Patient-Patient Centered Outcomes Research最新文献

Background: Understanding the treatment experiences and satisfaction of people with HIV using two-drug regimens, including dolutegravir/lamivudine (DTG/3TC), is crucial to assess ongoing needs and meet Ending the HIV Epidemic goals.

Methods: PAIRED is a mixed-methods study comprising a quantitative cross-sectional survey (using validated instruments to assess treatment satisfaction, adherence, and health-related quality of life [QOL]) and qualitative interviews. Adults in the USA who switched to DTG/3TC while virologically suppressed and had been using DTG/3TC for ≥ 3 months were eligible. All participants were surveyed, and a sub-set participated in in-depth interviews. Thematic analysis was performed to discern overarching interview themes.

Results: Overall, 474 participants completed the survey (assigned female sex at birth, 31%; non-white, 48%; aged ≥ 50 years, 50%); 20 of them participated in qualitative interviews. Treatment satisfaction, adherence, and QOL scores were high. Six themes were extracted from interviews: (1) fewer drugs are important in HIV treatment because of associated reductions in long-term drug exposure and perceived risk of toxicity; (2) high DTG/3TC satisfaction is largely due to achieved expectations regarding efficacy and safety; (3) DTG/3TC simplicity and convenience enable more freedom and autonomy; (4) treatment advancements led to HIV no longer being a "death sentence"; (5) managing HIV is no longer the main concern relative to other health conditions; and (6) some HIV unmet needs remain.

Conclusions: A large representative population with HIV had primarily positive experiences and few needs after switching to DTG/3TC. These data complement clinical trial data and support DTG/3TC effectiveness and tolerability in real-world settings.

Background: Early detection of cancer reduces mortality and morbidity, but conventional screening methods often face challenges such as invasiveness, limited accessibility and high resource demands. Emerging cancer screening technologies could overcome these barriers, yet their adoption depends heavily on public acceptance. This systematic review synthesises evidence from discrete choice experiment (DCE) studies examining population preferences for emerging cancer screening technologies.

Methods: A systematic review was conducting following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. PubMed, Embase, Scopus, Web of Science and EconLit were systematically searched for studies published up to August 2025. Eligible studies employed DCEs to assess preferences for modalities including liquid biopsy, multi-cancer early detection tests, artificial intelligence reading systems, genetic and genomic testing, mobile Health applications, nanopill-based screening and breath analysis. Studies were screened independently by two reviewers and assessed for reporting quality using the DIRECT checklist. Data were summarised using descriptive statistics and narrative synthesis.

Results: In total, 23 studies published between 2014 and 2025 were included, most focusing on genomic testing (n = 8) or liquid biopsy (n = 5). Across studies, 32 attributes were identified and categorised under clinical risk, modality characteristics, screening process, outcomes, or ethical considerations. Respondents generally favoured screening programmes that were clinically effective, accessible and minimally disruptive. Sensitivity was most frequently reported as the most influential attribute. Modality specific attributes influencing public preferences were identified, including data privacy and insurability for genetic and genomic testing, and the role of test providers for mHealth technologies and AI screening tools. Heterogeneity in preferences, attribute importance and predicted uptake were observed across many studies, highlighting differential attitudes towards cancer screening and modality specific attributes within the population.

Conclusions: This systematic review provides the first synthesis of DCE evidence across a wide range of emerging cancer screening technologies. Findings reveal that public preferences were shaped not only by clinical performance, but also by convenience, cost and the perceived credibility of new modalities. Considerable heterogeneity in preferences signals the need for tailored strategies to engage different population subgroups and avoid widening disparities in uptake. As these technologies move towards clinical adoption, robust governance and regulatory safeguards will be essential to ensure their translation into equitable and effective cancer screening programmes.

Background: Patient and public involvement (PPI) in health research is gaining global momentum through initiatives such as INVOLVE (UK), the Patient-Centred Outcomes Research Institute (USA), and the Strategy for Patient-Oriented Research (Canada). However, its implementation in Africa remains limited and lacks context-specific guidance. To address this gap, the Sickle Africa Data Coordinating Center supported the development of the Patients as Partners in Sickle Cell Disease Research (SCD-PAPIR) Framework to guide meaningful patient engagement and involvement in SCD research in Africa.

