in the quantitative interpretation of patient-reported outcome measure response options

PurposeUncertainties exist in how respondents interpret response options in patient-reported outcome measures (PROMs), particularly across different domains and for different scale labels. The current study assessed how respondents quantitatively interpret common response options.MethodsMembers of the general public were recruited to this study via an online panel, stratified by age, gender, and having English as a first language. Participants completed background questions and were randomised to answer questions on one of three domains (i.e. loneliness (negatively phrased), happiness or activities (positively phrased)). Participants were asked to provide quantitative interpretations of response options (e.g. how many times per week is equal to “often”) and to order several common response options (e.g. occasionally, sometimes) on a 0–100 slider scale. Chi-squared tests and regression analyses were used to assess whether response options were interpreted consistently across domains and respondent characteristics.ResultsData from 1377 participants were analysed. There was general consistency in quantifying the number of times over the last 7 days to which each response option referred. Response options were consistently assigned a lower value in the loneliness than happiness and activities domains. Individual differences, such as age and English as a second language, explained some significant variation in responses, but less than domain.ConclusionMembers of the public quantify common response options in a similar way, but their quantification is not equivalent across domains or every type of respondent. Recommendations for the use of certain scale labels over others in PROM development are provided.

Cognitive interviewing in patient-reported outcome measures: A systematic review of methodological processes.

Patient Reported Outcome Measures (PROMs) are standardized surveys assessing functional status, health, and wellbeing. Contemporary movement towards the evaluation of services through consideration of the patient perspective is reflected in the development and use of PROMs. Cognitive Interviewing (CI) is a promising method of developing and refining PROMs, however, there is variability in the CI procedures implemented. The objectives of this study were to assess the methodological-reporting quality of studies using CI in the development of PROMs and examine the methodological processes used in studies using CI in the development of PROMs. A systematic literature search was carried out to identify studies using CI to develop PROMs. Quality assessment was carried out according to an appraisal framework. Data regarding CI processes were extracted and synthesized. A total of 19 studies were reviewed, which were assessed to be of high quality. Participant and study characteristics, CI processes, and method of reporting findings were presented. This systematic review found studies evaluating or developing PROMs using CI varied in terms of the processes used across all aspects of the methodology. CI studies of PROMs were more systematically reported when based upon a theoretical model, whether used to guide data collection, analysis, or both. The Cognitive Interviewing Reporting Framework provides a helpful structure for planning, conducting, and reporting CI studies. It is recommended that future research assesses the relative utility of different approaches to CI for identifying problems with PROMs, so as to enable more informed methodological choices.

Patient reported outcomes measures (PROMs): definitions and perspectives for their use as quality indicators in emergency rooms.

There is a growing emphasis on including patients' perspectives on outcomes as a measure of quality care. This recent interest has spawned the rapid development and testing of patient-reported outcome measures (PROMs), through which patients report on their perception of the outcomes of the care they received. Their use has moved significantly from medical research toward applications in the clinical setting, quality measurement, and system accountability. While the use of as measures of clinical or treatment effectiveness, especially in chronic conditions, is widespread, their use in the emergency department (ED) setting is currently limited. In ED care, PROMs could offer promise since 80% of patients are discharged without any other way to track outcome. We explore the development and use of PROMs in publications on benchmarks and patient-centered care.

The Patient Experience: Symptoms and Impact of Dry Age-Related Macular Degeneration.

