Patient-Patient Centered Outcomes Research最新文献

Cognitive interviewing is a technique that can be used to improve and refine questionnaire items. Cognitive interviewing is now a commonly used method to improve the reliability and validity of clinical outcome assessment instruments by identifying problems respondents have in understanding and answering the draft questionnaire items, and then to revise the items to improve understanding and response accuracy. Although practitioners are in general agreement regarding the basic principles and aims of the cognitive interview, there is considerable variation in practitioners' approaches. The aim of this paper is to put forward a few recommendations regarding best practice in the application of cognitive interviews as part of the developmental process of clinical outcome assessment instruments. The paper is structured in order that each section addresses a discrete and key element of the cognitive interview process that ranges from selecting the appropriate methodology, development of the interview guide, interviewer recruitment and training, and determining sample size through to approaches for analysis and writing the cognitive interview report. Each element of the cognitive interview process is divided into (1) the rational underpinning the methodology; (2) the recommended procedural steps; and (3) an overview of lessons learnt from previous research.

Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematological disease, with symptoms including fatigue, difficulty breathing, and hemoglobinuria, which negatively affect health-related quality of life.

Objective: In-trial interviews with patients with PNH, enrolled across three clinical trials, were conducted to understand patient experiences and satisfaction after receiving iptacopan, a novel treatment for PNH.

Methods: Adult participants with PNH were recruited and consented into the qualitative interviews across three phase II/III iptacopan trials (NCT04558918, NCT04820530, and NCT04747613). Interview documents were developed and approved by the relevant ethics authorities in target countries. Trained qualitative interviewers used a semi-structured interview guide to elicit information on PNH-related symptoms and impacts participants experienced prior to the trial, how these changed after receiving iptacopan, and their treatment experience and satisfaction with iptacopan.

Results: Interviews were conducted with 61 participants in eight countries. Participants reported a broad range of symptoms and impacts associated with PNH, including fatigue, tiredness, frustration, difficulty doing usual activities, and limitation to social activities. Interview participants reported improvements in the signs, symptoms, and impacts of PNH after receiving iptacopan, and the majority of participants considered those improvements to be meaningful. Participants reported being either "very satisfied" or "satisfied" with their treatment experience of iptacopan.

Conclusion: Findings from these interviews provide valuable patient-reported data on the positive treatment experience and meaningful improvements in PNH symptoms and health-related quality-of-life impacts patients reported after receiving iptacopan. These qualitative reports from patients support and contextualize the positive efficacy results demonstrated in the three iptacopan clinical trials. NCT04558918 (16 Sep 2020), NCT04820530 (25 Mar 2021), NCT04747613 (09 Feb 2021).

Conducting applied qualitative health research studies often involves discussion of sensitive topics that may impact the emotional safety of participants and researchers. While generic guidance exists to support researchers in prioritizing participant and researcher emotional safety, specific considerations for conducting virtual qualitative interviews to develop patient-reported outcome measures (PROMs) remain limited. This article provides a framework to support PROM developers in prioritizing participant and researcher emotional safety when conducting virtual qualitative interviews. This framework is informed by the strategies developed and applied in the GENDER-Q Youth study, an interpretive descriptive study to develop a PROM for youth receiving gender-affirming care (GENDER-Q Youth). The GENDER-Q Youth study involved virtual concept elicitation interviews with transgender and gender diverse youth (aged 12 years and older) to understand important care-related experiences and outcomes. The interview data were then used to develop draft scales. Virtual cognitive debriefing interviews were conducted with concept elicitation participants to obtain feedback on the draft scales. Strategies to promote participant and researcher emotional safety were developed and implemented throughout data generation (i.e., concept elicitation and cognitive debriefing interviews) and data analysis. On the basis of knowledge gained from creating and applying safety strategies in the GENDER-Q Youth study, a framework was developed to support researchers in prioritizing participant and researcher emotional safety when conducting their respective virtual PROM development studies. This framework offers considerations to support researchers before data generation (e.g., scheduling interviews when support will be available, should an emotional safety concern arise), during data generation (e.g., conducting check-ins with participants), after data generation (e.g., providing opportunities for the interviewing researcher to debrief), and during data analysis (e.g., conducting check-ins with research team members). This framework can help PROM developers identify threats to emotional safety that may occur before, during, and after virtual data generation and during data analysis and facilitate the development of strategies and plans to mitigate these risks.

