Health-Related Quality of Life in Patients with Adult-Onset Myotonic Dystrophy Type 1: A Systematic Review
ConclusionsHRQoL in adult-onset DM1 has been extensively studied using the SF-36 and the INQoL, but current estimates are inconclusive, and little is known of the impact of the disease as measured using other instruments. Our data synthesis should help characterize the patient burden of DM1 and inform future studies of HRQoL in this indication. (Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - February 4, 2019 Category: International Medicine & Public Health Source Type: research

Patient-Community Perspectives on Real-World Evidence: Enhancing Engagement, Understanding, and Trust
ConclusionPatients see great possibility in using RWE to understand how a treatment works —to find someone that “looks like me” as assurance of how a treatment might benefit them personally. Patient groups will play a critical role in helping to educate constituents on understanding, contributing to, and using RWE. To maximize patient uptake and the co-development and application o f RWE, patient groups require education and tools. (Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - January 22, 2019 Category: International Medicine & Public Health Source Type: research

Patient and Oncologist Preferences for the Treatment of Adults with Advanced Soft Tissue Sarcoma: A Discrete Choice Experiment
ConclusionsBoth patients with STS and oncologists preferred a treatment that maximizes the life of patients while avoiding hospitalizations. (Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - January 19, 2019 Category: International Medicine & Public Health Source Type: research

EQ-5D-5L is More Responsive than EQ-5D-3L to Treatment Benefit of Cataract Surgery
ConclusionsThe EQ-5D-5L utilities tend to be more responsive than the EQ-5D-3L utilities to treatment benefits of cataract surgery. The HUI3 utilities are more responsive than both the EQ-5D-5L and SF-6D, and SF-6D utilities may be slightly more responsive than the EQ-5D-5L for assessing patients undergoing cataract surgery. (Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - January 4, 2019 Category: International Medicine & Public Health Source Type: research

The Value of Treatment Processes in Germany: A Discrete Choice Experiment on Patient Preferences in Complementary and Conventional Medicine
ConclusionsThe time physicians take and the extent to which they listen attentively are most important and are equally important to all patients. These results may contribute to the debate about more patient-centered healthcare. They support a strengthening of medical consultations in the German healthcare system. We suggest giving physicians the opportunity to spend more time with their patients, which may be achieved by changing the general conditions of remuneration (e.g., improved reimbursement of medical consultations).German Clinical Trial RegisterDRKS00013160. (Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - December 19, 2018 Category: International Medicine & Public Health Source Type: research

Elicitation of Health-Related Utility in Perianal Fistula in Crohn ’s Disease
ConclusionLow utility values were assigned to the non-remission health states for perianal fistulae in CD by the general public and patients with CD. This demonstrates the high humanistic burden of inadequately managed perianal fistula in CD. (Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - December 17, 2018 Category: International Medicine & Public Health Source Type: research

Symptoms and Impacts in Metastatic Castration-Resistant Prostate Cancer: Qualitative Findings from Patient and Physician Interviews
ConclusionsChemotherapy-na ïve patients with metastatic castration-resistant prostate cancer experience a substantial burden from their condition. Furthermore, as castration-resistant prostate cancer progresses from the non-metastatic stage to the early metastatic (pre-chemotherapy) stage, certain symptoms become more common and disturb patients’ lives to a greater extent. The resulting conceptual model for metastatic castration-resistant prostate cancer highlights areas that are not adequately assessed with current patient-reported outcome instruments. (Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - December 6, 2018 Category: International Medicine & Public Health Source Type: research

Comment on: Tools Measuring Quality of Death, Dying, and Care, Completed After Death: Systematic Review of Psychometric Properties
(Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - December 3, 2018 Category: International Medicine & Public Health Source Type: research

Authors ’ Reply to Montoya et al. Comment On: “Tools Measuring Quality of Death, Dying, and Care, Completed After Death: Systematic Review of Psychometric Properties”
(Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - December 3, 2018 Category: International Medicine & Public Health Source Type: research

Creation of a Decision Support Tool for Expectant Parents Facing Threatened Periviable Delivery: Application of a User-Centered Design Approach
ConclusionOur results suggest that a DST that combines unbiased information and understandable outcomes with family testimonials would be meaningful for periviable SDM. User-centered design was found to be a useful method for creating a DST prototype that may lead to improved effectiveness, usability, uptake, and dissemination in the future, by leveraging the expertise of a wide range of stakeholders. (Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - November 29, 2018 Category: International Medicine & Public Health Source Type: research