IntroductionNo published literature systematically explores the dry age-related macular degeneration (AMD) patient experience. To inform the development of patient-reported outcome measures (PROMs), the important and relevant signs, symptoms, and impacts for patients with dry AMD were identified.MethodsA holistic approach was used to capture, define, and organize the signs, symptoms, and impacts that are important to patients with dry AMD. Qualitative evidence was identified through a targeted literature review and clinician (N = 5) and patient (N = 20) interviews. The targeted review was expanded to include patients with AMD, as few studies specific to dry AMD were identified. The qualitative evidence was incorporated into a conceptual model that included the signs, symptoms, and impacts of dry AMD affecting the patient experience.ResultsTwenty-nine articles (dry AMD, N = 5; general AMD, N = 24) exploring health-related quality-of-life evidence in patients with AMD were identified. Concepts identified and included in the preliminary, literature-based model included signs and symptoms related to general vision loss and general impacts (e.g., dependency on others, poor spatial perception/mobility, difficulty reading, emotional affects). No concepts unique to dry AMD were identified. Interviewed clinicians refined the literature-based model. Across all visual acuity severities, ≥ 80% of patients reported difficulty driving, reading, and completing activities of daily living, along with frustration and dependency on others; all patients reported blurred vision. The final model included 35 signs, symptoms, and impacts, with 19 considered salient.ConclusionsTo better understand the patient experience, we captured, defined, and organized signs, symptoms, and impacts into a dry AMD conceptual model. This model can aid in the development of PROMs reflecting the experience of patients with dry AMD.Supplementary InformationThe online version contains supplementary material available at 10.1007/s40123-020-00325-y.

A narrative review of current evidence supporting the implementation of electronic patient-reported outcome measures in the management of chronic diseases.

An application of telemedicine of growing interest and relevance is the use of personal computers and mobile devices to collect patient-reported outcomes (PROs). PROs are self-reports of patients’ health status without interpretation by anyone else. The tools developed to assess PROs are known as patient-reported outcomes measures (PROMs). The technological innovations that have led to an increased ownership of electronic devices have also facilitated the development of electronic PROMs (ePROMs). ePROMs are a conduit for telemedicine in the care of patients with chronic diseases. Various studies have demonstrated that the use of ePROMs in routine clinical practice is both acceptable and feasible with patients increasingly expressing a preference for an electronic mode of administration. There is increasing evidence that the use of electronic patient-reported outcome (ePROMs) could have significant impacts on outcomes valued by patients, healthcare providers and researchers. Whilst the development and implementation of these systems may be initially costly and resource-intensive, patient preferences and existing evidence to support their implementation suggests the need for continued research prioritisation in this area. This narrative review summarises and discusses evidence of the impact of ePROMs on clinical parameters and outcomes relevant to chronic diseases. We also explore recently published literature regarding issues that may influence the robust implementation of ePROMs for routine clinical practice.

Evaluating quality in acute care using patient reported outcome measures: a scoping review

The aim of this scoping review is to identify patient reported outcome measures (PROMs) in acute care settings, assess their psychometric properties and provide recommendations for their use in daily practice. We performed a search in the PubMed database to identify publications concerning PROMs in an acute care setting. The COSMIN checklist was used to assess the psychometric properties of the reported PROMs. We found 1407 publications and included 14 articles, describing 15 measures. Most publications provided limited information on psychometric properties. Three generic PROMs were deemed of adequate quality for use in acute care. We recommend future development and evaluation of PROMs focussing on acute care to further evaluate and improve the quality of acute care.

Young people’s perspectives on patient-reported outcome measures in inflammatory arthritis: results of a multicentre European qualitative study from a EULAR task force

IntroductionAlthough patient-reported outcome measures (PROMs) are increasingly used in clinical practice and research, it is unclear whether these instruments cover the perspective of young people with inflammatory arthritis (IA). The...

Quality of life following a lower limb reconstructive procedure: a protocol for the development of a conceptual framework

IntroductionLower limb conditions requiring reconstructive surgery can be either congenital or acquired from trauma, infection or other medical conditions. Patient-reported outcome measures (PROMs) are often used by healthcare professionals to...

Patient-Reported Outcome Measures in Lymphedema: A Systematic Review and COSMIN Analysis.

Lymphedema is a chronic and debilitating condition that affects many cancer survivors. Patient-reported outcome measures (PROMs) can give valuable insight into the impact of lymphedema on a patient's quality of life and can play an essential role in treatment decisions. This study aims to (1) identify PROMs used to assess health-related quality of life (HRQoL) in patients with lymphedema; and (2) assess the quality of the lymphedema-specific PROMs. We performed a systematic search to identify articles on lymphedema, quality of life, and PROMs. An overview was created of all PROMs used to assess HRQoL in patients with lymphedema. The methodological quality of the lymphedema-specific PROMs was assessed using the Consensus-based Standards for the Selection of Health Measurement Instruments (COSMIN) criteria. A total of 235 articles met the inclusion criteria, of which 200 described studies using one or more PROMs as an outcome measure in patients with lymphedema. The other 35 studies described the development and/or validation of a lymphedema-specific PROM. The COSMIN assessment demonstrated that none of these PROMs met all quality standards for development. The use of PROMs in lymphedema is increasing; however, based on our findings, we cannot fully support the use of any of the existing instruments. A well-developed lymphedema-specific PROM, based on patient input, is needed to gain better insight into the impact of this condition, and can be used to measure the effect of possible medical and surgical treatments.

