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.

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.

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.

Suitability of Preference Methods Across the Medical Product Lifecycle: A Multicriteria Decision Analysis.

Objectives: This study aimed to understand the importance of criteria describing methods (eg, duration, costs, validity, and outcomes) according to decision makers for each decision point in the medical product lifecycle (MPLC) and to determine the suitability of a discrete choice experiment, swing weighting, probabilistic threshold technique, and best-worst scale cases 1 and 2 at each decision point in the MPLC.

Methods: Applying multicriteria decision analysis, an online survey was sent to MPLC decision makers (ie, industry, regulatory, and health technology assessment representatives). They ranked and weighted 19 methods criteria from an existing performance matrix about their respective decisions across the MPLC.

Social determinants of health associated with COVID-19 severity during pregnancy: a multinational cohort study (in the International Registry of Coronavirus Exposure in Pregnancy).

Background: The COVID-19 pandemic has caused morbidity and mortality, particularly among vulnerable populations. We aimed to assess social and demographic characteristics associated with COVID-19 severity among symptomatic participants during pregnancy.

Methods: The International Registry of Coronavirus Exposure in Pregnancy is a multinational, longitudinal observational cohort study of adult participants tested for SARS-CoV-2 or who received clinical diagnosis of COVID-19 during pregnancy (NCT04366986).

Preferences for preventive treatments for rheumatoid arthritis: discrete choice survey in the UK, Germany and Romania.

Objective: To quantify preferences for preventive therapies for rheumatoid arthritis (RA) across three countries.

Methods: A web-based survey including a discrete choice experiment was administered to adults recruited via survey panels in the UK, Germany and Romania. Participants were asked to assume they were experiencing arthralgia and had a 60% chance of developing RA in the next 2 years and completed 15 choices between no treatment and two hypothetical preventive treatments.

Systematic review of quantitative preference studies of treatments for rheumatoid arthritis among patients and at-risk populations.

Treatments used for rheumatoid arthritis (RA) are under investigation for their efficacy to prevent RA in at risk groups. It is therefore important to understand treatment preferences of those at risk. We systematically reviewed quantitative preference studies of drugs to treat, or prevent RA, to inform the design of further studies and trials of RA prevention.

Exploring Decisional Conflict With Measures of Numeracy and Optimism in a Stated Preference Survey.

Objectives: Low optimism and low numeracy are associated with difficulty or lack of participation in making treatment-related health care decisions. We investigated whether low optimism and low self-reported numeracy scores could help uncover evidence of decisional conflict in a discrete-choice experiment (DCE).

Methods: Preferences for a treatment to delay type 1 diabetes were elicited using a DCE among 1501 parents in the United States.

Treatment preferences for preventive interventions for rheumatoid arthritis: protocol of a mixed methods case study for the Innovative Medicines Initiative PREFER project.

Introduction: Amidst growing consensus that stakeholder decision-making during drug development should be informed by an understanding of patient preferences, the Innovative Medicines Initiative project 'Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle' (PREFER) is developing evidence-based recommendations about how and when patient preferences should be integrated into the drug life cycle. This protocol describes a PREFER clinical case study which compares two preference elicitation methodologies across several populations and provides information about benefit-risk trade-offs by those at risk of rheumatoid arthritis (RA) for preventive interventions.

Methods And Analysis: This mixed methods study will be conducted in three countries (UK, Germany, Romania) to assess preferences of (1) first-degree relatives (FDRs) of patients with RA and (2) members of the public.

Quantitative Benefit-Risk Assessment: State of the Practice Within Industry.

Background: Benefit-risk assessments for medicinal products and devices have advanced significantly over the past decade. The purpose of this study was to characterize the extent to which the life sciences industry is utilizing quantitative benefit-risk assessment (qBRA) methods.

Methods: Semi-structured interviews were conducted with a sample of industry professionals working in drug and/or medical device benefit-risk assessments (n = 20).

Parent Preferences for Delaying Insulin Dependence in Children at Risk of Stage III Type 1 Diabetes.

Autoantibody screening in type 1 diabetes (T1D) may reduce the chances of potentially life-threatening diabetic ketoacidosis (DKA) at diagnosis by allowing individuals at risk of progression to more actively monitor for and/or manage progression to insulin dependence. We investigated parents' preferences for treatments to delay the onset of insulin dependence in children who are at high risk of developing Stage III T1D. A web-based survey ( = 1501) was administered to a stratified sample of parents (children <18 years) in the United States from an online panel.

Something Is Better Than Nothing: The Value of Active Intervention in Stated Preferences for Treatments to Delay Onset of Alzheimer's Disease Symptoms.

Background: The objective of the study was to understand respondents' willingness to accept hypothetical treatment-related risks in return for the benefit of additional time with normal memory from potential Alzheimer's disease interception therapies.

Methods: A US web-based discrete-choice survey was administered to respondents ages 60 to 85 years with no Alzheimer's disease diagnosis and no cognitive symptoms. Choice questions required respondents to indicate whether they preferred a constant, no-treatment condition described as 4 years of normal memory followed by 3 years of cognitive impairment and 5 years of dementia or an interception treatment with chosen risks of disabling stroke and death, but with increased duration of normal memory.

Correction to: Effect of inhaled corticosteroid particle size on asthma efficacy and safety outcomes: a systematic literature review and meta-analysis.

