Patient-Patient Centered Outcomes Research最新文献

{"title":"Routine Quality-of-Life Measurement in Residential Aged Care: Staff, Resident, and Family Perspectives.","authors":"Andrew Simon Gilbert, Frances Batchelor, Nancy Devlin, Briony Dow, Brendan Mulhern, Rosalie Viney, Tessa Peasgood, Lidia Engel","doi":"10.1007/s40271-025-00729-7","DOIUrl":"10.1007/s40271-025-00729-7","url":null,"abstract":"<p><strong>Background and objectives: </strong>There is interest in routinely measuring quality of life (QoL) in aged care homes, evidenced by the Australian Government's implementation of QoL as a mandatory quality indicator. This study explores views of aged care staff, residents, and family members on the benefits, challenges, and feasibility of implementing routine QoL measures.</p><p><strong>Methods: </strong>Qualitative data were gathered to explore staff, resident, and family perspectives on QoL measurement in aged care homes, including purpose, benefits, implementation, and best practice. Two staff workshops were conducted at different aged care homes, and semi-structured interviews were held with 29 proxies (9 family members and 20 staff) and 24 residents. Workshops and interviews were transcribed verbatim, and thematically analysed via a qualitative interpretive approach using NVivo software.</p><p><strong>Results: </strong>Analysis yielded four key themes: (1) benefits of routine QoL measurement; (2) challenges in implementation; (3) best practice for collecting surveys; (4) validity concerns. Identified benefits included potentially improved care, monitoring service performance, and informing family members. Staff participants recommended integrating measures into existing care planning and having oversight from a registered nurse. Participants identified potential implementation challenges, including administrative burden, time and resourcing constraints, conflicts of interest, and resistance from staff, providers, and residents.</p><p><strong>Conclusions: </strong>This study identifies potential benefits to implementing routine QoL measurement in residential aged care homes. To maximise these benefits, it is important to consider how measurement can be integrated in ways that contribute to existing care planning and practices.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"211-223"},"PeriodicalIF":3.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11985636/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143082004","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":"Patient Experience of Treatment with Tirzepatide for Weight Management: Exit Interviews from SURMOUNT-4.","authors":"Chloe Carmichael, Irina Jouravskaya, Elizabeth Collins, Danielle Burns, Jiat Ling Poon, Helen Kitchen, Donna Mojdami, Madhumita Murphy, Nadia Ahmad, Chisom Kanu","doi":"10.1007/s40271-025-00730-0","DOIUrl":"10.1007/s40271-025-00730-0","url":null,"abstract":"<p><strong>Background and objectives: </strong>Tirzepatide is a glucose-dependent insulinotropic polypeptide and glucagon-like peptide-1 receptor agonist, which was approved in 2023 by the US Food and Drug Administration for weight management in adults with obesity or overweight. The purpose of this study was to conduct qualitative exit interviews with participants who had participated in the SURMOUNT-4 clinical trial, to better understand the patient experience of tirzepatide.</p><p><strong>Methods: </strong>Online exit interviews were conducted with adults from the USA who had participated in the SURMOUNT-4 clinical trial for weight management, recruited from 16 US-based SURMOUNT-4 clinical sites. Interviews utilized a semi-structured interview guide, and included questions related to receiving tirzepatide, using a single-use injection pen device, and the overall trial experience. Interviews were audio recorded and transcribed, and analyzed using a content analysis.</p><p><strong>Results: </strong>Eighty-six adults (83% female; mean age 49.9 years) participated in the interviews. All participants shared at least one perceived benefit of tirzepatide experienced during the open-label phase of SURMOUNT-4, including improved appetite control, increased energy, or improved clothing fit. Despite the gastrointestinal side effects experienced, many participants liked the efficacy of tirzepatide, and reported that the single-use injection pen device for administering the study medication was easy to use. Most participants were willing to continue taking tirzepatide.</p><p><strong>Conclusions: </strong>Study findings showed that beyond the direct pharmacological effects of treatment with tirzepatide, participants reported a wide range of perceived improvements across several aspects of their lives. Participants also reported a few negative experiences, including side effects. It is possible that the participants who had a more positive experience were more inclined to participate in the exit interviews. This study highlights the value of exit interviews, which can provide more learning about patient experiences during a clinical trial.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"225-236"},"PeriodicalIF":3.