Patient Voice Research Articles

“It would have been more constructive if she had given me points to improve on”: Student perceptions of patient feedback in dietetics

Introduction: The voice of the patient has historically been peripheral in many health professional programs. Methods: We investigated the merit of health professional students seeking patient feedback on the effectiveness of their clinical consultations as part of usual clinical placements. Dietetic students (n = 34) formally sought patient feedback after usual consultations (n = 48) then completed a reflective feedback tool. The experience of seeking and receiving patient feedback was then explored through student written narratives, facilitated with a focus group (n = 4). The student reflective forms, and recorded and transcribed focus group, were inductively analysed for themes. Results: Despite the positive perception of the educational task, students did not value the overwhelmingly positive feedback they received from patients, preferring more critical clinical educator feedback. Conclusion: The lack of value attributed to patient perspectives in the learning process raises questions about the success of the current teaching of patient-centred care despite the aspiration to legitimise the patient voice as central to health professional education.

Patient and public involvement and engagement in the development of innovative patient-centric early phase dose-finding trial designs

BackgroundIn light of the FDA’s Project Optimus initiative, there is fresh interest in leveraging Patient-reported Outcome (PRO) data to enhance the assessment of tolerability for investigational therapies within early phase dose-finding oncology trials. Typically, dose escalation in most trial designs is solely reliant on clinician assessed adverse events. Research has shown a disparity between patients and clinicians when assessing whether an investigational therapy is tolerable, leading to the recommendation of potentially intolerable doses for further investigation in subsequent trials.It is also increasingly recognized that patient and public involvement and engagement (PPIE) plays a pivotal role in enriching trial design and conduct. However, to our knowledge, no PPIE has explored the optimal integration of PROs in the development of advanced statistical trial designs within early phase dose-finding oncology trials.MethodsA virtual PPIE session was held with nine participants on 18th October 2023 to discuss the incorporation of PROs within a dose-finding trial design. This cross disciplinary session was developed and led by a team of statisticians, clinical specialists, qualitative experts, and trial methodologists. Following the session, in-depth perspectives were provided by two patient advocates who actively engaged in the PPIE session. We discuss the importance of PPIE in shaping advanced dose-finding trial designs, share insights from patients on integrating PROs to inform treatment tolerability, and present a template for meaningful patient involvement in trial design development.ResultsParticipants generally supported the introduction of PROs within dose-finding trials but showed some apprehensiveness as to how PROs may reduce the size of the recommended dose (and potentially efficacious effect). Some participants shared that they may be reluctant to record the real severity of their symptoms via PROs if it would mean that they would have to discontinue treatment. They discussed that PROs could be used to assess tolerability rather than toxicity of a dose.ConclusionsAmplifying patient voice in the development of patient-centric dose-finding trial designs is now essential. This paper offers an exemplary illustration of how trialists and methodologists can effectively incorporate patient voice in the future development of advanced dose-finding trial designs.

OTHR-22. SUCCESSFUL DEVELOPMENT OF RESOURCES FOR DMG/DIPG PATIENT FAMILIES THROUGH A NETWORK OF FAMILIES, RESEARCHERS, AND CARE TEAMS

Abstract The DIPG community has been severely affected by a lack of resources and awareness due to the relative rarity of the disease. In recent years, this has led to a network of patient families, researchers, and clinicians who have worked together to develop resources that improve clinical care, research, and patient family support. These resources address issues in care equity such as access to clinical trials, accurate information, and consults with leading physicians in the field. These initiatives span across multiple institutions and are predominantly little to no cost for the families. This relationship is mutually beneficial; data from patient families inform researchers and clinicians. In turn, researchers and clinicians use these findings to support other families. Over the last five years, many of the resources developed and/or utilized by the DIPG community have been successful. Since May 2021, 42% of post-mortem tissue donations through Gift From a Child have been DIPG or DMG. Data from this tissue is shared openly through the Children’s Brain Tumor Network, ensuring that a diverse patient population is represented in future research projects. DIPG One-link helps patient families navigate vetted resources for care at any point after diagnosis. Resources include patient navigators, information about research and clinical trials, emotional and grief support, places to safely share their data, and referrals to the DIPG/DMG National Tumor Board. All of these resources have shown an overall increase in use since implementation. There has also been an increase in access by patient populations and care teams that previously would have been unable to access such important resources. The direct involvement of patient families ensures that patient voices are represented and valued. These resources are essential to removing institutional, financial, geographic, and health literacy barriers, and ensuring all future patients have equitable access to care.

