Patient-Patient Centered Outcomes Research最新文献

{"title":"Patients' Preferences for Connected Insulin Pens: A Discrete Choice Experiment Among Patients with Type 1 and Type 2 Diabetes.","authors":"Jaein Seo,&nbsp;Sebastian Heidenreich,&nbsp;Esraa Aldalooj,&nbsp;Jiat Ling Poon,&nbsp;Erik Spaepen,&nbsp;Elizabeth L Eby,&nbsp;Rachel S Newson","doi":"10.1007/s40271-022-00610-x","DOIUrl":"https://doi.org/10.1007/s40271-022-00610-x","url":null,"abstract":"<p><strong>Background: </strong>This study quantified how people with diabetes value the unique features of connected insulin pens and related mobile apps, and the underlying reasons for preferring connected versus non-connected insulin pens.</p><p><strong>Methods: </strong>A discrete choice experiment (DCE) was conducted in the USA and UK to elicit preferences of adults (≥ 18 years) with type 1 or 2 diabetes for attributes of insulin pens. Attributes included device type, dosing support, glucose monitoring, additional app features, and data sharing. Relative attribute importance (RAI) scores were calculated to capture the relative importance of an attribute. Predicted choice probabilities were obtained to compare different profiles for connected and non-connected insulin pens.</p><p><strong>Results: </strong>The DCE was completed by 540 participants (58.9% male; 90.7% Caucasian; mean age, 58.3 years; 69.4% type 2 diabetes). Participants most valued the possibility of using a connected insulin pen with dosing support and automated dose logging (RAI = 39.9%), followed by automatic transfer of glucose levels (RAI = 29.0%), additional features of tracking diet and physical activity (RAI = 14.6%), data sharing (RAI = 13.6%), and device type (RAI = 2.9%). All profiles of connected insulin pens were preferred over a non-connected pen (p < 0.001), and pen profiles with advanced features were preferred over those without (p < 0.001). Preferences differed by age but not diabetes type, country of residence, or insulin regimen.</p><p><strong>Conclusion: </strong>People with diabetes in the USA and UK prefer connected over non-connected insulin pens due largely to the availability of automated logging of dose and glucose levels.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2023-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/58/a9/40271_2022_Article_610.PMC9911509.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9440324","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":"Patient-Reported Outcomes for Fully Vaccinated COVID-19 Patients Over 6 Weeks: The Experiences of Clinical Breakthrough Cases.","authors":"Keri Vartanian,&nbsp;Daniel Fish,&nbsp;Benjamin Gronowski,&nbsp;Natalie Kenton,&nbsp;Ari Robicsek","doi":"10.1007/s40271-022-00605-8","DOIUrl":"https://doi.org/10.1007/s40271-022-00605-8","url":null,"abstract":"<p><strong>Background: </strong>While coronavirus disease 2019 (COVID-19) vaccines have high rates of efficacy, fully vaccinated individuals can become infected with COVID-19. Among this population, symptoms tend to be less severe and shorter lasting. Less is known about how vaccinated individuals who contract COVID-19 experience the disease through patient-reported outcomes (PROs) and how this changes over time.</p><p><strong>Objective: </strong>The aim of this study was to describe the physical, mental, and social health PROs for fully vaccinated individuals who contracted COVID-19 over a 6-week period.</p><p><strong>Design: </strong>Prospective design using the Patient-Reported Outcomes Measurement Information System short-form (PROMIS-10) collected through a mobile application-based platform.</p><p><strong>Participant: </strong>1114 fully vaccinated patients who tested positive for COVID-19 at a large US health system and engaged with the study on or after 1 March 2021 and reported onset of illness prior to 1 November 2021.</p><p><strong>Main measures: </strong>Global physical and mental health PROMIS-10 T-scores for the 6-week period, component PROMIS-10 questions for the 6-week period, and component PROMIS-10 questions restricted to a subset of participants for the first month to measure individual recovery were analyzed.</p><p><strong>Key results: </strong>Mean global physical and mental health T-scores increased over time and remained within one standard deviation of the population mean. At baseline, at least 40% of participants reported good health for all component questions except Fatigue (25%), and the proportion reporting good health increased over time for all questions, with the largest improvements in Fatigue (25.5 to 67.5%), Pain (59.1 to 82.8%), and Emotional Problems (42.3 to 62.5%). Over the first month, the greatest positive changes in individual recovery were observed for Fatigue (65.0%), Pain (53.0%), and Emotional Problems (41.1%); at least 30% of respondents reported no change in at least one category, and the greatest decreases were for Usual Social Activities (23.9%), Social Satisfaction (23.2%), and Mental Health (21.8%).