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11985590/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143477193","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":"Qualitative In-trial Interviews: Methods, Challenges, and Best Practice.","authors":"Nicola Williamson, Chloe Howse, Nicola Hodson, Julia Stein, Rob Arbuckle","doi":"10.1007/s40271-024-00726-2","DOIUrl":"10.1007/s40271-024-00726-2","url":null,"abstract":"<p><p>Qualitative in-trial interviews with clinical trial participants are a means of providing rich, in-depth patient experience data to supplement and complement data captured by clinical outcome assessments and other clinical trial efficacy endpoints. Such in-trial interview data can be used to build understanding of disease and treatment experiences, evaluate content validity of clinical outcome assessments, aid interpretation of scores and meaningful changes, inform trial design feasibility and operational considerations, and provide supportive evidence regarding safety, efficacy, and effectiveness. Despite the rapid growth of in-trial interviews as part of clinical development programs in the pharmaceutical industry in recent years, published guidelines regarding the methods, conduct, and implementation of in-trial interviews are scarce. Drawing on published examples and the authors' experiences of conducting in-trial interview studies, this article provides an overview of best practice methods for implementing this methodology (including considerations for study design, sample size, interview conduct, and analysis) and the value of in-trial interview data to answer specific research questions. Operational and logistical considerations are outlined, including recommendations for country selection, site selection, training and communication, adverse event safety reporting, and data management and handling. Well-designed and carefully implemented in-trial interviews can lead to generation of insightful patient experience data that are truly of value to inform regulators, health technology agencies, clinicians, patients, and caregivers about product attributes and the impact of diseases and treatments on patients' lives.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"199-209"},"PeriodicalIF":3.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143081957","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":"Unveiling Preferences in Closed Communities: Development of a Discrete Choice Experiment (DCE) Questionnaire to Elicit Ultra-Orthodox Women Preferences for Video Consultations in Primary Care.","authors":"Irit Chudner, Anat Drach-Zahavy, Batya Madjar, Leah Gelman, Sonia Habib","doi":"10.1007/s40271-025-00734-w","DOIUrl":"10.1007/s40271-025-00734-w","url":null,"abstract":"<p><strong>Background: </strong>Video consultations in primary care settings demonstrate substantial benefits, including improved accessibility, reduced waiting times, and enhanced health management. These services could particularly benefit ultra-Orthodox women in Israel, who typically manage large families and face unique healthcare access challenges as primary caregivers. However, eliciting preferences within this closed religious community presents distinct methodological challenges because of cultural sensitivities and religious restrictions regarding technology use.</p><p><strong>Objective: </strong>We aimed to develop and validate a culturally sensitive, discrete choice experiment questionnaire for eliciting ultra-Orthodox women's preferences regarding video versus in-clinic consultations in primary care settings.</p><p><strong>Methods: </strong>A three-stage mixed-methods approach was employed: (1) 33 semi-structured interviews with key stakeholders (women, men, rabbis, and healthcare providers) to identify attributes and levels; (2) an attribute-ranking exercise with 88 ultra-Orthodox women to refine attributes; and (3) cognitive interviews with 15 women to validate the discrete choice experiment questionnaire.</p><p><strong>Results: </strong>Four key attributes emerged as most important for ultra-Orthodox women when choosing between video and in-clinic consultations: (1) consultation timing (regular hours/after 20:00); (2) travel time; (3) waiting time; and (4) familiarity with the healthcare provider. Importantly, the study revealed the necessity for a dedicated device exclusively for healthcare provider communication, closed to open Internet networks, as a fundamental prerequisite for implementing video consultations in this community. Additional unique findings emerged through this methodological process, contributing to the understanding of technological adoption in closed religious patients' communities.</p><p><strong>Conclusions: </strong>This study provides a comprehensive example of implementing pre-discrete choice experiment stages while addressing unique considerations of a special population. The findings provide a framework for developing inclusive telemedicine services for traditionally underserved populations.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"263-277"},"PeriodicalIF":3.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11985673/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143606819","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":"Stated-Preference Survey Design and Testing in Health Applications.","authors":"Deborah A Marshall, Jorien Veldwijk, Ellen M Janssen, Shelby D Reed","doi":"10.1007/s40271-023-00671-6","DOIUrl":"10.1007/s40271-023-00671-6","url":null,"abstract":"<p><p>Following the conceptualization of a well-formulated and relevant research question, selection of an appropriate stated-preference method, and related methodological issues, researchers are tasked with developing a survey instrument. A major goal of designing a stated-preference survey for health applications is to elicit high-quality data that reflect thoughtful responses from well-informed