High Proportion of Subjective Component to the Disease Activity Score is Associated with Favorable Response to Abatacept in Rheumatoid Arthritis
ConclusionsThe DAS28-P could be predictive of response to abatacept. A higher baseline DAS28-P is associated with a favorable therapeutic response to abatacept.Trial registrationTrial name, Korean Post-marketing Surveillance for Orencia®. Trial registration number, NCT01583244. Registered on April 20, 2012. (Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - November 28, 2018 Category: International Medicine & Public Health Source Type: research

Issues in the Design of Discrete Choice Experiments
(Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - November 16, 2018 Category: International Medicine & Public Health Source Type: research

Patient Perspectives on the Value of Patient Preference Information in Regulatory Decision Making: A Qualitative Study in Swedish Patients with Rheumatoid Arthritis
ConclusionsPatients thought PPI could be valuable to consider in regulatory decisions. It is essential for patients to be well-informed when asked for their preferences. Research on information materials to inform patients in preference studies is needed to increase the value of PPI in regulatory decision making. (Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - November 15, 2018 Category: International Medicine & Public Health Source Type: research

Healthcare Experience and their Relationship with Demographic, Disease and Healthcare-Related Variables: A Cross-Sectional Survey of Patients with Chronic Diseases Using the IEXPAC Scale
AbstractBackgroundPatient experience is acknowledged as a principal aspect of quality healthcare delivery, and it has implications with regard to outcomes.ObjectivesOur objective was to evaluate the healthcare experience of patients with chronic diseases to identify patient-perceived healthcare gaps and to assess the influence of demographic and healthcare-related variables on patient experiences.MethodsA cross-sectional survey was delivered to adult patients with chronic diseases: diabetes mellitus (DM), human immunodeficiency virus (HIV) infection, inflammatory bowel disease (IBD) or rheumatic diseases. Patient experienc...
Source: The Patient - Patient-Centered Outcomes Research - November 15, 2018 Category: International Medicine & Public Health Source Type: research

Multi-Method Patient-Engagement Approach: A Case Example from a PCORI-Funded Training Project
(Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - November 3, 2018 Category: International Medicine & Public Health Source Type: research

Implementing a Health Confidence Tool at Time of Discharge
(Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - October 30, 2018 Category: International Medicine & Public Health Source Type: research

Validity of the Patient Experiences and Satisfaction with Medications (PESaM) Questionnaire
ConclusionsThe PESaM questionnaire is a unique patient-reported outcome measure evaluating patient experiences and satisfaction with medications. It has been developed in conjunction with patients, ensuring coverage of domains and issues relevant from the patient ’s perspective. This study has shown promising validity of the generic module of the PESaM questionnaire. Further research is recommended to assess reliability in greater detail as well as the responsiveness of the measure.Trial registrationThe study is registered in The Netherlands National Trial Register (Trial Code 5860). (Source: The Patient - Patient-Ce...
Source: The Patient - Patient-Centered Outcomes Research - October 26, 2018 Category: International Medicine & Public Health Source Type: research

Symptoms and Concerns Among Children and Young People with Life-Limiting and Life-Threatening Conditions: A Systematic Review Highlighting Meaningful Health Outcomes
ConclusionBurdensome symptoms and concerns affect young people with malignant and nonmalignant conditions and occur across the disease trajectory; pediatric palliative care should not be limited to the end-of-life phase. A child –family-centered framework of health outcomes, spanning the patient, family, and quality of service levels is proposed to inform service development. Future research should address gaps identified across the literature (i.e., the involvement of young people in research, evidence for developing c ountries, and a focus on nonmalignant conditions. (Source: The Patient - Patient-Ce...
Source: The Patient - Patient-Centered Outcomes Research - October 26, 2018 Category: International Medicine & Public Health Source Type: research

Development of a Discrete-Choice Experiment (DCE) to Elicit Adolescent and Parent Preferences for Hypodontia Treatment
ConclusionsIncluding the target respondent group in all stages of DCE development ensured the final DCE survey was valid and acceptable. DCE methods appear to be a useful tool for exploring joint decision making alongside conventional preference elicitation. (Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - October 26, 2018 Category: International Medicine & Public Health Source Type: research

