Economic Evaluation in Kidney Transplantation: A Scoping Review and Novel PESTLE Stakeholder Gaps Analysis.

Aim: The objective of this study was to systematically review the literature related to the economic evaluation of kidney transplantation to determine the extent of current research and identify gaps for future research.

Subject And Methods: We searched 4 medical and 2 economic electronic databases as well as hand-searching reference lists of review articles and other pertinent articles. Exclusion criteria included articles that did not include original work (ie, reviews), were not in English, and were not journal articles or economic working papers (eg, commentaries, theses, abstracts).

Role Preferences in Medical Decision Making: Relevance and Implications for Health Preference Research.

Health preference research (HPR) is being increasingly conducted to better understand patient preferences for medical decisions. However, patients vary in their desire to play an active role in medical decisions. Until now, few studies have considered patients' preferred roles in decision making.

Does price disclosure in pharmaceutical advertising result in price transparency? Evidence from a randomized experiment.

Background: Policies that mandate list price disclosure in direct-to-consumer pharmaceutical advertising (DTCPA) cite price transparency among the benefits. The expectation is that price transparency will lead to changes in consumer behavior that will ultimately lower healthcare costs.

Objective: The objective of this study was to assess the impact of price transparency on perceived level of information and consumer behaviors, specifically intentions to seek treatment and intentions to comparison shop.

Social and environmental determinants of health among children with long-term movement impairment.

The healthcare research community increasingly recognizes the need to address social (SDOH) and environmental determinants of health (EDOH) to optimize health and healthcare. This is particularly relevant to disability and functioning and to those with child onset conditions that impair mobility and impact functioning and participation. Using the World Health Organization (WHO)'s International Classification of Functioning, Disability, and Health (ICF) as a comprehensive framework, this paper aims to discuss our understanding of the relationships between social and EDOH and outcomes among people with impaired mobility that impacts functioning.

Best-Worst Scaling and the Prioritization of Objects in Health: A Systematic Review.

Background And Objective: Best-worst scaling is a theory-driven method that can be used to prioritize objects in health. We sought to characterize all studies of best-worst scaling to prioritize objects in health, to assess trends of using best-worst scaling in prioritization over time, and to assess the relationship between a legacy measure of quality (PREFS) and a novel assessment of subjective quality and policy relevance.

Methods: A systematic review identified studies published through to the end of 2021 that applied best-worst scaling to study priorities in health (PROSPERO CRD42020209745), updating a prior review published in 2016.

Proximal Femoral Focal Deficiency/Congenital Femoral Deficiency: Evaluation and Management.

Proximal focal femoral deficiency is a congenital transverse deficiency in which the femur is globally smaller with a typical proximal deformity at the hip that may include distal involvement of knees, leg, and feet. Congenital femoral deficiency (CFD) describes a broader spectrum of longitudinal deficiency inclusive of proximal focal femoral deficiency. CFD may also include lateral distal femoral hypoplasia, knee cruciate ligament deficiency, rotational instability, patellar dislocation, fibular hemimelia, ray absence, and contralateral limb involvement.

Attention to price disclosures in direct-to-consumer prescription drug advertising and the impact on drug perceptions.

Background: Mandatory disclosure of the price of prescription drugs within direct-to-consumer pharmaceutical advertisements (DTCA) has been proposed as a potential means of curbing rising drug costs in the United States. While price transparency in DTCA has widespread public support, empirical evidence regarding the effects of such drug prices disclosures remains limited.

Objectives: This study assessed the degree to which a price disclosure was noticed, the individual characteristics associated with price disclosure recognition, and the impact on perceived drug affordability, effectiveness, and safety.

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.

Do Patient Preferences Align With Value Frameworks? A Discrete-Choice Experiment of Patients With Breast Cancer.

Assess patient preferences for aspects of breast cancer treatments to evaluate and inform the usual assumptions in scoring rubrics for value frameworks. A discrete-choice experiment (DCE) was designed and implemented to collect quantitative evidence on preferences from 100 adult female patients with a self-reported physician diagnosis of stage 3 or stage 4 breast cancer. Respondents were asked to evaluate some of the treatment aspects currently considered in value frameworks.

Reporting Formative Qualitative Research to Support the Development of Quantitative Preference Study Protocols and Corresponding Survey Instruments: Guidelines for Authors and Reviewers.

Background: Formative qualitative research is foundational to the methodological development process of quantitative health preference research (HPR). Despite its ability to improve the validity of the quantitative evidence, formative qualitative research is underreported.

Objective: To improve the frequency and quality of reporting, we developed guidelines for reporting this type of research.

Collecting patient preference information using a Clinical Data Research Network: demonstrating feasibility with idiopathic pulmonary fibrosis.