respondents. Achieving this goal requires researchers to design engaging surveys that maximize response rates, minimize hypothetical bias, and collect all the necessary information needed to answer the research question. Designing such a survey requires researchers to make numerous interrelated decisions that build upon the decision context, selection of attributes, and experimental design. Such decisions include considering the setting(s) and study population in which the survey will be administered, the format and mode of administration, and types of contextual information to collect. Development of a survey is an interactive process in which feedback from respondents should be collected and documented through qualitative pre-test interviews and pilot testing. This paper describes important issues to consider across all major steps required to design and test a stated-choice survey to elicit patient preferences for health preference research.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"187-197"},"PeriodicalIF":3.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139643317","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":"Using Best-Worst Scaling Survey to Investigate the Relative Importance of Attributes Associated with Public Hospital Outpatient Appointments.","authors":"Tilley Pain, Amy Brown, Gail Kingston, Stephen Perks, Corey Patterson, Nerida Firth, Jessica Lester, Luke Sherwood, Sonja Brennan, Deborah Street","doi":"10.1007/s40271-025-00732-y","DOIUrl":"10.1007/s40271-025-00732-y","url":null,"abstract":"<p><strong>Introduction: </strong>Obtaining patient input before healthcare redesign improves patient experience. The Townsville Hospital and Health Service, a regional Australian public health service, seeks to reduce the long wait list for medical specialist appointments by introducing allied health substitution models of care for low-acuity patients. This paper describes a best worst scaling survey conducted to refine attributes associated with outpatient appointments which will be used in a future discrete choice experiment (DCE).</p><p><strong>Methods: </strong>A literature review was conducted to identify attributes associated with medical specialist outpatient appointments and allied health substitution models. An object (or case 1) best worst scaling (BWS) survey was designed using blocks of a balanced incomplete block design and analysed using multinomial logit and mixed logit models. Patients waiting at local specialist outpatient clinics were invited to complete the survey via an iPad. The interviewer collected field notes, which were analysed using content analysis.</p><p><strong>Results: </strong>A total of 12 attributes were identified in the literature review and one from local discussion. The 167 completed responses demonstrated the ranking of attributes were diagnostic accuracy, symptom relief, continuity of care, satisfaction with care, healthcare professional, manner and communication, time on waitlist and onward referral. The least important attributes were reassurance offered, appointment wait time, cost and appointment duration.</p><p><strong>Conclusions: </strong>This BWS survey allows us to reduce the attributes for inclusion in the DCE from 13 to 8. Diagnostic accuracy and symptom relief were of most importance, and appointment wait time and duration were of least importance. This suggests that patients would be willing to be attend different models of care such as allied health primary contact model if clinical outcomes were equivalent to the current medical-led models.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"237-247"},"PeriodicalIF":3.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11985688/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143505319","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":"Incorporating Best-Worst Scaling (BWS) Questions into Focus Groups to Improve Understanding of Patient Preferences and Refine BWS Attributes.","authors":"Deborah A Marshall, Karen V MacDonald, Nitya Suryaprakash, Raza M Mirza, Maida J Sewitch, Geoffrey C Nguyen","doi":"10.1007/s40271-025-00736-8","DOIUrl":"https://doi.org/10.1007/s40271-025-00736-8","url":null,"abstract":"<p><strong>Background and objective: </strong>Best-worst scaling (BWS) is a stated preference elicitation method used for prioritizing attributes of healthcare interventions. Best-worst scaling attribute development is commonly based on literature review, qualitative work, and methodological/clinical expert input. There is limited research incorporating BWS in focus groups as part of the attribute development process. We sought to explore how incorporating BWS questions using the list of potential attributes in focus groups could be used to improve understanding of patient preferences and refine the list of potential BWS attributes as part of the attribute development process.</p><p><strong>Methods: </strong>We administered BWS questions on healthcare priorities for inflammatory bowel disease in five focus groups with Canadian patients with inflammatory bowel disease to (1) understand the \"what,\" \"how,\" and \"why\" of participant choices and (2) note how participants understand the attributes and the language they use to refine the list of potential BWS attributes. A list of 20 potential attributes was used to generate the BWS questions. We coded most/least important choices (\"what\") and used a thematic analysis to derive subthemes indicating \"how\" and \"why\" participants made their choices. We coded how participants understood the attributes/BWS questions and language used when discussing the attributes.