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Measuring Patient Preferences to Inform Clinical Trial Design: An Example in Rheumatoid Arthritis. 测量患者偏好为临床试验设计提供信息:以类风湿关节炎为例。
IF 3.4 3区 医学
Patient-Patient Centered Outcomes Research Pub Date : 2025-03-01 Epub Date: 2024-12-12 DOI: 10.1007/s40271-024-00724-4
Gillian R Currie, Jan Storek, Karen V MacDonald, Glen Hazlewood, Caylib Durand, John F P Bridges, Dianne Mosher, Deborah A Marshall
{"title":"Measuring Patient Preferences to Inform Clinical Trial Design: An Example in Rheumatoid Arthritis.","authors":"Gillian R Currie, Jan Storek, Karen V MacDonald, Glen Hazlewood, Caylib Durand, John F P Bridges, Dianne Mosher, Deborah A Marshall","doi":"10.1007/s40271-024-00724-4","DOIUrl":"10.1007/s40271-024-00724-4","url":null,"abstract":"<p><strong>Background: </strong>Allogeneic bone marrow transplantation (BMT) may be a curative treatment for patients with rheumatoid arthritis (RA), but it has serious risks, including death. It is uncertain whether patients would accept the risks and benefits of BMT and participate in clinical trials. We conducted a discrete choice experiment (DCE) to quantify risk tolerance and benefit-risk trade-offs to inform the design of a clinical trial for BMT.</p><p><strong>Methods: </strong>We conducted a DCE with three attributes (three levels each): chance of stopping disease progression (50-90%), increased chance of death in year after transplant (3-15%), and chance of chronic graft-versus-host disease (cGVHD) (3-15%). An orthogonal main effects design of nine binary choice tasks were presented for two scenarios: one considering their current situation and a second scenario where the patient has failed seven anti-rheumatic drugs. Participants were recruited from the Rheum4U inflammatory arthritis registry. Choice data were analyzed using a logit model accounting for multiple responses per participant.</p><p><strong>Results: </strong>Sixty patients participated. Most (82%) had severe disease, and the median number of anti-rheumatic drugs previously taken was 6 (range 0-18). As expected, an increased chance of stopping disease progression increases the probability of choosing BMT, while increased chance of both risks decreases the probability. Patients were willing to accept a 3% increase in risk of death or 6% increase in chance of chronic GVHD for a 10% increase in the chance of stopping disease progression. For the most clinically likely BMT risk-benefit profiles, and the likely initial target population of patients who have failed multiple biologics, between 72% and 91% of patients would choose BMT.</p><p><strong>Conclusions: </strong>Patients with RA are willing to accept substantial risks for a chance to stop disease progression with BMT, suggesting that a pilot trial of BMT for RA could successfully recruit patients. Preference studies have an important role in informing patient-centered clinical trial planning and design.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"161-171"},"PeriodicalIF":3.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142815025","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}
引用次数: 0
What Next for the Science of Patient Preference? Interoperability, Standardization, and Transferability.
IF 3.4 3区 医学
Patient-Patient Centered Outcomes Research Pub Date : 2025-03-01 Epub Date: 2025-01-28 DOI: 10.1007/s40271-025-00727-9
Kevin Marsh, Juan Marcos Gonzalez Sepulveda, Conny Berlin, Bennett Levitan, Marco Boeri, Catharina G M Groothuis-Oudshoorn, Norah L Crossnohere, Cecilia Jimenez-Moreno, Barry Liden, Isabelle Stoeckert, Jorien Veldwijk, Stephen Watt, Brett Hauber
{"title":"What Next for the Science of Patient Preference? Interoperability, Standardization, and Transferability.","authors":"Kevin Marsh, Juan Marcos Gonzalez Sepulveda, Conny Berlin, Bennett Levitan, Marco Boeri, Catharina G M Groothuis-Oudshoorn, Norah L Crossnohere, Cecilia Jimenez-Moreno, Barry Liden, Isabelle Stoeckert, Jorien Veldwijk, Stephen Watt, Brett Hauber","doi":"10.1007/s40271-025-00727-9","DOIUrl":"10.1007/s40271-025-00727-9","url":null,"abstract":"<p><p>Using patient preference information (PPI) to incorporate patient voices into the drug development lifecycle can help align therapies with the needs and values of patients. However, several barriers have limited the use of PPI, including a lack of clarity on its use by decision-makers, a need for greater decision-maker trust in PPI, and a lack of time, budgets, and access to specialist expertise. The value proposition for PPI could be enhanced by making it FAIR: Findable, Accessible, Interoperable, and Reusable. To support the development of a research agenda to deliver FAIR PPI, we reviewed related endeavors in the development of repositories of existing studies, disease models, benefit transfer, and common data standards. We concluded that developing FAIR PPI would require advances in the science of PPI, including the establishment of a consortium, mirroring the Clinical Data Interchange Standards Consortium (CDISC) or Observational Medical Outcomes Partnership (OPOM), to develop PPI data standards, and research into the sources of variation in patient preferences. This will require the science of PPI to graduate from being a body of empirical observations to developing theories that explain variations in patient preferences, simultaneously driving both efficiency in the generation of PPI and trust in PPI.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"101-108"},"PeriodicalIF":3.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143054063","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}
引用次数: 0
Content Validity Assessment of the Insomnia Daytime Symptoms and Impacts Questionnaire (IDSIQ) Among Adults with Insomnia and Nocturia.