</p><p><strong>Conclusions: </strong>This study provides an important step towards better understanding the impact of 'breakthrough' COVID-19 infections on clinically engaged, fully vaccinated patients' physical and mental health to improve support for their treatment and recovery.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2023-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/63/86/40271_2022_Article_605.PMC9638265.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9081907","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":"Patient Preferences in the Management of Hidradenitis Suppurativa: Results of a Multinational Discrete Choice Experiment in Europe.","authors":"Damon Willems,&nbsp;Eva-Lotta Hinzpeter,&nbsp;Hessel H Van der Zee,&nbsp;Christopher J Sayed,&nbsp;John R Ingram,&nbsp;Charlotte Beaudart,&nbsp;Silvia M A A Evers,&nbsp;Mickael Hiligsmann","doi":"10.1007/s40271-022-00614-7","DOIUrl":"https://doi.org/10.1007/s40271-022-00614-7","url":null,"abstract":"<p><strong>Background and objective: </strong>Hidradenitis suppurativa is a chronic inflammatory skin disease that can lead to a substantial reduction in quality of life. Recent studies revealed high levels of unmet care needs of patients with hidradenitis suppurativa, but their preferences in treatment decision making have scarcely been investigated. This study aimed to reveal which treatment attributes adult patients with HS in Europe consider most important in treatment decision-making.</p><p><strong>Methods: </strong>A discrete choice experiment was conducted with adult patients with hidradenitis suppurativa in Europe to reveal which treatment attributes are most important when making treatment decisions. Participants were presented with 15 sets of two treatment options and asked for each to choose the treatment they preferred. The treatments were characterized by six attributes informed by a prior literature review and qualitative research: effectiveness, pain reduction, duration of treatment benefit, risk of mild adverse event, risk of serious infection, and mode of administration. A random parameter logit model was used to estimate patients' preferences with additional subgroup and latent class models used to explore any differences in preferences across patient groups.</p><p><strong>Results: </strong>Two hundred and nineteen adult patients with hidradenitis suppurativa were included in the analysis (90% women, mean age 38 years). For all six treatment attributes, significant differences were observed between levels. Given the range of levels of each attribute, the most important treatment attributes were effectiveness (47.9%), followed by pain reduction (17.3%), annual risk of a mild adverse event (14.4%), annual risk of a serious infection (10.3%), mode of administration (5.3%), and duration of treatment benefit (4.8%). Higher levels of effectiveness, namely a 75% or 100% reduction in the abscess and inflammatory nodule count, were preferred over levels of effectiveness primarily investigated in randomized clinical trials of hidradenitis suppurativa (a 50% reduction). Results were largely consistent across subgroups and three latent class groups were identified.</p><p><strong>Conclusions: </strong>This study revealed the most important treatment characteristics for patients with hidradenitis suppurativa that can help inform joint patient-physician decision making in the management of hidradenitis suppurativa. Designing future hidradenitis suppurativa treatments according to stated preferences, namely, to offer higher levels of effectiveness and pain improvement without higher risks of adverse events, may increase patients' treatment concordance and lead to improved disease management outcomes.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2023-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/b2/aa/40271_2022_Article_614.PMC9911507.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9143344","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":"Constructing Health State Descriptions for Low-Risk Thyroid Cancer: Stakeholder Engagement and Formative Qualitative Research.","authors":"Erin G Roth,&nbsp;Justin Kim,&nbsp;Julia F Slejko,&nbsp;C Daniel Mullins,&nbsp;Jennifer L Doyle,&nbsp;David L Levitt,&nbsp;Miguel Melendez,&nbsp;Kyle J Fletke,&nbsp;Yinin Hu","doi":"10.1007/s40271-022-00597-5","DOIUrl":"https://doi.org/10.1007/s40271-022-00597-5","url":null,"abstract":"<p><strong>Overview: </strong>This paper describes stakeholder involvement and formative qualitative research in the creation of health state descriptions (HSDs) or vignettes for low-risk thyroid cancer. The aim of this project was to engage stakeholders in the contribution of a novel set of HSDs, an important first step in the process of assessing value in thyroid cancer health states.