PCR90 Patient Voice and Patient Engagement in Value Assessment Frameworks and HTA Decision-Making for Gene Therapies

PCR90 Patient Voice and Patient Engagement in Value Assessment Frameworks and HTA Decision-Making for Gene Therapies

Patient-driven research priorities for patient-centered measurement

BackgroundPatient-centred measurement (PCM) emphasizes a holistic approach wherein the voices of patients are reflected in the standardized use of patient-reported outcome and experience measures and are represented throughout the continuum of measurement activities. Given the challenges of routinely integrating patient self-reports into clinical care decisions, the perspectives of all healthcare system stakeholders, especially patients, is necessary to advance the science of PCM. The purpose of the analysis we report on here was to identify patient-driven research priorities for advancing the science of PCM.MethodsWe analyzed data from seven focus groups that were conducted across British Columbia, Canada and that included a total of 73 patients, using qualitative inductive analysis and constant comparative methods.ResultsWe found that the patients conveyed a desire for PCM to contribute to healthcare decisions, specifically that their individual healthcare needs and related priorities as they see them are always front and centre, guiding all healthcare interactions. The patients’ commentaries highlighted intersecting priorities for research on advancing the science of PCM that would help transform care by (1) enhancing the patient-provider relationship, (2) giving voice to patients’ stories, (3) addressing inclusivity, (4) ensuring psychological safety, (5) improving healthcare services and systems to better meet patient needs, and (6) bolstering healthcare system accountability.ConclusionsThese priorities provide direction for future research efforts that would be positioned to make progress towards better health, better care, and better use of resources for individuals and for society.

Centering marginalized voices in advocacy for equitable policy change in kidney disease.

Inequities in kidney disease are a result of differences in healthcare access and inequitable structural policies that lead to downstream social challenges. An individual with kidney disease sits at the intersection of a variety of governmental and institutional policies that directly affect their access to kidney healthcare and different care delivery models. However, their voice in policy change is often neglected by stakeholders with more structural power. Marginalized individuals with kidney disease are disproportionately affected by kidney disease and inequitable policies can further these health disparities. The review aims to describe how marginalized individuals can be centered in research and lead in advocacy efforts to promote equitable policy change. The marginalized patient voice is critical in advocacy to promote equitable policy change. We discuss examples illustrating research and advocacy methods which center and partner with marginalized communities to catalyze effective policy interventions. Centralizing the patient voice when engaging in advocacy can identify and contextualize the effects of inequitable public policy and improve advocacy efforts.

The Danger of Counter-Transference and Need for Patient Voice in A. M. Homes’s In a Country of Mothers (1993) and Lidia Yuknavitch’s Dora: A Headcase (2012): “Story It”

Abstract A. M. Homes’s In a Country of Mothers (1993) and Lidia Yuknavitch’s Dora: A Headcase (2012) offer fictional representations of the therapeutic process. Each features a female patient who is victimized by a therapist who allows their own counter-transference to prevent the patient’s voice from emerging. Instead of healing, these transferences impose therapist-directed narratives on young women who need to tell their own stories of loss and confusion. After tracing Freud’s changing ideas on transference, this article presents literary examples of counter-transference gone awry. In a Country of Mothers features a therapist who believes her patient is the daughter she gave up for adoption and who uses her own counter-transference to propel a dangerous relationship between the two women; Dora: A Headcase offers a modern-day rewriting of Freud’s “Dora” case study by a teen who resists the counter-transference of her therapist by writing her own story. This examination of literary counter-transference problematizes the supposed neutrality of the therapist and stresses the importance of patient voice in psychotherapeutic healing.

