Patient-Patient Centered Outcomes Research最新文献

Introduction: Compared with early stages (eBC) metastatic BC (mBC) is incurable. In mBC, aggressive treatment may increase the duration of survival but may also cause severe treatment side effects. A better understanding how patients with BC value different aspects of drug therapy might improve treatment effectiveness, satisfaction and adherence. This systematic review aims to identify and summarise studies evaluating patient preferences for drug therapy of BC and to compare preferences of patients with eBC and mBC.

Methods: The systematic review followed the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. The electronic databases PubMed and Web of Science were searched on 22 June 2023. All studies published to this point were considered. Original studies reporting patient preferences on BC drug therapy determined by any type of choice experiment were eligible. A narrative synthesis of the effect measures presented as relative importance ratings, trade-offs (required benefit to make a therapy worthwhile) or monetary values of the treatment attributes was reported for each study. Risk of bias assessment for individual studies was performed using the checklist for observational studies from the STROBE Statement and the checklist from 'Conducting Discrete Choice Experiments to Inform Healthcare Decision Making: A User's Guide'. The study protocol was registered at the PROSPERO database (CRD42022377031).

Results: A total of 34 studies met the inclusion criteria were included in the analysis evaluating the preferences of patients with eBC (n = 18), mBC (n = 10) or any stage BC (n = 6) on, for example, chemotherapy, endocrine therapy, hormonal therapy or CKD4/6-inhibitors using different types of choice experiments. Regardless of the stage, most patients valued treatment effectiveness in terms of survival gains higher than potential adverse drug reactions (ADRs). Treatment cost, mode of administration, treatment regimen and monitoring aspects were considered as least important treatment attributes. In addition, preferences concerning 16 different types of ADRs were described, showing high heterogeneity within BC stages. Yet, comparable results across BC stages were observed.

Conclusions: Regardless of the stage, patients with BC consistently valued survival gains as the most important attribute and were willing to accept the risk of potential ADRs. Incorporating patient preferences in shared decision making may improve the effectiveness of interventions by enhancing adherence to drug therapy in patients suffering from BC.

Background: There is evidence supporting the value of patient engagement (PE) in research to patients and researchers. However, there is little research evidence on the influence of PE throughout the entire research process as well as the outcomes of research engagement. The purpose of our study is to add to this evidence.

Methods: We used a convergent mixed method design to guide the integration of our survey data and observation data to assess the influence of PE in two groups, comprising patient research partners (PRPs), clinicians, and researchers. A PRP led one group (PLG) and an academic researcher led the other (RLG). Both groups were given the same research question and tasked to design and conduct an inflammatory bowel disease (IBD)-related patient preference study. We administered validated evaluation tools at three points and observed PE in the two groups conducting the IBD study.

Results: PRPs in both groups took on many operational roles and influenced all stages of the IBD-related qualitative study: launch, design, implementation, and knowledge translation. PRPs provided more clarity on the study design, target population, inclusion-exclusion criteria, data collection approach, and the results. PRPs helped operationalize the project question, develop study material and data collection instruments, collect data, and present the data in a relevant and understandable manner to the patient community. The synergy of collaborative partnership resulted in two projects that were patient-centered, meaningful, understandable, legitimate, rigorous, adaptable, feasible, ethical and transparent, timely, and sustainable.

Conclusion: Collaborative and meaningful engagement of patients and researchers can influence all stages of qualitative research including design and approach, and outputs.

Background: Individuals living with transfusion-dependent β-thalassemia (TDT) experience reduced health-related quality of life due to fatigue and chronic pain, which cause disruptions to daily life. Currently, limited qualitative data exist that describe these impacts.

Objective: This study aimed to examine the ways in which symptoms and current treatments of TDT impact health-related quality of life, to holistically describe the humanistic burden of TDT, and to identify the unmet needs of individuals living with TDT.

Methods: Adults (aged ≥ 18 years) with TDT and caregivers of adolescents (aged 12‒17 years) with TDT participated in semi-structured one-on-one virtual interviews and focus group discussions. Interviews were conducted in the USA and UK and lasted approximately 60 minutes. After transcription, the interviews were analyzed thematically using a framework approach.

