Patient-Patient Centered Outcomes Research最新文献

{"title":"Preferences for Adjuvant Immunotherapy in Adults with Resected Stage III Melanoma-A Discrete Choice Experiment.","authors":"Ann Livingstone, Kirsten Howard, Alexander M Menzies, Georgina V Long, Martin R Stockler, Rachael L Morton","doi":"10.1007/s40271-023-00635-w","DOIUrl":"10.1007/s40271-023-00635-w","url":null,"abstract":"<p><strong>Objectives: </strong>This study aimed to quantify adult preferences for adjuvant immunotherapy for resected melanoma and the influence of varying levels of key attributes and baseline characteristics.</p><p><strong>Methods: </strong>A D-efficient design generated 12 choice tasks for two alternative treatments, adjuvant immunotherapy or no adjuvant immunotherapy. Recruitment to the online discrete choice experiment (DCE) occurred via survey dissemination by eight Australian melanoma consumer and professional groups, targeting adults with resected stage III melanoma, considering or having received adjuvant immunotherapy. The DCE included six attributes with two to three levels each, including 3-year risk of recurrence, mild, permanent and fatal adverse events (AEs), drug regimen and annual out-of-pocket costs. A mixed multinomial logit model was used to estimate preferences and calculate marginal rates of substitution and marginal willingness to pay (mWTP).</p><p><strong>Results: </strong>The DCE was completed by 116 respondents, who chose adjuvant immunotherapy over no adjuvant immunotherapy in 70% of choice tasks. Respondents preferred adjuvant immunotherapy when associated with reduced: probabilities of recurrence, permanent and fatal AEs, and out-of-pocket costs. mWTP for an absolute reduction of 1% in 3-year risk of recurrence was less for respondents with lower rather than higher incomes, AU$794 (US$527) and AU$2190 (US$1454) per year. Respondents accepted an additional 4% chance of a permanent AE to reduce their absolute risk of 3-year recurrence by 1%. Respondents were willing to accept an extra 2% chance of 3-year recurrence to lower their chance of a fatal AE by 1%.</p><p><strong>Conclusions: </strong>Almost three-quarters of respondents chose adjuvant immunotherapy over no adjuvant immunotherapy, preferring treatment that improved efficacy and safety. Findings may inform decisions about access to adjuvant immunotherapy following surgery for melanoma.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":"16 5","pages":"497-513"},"PeriodicalIF":3.6,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/51/ca/40271_2023_Article_635.PMC10409831.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9970931","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Including Young Children in the Development and Testing of Patient Reported Outcome (PRO) Instruments: A Scoping Review of Children's Involvement and Qualitative Methods.","authors":"Victoria Gale, Jill Carlton","doi":"10.1007/s40271-023-00637-8","DOIUrl":"10.1007/s40271-023-00637-8","url":null,"abstract":"<p><strong>Background: </strong>Qualitative research during the development/testing of Patient Reported Outcome Measures (PROMs) is recommended to support content validity. However, it is unclear if and how young children (≤ 7 years) can be involved in this research because of their unique cognitive needs.</p><p><strong>Objectives: </strong>Here we investigate the involvement of children (≤ 7 years) in qualitative research for PROM development/testing. This review aimed to identify (1) which stages of qualitative PROM development children ≤ 7 years had been involved in, (2) which subjective health concepts had been explored within qualitative PROM development with this age group, and (3) which qualitative methods had been reported and how these compared with existing methodological recommendations.</p><p><strong>Methods: </strong>This scoping review systematically searched three electronic databases (searches re-run prior to final analysis on 29 June 2022) with no date restrictions. Included studies had samples of at least 75% aged ≤ 7 years or reported distinct qualitative methods for children ≤ 7 years in primary qualitative research to support concept elicitation or PROM development/testing. Articles not in English and PROMs that did not enable children ≤ 7 years to self-report were excluded. Data on study type, subjective health and qualitative methods were extracted and synthesised descriptively. Methods were compared with recommendations from guidance.