Evaluation of Voice in Patients with Antineutrophil Cytoplasmic Antibody Associated Vasculitides

Evaluation of Voice in Patients with Antineutrophil Cytoplasmic Antibody Associated Vasculitides

Bringing the patient voice to life at this year's conference

This year's British Association of Neuroscience Nurses'conference will be held between 4–5 October, 2024, at Newcastle Upon Tyne, and will be bringing the patient voice to the fore. With two key-note speakers sharing their experiences as ex-patients with head injuries, the conference hopes to provide neuroscience nurses with a better understanding of patient perspective.

Amplifying the Patient's Voice in Oncology Early-Phase Clinical Trials: Solutions to Burdens and Barriers.

Dose-finding oncology trials (DFOTs) provide early access to novel compounds of potential therapeutic benefit in addition to providing critical safety and dosing information. While access to trials for which a patient is eligible remains the largest barrier to enrollment on clinical trials, additional direct and indirect barriers unique to enrollment on DFOTs are often overlooked but worthy of consideration. Direct barriers including financial costs of care, travel and time investments, and logical challenges including correlative study designs are important to bear in mind when developing strategies to facilitate the patient experience on DFOTs. Indirect barriers such as strict eligibility criteria, washout periods, and concomitant medication restrictions should be accounted for during DFOT design to maintain the fidelity of the trial without being overly exclusionary. Involving patients and advocates and incorporating patient-reported outcomes (PROs) throughout the process, from initial DFOT design, through patient recruitment and participation, is critical to informing strategies to minimize identified barriers to offer the benefit of DFOTs to all patients.

The Progress Tracker Breast Cancer Registry: Feasibility of a longitudinal patient-led, patient-reported outcomes (PROM) registry.

e23169 Background: A breast cancer diagnosis has the greatest disability-adjusted life years lost of all cancers and significant impact on physical, psychoemotional, social and overall global functioning. Directed by Breast Cancer Canada, a registered non-profit patient-led organization and data managed by the University of Calgary’s POET Program, PROgress Tracker uses a novel, peer-to-peer support model for recruitment, engagement and retention, and is the first national longitudinal and non-interventional PROMs registry. Our aim is to demonstrate the feasibility and potential of this registry in centering patient voice and lived experience to transform breast cancer management. Methods: PROgress Tracker, with 10-year enrollment goal of 50,000 Canadians with Stage 0-IV breast cancer, extends a series of validated PROMs (PROMIS, BREAST-Q, FACT-B, GAD, PHQ-9, COST-FACIT, ISI and ESASr-CA) via a digital platform every 3 months for up to 10 years. Dynamic customization of additional PROMs (PRO-CTCAE, BREAST-Q) based on patient and treatment-specific trigger questions assess evolving components of wellbeing, including financial stress, work life and adverse treatment effects. Results: Recruitment began in October 2023. 162 participants from all Canadian regions including remote/rural geographic areas have shared baseline demographic and clinical data including stage, genetic/molecular tumor markers, treatment type(s) and completed an initial series of PROMs. Comprehensive PROMs including sensitive topics (finances, sexuality, mental health) were completed with minimal missing data ( < 5%); 3-month follow-up surveys show a current retention rate of 57%. Preliminary analysis: median age of participants 54 yrs, 95% identify as Caucasian, and 57% are currently working. 46% reported financial stress (99% response rate); 63% reported ongoing symptoms requiring follow-up. 6% were BRCA+ and 19% triple negative, 7% experienced recurrence, 3% Stage IV, 75% received systemic and/or radiation therapy, 38% received targeted therapy. Conclusions: The lived experience of breast cancer is integral to patient-centred change in the era of precision/personalized medicine and is captured by PROgress Tracker using a diverse set of periodic PROMs to understand the evolution of global measures of wellbeing over time. Initial data indicates that this novel peer to peer model via digital administration of comprehensive and longitudinal PROMs is feasible. This initiative demonstrates attaining this scope of data is achievable, has interest and participation from the national breast cancer community and growing capacity to accrue important, real-time data to inform best practice and identify additional supports required for breast cancer care. While early feasibility is evident, further outreach and recruitment initiatives will ensure diversity of participants.

