Patient-Patient Centered Outcomes Research最新文献

{"title":"Reporting of Patient and Public Involvement in Technology Appraisal and Assessment Reports: A Rapid Scoping Review.","authors":"Eugenie Evelynne Johnson, Cyril Onwuelazu Uteh, Emma Belilios, Fiona Pearson","doi":"10.1007/s40271-024-00721-7","DOIUrl":"10.1007/s40271-024-00721-7","url":null,"abstract":"<p><strong>Background: </strong>The National Institute for Health and Care Excellence (NICE) produces guidance on the use of health technologies (including new and existing medicines, medical devices, diagnostics and interventional procedures) in the National Health Service. Technology Appraisals inform recommendations on the use of new and existing health technologies. As part of its health technology evaluation process, NICE ask independent research groups known as Evidence or External Assessment Groups (EAGs) to assess or evaluate the available evidence surrounding health technologies. Although patients and the public are involved in the wider NICE Heath Technology Evaluation and Assessment process, little is known about the extent to which patient and public involvement and engagement (PPIE) is undertaken and documented in EAG Reports.</p><p><strong>Objectives: </strong>This rapid scoping review aimed to discover the extent to which PPIE is currently undertaken and documented in EAG Reports, which feed into the wider NICE health technology assessment process, and whether EAG Reports contain a plain language summary.</p><p><strong>Methods: </strong>We searched the NICE website for guidance published between 27 September, 2022 and 27 September, 2023. All records were downloaded directly from the NICE website into an Excel spreadsheet for extraction. Evaluations that were terminated before guidance was published or where an EAG Report was not available as supporting evidence were excluded. One researcher charted information regarding the type of each EAG Report, whether a plain language summary was included, and whether documentation of PPIE was included in the EAG Report either within a stand-alone section or throughout the main text of the report. A second researcher checked charted information for 20% of these records. We tabulated data and described PPIE conduct and documentation in included EAG Reports within a narrative synthesis.</p><p><strong>Results: </strong>A total of 97 EAG Reports were included in this rapid scoping review, the majority of which were documenting Single Technology Appraisals (N = 55). Of the 97 EAG Reports, 11 included a plain language summary. Of these 11 reports, two were Multiple Technology Appraisals, five were Diagnostic Assessment Reviews and four were Early Value Assessments. One Early Value Assessment, one Diagnostic Assessment Review and one Multiple Technology Appraisal reported that they did not conduct PPIE because of time constraints and noted that patients were involved in the wider NICE Appraisal process. Two Early Value Assessments that explicitly reported on PPIE used heterogenous methods of involvement.</p><p><strong>Conclusions: </strong>There is currently limited PPIE documented in EAG Reports and inclusion of a plain language summary is uncommon. Further guidance is required to assist EAGs with embedding PPIE and a plain language summary into their Reports taking into consideration the ultra-rapi","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"109-114"},"PeriodicalIF":3.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142565220","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Assessing Preferences of Patients with Chronic Spontaneous Urticaria for Injectable Treatment Profiles.","authors":"Ana Maria Giménez-Arnau, Maria-Magdalena Balp, Andrii Danyliv, Tonya Winders, James O'Donoghue, Jörn Kleebach, Samantha Morrison, Shaun Walsh, Maike Mueller, Daniela Lopez-Ortiz, Marcus Maurer, Jonathan A Bernstein","doi":"10.1007/s40271-024-00725-3","DOIUrl":"10.1007/s40271-024-00725-3","url":null,"abstract":"<p><strong>Background: </strong>In the context of injectable biologic products approved or in development for chronic spontaneous urticaria (CSU), it is important to capture which treatment attributes matter most to patient and what trade-offs patients are willing to make.</p><p><strong>Objectives: </strong>The CHOICE-CSU study aimed to quantify patient preferences toward injectable treatment attributes among patients with CSU, inadequately controlled by H1-antihistamines.</p><p><strong>Methods: </strong>This was a two-phase cross-sectional patient preference study in adult patients with a diagnosis of CSU, inadequately controlled by H1-antihistamines. A qualitative phase collected patients' insights and relevant treatment attributes that mattered to them, and the outputs were used for the quantitative phase to create the actual injectable treatment profiles with attributes and levels such as: efficacy, safety, and mode of administration. The quantitative phase used discrete choice experiment (DCE) methodology. Eligible patients were asked to make hypothetical choices between 12 treatment profile pairs, created by Sawtooth Software^TM. The DCE data were analyzed using hierarchical Bayesian logistic regression models, enabling the quantification of the relative importance of each attribute/level during the decision-making process.