Patient preferences in genetic newborn screening for rare diseases: study protocol.

Introduction: Rare diseases (RDs) collectively impact over 30 million people in Europe. Most individual conditions have a low prevalence which has resulted in a lack of research and expertise in this field, especially regarding genetic newborn screening (gNBS). There is increasing recognition of the importance of incorporating patients' needs and general public perspectives into the shared decision-making process regarding gNBS.

A socially interdependent choice framework for social influences in healthcare decision-making: a study protocol.

Objectives: Current choice models in healthcare (and beyond) can provide suboptimal predictions of healthcare users' decisions. One reason for such inaccuracy is that standard microeconomic theory assumes that decisions of healthcare users are made in a social vacuum. Healthcare choices, however, can in fact be (entirely) socially determined.

Stated-Preference Survey Design and Testing in Health Applications.

Following the conceptualization of a well-formulated and relevant research question, selection of an appropriate stated-preference method, and related methodological issues, researchers are tasked with developing a survey instrument. A major goal of designing a stated-preference survey for health applications is to elicit high-quality data that reflect thoughtful responses from well-informed respondents. Achieving this goal requires researchers to design engaging surveys that maximize response rates, minimize hypothetical bias, and collect all the necessary information needed to answer the research question.

Can the General Public Be a Proxy for an "At-Risk" Group in a Patient Preference Study? A Disease Prevention Example in Rheumatoid Arthritis.

Background: When selecting samples for patient preference studies, it may be difficult or impractical to recruit participants who are eligible for a particular treatment decision. However, a general public sample may not be an appropriate proxy.

Objective: This study compares preferences for rheumatoid arthritis (RA) preventive treatments between members of the general public and first-degree relatives (FDRs) of confirmed RA patients to assess whether a sample of the general public can be used as a proxy for FDRs.

Comparing Discrete Choice Experiment with Swing Weighting to Estimate Attribute Relative Importance: A Case Study in Lung Cancer Patient Preferences.

Introduction: Discrete choice experiments (DCE) are commonly used to elicit patient preferences and to determine the relative importance of attributes but can be complex and costly to administer. Simpler methods that measure relative importance exist, such as swing weighting with direct rating (SW-DR), but there is little empirical evidence comparing the two. This study aimed to directly compare attribute relative importance rankings and weights elicited using a DCE and SW-DR.

Prosocial Behaviour and Antibiotic Resistance: Evidence from a Discrete Choice Experiment.

Introduction: The health of a community depends on the health of its individuals; therefore, individual health behaviour can implicitly affect the health of the entire community. This is particularly evident in the case of infectious diseases. Because the level of prosociality in a community might determine the effectiveness of health programmes, prosocial behaviour may be a crucial disease-control resource.

Treatment Preferences of Adolescents and Young Adults with Depressive Symptoms: A Discrete Choice Experiment.

Background: Depression in adolescents and young adults is common and causes considerable disease burden while hampering their development, leading to adverse consequences in later life. Although treatment is available, young people are a vulnerable group regarding uptake and completion of treatment. To improve this, insight into youth's preferences for treatment is essential.

Research Priorities to Increase Confidence in and Acceptance of Health Preference Research: What Questions Should be Prioritized Now?

Background And Objective: There has been an increase in the study and use of stated-preference methods to inform medicine development decisions. The objective of this study was to identify prioritized topics and questions relating to health preferences based on the perspective of members of the preference research community.

Methods: Preference research stakeholders from industry, academia, consultancy, health technology assessment/regulatory, and patient organizations were recruited using professional networks and preference-targeted e-mail listservs and surveyed about their perspectives on 19 topics and questions for future studies that would increase acceptance of preference methods and their results by decision makers.

Precision cancer medicine and the doctor-patient relationship: a systematic review and narrative synthesis.

Background: The implementation of precision medicine is likely to have a huge impact on clinical cancer care, while the doctor-patient relationship is a crucial aspect of cancer care that needs to be preserved. This systematic review aimed to map out perceptions and concerns regarding how the implementation of precision medicine will impact the doctor-patient relationship in cancer care so that threats against the doctor-patient relationship can be addressed.

Methods: Electronic databases (Pubmed, Scopus, Web of Science, Social Science Premium Collection) were searched for articles published from January 2010 to December 2021, including qualitative, quantitative, and theoretical methods.

Public Preferences for Digital Health Data Sharing: Discrete Choice Experiment Study in 12 European Countries.

Background: With new technologies, health data can be collected in a variety of different clinical, research, and public health contexts, and then can be used for a range of new purposes. Establishing the public's views about digital health data sharing is essential for policy makers to develop effective harmonization initiatives for digital health data governance at the European level.

Objective: This study investigated public preferences for digital health data sharing.

Patients accept therapy using embryonic stem cells for Parkinson's disease: a discrete choice experiment.

Background: New disease-modifying ways to treat Parkinson's disease (PD) may soon become a reality with intracerebral transplantation of cell products produced from human embryonic stem cells (hESCs). The aim of this study was to assess what factors influence preferences of patients with PD regarding stem-cell based therapies to treat PD in the future.

Methods: Patients with PD were invited to complete a web-based discrete choice experiment to assess the importance of the following attributes: (i) type of treatment, (ii) aim of treatment, (iii) available knowledge of the different types of treatments, (iv) effect on symptoms, and (v) risk for severe side effects.

Maximum Acceptable Risk Estimation Based on a Discrete Choice Experiment and a Probabilistic Threshold Technique.