</p><p><strong>Methods: </strong>We draw upon formative, descriptive qualitative methods, following a multi-stage framework of data collection. We conducted individual semi-structured interviews, cognitive interviews, and focus groups with thyroid cancer patients, community providers, academic subspecialists, and participants with no thyroid cancer diagnosis (N = 31). The HSDs went through several iterations over the course of a year, in collaboration with a highly engaged community advisory board, laying the groundwork for HSDs that are comprehensible, comparable, and appropriate for stated-preference research.</p><p><strong>Findings: </strong>Thyroid cancer survivors compared their experiences with those described in the HSDs. Feedback included concern for the emotional well-being of study participants who would be reading them. Providers were attuned to the need for clinical accuracy and made suggestions to reflect their clinical experience, including for patients with complications or disease progression. The pilot participants with no thyroid cancer were particularly valuable in promoting the need to simplify language and maximize readability.</p><p><strong>Discussion: </strong>Stakeholder engagement was critical to being responsive to feedback as the iterations were refined and presented. Continuous engagement and consultation with multiple sources strengthened the HSDs. A secondary outcome from this project is that stakeholders expressed interest in adapting the HSDs into decision aids for people newly diagnosed with low-risk thyroid cancer.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9381756","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":"Patient Preferences for Multi-Cancer Early Detection (MCED) Screening Tests.","authors":"Heather Gelhorn,&nbsp;Melissa M Ross,&nbsp;Anuraag R Kansal,&nbsp;Eric T Fung,&nbsp;Michael V Seiden,&nbsp;Nicolas Krucien,&nbsp;Karen C Chung","doi":"10.1007/s40271-022-00589-5","DOIUrl":"https://doi.org/10.1007/s40271-022-00589-5","url":null,"abstract":"<p><strong>Background: </strong>Emerging blood-based multi-cancer early detection (MCED) tests can detect a variety of cancer types across stages with a range of sensitivity, specificity, and ability to predict the origin of the cancer signal. However, little is known about the general US population's preferences for MCED tests.</p><p><strong>Objective: </strong>To quantify preferences for MCED tests among US adults aged 50-80 years using a discrete choice experiment (DCE).</p><p><strong>Methods: </strong>To quantify preferences for attributes of blood-based MCED tests, an online DCE was conducted with five attributes (true positives, false negatives, false positives, likelihood of the cancer type unknown, number of cancer types detected), among the US population aged 50-80 years recruited via online panels and social media. Data were analyzed using latent class multinomial logit models and relative attribute importance was obtained.</p><p><strong>Results: </strong>Participants (N = 1700) were 54% female, mean age 63.3 years. Latent class modeling identified three classes with distinct preferences for MCED tests. The rank order of attribute importance based on relative attribute importance varied by latent class, but across all latent classes, participants preferred higher accuracy (fewer false negatives and false positives, more true positives) and screenings that detected more cancer types and had a lower likelihood of cancer type unknown. Overall, 72% of participants preferred to receive an MCED test in addition to currently recommended cancer screenings.</p><p><strong>Conclusions: </strong>While there is significant heterogeneity in cancer screening preferences, the majority of participants preferred MCED screening and the accuracy of these tests is important. While the majority of participants preferred adding an MCED test to complement current cancer screenings, the latent class analyses identified a small (16%) and specific subset of individuals who value attributes differently, with particular concern regarding false-negative and false-positive test results, who are significantly less likely to opt-in.