Patient-Patient Centered Outcomes Research最新文献

This research explores the growing importance of qualitative in-trial research (ITR) in regulatory and health technology assessment (HTA) decision making. Since 2020, the Food and Drug Administration (FDA) and European Medicines Agency (EMA) have emphasized incorporating patient experience data into drug development and approval. We reviewed regulatory guidance documents, drug approval records, and HTA reports from January 2017 to March 2025. This included labels and reviews for new drug applications from the FDA and EMA, the Patient-Focused Drug Development guidance series, guidance on rare diseases, oncology, and gene therapy, and methodological guidance from HTA bodies in Scotland, the UK, France, Germany, Italy, and Spain. From more than 1000 drug applications assessed by both regulatory agencies, only ten and eight products (from the FDA and EMA, respectively) included ITR, with 55% of these for rare diseases. Both agencies used ITR data to gain insights into symptoms and patient experiences, the relevance of patient-reported outcome concepts, and meaningful changes in symptoms or treatment benefits; and to support the interpretation of meaningful score or endpoint changes. Two products included ITR data in both FDA and EMA reviews/labels. Three HTA bodies published guidance documents on qualitative research, with only two products out of eight reviewed including qualitative data in HTA reports.Despite increasing use, ITR in regulatory submissions and HTA reviews remains limited. Early planning and alignment of ITR objectives with regulatory and HTA requirements are needed to enhance the relevance and impact of qualitative evidence in drug development and healthcare decision making.

Background: Although there are many long-term prophylactic treatments available for hereditary angioedema, few studies have assessed patient preferences for these treatments.

Objective: We aimed to assess patient preferences for long-term prophylactic treatment attributes, including treatment trade-offs and the likelihood of starting or switching to a new treatment.

Methods: An online discrete-choice experiment survey instrument was developed and administered to adults in the USA with a self-reported diagnosis of hereditary angioedema. Respondents evaluated a series of choices between pairs of hypothetical long-term prophylactic hereditary angioedema treatment alternatives.

Results: A total of 250 respondents (81.6% female; mean [standard deviation] age, 39 [11] years) completed the survey. Respondents placed the most importance on a reduction in attack frequency (conditional relative attribute importance = 31.7%), a reduced risk of a gastrointestinal side effect (conditional relative attribute importance = 18.5%), and treatments taken as an oral tablet compared with injections (conditional relative attribute importance = 18.1%). Respondents were more willing to accept increases in injection-site reactions compared with their willingness to accept gastrointestinal side effects in these trade-offs. A total of 197 respondents (78.8%) stated they were open to starting a new medication with their preferred mode of administration.

Conclusions: A reduction in attack frequency is the most important treatment feature for adults living with hereditary angioedema. The heterogeneity in patient perspectives highlights the need for patient-physician communication when making decisions about initiating a new long-term prophylactic treatment for hereditary angioedema.

Background: Early pregnancy loss (EPL) is associated with significant emotional burden. While multiple management options exist, psychological aspects are often underrepresented in decision-making support. The objective of this study is to identify criteria that are important to women when choosing between expectant, medical, and surgical management of EPL, with particular attention to psychological aspects.

Methods: A mixed-methods study using the Analytic Hierarchy Process (AHP) was conducted. Criteria were identified via literature review and a focus group. Thirty-seven women with EPL history completed structured telephone interviews with pairwise comparisons. Quantitative data were analyzed to calculate criterion weights and assess consistency. Logistic regression explored factors associated with inconsistent responses.

Results: Among 37 participants, 'Minimizing psychological distress' emerged as the most important criterion (local weight = 0.381), with sub-criteria such as 'Experiencing miscarriage as a natural process' (0.384; 0.252) and 'Avoiding hospital treatment' (0.355; 0.425) ranked most important for women preferring expectant and medical management, respectively. Women preferring surgical management prioritized 'Short time to miscarriage completion' (0.389). Sixty-five percent of participants demonstrated acceptable preference consistency (consistency ratio ≤ 0.2). Lower education showed the greatest and statistically significant negative influence on the consistency of the preferences in the regression models.

Conclusion: Preferences varied by treatment choice, indicating that psychological distress is defined differently across women. The AHP method enabled nuanced insights into individual decision-making. Emotional factors may influence preference patterns and should be considered in clinical counselling. These findings support the need for individualized counselling and shared decision-making. The results may inform the development of clinical tools and guidelines.

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: People receiving treatment for advanced cancer invest substantial portions of their survival time receiving healthcare, labelled the 'time toxicity' of treatment. Although qualitative research has examined the impact of time burden on patients and their caregivers, its influence on treatment decision-making is unclear.

Objective: Our objective was to explore treatment decision-making with patients with advanced gastrointestinal cancer, their caregivers, and oncologists, and unmask the role of time burden in those decisions. The objective was to inform the design of a subsequent discrete-choice experiment (DCE) investigating the importance of time burden in treatment decision-making.

Methods: A two-step process was used. Factors relevant to treatment decision-making were discussed as part of semi-structured interviews. Responses were analysed using thematic analysis with a focus on measurable themes relevant to the development of candidate attributes for a DCE. Second, we reviewed stated-preferences studies in the field of treatment decision-making in cancer and compared the results with the candidate attributes identified from interviews.