</p><p><strong>Results: </strong>Across the 36 participants, the most frequently chosen most/least important attributes were summarized. Three subthemes explaining the \"how\" and \"why\" of participant choices were identified: perceived gain; influence of individual experiences; current health state and personal circumstances. Participants identified challenges understanding specific attributes and BWS questions, and provided suggestions for modifications to attribute language/descriptions. Administering BWS questions in focus groups provided: (1) insight into the assumptions participants made when completing the BWS questions; (2) clarity in language and attribute descriptions, and challenges participants had when completing the BWS questions that can be used to refine the list of potential attributes as part of the attribute development process; and (3) understanding of which attributes were most/least important and why to identify potential attributes to remove during the next steps of the attribute development process.</p><p><strong>Conclusions: </strong>Best-worst scaling questions conducted within focus groups can stimulate discussions around relative importance and prioritization of attributes. Through open dialogue, this method can unveil unforeseen responses or identify areas that are unclear and enable a transparent approach to refine the list of potential attributes as part of the attribute development process.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2025-03-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143634594","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":"How do Design Characteristics Affect Respondent Engagement? Assessing Attribute Non-attendance in Discrete Choice Experiments Valuing the EQ-5D-5L.","authors":"Peiwen Jiang, Deborah Street, Richard Norman, Rosalie Viney, Mark Oppe, Brendan Mulhern","doi":"10.1007/s40271-025-00735-9","DOIUrl":"https://doi.org/10.1007/s40271-025-00735-9","url":null,"abstract":"<p><strong>Introduction: </strong>Discrete choice experiments (DCEs) are increasingly applied to develop value sets for health-related quality-of-life instruments, but respondents may adopt various simplifying heuristics that affect the resulting health state values. Attribute level overlap can make these DCE tasks easier and thereby increase respondent engagement. This study uses choice tasks involving EQ-5D-5L health states to compare designs with and without overlap, constructed using different methods (generator-developed design, Ngene, SAS, and Bayesian D-efficient design) to assess respondent non-attendance to attributes.</p><p><strong>Methods: </strong>A multi-arm DCE using the EQ-5D-5L was conducted in the Australian general population. The performance of designs with various properties was compared using the level of respondent engagement. Respondent engagement was quantified through the inferred attribute non-attendance (ANA) estimated by the equality constrained latent class model. Utility decrements derived using all respondents (i.e., including non-attendees) were compared with estimates obtained only from those who attended to all EQ-5D-5L attributes.</p><p><strong>Results: </strong>The inclusion of overlap improved full attendance rates from 22.3-28.4% to 28.2-54.2%. Within designs with overlap, modified Fedorov designs (constructed using either Ngene or SAS macros) had higher full attendance rates than other designs. The relative attribute importance of the EQ-5D-5L also differed significantly before and after data exclusion using ANA analysis, but there was no clear pattern in the differences.</p><p><strong>Conclusions: </strong>This study found evidence to support the use of modified Fedorov designs (constructed using Ngene or SAS) with attribute overlap to reduce ANA and improve respondent engagement in DCE studies. It highlights the potential value of ANA analysis as a quality-control tool for the inclusion and exclusion of respondents in future health valuation work for the EQ-5D-5L.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2025-03-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143634461","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":"Reporting of Patient and Public Involvement in Technology Appraisal and Assessment Reports: A Rapid Scoping Review.","authors":"Eugenie Evelynne Johnson, Cyril Onwuelazu Uteh, Emma Belilios, Fiona Pearson","doi":"10.1007/s40271-024-00721-7","DOIUrl":"10.1007/s40271-024-00721-7","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;The National Institute for Health and Care Excellence (NICE) produces guidance on the use of health technologies (including new and existing medicines, medical devices, diagnostics and interventional procedures) in the National Health Service. Technology Appraisals inform recommendations on the use of new and existing health technologies. As part of its health technology evaluation process, NICE ask independent research groups known as Evidence or External Assessment Groups (EAGs) to assess or evaluate the available evidence surrounding health technologies. Although patients and the public are involved in the wider NICE Heath Technology Evaluation and Assessment process, little is known about the extent to which patient and public involvement and engagement (PPIE) is undertaken and documented in EAG Reports.