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.

Objective: Nearly 30% of kidneys from deceased donors are discarded annually in the USA. A recent study indicated that a significant number of patients would accept lower-quality kidneys to avoid long waits. We expand on previous work to assess how the distribution of patient preferences for lower-quality kidneys would change with patient time on the transplant list.

Methods: We conducted a discrete-choice experiment with US pre-transplant patients waitlisted for kidneys from deceased donors. Respondents were asked to evaluate tradeoffs between expected graft survival and waiting time. We used a logit-based regression with patient covariates to explain membership of three patient-preference phenotypes previously identified with these data. Specifically, we tested the degree to which phenotype membership changed with waiting time and how such changes were moderated by observable patient characteristics such as age, insulin use, recipient function, time on dialysis, and household income.

Results: Waiting time had a nonlinear effect on phenotype probabilities, with more patients expected to be willing to accept lower-quality kidneys as waiting time increases. Patients with longer insulin dependence, lower income, and limited function were more likely to accept lower-quality kidneys. Higher income was significantly associated with the probability of being willing to wait for better future kidneys. Dialysis time had no significant effect.

Conclusions: Our analysis provides insights into time-varying effects using cross-sectional data. Results suggest that patient preferences for organ acceptability vary with waiting time and are moderated by health status and socioeconomic factors. Longer waits and worse health statuses were generally associated with greater willingness to accept lower-quality kidneys.

Background: Patient and public involvement (PPI) is crucial for aligning research with public needs, reducing research waste, and enhancing the relevance and quality of evidence. Evaluating PPI is necessary to ensure its effectiveness. However, despite its recognised importance, researchers have reported a lack of robust tools for evaluating PPI systematically. To clarify which tools are used to evaluate PPI in health research, we conducted a scoping review.

Objective: We aimed to identify and map evaluation tools that have been used in empirical health research studies to assess PPI, and to describe reported outcomes related to PPI.

Methods: A scoping review was conducted in accordance with Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews (PRISMA-ScR) guidelines. A comprehensive search was undertaken in MEDLINE, Embase, CINAHL and Scopus to identify studies published between 2021 and 2024 describing evaluation tools for PPI in health research contexts. Studies evaluating PPI were included, irrespectively of tool validation. Study selection and data charting were guided by principles from structured extraction frameworks and results were synthesised descriptively and narratively.

Results: Thirty studies were included. Positive personal outcomes for PPI partners were reported, including increased well-being and skill development. Despite the existence of robust validated evaluation tools, many were adapted or developed de novo. An 'us vs them' dynamic was noted, reflecting differing engagement levels between PPI partners and researchers during evaluations. The need for additional training for both PPI partners and researchers to enhance collaboration was a recurring theme.

Conclusions: Patient and public involvement evaluation tools are often developed or adapted to fit specific contexts, with multiple methods used for assessment. Challenges include low researcher response rates in evaluations and the need for better researcher preparedness for PPI.

One-to-one interviews and focus groups are used to generate qualitative data about patients' health outcomes and inform the development of patient-reported outcome measures (PROMs). In the development of PROMs for young people, visual elicitation tools can be used with one-to-one interviews and focus groups to enhance the data generation process and data quality. This article aims to (1) provide a detailed description of how visual elicitation tools can be applied in the development of youth-specific PROMs using the GENDER-Q Youth study as an example and (2) share the lessons learned from the GENDER-Q Youth study with PROM developers who are considering using visual elicitation tools in their own virtual qualitative studies. This article discusses processes that took place within the context of a mixed-methods, multi-step study to develop a PROM for youth receiving gender-affirming care called GENDER-Q Youth. Step one was an applied qualitative health research study using an interpretive description approach. Virtual concept elicitation interviews were conducted with transgender and gender diverse youth aged 12-18 years at recruitment using an optional timeline-based visual elicitation tool (i.e., before interviews, youth were invited to create a timeline about their gender-affirming care journeys). The research team navigated ethical and logistical challenges associated with using timeline activities during the data generation process. These challenges occurred during the pre-interview stage (e.g., mailing activity supplies) and during interviews (e.g., incorporating the timeline activity into the interview). Details about the approach used by the research team, challenges faced, and lessons learned are discussed. When conducting one-to-one virtual concept elicitation interviews, visual elicitation tools have the potential to enhance the quality of data generated about participants' outcomes and experiences of healthcare. Visual elicitation tools can also improve the interview experiences of both participants and researchers and are feasible to implement within the context of qualitative PROM development research with young people.

Aim: Patient-perceived treatment tolerability can affect patient ability and willingness to remain on therapy. We sought to examine completion rates for a single item of overall side effect bother at baseline and at the first on-treatment assessment, the association between this item with other patient-reported outcomes (PROs) and the odds of early discontinuation due to clinician-assessed adverse events or reasons other than disease progression.

Methods: Data were from three commercial cancer trials in solid tumours, focusing on the safety population. The GP5 item from the Functional Assessment of Cancer Therapy (FACT) was used for side effect bother. Other PROs included items on specific symptoms, functional impacts and global health status, all drawn from validated measures. Descriptive statistics were used for completion rates, and correlation and logistic regression analyses were used to examine associations. GP5 was dichotomised as 0-1 ('low') versus 2-4 ('high').

Results: Completion rates were at or above 90% at baseline for all items. GP5 completion rates were 5% lower than completion rates for other items (89.8% versus 94.9%) at baseline, but this was not seen after baseline. Among patients with non-missing baseline GP5, 11.8-15.7% of cancer treatment-naïve patients reported high bother, compared with 23.9% of treatment-experienced patients. Patients with high bother at baseline had higher odds of early discontinuation compared with those with low bother, but this was not statistically significant after covariate adjustment.

Conclusions: Continued collection of the GP5 item and concomitant work aiming to understand reasons for missingness as well as interpretation is important for evaluating tolerability in cancer trials.