Patient-Patient Centered Outcomes Research最新文献

{"title":"Attitudes Toward the Adoption of Remote Patient Monitoring and Artificial Intelligence in Parkinson's Disease Management: Perspectives of Patients and Neurologists.","authors":"Carlos Antonio Godoy Junior, Francesco Miele, Laura Mäkitie, Eleonora Fiorenzato, Maija Koivu, Lytske Jantien Bakker, Carin Uyl-de Groot, William Ken Redekop, Welmoed Kirsten van Deen","doi":"10.1007/s40271-023-00669-0","DOIUrl":"10.1007/s40271-023-00669-0","url":null,"abstract":"<p><strong>Objective: </strong>Early detection of Parkinson's Disease (PD) progression remains a challenge. As remote patient monitoring solutions (RMS) and artificial intelligence (AI) technologies emerge as potential aids for PD management, there's a gap in understanding how end users view these technologies. This research explores patient and neurologist perspectives on AI-assisted RMS.</p><p><strong>Methods: </strong>Qualitative interviews and focus-groups were conducted with 27 persons with PD (PwPD) and six neurologists from Finland and Italy. The discussions covered traditional disease progression detection and the prospects of integrating AI and RMS. Sessions were recorded, transcribed, and underwent thematic analysis.</p><p><strong>Results: </strong>The study involved five individual interviews (four Italian participants and one Finnish) and six focus-groups (four Finnish and two Italian) with PwPD. Additionally, six neurologists (three from each country) were interviewed. Both cohorts voiced frustration with current monitoring methods due to their limited real-time detection capabilities. However, there was enthusiasm for AI-assisted RMS, contingent upon its value addition, user-friendliness, and preservation of the doctor-patient bond. While some PwPD had privacy and trust concerns, the anticipated advantages in symptom regulation seemed to outweigh these apprehensions.</p><p><strong>Discussion: </strong>The study reveals a willingness among PwPD and neurologists to integrate RMS and AI into PD management. Widespread adoption requires these technologies to provide tangible clinical benefits, remain user-friendly, and uphold trust within the physician-patient relationship.</p><p><strong>Conclusion: </strong>This study offers insights into the potential drivers and barriers for adopting AI-assisted RMS in PD care. Recognizing these factors is pivotal for the successful integration of these digital health tools in PD management.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11039525/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139106899","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":"Patients' Preferences for Systemic Lupus Erythematosus Treatments-A Discrete Choice Experiment.","authors":"Hannah Collacott, Andrea Phillips-Beyer, Nicolas Krucien, Bruno Flamion, Kevin Marsh","doi":"10.1007/s40271-023-00670-7","DOIUrl":"10.1007/s40271-023-00670-7","url":null,"abstract":"<p><strong>Background: </strong>Symptoms of systemic lupus erythematosus (SLE) vary between patients, but those of increased disease activity typically include musculoskeletal and mucocutaneous manifestations such as joint pain, swelling, and rashes. Several treatment options are available to patients with SLE with variable efficacy. Many treatments, especially corticosteroids, cause unwanted side effects, although little is currently known about patients' preferences for treatments of SLE.</p><p><strong>Objective: </strong>We aimed to identify which attributes of SLE treatment are valued by patients and to quantify their relative importance.</p><p><strong>Methods: </strong>Adult participants with moderate-to-severe SLE were asked to make a series of choices between two hypothetical treatments in an online discrete choice experiment (DCE). A latent class model (LCL) was estimated to analyze choice data. Relative attribute importance (RAI) was calculated to determine the importance of each attribute to participants.</p><p><strong>Results: </strong>A total of 342 participants from the USA completed the survey. A three-class LCL model was found to have the best fit. Class 1 (non-attenders) had non-significant preferences across all attributes. To achieve a better fit, a constrained LCL (cLCL) model was run with the two remaining classes. The most important attributes for participants in class 2 (benefit-seekers) were joint pain (RAI = 32.0%), non-joint pain (RAI = 21.8%), fatigue (RAI = 20.1%), and skin rashes and itching (RAI = 19.1%). The most important attributes for participants in class 3 (risk-avoiders) were risk of non-severe side effects from corticosteroids (RAI = 28.4%), risk of severe side effects from corticosteroids (RAI = 21.4%), and the risk of infections (RAI = 19.2%). Risk-avoiders were more likely to have been diagnosed with SLE for a longer period (>1 year) and were more likely to have experience with oral corticosteroids.</p><p><strong>Conclusions: </strong>SLE patients fall into two groups with distinct preferences: benefit-seekers, who prioritize reducing the impact of disease symptoms, and risk-avoiders, who prioritize avoiding treatment risks. The implication of this finding will depend on the reasons for these differences, which warrant further research. Our study suggests that these differences arise due to the impact of disease and treatment experience on preferences. If so, well-informed patients may not be willing to tolerate the risks associated with oral corticosteroids in exchange for their benefits.