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;This rapid scoping review aimed to discover the extent to which PPIE is currently undertaken and documented in EAG Reports, which feed into the wider NICE health technology assessment process, and whether EAG Reports contain a plain language summary.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;We searched the NICE website for guidance published between 27 September, 2022 and 27 September, 2023. All records were downloaded directly from the NICE website into an Excel spreadsheet for extraction. Evaluations that were terminated before guidance was published or where an EAG Report was not available as supporting evidence were excluded. One researcher charted information regarding the type of each EAG Report, whether a plain language summary was included, and whether documentation of PPIE was included in the EAG Report either within a stand-alone section or throughout the main text of the report. A second researcher checked charted information for 20% of these records. We tabulated data and described PPIE conduct and documentation in included EAG Reports within a narrative synthesis.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;A total of 97 EAG Reports were included in this rapid scoping review, the majority of which were documenting Single Technology Appraisals (N = 55). Of the 97 EAG Reports, 11 included a plain language summary. Of these 11 reports, two were Multiple Technology Appraisals, five were Diagnostic Assessment Reviews and four were Early Value Assessments. One Early Value Assessment, one Diagnostic Assessment Review and one Multiple Technology Appraisal reported that they did not conduct PPIE because of time constraints and noted that patients were involved in the wider NICE Appraisal process. Two Early Value Assessments that explicitly reported on PPIE used heterogenous methods of involvement.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;There is currently limited PPIE documented in EAG Reports and inclusion of a plain language summary is uncommon. Further guidance is required to assist EAGs with embedding PPIE and a plain language summary into their Reports taking into consideration the ultra-rapi","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"109-114"},"PeriodicalIF":3.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142565220","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":"Assessing Preferences of Patients with Chronic Spontaneous Urticaria for Injectable Treatment Profiles.","authors":"Ana Maria Giménez-Arnau, Maria-Magdalena Balp, Andrii Danyliv, Tonya Winders, James O'Donoghue, Jörn Kleebach, Samantha Morrison, Shaun Walsh, Maike Mueller, Daniela Lopez-Ortiz, Marcus Maurer, Jonathan A Bernstein","doi":"10.1007/s40271-024-00725-3","DOIUrl":"10.1007/s40271-024-00725-3","url":null,"abstract":"<p><strong>Background: </strong>In the context of injectable biologic products approved or in development for chronic spontaneous urticaria (CSU), it is important to capture which treatment attributes matter most to patient and what trade-offs patients are willing to make.</p><p><strong>Objectives: </strong>The CHOICE-CSU study aimed to quantify patient preferences toward injectable treatment attributes among patients with CSU, inadequately controlled by H1-antihistamines.</p><p><strong>Methods: </strong>This was a two-phase cross-sectional patient preference study in adult patients with a diagnosis of CSU, inadequately controlled by H1-antihistamines. A qualitative phase collected patients' insights and relevant treatment attributes that mattered to them, and the outputs were used for the quantitative phase to create the actual injectable treatment profiles with attributes and levels such as: efficacy, safety, and mode of administration. The quantitative phase used discrete choice experiment (DCE) methodology. Eligible patients were asked to make hypothetical choices between 12 treatment profile pairs, created by Sawtooth Software^TM. The DCE data were analyzed using hierarchical Bayesian logistic regression models, enabling the quantification of the relative importance of each attribute/level during the decision-making process.</p><p><strong>Results: </strong>A total of 450 respondents participated in the DCE. The key attributes driving respondent preference amongst injectable treatment options were type of administration device (relative importance 18.5%), complete control of urticaria (relative importance 17.4%), and resolution of angioedema (relative importance 16.4%). Keeping all other attributes and levels equal, the predicted choice share was higher for a profile with an auto-injector versus one with a pre-filled syringe (72.9% versus 27.1%).</p><p><strong>Conclusions: </strong>The CHOICE-CSU study is the first study to provide a quantitative assessment of preferences that patients with CSU, inadequately controlled by H1-antihistamines, have for injectable treatment attributes. Symptom-free periods are the most important overriding therapy goal for patients, and patients will accept some inconveniences, such as administration mode, to achieve this. Additionally, when efficacy is equivalent, administration ease of injectable therapies is valued by patients. As new CSU oral treatment options emerge, additional testing of patient preference toward oral treatments will be required.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"173-185"},"PeriodicalIF":3.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11832561/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143054061","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}