Patient-Patient Centered Outcomes Research最新文献

Background: People with relapsing-remitting multiple sclerosis can benefit from disease-modifying treatments (DMTs). Several DMTs are available that vary in their efficacy, side-effect profile and mode of administration.

Objective: We aimed to measure the preferences of people with relapsing-remitting multiple sclerosis for DMTs using a discrete choice experiment and to assess which stated preference attributes correlate with the attributes of the DMTs they take in the real world.

Methods: Discrete choice experiment attributes were developed from literature reviews, interviews and focus groups. In a discrete choice experiment, participants were shown two hypothetical DMTs, then chose whether they preferred one of the DMTs or no treatment. A mixed logit model was estimated from responses and individual-level estimates of participants' preferences conditional on their discrete choice experiment choices calculated. Logit models were estimated with stated preferences predicting current real-world on-treatment status, DMT mode of administration and current DMT.

Results: A stated intrinsic preference for taking a DMT was correlated with currently taking a DMT, and stated preferences for mode of administration were correlated with the modes of administration of the DMTs participants were currently taking. Stated preferences for treatment effectiveness and adverse effects were not correlated with real-world behaviour.

Conclusions: There was variation in which discrete choice experiment attributes correlated with participants' real-world DMT choices. This may indicate patient preferences for treatment efficacy/risk are not adequately taken account of in prescribing. Treatment guidelines must ensure they take into consideration patients' preferences and improve communication around treatment efficacy/risk.

Objectives: Identify Australian public preferences for antibiotic treatments in the context of antibiotic stewardship.

Methods: A discrete choice experiment (DCE) was conducted in Australia to investigate the importance of seven attributes associated with antibiotic treatments and related stewardship practices: contribution to antimicrobial resistance (AMR), treatment duration, side effects, days needed to recover, days before taking antibiotics, treatment failure and out-of-pocket costs. The DCE data were analysed using conditional logit, mixed logit and latent class conditional logit models. The relative importance of each attribute was calculated.

Results: A total of 1882 respondents completed the survey; the main study sample consist of 1658 respondents (mean age 48 years) who passed quality checks. All seven attributes significantly influenced respondents' preferences for antibiotic treatments. Based on the designed attribute levels in the DCE, on average, out-of-pocket costs (32.8%) and contribution to antibiotic resistance (30.3%) were the most important attributes, followed by side effects (12.9%). Days before starting medication was least important (3.9%). Three latent classes were identified. Class 1 (including respondents who were more likely to be older and more health literate; 24.5%) gave contribution to antibiotic resistance greater importance in treatment preferences. Class 2 (including respondents more likely to report poorer health; 25.2%) gave out-of-pocket costs greater importance. The remaining (50.4%), who were generally healthier, perceived side effects as the most important attribute.

Conclusions: Despite concerted public awareness raising campaigns, our results suggest that several factors may influence the preferences of Australians when considering antibiotic use. However, for those more likely to be aware of the need to preserve antibiotics, out-of-pocket costs and limiting the contribution to antibiotic resistance are the dominant influence. Delays in starting treatment were not important for any latent class, suggesting public tolerance for this measure. These results could help inform strategies to promote prudent antibiotic stewardship.

Purpose: To evaluate the measurement properties of the 15-item Singapore Caregiver Quality of Life Scale (SCQOLS-15) in family caregivers of patients with heart diseases.

Methods: The SCQOLS-15 survey was self-administered by family caregivers of patients with chronic heart diseases, at baseline and 1 week later. The criterion validity of SCQOLS-15 and its domain scores was assessed by calculating the Spearman correlation coefficient (ρ) with the Brief Assessment Scale for Caregivers (BASC), Caregiver Reaction Assessment (CRA), and their sub-scores. Known-group validity was assessed using the New York Heart Association (NYHA) functional class. Test-retest reliability was evaluated using the intraclass correlation coefficient (ICC).

Results: Of the 327 caregivers included, 65% were adult children and 28% were spouses. The distribution of NYHA classes of the patients was I: 27%, II: 40%, III: 24%, and IV: 9%. There was a positive correlation between the SCQOLS-15 and BASC total scores (ρ = 0.7). SCQOLS-15 domain scores were also correlated with BASC and CRA sub-scores as per a priori hypotheses, with absolute values of ρ ranging from 0.4 to 0.6. The mean values of SCQOLS-15 total and all domain scores were lower among caregivers of patients with NYHA class III/IV compared to those of class I/II patients (each P < 0.05). Among 146 caregivers who completed the follow-up and self-rated a stable quality-of-life, ICCs for test-retest reliability of SCQOLS-15 total and all domain scores were ≥ 0.8.

Conclusion: The SCQOLS-15 is a valid and reliable instrument for measuring the quality of life in caregivers of heart disease patients.

Background: Multiple studies have indicated a socioeconomic impact of cancer and cancer care on patients and their families. Existing instruments designed to measure this impact lack consensus in their conceptualization of the issue. Further, various terminologies have been used in the literature (e.g., financial burden, financial hardship, financial stress) without clear definitions and consistent conceptual background. Based on a targeted review of existing models addressing the socioeconomic impact of cancer, our goal was to develop a comprehensive framework from a European perspective.

