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Perceptions and Acceptance of a Prophylactic Vaccine for Human Immunodeficiency Virus (HIV): A Qualitative Study 对人类免疫缺陷病毒(HIV)预防性疫苗的看法和接受程度:定性研究
The Patient - Patient-Centered Outcomes Research Pub Date : 2024-04-06 DOI: 10.1007/s40271-024-00686-7
{"title":"Perceptions and Acceptance of a Prophylactic Vaccine for Human Immunodeficiency Virus (HIV): A Qualitative Study","authors":"","doi":"10.1007/s40271-024-00686-7","DOIUrl":"https://doi.org/10.1007/s40271-024-00686-7","url":null,"abstract":"<h3>Abstract</h3> <span> <h3>Background</h3> <p>Despite advances in human immunodeficiency virus (HIV) prevention methods, such as the advent of pre-exposure prophylaxis (PrEP), the number of people with newly acquired HIV remains high, particularly in at-risk groups. A prophylactic HIV vaccine could contribute to reduced disease prevalence and future transmission and address limitations of existing options, such as suboptimal long-term adherence to PrEPs.</p> </span> <span> <h3>Methods</h3> <p>This qualitative study aimed to capture perceptions towards and acceptance of prophylactic HIV vaccination in three adult populations in the United States: the general population, ‘at-risk’ individuals (e.g. men who have sex with men, transgender individuals, gender-nonconforming individuals, and individuals in a sexual relationship with a person living with HIV), and parents/caregivers of children aged 9–17 years. Interviews were conducted with 55 participants to explore key drivers and barriers to HIV vaccine uptake, and a conceptual model was developed.</p> </span> <span> <h3>Results</h3> <p>The sample was diverse; participants were 51% female, aged 20–57 years (mean 37 years), 33% with high school diploma as highest education level, and identified as White (42%), Black or African American (35%), of Hispanic, Latino, or Spanish origin (22%), or other races/ethnicities (8%) [groupings are not mutually exclusive]. Perceptions were influenced by individual, interpersonal, community, institutional, and structural factors. Overall, 98% of participants thought vaccination would be beneficial in preventing HIV. Key considerations/barriers included perceived susceptibility, i.e. whether participants felt there was a risk of contracting HIV (discussed by 90%); the clinical profile of the vaccine (e.g. the adverse effect profile [98%], and vaccine efficacy [85%], cost [73%] and administration schedule [88%]); and concerns around potential vaccine-induced seropositivity (VISP; 62%). Stigma was not found to be an important barrier, with a general view that vaccination status was personal. Participants in the ‘at-risk’ group were the most likely to accept an HIV vaccine (70%). Unique concerns in the subgroups included how a potential vaccine’s clinical profile compared with PrEP, voiced by those receiving/considering PrEP, and considerations of children’s views on the topic, voiced by parents/caregivers.</p> </span> <span> <h3>Conclusions</h3> <p>Understanding these factors could help develop HIV vaccine research strategies and contribute toward public health messaging to support future HIV vaccination programs.</p> </span>","PeriodicalId":501651,"journal":{"name":"The Patient - Patient-Centered Outcomes Research","volume":"52 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-04-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140590466","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Diabetes Research Matters: A Three-Round Priority-Setting Survey Consultation with Adults Living with Diabetes and Family Members in Australia 糖尿病研究事关重大:与澳大利亚成年糖尿病患者及其家人进行的三轮优先事项确定调查咨询
The Patient - Patient-Centered Outcomes Research Pub Date : 2024-04-06 DOI: 10.1007/s40271-024-00688-5
{"title":"Diabetes Research Matters: A Three-Round Priority-Setting Survey Consultation with Adults Living with Diabetes and Family Members in Australia","authors":"","doi":"10.1007/s40271-024-00688-5","DOIUrl":"https://doi.org/10.1007/s40271-024-00688-5","url":null,"abstract":"<h3>Abstract</h3> <span> <h3>Objective</h3> <p>We aimed to identify the health and quality-of-life research priorities of Australians with diabetes or family members.</p> </span> <span> <h3>Methods</h3> <p>Through an iterative, three-step, online survey process we (1) qualitatively generated research topics (long list) in response to one question “What research is needed to support people with diabetes to live a better life?”; (2) determined the most important research questions (short list); and (3) ranked research questions in order of importance (priorities). We aimed to recruit <em>N</em> = 800 participants, with approximate equal representation of diabetes type and family members.