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139547097","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":"Key Insights into Developing Qualitative Concept Elicitation Work for Outcome Measures with Children and Young People.","authors":"Samantha Husbands, Paul Mark Mitchell, Joanna Coast","doi":"10.1007/s40271-023-00663-6","DOIUrl":"10.1007/s40271-023-00663-6","url":null,"abstract":"<p><p>Qualitative concept elicitation can develop meaningful patient-reported outcome measures for children and young people; however, the methods used for concept elicitation are often underreported for this population. This paper provides in-depth insight into the methods used for concept elicitation with children and young people, with a focus on key stages of concept elicitation that are challenging or unique to doing this research with children. Drawing on our experiences of developing wellbeing measures for children and young people aged 6-15 years, we detail the processes followed in our qualitative concept elicitation work, covering issues related to sampling and recruitment, encouraging informed assent and freedom over children and young people's involvement in concept elicitation, and the use of creative and participatory methods to develop measure items. We provide reflections on the approaches taken to navigate challenging aspects of concept elicitation with children and young people. Our reflections suggest that using existing links and online recruitment methods can help to navigate organisational gatekeepers, and using appropriate processes to develop study information and obtain informed assent can ensure that research is inclusive and that children have the freedom to decide whether to be involved. Our adaptation of a creative and participatory activity to generate concepts for measure items suggests that such approaches can be engaging and may help to give children greater control over their participation. In detailing our methods, we hope to have developed a useful resource for other researchers, while highlighting the value of transparent reporting in this area.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11039412/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139730988","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":"Pretesting Discrete-Choice Experiments: A Guide for Researchers.","authors":"Nicola B Campoamor, Christi J Guerrini, Whitney Bash Brooks, John F P Bridges, Norah L Crossnohere","doi":"10.1007/s40271-024-00672-z","DOIUrl":"10.1007/s40271-024-00672-z","url":null,"abstract":"<p><p>Discrete-choice experiments (DCEs) are a frequently used method to explore the preferences of patients and other decision-makers in health. Pretesting is an essential stage in the design of a high-quality choice experiment and involves engaging with representatives of the target population to improve the readability, presentation, and structure of the preference instrument. The goal of pretesting in DCEs is to improve the validity, reliability, and relevance of the survey, while decreasing sources of bias, burden, and error associated with preference elicitation, data collection, and interpretation of the data. Despite its value to inform DCE design, pretesting lacks documented good practices or clearly reported applied examples. The purpose of this paper is: (1) to define pretesting and describe the pretesting process specifically in the context of a DCE, (2) to present a practical guide and pretesting interview discussion template for researchers looking to conduct a rigorous pretest of a DCE, and (3) to provide an illustrative example of how these resources were operationalized to inform the design of a complex DCE aimed at eliciting tradeoffs between personal privacy and societal benefit in the context of a police method known as investigative genetic genealogy (IGG).</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10894089/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139742663","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":"Impact of Difelikefalin on the Health-Related Quality of Life of Haemodialysis Patients with Moderate-To-Severe Chronic Kidney Disease-Associated Pruritus: A Single-Arm Intervention Trial.","authors":"James Fotheringham, Julian Guest, Joerg Latus, Edgar Lerma, Isabelle Morin, Thilo Schaufler, Marco Soro, Sonja Ständer, Steven Zeig","doi":"10.1007/s40271-023-00668-1","DOIUrl":"10.1007/s40271-023-00668-1","url":null,"abstract":"<p><strong>Objective: </strong>Chronic kidney disease-associated pruritus (CKD-aP) can have a substantial negative impact on health-related quality of life (HRQoL), including an increased risk of depression, anxiety and sleep disturbance. This trial aimed to assess the impact of intravenous difelikefalin on HRQoL in haemodialysis patients with moderate-to-severe CKD-aP.</p><p><strong>Methods: </strong>Post hoc analysis of an open-label, multicentre, single-arm intervention trial assessed pruritus severity and HRQoL at baseline and at 12 weeks of difelikefalin treatment using Worst Itching Intensity Numerical Rating Scale (WI-NRS), Sleep Quality Numeric Rating Scale (SQ-NRS), 5-D itch scale, Skindex-10 scale, EQ-5D-5L with Pruritus Bolt-On (EQ-PSO).