Results: A total of ten interviews/focus group discussions (six interviews and four focus group discussions) were conducted with 14 adults with TDT and two caregivers of adolescents with TDT. A framework analysis revealed five themes describing health-related quality of life (negative impacts on daily activities, social life, family life, work and education, and psychological well-being) and three themes describing the lived experience of TDT (impact of red blood cell transfusions and iron chelation therapy, treatment, and stigma). Physical, psychological, and treatment-related factors contributed to negative impacts on daily activities, social and family life, and work and education. Concerns about reduced lifespan, relationships and family planning, and financial independence were detrimental to participants' mental well-being. Participants reported having high resilience to the many physical and psychological challenges of living with TDT. A lack of TDT-specific knowledge among healthcare professionals, particularly regarding chronic pain associated with the disease, left some participants feeling ignored or undermined. Additionally, many participants experienced stigma and were reluctant to disclose their disease to others.

Conclusions: Individuals living with TDT experience substantial negative impacts on health-related quality of life that disrupt their daily lives, disruptions that are intensified by inadequate healthcare interactions, demanding treatment schedules, and stigma. Our study highlights the unmet needs of individuals living with TDT, especially for alternative treatments that reduce or eliminate the need for red blood cell transfusions and iron chelation therapy.

Background and objective: Consumer engagement improves research quality and relevance but can be difficult to implement. This study aimed to explore the motivations and understand the barriers, if any, experienced by consumers before and when partnering with cancer research teams.

Methods: Semi-structured interviews were conducted with consumers and the results analysed thematically. Two groups were recruited: consumers who were members of the consumer registry and patients who did not have previous experience of being a consumer in a researcher partnership.

Results: Twenty-one interviews were conducted with a total of 22 participants aged between 26 and 74 years. Consumers motivation was driven by altruism to help others and personal benefits. Barriers to beginning and maintaining consumer engagement included consumers' perceptions of being appreciated by researchers and meaningful communication between researchers and consumers.

Conclusions: Australian policy has made important steps towards consumer engagement in research. This study showed that demonstrating an appreciation for consumers and effective communication are key areas to consider when designing implementation strategies of these policies in the cancer research space in the future.

Background: Chinese rural women aged 35-64 years are encouraged to complete breast cancer screening (BCS) free of charge. However, it is challenging to reach a satisfying BCS uptake rate. In this study, rural women's preferences and preferences heterogeneity were measured for the development of strategies to enhance participation in BCS.

Methods: A cross-sectional survey with a discrete choice experiment (DCE) was conducted via convenience sampling via face-to-face interviews in Jiangsu, China. Six DCE attributes were identified through a systematic literature review; our previous study of Chinese rural women's BCS intentions; a qualitative work involving in-depth interviews with rural women (n = 13), medical staff (n = 4), and health care managers (n = 2); and knowledge of realistic and actionable policy. The D-efficient design was generated using Ngene 1.3.0. A mixed logit model (MXL) in Stata 18.0 was used to estimate the main effect of attribute levels on rural women's preferences. The relative importance and willingness to utilize BCS services (WTU) were also estimated. The heterogeneous preferences were analyzed by a latent class model (LCM). Sociodemographic status was used to predict the characteristics of class membership. The WTU for different classes was also calculated.

Results: A total of 451 rural women, aged 35-64 years, were recruited. The MXL results revealed that the screening interval (SI) was the most important attribute for rural women with regard to utilizing BCS services, followed by the level of screening, the attitude of medical staff, ways to get knowledge and information, people who recommend screening, and time spent on screening (TSS). Rural women preferred a BCS service with a shorter TSS; access to knowledge and information through multiple approaches; a shorter SI; a recommendation from medical staff or workers from the village or community, and others; the enthusiasm of medical staff; and medical staff with longer tenures in the field. Two classes named "process driven" and "efficiency driven" were identified by the preference heterogeneity analysis of the LCM.

Conclusion: There is a higher uptake of breast cancer screening when services are tailored to women's preferences. The screening interval was the most important attribute for rural women in China with a preference for a yearly screening interval versus longer intervals.

Background: Patients with cancer may progress through multiple treatments with differing adverse effect profiles. Moreover, pathways may be fixed or flexible in allowing for escalation or de-escalation of treatment depending on interim outcomes. We sought to develop a methodology capable of estimating preferences for the entirety of a pathway involving a sequence of different treatments.