</p> </span> <span> <h3>Results</h3> <p>Participants (<em>N</em> = 661) were adults (aged 18+ years) in Australia with a self-reporting diagnosis of diabetes (type 1, <em>n</em> = 302; type 2, <em>n</em> = 204; prior/current gestational, <em>n</em> = 58; less common types, <em>n</em> = 22, or a family member, <em>n</em> = 75). Retention rates for Surveys 2 and 3 were 47% (<em>n</em> = 295) and 50% (<em>n</em> = 316), respectively. From 1549 open-text responses, 25 topics and 125 research questions were identified thematically. Research priorities differed by cohort, resulting in specific lists developed and ranked by each cohort. The top-ranked research question for the type 1 diabetes cohort was “How can diabetes technology be improved …?” and for the type 2 diabetes cohort: “How can insulin resistance be reversed …?”. One question was common to the final lists of all cohorts: “What are the causes or triggers of diabetes?” Within cohorts, the top priorities were perceived as being of similar importance.</p> </span> <span> <h3>Conclusions</h3> <p>The research priorities differ substantially by diabetes type and for family members. These findings should inform funding bodies and researchers, to align future research and its communication with community needs.</p> </span>","PeriodicalId":501651,"journal":{"name":"The Patient - Patient-Centered Outcomes Research","volume":"2017 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-04-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140590918","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Community Preferences for the Care of Older People at the End of Life: How Important is the Disease Context? 社区对临终老年人护理的偏好:疾病背景有多重要?
The Patient - Patient-Centered Outcomes Research Pub Date : 2024-03-18 DOI: 10.1007/s40271-024-00675-w
Patricia Kenny, Deborah J. Street, Jane Hall, Meera R. Agar, Jane Phillips
{"title":"Community Preferences for the Care of Older People at the End of Life: How Important is the Disease Context?","authors":"Patricia Kenny, Deborah J. Street, Jane Hall, Meera R. Agar, Jane Phillips","doi":"10.1007/s40271-024-00675-w","DOIUrl":"https://doi.org/10.1007/s40271-024-00675-w","url":null,"abstract":"<h3 data-test=\"abstract-sub-heading\">Background</h3><p>Population preferences for care at the end of life can inform palliative care policy and direction. Research investigating preferences for care at the end of life has focused predominantly on the context of advanced cancer, with relatively little attention to other life-limiting illnesses that are common causes of death.</p><h3 data-test=\"abstract-sub-heading\">Objectives</h3><p>We aimed to investigate preferences for the care of older people at the end of life in three different disease contexts. The purpose was to understand if population preferences for care in the last 3 weeks of life would differ for patients dying from cancer, heart failure or dementia.</p><h3 data-test=\"abstract-sub-heading\">Methods</h3><p>Three discrete choice experiments were conducted in Australia with a general population sample using similar methods but different end-of-life disease contexts. Some attributes were common across the three experiments and others differed to accommodate the specific disease context. Each survey was completed by a different panel sample aged ≥45 years (cancer, <i>n</i> = 1548; dementia, <i>n</i> = 1549; heart failure, <i>n</i> = 1003). Analysis was by separate mixed logit models.</p><h3 data-test=\"abstract-sub-heading\">Results</h3><p>The most important attributes across all three surveys were costs to the patient and family, patient symptoms and informal carer stress. The probability of choosing an alternative was lowest (0.18–0.29) when any one of these attributes was at the least favourable level, holding other attributes constant across alternatives. The cancer survey explored symptoms more specifically and found patient anxiety with a higher relative importance score than the symptom attribute of pain. Dementia was the only context where most respondents preferred to not have a medical intervention to prolong life; the probability of choosing an alternative with a feeding tube was 0.40 (95% confidence interval 0.36–0.43).</p><h3 data-test=\"abstract-sub-heading\">Conclusions</h3><p>This study suggests a need for affordable services that focus on improving patient and carer well-being irrespective of the location of care, and this message is consistent across different disease contexts, including cancer, heart failure and dementia. It also suggests some different considerations in the context of people dying from dementia where medical intervention to prolong life was less desirable.</p>","PeriodicalId":501651,"journal":{"name":"The Patient - Patient-Centered Outcomes Research","volume":"96 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-03-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140146536","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Research Priorities to Increase Confidence in and Acceptance of Health Preference Research: What Questions Should be Prioritized Now? 提高对健康偏好研究的信心和接受度的研究重点:现在应优先考虑哪些问题?
