Patient-Patient Centered Outcomes Research最新文献

筛选
英文 中文
Treatment Preferences in Adult Patients with Type 2 and Type 3 Spinal Muscular Atrophy: Evidence from a Discrete Choice Experiment in China. 成人2型和3型脊髓性肌萎缩症患者的治疗偏好:来自中国离散选择实验的证据
IF 3.1 3区 医学
Patient-Patient Centered Outcomes Research Pub Date : 2026-05-04 DOI: 10.1007/s40271-026-00818-1
Jiaqi Yuan, Shunping Li, Lei Dou, Huanping Xing, Liyong Lu, Shan Jiang
{"title":"Treatment Preferences in Adult Patients with Type 2 and Type 3 Spinal Muscular Atrophy: Evidence from a Discrete Choice Experiment in China.","authors":"Jiaqi Yuan, Shunping Li, Lei Dou, Huanping Xing, Liyong Lu, Shan Jiang","doi":"10.1007/s40271-026-00818-1","DOIUrl":"https://doi.org/10.1007/s40271-026-00818-1","url":null,"abstract":"<p><strong>Introduction: </strong>Spinal muscular atrophy (SMA) is a rare autosomal recessive hereditary neuromuscular disease that leads to progressive muscle weakness and atrophy. Available treatments differ significantly in terms of effectiveness, administration, costs, and side-effect profiles. Currently, evidence regarding patient treatment preferences remains scarce, especially in developing countries. This study aimed to provide evidence on patient preferences toward SMA treatments.</p><p><strong>Methods: </strong>We conducted a discrete choice experiment (DCE) among patients with SMA in China. Attributes were selected on the basis of a systematic literature review, in-depth patient and expert interviews, and validation through a focus group discussion. Five attributes were included: mode of administration, annual out-of-pocket costs, motor function, respiratory function, and incidence of side effects. A mixed logit model was applied for preference estimation. On the basis of the model results, we derived attribute relative importance (RI), conducted scenario analyses to predict the uptake of hypothetical treatment profiles, and performed willingness-to-pay (WTP) analyses to quantify the monetary value of attribute improvements.</p><p><strong>Results: </strong>A total of 202 patients completed the questionnaire, with 190 included in the final analysis. Respondents had a mean age of 29.2 years, were predominantly diagnosed with type 3 SMA (67%), and mainly received parental care (70%). Annual out-of-pocket expenses (RI = 56%) emerged as the most influential attribute, followed by motor function improvements (RI = 20%), incidence of side effects (RI = 13%), respiratory function (RI = 8%), and mode of administration (RI = 3%). Patients expressed a substantial willingness to pay of renminbi (RMB) 32,217 annually for improvements in motor function. Scenario analyses indicated that 95% of patients would prefer a new treatment profile providing enhanced motor function, even at higher costs.</p><p><strong>Conclusions: </strong>Adult patients with type 2 or type 3 SMA in China strongly prefer affordable treatments with oral administration, improved motor and respiratory functions, and minimal side effects. Understanding these preferences can guide clinical decision-making, supporting personalized treatment strategies that may ultimately enhance treatment adherence and clinical outcomes.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2026-05-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147823404","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
Focus Group-Based Cognitive Interviews: A Valid Method in the Development and Evaluation of Clinical Outcome Assessment Measures? 基于焦点小组的认知访谈:临床结果评估方法开发和评估的有效方法?