Results: Interviews with 45 participants (20 patients, 10 caregivers,15 gastrointestinal oncologists; 53% metropolitan) revealed 4 themes and 6 candidate attributes: expected survival benefit of treatment, impact of physical side effects, effect on day-to-day functioning, route of administration, healthcare contact days, and planned length of the treatment course. Review of 45 published studies yielded no additional attributes.

Conclusions: This study identified six candidate attributes for a forthcoming DCE on time burden in advanced cancer care. These findings support growing efforts to quantify and address time toxicity in cancer treatment decision-making.

Background: China faces the world's largest diabetes burden, with a 12.4% prevalence. A wide range of second-line therapies is available for patients with type 2 diabetes mellitus (T2DM), each with distinct characteristics. Although current guidelines advocate for a patient-centered approach in selecting second-line treatments, empirical evidence on Chinese patient's preferences for these therapies remains limited. We therefore elicited population‑level preferences using a Best-Worst Scaling profile‑case (BWS‑2) survey to inform decision making.

Method: A seven-attribute instrument was designed through literature review, expert input, and a BWS object-case study used for attribute refinement (reported elsewhere). The final study employed BWS‑2 to elicit preferences across benefit, risk, and cost attributes. An orthogonal main‑effects design generated the choice sets. The survey was conducted through in-person interviews with patients with T2DM at healthcare institutions in three Chinese regions (eastern, central, and western). Data were analyzed using counting approach, conditional, and mixed logit regression. Preference heterogeneity was explored with subgroup analyses and latent class analysis (LCA).

Results: Among 1517 respondents, counting approach and logit estimates were consistent; most attribute coefficients were statistically significant in logit analysis, with patients showing the highest preference for reduction in HbA1c, cardiovascular protection, and zero out-of-pocket costs compared with the reference levels. Weight change and mode of administration exerted modest influence. LCA identified six unobserved subgroups. Preferences heterogeneity were also explored by splitting the sample according to patient urban-rural residence and income characteristics.

Conclusions: Chinese patients with T2DM prioritized HbA1c reduction, cardiovascular benefits, and cost, while weight change was rated as the least important attribute. LCA further revealed preference heterogeneity, including limited concern for adverse events and neutral attitudes toward all attributes.

Purpose: The benefits and barriers of using patient-reported outcome measures (PROMs) in routine clinical practice have been examined extensively, but there has been limited in-depth exploration of the experience of PROMs by stakeholders and consumers. This study sought to understand the experiences of children with life-altering skin conditions, their caregivers and treating health professionals regarding the routine use of electronic PROMs.

Methods: Using interpretive description, data were gathered through qualitative interviews and researcher observations in two outpatient clinics within a major metropolitan paediatric hospital. In total, 48 interviews were conducted with children (aged 11-16 years) attending burn scar or vascular anomalies clinics, their caregivers (children of all ages) and treating health professionals.

Results: The therapeutic relationship was a central organising concept and crucial for providing person-centred care. Within the therapeutic relationship, ePROMs were used to prioritise where to start alongside a high-quality conversation. Study themes identified were shaping (or not shaping) care, taking a considered approach to the use of ePROMs, and aligning values and priorities with the everyday reality. ePROMs did not shape care or capture the priorities of all children and caregivers. A spectrum of sentiment from negative to positive was expressed by families regarding the routine use of ePROMs, differing to the predominantly positive sentiment by health professionals.

Conclusions: ePROMs should be used alongside high-quality conversations to assist in eliciting, understanding and evaluating what matters to children and caregivers but must be used within a therapeutic relationship.

Objectives: The EuroQol Valuation Technology (EQ-VT) uses composite time trade-off (cTTO) with a 10-year lead-time to value health states worse-than-dead (WTD) ranging between - 1 and 0 (dead). While WTD responses are common in EQ-5D-5L studies, their drivers remain understudied in Asia. This mixed-methods study explored socio-demographic factors and reasoning behind WTD preferences in Singapore.

Methods: We recruited 500 adult Singaporeans using quota sampling. Each participant completed 20 cTTO tasks through computer-assisted interviews, followed by open- and closed-ended questions. Two-part regression models assessed both the likelihood and the extent of WTD ratings, and qualitative content analysis evaluated participants' reasoning for their health state valuations.

Results: Of the 500 participants (mean age 48.1 years; 52.6% had tertiary education), 76.8% identified as Chinese. Overall, 33.0% assigned WTD values, and 16.1% assigned '- 1' values. Age, education, marital status, interviewer and caregiving experience were associated with WTD ratings, though none remained significant in the multivariable analysis. Similar factors were linked to '- 1' values, with middle age being the only factor that remained significant in the multivariable analysis. Qualitative data showed that middle-aged participants often cited worries about imposing physical, mental, or financial burdens on their families.

Conclusions: Preferences for immediate death over living in poor health are common in Singapore, particularly among middle-aged respondents. Concerns about burdening family members appear to drive these preferences, reflecting broader cultural values. These insights may clarify the high frequency of '-1' values in EQ-5D valuation studies across Asia.