The Patient - Patient-Centered Outcomes Research Pub Date : 2023-12-16 DOI: 10.1007/s40271-023-00650-x
Rachael L. DiSantostefano, Ian P. Smith, Marie Falahee, Aura Cecilia Jiménez-Moreno, Serena Oliveri, Jorien Veldwijk, G. Ardine de Wit, Ellen M. Janssen, Conny Berlin, Catharina G. M. Groothuis-Oudshoorn
{"title":"Research Priorities to Increase Confidence in and Acceptance of Health Preference Research: What Questions Should be Prioritized Now?","authors":"Rachael L. DiSantostefano, Ian P. Smith, Marie Falahee, Aura Cecilia Jiménez-Moreno, Serena Oliveri, Jorien Veldwijk, G. Ardine de Wit, Ellen M. Janssen, Conny Berlin, Catharina G. M. Groothuis-Oudshoorn","doi":"10.1007/s40271-023-00650-x","DOIUrl":"https://doi.org/10.1007/s40271-023-00650-x","url":null,"abstract":"<h3 data-test=\"abstract-sub-heading\">Background and Objective</h3><p>There has been an increase in the study and use of stated-preference methods to inform medicine development decisions. The objective of this study was to identify prioritized topics and questions relating to health preferences based on the perspective of members of the preference research community.</p><h3 data-test=\"abstract-sub-heading\">Methods</h3><p>Preference research stakeholders from industry, academia, consultancy, health technology assessment/regulatory, and patient organizations were recruited using professional networks and preference-targeted e-mail listservs and surveyed about their perspectives on 19 topics and questions for future studies that would increase acceptance of preference methods and their results by decision makers. The online survey consisted of an initial importance prioritization task, a best-worst scaling case 1 instrument, and open-ended questions. Rating counts were used for analysis. The best-worst scaling used a balanced incomplete block design.</p><h3 data-test=\"abstract-sub-heading\">Results</h3><p>One hundred and one participants responded to the survey invitation with 66 completing the best-worst scaling. The most important research topics related to the synthesis of preferences across studies, transferability across populations or related diseases, and method topics including comparison of methods and non-discrete choice experiment methods. Prioritization differences were found between respondents whose primary affiliation was academia versus other stakeholders. Academic researchers prioritized methodological/less studied topics; other stakeholders prioritized applied research topics relating to consistency of practice.</p><h3 data-test=\"abstract-sub-heading\">Conclusions</h3><p>As the field of health preference research grows, there is a need to revisit and communicate previous work on preference selection and study design to ensure that new stakeholders are aware of this work and to update these works where necessary. These findings might encourage discussion and alignment among different stakeholders who might hold different research priorities. Research on the application of previous preference research to new contexts will also help increase the acceptance of health preference information by decision makers.</p>","PeriodicalId":501651,"journal":{"name":"The Patient - Patient-Centered Outcomes Research","volume":"22 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-12-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138681085","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Preferences of Patients with Amyotrophic Lateral Sclerosis for Intrathecal Drug Delivery: Choosing between an Implanted Drug-Delivery Device and Therapeutic Lumbar Puncture 肌萎缩侧索硬化症患者对鞘内给药的偏好:在植入式给药装置和治疗性腰椎穿刺之间做出选择
The Patient - Patient-Centered Outcomes Research Pub Date : 2023-12-14 DOI: 10.1007/s40271-023-00665-4
Jaein Seo, Sandeep Saurkar, Gabriela S. Fernandez, Anup Das, Stephen A. Goutman, Sebastian Heidenreich