</p><p><strong>Results: </strong>A total of 222 patients received ≥ 1 dose of difelikefalin, and 197 patients completed 12 weeks of difelikefalin treatment. Clinically meaningful changes from baseline to 12 weeks were observed in all disease-specific measures: 73.7% of patients achieved a ≥ 3-point reduction in the weekly mean of 24 h WI-NRS scores and 66% of patients experienced ≥ 3-point improvements in SQ-NRS scores. Improvements were also observed in all Skindex-10 scale and 5-D itch scale domain scores. The percentage of patients reporting no problems in all EQ-PSO domains increased from 1.4 to 24.7% (p < 0.001), respectively. Patients' generic HRQoL EQ-5D-5L mean utility and EQ-5D visual analogue scale scores increased from baseline to 12 weeks: mean changes 0.04 (p = 0.001) and 2.8 (p = 0.046), respectively.</p><p><strong>Conclusions: </strong>Patients undergoing haemodialysis with moderate-to-severe CKD-aP receiving difelikefalin reported experiencing clinically meaningful improvements in both their pruritus symptoms and itch-related QoL.</p><p><strong>Clinicaltrials: </strong>gov registration number, NCT03998163; first submitted, 7 May 2019.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10894140/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139405167","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":"Prosocial Behaviour and Antibiotic Resistance: Evidence from a Discrete Choice Experiment.","authors":"Mirko Ancillotti, Samare P I Huls, Eva M Krockow, Jorien Veldwijk","doi":"10.1007/s40271-023-00666-3","DOIUrl":"10.1007/s40271-023-00666-3","url":null,"abstract":"<p><strong>Introduction: </strong>The health of a community depends on the health of its individuals; therefore, individual health behaviour can implicitly affect the health of the entire community. This is particularly evident in the case of infectious diseases. Because the level of prosociality in a community might determine the effectiveness of health programmes, prosocial behaviour may be a crucial disease-control resource. This study aimed to extend the literature on prosociality and investigate the role of altruism in antibiotic decision making.</p><p><strong>Methods: </strong>A discrete choice experiment was conducted to assess the influence of altruism on the general public's preferences regarding antibiotic treatment options. The survey was completed by 378 Swedes. Latent class analysis models were used to estimate antibiotic treatment characteristics and preference heterogeneity. A three-class model resulted in the best model fit, and altruism significantly impacted preference heterogeneity.</p><p><strong>Results: </strong>Our findings suggest that people with higher altruism levels had more pronounced preferences for treatment options with lower contributions to antibiotic resistance and a lower likelihood of treatment failure. Furthermore, altruism was statistically significantly associated with sex, education, and health literacy.</p><p><strong>Conclusions: </strong>Antibiotic awareness, trust in healthcare systems, and non-discriminatory priority setting appear to be structural elements conducive to judicious and prosocial antibiotic behaviour. This study suggests that prosocial messages could help to decrease the demand for antibiotic treatments.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10894120/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138808580","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":"How to Present a Decision Object in Health Preference Research: Attributes and Levels, the Decision Model, and the Descriptive Framework.","authors":"Axel C Mühlbacher, Esther W de Bekker-Grob, Oliver Rivero-Arias, Bennett Levitan, Caroline Vass","doi":"10.1007/s40271-024-00673-y","DOIUrl":"https://doi.org/10.1007/s40271-024-00673-y","url":null,"abstract":"<p><p>In health preference research (HPR) studies, data are generated by participants'/subjects' decisions. When developing an HPR study, it is therefore important to have a clear understanding of the components of a decision and how those components stimulate participant behavior. To obtain valid and reliable results, study designers must sufficiently describe the decision model and its components. HPR studies require a detailed examination of the decision criteria, detailed documentation of the descriptive framework, and specification of hypotheses. The objects that stimulate subjects' decisions in HPR studies are defined by attributes and attribute levels. Any limitations in the identification and presentation of attributes and levels can negatively affect preference elicitation, the quality of the HPR data, and study results. This practical guide shows how to link the HPR question to an underlying decision model. It covers how to (1) construct a descriptive framework that presents relevant characteristics of a decision object and (2) specify the research hypotheses. The paper outlines steps and available methods to achieve all this, including the methods' advantages and limitations.