</p><p><strong>Results: </strong>Of 19 included studies, 15 reported concept elicitation research and 4 reported cognitive interviewing. Most explored quality of life (QoL)/health-related quality of life (HRQoL). Some concept elicitation studies reported that creative/participatory activities had supported children's engagement, but results and reporting detail varied considerably across studies. Cognitive interviewing studies reported less methodological detail and fewer methods adapted for young children compared with concept elicitation studies. They were limited in scope regarding assessments of content validity, mostly focussing on clarity while relevance and comprehensiveness were explored less.</p><p><strong>Discussion: </strong>Creative/participatory activities may be beneficial in concept elicitation research with children ≤ 7 years, but future research needs to explore what contributes to the success of young children's involvement and how researchers can adopt flexible methods. Cognitive interviews with young children are limited in frequency, scope and reported methodological detail, potentially impacting PROM content validity for this age group. Without detailed reporting, it is not possible to determine the feasibility and usefulness of children's (≤ 7 years) involvement in qualitative research to support PROM development and assessment.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":"16 5","pages":"425-456"},"PeriodicalIF":3.4,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9954735","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A New Framework for Co-Creating Telehealth for Cancer Care with the Patient Community.","authors":"Bonnie Addario,&nbsp;Violeta Astratinei,&nbsp;Louise Binder,&nbsp;Jan Geissler,&nbsp;Marcia K Horn,&nbsp;Linda U Krebs,&nbsp;Bryan Lewis,&nbsp;Kathy Oliver,&nbsp;Andrew Spiegel","doi":"10.1007/s40271-023-00642-x","DOIUrl":"https://doi.org/10.1007/s40271-023-00642-x","url":null,"abstract":"<p><p>The increased use of telehealth in cancer care during the coronavirus disease 2019 pandemic has added to our knowledge and experience of the modality with benefits in terms of efficacy, cost, and patient and healthcare professional experience reported. However, telehealth has also been found not to be universally available to all patients with cancer, nor to be appropriate for every healthcare interaction; additionally, not all patients prefer it. Now that coronavirus disease restrictions have essentially ended and an opportunity to re-assess telehealth provision in cancer care presents, we offer a framework that aims to ensure that the needs and preferences of the patient community are included in the development of telehealth provision. Stakeholders in this process include patients, patient advocates, healthcare providers, healthcare services commissioners, managers, and policy makers. The framework outlines how patient advocates can work with other stakeholders as equal partners at all stages of telehealth service development. The patient advocate community has a unique understanding of the patient perspective as well as expertise in healthcare design and delivery. This enables advocates to contribute to shaping telehealth provision, from policy and guideline formulation to patient navigation. Appropriate resources, education and training may be needed for all stakeholders to support the development of an effective telehealth system. Together with other stakeholders, patient advocates can make an important contribution to optimizing appropriate patient-centred telehealth provision in cancer care.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":"16 5","pages":"415-423"},"PeriodicalIF":3.6,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/da/32/40271_2023_Article_642.PMC10409807.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9971997","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Decision Making About Disease-Modifying Treatments for Relapsing-Remitting Multiple Sclerosis: Stated Preferences and Real-World Choices.","authors":"Edward J D Webb,&nbsp;David Meads,&nbsp;Ieva Eskytė,&nbsp;Helen L Ford,&nbsp;Hilary L Bekker,&nbsp;Jeremy Chataway,&nbsp;George Pepper,&nbsp;Joachim Marti,&nbsp;Yasmina Okan,&nbsp;Sue H Pavitt,&nbsp;Klaus Schmierer,&nbsp;Ana Manzano","doi":"10.1007/s40271-023-00622-1","DOIUrl":"https://doi.org/10.1007/s40271-023-00622-1","url":null,"abstract":"<p><strong>Background: </strong>People with relapsing-remitting multiple sclerosis can benefit from disease-modifying treatments (DMTs). Several DMTs are available that vary in their efficacy, side-effect profile and mode of administration.