Meeting Report for the 2023 International Scientific Conference on Complex Lymphatic Anomalies

Objectives: Complex lymphatic anomalies (CLAs) are rare diseases with variable clinical manifestations caused by the abnormal development of lymphatic vessels. The Lymphatic Malformation Institute (LMI), Lymphangiomatosis & Gorham’s Disease Alliance (LGDA), and LGD Alliance Europe sponsored an international conference on CLAs so leaders in the field could discuss recent advances in research on CLAs. Methods: The conference occurred in Dallas, Texas, between September 29th and September 30th, 2023. The event had 22 distinguished speakers and 38 attendees. Additional researchers, clinicians, and patients attended the conference virtually. Results: In this article, we summarize the key takeaways from the meeting. The conference emphasized the crucial role of the patient voice in guiding research, and attendees heard stories from patients and parents of children with CLAs. The conference covered topics such as the genetic causes of CLAs, preclinical models, clinical advances, new technologies for CLA research, lymphatic imaging, and patient-centered research. Videos of all the presentations can be found on the LGDA website. Conclusions: The meeting revealed that while progress has been made, many challenges still exist surrounding the diagnosis and treatment of CLAs. Sustained research efforts are necessary to fill the gaps in knowledge and improve the care and quality of life of CLA patients.

Moving beyond the 'PPG': co-production and inclusion health.

We frequently fail to meaningfully incorporate patient voice in the development of health services, in particular the voices of those who are most disadvantaged. To share learning from a co-production project to improve primary care experience for those with multiple disadvantage and lived experience of trauma. We formed a collective of women (Bridging Gaps). Group members had lived experience of poverty, mental health challenges, addictions, homelessness, and sexual exploitation. Other members were researchers, GPs, and members of a third-sector organisation. We worked alongside local GPs to change how services were delivered. We collected interviews (n = 9), ethnographic data from group meetings (n = 3), and reflective notes from group members (n = 19). Data on our process were analysed using a framework approach drawn from the principles of trauma-informed care. We highlight the challenges for those with multiple disadvantages and trauma experience to meaningfully and safely engage in traditional Patient Participation Group (PPG) models. True co-production of services requires adequate resources and close collaborative working with local community organisations. Groups must be facilitated by those with relevant experience and the ability to both notice and manage power dynamics within the room. With sufficient support, co-production models have potential to empower group participants and improve health services. Partnership working between GPs, the third sector, and other organisations is vital. This can allow GPs to benefit from the expertise of those with relevant lived experience in tackling health inequalities.

Key Opinion Leaders' Interviews to Inform the Future of Benefit-Risk Planning in the Medical Total Product Life Cycle of Global Pharmaceutical and Medical Device Organizations.

Key opinion leader (KOL) interviews were conducted by the Benefit-Risk Assessment Planning(BRAP) Taskforce to seek expert opinion mainly from industry and regulatory bodies, about the current status and future direction of benefit-risk assessment (BRA) planning in the lifecycle of medical product development. The findings from these interviews are intended to help communication concerning planning for BRA between industry and regulators and shape future guidance. Key opinion leader interviews consisted of 5 questions related to BRA planning, which were administered to volunteers (mainly clinicians and statisticians) within a pool of experienced pharmaceutical and medical device professionals representing academia, industry, regulatory agenciesand a patient group. The interviewees' responses to the 5 questions were summarized. To analyze the qualitative data, a Coding System was developed to label themes arising from the interviews. The key findings from the interviews were summarized into a Master Template. A quantitative analysis based on descriptive statistics was also conducted. Of the 27 interviewees, there were 11 professionals from regulatory agencies, 11 from industry, 4 from academia and 1 from a patient advocacy group. Key findings based on the comments provided by 48% of the interviewees indicated the need of incorporating BRA into other (e.g., existing) processes with the importance of alignment between processes being stressed in the comments provided by 59% of the interviewees. Commencing BRA early in the product lifecycle was emphasized in comments provided by 44% of the interviewees. Among other needs identified were an appropriate contextualization of benefits and risks (based on comments provided by 41% of interviewees) through adoption of an integrated approach with structured support by regulatory agencies and a need for understanding the audience with better communication of benefit-risk (BR) among all stakeholders (based on comments provided by 44% of the interviewees). Almost all comments provided by interviewees (96%) highlighted the importance of utilizing patient experience/preference to guide new product development and BRA. Comments provided by 74% of the interviewees expressed the need to understand patient tolerance for risk and trade-offs, with a majority (78%) of interviewees highlighting how to gather information, and 59% stressing the need for the selection and development of appropriate methodologies as important considerations for enhancing the quality and relevance of the data collected from patients. Interviewees indicated that BRA should commence early in the medical product development and inform decision-making throughout the product lifecycle. Better planning and integration of BRA into existing processes within industry would be valuable. The importance of incorporating the patient voice into BRA and medical product development was emphasized. Other key findings from the KOL interviews included a need for improved communication of BR information, and establishment of methodologies for performing BRA and soliciting patient input.