Objective: We aimed to empirically compare maximum acceptable risk results estimated using both a discrete choice experiment (DCE) and a probabilistic threshold technique (PTT).

Methods: Members of the UK general public (n = 982) completed an online survey including a DCE and a PTT (in random order) measuring their preferences for preventative treatment for rheumatoid arthritis. For the DCE, a Bayesian D-efficient design consisting of four blocks of 15 choice tasks was constructed including six attributes with varying levels.

Discrete choice experiment versus swing-weighting: A head-to-head comparison of diabetic patient preferences for glucose-monitoring devices.

Introduction: Limited evidence exists for how patient preference elicitation methods compare directly. This study compares a discrete choice experiment (DCE) and swing-weighting (SW) by eliciting preferences for glucose-monitoring devices in a population of diabetes patients.

Methods: A sample of Dutch adults with type 1 or 2 diabetes (n = 459) completed an online survey assessing their preferences for glucose-monitoring devices, consisting of both a DCE and a SW exercise.

Balancing benefits and risks in lung cancer therapies: patient preferences for lung cancer treatment alternatives.

Background: In the treatment of Non-Small Cell Lung Cancer (NSCLC) the combination of Immuno- Oncotherapy (IO) and chemotherapy (CT) has been found to be superior to IO or CT alone for patients' survival. Patients and clinicians are confronted with a preference sensitive choice between a more aggressive treatment with a greater negative effect on quality of life versus alternatives that are less effective but have fewer side effects.

Objectives: The aims of this study were to: (a) quantify patients' preferences for relevant attributes related to Immuno-Oncotherapy treatment alternatives, and (b) evaluate the maximum acceptable risk (MAR)/Minimum acceptable benefit (MAB) that patients would accept for treatment alternatives.

Public Preferences for Introducing a COVID-19 Certificate: A Discrete Choice Experiment in the Netherlands.

Objective: Here we investigate public preferences for coronavirus disease 2019 (COVID-19) certificates in the Netherlands, and whether these preferences differ between subgroups in the population.

Methods: A survey including a discrete choice experiment was administered to 1500 members of the adult population of the Netherlands. Each participant was asked to choose between hypothetical COVID-19 certificates that differed in seven attributes: the starting date, and whether the certificate allowed gathering with multiple people, shopping without appointment, visiting bars and restaurants, visiting cinemas and theatres, attending events, and practising indoor sports.

Taking the Shortcut: Simplifying Heuristics in Discrete Choice Experiments.

Health-related discrete choice experiments (DCEs) information can be used to inform decision-making on the development, authorisation, reimbursement and marketing of drugs and devices as well as treatments in clinical practice. Discrete choice experiment is a stated preference method based on random utility theory (RUT), which imposes strong assumptions on respondent choice behaviour. However, respondents may use choice processes that do not adhere to the normative rationality assumptions implied by RUT, applying simplifying decision rules that are more selective in the amount and type of processed information (i.

Quantitative Benefit-Risk Assessment in Medical Product Decision Making: A Good Practices Report of an ISPOR Task Force.

Benefit-risk assessment is commonly conducted by drug and medical device developers and regulators, to evaluate and communicate issues around benefit-risk balance of medical products. Quantitative benefit-risk assessment (qBRA) is a set of techniques that incorporate explicit outcome weighting within a formal analysis to evaluate the benefit-risk balance. This report describes emerging good practices for the 5 main steps of developing qBRAs based on the multicriteria decision analysis process.

Illustrating Emerging Good Practices for Quantitative Benefit-Risk Assessment: A Hypothetical Case Study of Systemic Biologic Treatments for Plaque Psoriasis.

Objectives: Quantitative benefit-risk assessment (qBRA) is a structured process to evaluate the benefit-risk balance of treatment options to support decision making. The ISPOR qBRA Task Force was recently established to provide recommendations for the design, conduct, and reporting of qBRA. This report presents a hypothetical case study illustrating how to apply the Task Force's recommendations toward a qBRA to inform the benefit-risk assessment of brodalumab at the time of initial marketing approval.

The Impact of Video-Based Educational Materials with Voiceovers on Preferences for Glucose Monitoring Technology in Patients with Diabetes: A Randomised Study.

Introduction: Ensuring patients have enough information about healthcare choices prior to completing a preference study is necessary to support the validity of the findings. Patients are commonly informed using text-based information with supporting graphics. Video-based information may be more engaging for the general patient population.

Diabetes patient preferences for glucose-monitoring technologies: results from a discrete choice experiment in Poland and the Netherlands.

Introduction: New glucose-monitoring technologies have different cost-benefit profiles compared with traditional finger-prick tests, resulting in a preference-sensitive situation for patients. This study aimed to assess the relative value adults with diabetes assign to device attributes in two countries.

Research Design And Methods: Adults with type 1 or 2 diabetes from the Netherlands (n=226) and Poland (n=261) completed an online discrete choice experiment.

Acceptable risks of treatments to prevent rheumatoid arthritis among first-degree relatives: demographic and psychological predictors of risk tolerance.

Objectives: To quantify tolerance to risks of preventive treatments among first-degree relatives (FDRs) of patients with rheumatoid arthritis (RA).

Methods: Preventive treatments for RA are under investigation. In a preference survey, adult FDRs assumed a 60% chance of developing RA within 2 years and made choices between no treatment and hypothetical preventive treatment options with a fixed level of benefit (reduction in chance of developing RA from 60% to 20%) and varying levels of risks.