{"title":"Preferences of Patients with Amyotrophic Lateral Sclerosis for Intrathecal Drug Delivery: Choosing between an Implanted Drug-Delivery Device and Therapeutic Lumbar Puncture","authors":"Jaein Seo, Sandeep Saurkar, Gabriela S. Fernandez, Anup Das, Stephen A. Goutman, Sebastian Heidenreich","doi":"10.1007/s40271-023-00665-4","DOIUrl":"https://doi.org/10.1007/s40271-023-00665-4","url":null,"abstract":"<h3 data-test=\"abstract-sub-heading\">Background</h3><p>Novel intrathecal treatments for amyotrophic lateral sclerosis (ALS) may require delivery using lumbar puncture (LP). Implanted drug-delivery devices (IDDDs) could be an alternative but little is known about patients’ preferences for intrathecal drug-delivery methods.</p><h3 data-test=\"abstract-sub-heading\">Objective</h3><p>We aimed to elicit preferences of patients with ALS for routine LP and IDDD use.</p><h3 data-test=\"abstract-sub-heading\">Methods</h3><p>A discrete choice experiment (DCE) and a threshold technique (TT) exercise were conducted online among patients with ALS in the US and Europe. In the DCE, patients made trade-offs between administration attributes. Attributes were identified from qualitative interviews. The TT elicited maximum acceptable risks (MARs) of complications from device implantation surgery. DCE data were analyzed using mixed logit to quantify relative attribute importance (RAI) as the maximum contribution of each attribute to a preference, and to estimate MARs of device failure. TT data were analyzed using interval regression. Four scenarios of LP and IDDD were compared.</p><h3 data-test=\"abstract-sub-heading\">Results</h3><p>Participants (<i>N</i> = 295) had a mean age of 57.7 years; most (74.2%) were diagnosed &lt; 3 years ago. Preferences were affected by device failure risk (RAI 28.6%), administration frequency (26.4%), administration risk (19.7%), overall duration (17.8%), and appointment location (7.5%). Patients accepted a 5.6% device failure risk to reduce overall duration from 2 h to 30 min and a 3.6% risk for administration in a local clinic instead of a hospital. The average MAR of complications from implantation surgery was 29%. Patients preferred IDDD over LP in three of four scenarios.</p><h3 data-test=\"abstract-sub-heading\">Conclusion</h3><p>Patients considered an IDDD as a valuable alternative to LP in multiple clinical settings.</p>","PeriodicalId":501651,"journal":{"name":"The Patient - Patient-Centered Outcomes Research","volume":"10 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138681277","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","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 Pharmacy Services: A Systematic Review of Studies Based on Discrete Choice Experiments 患者对药房服务的偏好:基于离散选择实验的研究系统回顾
The Patient - Patient-Centered Outcomes Research Pub Date : 2023-12-13 DOI: 10.1007/s40271-023-00652-9
Margaux Riboulet, Anne-Laure Clairet, Mohamed Bennani, Virginie Nerich
{"title":"Patient Preferences for Pharmacy Services: A Systematic Review of Studies Based on Discrete Choice Experiments","authors":"Margaux Riboulet, Anne-Laure Clairet, Mohamed Bennani, Virginie Nerich","doi":"10.1007/s40271-023-00652-9","DOIUrl":"https://doi.org/10.1007/s40271-023-00652-9","url":null,"abstract":"<h3 data-test=\"abstract-sub-heading\">Background and Objective</h3><p>In recent years, the mission of pharmacists has evolved and the model of the caregiver–patient relationship has been challenged by a new patient-centered approach. A challenge to providing personalized care is the assessment of patient preferences. We aimed to systematically identify published discrete choice experiments related to patient preferences for pharmacy services and to assess the quality of the selected articles.</p><h3 data-test=\"abstract-sub-heading\">Methods</h3><p>A systematic review of the literature was conducted in two databases (PubMed and Embase, until March 2023) according to Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) recommendations. The quality of the selected articles was assessed according to the International Society for Pharmacoeconomics and Outcomes Research conjoint analysis checklist.</p><h3 data-test=\"abstract-sub-heading\">Results</h3><p>Among the 421 articles identified, 16 published studies were included and analyzed. They were mostly published in pharmaceutical (<i>n</i> = 6), economic (<i>n</i> = 4), and public health (<i>n</i> = 4) journals. Only two articles concerned the field of hospital pharmacy. Only five presented the specific pharmacy service studied: filling of prescriptions, management of symptoms, treatment delivery, and components of pharmacist counseling. Five articles focused on pharmacy services related to a specific disease. None fully fulfilled the International Society for Pharmacoeconomics and Outcomes Research checklist, only partially fulfilled.</p><h3 data-test=\"abstract-sub-heading\">Conclusions</h3><p>According to the identified studies, cost, time, logistics (organizational criteria), and pharmacists’ courtesy and skills were consistently cited as factors influencing patient preferences for pharmacy services. The disclosure of patient preferences is a complex and under-researched topic, especially in the field of pharmacy, but interest is growing. As the mission of pharmacists evolves to bring them closer to patients, the better understanding of patient preferences in pharmacy services will allow for better targeting and better integration of patient profiles in patient management.</p>","PeriodicalId":501651,"journal":{"name":"The Patient - Patient-Centered Outcomes Research","volume":"10 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-12-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138627784","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