Methods: An iterative, participatory approach was adopted. The process involved the creation of a SCD PAPIR working group comprising SCD patient advocates in 14 African countries; 18 virtual working group meetings, one public webinar, and three in-person workshops. The framework was co-created through facilitated discussions reflecting on prior engagement in SCD research as a patient partner, and best practices for effective patient-researcher collaborations. Patient-only sessions and leadership roles were integrated to ensure safe spaces and to minimise power imbalances.

Results: The SCD-PAPIR Framework positions SCD patients and their caregivers as citizen researchers. Key to the framework is the principle of subsidiarity, which affirms the agency and experiential knowledge of patients while calling for epistemic humility from researchers. Its core pillars include valuing patient expertise, transparent communication, resource sharing, patient empowerment, collective learning, amplification of patient voice, continuous feedback, and shared benefits. Best practices emphasise two-way communication, addressing power asymmetries, co-learning and patient empowerment, co-ownership of outputs, and formalised PAPIR structures.

Conclusion: The SCD-PAPIR Framework provides a contextually grounded model for patient-engaged research in Africa and contributes to efforts to decolonise health research by positioning patients as co-creators of knowledge, and not merely a data source. The effective implementation of the framework will require investment in institutionalising PAPIR in SCD research. Future work should focus on designing implementation toolkits, developing PPI training modules for researchers and patient advocates, and adapting the framework to other health conditions.

Background and objective: Best-worst scaling (BWS) is a stated preference elicitation method used for prioritizing attributes of healthcare interventions. Best-worst scaling attribute development is commonly based on literature review, qualitative work, and methodological/clinical expert input. There is limited research incorporating BWS in focus groups as part of the attribute development process. We sought to explore how incorporating BWS questions using the list of potential attributes in focus groups could be used to improve understanding of patient preferences and refine the list of potential BWS attributes as part of the attribute development process.

Methods: We administered BWS questions on healthcare priorities for inflammatory bowel disease in five focus groups with Canadian patients with inflammatory bowel disease to (1) understand the "what," "how," and "why" of participant choices and (2) note how participants understand the attributes and the language they use to refine the list of potential BWS attributes. A list of 20 potential attributes was used to generate the BWS questions. We coded most/least important choices ("what") and used a thematic analysis to derive subthemes indicating "how" and "why" participants made their choices. We coded how participants understood the attributes/BWS questions and language used when discussing the attributes.

Results: Across the 36 participants, the most frequently chosen most/least important attributes were summarized. Three subthemes explaining the "how" and "why" of participant choices were identified: perceived gain; influence of individual experiences; current health state and personal circumstances. Participants identified challenges understanding specific attributes and BWS questions, and provided suggestions for modifications to attribute language/descriptions. Administering BWS questions in focus groups provided: (1) insight into the assumptions participants made when completing the BWS questions; (2) clarity in language and attribute descriptions, and challenges participants had when completing the BWS questions that can be used to refine the list of potential attributes as part of the attribute development process; and (3) understanding of which attributes were most/least important and why to identify potential attributes to remove during the next steps of the attribute development process.

Conclusions: Best-worst scaling questions conducted within focus groups can stimulate discussions around relative importance and prioritization of attributes. Through open dialogue, this method can unveil unforeseen responses or identify areas that are unclear and enable a transparent approach to refine the list of potential attributes as part of the attribute development process.

Background and objective: The COVID-19 pandemic has significantly influenced vaccination strategies and public health policies. Discrete choice experiments have emerged as a valuable tool for understanding preferences regarding vaccination. This study systematically reviews discrete choice experiments conducted on COVID-19 public vaccination preferences to identify key determinants influencing vaccine uptake and to assess methodological approaches used in these studies.

Methods: A systematic literature search was conducted across major databases, including PubMed, Scopus, and Web of Science, to identify discrete choice experiments focusing on COVID-19 vaccination preferences up to 31 December, 2024. Attribute categorization into five dimensions Outcome, Process, Cost, Trust, and Framing was performed and quality appraised according to the DIRECT checklist. Conditional relative importance as well as geographical differences were assessed.

Results: The review identified 58 studies employing discrete choice experiments that assessed public COVID-19 vaccine preferences. Among attribute categories, outcome-related factors were the most frequently used and had the highest relative importance. Other commonly evaluated attributes included cost, origin/brand, and required doses. A notable geographic disparity was observed, with studies being unevenly distributed across different regions. Methodological heterogeneity was observed in attribute selection and experimental design.