Developing Sexual and Reproductive Health Educational Resources for Young Women with Cystic Fibrosis: A Structured Approach to Stakeholder Engagement
ConclusionsThis study illuminates a path for a formal process of stakeholder engagement and evaluation in educational resource development centered on the SRH care needs of AYA women with CF. Similar systematic, planned processes could be extended to other populations and aspects of healthcare. (Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - October 26, 2018 Category: International Medicine & Public Health Source Type: research

Acknowledgement to Referees
(Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - October 24, 2018 Category: International Medicine & Public Health Source Type: research

Parent Empowerment in Pediatric Healthcare Settings: A Systematic Review of Observational Studies
The objective of this systematic review was to synthesize potential antecedents and consequences of parent empowerment in healthcare settings.Eligibility CriteriaThe inclusion criteria were (1) studies with results about parent empowerment in the context of children ’s healthcare or healthcare providers; and (2) qualitative studies, observational studies, and systematic reviews of such studies.Information SourcesWe searched the databases of PubMed, Web of Science, and Google Scholar (2006 –2017) and reference lists.Included StudiesForty-four articles met the inclusion criteria.Synthesis of ResultsWe identified ...
Source: The Patient - Patient-Centered Outcomes Research - October 17, 2018 Category: International Medicine & Public Health Source Type: research

Impact of Carotid Artery Stenosis on Quality of Life: A Systematic Review
ConclusionThe findings from the review identified the important themes that affect patients with carotid stenosis disease. The current generic and disease-specific PROMs do not cover all themes that impact the HRQoL of patients suffering with this disease. The proposed themes can be used to develop a new disease-specific PROM to measure HRQoL. (Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - October 17, 2018 Category: International Medicine & Public Health Source Type: research

A Scoping Review of Key Health Items in Self-Report Instruments Used Among Solid Organ Transplant Recipients
AbstractThe overall aim of this scoping review of the literature is twofold: (1) to provide an overview of all instruments that have been used to assess health-related quality of life (HRQoL) after solid organ transplantation and (2) to provide a list of health items they include to support future studies on the development of a new-generation HRQoL instrument. All studies that administered any form of HRQoL instrument to post-transplant solid organ recipients were identified in a comprehensive search of PubMed (MEDLINE), Embase, and Web of Science, with a cut-off date of May 2018. The search used various combinations of t...
Source: The Patient - Patient-Centered Outcomes Research - October 16, 2018 Category: International Medicine & Public Health Source Type: research

Development of an Item Pool for a Needs-Based Measure of Quality of Life of Carers of a Family Member with Dementia
ConclusionsThe resulting dementia carer-specific item pool reflects the accounts of a diverse sample of those who provide care for a person with dementia, allowing them to define the nature of the impact on their lives and resulting in a valid, acceptable set of items. (Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - October 12, 2018 Category: International Medicine & Public Health Source Type: research

Patient-Consent Disconnects in Clinical Research
(Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - October 1, 2018 Category: International Medicine & Public Health Source Type: research

Acceptability of Patient-Reported Outcome and Experience Measures for Hepatitis C Treatment Among People Who Use Drugs
ConclusionsThe findings show that utilising a participatory approach to the development of PRMs for DAA HCV treatment with PWID not only provides a way to measure experiences and outcomes of treatment from the patient perspective, but also provides a means for highly marginalised patient groups to have a say in and negotiate their care in ways that might not otherwise be possible. (Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - October 1, 2018 Category: International Medicine & Public Health Source Type: research

The Parker Model: Applying a Qualitative Three-Step Approach to Optimally Utilize Input from Stakeholders When Introducing New Device Technologies in the Management of Chronic Rheumatic Diseases
ConclusionsThis study is the first to apply a composite, qualitative research model when introducing an e-Device for the treatment and management of rheumatic disease. Results show that input from patients and other stakeholders using the Parker Model can add value to the development and implementation of an e-Device. (Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - October 1, 2018 Category: International Medicine & Public Health Source Type: research