</p><p><strong>Objective: </strong>We aimed to measure the preferences of people with relapsing-remitting multiple sclerosis for DMTs using a discrete choice experiment and to assess which stated preference attributes correlate with the attributes of the DMTs they take in the real world.</p><p><strong>Methods: </strong>Discrete choice experiment attributes were developed from literature reviews, interviews and focus groups. In a discrete choice experiment, participants were shown two hypothetical DMTs, then chose whether they preferred one of the DMTs or no treatment. A mixed logit model was estimated from responses and individual-level estimates of participants' preferences conditional on their discrete choice experiment choices calculated. Logit models were estimated with stated preferences predicting current real-world on-treatment status, DMT mode of administration and current DMT.</p><p><strong>Results: </strong>A stated intrinsic preference for taking a DMT was correlated with currently taking a DMT, and stated preferences for mode of administration were correlated with the modes of administration of the DMTs participants were currently taking. Stated preferences for treatment effectiveness and adverse effects were not correlated with real-world behaviour.</p><p><strong>Conclusions: </strong>There was variation in which discrete choice experiment attributes correlated with participants' real-world DMT choices. This may indicate patient preferences for treatment efficacy/risk are not adequately taken account of in prescribing. Treatment guidelines must ensure they take into consideration patients' preferences and improve communication around treatment efficacy/risk.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":"16 5","pages":"457-471"},"PeriodicalIF":3.6,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10317912","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Exploring the Preferences of the Australian Public for Antibiotic Treatments: A Discrete Choice Experiment.","authors":"Chris Degeling, Trent Yarwood, Alberto Nettel-Aguirre, Judy Mullan, Nina Reynolds, Gang Chen","doi":"10.1007/s40271-023-00640-z","DOIUrl":"10.1007/s40271-023-00640-z","url":null,"abstract":"<p><strong>Objectives: </strong>Identify Australian public preferences for antibiotic treatments in the context of antibiotic stewardship.</p><p><strong>Methods: </strong>A discrete choice experiment (DCE) was conducted in Australia to investigate the importance of seven attributes associated with antibiotic treatments and related stewardship practices: contribution to antimicrobial resistance (AMR), treatment duration, side effects, days needed to recover, days before taking antibiotics, treatment failure and out-of-pocket costs. The DCE data were analysed using conditional logit, mixed logit and latent class conditional logit models. The relative importance of each attribute was calculated.</p><p><strong>Results: </strong>A total of 1882 respondents completed the survey; the main study sample consist of 1658 respondents (mean age 48 years) who passed quality checks. All seven attributes significantly influenced respondents' preferences for antibiotic treatments. Based on the designed attribute levels in the DCE, on average, out-of-pocket costs (32.8%) and contribution to antibiotic resistance (30.3%) were the most important attributes, followed by side effects (12.9%). Days before starting medication was least important (3.9%). Three latent classes were identified. Class 1 (including respondents who were more likely to be older and more health literate; 24.5%) gave contribution to antibiotic resistance greater importance in treatment preferences. Class 2 (including respondents more likely to report poorer health; 25.2%) gave out-of-pocket costs greater importance. The remaining (50.4%), who were generally healthier, perceived side effects as the most important attribute.</p><p><strong>Conclusions: </strong>Despite concerted public awareness raising campaigns, our results suggest that several factors may influence the preferences of Australians when considering antibiotic use. However, for those more likely to be aware of the need to preserve antibiotics, out-of-pocket costs and limiting the contribution to antibiotic resistance are the dominant influence. Delays in starting treatment were not important for any latent class, suggesting public tolerance for this measure. These results could help inform strategies to promote prudent antibiotic stewardship.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":"16 5","pages":"555-567"},"PeriodicalIF":3.4,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/8f/9c/40271_2023_Article_640.PMC10409829.