Methods: Patients with early breast cancer completed an online discrete choice experiment to assess preferences for eight key early breast cancer attributes. Hierarchical Bayesian modeling was used to calculate attribute-level preference weights. Preference weights for hypothetical pathways were estimated by summing the respective weights for efficacy, flexible or fixed pathway, duration, administration regimen, and adverse event risk, the last two of which were time-adjusted by multiplying each weight by the proportion of time spent on a selected treatment.

Results: Increases in the risk of a serious adverse event were most influential in treatment pathway preferences, followed by increases in efficacy and decreases in overall pathway duration. Patients preferred a flexible pathway versus a fixed pathway. Pathway preference estimates fluctuated in a logically consistent manner. Switching from a flexible to a fixed pathway yielded a significantly lower pathway preference. For this same pathway, when adjuvant treatment was replaced with a treatment with a more favorable toxicity profile and shorter duration, it offset the negative impact of the more toxic neoadjuvant chemotherapy.

Conclusions: This novel methodology accounts for patient preference throughout a sequence of treatments, allowing for comparison of preferences across complex treatment pathways.

Choice-based preference elicitation methods such as the discrete choice experiment (DCE) present hypothetical choices to respondents, with an expectation that these hypothetical choices accurately reflect a 'real world' health-related decision context and that consequently the choice data can be held to be a true representation of the respondent's health or treatment preferences. For this to be the case, careful consideration needs to be given to the format of the choice task in a choice experiment. The overarching aim of this paper is to highlight important aspects to consider when designing and 'setting up' the choice tasks to be presented to respondents in a DCE. This includes the importance of considering the potential impact of format (e.g. choice context, choice set presentation and size) as well as choice set content (e.g. labelled and unlabelled choice sets and inclusion of reference alternatives) and choice questions (stated choice versus additional questions designed to explore complete preference orders) on the preference estimates that are elicited from studies. We endeavoure to instil a holistic approach to choice task design that considers format alongside content, experimental design and analysis.

Background: Stakeholders increasingly expect research and care delivery to be guided by and to optimize patient experiences. However, standardized tools to engage patients to gather high-quality data about their experiences, priorities, and desired outcomes are not publicly available. The objective of this study was to develop and test a Toolbox with a disease-agnostic interview guide template and accompanying resources to assist researchers in engaging patients living with chronic disease in a dialogue about their experiences.

Methods: Guided by a multidisciplinary workgroup, a targeted literature review (PubMed) was conducted, followed by group discussions to identify/thematically organize patient experience concepts, development of a conceptual model, and drafting of an interview guide template and patient-facing visual. Materials were tested/refined via cognitive (n = 5) and pilot (n = 30) interviews conducted virtually with US patients diagnosed with chronic/potentially disabling conditions from December 2020 to April 2021. Patient-facing tools were reviewed by health literacy experts for applicability/accessibility. English-speaking adults who self-reported receiving a chronic condition diagnosis at least 6 months prior participated in a 60-90 min interview.

Results: Patient experience concepts were organized thematically under three domains: (1) life before a diagnosis, (2) experiences getting a diagnosis, and (3) experiences living with a diagnosis. A plain language consent sheet template, interview guide template, and patient experience conceptual model were developed and revised based on input from interviewees, interviewers, and the workgroup.

Conclusions: A disease-agnostic patient-engagement Toolbox was developed and tested to capture patient experience data. These materials can be customized based on study objectives and leveraged by various stakeholders to identify opportunities to enhance the patient centricity of healthcare delivery and research.

Interest in using patient preference (PP) data alongside traditional economic models in health technology assessment (HTA) is growing, including using PP data to quantify non-health benefits. However, this is limited by a lack of standardised methods. In this article, we describe a method for using discrete choice experiment (DCE) data to estimate the value of non-health benefits in terms of quality-adjusted survival equivalence (QASE), which is consistent with the concept of value prevalent among HTA agencies. We describe how PP data can be used to estimate QASE, assess the ability to test the face-validity of QASE estimates of changes in mode of administration calculated from five published DCE oncology studies and review the methodological and normative considerations associated with using QASE to support HTA. We conclude that QASE may have some methodological advantages over alternative methods, but this requires DCEs to estimate second-order effects between length and quality of life. In addition, empirical work has yet to be undertaken to substantiate this advantage and demonstrate the validity of QASE. Further work is also required to align QASE with normative objectives of HTA agencies. Estimating QASE would also have implications for the conduct of DCEs, including standardising and defining more clear attribute definitions.