After publication of this work [1] it was noticed that the author name Rachael L. DiSantostefano was not spelt correctly as there was a space in her surname between 'Di' and 'Santostefano'. The publisher apologises for this error.

The feasibility of using multiple databases to study rare outcomes: the potential effect of long-acting beta agonists with inhaled corticosteroid therapy on asthma mortality.

Purpose: Long-acting beta agonists (LABAs) when used without concomitant inhaled corticosteroids (ICS) increase the risk of asthma-related deaths, but the effect on asthma-related death of LABA used in combination with ICS therapy is unknown. To address this question, we explored the feasibility of conducting an observational study using multiple US health care data sources.

Methods: Retrospective cohort study to evaluate the likelihood of getting an upper 95% confidence limit ≤1.

The frequency of, and adherence to, single maintenance and reliever therapy instructions in asthma: a descriptive analysis.

Inhaled corticosteroid/long-acting β2-agonist (ICS/LABA) fixed-dose combinations are recommended regular maintenance options for asthma. ICS/LABAs containing formoterol may also be indicated for single maintenance and reliever therapy (SMART). This analysis evaluated the frequency of SMART dosing of budesonide/formoterol fixed-dose combination (BFC) in the United Kingdom.

Identification of responders to inhaled corticosteroids in a chronic obstructive pulmonary disease population using cluster analysis.

Objectives: To identify clusters of patients who may benefit from treatment with an inhaled corticosteroid (ICS)/long-acting β2 agonist (LABA) versus LABA alone, in terms of exacerbation reduction, and to validate previously identified clusters of patients with chronic obstructive pulmonary disease (COPD) (based on diuretic use and reversibility).

Design: Post hoc supervised cluster analysis using a modified recursive partitioning algorithm of two 1-year randomised, controlled trials of fluticasone furoate (FF)/vilanterol (VI) versus VI alone, with the primary end points of the annual rate of moderate-to-severe exacerbations.

Setting: Global.

Relationship between blood eosinophils and clinical characteristics in a cross-sectional study of a US population-based COPD cohort.

Background: Current evidence suggests that blood eosinophil levels (Eos) are associated with chronic obstructive pulmonary disease (COPD) treatment response and natural history. This analysis investigated the relationship between Eos levels and clinical characteristics in a representative cohort of US subjects with spirometry-defined COPD.

Methods: Cross-sectional data from the National Health And Nutrition Examination Survey (NHANES 2007-2010) of subjects ≥ 40 years with spirometry-defined COPD and Eos data (n = 948) were analyzed.

Medication Guide Reading Behaviors and Attitudes Among Subjects With Migraine, Asthma, or COPD.

Background: This cross-sectional survey describes attitudes and reading behaviors toward medication guides among 785 subjects with migraine, asthma, or COPD who reported recent use of Treximet (sumatriptan/naproxen sodium) or Advair (fluticasone propionate/salmeterol).

Results: The survey demonstrated that the majority (82%) of subjects had read their medication guide, but most read it exactly once and did not read it thoroughly. Patients did not read medication guides with each refill, with the most frequent reasons being that they did not expect the information to have changed and that a doctor would tell them what they needed to know.

Patient Comprehension of Medication Guides for Asthma and Chronic Obstructive Pulmonary Disease Medications.

Purpose: This study assessed patients' comprehension of the Advair and Serevent medication guides (MGs) and MG reading behaviors with the goal to improve risk communication.

Methods: After reading their assigned MGs, 452 adults with asthma or chronic obstructive pulmonary disease participated in structured interviews to assess comprehension of safety risks in the Advair MG (asthma, n = 150; chronic obstructive pulmonary disease, n = 153) and Serevent MG (asthma, n = 149). Generalized estimating equations for correlated binary data were used to identify factors associated with correct responses.

Validation of chronic obstructive pulmonary disease recording in the Clinical Practice Research Datalink (CPRD-GOLD).

Objectives: The optimal method of identifying people with chronic obstructive pulmonary disease (COPD) from electronic primary care records is not known. We assessed the accuracy of different approaches using the Clinical Practice Research Datalink, a UK electronic health record database.

Setting: 951 participants registered with a CPRD practice in the UK between 1 January 2004 and 31 December 2012.

Risk of pneumonia with inhaled corticosteroid versus long-acting bronchodilator regimens in chronic obstructive pulmonary disease: a new-user cohort study.

Introduction: Observational studies using case-control designs have showed an increased risk of pneumonia associated with inhaled corticosteroid (ICS)-containing medications in patients with chronic obstructive pulmonary disease (COPD). New-user observational cohort designs may minimize biases associated with previous case-control designs.

Objective: To estimate the association between ICS and pneumonia among new users of ICS relative to inhaled long-acting bronchodilator (LABD) monotherapy.

Risk of pneumonia with inhaled corticosteroid/long-acting β2 agonist therapy in chronic obstructive pulmonary disease: a cluster analysis.

Background: Pneumonia poses a significant risk in patients with moderate to severe chronic obstructive pulmonary disease but data are limited on the disease phenotypes most susceptible to pneumonia.

Methods: Cluster analysis using a data-driven recursive partitioning algorithm was employed using baseline data from two pooled one-year randomized exacerbation trials (n=3,255) of fluticasone furoate/vilanterol or vilanterol alone to identify distinct patient groups at greatest risk of pneumonia or serious (hospitalization or death) pneumonia.

Results: Five clusters were identified.