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10343205","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Measurement Properties of the 15-Item Singapore Caregiver Quality of Life Scale (SCQOLS-15) in Family Caregivers of Patients with Heart Diseases.","authors":"Mihir Gandhi,&nbsp;Ru-San Tan,&nbsp;Shir Lynn Lim,&nbsp;Irene Teo,&nbsp;Grace Yang,&nbsp;Kai Lee Woo,&nbsp;Yin Bun Cheung","doi":"10.1007/s40271-023-00634-x","DOIUrl":"10.1007/s40271-023-00634-x","url":null,"abstract":"<p><strong>Purpose: </strong>To evaluate the measurement properties of the 15-item Singapore Caregiver Quality of Life Scale (SCQOLS-15) in family caregivers of patients with heart diseases.</p><p><strong>Methods: </strong>The SCQOLS-15 survey was self-administered by family caregivers of patients with chronic heart diseases, at baseline and 1 week later. The criterion validity of SCQOLS-15 and its domain scores was assessed by calculating the Spearman correlation coefficient (ρ) with the Brief Assessment Scale for Caregivers (BASC), Caregiver Reaction Assessment (CRA), and their sub-scores. Known-group validity was assessed using the New York Heart Association (NYHA) functional class. Test-retest reliability was evaluated using the intraclass correlation coefficient (ICC).</p><p><strong>Results: </strong>Of the 327 caregivers included, 65% were adult children and 28% were spouses. The distribution of NYHA classes of the patients was I: 27%, II: 40%, III: 24%, and IV: 9%. There was a positive correlation between the SCQOLS-15 and BASC total scores (ρ = 0.7). SCQOLS-15 domain scores were also correlated with BASC and CRA sub-scores as per a priori hypotheses, with absolute values of ρ ranging from 0.4 to 0.6. The mean values of SCQOLS-15 total and all domain scores were lower among caregivers of patients with NYHA class III/IV compared to those of class I/II patients (each P < 0.05). Among 146 caregivers who completed the follow-up and self-rated a stable quality-of-life, ICCs for test-retest reliability of SCQOLS-15 total and all domain scores were ≥ 0.8.</p><p><strong>Conclusion: </strong>The SCQOLS-15 is a valid and reliable instrument for measuring the quality of life in caregivers of heart disease patients.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":"16 5","pages":"485-495"},"PeriodicalIF":3.6,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9960613","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Developing a Conceptual Framework for Socioeconomic Impact Research in European Cancer Patients: A 'Best-Fit' Framework Synthesis.","authors":"Phu Duy Pham,&nbsp;Michael Schlander,&nbsp;Rachel Eckford,&nbsp;Karla Hernandez-Villafuerte,&nbsp;Jasper Ubels","doi":"10.1007/s40271-023-00632-z","DOIUrl":"https://doi.org/10.1007/s40271-023-00632-z","url":null,"abstract":"<p><strong>Background: </strong>Multiple studies have indicated a socioeconomic impact of cancer and cancer care on patients and their families. Existing instruments designed to measure this impact lack consensus in their conceptualization of the issue. Further, various terminologies have been used in the literature (e.g., financial burden, financial hardship, financial stress) without clear definitions and consistent conceptual background. Based on a targeted review of existing models addressing the socioeconomic impact of cancer, our goal was to develop a comprehensive framework from a European perspective.</p><p><strong>Method: </strong>A 'best-fit' framework synthesis was applied. First, we systematically identified existing models to generate a priori concepts. Second, we systematically identified relevant European qualitative studies and coded their results against these a priori concepts. Inclusion and exclusion criteria were predefined and applied thoroughly in these processes. Thematic analysis and team discussions were applied to finalize the (sub)themes in our proposed conceptual framework. Third, we examined model structures and quotes from qualitative studies to explore relationships among (sub)themes. This process was repeated until no further change in (sub)themes and their relationships emerged.</p><p><strong>Result: </strong>Eighteen studies containing conceptual models and seven qualitative studies were identified. Eight concepts and 20 sub-concepts were derived from the included models. After coding the included qualitative studies against the a priori concepts and following discussions among team members, seven themes and 15 sub-themes were included in our proposed conceptual framework. Based on the identified relationships, we categorized themes into four groups: causes, intermediate consequences, outcomes and risk factors.