Patient-Patient Centered Outcomes Research最新文献

Background and objectives: Amyotrophic lateral sclerosis (ALS) is a rapidly progressive and fatal neurological disease that leads to death within 2-5 years of diagnosis for more than 80% of people living with ALS (PLWALS). The American Academy of Neurology (AAN) developed practice parameters-general principles to guide clinicians in managing ALS-encouraging multidisciplinary care (MDC) but does not recommend specific healthcare delivery models. Three healthcare delivery models have evolved: a traditional model, a triage model, and a non-triage model. This study aims to describe preferences for and satisfaction with various models, among PLWALS and their caregivers (CALS), along with their perceptions of how their care aligns with AAN guidelines.

Methods: A cross-sectional observational study utilizing a web-based survey was distributed via email to PLWALS and CALS. Three multi-assessment questionnaires were developed and tailored for PLWALS, CALS, and former CALS. Best-worst scaling (object case) data were analyzed using a best-minus-worst approach and descriptive statistics were calculated from means, t-tests and chi-square.

Results: The combined sample included 378 respondents: 254 PLWALS (67.20%) and 124 CALS (32.80%; composed of 79 current caregivers [20.90%] and 45 former caregivers [11.90%]). The mean respondent age was 61.09 years (SD 11.1). The majority of the sample was white (92.86%), insured by Medicare (61.11%), and married/partnered (79.10%). Respondents preferred a non-triage model the most and a traditional model the least; 88.20% (CI: 84.92-91.49) were extremely likely to choose a non-triage model if given the choice and 83.12% (CI: 79.29-86.92) of respondents ranked non-triage as most preferred. A traditional model was ranked as the least preferred model in 75.28% (CI: 70.78-79.78) of respondents. The most important factors driving respondent preferences were ALS expertise and team-based care. Overall, respondents are satisfied with their care teams. PLWALS utilizing non-triage MDC models reported more adherence to quality care measures compared with those utilizing triage and traditional models.

Discussion: Respondent preference for non-triage models is consistent with the importance they place on the features of non-triage models. However, these findings should be understood in the context of our sample in which a large majority of respondents were receiving care via a non-triage model. To ensure ALS care delivery is patient-centered, practice parameters that aim to guide clinicians in managing ALS should provide more guidance to MDCs to deliver care aligned with patient preferences and values. Efforts should focus on sustainable financial models that can better facilitate non-triage models of care.

Background and objective: Anti-vascular endothelial growth factor agents are the first-line treatment for macular edema associated with neovascularization or increased vascular permeability, yet patient adherence remains suboptimal. This study aimed to quantify how patient preferences for therapy-related attributes of anti-vascular endothelial growth factor agents influence trade-offs among effectiveness, safety, required injection regimen, and cost.

Methods: A face-to-face survey-based discrete choice experiment was conducted across six Chinese cities from October 2023 to April 2024 among adults with diabetic macular edema or neovascular age-related macular degeneration. Two separate discrete choice experiment sets were developed based on literature review, focus groups, best-worst scaling, and reference to package inserts and clinical trial data. Each patient was asked to choose a preferred alternative from each of the ten choice sets. Mixed logit models estimated patient preference weights for each attribute level, relative attribute importance, and trade-offs between benefits, risks, injection regimens, and cost. Latent class models explored preference heterogeneity.

Results: A total of 355 participants who passed quality checks, most preferred anti-vascular endothelial growth factor agents with longer or more flexible maintenance-phase injection intervals, greater macular edema reduction, improved visual acuity, lower risk of serious adverse events, and lower cost. For patients with diabetic macular edema, injection frequency was the most important attribute. They were willing to pay an additional ¥5045.26 ($708.60) to extend injection intervals from monthly to treat-and-extend regimen, or accept a 12.97% increased risk, or forgo a 21.76% macular edema reduction, or a 14.76% visual acuity improvement. For patients with neovascular age-related macular degeneration, visual acuity improvement was the most important. They were willing to pay ¥16,586.29 ($2329.54) to improve visual acuity from 20% to 43%, or accept a 17.30% increased risk, or forgo an 18.64% macular edema reduction, or an 11.88% visual acuity improvement. Patients with diabetic macular edema with senior high school education or above, or with cross-region treatment history, placed greater emphasis on visual acuity improvement, while those with more than three prior injections prioritized lower injection frequency.

Conclusions: Eliciting individual preferences can support ophthalmologists in making patient-centered recommendations during shared decision making. Understanding trade-offs across different patients is also valuable for pharmaceutical companies and payers. Future research should further explore the balance between cost, risk, and potential benefits that patients are willing to accept.

