Patient-Patient Centered Outcomes Research最新文献

{"title":"The Pogo-ization of Post-Pandemic Vaccine Policy.","authors":"Peter J Pitts","doi":"10.1007/s40271-023-00626-x","DOIUrl":"https://doi.org/10.1007/s40271-023-00626-x","url":null,"abstract":"","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2023-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10092907/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9445079","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":"Correction to: How Does the Public Evaluate Vaccines for Low‑Incidence, Severe‑Outcome Diseases? A General‑Population Choice Experiment.","authors":"F Reed Johnson, Angelyn Fairchild, Dale Whittington, Amit K Srivastava, Juan Marcos Gonzalez, Liping Huang","doi":"10.1007/s40271-023-00620-3","DOIUrl":"https://doi.org/10.1007/s40271-023-00620-3","url":null,"abstract":"","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2023-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10320800/pdf/40271_2023_Article_620.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10116511","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":"So You Want to Build Your Disease's First Online Patient Registry: An Educational Guide for Patient Organizations Based on US and European Experience.","authors":"Paul Wicks,&nbsp;Lindsey Wahlstrom-Edwards,&nbsp;Sam Fillingham,&nbsp;Andrea Downing,&nbsp;Elin Haf Davies","doi":"10.1007/s40271-023-00619-w","DOIUrl":"https://doi.org/10.1007/s40271-023-00619-w","url":null,"abstract":"<p><p>Patient registries fulfill a number of key roles for clinicians, researchers, non-profit organizations, payers, and policy makers. They can help the field understand the natural history of a condition, determine the effectiveness of interventions, measure safety, and audit the quality of care provided. Successful registries in cystic fibrosis, Duchenne's muscular dystrophy, and other rare diseases have become a model for accelerating progress. However, the complex tasks required to develop a modern registry can seem overwhelming, particularly for those who are not from a technical background. In this Education article, a team of co-authors from across patient advocacy, technology, privacy, and commercial perspectives who have worked on a number of such projects offer a \"Registry 101\" primer to help get started. We will outline the promise and potential of patient registries with worked case examples, identify some of the key technical considerations you will need to consider, describe the type of data you might want to collect, consider privacy risks to protect your users, sketch out some of the paths towards long-term financial sustainability we have observed, and conclude with plans to mitigate some of the challenges that can occur and signpost interested readers to further resources. While rapid growth in the digital health market has presented numerous opportunities to those at the beginning of their journey, it is important to start with the long-term goals in mind and to benefit from the learnings of those who have walked this path before.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2023-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/90/80/40271_2023_Article_619.PMC10031688.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9408914","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":"Systematic Review of the Effect of a One-Day Versus Seven-Day Recall Duration on Patient Reported Outcome Measures (PROMs).","authors":"Tessa Peasgood,&nbsp;Julia M Caruana,&nbsp;Clara Mukuria","doi":"10.1007/s40271-022-00611-w","DOIUrl":"https://doi.org/10.1007/s40271-022-00611-w","url":null,"abstract":"<p><strong>Background: </strong>There is ongoing uncertainty around the most suitable recall period for patient-reported outcome measures (PROMs).</p><p><strong>Method: </strong>This systematic review integrates quantitative and qualitative literature across health, economics, and psychology to explore the effect of a one-day (or '24-h') versus seven-day (or 'one week') recall period. The following databases were searched from database inception to 30 November 2021: MEDLINE, EMBASE, PsycINFO, Web of Science, EconLit, CINAHL Complete, Cochrane Library, and Sociological Abstracts. Studies were included that compared a one-day (or '24-h') versus seven-day (or weekly) recall period condition on patient-reported scores for PROM and Health-Related Quality-of-Life (HRQoL) instrument scores in adult populations (aged 18 and above) or combined paediatric and adult populations with a majority of respondents aged over 18 years. Studies were excluded if they assessed health behaviours only, used ecological momentary assessment to derive an index of daily recall, or incorporated clinician reports of patient symptoms. We extracted results relevant to six domains with generic health relevance: physical functioning, pain, cognition, psychosocial wellbeing, sleep-related symptoms and aggregated disease-specific signs and symptoms. Quantitative studies compared weekly recall scores with the mean or maximum score over the last seven days or with the same-day recall score.