</p><p><strong>Conclusion: </strong>We propose a Socioeconomic Impact Framework based on a targeted review and synthesis of existing models in the field and adapted to the European perspective. Our work contributes as an input to a European consensus project on socioeconomic impact research by an Organization European Cancer Institute (OECI) Task Force.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":"16 5","pages":"515-536"},"PeriodicalIF":3.6,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/e7/b6/40271_2023_Article_632.PMC10409844.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9961502","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Preferences of People Living with HIV for Long-Acting Antiretroviral Treatment in Germany: Evidence from a Discrete Choice Experiment.","authors":"Martin Emmert,&nbsp;Stefan Rohrbacher,&nbsp;Jennifer Jahn,&nbsp;Katharina Fernando,&nbsp;Michael Lauerer","doi":"10.1007/s40271-023-00641-y","DOIUrl":"https://doi.org/10.1007/s40271-023-00641-y","url":null,"abstract":"<p><strong>Objective: </strong>This study aimed to elicit preferences for attributes of current and novel long-acting antiretroviral therapy for human immunodeficiency virus treatment.</p><p><strong>Methods: </strong>Primary survey data were collected (July-October 2022) on a sample of 333 people living with human immunodeficiency virus in Germany from a patient recruitment agency. Respondents were invited by e-mail to respond to a web-based questionnaire. After performing a systematic literature review, we conducted qualitative semi-structured interviews to identify and select the key attributes of drug therapy for patients' preferences for human immunodeficiency virus treatment. Based on this, a discrete choice experiment survey elicited preferences for long-acting antiretroviral therapy characteristics, including the type of medication, frequency of dosing, the location of treatment, the risk of both short-term and long-term side effects, as well as possible interactions with other medications or (party) drugs. A statistical data analysis was performed using multinomial logit models. An additional latent class multinomial logit was performed to evaluate subgroup differences.</p><p><strong>Results: </strong>Overall, 226 respondents (86% male, mean age 46.1 years) were included in the analysis. The frequency of dosing (36.1%) and the risk of long-term side effects (28.2%) had the greatest influence on preferences. The latent class analysis identified two patient groups. While the first class (n = 135; 87% male, mean age 44.4 years) found the frequency of dosing (44.1%) to be most important, the second class (n = 91; 85% male, mean age 48.6 years) focused on the risk of long-term side effects (50.3%). The evaluation of structural variables showed that male respondents, those living in small cities or villages, and those with better health status results were significantly more likely to be assigned to the second class (p < 0.05 each).</p><p><strong>Conclusions: </strong>All attributes included in our survey were important to participants when choosing an antiretroviral therapy. We found evidence that the frequency of dosing as well as the risk of long-term side effects have a particular impact on the acceptance of novel therapy regimens and should be considered in order to optimize adherence and satisfaction.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":"16 5","pages":"537-553"},"PeriodicalIF":3.6,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/14/cb/40271_2023_Article_641.PMC10409836.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9961980","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Design and Administration of Patient-Centred Outcome Measures: The Perspectives of Children and Young People with Life-Limiting or Life-Threatening Conditions and Their Family Members.","authors":"Lucy Coombes,&nbsp;Daney Harðardóttir,&nbsp;Debbie Braybrook,&nbsp;Anna Roach,&nbsp;Hannah Scott,&nbsp;Katherine Bristowe,&nbsp;Clare Ellis-Smith,&nbsp;Julia Downing,&nbsp;Myra Bluebond-Langner,&nbsp;Lorna K Fraser,&nbsp;Fliss E M Murtagh,&nbsp;Richard Harding","doi":"10.1007/s40271-023-00627-w","DOIUrl":"https://doi.org/10.1007/s40271-023-00627-w","url":null,"abstract":"<p><strong>Background: </strong>Self-reported health data from children with life-limiting conditions is rarely collected. To improve acceptability and feasibility of child and family-centred outcome measures for children, they need to be designed in a way that reflects preferences, priorities and abilities.