IF 3.1 3区 医学
Patient-Patient Centered Outcomes Research Pub Date : 2026-05-01 Epub Date: 2026-02-09 DOI: 10.1007/s40271-026-00802-9
Keith Meadows
{"title":"Focus Group-Based Cognitive Interviews: A Valid Method in the Development and Evaluation of Clinical Outcome Assessment Measures?","authors":"Keith Meadows","doi":"10.1007/s40271-026-00802-9","DOIUrl":"10.1007/s40271-026-00802-9","url":null,"abstract":"","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"353-354"},"PeriodicalIF":3.1,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146151228","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-Reported Outcomes in Early-Phase Oncology Clinical Trials: A Stepping Stone to a Patient-Centered Drug Development. 早期肿瘤临床试验中患者报告的结果:以患者为中心的药物开发的垫脚石。
IF 3.1 3区 医学
Patient-Patient Centered Outcomes Research Pub Date : 2026-05-01 Epub Date: 2025-11-11 DOI: 10.1007/s40271-025-00788-w
Frederic Fiteni, Adeline Meilhoc, Olivier Blin, Estelle Haenel
{"title":"Patient-Reported Outcomes in Early-Phase Oncology Clinical Trials: A Stepping Stone to a Patient-Centered Drug Development.","authors":"Frederic Fiteni, Adeline Meilhoc, Olivier Blin, Estelle Haenel","doi":"10.1007/s40271-025-00788-w","DOIUrl":"10.1007/s40271-025-00788-w","url":null,"abstract":"<p><p>In phase I clinical trials, the recommended phase II dose (RP2D) is usually set at or near the maximum tolerated dose (MTD), which is determined based on the observation of dose-limiting toxicities (DLTs). Clinicians typically evaluate toxicities using the National Cancer Institute-Common Terminology Criteria for Adverse Events (NCI-CTCAE), with grade three or higher toxicities classified as DLTs. However, it has been repeatedly demonstrated that physicians tend to underestimate patient's symptoms. Therefore, patient-reported outcomes (PROs), especially the NCI PRO-CTCAE questionnaire, can complement clinician assessments by providing direct patient input on adverse events. This integration could lead to a more accurate definition of DLT and better informed RP2D decisions. Moreover, PROs could optimize sample size strategies in later-stage trials and enable comparison of health-related quality of life (HRQoL) data with synthetic control arms to confirm the benefit of a drug, especially in rare oncogene-driven subsets. Whilst stakeholders and regulatory authorities acknowledge the value of integrating PROs early in drug development, they emphasize the lack of methodological guidelines to support broader adoption. The integration of PROs represents an opportunity to improve the patient-centeredness of phase I trials, ultimately strengthening the drug development process.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"355-359"},"PeriodicalIF":3.1,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145490835","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 in Anticoagulation Treatment: A Review of Discrete Choice Experiments. 患者抗凝治疗偏好:离散选择实验综述。
IF 3.1 3区 医学
Patient-Patient Centered Outcomes Research Pub Date : 2026-05-01 Epub Date: 2025-11-28 DOI: 10.1007/s40271-025-00792-0
Ilja M Brugman, Linda van Eikenhorst, Caroline Schlinkert, Cordula Wagner
{"title":"Patient Preferences in Anticoagulation Treatment: A Review of Discrete Choice Experiments.","authors":"Ilja M Brugman, Linda van Eikenhorst, Caroline Schlinkert, Cordula Wagner","doi":"10.1007/s40271-025-00792-0","DOIUrl":"10.1007/s40271-025-00792-0","url":null,"abstract":"<p><p>Patient engagement is expected to improve the safety and efficacy of anticoagulant medication. By identifying the factors and patient characteristics influencing patients' decisions regarding anticoagulant medication, we aim to support healthcare professionals regarding patient engagement. This review of discrete choice experiments (DCEs) explores the considerations and preferences of patients with various underlying conditions when choosing their anticoagulant medication. Two international databases were searched in March 2024: PubMed and Web of Science Core Collection. The search was updated in July 2025. Eligible studies included original DCE studies that explored the considerations and preferences of patients and covered long-term anticoagulant use beyond the hospital setting (direct oral anticoagulants, vitamin K antagonists, antiplatelet agents, and low molecular weight heparins). The initial search identified a total of 174 records, after which two authors independently assessed the articles for both the title and abstract and the full-text inclusion rounds. After the update, this resulted in the inclusion of 13 articles. The PRISMA 2020 statement was followed and the quality of the included studies was assessed. The results show that overall patients prioritize safety and effectiveness of anticoagulants over convenience factors. If only convenience factors were taken into account, frequency of administration emerges as the most important. Subgroup analysis showed that patient preferences vary based on geographic, demographic, and socioeconomic factors, health status, as well as previous experiences with anticoagulation medication or related health issues. By acknowledging the impact of these diverse factors on patient preferences, healthcare professionals can better support safe and effective anticoagulant care tailored to the needs of individual patients.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"361-384"},"PeriodicalIF":3.1,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145642483","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