</p><p><strong>Results: </strong>A total of 450 respondents participated in the DCE. The key attributes driving respondent preference amongst injectable treatment options were type of administration device (relative importance 18.5%), complete control of urticaria (relative importance 17.4%), and resolution of angioedema (relative importance 16.4%). Keeping all other attributes and levels equal, the predicted choice share was higher for a profile with an auto-injector versus one with a pre-filled syringe (72.9% versus 27.1%).</p><p><strong>Conclusions: </strong>The CHOICE-CSU study is the first study to provide a quantitative assessment of preferences that patients with CSU, inadequately controlled by H1-antihistamines, have for injectable treatment attributes. Symptom-free periods are the most important overriding therapy goal for patients, and patients will accept some inconveniences, such as administration mode, to achieve this. Additionally, when efficacy is equivalent, administration ease of injectable therapies is valued by patients. As new CSU oral treatment options emerge, additional testing of patient preference toward oral treatments will be required.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"173-185"},"PeriodicalIF":3.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11832561/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143054061","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Preferences of Cancer Survivors for Follow-Up Care: A Systematic Review of Discrete Choice Experiments.","authors":"Tongyu Zhang, Zhuobing Bai, Bingyan Zhao, Yu Chen, Chunmei Zhang","doi":"10.1007/s40271-024-00722-6","DOIUrl":"10.1007/s40271-024-00722-6","url":null,"abstract":"<p><strong>Background and objective: </strong>Cancer survivors frequently encounter multiple challenges, including physical, psychological, social, emotional, and financial difficulties. These challenges significantly impact their quality of life and recovery process. This systematic review intends to delineate and assess the evidence gathered from discrete choice experiments, aiming to unravel the preferences of cancer survivors towards their follow-up care.</p><p><strong>Methods: </strong>A systematic literature review was conducted across PubMed, Web of Science, Embase, CINAHL, PsycINFO, CNKI, Wanfang, and SinoMed databases up to July 2024. Two reviewers independently conducted the screening, data extraction, and quality appraisal of the studies. The collected data were synthesized through a narrative approach. Additionally, semi-quantitative analyses were used to evaluate the frequency, importance, and significance of each attribute.</p><p><strong>Results: </strong>Eight studies conducted across four countries were analyzed, yielding 41 distinct attributes grouped into eight main categories and 14 subcategories, further refined by subject matter. Despite the differences in payment systems between countries, the type of provider topped the list of preferences for cancer survivors' follow-up care. Next in preference was the contact modality. Service quality (comprehensive plans, personalized service, detailed information) was also valued. However, opinions diverged on continuity, additional support, and service frequency.</p><p><strong>Conclusions: </strong>The attributes of follow-up care preferences integrated in this review demonstrate a diverse range among cancer survivors. Generally, they prioritize skilled professionals, flexible contact modality, and other high-quality follow-up care elements. Future studies should analyze cancer survivors' preferences for follow-up care from the perspectives of different stakeholders, and further consider internal and external factors to ensure authentic decision making.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"115-129"},"PeriodicalIF":3.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142559314","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":"Patients and Spine Surgeons' Perspectives regarding Decision-Making and Outcomes in Lumbar Surgery: An Exploratory, Qualitative Study.","authors":"Ling Jie Cheng, Nan Luo, Le Ann Chen, Jing Ying Cheng, Gabriel Ka Po Liu, Wenru Wang, Hwee Weng Dennis Hey, Vivien Xi Wu","doi":"10.1007/s40271-024-00719-1","DOIUrl":"10.1007/s40271-024-00719-1","url":null,"abstract":"<p><strong>Background: </strong>Decision-making in lumbar surgery for degenerative conditions is influenced by various factors, including patient expectations and empowerment. The role of patient-reported outcomes (PROs) in guiding these decisions is underexplored. This study aims to understand the perspectives of patients and spine surgeons in decision-making for lumbar surgery and explore their perspectives on the relevance and influence of pre- and post-surgery PROs.</p><p><strong>Methods: </strong>An exploratory qualitative study was conducted between February 2022 and November 2023. A total of 15 patients with degenerative lumbar conditions and 9 spine surgeons from 5 tertiary public hospitals in Singapore were recruited. Data were analyzed using a framework analysis approach, ensuring validity through member checking, reflexive journals, and data source triangulation.