Method: A 'best-fit' framework synthesis was applied. First, we systematically identified existing models to generate a priori concepts. Second, we systematically identified relevant European qualitative studies and coded their results against these a priori concepts. Inclusion and exclusion criteria were predefined and applied thoroughly in these processes. Thematic analysis and team discussions were applied to finalize the (sub)themes in our proposed conceptual framework. Third, we examined model structures and quotes from qualitative studies to explore relationships among (sub)themes. This process was repeated until no further change in (sub)themes and their relationships emerged.

Result: Eighteen studies containing conceptual models and seven qualitative studies were identified. Eight concepts and 20 sub-concepts were derived from the included models. After coding the included qualitative studies against the a priori concepts and following discussions among team members, seven themes and 15 sub-themes were included in our proposed conceptual framework. Based on the identified relationships, we categorized themes into four groups: causes, intermediate consequences, outcomes and risk factors.

Conclusion: We propose a Socioeconomic Impact Framework based on a targeted review and synthesis of existing models in the field and adapted to the European perspective. Our work contributes as an input to a European consensus project on socioeconomic impact research by an Organization European Cancer Institute (OECI) Task Force.

Objective: This study aimed to elicit preferences for attributes of current and novel long-acting antiretroviral therapy for human immunodeficiency virus treatment.

Methods: Primary survey data were collected (July-October 2022) on a sample of 333 people living with human immunodeficiency virus in Germany from a patient recruitment agency. Respondents were invited by e-mail to respond to a web-based questionnaire. After performing a systematic literature review, we conducted qualitative semi-structured interviews to identify and select the key attributes of drug therapy for patients' preferences for human immunodeficiency virus treatment. Based on this, a discrete choice experiment survey elicited preferences for long-acting antiretroviral therapy characteristics, including the type of medication, frequency of dosing, the location of treatment, the risk of both short-term and long-term side effects, as well as possible interactions with other medications or (party) drugs. A statistical data analysis was performed using multinomial logit models. An additional latent class multinomial logit was performed to evaluate subgroup differences.

Results: Overall, 226 respondents (86% male, mean age 46.1 years) were included in the analysis. The frequency of dosing (36.1%) and the risk of long-term side effects (28.2%) had the greatest influence on preferences. The latent class analysis identified two patient groups. While the first class (n = 135; 87% male, mean age 44.4 years) found the frequency of dosing (44.1%) to be most important, the second class (n = 91; 85% male, mean age 48.6 years) focused on the risk of long-term side effects (50.3%). The evaluation of structural variables showed that male respondents, those living in small cities or villages, and those with better health status results were significantly more likely to be assigned to the second class (p < 0.05 each).

Conclusions: All attributes included in our survey were important to participants when choosing an antiretroviral therapy. We found evidence that the frequency of dosing as well as the risk of long-term side effects have a particular impact on the acceptance of novel therapy regimens and should be considered in order to optimize adherence and satisfaction.

Background: Self-reported health data from children with life-limiting conditions is rarely collected. To improve acceptability and feasibility of child and family-centred outcome measures for children, they need to be designed in a way that reflects preferences, priorities and abilities.

Objectives: The aim was to identify preferences for patient-reported outcome measure design (recall period, response format, length, administration mode) to improve the feasibility, acceptability, comprehensibility and relevance of a child and family-centred outcome measure, among children with life-limiting conditions and their family members.

Method: A semi-structured qualitative interview study seeking the perspectives of children with life-limiting conditions, their siblings and parents on measure design was conducted. Participants were purposively sampled and recruited from nine UK sites. Verbatim transcripts were analysed using framework analysis.

Results: A total of 79 participants were recruited: 39 children aged 5-17 years (26 living with a life-limiting condition; 13 healthy siblings) and 40 parents (of children aged 0-17 years). Children found a short recall period and a visually appealing measure with ten questions or fewer most acceptable. Children with life-limiting conditions were more familiar with using rating scales such as numeric and Likert than their healthy siblings. Children emphasised the importance of completing the measure alongside interactions with a healthcare professional to enable them to talk about their responses. While parents assumed that electronic completion methods would be most feasible and acceptable, a small number of children preferred paper.

Conclusions: This study demonstrates that children with life-limiting conditions can engage in communicating preferences regarding the design of a patient-centred outcome measure. Where possible, children should be given the opportunity to participate in the measure development process to enhance acceptability and uptake in clinical practice. Results of this study should be considered in future research on outcome measure development in children.