Formative Research in the Codesign of a Discrete Choice Experiment with Patients with Myasthenia Gravis: Selecting and Refining Attributes, Levels and Scenarios. 重症肌无力患者离散选择实验共同设计的形成性研究:选择和精炼属性、水平和场景。
IF 3.1 3区 医学
Patient-Patient Centered Outcomes Research Pub Date : 2026-05-01 Epub Date: 2025-12-17 DOI: 10.1007/s40271-025-00796-w
Gozde Aydin, Xin Zhang, Dennis Petrie, Anneke Van der Walt, Stephen Reddel, Katherine Buzzard, Natalie Windle, Susan White, Catherine Bergin, Laura Fanning
{"title":"Formative Research in the Codesign of a Discrete Choice Experiment with Patients with Myasthenia Gravis: Selecting and Refining Attributes, Levels and Scenarios.","authors":"Gozde Aydin, Xin Zhang, Dennis Petrie, Anneke Van der Walt, Stephen Reddel, Katherine Buzzard, Natalie Windle, Susan White, Catherine Bergin, Laura Fanning","doi":"10.1007/s40271-025-00796-w","DOIUrl":"10.1007/s40271-025-00796-w","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Introduction: &lt;/strong&gt;Given the substantial burden faced by patients with myasthenia gravis (MG), including the disease itself, treatment-related side effects, the invasiveness of certain interventions and the time spent in seeking and receiving care, it is essential to understand patient preferences to inform resource allocation and enhance patients' quality of life.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;The aim of this study was to co-design the MG treatment choice tasks with patients for a discrete choice experiment (DCE) using a multi-stage, mixed-methods approach involving the nominal group technique (NGT) and focus groups.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;MG patients in Australia were invited to participate in two of eight 1-hour online sessions in July and August 2024. Each session included four to six patients and was facilitated by three researchers. Participants were presented with three MG scenarios: 'mild exacerbation', 'severe exacerbation' and 'maintenance phase'. During NGT sessions, patients shared their experiences with MG treatments, proposed DCE attributes and ranked their importance for each scenario. Attribute rankings were normalised to a 0-1 scale, averaged within each session and combined using a weighted mean adjusted for group size. The rankings were used to prioritise attributes for inclusion in DCEs. In subsequent focus groups, patients reviewed draft DCE scenario descriptions and provided feedback on the clarity and relevance of attribute descriptions and levels. Throughout the process, research team discussions were held to discuss clinical and methodological relevance of the emerging results.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Nineteen patients with MG aged 35-82 years (12 females, 7 males) participated in four online NGT and four focus group sessions, identifying 31 attributes across treatment administration (n = 10), side effects (n = 16) and treatment outcomes (n = 5). Attribute rankings varied by scenario, with 'recommended by neurologist', health care workers' knowledge of the treatment' and 'easy access to treatment' being consistently top-ranked among treatment administration attributes. Side effects ranged broadly, from gastrointestinal issues to skin cancer. For treatment outcome attributes, 'chance to improve' was most highly ranked for mild and severe exacerbation scenarios, whereas 'risk of getting severely worse' and 'duration of effectiveness' were equally top-ranked for the maintenance scenario. Attribute rankings and patient perspectives were balanced with DCE methodological considerations and clinician input to finalise attributes for each scenario, including grouping similar side effects to describe a few attributes and retaining the 'cost to patient' attribute despite it being a low patient priority to allow trade-off analysis in the resulting choice data. A final list of seven attributes was selected for both the mild exacerbation and maintenance scenarios. Discussions from focus groups and r","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"435-448"},"PeriodicalIF":3.1,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145769988","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 Is Important to Patients? A Mixed Methods Study to Understand Patient Experience Commonalities Across Three Conditions. 对患者来说什么是重要的?一项混合方法研究,了解三种情况下患者体验的共性。
IF 3.1 3区 医学
Patient-Patient Centered Outcomes Research Pub Date : 2026-05-01 Epub Date: 2026-02-06 DOI: 10.1007/s40271-025-00799-7
Kayleigh R Majercak, Eleanor M Perfetto, C Daniel Mullins