</p><p><strong>Results: </strong>Overall, across the 24 quantitative studies identified, 158 unique results were identified. Symptoms tended to be reported as more severe and HRQoL lower when assessed with a weekly recall than a one-day recall. A narrative synthesis of 33 qualitative studies integrated patient perspectives on the suitability of a one-day versus seven-day recall period for assessing health state or quality of life. Participants had mixed preferences, some noted the accuracy of recall for the one-day period but others preferred the seven-day recall for conditions characterised by high symptom variability, or where PROMs concepts required integration of infrequent experiences or functioning over time.</p><p><strong>Conclusion: </strong>This review identified a clear trend toward higher symptom scores and worse quality of life being reported for a seven-day compared to a one-day recall. The review also identified anomalies in this pattern for some wellbeing items and a need for further research on positively framed items. A better understanding of the impact of using different recall periods within PROMs and HRQoL instruments will help contextualise future comparisons between instruments. Questionnaires ask patients about their health over different time periods (e.g., \"what were your symptoms like over the last week?\" versus \"what were your symptoms like today?\"). Studies find that people may report their symptoms as more severe when they are asked to think about their symptoms over t","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2023-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/d9/96/40271_2022_Article_611.PMC10121527.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9755525","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":"Comparing Outcomes of a Discrete Choice Experiment and Case 2 Best-Worst Scaling: An Application to Neuromuscular Disease Treatment.","authors":"Vikas Soekhai,&nbsp;Bas Donkers,&nbsp;Jennifer Viberg Johansson,&nbsp;Cecilia Jimenez-Moreno,&nbsp;Cathy Anne Pinto,&nbsp;G Ardine de Wit,&nbsp;Esther de Bekker-Grob","doi":"10.1007/s40271-023-00615-0","DOIUrl":"https://doi.org/10.1007/s40271-023-00615-0","url":null,"abstract":"<p><strong>Background and objectives: </strong>Case 2 best-worst scaling (BWS-2) is an increasingly popular method to elicit patient preferences. Because BWS-2 potentially has a lower cognitive burden compared with discrete choice experiments, the aim of this study was to compare treatment preference weights and relative importance scores.</p><p><strong>Methods: </strong>Patients with neuromuscular diseases completed an online survey at two different moments in time, completing one method per occasion. Patients were randomly assigned to either first a discrete choice experiment or BWS-2. Attributes included: muscle strength, energy endurance, balance, cognition, chance of blurry vision, and chance of liver damage. Multinomial logit was used to calculate overall relative importance scores and latent class logit was used to estimate heterogeneous preference weights and to calculate the relative importance scores of the attributes for each latent class.</p><p><strong>Results: </strong>A total of 140 patients were included for analyses. Overall relative importance scores showed differences in attribute importance rankings between a discrete choice experiment and BWS-2. Latent class analyses indicated three latent classes for both methods, with a specific class in both the discrete choice experiment and BWS-2 in which (avoiding) liver damage was the most important attribute. Ex-post analyses showed that classes differed in sex, age, level of education, and disease status. The discrete choice experiment was easier to understand compared with BWS-2.</p><p><strong>Conclusions: </strong>This study showed that using a discrete choice experiment and BWS-2 leads to different outcomes, both in preference weights as well as in relative importance scores, which might have been caused by the different framing of risks in BWS-2. However, a latent class analysis revealed similar latent classes between methods. Careful consideration about method selection is required, while keeping the specific decision context in mind and pilot testing the methods.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2023-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/fc/c1/40271_2023_Article_615.PMC10121531.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9755036","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 for Attributes that Characterise Alternative Models of Care in Gastroenterology: A Discrete Choice Experiment.","authors":"Rumbidzai N Mutsekwa,&nbsp;Katrina L Campbell,&nbsp;Russell Canavan,&nbsp;Brendan Mulhern,&nbsp;Rebecca L Angus,&nbsp;Joshua M Byrnes","doi":"10.1007/s40271-022-00609-4","DOIUrl":"https://doi.org/10.1007/s40271-022-00609-4","url":null,"abstract":"<p><strong>Objectives: </strong>Increased demand for gastroenterology services has resulted in growing waitlists, with patients at risk of exceeding clinically recommended wait-times. Given limited healthcare resources, expanded scope models of care are an option to help address this demand, but little is known about patient preferences for these models of care.