The Patient - Patient-Centered Outcomes Research最新文献

Background

As we adopt value models to inform drug reimbursement, coverage, clinical trials, and treatment choices, aligning these models to reflect patient values and preferences becomes increasingly relevant. In this study, we focus on colorectal cancer (CRC), which is highly prevalent and a leading cause of death in Canada, and new drug treatment options are costly.

Objective

The aim of this study was to understand how the values and experiences of people with CRC and their caregivers inform their perspectives about new and emerging colorectal cancer drug treatments.

Methods

We applied qualitative methods to identify key personal, social, and system factors about how the values of people with CRC and their caregivers’ values influence their treatment decision-making in a more holistic manner. Canadian adults (>18 years) living with non-metastatic or metastatic CRC and caregivers were recruited from oncology clinics and Colorectal Cancer Canada (CCC) using purposive sampling. Participants engaged in structured interviews by telephone. Interviews were transcribed verbatim and analyzed thematically guided by a qualitative phenomenological approach and Sherwin’s ethical theory of relational autonomy using NVivo software.

Results

We conducted structured interviews with 12 people with CRC and six of their caregivers, and elicited patient and caregiver values and their influence on treatment decision-making context of key personal, social, and system factors. Thematic analysis of transcripts led to the development of four overarching and intersecting themes that were identified as influencing people with CRC and their caregivers’ treatment decision-making: treatment outcomes and effectiveness, intrapersonal and interpersonal factors, quality of life, and survivorship and prognosis.

Discussion

Our findings suggest intersecting influences of patient-, community-, and social network-, and systemic-level factors that influence patients’ decisions on treatment. Perceived clinical benefit, requirements of treatment, available information, the impact of treatment on social relationships and daily life, and the impact of social support were key factors described by participants. To our knowledge, this is the first study to utilize the theory of relational autonomy to understand patient and caregiver values in the context of treatment decisions in CRC. Using these findings, a continued exploration of people with CRC’s values in treatment decision-making and how much patients value or weight the different aspects of treatment would help further advance patient care and guide healthcare system decision-making.

As the pharmaceutical industry advances towards more patient-focused product development, it is well recognized that meaningful patient engagement is required for the authentic patient voice to inform research and regulatory decisions. However, for this to happen systematically and consistently across the industry, there is still a need to evaluate and communicate the value of patient engagement to all stakeholders. Evaluating engagement also informs process improvement, elevating the value further. We describe the development of a conceptual, yet practical, framework for measuring the impact of engagement to achieve this. The framework depicts how metrics can be used to capture and assess the inputs, outputs, and value of patient engagement across the medicines lifecycle. Although conceived in the context of systems and processes within one company, Novartis, the framework was co-created with patient advisors and designed to be both patient-relevant and adaptable for any pharmaceutical organization. The adoption and evolution of the framework will help to demonstrate the value—to patients, healthcare systems, and businesses—of integrating patient engagement into core activities across the medicines lifecycle. We encourage the pharmaceutical industry to apply impact measurement to build a robust evidence base, through measuring, publishing, and communicating the value of patient engagement.

Introduction

Poor adherence to anti-osteoporosis treatment is a well-recognized problem, partly due to misalignment with patient preferences. In recent years, several quantitative preference studies have been conducted. This study aimed to systematically review stated preference research to provide a comprehensive overview of patient preferences in osteoporosis, in particular on conditional relative attribute importance and preference heterogeneity.

Methods

This systematic review was conducted in MEDLINE and Embase up to February 29th, 2024. It includes all English-language, peer-reviewed, stated preference articles related to osteoporosis management and treatment in patients with or at risk of osteoporosis. Conditional relative importance of attributes as well as heterogeneity was assessed, and attributes classified into outcome, process, and cost attributes. Quality assessment was performed using a combined checklist of Purpose, Respondents, Explanation, Findings, and Significance (PREFS) and International Society for Pharmacoeconomics and Outcomes Research (ISPOR) items.

Results

Fourteen studies including 4714 participants were evaluated. Attributes were mostly classified as process related (50%) and outcome related (40%), both of which significantly influence patient preferences. In pairwise attribute comparison, efficacy was dominant over cost, administration, and side-effects. Preference heterogeneity was observed in the majority of studies (86%). Quality assessment indicated an overall improvement in study quality over time, with recent studies adhering more closely to established methodological standards.

Conclusions

The findings highlight the importance of considering patient preferences in the management of osteoporosis, underscoring the need for a patient-centered approach. The readiness of patients to engage in trade-offs between attributes suggests that healthcare providers should ensure treatments are aligned with individual patient preferences to improve adherence and optimize outcomes.