{"title":"What Is Important to Patients? A Mixed Methods Study to Understand Patient Experience Commonalities Across Three Conditions.","authors":"Kayleigh R Majercak, Eleanor M Perfetto, C Daniel Mullins","doi":"10.1007/s40271-025-00799-7","DOIUrl":"10.1007/s40271-025-00799-7","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;The objective of this study was to identify if commonalities exist across conditions on the way different conditions and their treatments impact patient health and daily life.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;Qualitative data were collected using in-depth interviews to identify patient experiences with asthma, colorectal cancer, and multiple sclerosis. Patient-organization partners supported recruitment efforts. Virtual, 1-h concept elicitation interviews were conducted with US adults in November to December 2022. Interviews included open-ended questions, using a semi-structured guide adapted from the National Health Council's Map My Experience Toolbox. In addition, in a final interview question, participants rated a list of common impacts derived from review and analysis of existing Voice-of-the-Patient (VoP) reports as \"important/not important.\" Patient data were aggregated to identify cross-cutting impacts meeting a threshold (i.e., 16 of 24 patients reported) or if reported as most bothersome. Resulting impacts were confirmed through review with partner patient organizations. Follow-up interviews were conducted with a subset of participants to identify aspects of the impact that make it important as well as to prioritize and rate the list of impacts.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;A total of 24 adults were interviewed; n = 8 per condition. Common symptoms (% of patients reporting) across conditions included: emotional health/anxiety/depression (95.8%), fatigue (83.3%), pain/discomfort (54.2%), nausea/vomiting (50.0%), gastrointestinal (GI) symptoms (45.8%), cognitive health (45.8%), and sleep disturbance (41.7%). Common function-related impacts (% of patients reporting) included: physical mobility (83.3%), activities of daily living (79.2%), work life (75.0%), emotional burden of disease (66.7%), and lifestyle changes (62.5%). Condition-specific impacts included symptoms such as cough for asthma, chronic sensory/sensitivity dysfunction symptoms for colorectal cancer, and neuromuscular symptoms for multiple sclerosis. For the final interview question, most patients rated the VoP-generated impact list as \"important,\" which sometimes conflicted with the concept elicitation interview findings. For example, pain/discomfort was reported by 54.2% of patients from the concept elicitation interviews versus 73.9% of patients rating the VoP list. Additional probing and prioritization of cross-cutting impacts were based on follow-up interviews, (n = 12) resulting in a prioritized list of \"direct\" impacts (n = 8) and \"probing\" impacts (n = 18). Patients reported intensity and impact on function as the most important aspects or attributes of the symptom-related impacts, and difficulty and missed days/ability to continue working were reported as the most important aspects for the function-related impacts.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;Across three medical conditions, a preliminary set of cross-cutting \"most important\" i","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"465-480"},"PeriodicalIF":3.1,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13124791/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146133670","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
Feasibility of Routine Quality-of-Life Measurement in Residential Aged Care: Results from a Pilot Study in Australia. 日常生活质量测量在住宅老年护理的可行性:来自澳大利亚试点研究的结果。
IF 3.1 3区 医学
Patient-Patient Centered Outcomes Research Pub Date : 2026-05-01 Epub Date: 2025-11-07 DOI: 10.1007/s40271-025-00787-x
Lidia Engel, Nancy Devlin, Briony Dow, Andrew Gilbert, Brendan Mulhern, Tessa Peasgood, Rosalie Viney, Frances Batchelor
{"title":"Feasibility of Routine Quality-of-Life Measurement in Residential Aged Care: Results from a Pilot Study in Australia.","authors":"Lidia Engel, Nancy Devlin, Briony Dow, Andrew Gilbert, Brendan Mulhern, Tessa Peasgood, Rosalie Viney, Frances Batchelor","doi":"10.1007/s40271-025-00787-x","DOIUrl":"10.1007/s40271-025-00787-x","url":null,"abstract":"<p><strong>Objective: </strong>This study presents findings from a pilot study that aimed to examine the feasibility of routine measurement of quality of life in residential aged care, including the examination of barriers to and facilitators of collecting and using that data to improve quality of care.</p><p><strong>Methods: </strong>This study was conducted at two not-for-profit residential aged care facilities in Melbourne, VIC, Australia. All residents were eligible to participate if consent was provided. Self-reported quality-of-life data were collected from residents, alongside proxy-reported data from aged care staff and relatives, primarily using the EQ-5D-5L in addition to a randomly assigned second measure (i.e. The Adult Social Care Outcomes Toolkit [ASCOT], Quality of Life-Aged Care Consumers [QOL-ACC], EQ Health and Wellbeing Instrument [EQ-HWB]). Feasibility was assessed in terms of missing data, residents' level of engagement and understanding, and difficulty experienced by staff and relatives in providing proxy reports. Perceived facilitators and barriers were identified via qualitative interviewers with staff who collected the data.