Content Validity of Patient-Reported Outcome Measures of Satisfaction With Primary Care for Musculoskeletal Complaints: A Systematic Review.

To evaluate the content validity of patient-reported outcome measures (PROMs) used to assess satisfaction in patients with musculoskeletal complaints who are treated in primary care. Systematic review of clinimetric measurement. A literature search in MEDLINE, Embase, and CINAHL was undertaken (up to January 2020) to identify studies of the development or evaluation of content validity of a PROM that aimed to assess patient satisfaction. A PROM was considered eligible if it aimed to measure satisfaction with care in patients with musculoskeletal complaints. Two independent reviewers performed study selection, quality assessment, and data extraction. Evaluation of content validity of the included PROMs was performed according to the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) guidance, which includes an evaluation of PROM development, content validity studies, PROM content, and quality of evidence using the modified Grading of Recommendations Assessment, Development and Evaluation (GRADE). Seven PROMs were identified. Their quality of development was inadequate. No studies evaluating the content validity of the satisfaction PROMs were retrieved. The content validity of the patient satisfaction PROMs was insufficient and supported by very low- quality evidence. In measuring satisfaction among patients with musculoskeletal complaints treated in primary care, none of the identified PROMs had adequate content validity. Future studies should address the relevance, comprehensiveness, and comprehensibility of PROMs used to measure patient satisfaction and emphasize patient involvement during the development of new instruments. J Orthop Sports Phys Ther 2021;51(3):94-102. Epub 12 Nov 2020. doi:10.2519/jospt.2021.9788.

Development of the Functional Assessment of Cancer Therapy-Carcinoid Syndrome Symptom Index

Objective: To develop a symptom-focused index to evaluate representative symptoms, treatment side effects, and emotional and functional well-being of patients with carcinoid syndrome (CS). Methods: The development of the Functional Assessment of Cancer Therapy-Carcinoid Syndrome Symptom Index (FACT-CSI) followed US Food and Drug Administration guidelines for the development of patient-reported outcome (PRO) measures and involved the following: (a) literature review; (b) interviews with 14 CS patients; (c) interviews with 9 clinicians; and (d) instrument development involving input from a range of PRO measure development and CS experts. The resulting draft instrument underwent cognitive interviews with 7 CS patients. Results: Forty-six CS sources were reviewed. Analysis of patient interviews produced 23 patient-reported symptoms. The most frequently endorsed physical symptoms were flushing, diarrhea, abdominal pain, fatigue, and food sensitivity/triggers. Seven priority CS emotional and functional themes were also identified by patients. Expert interviews revealed 12 unique priority symptoms – the most common being diarrhea, flushing, wheezing, edema, abdominal pain/cramping, fatigue, and 8 emotional and functional concerns. Through an iterative process of team and clinical collaborator meetings, data review, item reduction and measure revision, 24 items were selected for the draft symptom index representing symptoms, emotional concerns, global assessment of treatment side effects, and functional well-being. Cognitive interview results demonstrated strong content validity, including positive endorsement of item clarity (>86% across items), symptom relevance (>70% for most items), and overall measure content (86%). Conclusions: The FACT-CSI is a content-relevant, symptom-focused index reflecting the highest priority and clinically relevant symptoms and concerns of people with CS.