</p><p><strong>Methods: </strong>Low-risk gastroenterology patients (n = 1198) referred to an outpatient tertiary service in Australia over a 2-year period were invited to participate in an unlabelled discrete choice experiment with seven attributes: primary healthcare professional, wait-time, continuity of care, consultation length, manner and communication skills, reassurance, and cost. These were developed using qualitative research, literature review, and stakeholders' experiences. A d-efficient fractional design was used to construct four blocks of 12 choice sets, with two alternatives. A 13th choice set was included as a data and quality check. Latent class and mixed logit regression were used for analysis. The resulting preference parameters for individual attributes were then used to calculate willingness to pay and willingness to wait.</p><p><strong>Results: </strong>Overall, the model based on the 347 respondents suggested no strong preference for professional background. All other attributes were statistically significant predictors of preference (p < 0.001), with respondents willing to make significant trade-offs (time and cost) before accepting deterioration in attributes. There was strong emphasis on manner and communication skills, with a clinician who listens and provides good explanations overwhelmingly the most important attribute. Latent class analysis identified two patient segments who differed in their preference for the primary treating healthcare professional (doctor or dietitian) based on exposure to either traditional medical or non-medical professional role substitution model.</p><p><strong>Conclusions: </strong>Patients have strong but varied preferences for gastroenterology services based on whether they have been exposed to expanded scope models of care. Design and implementation of new models of care need to consider strategies to overcome any perceived loss in utility or deterioration in healthcare quality for those unfamiliar with professional role substitution.</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":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9088901","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":"Long-Term Improvement in the Patient-Reported Outcomes of Rectal Bleeding, Stool Frequency, and Health-Related Quality of Life with Tofacitinib in the Ulcerative Colitis OCTAVE Clinical Program.","authors":"David P Hudesman, Joana Torres, Leonardo Salese, John C Woolcott, Rajiv Mundayat, Chinyu Su, Mahmoud H Mosli, Jessica R Allegretti","doi":"10.1007/s40271-022-00603-w","DOIUrl":"10.1007/s40271-022-00603-w","url":null,"abstract":"<p><strong>Background: </strong>Tofacitinib is an oral small molecule Janus kinase inhibitor for the treatment of ulcerative colitis (UC). The tofacitinib OCTAVE clinical program included phase III induction (OCTAVE Induction 1 and 2) and maintenance (OCTAVE Sustain) studies, and an open-label, long-term extension study (OCTAVE Open).</p><p><strong>Objective: </strong>This post hoc analysis assessed selected long-term, disease-specific patient-reported outcome (PRO) and health-related quality-of-life (HRQoL) measurements in patients with UC receiving tofacitinib in the OCTAVE clinical program.</p><p><strong>Methods: </strong>Analyses included patients from OCTAVE Open assigned to tofacitinib 5 mg twice daily (subpopulation in remission at Week 52 of OCTAVE Sustain). OCTAVE Open data from the final analyses are shown to Month 48. Endpoints included rectal bleeding subscore (RBS) = 0, stool frequency subscore (SFS) ≤ 1, and HRQoL measure, Inflammatory Bowel Disease Questionnaire (IBDQ) remission (IBDQ total score ≥ 170); with non-responder imputation for missing data at all visits, and last observation carried forward for visits after a patient advanced to the next study (NRI-LOCF). Observed cases were also assessed.</p><p><strong>Results: </strong>At Month 48, of 175 patients, 95 (54.3%) and 96 (54.9%) achieved/maintained RBS = 0 and SFS ≤ 1, respectively (NRI-LOCF). Additionally, 93 (53.1%) patients achieved/maintained IBDQ remission at Month 48 (NRI-LOCF).</p><p><strong>Conclusions: </strong>Among patients who entered OCTAVE Open in remission, most maintained normalization of rectal bleeding and improvement in stool frequency for ≤ 4 years of follow-up in OCTAVE Open. IBDQ remission was also generally maintained in OCTAVE Open. These data show robust maintenance of key UC PROs and durability of response with tofacitinib 5 mg twice daily.</p><p><strong>Trial registration: </strong>http://www.</p><p><strong>Clinicaltrials: </strong>gov (NCT01465763 [21/10/2011]; NCT01458951 [21/10/2011]; NCT01458574 [21/10/2011]; NCT01470612 [21/10/2011]).</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2023-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/28/00/40271_2022_Article_603.PMC9911479.