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Background:A national priority in the United States is to promote patient engagement in cancer genomics research, especially among diverse and understudied populations. Several cancer genomics research programs have emerged to accomplish this priority, yet questions remain about the meaning and methods of patient engagement. This study explored how cancer genomics research programs define engagement and what strategies they use to engage patients across stages in the conduct of research.Methods:An environmental scan was conducted of cancer genomics research programs focused on patient engagement. Research programs were identified and characterized using materials identified from publicly available sources (e.g., websites), a targeted literature review, and interviews with key informants. Descriptive information about the programs and their definitions of engagement, were synthesized using thematic analysis. The engagement strategies were synthesized and mapped to different stages in the conduct of research, including recruitment, consent, data collection, sharing results, and retention.Results:Ten research programs were identified, examples of which include the Cancer Moonshot Biobank, the MyPART Network, NCI-CONNECT, and the Participant Engagement and Cancer Genome Sequencing (PE-CGS) Network. All programs aimed to include understudied or underrepresented populations. Based on publicly available information, four programs explicitly defined engagement. These definitions similarly characterized engagement as being interpersonal, reciprocal, and continuous. Five general strategies of engagement were identified across the programs: 1) digital (such as websites) and 2) non-digital communications (such as radio broadcasts, or printed brochures); 3) partnering with community organizations; 4) providing incentives; and 5) affiliating with non-academic medical centers. Digital communications were the only strategy used across all stages of the conduct of research. Programs tailored these strategies to their study goals, including overcoming barriers to research participation among diverse populations.Conclusion:Programs studying cancer genomics are deeply committed to increasing research participation among diverse populations through patient engagement. Yet, the field needs to reach a consensus on the meaning of patient engagement, develop a taxonomy of patient engagement measures in cancer genomics research, and identify optimal strategies to engage patients in cancer genomics. Addressing these needs could enable patient engagement to fulfill its potential and accelerate the pace of cancer genomic discoveries.

ImportanceThere are no approved treatments for nonalcoholic fatty liver disease (NAFLD) despite its association with obesity and increased risk of cardiovascular disease (CVD).ObjectiveTo examine the association between bariatric surgery and CVD risk in individuals with severe obesity and NAFLD.Design, Setting, and ParticipantsThis large, population-based retrospective cohort study obtained data from the MarketScan Commercial Claims and Encounters database from January 1, 2007, to December 31, 2017. Participants included insured adults aged 18 to 64 years with NAFLD and severe obesity (body mass index ≥40) without a history of bariatric surgery or CVD before NAFLD diagnosis. Baseline characteristics were balanced between individuals who underwent surgery (surgical group) and those who did not (nonsurgical group) using inverse probability of treatment weighting. Data were analyzed from March 2020 to April 2021.ExposuresBariatric surgery (Roux-en-Y gastric bypass, sleeve gastrectomy, and other bariatric procedures) vs nonsurgical care.Main Outcomes and MeasuresThe main outcome was the incidence of cardiovascular events (primary or secondary composite CVD outcomes). The primary composite outcome included myocardial infarction, heart failure, or ischemic stroke, and the secondary composite outcome included secondary ischemic heart events, transient ischemic attack, secondary cerebrovascular events, arterial embolism and thrombosis, or atherosclerosis. Cox proportional hazards regression models with inverse probability treatment weighting were used to examine the associations between bariatric surgery, modeled as time varying, and all outcomes.ResultsThe study included 86 964 adults (mean [SD] age, 44.3 [10.9] years; 59 773 women [68.7%]). Of these individuals, 30 300 (34.8%) underwent bariatric surgery and 56 664 (65.2%) received nonsurgical care. All baseline covariates were balanced after applying inverse probability treatment weighting. In the surgical group, 1568 individuals experienced incident cardiovascular events compared with 7215 individuals in the nonsurgical group (incidence rate difference, 4.8 [95% CI, 4.5-5.0] per 100 person-years). At the end of the study, bariatric surgery was associated with a 49% lower risk of CVD (adjusted hazard ratio [aHR], 0.51; 95% CI, 0.48-0.54) compared with nonsurgical care. The risk of primary composite CVD outcomes was reduced by 47% (aHR, 0.53 [95% CI, 0.48-0.59), and the risk of secondary composite CVD outcomes decreased by 50% (aHR, 0.50; 95% CI, 0.46-0.53) in individuals with vs without surgery.Conclusions and RelevanceResults of this study suggest that, compared with nonsurgical care, bariatric surgery was associated with significant reduction in CVD risk in individuals with severe obesity and NAFLD.