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9381251","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":"Conducting Patient-Oriented Research in Low-Income and Middle-Income Countries: A Scoping Review.","authors":"Bukola Mary Ibitoye,&nbsp;Bernie Garrett,&nbsp;Manon Ranger,&nbsp;Jennifer Stinson","doi":"10.1007/s40271-022-00592-w","DOIUrl":"https://doi.org/10.1007/s40271-022-00592-w","url":null,"abstract":"<p><strong>Background: </strong>Patient-oriented research involves extensive collaboration with patients, their families, caregivers, clinicians and other relevant stakeholders to identify and investigate problems and outcomes relevant to patients. Patient-oriented research can help develop effective patient-centred interventions. Patient-oriented research is an increasingly used approach in high-income countries, but it is unclear how patients are engaged in research in low-income and middle-income countries (LMICs).</p><p><strong>Objectives: </strong>The aim of this scoping review was to explore how patient-oriented research is conducted in LMICs. The objectives were to determine the levels of involvement of patients in the research, how studies have impacted healthcare and patient outcomes in these countries, the reported benefits of patient-oriented research on the research process and the reported challenges of conducting patient-oriented research in LMICs.</p><p><strong>Methods: </strong>A scoping review was conducted using the methodological framework suggested by Arksey and O'Malley and the Joanna Briggs Institute guidelines for conducting scoping reviews. The eligibility criteria were any healthcare research using any research design that involved patients of any age group in the research process. Six databases were searched from their inception till January 4, 2022: MEDLINE, Embase, Cumulated Index to Nursing and Allied Health Literature, PsychInfo, Cochrane Central Register of Controlled Trials and EBM Reviews. The reference lists of relevant articles and Google Scholar were combed as well. Data extraction was performed with a self-developed data extraction guide. The findings were narratively summarised.</p><p><strong>Results: </strong>Thirteen articles were included in this scoping review, representing eight LMICs in Africa and Asia. The majority of studies (38%, n = 5) focused on patients living with human immunodeficiency virus. More than half of the studies (n = 8, 62%) were conducted in the adult population, 31% (n = 4) of the studies involved children and/or adolescents. For most of the studies (92%, n = 12), the participants served as consultants; for one study, the authors identified the participants as co-researchers. Across the studies, information regarding patient-oriented research activities was not consistently reported in the same manner (i.e. different locations in the article), with very limited information in some cases. None of the studies used a patient-oriented research framework and the majority did not report on how patient-oriented research impacted healthcare and patient outcomes. Patient-oriented research was beneficial in identifying relevant patients' needs and improving collaboration among stakeholders, but it also led to extended research timelines and increased financial costs for the researchers and patients.</p><p><strong>Conclusions: </strong>Researchers in LMICs are incorporating patient-oriented res","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10819909","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 Author Instructions Do Health Journals Provide for Writing Plain Language Summaries? A Scoping Review.","authors":"Karen M Gainey,&nbsp;Jenna Smith,&nbsp;Kirsten J McCaffery,&nbsp;Sharon Clifford,&nbsp;Danielle M Muscat","doi":"10.1007/s40271-022-00606-7","DOIUrl":"https://doi.org/10.1007/s40271-022-00606-7","url":null,"abstract":"<p><strong>Background: </strong>Plain language summaries (PLSs) are intended for a non-expert audience in order to make health research accessible and understandable to the public. This is important because most research is written with jargon and at a high reading level. However, there is a high degree of variability in the instructions for writing PLSs, which may impede their usefulness as a tool for communicating health research to the public.</p><p><strong>Objective: </strong>The aim of this scoping review was to conduct a detailed analysis of the author instructions for PLSs provided by leading biomedical and health journals.