Assessing the impacts of having a child with achondroplasia on parent well-being

PurposeThis study’s purpose was to develop a better understanding of the experiences of parents of children with achondroplasia and to provide qualitative evidence to support the development of a patient-reported outcome (PRO) measure of parent impacts.MethodsConcept elicitation (CE) individual/focus group interviews were conducted with parents of children aged 2 to < 12 years with achondroplasia in the United States and Spain. The qualitative analysis informed the PRO measure development. Cognitive debriefing (CD) interviews were conducted to ensure parent understanding and item relevance.ResultsThirty-six parents participated in individual/focus group CE interviews. The analysis identified parent impacts in four domains, including caretaking responsibilities, emotional well-being, family, and work, and results informed the development of the Achondroplasia Parent Experience Measure (APEM). Caretaking responsibilities included managing child’s medical care (92%), helping child with self-care (67%), advocating for child (64%), assisting child (56%), and observing/monitoring child (e.g., to ensure safety; 47%). Impacts on parents’ emotional well-being included worry about the future (75%), worry about child’s physical health (67%), safety concerns (50%), feeling stressed/overwhelmed (44%), and worry about child’s social relationships (42%). Impacts on family and work included family strain (56%), limiting/adapting family activities (42%), and missed work time (50%). CD interviews with an additional 16 parents of children with achondroplasia confirmed understanding and item relevance.ConclusionThe results improve our understanding of the experiences of parents of children with achondroplasia and provide qualitative evidence to support the content validity of the APEM. A psychometric study is needed to validate the measure.

The development and cognitive testing of the positive outcomes HIV PROM: a brief novel patient-reported outcome measure for adults living with HIV

BackgroundPeople living with HIV experience burdensome multidimensional symptoms and concerns requiring person-centred care. Routine use of patient reported outcome measures can improve outcomes. There is no brief patient reported outcome measure (PROM) that currently reflects the breadth of concerns for people living with HIV. This study aimed to develop and cognitively test a brief novel patient reported outcome measure for use within routine adult HIV care– the “Positive Outcomes” HIV PROM.MethodsDevelopment followed the COSMIN taxonomy and guidance for relevance and comprehensiveness, and Rothrock guidance on development of valid patient reported outcome measures. The Positive Outcomes HIV PROM was developed by a steering group (people living with HIV, HIV professionals and health services researchers) using findings from a previously reported qualitative study of priority outcomes for people living with HIV. The prototype measure was cognitively tested with a purposive sample of people living with HIV.ResultsThe Positive Outcomes HIV PROM consists of 23 questions (22 structured, and one open question) informed by the priorities of key stakeholders (n = 28 people living with HIV, n = 21 HIV professionals and n = 8 HIV commissioners) to ensure face and content validity, and refined through cognitive testing (n = 6 people living with HIV). Cognitive testing demonstrated high levels of acceptability and accessibility.ConclusionsThe Positive Outcomes HIV PROM is the first brief patient reported outcome measure reflecting the diverse needs of people living with HIV designed specifically for use in the clinical setting to support patient assessment and care, and drive service quality improvement. It is derived from primary data on the priority outcomes for people living with HIV and is comprehensive and acceptable. Further psychometric testing is required to ensure reliability and responsiveness.

The International Consultation on Incontinence Questionnaires (ICIQ): An update on status and direction.

In its 22nd year, the International Consultation on Incontinence Questionnaire (ICIQ) project continues to promote its primary objective; the development and dissemination of patient completed questionnaires for the standardized, high-quality assessment of urinary, bowel, and vaginal symptoms. The paper gives an update on the ICIQ in the context of current scientific developments of PROM design and regulatory requirements, and plans are outlined for its future direction. An online library of all questionnaires, including over 300 translations has been launched to facilitate access through a semi-automated registration system. The ICIQ project continues to update its protocol to meet current scientific standards and incorporate methodological advances within PROM development. Nineteen psychometrically validated patient-reported outcome measures (PROMs) are published for use in clinical practice and research, including amongst others, a bladder diary, male and female lower urinary tract symptoms, long term catheter, pad use, and bowel symptom questionnaires. The original ICIQ-UI Short Form for the assessment of urinary incontinence continues to be the most internationally used questionnaire and has been translated into over 60 languages. New questionnaires which are under development include the ICIQ-Underactive Bladder and ICIQ-Satisfaction. We anticipate the new website and online library will further facilitate the ease of dissemination and availability of the questionnaires for clinical practice and research. The ICIQ continues to successfully achieve its primary objective. Going forward, a greater focus on promoting routine clinical use and the potential for electronic integration into databases and medical records is envisaged.