A Review of the Use of EQ-5D for Clinical Outcome Assessment in Health Technology Assessment, Regulatory Claims, and Published Literature 在健康技术评估、监管要求和已发表文献中使用 EQ-5D 进行临床结果评估的综述
The Patient - Patient-Centered Outcomes Research Pub Date : 2023-12-12 DOI: 10.1007/s40271-023-00662-7
Caroline Shaw, Louise Longworth, Bryan Bennett, Louise McEntee-Richardson, James W. Shaw
{"title":"A Review of the Use of EQ-5D for Clinical Outcome Assessment in Health Technology Assessment, Regulatory Claims, and Published Literature","authors":"Caroline Shaw, Louise Longworth, Bryan Bennett, Louise McEntee-Richardson, James W. Shaw","doi":"10.1007/s40271-023-00662-7","DOIUrl":"https://doi.org/10.1007/s40271-023-00662-7","url":null,"abstract":"<p>The aim is to identify the extent to which EQ-5D is used as a clinical outcome assessment (COA) endpoint in a non-economic context in health technology assessment (HTA) decisions, regulatory labelling claims and published literature. Drug technology appraisals (TAs) published by HTA agencies in England, France, Germany and the USA between 2019 and 2021 were identified. Product labelling for drugs approved by the European Medicines Agency (EMA) and US Food and Drug Administration (FDA) between 2016 and 2021 were also identified. A systematic literature review (SLR) was also performed. Documents reporting EQ-5D in the context of economic evaluation only were excluded. EQ-5D data were reported for COA in 195 of 1072 (18%) published TAs, with the majority reported for Germany (<i>n</i> = 138). The EQ-5D visual analogue scale (EQ-VAS) was reported most frequently, in 68% of all TAs, and accounted for 100% of Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen (IQWiG) and 94% of Gemeinsamer Bundesausschuss (G-BA) TAs. In total, 320 drugs were approved or reviewed by the EMA and 735 by the FDA. Of these, 15 reported EQ-5D data from the EMA and 35 from the FDA; however, all EQ-5D data submitted to the FDA were reported in supporting documentation. Reporting of both EQ-5D index and EQ-VAS was most frequent, occurring in 32% of all documents. For the SLR, 329 of 4248 (8%) retrieved records were included. Reporting of both EQ-5D index and EQ-VAS was most frequent, occurring in 36% of studies. Clinical evaluation of recent drug approvals, based on regulatory, HTA and systematic literature reviews, demonstrated limited use of EQ-5D outside the context of economic evaluations. This may be due to the likelihood that the EQ-5D may lack sensitivity to detect improvement in conditions with small expected therapeutic benefit, or because the EQ-5D is not considered an adequate COA tool for clinical evaluation of treatment benefit. EQ-5D, as a COA, was more likely to be used in clinical evaluation of cancer drugs than drugs for treatment in any other disease category. HTA bodies were more likely to use the EQ-5D for COA, especially in Germany.</p>","PeriodicalId":501651,"journal":{"name":"The Patient - Patient-Centered Outcomes Research","volume":"17 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138573078","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Current Practices and Challenges When Submitting Patient Experience Data for Regulatory Decisions by the US Food and Drug Administration: An Industry Survey 美国食品和药物管理局为监管决策提交患者体验数据时的当前做法和挑战:行业调查
The Patient - Patient-Centered Outcomes Research Pub Date : 2023-12-12 DOI: 10.1007/s40271-023-00653-8
Cathy Anne Pinto, Tommi Tervonen, Cecilia Jimenez-Moreno, Bennett Levitan, Montse Soriano Gabarró, Cynthia Girman, Josephine M. Norquist, Brett Hauber