Background: Advanced cell therapies, including chimeric antigen receptor T cell (CAR T cell) therapies, offer novel opportunities for the treatment of advanced blood cancers such as lymphoma and leukaemia. However, as these therapies are relatively new, there is limited information on the experiences of patients and informal caregivers of the treatment which may influence the uptake of these therapies. The aim of this qualitative study was to explore their experiences to facilitate the identification of specific issues that should be addressed to positively impact patient outcomes and experiences of care.

Methods: We conducted semi-structured qualitative interviews with 26 CAR T cell recipients from four UK CAR T cell centres who were ≥ 1 month post-treatment and nine caregivers. Interviews explored participants' experiences of CAR T cell therapy and perspectives on key issues. Data from the interviews were inductively coded using thematic analysis.

Results: The main themes identified included (1) opinions of information provision, (2) experiences of protective isolation, (3) impacts of illness and treatment, (4) reflections on support needed and received, and (5) role of charities and support groups. Participants highlighted the need for support specifically for caregivers and provided practical suggestions for improving the experiences of future patients.

Conclusions: Interventions to address the issues raised need to be co-developed with key stakeholders, including patients, caregivers, healthcare professionals, policymakers and charitable organisations.

Background: Vasomotor symptoms (VMS; hot flashes) significantly impact women's health-related quality of life during the menopausal transition. Two phase III trials (OASIS 1 and 2) were conducted to investigate the efficacy and safety of elinzanetant for the treatment of moderate-to-severe VMS associated with menopause. This exit interview study explored the impact of VMS on women's mood and work/productivity before and since treatment.

Methods: A total of 40 postmenopausal women from the USA who participated in OASIS 1 and 2 (receiving elinzanetant for 26 weeks or placebo for 12 weeks followed by elinzanetant for 14 weeks) took part in a 60-min exit interview. Interviews were conducted via telephone by trained qualitative interviewers using a semi-structured interview guide; concept-elicitation techniques, followed by focused questioning, were used to explore concepts of interest. Interview transcripts were analyzed using thematic analysis methods in Atlas.ti. Saturation analysis was conducted to determine the appropriateness of the sample size.

Results: Twelve mood concepts and seven work/productivity concepts were reported to be associated with VMS before taking the study medication. Most commonly reported mood concepts included reduced happiness (60.0%), embarrassment (50.0%), and mood swings (45.0%). Most commonly reported work/productivity concepts included reduced concentration (77.5%) and reduced productivity (67.5%). Most participants reported improvements since taking the study medication (mood: ≥ 82.4%; work/productivity: ≥ 80.0%), which contributed to other positive changes (e.g., in social wellbeing). Improvements were considered meaningful (≥ 72.2%) and highly satisfying (≥ 71.4%).

Conclusion: This study provides novel insights into women's experiences of VMS-associated impacts on mood and work/productivity, highlighting the emotional and economic burdens of VMS. Data support and contextualize the treatment benefits of elinzanetant on mood and work/productivity that are meaningful to women.

Preference information describes the relative desirability or acceptability of specified alternatives that differ across health states, interventions, or services. Studies that generate preference information are being designed to support patient-centered decision making across all stages of the medical product lifecycle, as well as in healthcare more generally. Ensuring high-quality preference research with the potential for impact requires transparent and thoughtful study design, a core aspect of which often includes the development of attributes. Good practices for attribute development in preference studies have started to emerge and demonstrate that developing attributes requires substantial time and effort. Resources to more easily and systematically identify potentially relevant attributes may support the accessibility, interoperability, and reusability of attributes, in turn improving the efficiency of preference study design and comparability of findings across studies. In this paper, we first describe the need for and potential benefit of tools that promote the purposeful re-use of attributes for preference studies. We next present a taxonomy for categorizing and describing attributes that could be applied to facilitate their identification. Finally, we apply this taxonomy to a prototype "attribute library," developed as a part of a Medical Device Innovation Consortium work group, to demonstrate the potential value of these resources to support the preference research community.

