Development of the coreHEM mental health patient-reported outcome measure - A novel mental health outlook measure for people with haemophilia.

Introduction: Currently, no quality-of-life instrument exists that captures the full experience of the mental health outlook (MHO), a coreHEM core outcome, in people with haemophilia, including the potential transformational experience of receiving gene therapy.

Aim: To describe the methods used to develop a content validated patient-reported outcome measure (PROM) that measures MHO for people with haemophilia.

Methods: A conceptual framework, developed from a literature/evidence review, was used to create an interview guide and draft a questionnaire.

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.

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.

Patient Preferences in Pulmonary Arterial Hypertension, a Latent Class Analysis to Identify Preference Heterogeneity.

Objectives: Pulmonary arterial hypertension (PAH) is a chronic, progressive disease of the pulmonary circulation characterized by vascular remodeling that, if untreated, can lead to right heart dysfunction and death. This analysis measured heterogeneity in patient preferences for PAH-specific treatment regimens.

Method: Adult patients with PAH with slight to marked limitations during physical activity were recruited through a patient organization in Germany.

Patient Preferences for Lung Cancer Interception Therapy.

Importance: Interception therapy requires individuals to undergo treatment to prevent a future medical event, but little is known about preferences of individuals at high risk for lung cancer and whether they would be interested in this type of treatment.

Objective: To explore preferences of individuals at high risk for lung cancer for potential interception therapies to reduce this risk.

Design, Setting, And Participants: This survey study used a discrete-choice experiment and included hypothetical lung cancer interception treatments with 4 attributes: reduction in lung cancer risk over 3 years, injection site reaction severity, nonfatal serious infection, and death from serious infection.

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.

A Roadmap for Increasing the Usefulness and Impact of Patient-Preference Studies in Decision Making in Health: A Good Practices Report of an ISPOR Task Force.

Many qualitative and quantitative methods are readily available to study patient preferences in health. These methods are now being used to inform a wide variety of decisions, and there is a growing body of evidence showing studies of patient preferences can be used for decision making in a wide variety of contexts. This ISPOR Task Force report synthesizes current good practices for increasing the usefulness and impact of patient-preference studies in decision making.

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.

Patient preferences for mitral valve regurgitation treatment: a discrete choice experiment.

Introduction: This study aimed to quantify patients' preferences for benefits and risks associated with treating degenerative mitral regurgitation (DMR) via open heart surgical repair versus a beating heart surgical approach.

Methods: A D-efficient main effects discrete choice experiment (DCE) survey with 10 choice tasks that involved trade-offs across six attributes varying between two and four levels each (procedure invasiveness, recovery intensity, risk of disabling stroke, risk of new onset atrial fibrillation, risk of symptom reappearance and risk of reintervention) was administered online to either clinically confirmed (n = 30) or self-reported DMR (n = 88) patients recruited from either cardiovascular clinics or online clinical patient databases. The error component logit (ECL) analysis combined both patient cohorts after performing a Swait-Louviere scale test.

Development of a severity scale to assess chronic lung disease after extremely preterm birth.

Objective: Chronic lung disease of prematurity (CLDP) is a frequent complication of prematurity. We aimed to identify what clinicians believe are the most important factors determining the severity of CLDP in extremely preterm infants (<28 weeks gestational age) after discharge from the neonatal intensive care unit (NICU) through 12 months corrected age (CA), and to evaluate how these factors should be weighted for scoring, to develop a CLDP severity scale.

Study Design: Clinicians completed a three-round online survey utilizing Delphi methodology.

Clinical characteristics and burden of illness in patients with hereditary angioedema: findings from a multinational patient survey.

Background: Hereditary angioedema (HAE) is a rare, debilitating, genetic disease characterized by unpredictable, recurrent, and potentially fatal swelling of the skin and mucous membranes. We conducted a noninterventional, cross-sectional, web-based survey of patients with a self-reported diagnosis of HAE type 1/2 in Australia, Austria, Canada, France, Germany, Spain, Switzerland, and the United Kingdom to gain a comprehensive real-world understanding of the characteristics of HAE and its burden from the perspective of the patient. The survey included questions on clinical and demographic characteristics, burden of disease, and treatment.

Line of therapy and patient preferences regarding lung cancer treatment: a discrete-choice experiment.

Objective: A growing literature on patient preferences informs decisions in research, regulatory science, and value assessment, but few studies have explored how preferences vary across patients with differing treatment experience. We sought to quantify patient preferences for the benefits and risks of lung cancer treatment and test how preferences differed by line of therapy (LOT).

Methods: Preferences were elicited using a discrete choice experiment (DCE) following rigorous patient and stakeholder engagement.

Do people have differing motivations for participating in a stated-preference study? Results from a latent-class analysis.

Background: Researchers and policy makers have long suspected that people have differing, and potentially nefarious, motivations for participating in stated-preference studies such as discrete-choice experiments (DCE). While anecdotes and theories exist on why people participate in surveys, there is a paucity of evidence exploring variation in preferences for participating in stated-preference studies.