</p><p><strong>Results: </strong>Three overarching categories emerged: \"expectations and outcomes,\" \"decision empowerment,\" and \"surgical experiences.\" There is a shift toward shared decision-making, highlighting the importance of patient-centric approaches. Surgical decisions are primarily influenced by PROs, particularly health-related quality of life (HRQoL) and pain relief. Patients prioritize pain relief and improvements in daily functioning, while surgeons also emphasize avoiding postoperative complications. Decision support tools, including PRO data, are essential but need better accessibility and integration within clinical settings. Positive surgical experiences are driven by clear communication, trust with surgeons, swift recovery, and no regrets post-surgery.</p><p><strong>Conclusions: </strong>Our study emphasizes the importance of patient-centered approaches in lumbar surgery decision-making, particularly regarding pre- and post-surgery PROs. Implementing these approaches may enhance patient satisfaction and surgical outcomes, urging the surgical community to prioritize informed, empathetic decision-making to improve healthcare quality.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"145-160"},"PeriodicalIF":3.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142717718","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":"What Do People Want from an AI-Assisted Screening App for Sexually Transmitted Infection-Related Anogenital Lesions: A Discrete Choice Experiment.","authors":"Nyi Nyi Soe, Phyu Mon Latt, Alicia King, David Lee, Tiffany R Phillips, Christopher K Fairley, Lei Zhang, Jason J Ong","doi":"10.1007/s40271-024-00720-8","DOIUrl":"10.1007/s40271-024-00720-8","url":null,"abstract":"<p><strong>Background: </strong>One of the World Health Organization (WHO) recommendations to achieve its global targets for sexually transmitted infections (STIs) is the increased use of digital technologies. Melbourne Sexual Health Centre (MSHC) has developed an AI-assisted screening application (app) called AiSTi for the detection of common STI-related anogenital skin conditions. This study aims to understand the community's preference for using the AiSTi app.</p><p><strong>Methods: </strong>We used a discrete choice experiment (DCE) to understand community preferences regarding the attributes of the AiSTi app for checking anogenital skin lesions. The DCE design included the attributes: data type; AI accuracy; verification of result by clinician; details of result; speed; professional support; and cost. The anonymous DCE survey was distributed to clients attending MSHC and through social media channels in Australia between January and March 2024. Participant preferences on various app attributes were examined using random parameters logit (RPL) and latent class analysis (LCA) models.</p><p><strong>Results: </strong>The median age of 411 participants was 32 years (interquartile range 26-40 years), with 64% assigned male at birth. Of the participants, 177 (43.1%) identified as same-sex attracted and 137 (33.3%) as heterosexual. In the RPL model, the most influential attribute was the cost of using the app (24.1%), followed by the clinician's verification of results (20.4%), the AI accuracy (19.5%) and the speed of receiving the result (19.1%). The LCA identified two distinct groups: 'all-rounders' (88%), who considered every attribute as important, and a 'cost-focussed' group (12%), who mainly focussed on the price. On the basis of the currently available app attributes, the predicted uptake was 72%. In the short term, a more feasible scenario of improving AI accuracy to 80-89% with clinician verification at a $5 cost could increase uptake to 90%. A long-term optimistic scenario with AI accuracy over 95%, no clinician verification and no cost could increase it to 95%.</p><p><strong>Conclusions: </strong>Preferences for an AI-assisted screening app targeting STI-related anogenital skin lesions are one that is low-cost, clinician-verified, highly accurate and provides results rapidly. An app with these key qualities would substantially improve user uptake.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"131-143"},"PeriodicalIF":3.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11832619/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142562431","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":"Transferability of Preferences; for Better or ….?","authors":"Jorien Veldwijk, Semra Ozdemir, Michael Bui, Juan Marcos Gonzalez, Catharina G M Groothuis-Oudshoorn, Brett Hauber, Tommi Tervonen","doi":"10.1007/s40271-025-00728-8","DOIUrl":"10.1007/s40271-025-00728-8","url":null,"abstract":"","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":" ","pages":"97-100"},"PeriodicalIF":3.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11832702/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143076446","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":"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}

{"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}

{"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}

{"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}