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-02-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139716661","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":"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":"https://doi.org/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":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-01-31","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":"Preferences and Willingness to Pay for Medication in Patients with Renal Cell Carcinoma in China: A Discrete-Choice Experiment.","authors":"Ruilin Ding, Rong Shao, Lingli Zhang, Jianzhou Yan","doi":"10.1007/s40271-023-00659-2","DOIUrl":"10.1007/s40271-023-00659-2","url":null,"abstract":"<p><strong>Objective: </strong>We aimed to assess the relative importance of attributes and the willingness to pay for pharmacological therapies among patients with renal cell carcinoma in China.</p><p><strong>Methods: </strong>Patients with renal cell carcinoma completed a D-efficient-designed, discrete-choice experiment online survey that presented a series of ten trade-off questions and one examining scenario. Based on the literature review and consultations with patients with renal cell carcinoma and clinicians, each question included a pair of hypothetical renal cell carcinoma medication profiles characterized by seven attributes including progression-free survival, objective response rate, medication regimen, fatigue, gastrointestinal reaction, hand-foot syndrome, and monthly out-of-pocket costs. Relative importance and willingness to pay were calculated using coefficients estimated by mixed logit regression in the main analysis. Subgroup analyses were conducted considering the heterogeneity of the participants, based on sex, education level, and income level, using conditional logit regression.</p><p><strong>Results: </strong>The analysis incorporated responses from 182 Chinese respondents. Except for the medication regimen, all attributes were statistically significant. Progression-free survival was the most important attribute, followed by objective response rate, monthly out-of-pocket costs, fatigue, gastrointestinal reaction, and hand-foot syndrome. Patients were willing to pay ￥2010.51 ($298.30), ￥494.93 ($73.43) for 1 unit improvement of progression-free survival, and objective response rate, and￥7558.93 ($1121.50), ￥6927.24 ($1027.78) to avoid experiencing fatigue and gastrointestinal reaction, respectively. Differences in preferences and willingness to pay were found according to patients' gender, income, and education level.</p><p><strong>Conclusions: </strong>In China, patients with renal cell carcinoma preferred medications with better efficacy (objective response rate and progression-free survival) and lower out-of-pocket costs. Heterogeneity can be found in preferences and willingness to pay based on patients' gender, income, and education levels.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138464262","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":"Determining Commonalities in the Experiences of Patients with Rare Diseases: A Qualitative Analysis of US Food and Drug Administration Patient Engagement Sessions.","authors":"Catherine Mease, Lewis J Fermaglich, Karen Jackler, Shawn Shermer, Kathleen L Miller","doi":"10.1007/s40271-023-00648-5","DOIUrl":"10.1007/s40271-023-00648-5","url":null,"abstract":"<p><strong>Background: </strong>Rare diseases are estimated to affect more than one in ten Americans. However, most patients with a rare disease face significant emotional, physical, and social challenges. To better understand the burden of disease and unmet needs, the US Food and Drug Administration (FDA) conducts and supports multiple patient engagement platforms. We analyzed summaries from these discussions to identify commonalities among patients with disparate rare diseases, the results of which could inform priorities for cross-disease policies and medical product development.</p><p><strong>Methods: </strong>We conducted a qualitative analysis of patient engagement session summaries to investigate shared experiences across rare diseases. Cross-disease similarities were identified within four dimensions: product development/regulatory, clinical/physical, social/psychological, and economic/financial. Summaries from 29 rare diseases were included in our analyses.</p><p><strong>Results: </strong>Within the product development/regulatory dimension, we observed that patients and caregivers across rare diseases shared the desire for development of medical products that cured their disease or improved their overall quality of life. In the clinical/physical dimension, we found that patients had numerous common symptoms, including pain and fatigue. In the social/psychological dimension, we observed significant negative impact on mental health. Within the economic/financial dimension, patients and caregivers shared that disease burden caused significant financial hardships.</p><p><strong>Conclusion: </strong>We found remarkable similarities among patients with rare diseases across all four dimensions. Our results indicate that, even among rare diseases with diverse etiologies, patients share numerous commonalties due to their diseases: a lack of effective treatment options, certain physical symptoms, mental health challenges, and financial concerns.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41219705","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}