{"title":"Current Practices and Challenges When Submitting Patient Experience Data for Regulatory Decisions by the US Food and Drug Administration: An Industry Survey","authors":"Cathy Anne Pinto, Tommi Tervonen, Cecilia Jimenez-Moreno, Bennett Levitan, Montse Soriano Gabarró, Cynthia Girman, Josephine M. Norquist, Brett Hauber","doi":"10.1007/s40271-023-00653-8","DOIUrl":"https://doi.org/10.1007/s40271-023-00653-8","url":null,"abstract":"<h3 data-test=\"abstract-sub-heading\">Objectives</h3><p>To understand industry practices and challenges when submitting patient experience data (PED) for regulatory decisions by the US Food and Drug Administration (FDA).</p><h3 data-test=\"abstract-sub-heading\">Methods</h3><p>A two-part online survey related to collection, submission, and use of PED by FDA in regulatory decision-making (part 1) and a best-worst exercise for prioritizing potential PED initiatives (part 2) was completed by industry and contract research organization (CRO) members with ≥ 2 years of recent experience with patient-reported outcome (PRO), natural history study (NHS), or patient preference (PP) data; and direct experience with FDA filings including PED.</p><h3 data-test=\"abstract-sub-heading\">Results</h3><p>A total of 50 eligible respondents (84% industry) completed part 1 of the survey, among which 46 completed part 2. Respondents mostly had PRO (86%) and PP (50%) experience. All indicated that FDA meetings should have a standing agenda item to discuss PED. Most (78%) reported meetings should occur before pivotal trials. A common challenge was justifying inclusion without knowing if and how data will be used. Most agreed that FDA and industry should co-develop the PED table in the FDA clinical review (74%), and the table should report reason(s) for not using PED (96%) in regulatory decision-making. Most important efforts to advance PED use in decision-making were a dedicated meeting pathway and expanded FDA guidance (51% each).</p><h3 data-test=\"abstract-sub-heading\">Conclusions</h3><p>FDA has policy targets expanding PED use, but challenges remain regarding pathways for PED submission and transparency in regulatory decision-making. Alignment on the use of existing meeting opportunities to discuss PED, co-development of the PED table, and expanded guidance are encouraged.</p>","PeriodicalId":501651,"journal":{"name":"The Patient - Patient-Centered Outcomes Research","volume":"13 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138573069","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Exploring the Preferences of Parents of Children with Myopia in Rural China for Eye Care Services Under Privatization Policy: Evidence from a Discrete Choice Experiment 探索私有化政策下中国农村近视儿童家长对眼科医疗服务的偏好:离散选择实验的证据
The Patient - Patient-Centered Outcomes Research Pub Date : 2023-12-10 DOI: 10.1007/s40271-023-00660-9
Wen Lin, Xiaodong Dong, Jack Hennessy, Junling Zhao, Xiaochen Ma
{"title":"Exploring the Preferences of Parents of Children with Myopia in Rural China for Eye Care Services Under Privatization Policy: Evidence from a Discrete Choice Experiment","authors":"Wen Lin, Xiaodong Dong, Jack Hennessy, Junling Zhao, Xiaochen Ma","doi":"10.1007/s40271-023-00660-9","DOIUrl":"https://doi.org/10.1007/s40271-023-00660-9","url":null,"abstract":"<h3 data-test=\"abstract-sub-heading\">Objectives</h3><p>This study aims to measure the preferences and valuations of parents of students with myopia parents for eye care service attributes in rural China, and to quantify the potential welfare impacts of privatization policy on children’s eye care services.</p><h3 data-test=\"abstract-sub-heading\">Methods</h3><p>A discrete choice experiment was designed and implemented among a sample of parents of children with myopia in rural China. We randomly selected 350 participants from the list of subjects obtained from local town schools and family doctors using a random number table method. The participants were asked to choose between two hypothetical scenarios defined by five attributes: provider type, distance, price, lenses type, and refractionists’ professional competencies. We estimate conditional logit and mixed logit models to approximate individual preferences for these attributes and estimate the welfare effects by calculating willingness to pay.</p><h3 data-test=\"abstract-sub-heading\">Results</h3><p>Respondents (<i>n</i> = 336) showed a significant preference for public providers of refractive error services, myopia control lenses, and professional refractionists (<i>P</i> &lt; 0.01 for each). Consumer welfare losses due to a prohibition of the public provision of refractive error services could be compensated by improving the quality of products and services delivered by private providers. Lastly, both parent and child demographics and previous experience of eye care service consumption are important predictors of willingness to pay for refractive error services.