Background

With the aim to optimize communication during HPV vaccination campaigns in France, we elicited parental preferences around HPV vaccination.

Methods

We conducted a single-profile discrete choice experiment (DCE) among parents of 11- to 14-year-old middle-school pupils, who completed an anonymous, self-administered, internet-based questionnaire during 2020–2021. The DCE comprised five attributes (vaccine-preventable disease, justification of optimal age, information on safety, indirect protection and coverage) of vaccination against an unnamed disease that were presented to respondents in ten choice tasks, or scenarios. We use fixed effect logit models to estimate attribute weights on theoretical vaccine acceptance, and random effect linear regression to estimate attribute coefficients on vaccine eagerness (decision and decision certainty). We estimated marginal effects of attributes on expected vaccine acceptance.

Results

Vaccination scenarios were accepted by 55.6–89.2% of the 1291 participants. The largest marginal effects on expected vaccine acceptance in the full sample arose from prevention of cancer versus genital warts (+ 11.3 percentage points); from a “severe side effect suspicion that was not scientifically confirmed” versus a statement about “more benefits than risks” (+ 8.9 percentage points), and information on 80% vaccine coverage in neighbouring countries versus on “insufficient coverage” (+ 4.2 percentage points). Explaining the early age of vaccination by sexual debut had a strong negative impact among French monolingual parents with lower education level (vs age-independent, OR 0.48, 95% CI 0.27–0.86), but not other socio-economic groups. After removing low-quality responses (unvaried certainty and short questionnaire completion), among serial non-demanders with children not vaccinated against HPV, only disease elimination impacted vaccine eagerness positively (coefficient 0.54, 0.06–1.02).

Discussion

Using DCEs to elicit parents’ preferences around communication messages, notably on cancer prevention, vaccine coverage and information about vaccine safety, could help to optimize HPV vaccination promotion efforts.

This paper focuses on survey administration and data collection methods employed for stated-preference studies in health applications. First, it describes different types of survey administration methods, encompassing web-based surveys, face-to-face (in-person) surveys, and mail surveys. Second, the concept of sampling frames is introduced, clarifying distinctions between the target population and survey frame population. The discussion then extends to different types of sampling methods, such as probability and non-probability sampling, along with an evaluation of potential issues associated with different sampling methods within the context of health preference research. Third, the paper provides information about different recruitment methods, including web-surveys, leveraging patient groups, and in-clinic recruitment. Fourth, a crucial aspect addressed is the calculation of response rate, with insights into determining an adequate response rate and strategies to improve response rates in stated-preference surveys. Lastly, the paper concludes by discussing data management plans and suggesting insights for future research in this field. In summary, this paper examines the nuanced aspects of survey administration and data collection methods in stated-preference studies, offering valuable guidance for researchers and practitioners in the health domain.

Purpose

To quantify the preferences for an oral antidiabetic drug (OAD) among patients with type 2 diabetes mellitus (T2DM) in China.

Methods

A discrete choice experiment (DCE) with hypothetical OAD profiles was performed among patients with T2DM recruited from both online and offline sources. Each patient completed 12 DCE choice tasks. The attributes, elicited through mixed methods, include blood glucose level decrease, blood glucose level stability, frequency of medication, gastrointestinal side effects, dose adjustment and out-of-pocket expense. The conditional logit regression model was used to analyze the data. Patients’ willingness-to-pay (WTP) was also calculated. Subgroup analyses based on patient characteristics were also conducted.

Results

A total of 741 respondents were included in the analysis sample, covering 456 respondents online and 285 offline. The result showed that all attributes and levels were statistically significant, except one level “dose adjustment required for patients with hepatic or renal insufficiency” in the attribute of dose adjustment. WTP results showed that patients were willing to pay 12.06 and 23.20 yuan, respectively to reduce the frequency of medication from “once per day” and “three times per day” to “once every 2 weeks”, respectively. Subgroup analyses showed that the frequency of medication (once versus two to three times per day) had the largest impact and influenced most coefficient estimates.

Conclusion

The results suggest that Chinese patients with T2DM prioritized better efficacy, less frequency of medication, lower gastrointestinal side effects, no dose adjustment required for patients with hepatic or renal insufficiency, and less out-of-pocket expense of OAD treatment.

The interest in quantifying stated preferences for health and healthcare continues to grow, as does the technology available to support and improve health preference studies. Technological advancements in the last two decades have implications and opportunities for preference researchers designing, administering, analysing, interpreting and applying the results of stated preference surveys. In this paper, we summarise selected technologies and how these can benefit a preference study. We discuss empirical evaluations of the technology in preference research, with examples from health where possible. The technologies reviewed include serious games, virtual reality, eye tracking, innovative formats and decision aids with values clarification components. We conclude with a critical reflection on the benefits and limitations of implementing (often costly) technology alongside stated preference studies.