</p><p><strong>Method: </strong>We screened 534 health journals covering 11 categories selected from the InCites Journal Citation Reports linked to the top 10 non-communicable diseases. We included journals published in English that recommended the inclusion of a PLS (as defined by the National Institute for Health Research) and provided authors with text-based instructions on how it should be written. Two independent reviewers extracted data pertaining to common elements identified in author instructions, such as word count/PLS length, content, structure, purpose, wording to support plain language, and the use of jargon, acronyms and abbreviations. Other aspects of PLSs were recorded, such as the label used (e.g., plain language summary, lay summary, and patient summary), journal publisher, consumer involvement and whether the PLS is optional or mandatory. We recorded the frequency of each element and qualitative details of specific instructions. A consumer representative provided ongoing and iterative feedback on the methods, results, and reporting of this study RESULTS: Despite reviewing 534 journals across 10 non-communicable disease areas and 11 journal categories, we found only 27 (5.1%) contained text-based instructions for PLS. Of the 27 journals included in this review, most (70%) did not require a PLS. Approximately 70% of journals with PLS instructions included advice about the use of jargon, abbreviations, and acronyms. Only one journal recommended the use of a readability tool, however five noted that the reading level of the audience or readability of the PLS should be considered. Author instructions were highly heterogeneous between journals. There was inconsistency regarding the word count/PLS length (e.g., between 100 and 850 words), structure (e.g., paragraphs or bullet points), and varying levels of detail for other elements in the instructions. Although only one journal recommended consumer involvement in the development of PLSs, many recommended authors consult those who are not an expert in their field to review their summary prior to submission.</p><p><strong>Conclusion: </strong>The development of consistent author instructions could enhance the effectiveness and use of PLSs. Such instructions should be developed with consumers to ensure they met the needs of a lay non-expert audience.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/62/cb/40271_2022_Article_606.PMC9813023.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10808077","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":"Student-Athlete Preferences for Sexual Violence Reporting: A Discrete Choice Experiment.","authors":"Alice M Ellyson,&nbsp;Avanti Adhia,&nbsp;Emily Kroshus,&nbsp;Davene R Wright","doi":"10.1007/s40271-022-00600-z","DOIUrl":"https://doi.org/10.1007/s40271-022-00600-z","url":null,"abstract":"<p><strong>Background: </strong>Sexual violence (SV) is prevalent among US college athletes, but formal reports are rare. Little is known about adaptations to institution-level reporting policies and procedures that could facilitate reporting.</p><p><strong>Methods: </strong>We conducted a discrete choice experiment (DCE) survey with 1004 student-athletes at ten Division I NCAA member institutions to examine how attributes of the reporting system influence the decision to formally report SV to their institution. Changes in utility values were estimated using multinomial logistic regression and mixed multinomial logistic regression. Importance scores were compared to understand student-athlete preferences.</p><p><strong>Results: </strong>In order of relative importance, the two attributes most preferred by student-athletes were higher probabilities of students perpetrating SV being found in violation of code of conduct policies (relative importance score = 33), and the availability of substance use amnesty policies (relative importance score = 24). Student-athletes with prior SV experiences were more likely to opt out of formally reporting in the DCE paired choice, had lower estimated utility values for all attributes, and had less between-person heterogeneity. While anonymous reporting and survivor-initiated investigations were preferred by student-athletes on average, there was considerable valuation heterogeneity between student-athletes (sizeable deviations from mean estimated utilities). These two attributes also varied in relative importance; anonymous reporting had higher relative importance after interacting levels with prior SV experiences and competitive status, but lower relative importance after interacting levels with whether a student-athlete played on men's or women's sports teams.</p><p><strong>Conclusions: </strong>Changes to reporting policies and procedures (e.g., transparency about SV reporting outcomes, implementing substance use amnesty policies) may be promising institution-level interventions to increase formal reporting of SV among student-athletes. More research is needed to understand preference heterogeneity between students and generalize these findings to broader student populations.