Rapid survey assessment: Barriers and facilitators to mammogram completion in English and Spanish-speaking patients in a federally qualified health center (FQHC) setting.

1587 Background: Mammography has decreased breast cancer mortality in the US by 40% since the 1980s; yet disparities in screening and outcomes persist in underserved populations. Many studies note language as a barrier to screening with lower mammogram completion amongst Spanish-speaking patients versus those with English language proficiency. At our health center, Spanish-speaking patients were more likely to complete screening mammography compared to patients with English proficiency (53% vs 31% in 2022). To understand this pattern and barriers and facilitators to breast cancer screening, we conducted a mixed methods rapid assessment survey as part of a quality improvement initiative. Methods: We developed a patient bilingual survey measuring knowledge, attitudes, and behaviors about breast cancer prevention and screening. We identified consecutive female patients (age 40-75 years) and extracted baseline demographic data from the electronic medical record. Surveys were distributed via our health center’s patient messaging service and responses stored on a secure platform. Data analysis included descriptive statistics, chi-squared testing between groups and qualitative analysis to identify common themes from free-text answers. Results: The overall response rate was 14% (N=732/5,332) in 30 days. Of the surveys completed, 37% were in Spanish. Spanish-speaking patients more strongly regarded mammography as safe and effective compared to English-speaking patients (p<0.001). Spanish-speaking patients were more likely to view getting a mammogram as a priority (79% vs 65% p<0.001) and noted speaking about breast cancer prevention in their communities more often (p<0.006) as an important factor. Spanish-speaking patients primarily cited cost, scheduling, and language (p<0.05) as barriers to mammogram completion, while English-speaking patients cited time constraints, transportation, and discomfort (p<0.05) more frequently. Spanish-speaking patients were more likely to prefer speaking with a trusted community member or attending health fairs than English-speaking patients (P<0.05). Conclusions: Our study demonstrates differences in attitudes, behaviors, and perceived barriers to mammography in English vs Spanish-speaking patients. The increased mammogram completion rate in Spanish-speaking patients may reflect more positive attitudes about screening and more discussion in the community amongst Spanish-speaking patients queried. Reported barriers to screening differed and will guide targeted QI interventions. We demonstrated feasibility and patient engagement in a diverse population using a message system built into our EHR in an FQHC setting. This survey study allows us to elevate patient voices, better understand gaps and preferences, and better tailor future interventions for cancer screening.

Semiotics of ICU Physicians' Views on End-of-life Care and Quality of Dying in a Critical Care Setting: A Qualitative Study.