Patient-Reported Symptoms Over 48  Weeks Among Participants in Randomized, Double-Blind, Phase III Non-inferiority Trials of Adults with HIV on Co-formulated Bictegravir, Emtricitabine, and Tenofovir Alafenamide versus Co-formulated Abacavir, Dolutegravir, and Lamivudine
AbstractBackgroundIntegrase strand transfer inhibitors (INSTIs) are recommended for first-line antiretroviral therapy in combination with two nucleos(t)ide reverse transcriptase inhibitors. Co-formulated bictegravir, emtricitabine, and tenofovir alafenamide (B/F/TAF), a novel, INSTI-based regimen, is currently approved in the US and EU for the treatment of HIV-1 infection and recommended as first-line treatment in current guidelines. In our current analysis, we aimed to determine changes in patient-reported symptoms over time among HIV-1-infected adults who initiated or switched to B/F/TAF versus another INSTI-based regime...
Source: The Patient - Patient-Centered Outcomes Research - October 1, 2018 Category: International Medicine & Public Health Source Type: research

Accounting for Scale Heterogeneity in Healthcare-Related Discrete Choice Experiments when Comparing Stated Preferences: A Systematic Review
ConclusionsScale heterogeneity is a prevalent issue in healthcare DCEs. Despite this, few published DCEs have discussed such issues, and fewer still have used formal methods to identify and account for the impact of scale heterogeneity. The use of formal methods to test for scale heterogeneity should be used, otherwise the results of DCEs potentially risk producing biased and potentially misleading conclusions regarding preferences for aspects of healthcare. (Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - October 1, 2018 Category: International Medicine & Public Health Source Type: research

Themes that Determine Quality of Life in Patients with Peripheral Arterial Disease: A Systematic Review
DiscussionThe findings from the review identified the important domains that affect patients living with peripheral arterial disease. None of the current generic and disease-specific patient-reported outcome measures provide a comprehensive measure for all themes that impact the daily living of patients with peripheral arterial disease. (Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - October 1, 2018 Category: International Medicine & Public Health Source Type: research

Patient Perspective on Acute Intermittent Porphyria with Frequent Attacks: A Disease with Intermittent and Chronic Manifestations
ConclusionsIn this study population of acute intermittent porphyria with frequent attacks, most patients had symptoms during and between attacks. In these patients, acute intermittent porphyria appears to have acute exacerbations as well as chronic day-to-day manifestations, and is not just intermittent as its name implies. As a result, patients reported limitations in their ability to function across multiple domains of their lives on a regular basis and not just during acute attacks. (Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - October 1, 2018 Category: International Medicine & Public Health Source Type: research

Patient Preferences for Managing Insomnia: A Discrete Choice Experiment
AbstractBackgroundDespite the rapid development of effective treatments, both pharmacological and non-pharmacological, insomnia management remains suboptimal at the practice interface. Patient preferences play a critical role in influencing treatment outcomes. However, there is currently a mismatch between patient preferences and clinician recommendations, partly perpetuated by a limited understanding of the patients ’ decision-making process.ObjectivesThe aim of our study was to empirically quantify patient preferences for treatment attributes common to both pharmacological and non-pharmacological insomnia treatment...
Source: The Patient - Patient-Centered Outcomes Research - October 1, 2018 Category: International Medicine & Public Health Source Type: research

Understanding How Chorea Affects Health-Related Quality of Life in Huntington Disease: An Online Survey of Patients and Caregivers in the United States
The objective of this study was to evaluate how chorea impacts HRQoL and overall functioning among HD patients participating on the PatientsLikeMe website (www.PatientsLikeMe.com).MethodsA survey was provided to HD participants and/or their caregivers via PatientsLikeMe (9 February 2017 –22 March 2017), comprising multiple-choice and open-ended questions designed to assess how chorea impacts HRQoL and overall functioning, and the importance of treating chorea. The HDQLIFE measurement system was used to evaluate patient-reported outcomes of chorea and compare Anxiety and Stigma sc ores in participants with high chorea...
Source: The Patient - Patient-Centered Outcomes Research - October 1, 2018 Category: International Medicine & Public Health Source Type: research

Patient-Consent Disconnects in Clinical Research
(Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - October 1, 2018 Category: International Medicine & Public Health Source Type: research

Symposium Title: Preference Evidence for Regulatory Decisions
(Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - October 1, 2018 Category: International Medicine & Public Health Source Type: research