Conclusions: This review emphasizes the importance of considering individual preferences into vaccination strategies to enhance uptake, particularly in preparation for future pandemics. The findings reveal that vaccine effectiveness and safety are key concerns for individuals. Future research could focus on increasing representation of underexamined regions in preference studies to better inform local policymakers in developing effective vaccination programs for future health crises.

Clinical trial registration: This review was prospectively registered in PROSPERO (International Prospective Register of Systematic Reviews) with the ID CRD42025543234.

Background: Breast cancer screening is considered an effective early detection strategy. Artificial intelligence (AI) may both offer benefits and create risks for breast screening programmes. To use AI in health screening services, the views and expectations of consumers are critical. This study examined the preferences of Australian women regarding AI use in breast cancer screening and the impact of information on preferences using discrete choice experiments.

Methods: The experiment presented two alternative screening services based on seven attributes (reading method, screening sensitivity, screening specificity, time between screening and receiving results, supporting evidence, fair representation, and who should be held accountable) to 2063 women aged between 40 and 74 years recruited from an online panel. Participants were randomised into two arms. Both received standard information on AI use in breast screening, but one arm received additional information on its potential benefits. Preferences for hypothetical breast cancer screening services were modelled using a random parameter logit model. Relative attribute importance and uptake rates were estimated.

Results: Participants preferred mixed reading (radiologist + AI system) over the other two reading methods. They showed a strong preference for fewer missed cases with a high attribute relative importance. Fewer false positives and a shorter waiting time for results were also preferred. Strength of preferences for mixed reading was significantly higher compared to two radiologists when additional information on AI is provided, highlighting the impact of information.

Conclusions: This study revealed the preferences among Australian women for the use of AI-driven breast cancer screening services. Results generally suggest women are open to their mammograms being read by both a radiologist and an AI-based system under certain conditions.

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.

Partnerships between patients and the medical research community are strengthening. Patient involvement in research processes through collaborative workstreams provides authentic insights and perspectives, enhances trust between stakeholders and the patient community, brings balance to authorship groups and adds value and contextualisation to publications. Here, patient advocates, representatives from patient and caregiver communities and pharmaceutical and medical communications professionals propose seven actions to advance patient authorship and collaboration in peer-reviewed publications. Drawing on research, personal experience and professional insight, they call for a shift in conventional publication development practices-from seeking reasons to include patient authors to requiring justification for their exclusion-thereby facilitating greater inclusion and representation of the patient voice. The authors advocate moving beyond the concept of 'patient-centricity' towards 'patient partnership' to reflect a collaborative approach and more equitable balance of power and benefits among stakeholders. They also emphasise the importance of involving patients holistically in publication steering committees to ensure that the publication landscape includes patient perspectives and represents lived experiences. Continued facilitation and strengthening of partnerships between patient and non-patient authors is noted as essential for improving communication, understanding and equity within authorship groups. To support the visibility and recognition of patient authors, they recommend the use of the 'patient author' affiliation metatag to better identify, search, filter and standardise publications with patient involvement, identify patient authors and help build an evidence base from which best practice and guidance can be developed. Additionally, the authors highlight the need to consider and develop guidance around compensation of patient authors to acknowledge the contribution and time commitments across the research process and enable greater diversity, equity and inclusion. Finally, they stress the importance of extending the reach of publications to wider audiences through enhanced accessibility formats and open access.

Health preference research plays a critical role in shaping healthcare policy and decision-making; however the underrepresentation of underserved populations challenges the validity and reliability of preference estimates. Despite efforts to diversify recruitment, health preference studies often have limited demographic diversity and non-representative sampling, leading to potentially biased findings that overlook the preferences of underserved populations. We discuss the importance of engaging underserved populations in health preference research from both ethical and research perspectives. We identify key challenges to the inclusion of underserved groups and outline strategies to address them, illustrating these with examples where possible. By prioritising inclusive and flexible methodologies, health preference researchers can generate more representative data, ensuring that estimates reflect the diverse needs and values of all populations. Ultimately, these efforts will support the development of more equitable, evidence-based, and impactful healthcare policies.