Background: Polygenic scores (PGS) capture a proportion of the genomic liability for cancer in unselected and high-risk cohorts, with meaningful application in improving risk-stratified screening and management. However, there are significant evidence gaps regarding future clinical implementation. Despite being key interest-holders, recipient views are underrepresented. The objective of this study was to explore recipients' views on the clinical implementation of PGS for hereditary cancer risk assessment in Australian cancer genetics clinics.

Methods: Three video-conferenced focus groups were conducted with recipients who had been given their breast and ovarian cancer PGS through the PRiMo trial. Nominal Group Technique was used to enable evaluation of implementation determinants and strategies, and priority setting. Descriptive and deductive content analyses were conducted utilising the Consolidated Framework for Implementation Research and the Expert Recommendations for Implementing Change compilation of facilitative strategies.

Results: Participants (N = 10) were female, with an average age of 36 years (range 18-70 years). Of these, 50% (N = 5) experienced a change in their hereditary cancer risk assessment due to their PGS. Participants prioritised the positive value and impact of PGS, and the behavioural characteristics of recipients, notably their knowledge and expectations of PGS and cancer genetics clinics, as major determinants of implementation success. Implementation strategies that prepared and supported recipients to access, engage, and use PGS were emphasised, with a focus on a clear results report, educational resources, in-clinic resources, and delivery of ongoing good clinical follow-up.

Conclusion: Evidence-based strategies should be deployed to address recipients' priority barriers to the clinical implementation of PGS for hereditary cancer risk assessment. Centralising recipient voices in implementation design will improve effectiveness and success.

Patient-reported outcome measures (PROMs) are widely implemented in Wales to achieve Value-Based Healthcare (VBHC). All patient-facing information in Wales must be available bilingually; therefore, Welsh-language PROM translation is necessary. Conceptually equivalent and patient-friendly translations of PROMs are essential to ensure that the data they collect are equivalent to that collected via their corresponding original language versions. As a result, the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) have developed best practice principles for PROM translations. This article reflects on a body of work undertaken over 8 years between 2016 and 2024 to translate a series of PROMs from English to Welsh by attempting to follow these principles. While effective in most areas, they do not provide guidance on reaching an appropriate register in languages with more than one register, dealing with multiple dialects, or how to recruit enough participants for cognitive debriefing when there are limited numbers of native speakers. Lessons have been learned while trying to manage these challenges in Wales, and recommendations for future practice are provided.

Background: Hospital overcrowding in Singapore has reached a critical level, primarily owing to an ageing population and an increased demand for medical services. Alternative care models such as hospital-at-home (H@H) programmes have been introduced to address this issue, utilising telehealth and home-based care. We conducted a choice experiment to determine the most preferred characteristics of a hospital-at-home programme in Singapore.

Methods: A desktop review, interviews with clinicians and an expert panel helped to decide on the attributes and levels of the choice sets. The attributes were the type of care team, communication method, care providers, out-of-pocket costs, additional services, and place of return to the hospital if needed. We obtained information from adult Singaporeans (age > 21 years) admitted to a hospital within the past 2 years using an online panel. The design was a fractional factorial study. A pilot study and a redesign helped to refine the choice experiment. The survey used two unlabelled alternatives.

Results: The total study sample consisted of 602 respondents. The respondents did not have a particular preference for the type of care team, communication method or additional services provider. However, there was a strong preference for having a doctor as part of the care team and being readmitted to a specific ward rather than the emergency department. The analysis of relative importance revealed that out-of-pocket costs (71%) and the composition of care providers (12%) were the most significant attributes in decision-making. We found an anticipated adoption rate of H@H was over 83% compared with inpatient care.

Conclusions: Clinically eligible patients in Singapore are highly likely to accept the H@H care model. An approved private provider could provide the service without reducing patient satisfaction and trust.