</p><p><strong>Objectives: </strong>The aim was to identify preferences for patient-reported outcome measure design (recall period, response format, length, administration mode) to improve the feasibility, acceptability, comprehensibility and relevance of a child and family-centred outcome measure, among children with life-limiting conditions and their family members.</p><p><strong>Method: </strong>A semi-structured qualitative interview study seeking the perspectives of children with life-limiting conditions, their siblings and parents on measure design was conducted. Participants were purposively sampled and recruited from nine UK sites. Verbatim transcripts were analysed using framework analysis.</p><p><strong>Results: </strong>A total of 79 participants were recruited: 39 children aged 5-17 years (26 living with a life-limiting condition; 13 healthy siblings) and 40 parents (of children aged 0-17 years). Children found a short recall period and a visually appealing measure with ten questions or fewer most acceptable. Children with life-limiting conditions were more familiar with using rating scales such as numeric and Likert than their healthy siblings. Children emphasised the importance of completing the measure alongside interactions with a healthcare professional to enable them to talk about their responses. While parents assumed that electronic completion methods would be most feasible and acceptable, a small number of children preferred paper.</p><p><strong>Conclusions: </strong>This study demonstrates that children with life-limiting conditions can engage in communicating preferences regarding the design of a patient-centred outcome measure. Where possible, children should be given the opportunity to participate in the measure development process to enhance acceptability and uptake in clinical practice. Results of this study should be considered in future research on outcome measure development in children.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":"16 5","pages":"473-483"},"PeriodicalIF":3.6,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/bc/56/40271_2023_Article_627.PMC10205035.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10016852","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Foundation for Patient-Centered Core Impact Sets: Key Learnings from Past and Existing Approaches.","authors":"Eleanor M Perfetto,&nbsp;T Rosie Love,&nbsp;Elisabeth M Oehrlein,&nbsp;Silke C Schoch,&nbsp;Suz Schrandt","doi":"10.1007/s40271-023-00630-1","DOIUrl":"https://doi.org/10.1007/s40271-023-00630-1","url":null,"abstract":"<p><p>Despite growing commitment to patient centricity, challenges persist in consistently identifying the impacts of disease and/or treatment that patients report as most important to them, especially across myriad potential downstream uses. Patient-centered core impact sets (PC-CIS), disease-specific lists of impacts that patients report as most important, are proposed as a solution. But, PC-CIS is a new concept, currently in the pilot stage with patient advocacy groups. We conducted an environmental scan to explore PC-CIS conceptual overlap with past/existing efforts [e.g., core outcome sets (COS)] and to inform general feasibility for further development and operationalization. With guidance and advice from an expert advisory committee, we conducted a search of the literature and relevant websites. Identified resources were reviewed for alignment with the PC-CIS definition, and key insights were gleaned. We identified 51 existing resources and five key insights: (1) no existing efforts identified meet the definition of PC-CIS as we have specified it in terms of patient centricity, (2) existing COS-development efforts are a valuable source of foundational resources for PC-CIS, (3) existing health-outcome taxonomies can be augmented with patient-prioritized impacts to create a comprehensive impact taxonomy, (4) current approaches/methods can inadvertently exclude patient priorities from core lists/sets and will need to be modified to protect the patient voice, and (5) there is need for clarity and transparency on how patients were engaged in individual past/existing efforts. PC-CIS is conceptually unique from past/existing efforts in its explicit emphasis on patient leadership and being patient driven. However, PC-CIS development can leverage many resources from the past/existing related work.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":"16 4","pages":"293-300"},"PeriodicalIF":3.6,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9684003","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}