IF 3.4 3区 医学
Patient-Patient Centered Outcomes Research Pub Date : 2025-02-27 DOI: 10.1007/s40271-025-00733-x
Miriam Kimel, Leah Kleinman, Tobias Di Marco, Antonio Olivieri, Andrea Schulz, Katherine Kirk, Andrea Phillips Beyer
{"title":"Content Validity Assessment of the Insomnia Daytime Symptoms and Impacts Questionnaire (IDSIQ) Among Adults with Insomnia and Nocturia.","authors":"Miriam Kimel, Leah Kleinman, Tobias Di Marco, Antonio Olivieri, Andrea Schulz, Katherine Kirk, Andrea Phillips Beyer","doi":"10.1007/s40271-025-00733-x","DOIUrl":"https://doi.org/10.1007/s40271-025-00733-x","url":null,"abstract":"<p><strong>Purpose: </strong>The study aimed to support use of the Insomnia Daytime Symptoms and Impacts Questionnaire (IDSIQ) in clinical trials for patients with concomitant insomnia disorder and nocturia.</p><p><strong>Methods: </strong>Eligible participants in this observational study were United States (US) residents aged ≥ 55 years who experienced self-reported symptoms reflecting the diagnostic criteria for insomnia disorder and nocturia (i.e., experienced ≥ 2 nocturnal voids per night within 30 days of screening). Concept elicitation (n = 10), confirmation of limited concepts (n = 5), and cognitive debriefing of the IDSIQ (n = 10) were conducted with 20 participants. A content analysis approach was used.</p><p><strong>Results: </strong>Most participants in the overall sample were female (n = 13/20, 65%) and white (n = 17/20, 85%), with a mean age of 60.9 years. Based on concept elicitation, most participants (n = 9/10, 90%) experienced insomnia 5-7 nights per week and nocturia 7 nights per week. Participants identified impacts to their emotions, social activities, physical abilities, cognitive function, daily activities, work, and family for insomnia disorder and nocturia. These concepts were confirmed with the five additional interviews. Concept saturation for both disorders was achieved in all participants by the seventh concept elicitation interview. All cognitive interview participants (n = 10) understood the IDSIQ instructions, items, and response options.</p><p><strong>Conclusions: </strong>Having concurrent insomnia disorder and nocturia is associated with daytime symptoms and impacts to functioning. All participants who were debriefed understood all IDSIQ items and response options. These findings suggest the IDSIQ is a content-valid instrument for use in clinical trials with patients with insomnia and comorbid nocturia.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2025-02-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143517259","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}
引用次数: 0
Using Best-Worst Scaling Survey to Investigate the Relative Importance of Attributes Associated with Public Hospital Outpatient Appointments.
IF 3.4 3区 医学
Patient-Patient Centered Outcomes Research Pub Date : 2025-02-26 DOI: 10.1007/s40271-025-00732-y
Tilley Pain, Amy Brown, Gail Kingston, Stephen Perks, Corey Patterson, Nerida Firth, Jessica Lester, Luke Sherwood, Sonja Brennan, Deborah Street
{"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":"https://doi.org/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":""},"PeriodicalIF":3.4,"publicationDate":"2025-02-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Patient Experience of Treatment with Tirzepatide for Weight Management: Exit Interviews from SURMOUNT-4.
IF 3.4 3区 医学
Patient-Patient Centered Outcomes Research Pub Date : 2025-02-22 DOI: 10.1007/s40271-025-00730-0
Chloe Carmichael, Irina Jouravskaya, Elizabeth Collins, Danielle Burns, Jiat Ling Poon, Helen Kitchen, Donna Mojdami, Madhumita Murphy, Nadia Ahmad, Chisom Kanu
{"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":"https://doi.org/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":""},"PeriodicalIF":3.4,"publicationDate":"2025-02-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Routine Quality-of-Life Measurement in Residential Aged Care: Staff, Resident, and Family Perspectives.