Despite growing commitment to patient centricity, challenges persist in consistently identifying the impacts of disease and/or treatment that patients report as most important to them, especially across myriad potential downstream uses. Patient-centered core impact sets (PC-CIS), disease-specific lists of impacts that patients report as most important, are proposed as a solution. But, PC-CIS is a new concept, currently in the pilot stage with patient advocacy groups. We conducted an environmental scan to explore PC-CIS conceptual overlap with past/existing efforts [e.g., core outcome sets (COS)] and to inform general feasibility for further development and operationalization. With guidance and advice from an expert advisory committee, we conducted a search of the literature and relevant websites. Identified resources were reviewed for alignment with the PC-CIS definition, and key insights were gleaned. We identified 51 existing resources and five key insights: (1) no existing efforts identified meet the definition of PC-CIS as we have specified it in terms of patient centricity, (2) existing COS-development efforts are a valuable source of foundational resources for PC-CIS, (3) existing health-outcome taxonomies can be augmented with patient-prioritized impacts to create a comprehensive impact taxonomy, (4) current approaches/methods can inadvertently exclude patient priorities from core lists/sets and will need to be modified to protect the patient voice, and (5) there is need for clarity and transparency on how patients were engaged in individual past/existing efforts. PC-CIS is conceptually unique from past/existing efforts in its explicit emphasis on patient leadership and being patient driven. However, PC-CIS development can leverage many resources from the past/existing related work.

Health-related discrete choice experiments (DCEs) information can be used to inform decision-making on the development, authorisation, reimbursement and marketing of drugs and devices as well as treatments in clinical practice. Discrete choice experiment is a stated preference method based on random utility theory (RUT), which imposes strong assumptions on respondent choice behaviour. However, respondents may use choice processes that do not adhere to the normative rationality assumptions implied by RUT, applying simplifying decision rules that are more selective in the amount and type of processed information (i.e., simplifying heuristics). An overview of commonly detected simplifying heuristics in health-related DCEs is lacking, making it unclear how to identify and deal with these heuristics; more specifically, how researchers might alter DCE design and modelling strategies to accommodate for the effects of heuristics. Therefore, the aim of this paper is three-fold: (1) provide an overview of common simplifying heuristics in health-related DCEs, (2) describe how choice task design and context as well as target population selection might impact the use of heuristics, (3) outline DCE design strategies that recognise the use of simplifying heuristics and develop modelling strategies to demonstrate the detection and impact of simplifying heuristics in DCE study outcomes.

Background and objective: The World Health Organization physical activity guidelines for people living with disability do not consider the needs of people living with moderate-to-severe traumatic brain injury. This paper describes the qualitative co-development of a discrete choice experiment survey to inform the adaption of these guidelines by identifying the physical activity preferences of people living with moderate-to-severe traumatic brain injury in Australia.

Methods: The research team comprised researchers, people with lived experience of traumatic brain injury and health professionals with expertise in traumatic brain injury. We followed a four-stage process: (1) identification of key constructs and initial expression of attributes, (2) critique and refinement of attributes, (3) prioritisation of attributes and refinement of levels and (4) testing and refining language, format and comprehensibility. Data collection included deliberative dialogue, focus groups and think-aloud interviews with 22 purposively sampled people living with moderate-to-severe traumatic brain injury. Strategies were used to support inclusive participation. Analysis employed qualitative description and framework methods.

Results: This formative process resulted in discarding, merging, renaming and reconceptualising attributes and levels. Attributes were reduced from an initial list of 17 to six: (1) Type of activity, (2) Out-of-pocket cost, (3) Travel time, (4) Who with, (5) Facilitated by and (6) Accessibility of setting. Confusing terminology and cumbersome features of the survey instrument were also revised. Challenges included purposive recruitment, reducing diverse stakeholder views to a few attributes, finding the right language and navigating the complexity of discrete choice experiment scenarios.

Conclusions: This formative co-development process significantly improved the relevance and comprehensibility of the discrete choice experiment survey tool. This process may be applicable in other discrete choice experiment studies.

Aims: The aim of this study was to explore the experiences, values and preferences of people living with relapsing multiple sclerosis (PLwRMS) focusing on their treatments and what drives their treatment preferences.

Methods: In-depth, semi-structured, qualitative telephone interviews were conducted using a purposive sampling approach with 72 PLwRMS and 12 health care professionals (HCPs, MS specialist neurologists and nurses) from the United Kingdom, United States, Australia and Canada. Concept elicitation questioning was used to elicit PLwRMS' attitudes, beliefs and preferences towards features of disease-modifying treatments. Interviews with HCPs were conducted to inform on HCPs' experiences of treating PLwRMS. Responses were audio recorded and transcribed verbatim and then subjected to thematic analysis.

Results: Participants discussed numerous concepts that were important to them when making treatment decisions. Levels of importance participants placed on each concept, as well as reasons underpinning importance, varied substantially. The concepts with the greatest variability in terms of how much PLwRMS found them to be important in their decision-making process were mode of administration, speed of treatment effect, impact on reproduction and parenthood, impact on work and social life, patient engagement in decision making, and cost of treatment to the participant. Findings also demonstrated high variability in what participants described as their ideal treatment and the most important features a treatment should have. HCP findings provided clinical context for the treatment decision-making process and supported patient findings.

Conclusions: Building upon previous stated preference research, this study highlighted the importance of qualitative research in understanding what drives patient preferences. Characterized by the heterogeneity of the RMS patient experience, findings indicate the nature of treatment decisions in RMS to be highly individualized, and the subjective relative importance placed on different treatment factors by PLwRMS to vary. Such qualitative patient preference evidence could offer valuable and supplementary insights, alongside quantitative data, to inform decision making related to RMS treatment.