</p><p><strong>Results: </strong>From 103 consenting participants, we gathered quality-of-life data through self-report (n = 90), staff proxy-report (n = 101) and family proxy-report (n = 49). Most residents (94%) were able to respond to the EQ-5D-5L questions and residents' level of engagement was rated by staff as good. Only a few missing values (0-10%) were recorded for the EQ-5D-5L. Qualitative findings indicate that while quality-of-life data collection has benefits, barriers include time pressures, residents being too unwell to self-report, staff uncertainty about responding on their behalf and issues with the measure itself.</p><p><strong>Conclusions: </strong>While it is feasible to routinely collect quality-of-life data in residential aged care, addressing the barriers identified will optimise the efficiency of the process and maximise the use of data to guide quality improvement strategies.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"407-421"},"PeriodicalIF":3.1,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13124758/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145472516","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
Beyond Numbers: Uses and Value of In-Trial Interview Data for Regulatory and Health Technology Assessment (HTA) Decision Making. 超越数字:试验中访谈数据在监管和卫生技术评估(HTA)决策中的用途和价值。
IF 3.1 3区 医学
Patient-Patient Centered Outcomes Research Pub Date : 2026-05-01 Epub Date: 2026-02-02 DOI: 10.1007/s40271-025-00795-x
Carla Dias-Barbosa, Miriam Kimel, Paulina Rolska-Wojcik, Karen Bailey, Olabimpe R Eseyin, Mona L Martin
{"title":"Beyond Numbers: Uses and Value of In-Trial Interview Data for Regulatory and Health Technology Assessment (HTA) Decision Making.","authors":"Carla Dias-Barbosa, Miriam Kimel, Paulina Rolska-Wojcik, Karen Bailey, Olabimpe R Eseyin, Mona L Martin","doi":"10.1007/s40271-025-00795-x","DOIUrl":"10.1007/s40271-025-00795-x","url":null,"abstract":"<p><p>This research explores the growing importance of qualitative in-trial research (ITR) in regulatory and health technology assessment (HTA) decision making. Since 2020, the Food and Drug Administration (FDA) and European Medicines Agency (EMA) have emphasized incorporating patient experience data into drug development and approval. We reviewed regulatory guidance documents, drug approval records, and HTA reports from January 2017 to March 2025. This included labels and reviews for new drug applications from the FDA and EMA, the Patient-Focused Drug Development guidance series, guidance on rare diseases, oncology, and gene therapy, and methodological guidance from HTA bodies in Scotland, the UK, France, Germany, Italy, and Spain. From more than 1000 drug applications assessed by both regulatory agencies, only ten and eight products (from the FDA and EMA, respectively) included ITR, with 55% of these for rare diseases. Both agencies used ITR data to gain insights into symptoms and patient experiences, the relevance of patient-reported outcome concepts, and meaningful changes in symptoms or treatment benefits; and to support the interpretation of meaningful score or endpoint changes. Two products included ITR data in both FDA and EMA reviews/labels. Three HTA bodies published guidance documents on qualitative research, with only two products out of eight reviewed including qualitative data in HTA reports.Despite increasing use, ITR in regulatory submissions and HTA reviews remains limited. Early planning and alignment of ITR objectives with regulatory and HTA requirements are needed to enhance the relevance and impact of qualitative evidence in drug development and healthcare decision making.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"385-405"},"PeriodicalIF":3.1,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13124756/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146108442","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
Patient Preferences for Long-Term Prophylactic Treatment in Hereditary Angioedema: A Discrete-Choice Experiment. 遗传性血管性水肿患者对长期预防性治疗的偏好:一个离散选择实验。
IF 3.1 3区 医学
Patient-Patient Centered Outcomes Research Pub Date : 2026-05-01 Epub Date: 2026-01-21 DOI: 10.1007/s40271-025-00798-8
Kathleen F Villa, Carol Mansfield, Aaron Yarlas, James A Ruggles, Laura Bordone, Cannon Kent, Caroline M Vass