IF 3.4 3区 医学
Patient-Patient Centered Outcomes Research Pub Date : 2025-02-03 DOI: 10.1007/s40271-025-00729-7
Andrew Simon Gilbert, Frances Batchelor, Nancy Devlin, Briony Dow, Brendan Mulhern, Rosalie Viney, Tessa Peasgood, Lidia Engel
{"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":"https://doi.org/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":""},"PeriodicalIF":3.4,"publicationDate":"2025-02-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Qualitative In-trial Interviews: Methods, Challenges, and Best Practice.
IF 3.4 3区 医学
Patient-Patient Centered Outcomes Research Pub Date : 2025-02-03 DOI: 10.1007/s40271-024-00726-2
Nicola Williamson, Chloe Howse, Nicola Hodson, Julia Stein, Rob Arbuckle
{"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":"https://doi.org/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":""},"PeriodicalIF":3.4,"publicationDate":"2025-02-03","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}
引用次数: 0
Patient Preferences for First-Line Treatment of Locally Advanced or Metastatic Urothelial Carcinoma: An Application of Multidimensional Thresholding. 患者对局部晚期或转移性尿路上皮癌一线治疗的偏好:多维阈值法的应用。
IF 3.4 3区 医学
Patient-Patient Centered Outcomes Research Pub Date : 2025-01-01 Epub Date: 2024-08-28 DOI: 10.1007/s40271-024-00709-3
Andrea B Apolo, Christine Michaels-Igbokwe, Nicholas I Simon, David J Benjamin, Mallory Farrar, Zsolt Hepp, Lisa Mucha, Sebastian Heidenreich, Katelyn Cutts, Nicolas Krucien, Natasha Ramachandran, John L Gore
{"title":"Patient Preferences for First-Line Treatment of Locally Advanced or Metastatic Urothelial Carcinoma: An Application of Multidimensional Thresholding.","authors":"Andrea B Apolo, Christine Michaels-Igbokwe, Nicholas I Simon, David J Benjamin, Mallory Farrar, Zsolt Hepp, Lisa Mucha, Sebastian Heidenreich, Katelyn Cutts, Nicolas Krucien, Natasha Ramachandran, John L Gore","doi":"10.1007/s40271-024-00709-3","DOIUrl":"10.1007/s40271-024-00709-3","url":null,"abstract":"<p><strong>Objectives: </strong>Patient preferences have the potential to influence the development of new treatments for locally advanced/metastatic urothelial carcinoma (la/mUC), and therefore we explored how patients with la/mUC value different attributes of first-line treatments.</p><p><strong>Methods: </strong>An online preference survey and multidimensional thresholding (MDT) exercise were developed following a targeted literature review and qualitative interviews with physicians, patients with la/mUC, and their caregivers. Treatment attributes included two benefits (overall response rate [ORR], pain related to bladder cancer [scored 0-100; 100 being the worst pain possible]) and four treatment-related risks (peripheral neuropathy, severe side effects, mild to moderate nausea, mild to moderate skin reactions). A Dirichlet regression was used to estimate average preference weights. Marginal utility and the reduction in ORR that patients would accept in exchange for a 10-point decrease or a 10% decrease in other attributes were calculated.</p><p><strong>Results: </strong>A total of 100 patients were recruited and self-completed the survey and MDT. Mean patient age was 64.9 years (standard deviation, 7.6), 54% were female, and 38% identified as white. All included treatment attributes had a statistically significant impact on preferences. Changes in ORR had the largest impact, followed by cancer-related pain and treatment-related risks. Patients were willing to accept an 8.4% decrease in ORR to reduce their pain level by 10 points or a 7.8% decrease in ORR to reduce the risk of peripheral neuropathy by 10%. For a 10% decrease in severe side effects, mild to moderate nausea, or skin reaction, patients would accept decreases in ORR of 5.5%, 3.7%, or 3.4%, respectively.</p><p><strong>Conclusions: </strong>Of the attributes tested, changes in ORR were most important to patients. Patients made tradeoffs between treatment attributes indicating that a lower ORR may be acceptable for an improvement in other attributes such as reduced cancer-related pain or the risk of treatment-related adverse events.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"77-87"},"PeriodicalIF":3.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11717873/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142094152","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}
引用次数: 0