While intensive care unit (ICU) mortality rates in India are higher when compared to countries with more resources, fewer patients with clinically futile conditions are subjected to limitation of life-sustaining treatments or given access to palliative care. Although a few surveys and audits have been conducted exploring this phenomenon, the qualitative perspectives of ICU physicians regarding end-of-life care (EOLC) and the quality of dying are yet to be explored. There are 22 eligible consultant-level ICU physicians working in multidisciplinary ICUs were purposively recruited and interviewed. The study data was analyzed using reflexive thematic analysis (RTA) with a critical realist perspective, and the study findings were interpreted using the lens of the semiotic theory that facilitated the development of themes. About four themes were generated. Intensive care unit physicians perceived the quality of dying as respecting patients' and families' choices, fulfilling their needs, providing continued care beyond death, and ensuring family satisfaction. To achieve this, the EOLC process must encompass timely decision-making, communication, treatment guidelines, visitation rights, and trust-building. The contextual challenges were legal concerns, decision-making complexities, cost-related issues, and managing expectations. To improve care, ICU physicians suggested amplifying patient and family voices, building therapeutic relationships, mitigating conflicts, enhancing palliative care services, and training ICU providers in EOLC. Effective management of critically ill patients with life-limiting illnesses in ICUs requires a holistic approach that considers the complex interplay between the EOLC process, its desired outcome, the quality of dying, care context, and the process of meaning-making by ICU physicians. Iyer S, Sonawane RN, Shah J, Salins N. Semiotics of ICU Physicians' Views on End-of-life Care and Quality of Dying in a Critical Care Setting: A Qualitative Study. Indian J Crit Care Med 2024;28(5):424-435.

Ownership of Patient Care: Medical Students’ Expectations, Experiences, and Evolutions Across the Core Clerkship Curriculum

Phenomenon : Ownership of patient care is a key element of professional growth and professional identity formation, but its development among medical students is incompletely understood. Specifically, how attitudes surrounding ownership of patient care develop, what experiences are most influential in shaping them, and how educators can best support this growth are not well known. Therefore, we studied the longitudinal progression of ownership definitions and experiences in medical students across their core clerkship curriculum. Approach : We conducted a series of four longitudinal focus groups with the same cohort of medical students across their core clerkship curriculum. Using workplace learning theory as a sensitizing concept, we conducted semi-structured interviews to explore how definitions, experiences, and influencers of ownership developed and evolved. Results were analyzed inductively using thematic analysis. Findings : Fifteen students participated in four focus groups spanning their core clerkship curriculum. We constructed four themes from responses: (1) students’ definitions of ownership of patient care evolved to include more central roles for themselves and more defined limitations; (2) student conceptions of patient care ownership became more relational and reciprocal over time as they ascribed a more active role to patients; (3) student assessment fostered ownership as an external motivator when it explicitly addressed ownership, but detracted from ownership if it removed students from patient care; and (4) structural and logistical factors impacted students’ ability to display patient care ownership. Insights : Student conceptions of ownership evolved over their core clerkship curriculum to include more patient care responsibility and more meaningful relational connections with patients, including recognizing patients’ agency in this relationship. This progression was contingent on interactions with real patients and students being afforded opportunities to play a meaningful role in their care. Rotation structures and assessment processes are key influencers of care ownership that merit further study, as well as the voice of patients themselves in these relationships.

Inpatients' experiences of falls: A qualitative meta-synthesis.