Background: Hypertension is a major risk factor for fatal and nonfatal cerebrovascular and cardiovascular events. Poor treatment adherence, a key measure impeding successful treatment, can be improved by incorporating patient preferences. We aimed to identify patient preferences for hypertension treatment outcomes by performing a quantitative exploratory field study using the analytic hierarchy process (AHP). We also explored the associations between patient preferences with prior cardiovascular and cerebrovascular events, sociodemographic factors, and health-related quality of life (HRQoL).

Methods: We quantified preferred outcomes by pairwise comparisons among individuals with hypertension, recruited in secondary and tertiary care settings. Individual weights were calculated using the eigenvector method. HRQoL was assessed via the EQ-5D-5L instrument, including its visual analogue scale.

Results: Data from 263 participants revealed that stroke (weight: 0.271) and death (0.270) were equally prioritized as the most worrisome hypertension treatment outcomes, followed by myocardial infarction (0.191), acute heart failure (0.176), and common adverse drug reactions (ADRs) (0.091). Among ADR, dyspnea was consistently ranked as the most concerning. Stratified analyses revealed variations in outcome prioritization: patients with prior stroke or acute heart failure strongly prioritized stroke avoidance, whereas those with myocardial infarction or multiple events emphasized avoiding death.

Conclusions: This is the first study to apply AHP to hypertension treatment preferences, identifying stroke and death as equally worrisome outcomes, clearly prioritized for avoidance. Thus, prolonging life is not universally the primary goal among patients. Integrating patient preferences into clinical decision-making may support a more patient-centered approach and improve treatment adherence and outcomes.

Background and objective: Atrial fibrillation is the most common sustained cardiac arrhythmia and is associated with an increased risk of stroke, heart failure and death. Genetic factors can contribute to atrial fibrillation pathogenesis and have the potential for atrial fibrillation risk stratification and clinical management. Using stated preference methods, this paper provides the first empirical evidence on consumer preferences, values and priorities for genomic testing in atrial fibrillation.

Methods: Two discrete choice experiment surveys were designed and administered to the Australian public. Participants were asked to imagine situations where they either developed atrial fibrillation symptoms (symptomatic patients survey, n = 503) or had close family members diagnosed with atrial fibrillation (at-risk relatives survey, n = 506). Each participant was given 12 hypothetical scenarios involving five key aspects of genomic testing. Choice data were analysed using panel error component mixed logit and latent class models.

Results: The two most valued benefits were clinical implications for monitoring personal cardiac complications other than atrial fibrillation and health implications for other family members. Cost was the main driver of preferences for testing. The value of genomic testing was estimated at $2900 for symptomatic patients and approximately 10% less for at-risk relatives. Uptake was predicted at around 82% for both groups if the test was publicly funded. People of lower financial or educational status and people without private health insurance were less likely to take-up genomic testing.

Conclusions: Genomic testing for atrial fibrillation has perceived value for symptomatic patients and at-risk relatives. Tailored educational programmes and targeted support are essential to improve access for socioeconomically disadvantaged groups.

Objective: Ensuring therapies align with women's expectations can improve adherence and is crucial for the effectiveness and safety of menopausal treatments, especially in light of the concerns raised after the Women's Health Initiative study. This systematic review aims to provide an overview of studies reporting data on women's preferences for menopausal symptoms and treatments to better guide research and development of therapies.

Design: A systematic literature review was conducted on Medline (via Ovid) and Embase. This review used a structured search strategy to identify all quantitative stated-preference evidence on menopausal symptoms or treatments, including conjoint analysis, discrete choice experiment, best-worst scaling, or quantitative preference survey, published before April 2025. A manual search complemented the process. The quality of the included conjoint analyses was assessed using the Discrete Choice Experiments Reporting Checklist (DIRECT), and quantitative surveys were evaluated using the Consensus-Based Checklist for Reporting of Survey Studies (CROSS) checklist. The whole conduct of the systematic review was performed in adherence to the Preferred Reporting Items for Systematic Review and Meta-analysis (PRISMA) 2020 statement (PROSPERO registration no. CRD42024614218).

Results: In total, 606 references were screened after removing duplicates, and 7 studies were included (4 conjoint analyses and 3 quantitative surveys). Five studies were conducted in the USA, one in Sweden, and another was conducted across five European countries and the USA. Two main topics were covered: (1) preferences regarding menopausal symptoms and treatments and (2) preferences toward local estrogen therapies. First, regarding preferences for menopausal treatment, the most important attribute categories were efficacy and long-term safety. Limited data were provided on other treatment characteristics. Studies reported that women are dissatisfied with their current therapeutic options. Second, two studies focused on local estrogen therapies and revealed a preference for vaginal tablets with applicators over other treatment modalities.

Conclusions: Research on menopausal treatment preferences is currently limited. Diverse study designs hampered the comparison and synthesis of the results. There is a need to conduct further conjoint analyses, such as discrete choice experiments (DCE), to better understand women's preferences and improve patient management and treatment options.

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.