What narrative devices do people with systemic sclerosis use to describe the experience of pain from digital ulcers: a multicentre focus group study at UK scleroderma centres

ObjectivesDigital ulcers (DUs) are a common complication in systemic sclerosis (SSc). No existing studies have specifically reported on the qualitative patient experience of DU pain, and our current patient-reported outcome...

Development of a Patient-Reported Outcome Measure for Mohs Reconstruction.

Objective: There are currently no validated patient-reported outcome measures (PROMs) to specifically evaluate quality-of-life (QOL) outcomes for patients undergoing Mohs micrographic surgery (MMS) reconstruction. Therefore, the objective of this report was to develop a short PROM that can be used to evaluate the outcome after MMS reconstruction from the patient's perspective. Methods: Following established guidelines for PROM development, a comprehensive literature review, concept elicitation interviews (n = 25), and cognitive debriefing interviews (n = 5) were performed. These data were used to develop a preliminary survey to measure patient QOL throughout the MMS reconstruction process. Results: Participants identified many items from existing validated QOL instruments as being irrelevant to their situation, indicating that the sensitivity of existing instruments may be relatively low for patients undergoing MMS. There was significant overlap between what patients frequently identified and what expert physicians believed would be important in terms of factors that affect patient QOL throughout this process. Cognitive debriefing interviews indicated that the survey can be quickly and easily administered. Conclusions: Following accepted standards, we have created a 12-item disease- and treatment-specific QOL PROM for individuals undergoing the MMS reconstruction process. Given the prevalence of MMS, this PROM could be an important tool for clinicians to assess outcomes in future interventional studies aimed at minimizing morbidity and maximizing QOL for these skin cancer patients. Psychometric testing and validation of this newly developed PROM are currently underway.

Hypertension specific patient-reported outcome measure. Part III: validation, responsiveness and reliability assessment

Aim. Health-related quality of life in patients with arterial hypertension (HTN) is still determined by only generic patient-reported outcome measures (PROMs), although disease-specific ones are more reliable and highly valid. Previously, we reported the results of development and item-selection process of the new Russian HTN-specific PROM. The purpose of this last stage was to confirm validity, reliability, responsiveness and sensitivity of the scale and to present its final version.Material and methods. Analysis was done using data from a mass survey of patients with Grades 1-3 HTN (n=359, aged 25 to 91 y. o.) and healthy volunteers (n=48, aged 23 to 65 y.o), 407 returned questionnaires. We conducted two exploratory factor analyses (EFA) with the intermediate version of the PROM (80 questions, 20 of them HTN-specific). The Cattel’s scree test was used to select the optimal number of factors. After removing the items with a low factor loadings, a confirmatory factor analysis (CFA) was performed to assess the model’s fitindexes adequacy. The core indexes to be measured were SRMR, RMSEA, CFI. Finally, the PROM’s reliability (Cronbach’s α and McDonald’s ω) and criterion validity (responsiveness) were evaluated.Results. Both EFA with oblique and varimax rotation showed 35 questions have factor loadings ≥0,5 and assigned to one of 5 factors. The basic structure of the PROM was retained for further CFA. Most of indices of fit measured met the requirements: SRMR was 0,08, RMSEA was 0,07 (90% CI (0,07-0,08)) and CFI was 0,08, which confirms the construct validity. Both Cronbach’s α and MacDonald’s ω of each of the domains were ≥0,80 and the whole scale was 0,89 confirming satisfactory reliability. Scores of the questionnaire were positively correlated with the severity of HTN (p&lt;0,001 for Grades 1-3 HTN) and between HTN and non-HTN patients (p=0,01 and 0,04 in psychologic and social domains respectively).Conclusion. Sequentially addressing of exploratory and confirmatory factor analyses and assessment of reliability and responsiveness allowed to form the final version of the patient-reported outcome measure for arterial hypertension. The new Russian-language questionnaire is a useful and feasible tool for routine practice and clinical trials.