Rare diseases present challenges for accessing patient populations to conduct surveys. Clinical Data Research Networks (CDRNs) offer an opportunity to overcome those challenges by providing infrastructure for accessing patients and sharing data. This study aims to demonstrate the feasibility of collecting patient preference information for a rare disease in a CDRN, using idiopathic pulmonary fibrosis as proof of concept.

Engaging patients and caregivers in prioritizing symptoms impacting quality of life for Duchenne and Becker muscular dystrophy.

Purpose: Patient preference information (PPI) have an increasing role in regulatory decision-making, especially in benefit-risk assessment. PPI can also facilitate prioritization of symptoms to treat and inform meaningful selection of clinical trial endpoints. We engaged patients and caregivers to prioritize symptoms of Duchenne and Becker muscular dystrophy (DBMD) and explored preference heterogeneity.

Care Management to Reduce Disparities and Control Hypertension in Primary Care: A Cost-effectiveness Analysis.

Background: Project ReD CHiP (reducing disparities and controlling hypertension in primary care) care management was a clinic-based intervention that aimed to improve blood pressure control through improved care coordination and provide self-management support to patients from racially and socioeconomically.

Objective: To evaluate the cost-effectiveness of ReD CHiP care management versus standard care to treat hypertension in diverse communities.

Research Design: Microsimulation model from a health care sector perspective over 15 years.

Seeing is Engaging: Vlogs as a Tool for Patient Engagement.

Video blogs (‘vlogs’ – pronounced as one syllable) are a popular platform for conveying know-how online and can be used as a tool for patient engagement. It can be challenging to engage patients in their own care, and the unique attributes of vlogs overcome some of the barriers to engagement such as high treatment burden and a lack of the sense of community. While some clinicians may distrust social media and question its place in patient care, we present The Frey Life, one example of a patient vlog, to show how the platform models and fosters engagement, and provides the patient perspective.

Developing a Patient-Centered Benefit-Risk Survey: A Community-Engaged Process.

Objectives: To provide a community-engaged process to inform the design of a stated-preferences experiment. The process involved integrating patients and caregivers of people with Duchenne/Becker muscular dystrophy, advocates, clinicians, and the sponsor in conceptualizing and developing a benefit-risk survey on the basis of phase III trial results.

Methods: Our community-engagement process for the development of a stated-preference survey included a set of five guiding principles with a foundation in the principles of community-engaged research.

Patient-centered benefit-risk assessment in duchenne muscular dystrophy.

Introduction: This study quantified caregiver and patient preferences for a therapeutic agent with demonstrated pulmonary benefits for Duchenne muscular dystrophy (DMD). Caregiver and patient differences were also explored.

Methods: A best-worst scaling survey (BWS) was administered to caregivers and patients.

Using Best-Worst Scaling to Investigate Preferences in Health Care.

Introduction: Best-worst scaling (BWS) is becoming increasingly popular to elicit preferences in health care. However, little is known about current practice and trends in the use of BWS in health care. This study aimed to identify, review and critically appraise BWS in health care, and to identify trends over time in key aspects of BWS.

A Scoping Review of Healthcare Costs for Patients with Cystic Fibrosis.

Background: Cystic fibrosis (CF), one of the most common life-shortening genetic diseases, has no cure, but people living with it have seen improvements in their health and survival. The rising life expectancy and increased availability of treatment options has likely increased the lifetime costs of people living with CF. In addition, a recent drug approval for a therapy that targets the cause of the disease is one of the most expensive drugs worldwide.

Serving Older Adults with Complex Care Needs: A New Benefit Option for Medicare.

Medicare was originally designed to protect beneficiaries from the financial burden of acute episodes of illness. As lifespans lengthen, Medicare must adapt to serve beneficiaries with substantial long-term physical or cognitive impairment who need personal care assistance. These beneficiaries often incur high out-of-pocket costs for Medicare-covered services as well as home and community care not covered by Medicare.

Caregiver preferences for emerging duchenne muscular dystrophy treatments: a comparison of best-worst scaling and conjoint analysis.

Background: Through Patient-Focused Drug Development, the US Food and Drug Administration (FDA) documents the perspective of patients and caregivers and are currently conducting 20 public meetings on a limited number of disease areas. Parent Project Muscular Dystrophy (PPMD), an advocacy organization for Duchenne muscular dystrophy (DMD), has demonstrated a community-engaged program of preference research that would complement the FDA's approach.

Objective: Our objective was to compare two stated-preference methods, best-worst scaling (BWS) and conjoint analysis, within a study measuring caregivers' DMD-treatment preferences.

Screening parents during child evaluations: exploring parent and child psychopathology in the same clinic.

Objective: Children of depressed and/or anxious parents are at increased risk for developing psychiatric disorders. Little research has focused on screening parents bringing their children for psychiatric evaluation, and few studies have included fathers or Hispanic children. This study had the following aims: 1) to identify current symptom rates in parents bringing their children for evaluation; and 2) to determine whether parental symptoms were associated with children's symptoms, diagnoses, and functioning.