{"title":"Patient Preferences for Long-Term Prophylactic Treatment in Hereditary Angioedema: A Discrete-Choice Experiment.","authors":"Kathleen F Villa, Carol Mansfield, Aaron Yarlas, James A Ruggles, Laura Bordone, Cannon Kent, Caroline M Vass","doi":"10.1007/s40271-025-00798-8","DOIUrl":"10.1007/s40271-025-00798-8","url":null,"abstract":"<p><strong>Background: </strong>Although there are many long-term prophylactic treatments available for hereditary angioedema, few studies have assessed patient preferences for these treatments.</p><p><strong>Objective: </strong>We aimed to assess patient preferences for long-term prophylactic treatment attributes, including treatment trade-offs and the likelihood of starting or switching to a new treatment.</p><p><strong>Methods: </strong>An online discrete-choice experiment survey instrument was developed and administered to adults in the USA with a self-reported diagnosis of hereditary angioedema. Respondents evaluated a series of choices between pairs of hypothetical long-term prophylactic hereditary angioedema treatment alternatives.</p><p><strong>Results: </strong>A total of 250 respondents (81.6% female; mean [standard deviation] age, 39 [11] years) completed the survey. Respondents placed the most importance on a reduction in attack frequency (conditional relative attribute importance = 31.7%), a reduced risk of a gastrointestinal side effect (conditional relative attribute importance = 18.5%), and treatments taken as an oral tablet compared with injections (conditional relative attribute importance = 18.1%). Respondents were more willing to accept increases in injection-site reactions compared with their willingness to accept gastrointestinal side effects in these trade-offs. A total of 197 respondents (78.8%) stated they were open to starting a new medication with their preferred mode of administration.</p><p><strong>Conclusions: </strong>A reduction in attack frequency is the most important treatment feature for adults living with hereditary angioedema. The heterogeneity in patient perspectives highlights the need for patient-physician communication when making decisions about initiating a new long-term prophylactic treatment for hereditary angioedema.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"481-495"},"PeriodicalIF":3.1,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13124884/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146020406","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
Women's Preferences in Early Pregnancy Loss Management: A Focus on Psychological Considerations Using the Analytic Hierarchy Process. 女性在早期妊娠损失管理中的偏好:使用层次分析法关注心理因素。
IF 3.1 3区 医学
Patient-Patient Centered Outcomes Research Pub Date : 2026-05-01 Epub Date: 2025-11-28 DOI: 10.1007/s40271-025-00793-z
Mirjam E C Peters, Andrea Icks, Charalabos-Markos Dintsios
{"title":"Women's Preferences in Early Pregnancy Loss Management: A Focus on Psychological Considerations Using the Analytic Hierarchy Process.","authors":"Mirjam E C Peters, Andrea Icks, Charalabos-Markos Dintsios","doi":"10.1007/s40271-025-00793-z","DOIUrl":"10.1007/s40271-025-00793-z","url":null,"abstract":"<p><strong>Background: </strong>Early pregnancy loss (EPL) is associated with significant emotional burden. While multiple management options exist, psychological aspects are often underrepresented in decision-making support. The objective of this study is to identify criteria that are important to women when choosing between expectant, medical, and surgical management of EPL, with particular attention to psychological aspects.</p><p><strong>Methods: </strong>A mixed-methods study using the Analytic Hierarchy Process (AHP) was conducted. Criteria were identified via literature review and a focus group. Thirty-seven women with EPL history completed structured telephone interviews with pairwise comparisons. Quantitative data were analyzed to calculate criterion weights and assess consistency. Logistic regression explored factors associated with inconsistent responses.</p><p><strong>Results: </strong>Among 37 participants, 'Minimizing psychological distress' emerged as the most important criterion (local weight = 0.381), with sub-criteria such as 'Experiencing miscarriage as a natural process' (0.384; 0.252) and 'Avoiding hospital treatment' (0.355; 0.425) ranked most important for women preferring expectant and medical management, respectively. Women preferring surgical management prioritized 'Short time to miscarriage completion' (0.389). Sixty-five percent of participants demonstrated acceptable preference consistency (consistency ratio ≤ 0.2). Lower education showed the greatest and statistically significant negative influence on the consistency of the preferences in the regression models.</p><p><strong>Conclusion: </strong>Preferences varied by treatment choice, indicating that psychological distress is defined differently across women. The AHP method enabled nuanced insights into individual decision-making. Emotional factors may influence preference patterns and should be considered in clinical counselling. These findings support the need for individualized counselling and shared decision-making. The results may inform the development of clinical tools and guidelines.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"423-434"},"PeriodicalIF":3.1,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13124774/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145642401","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
0
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
相关产品
×
本文献相关产品
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:604180095
Book学术官方微信
小红书