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9081909","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":"The Perception of Women in Rural and Remote Scotland About Intrapartum Care: A Qualitative Study.","authors":"Verity Watson, Helen Bryers, Nicolas Krucien, Seda Erdem, Mary Burnside, Hugo C van Woerden","doi":"10.1007/s40271-022-00608-5","DOIUrl":"10.1007/s40271-022-00608-5","url":null,"abstract":"<p><strong>Background: </strong>The views of mothers are important in shaping policy and practice regarding options for intrapartum care. Mothers in rural and remote areas face unique challenges accessing services, and these need to be well understood. Therefore, our aim was to understand the compromises that women who live in remote and rural settings, more than 1 h from a maternity unit, face regarding intrapartum care.</p><p><strong>Methods: </strong>Qualitative semi-structured telephone interviews (n = 14) were undertaken in rural Scotland with 13 women who had young children and one who was pregnant. Interviews were transcribed and thematically analysed by two researchers.</p><p><strong>Results: </strong>Key themes identified were women's perceptions about risk and the safety of different pathways of maternity care and birth locations; the actual and perceived distance between home and the place of birth, and the type of maternity care available at a place of birth. Mothers in rural and remote areas face particular challenges in choosing where to have their babies. In addition to clinical decisions about 'place of birth' agreed with healthcare professionals, they have to mentally juggle the implications of giving birth when at a distance from family support and away from familiar surroundings. It was clear that many women from rural communities have a strong sense of 'place' and that giving birth in a geographical location, community and culture that feels familiar is important to many of them.</p><p><strong>Conclusions: </strong>Health care staff need to appreciate the impact of non-clinical factors that are important to mothers in remote and rural areas and acknowledge these, even when they cannot be accommodated. Local and national policy also needs to reflect and respond to the practical challenges faced by rurality.</p>","PeriodicalId":51271,"journal":{"name":"Patient-Patient Centered Outcomes Research","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2023-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9440302","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":"How Does the Public Evaluate Vaccines for Low-Incidence, Severe-Outcome Diseases? A General-Population Choice Experiment.","authors":"F Reed Johnson,&nbsp;Angelyn Fairchild,&nbsp;Dale Whittington,&nbsp;Amit K Srivastava,&nbsp;Juan Marcos Gonzalez,&nbsp;Liping Huan","doi":"10.1007/s40271-022-00602-x","DOIUrl":"https://doi.org/10.1007/s40271-022-00602-x","url":null,"abstract":"<p><strong>Background: </strong>Because immunizing large numbers of healthy people could be required to reduce a relatively small number of infections, disease incidence has a large impact on cost effectiveness, even if the infection is associated with very serious health outcomes. In addition to cost effectiveness, the US Advisory Committee on Immunization Practices requires evidence of stakeholders' values and preferences to help inform vaccine recommendations. This study quantified general-population preferences for vaccine trade-offs among disease severity, disease incidence, and other vaccine features.</p><p><strong>Methods: </strong>We developed a best-practice discrete choice experiment survey and administered it to 1185 parents of children aged 12-23 years and 1203 young adults aged 18-25 years from a national opt-in consumer panel. The data were analyzed using exploded-logit latent-class analysis.</p><p><strong>Results: </strong>Latent-class analysis identified two classes with similar relative-importance weights in both samples. One of the two classes represented about half the samples and had preferences consistent with well-structured, logically ordered, and acceptably precise stated-preference utility. Preferences for the other half of the samples were poorly defined over the ranges of vaccine and disease attributes evaluated. Both parents and young adults in the first class evaluated protection from a disease with 1 in 100 incidence and full recovery at home as having statistically the same preference utility as a disease with 1 in 1 million incidence requiring hospitalization and resulting in permanent deafness.</p><p><strong>Conclusions: </strong>The results suggest that vaccines that protect against low-incidence, severe-outcome diseases, provide 'peace of mind' benefits not captured by standard health-outcome metrics. The fact that half the respondents had poorly defined vaccine preferences is a reminder of the challenges of implementing patient-centric vaccine decision making.</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://www.ncbi.nlm.nih.gov/pmc/articles/PMC9744670/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9087285","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":"Rethinking Patient Engagement in Cancer Research.","authors":"Anne L R Schuster,&nbsp;Heather Hampel,&nbsp;Electra D Paskett,&nbsp;John F P Bridges","doi":"10.1007/s40271-022-00604-9","DOIUrl":"https://doi.org/10.1007/s40271-022-00604-9","url":null,"abstract":"","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/de/23/40271_2022_Article_604.PMC9911482.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9561365","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}