Identify and synthesize published qualitative research reporting inpatient experiences of a fall to determine novel insights and understandings of this longstanding complex problem. Qualitative meta-synthesis. Online databases were searched to systematically identify published research reporting inpatient experiences of a fall. The included studies were inductively analysed and interpreted then reported as a meta-synthesis. Databases Ovid MEDLINE, Embase, Ovid Emcare, CINAHL Complete, Scopus and ProQuest Dissertations and Theses Global were searched on 3rd August, 2023. From 10 included publications, four new themes of inpatients' experiences of a fall were constructed. Themes one, two and three related to antecedents of patient falls, and theme four related to consequences. Theme one, 'My foot didn't come with me: Physiological and anatomical changes', encompassed patients' experiences of medical conditions, medication, and anatomical changes. These aspects contributed to alterations in balance and strength, and misconceptions of capability in activities of daily (inpatient) living. Theme two, 'I was in a hurry: Help-seeking', encompassed patients' experiences striving for independence while balancing power and control, minimizing their own needs over care of others', and unavailability of support. Theme three, 'I couldn't find the call light: Environment and equipment', encompassed patients' experiences of not being able to reach or use equipment, and environment changes. Theme four, 'It was my fault too: Blame and confidence', encompassed patients' expressions of blame after their fall, blame directed at both themselves and/or others, and impacts on confidence and fear in mobilizing. Inpatient falls are embedded in a complexity of individual, relational, and environmental factors, yet there are potential ways forward both informed and led by the patient's voice. Strength-based approaches to address the tenuous balance between independence and support may be one opportunity to explore as a next step in complementing the existing multifaceted interventions. Inpatient falls are a complex and costly health safety and quality problem. Despite global initiatives in the prevention of inpatient falls, they remain intractable. This meta-synthesis provides an in-depth exploration of extant qualitative data on patients' experiences of falls in hospitals. Four themes were constructed expressing the inpatients' experiences: physiological and anatomical changes, help-seeking, environment and equipment, and blame and confidence. Novel considerations for future investigation are offered, drawing from self-determination theory and positive psychological interventions. This meta-synthesis elicits new considerations for future interventions based on people's experiences of their fall in hospital, offering healthcare professionals novel directions in fall prevention. The review was reported according to the Enhancing transparency in reporting the synthesis of qualitative research statement (ENTREQ; Tong etal., 2012). No Patient or Public Contribution. PROSPERO CRD42023445279.

Determining the minimally clinically important difference for the pediatric liver transplant quality of life questionnaire.

The Pediatric Liver Transplant Quality of Life (PeLTQL) questionnaire is a disease-specific patient reported outcome measure for pediatric liver transplant (LT) recipients. To-date, threshold values above which a change in PeLTQL score is considered meaningful to patients are unavailable. This study proposes the first values for the minimally clinically important difference (MCID) for the PeLTQL. In this retrospective cohort study, anchor and distribution-based methods were used to estimate the MCID for the PeLTQL. Questionnaires completed between March 2013, and July 2022 were included if data from two sequential visits were available. An internal anchor question was used for anchor-based determination of the MCID. A final MCID estimate was ascertained from triangulation of all methods. PeLTQL data from 65 LT recipients (26 [40%] male, 17 [42%] biliary atresia, median age at LT 3.08 years [IQR 0.99-7.30]), and their caregivers were included for analysis. Median patient age at time of baseline PeLTQL completion was 13.84 (10.90-15.86) years. The MCID for self-PeLTQL total scores ranged from 4.53 to 8.46, and from 4.47 to 8.85 for proxy responses. By triangulation, the MCID of the PeLTQL total score was 6.45 and 6.78 for self and proxy responses respectively. A change in PeLTQL score of 6.5 or more points suggests a change in health status that is meaningful to the patient, providing the clinical team an opportunity to engage the patient's voice in reassessing current health status and management strategies.

Giving patients a voice for healthcare reform in Austria: the qualitative voice-study.

Inclusion of patients in healthcare service and system planning is an increasingly important tool to improve healthcare systems worldwide. In 2012, a focused healthcare reform was initiated in Austria to strengthen the primary care sector which is still underway in 2023. The aim of this study was to assess the perceptions, desires, and needs of patients in terms of primary care as a necessary building block of the Austrian healthcare reform. This study was designed as an exploratory qualitative study using semi-structured interviews between the years 2013 and 2018. Interviews with patients focused on positive and negative experiences with regard to general practice (GP) consultations and perceptions of the primary care system in general, as well as desires for improvement. Qualitative content analysis was used to analyse the material using the software atlas.ti. Altogether, 41 interviews were conducted with seven categories identified. These categories include organization and time management around consultation, access, and availability including opening hours, human and professional aspects of consultation, infrastructure and hygiene of the waiting room, healthcare system factors, as well as non-clinical/administrative staff. Appreciating and responding to patients' perceptions and needs, healthcare reform in Austria should include improvements regarding consultation/waiting time, coordination, and navigation in Primary Care. Successful healthcare reform has to include the patient voice.