Methods: We used a DCE to estimate preferences for participating in preference research among an online survey panel sample.

Comparing the Preferences of Patients and the General Public for Treatment Outcomes in Type 2 Diabetes Mellitus.

Background: Healthcare treatments and interventions are traditionally evaluated from the societal perspective, but a more patient-centric perspective has been proposed in recent years. We sought to compare preferences of patients and the general public for treatment outcomes of type 2 diabetes using both best-worst scaling (BWS) and rating approaches.

Methods: A survey evaluating the treatment priorities for type 2 diabetes was conducted in the United States.

Patient preferences for reducing bowel adverse events following prostate radiotherapy.

Background: The Extended Prostate Cancer Index Composite (EPIC) instrument is a commonly used patient reported outcome (PRO) tool in prostate cancer clinical trials. Summary scores for EPIC subscales are calculated by averaging patient scores for attributes (e.g.

Analysis of Patient Preferences in Lung Cancer - Estimating Acceptable Tradeoffs Between Treatment Benefit and Side Effects.

Objective: Increased treatment options and longer survival for lung cancer have generated increased interest in patient preferences. Previous studies of patient preferences in lung cancer have not fully explored preference heterogeneity. We demonstrate a method to explore preference heterogeneity in the willingness of patients with lung cancer and caregivers to trade progression-free survival (PFS) with side effects.

Increasing the Patient-Centeredness of Health Economics and Outcomes Research Through Patient Engagement in Core Outcome Set Development.

Core outcome sets (COS) are becoming increasingly popular in clinical research and can provide important inputs for further health economics and outcomes research (HEOR) studies. Use of standard, consistently reported outcomes can demonstrate and allow differentiation of the effectiveness and value of different treatments. Incorporating patient values during COS development increases the patient centeredness of evidence available across decision-making contexts.

Prioritizing outcome preferences in patients with ocular hypertension and open-angle glaucoma using best-worst scaling.

Purpose: To quantify patients' preferences for glaucoma outcomes and use this information to prioritize outcomes that are important to patients.

Design: A cross-sectional study using best-worst scaling object case (BWS).

Participants: Two hundred seventy-four participants newly diagnosed with ocular hypertension or mild to moderate open angle glaucoma from three private practices and one academic medical center in the United States.

Evaluating Patient Preferences of Maintenance Therapy for the Treatment of Chronic Obstructive Pulmonary Disease: A Discrete Choice Experiment in the UK, USA and Germany.

Introduction: With increasing availability of different treatments for chronic obstructive pulmonary disease (COPD), we sought to understand patient preferences for COPD treatment in the UK, USA, and Germany using a discrete choice experiment (DCE).

Methods: Qualitative research identified six attributes associated with COPD maintenance treatments: ease of inhaler use, exacerbation frequency, frequency of inhaler use, number of different inhalers used, side effect frequency, and out-of-pocket costs. A DCE using these attributes, with three levels each, was designed and tested through cognitive interviews and piloting.

Patient preferences for attributes of tyrosine kinase inhibitor treatments for EGFR mutation-positive non-small-cell lung cancer.

EGFR-tyrosine kinase inhibitors (TKIs) vary in efficacy, side effects (SEs) and dosing regimen. We explored EGFR-TKI treatment attribute preferences in EGFR mutation-positive metastatic non-small-cell lung cancer. Patients completed a survey utilizing preference elicitation methods: direct elicitation of four EGFR-TKI profiles describing progression-free survival (PFS), severe SE risk, administration; discrete choice experiment involving 12 choice tasks.

Examining Generalizability of Older Adults' Preferences for Discussing Cessation of Screening Colonoscopies in Older Adults with Low Health Literacy.

Background/objectives: Many older adults receive unnecessary screening colonoscopies. We previously conducted a survey using a national online panel to assess older adults' preferences for how clinicians can discuss stopping screening colonoscopies. We sought to assess the generalizability of those results by comparing them to a sample of older adults with low health literacy.

How Do Older Adults Consider Age, Life Expectancy, Quality of Life, and Physician Recommendations When Making Cancer Screening Decisions? Results from a National Survey Using a Discrete Choice Experiment.

Older adults with limited life expectancy frequently receive cancer screening, although on average, harms outweigh benefits. We examined the influence of life expectancy on older adults' cancer screening decisions relative to three other factors. Adults aged 65+ years ( = 1272) were recruited from a national online survey panel.

The evolving role of patient preference studies in health-care decision-making, from clinical drug development to clinical care management.

: There is a growing trend of using patient preference studies to help incorporate the patient perspective into clinical drug development, care management, and health-care decision-making. Collecting and interpreting patient preference data is integral to multi-stakeholder engagement, patient-centric drug development, and clinical care management. Operationally, challenges exist in understanding 'when' and 'how' to embark on patient preference studies.