</p><h3 data-test=\"abstract-sub-heading\">Conclusions</h3><p>The privatization policy on children’s eye care services would not cater to the preferences of rural consumers, inevitably leading to welfare losses<i>.</i> However, reduced consumer welfare could be compensated by improving the quality of products and service delivery from private providers. These results could help inform strategies to improve and reduce inequities in access to high-quality eye care services in rural China.</p>","PeriodicalId":501651,"journal":{"name":"The Patient - Patient-Centered Outcomes Research","volume":"21 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-12-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138567953","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
A Best–Worst Scaling Study of the General Population's Preferences for Activities in Living Arrangements for Persons With Dementia 关于普通人群对痴呆症患者生活安排活动偏好的最佳-最差比例研究
The Patient - Patient-Centered Outcomes Research Pub Date : 2023-12-08 DOI: 10.1007/s40271-023-00661-8
Christian Speckemeier, Carina Abels, Klemens Höfer, Anke Walendzik, Jürgen Wasem, Silke Neusser
{"title":"A Best–Worst Scaling Study of the General Population's Preferences for Activities in Living Arrangements for Persons With Dementia","authors":"Christian Speckemeier, Carina Abels, Klemens Höfer, Anke Walendzik, Jürgen Wasem, Silke Neusser","doi":"10.1007/s40271-023-00661-8","DOIUrl":"https://doi.org/10.1007/s40271-023-00661-8","url":null,"abstract":"<h3 data-test=\"abstract-sub-heading\">Background</h3><p>Activity involvement is a central element of dementia care. However, few studies have examined preferences for types of activities for persons who may be in need of care in the future. In this study, a best–worst scaling (BWS) was conducted to gather insights on preferred activities in small-scale living arrangements for dementia in the general population aged 50–65 years from rural and urban regions.</p><h3 data-test=\"abstract-sub-heading\">Methods</h3><p>BWS tasks were developed based on literature searches and focus groups. The final BWS contains ten activities, namely ‘interaction with animals’, ‘gardening’, ‘painting, handicrafts, manual activities’, ‘household activities (e.g., folding laundry, cooking)’, ‘watching television’, ‘practicing religion’, ‘listening to music and singing familiar songs’, ‘conversations about the past’, ‘walks and excursions’, ‘sport activities (e.g., gymnastics)’. Each participant had to fill out subsets of four objects each and identify them as best and worst. A postal survey was sent to a total of 4390 persons from rural and urban regions aged between 50 and 65 years. Results were analyzed by count analysis and logit models. It was examined if preferences differ with respect to gender, religiousness, and informal caregiving experience.</p><h3 data-test=\"abstract-sub-heading\">Results</h3><p>A total of 840 questionnaires were returned, and 627 surveys were included in the analysis. In the rural sample, the highest relative importance (RI) was assigned to ‘walks and excursions’ (RI: 100%), ‘sport activities (e.g., gymnastics)’ (RI: 56%), ‘gardening’ (RI: 28%), and ‘interaction with animals’ (26%). In the urban sample, ‘walks and excursions’ (RI: 100%), ‘sport activities (e.g., gymnastics)’ (RI: 37%), ‘interaction with animals’ (25%) and ‘gardening’ (RI: 22%) were perceived as most important. In both groups, household activities and practicing religion were of lowest importance. Importance ranks differed between subgroups. Results of the logit models with random effects showed the same order as results from the count analyses.</p><h3 data-test=\"abstract-sub-heading\">Discussion</h3><p>Our findings show that respondents generally favored activities with an outdoor component, while the household activities that are part of many concepts were of low importance to respondents. Thus, our study can inform the design of a preference-based specific range of activities in small-scale living arrangements for dementia.</p>","PeriodicalId":501651,"journal":{"name":"The Patient - Patient-Centered Outcomes Research","volume":"76 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-12-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138564200","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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