Evaluating Comprehensibility of 157 Patient-Reported Outcome Measures (PROMs) in the Nationwide Dutch Outcome-Based Healthcare Program: More Attention for Comprehensibility of PROMs is Needed. 评估荷兰全国基于结果的医疗保健计划中 157 项患者报告结果指标 (PROM) 的可理解性:需要更加关注 PROMs 的可理解性。
IF 3.4 3区 医学
Patient-Patient Centered Outcomes Research Pub Date : 2025-01-01 Epub Date: 2024-08-13 DOI: 10.1007/s40271-024-00710-w
Attie Tuinenburg, Domino Determann, Elise H Quik, Esmee M van der Willik, Geeske Hofstra, Joannes M Hallegraeff, Ingrid Vriend, Lisanne Warmerdam, Hester E van Bommel, Gudule Boland, Martijn A H Oude Voshaar
{"title":"Evaluating Comprehensibility of 157 Patient-Reported Outcome Measures (PROMs) in the Nationwide Dutch Outcome-Based Healthcare Program: More Attention for Comprehensibility of PROMs is Needed.","authors":"Attie Tuinenburg, Domino Determann, Elise H Quik, Esmee M van der Willik, Geeske Hofstra, Joannes M Hallegraeff, Ingrid Vriend, Lisanne Warmerdam, Hester E van Bommel, Gudule Boland, Martijn A H Oude Voshaar","doi":"10.1007/s40271-024-00710-w","DOIUrl":"10.1007/s40271-024-00710-w","url":null,"abstract":"<p><strong>Introduction: </strong>Patient-reported outcomes measures (PROMs) are increasingly prevalent in healthcare and used for shared decision-making and healthcare quality evaluation. However, the extent to which patients with varying health literacy levels can complete PROMs is often overlooked. This may lead to biased aggregated data and patients being excluded from studies or other PROM collection initiatives. This cross-sectional study evaluates the comprehensibility of 157 well-known and widely used PROM scales using a comprehensibility checklist.</p><p><strong>Methods: </strong>Pairs of two independent raters scored 157 PROM scales designed for adults included in the 35 sets of outcome information developed as part of the Dutch Outcome-Based Healthcare Program. The PROM scales were scored on the eight comprehensibility domains of the Pharos Checklist for Questionnaires in Healthcare (PCQH). Interrater agreement of domain ratings was assessed using Intraclass Correlation Coefficients or Cohen's kappa. Subsequently, final ratings were established through discussion and used to evaluate the domain-specific comprehensibility rating for each PROM scale.</p><p><strong>Results: </strong>Comprehensibility of a large number of PROM scales (n = 157), which cover a wide range of diseases and conditions across Dutch medical specialist care, was assessed. While most PROM scales were written at an accessible language level, with minimal use of medical terms, instruction clarity, number of questions, and response options emerged as significant issues, affecting a substantial proportion of PROM scales. Interrater agreement was high for most domains of the PCQH.</p><p><strong>Conclusion: </strong>This study highlights the need for greater attention to the comprehensibility of PROMs to ensure their accessibility to all patients, including those with low health literacy. The PCQH can be a valuable tool in PROM development in addition to qualitative methods and in selection processes enabling comparison of comprehensibility between PROMs. However, the PCQH needs further development and validation for these purposes. Enhancing the comprehensibility of PROMs is essential for their effective incorporation in healthcare evaluation and decision-making processes.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"65-76"},"PeriodicalIF":3.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11717823/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141977216","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}
引用次数: 0
A Framework to Promote Implementation of Patient-Reported Outcomes in Institutions Caring for Vulnerable and Underserved Cancer Populations. 促进在照顾弱势和未得到充分服务的癌症患者的机构中实施 "患者报告结果 "的框架。
IF 3.4 3区 医学
Patient-Patient Centered Outcomes Research Pub Date : 2025-01-01 Epub Date: 2024-06-22 DOI: 10.1007/s40271-024-00703-9
Anne L R Schuster, Norah L Crossnohere, Eric Adjei Boakye, Rebekah Angove, Billie Baldwin, Esteban A Barreto, Ronald C Chen, Theresa W Gillespie, Betty Hamilton, Nadine Jackson McCleary, Maimah Karmo, Tara Kaufmann, William Lee, Vikas Mehta, Larissa Meyer, Kriti Mittal, Leah Owens, Rachel Peterson, Andrea Pusic, Anne Marie Rainey, Angelique Richardson, Lauren Shapiro, Bethany Sibbitt, Cardinale Smith, Mary Vargo, Andrew Vickers, Michael Brundage, Claire Snyder
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