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10817177","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":"Improving Interpretation of Evidence Relating to Quality of Life in Health Technology Assessments of Rare Disease Treatments.","authors":"Elena Nicod,&nbsp;Andrew J Lloyd,&nbsp;Thomas Morel,&nbsp;Michela Meregaglia,&nbsp;Sheela Upadhyaya,&nbsp;Amanda Whittal,&nbsp;Karen Facey,&nbsp;Michael Drummond","doi":"10.1007/s40271-022-00598-4","DOIUrl":"https://doi.org/10.1007/s40271-022-00598-4","url":null,"abstract":"<p><p>Rare diseases are often severe, debilitating, life-limiting conditions, many of which occur in childhood. These complex conditions have a wide range of clinical manifestations that have a substantial impact on the lives of patients, carers and families and often produce heterogeneous clinical outcomes. Therefore, the evaluation of quality-of-life (QoL) impacts is important. In health technology assessment (HTA), patient-reported outcome measures (PROMs) and/or health state utility values (HSUVs) are used to determine QoL impacts of new treatments, but their use in rare diseases is challenging due to small and heterogeneous populations and limited disease knowledge. This paper describes challenges associated with the use of patient-reported outcomes (PROs)/HSUVs to evaluate QoL in HTA of rare disease treatments (RDTs) and identifies five recommendations to ensure appropriate interpretation of QoL impacts. These were derived from mixed methods research (literature reviews, appraisal document analyses, appraisal committee observations and interviews) examining the use of PROs/HSUVs in HTA of RDTs. They highlight that HTAs of RDTs must (1) understand the QoL impacts of the disease and of treatments; (2) critically assess PRO data, recognising the nuances in development and administration of PROMs/HSUVs, considering what is feasible and what matters most to the patient population; (3) recognise that lack of significant effect on a PRO does not imply no QoL benefit; (4) use different forms of evidence to understand QoL impacts, such as patient input; and (5) provide methodological guidance to capture QoL impacts on patients/carers.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10874644","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Preferences for Treatments for Major Depressive Disorder: Formative Qualitative Research Using the Patient Experience.","authors":"Susan dosReis,&nbsp;Laura M Bozzi,&nbsp;Beverly Butler,&nbsp;Richard Z Xie,&nbsp;Richard H Chapman,&nbsp;Jennifer Bright,&nbsp;Erica Malik,&nbsp;Julia F Slejko","doi":"10.1007/s40271-022-00596-6","DOIUrl":"https://doi.org/10.1007/s40271-022-00596-6","url":null,"abstract":"<p><strong>Objectives: </strong>The goals of this formative research are to elicit attributes of treatment and desired outcomes that are important to individuals with major depressive disorder (MDD), to develop a stated preference instrument, and to pre-test the instrument.</p><p><strong>Methods: </strong>A three-phase survey study design elicited the patient's journey with MDD to design and pre-test the discrete choice experiment (DCE) instrument. Participants were 20 adults aged ≥ 18 with MDD who did not also have bipolar disorder or post-partum depression. We engaged patient advocates and a multi-disciplinary stakeholder advisory group to select and refine attributes for inclusion in a DCE instrument. The DCE was incorporated into a survey that also collected depression treatment and management and sociodemographic characteristics. The DCE was pre-tested with ten adults with MDD.</p><p><strong>Results: </strong>Six attributes were included in the DCE: mode of treatment (medicine only, psychotherapy only, all modalities including brain stimulation), time to treatment effect (6, 9, 12 weeks), days of hopefulness (2, 4, 6 days/week), effect on productivity (40%, 60%, 90% increase), relations with others (strained, improved, no impact), and out-of-pocket costs ($30, $60, $90/month). The DCE test led to the refinement of mode of treatment (medicine, medicine and psychotherapy, and all modalities); time to treatment effect (4, 6, 9 weeks); monthly out-of-pocket costs ($30, $90, $270).</p><p><strong>Conclusions: </strong>MDD treatment preferences revealed trade-offs among mode of treatment, time to treatment effect, functional outcomes, and cost. The findings demonstrate the potential for meaningfully incorporating the patient experience in preference measures.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9483243/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10820392","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}