Background

Malignant pleural mesothelioma (MPM) is a rare and usually fatal malignancy frequently linked to occupational asbestos exposures and associated with poor prognosis and considerable humanistic burden. The study aimed to develop conceptual models of the health-related quality of life (HRQoL) impact on patients with and receiving treatment for MPM, and the burden on their caregivers.

Methods

This multi-country study (Australia and United Kingdom) adopted a qualitative methodology to conduct semi-structured, independent interviews with people with MPM (n = 26), current caregivers (n = 20), and caregivers of people who had recently died because of MPM (n = 4). Participants were recruited using a purposive sampling approach and interviews conducted via telephone between January 2021 and January 2022. Transcripts were analysed using thematic analysis and used to construct conceptual models.

Results

Patient analysis yielded four overarching themes: (1) debilitating burden of breathlessness and fatigue; (2) physical mesothelioma symptoms experienced by patients; (3) distress of MPM on the self and family; and (4) treatment is worth ‘having a go’ despite the potential impact on symptoms. Caregiver analysis yielded five core themes: (1) daily life limited by caregiving duties; (2) emotional well-being and the need for support; (3) the relational role shift to caregiver; (4) time spent providing care negatively impacts work and productivity; and (5) positive aspects and outcomes of caregiving.

Conclusions

This study highlights the substantial daily and emotional HRQoL impact that MPM symptoms have on patients and caregivers. Both groups reduced work, productivity, and social and leisure activities. There was evidence of positive HRQoL impacts as a result of immunotherapy and radiotherapy, but less for chemotherapy. Caregiver impacts were intensified during the end-of-life period and persisted following patient death. Evident is a need for increased psychological support, information, and advice for caregivers, increased during the end-of-life period.

Background

It is essential to consider the evidence of consumer preferences and their specific needs when determining which strategies to use to improve patient attendance at scheduled healthcare appointments.

Objectives

This study aimed to identify key attributes and elicit healthcare consumer preferences for a healthcare appointment reminder system.

Methods

A discrete choice experiment was conducted in a general Australian population sample. The respondents were asked to choose between three options: their preferred reminder (A or B) or a ‘neither’ option. Attributes were developed through a literature review and an expert panel discussion. Reminder options were defined by four attributes: modality, timing, content and interactivity. Multinomial logit and mixed multinomial logit models were estimated to approximate individual preferences for these attributes. A scenario analysis was performed to estimate the likelihood of choosing different reminder systems.

Results

Respondents (n = 361) indicated a significant preference for an appointment reminder to be delivered via a text message (β = 2.42, p < 0.001) less than 3 days before the appointment (β = 0.99, p < 0.001), with basic details including the appointment cost (β = 0.13, p < 0.10), and where there is the ability to cancel or modify the appointment (β = 1.36, p < 0.001). A scenario analysis showed that the likelihood of choosing an appointment reminder system with these characteristics would be 97%.

Conclusions

Our findings provide evidence on how healthcare consumers trade-off between different characteristics of reminder systems, which may be valuable to inform current or future systems. Future studies may focus on exploring the effectiveness of using patient-preferred reminders alongside other mitigation strategies used by providers.

Background and objective

The history of clinical trials is fraught with unethical practices. Since 1945, robust frameworks have evolved to standardise the collection and reporting of safety data, most notably, the Common Terminology Criteria for Adverse Events (CTCAE) from the National Cancer Institute; used by investigators to report side effects experienced by participants. As medicine moves into the patient-centred model, interest has been growing to collect data on adverse events directly from participants (patient-reported adverse events). The aim of this systematic scoping review was to investigate the inclusion of patient-reported adverse event data within safety/tolerability analyses and explore the collection and reporting of patient-reported adverse event data.

Methods and results

A database search was undertaken and the Covidence platform was used to manage the review; results were analysed descriptively. Sixty-eight studies were included in the analysis. An increase in the number of studies that incorporate patient-reported adverse event data was seen by year. Seventy instruments were used for the collection of patient-reported adverse event data with recall period, mode, frequency and site of administration varying across studies; the duration of data collection ranged from 28 days to 6 years. Frequently, information on these details was omitted from publications. The number of instruments used by studies to collect patient-reported adverse event data ranged from one to seven instruments.

Conclusions

Despite growing calls for the inclusion of patient-reported adverse events, this has not yet translated into published reports. The collection and reporting of these data were variable and conducted using instruments that were not designed for purpose. To address these inconsistencies, standardisation of data collection and reporting using a purpose-built validated instrument is required.