Adaptation, Acceptance and Adaptive Preferences in Health and Capability Well-Being Measurement Amongst Those Approaching End of Life
ConclusionDespite adapting to their conditions, the reference group for individuals approaching EoL largely remained a healthy, capable population, and most did not show evidence of adaptive preferences. (Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - October 1, 2018 Category: International Medicine & Public Health Source Type: research

Key Issues and Potential Solutions for Understanding Healthcare Preference Heterogeneity Free from Patient-Level Scale Confounds
(Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - October 1, 2018 Category: International Medicine & Public Health Source Type: research

Acceptability of Patient-Reported Outcome and Experience Measures for Hepatitis C Treatment Among People Who Use Drugs
ConclusionsThe findings show that utilising a participatory approach to the development of PRMs for DAA HCV treatment with PWID not only provides a way to measure experiences and outcomes of treatment from the patient perspective, but also provides a means for highly marginalised patient groups to have a say in and negotiate their care in ways that might not otherwise be possible. (Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - October 1, 2018 Category: International Medicine & Public Health Source Type: research

How Do Members of the Duchenne and Becker Muscular Dystrophy Community Perceive a Discrete-Choice Experiment Incorporating Uncertain Treatment Benefit? An Application of Research as an Event
AbstractBackgroundBest –worst scaling methods have been used in several Duchenne and Becker muscular dystrophy (DBMD) studies to quantify patient and caregiver priorities and preferences and promote patient-focused drug development (PFDD). We sought to assess the extent to which different members of the DBMD community w ould accept a discrete-choice experiment (DCE) that incorporates uncertainty regarding individual-level benefit.MethodsA community advisory board encouraged the development and testing of a DCE to further examine treatment preferences in DBMD and to facilitate the inclusion of a policy-relevant uncert...
Source: The Patient - Patient-Centered Outcomes Research - September 27, 2018 Category: International Medicine & Public Health Source Type: research

General Population vs. Patient Preferences in Anticoagulant Therapy: A Discrete Choice Experiment
ConclusionsThe general population and patients with cardiovascular disease had slightly different preferences for treatment outcomes. The differences can potentially influence estimated benefits and risks and patient-centered treatment decisions. (Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - September 21, 2018 Category: International Medicine & Public Health Source Type: research

Tools Measuring Quality of Death, Dying, and Care, Completed after Death: Systematic Review of Psychometric Properties
ConclusionFour tools demonstrated some promise, but no single tool was consistent across all psychometric properties assessed. All tools identified would benefit from further psychometric testing. (Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - August 24, 2018 Category: International Medicine & Public Health Source Type: research

Treatment Decisions for Advanced Non-Squamous Non-Small Cell Lung Cancer: Patient and Physician Perspectives on Maintenance Therapy
ConclusionThis study identified challenges and motivators influencing advanced NSCLC patients ’ decisions to accept or refuse therapy, as well as patient and disease characteristics associated with physician’s treatment recommendations for MT. (Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - August 21, 2018 Category: International Medicine & Public Health Source Type: research

“I Was Trying to Do the Maths”: Exploring the Impact of Risk Communication in Discrete Choice Experiments
ConclusionMost women completed the DCE in line with economic theory; however, violations were identified. Women appeared to visualise risk whether they received icon arrays or percentages only. Providing clear instructions and graphics to aid interpretation of risk and qualitative piloting to verify understanding is recommended. Further investigation is required to determine if the process of verbalising thoughts changes the behaviour of respondents. (Source: The Patient - Patient-Centered Outcomes Research)
Source: The Patient - Patient-Centered Outcomes Research - August 11, 2018 Category: International Medicine & Public Health Source Type: research

Including Opt-Out Options in Discrete Choice Experiments: Issues to Consider
We present our code written in the R statistical language so that others can explore these issues in their own data. In this practical guideline, we generate synthetic data on medication choice and use Monte Carlo simulation. We consider three different definitions for the opt-out alternative and four candidate models for each definition. We apply a frequentist-based multimodel inference approach and use performance indicators to assess the relative suitability of each candidate model in a range of settings. We show that misspecifying the opt-out effect has repercussions for marginal willingness to pay estimation and the f...
Source: The Patient - Patient-Centered Outcomes Research - August 2, 2018 Category: International Medicine & Public Health Source Type: research