Duchenne muscular dystrophy is the most common form of muscular dystrophy diagnosed in childhood but is not routinely screened for prenatally or at birth in the United States. We sought to characterize the diagnostic experiences of families and describe their preferences for newborn screening (NBS). We conducted a registry-based survey of families with Duchenne and Becker muscular dystrophy that included open- and closed-ended questions regarding the journey to a diagnosis, preferences for when to learn of a diagnosis, and how knowledge of a diagnosis would impact life decisions. Open-ended responses were analyzed thematically, and closed-ended responses were analyzed descriptively. Sixty-five families completed the survey. The average ages of first concern and diagnosis were 2 and 4 years, respectively. One-third of families (30%) indicated that they would prefer to receive a diagnosis in the newborn period irrespective of treatment options available, and nearly all of the remaining families (93%) indicated that they would want to learn about a diagnosis if there were treatments that worked well during the newborn period. All families (100%) indicated that a diagnosis in the newborn period would impact life decisions. We identified three overarching themes, which described the stages of the diagnostic journey, including having concerns about the child, seeking answers, and receiving the diagnosis. NBS can facilitate improved health outcomes through early access to care, and inform families on major health and nonhealth decisions. The preferences and experiences of families and other stakeholders should be considered when determining the potential value and benefit of expanding NBS programs.

BACKGROUND The approval of novel therapies for patients diagnosed with hematologic malignancies have improved survival outcomes but increased the challenge of aligning chemotherapy choices with patient preferences. We previously developed paper versions of a discrete choice experiment (DCE) and a best-worst scaling (BWS) instrument to quantify the treatment outcome preferences of patients with hematologic malignancies to inform shared decision making. OBJECTIVE We aim to develop an electronic health care tool (EHT) to guide clinical decision making that uses either a BWS or DCE instrument to capture patient preferences. The primary objective of this study is to use both qualitative and quantitative methods to evaluate the perceived usability, cognitive workload (CWL), and performance of electronic prototypes that include the DCE and BWS instrument. METHODS This mixed methods study includes iterative co-design methods that will involve healthy volunteers, patient-caregiver pairs, and health care workers to evaluate the perceived usability, CWL, and performance of tasks within distinct prototypes. Think-aloud sessions and semistructured interviews will be conducted to collect qualitative data to develop an affinity diagram for thematic analysis. Validated assessments (Post-Study System Usability Questionnaire [PSSUQ] and the National Aeronautical and Space Administration’s Task Load Index [NASA-TLX]) will be used to evaluate the usability and CWL required to complete tasks within the prototypes. Performance assessments of the DCE and BWS will include the evaluation of tasks using the Single Easy Questionnaire (SEQ), time to complete using the prototype, and the number of errors. Additional qualitative assessments will be conducted to gather participants’ feedback on visualizations used in the Personalized Treatment Preferences Dashboard that provides a representation of user results after completing the choice tasks within the prototype. RESULTS Ethical approval was obtained in June 2021 from the Institutional Review Board of the University of North Carolina at Chapel Hill. The DCE and BWS instruments were developed and incorporated into the PRIME (Preference Reporting to Improve Management and Experience) prototype in early 2021 and prototypes were completed by June 2021. Heuristic evaluations were conducted in phase 1 and completed by July 2021. Recruitment of healthy volunteers began in August 2021 and concluded in September 2021. In December 2021, our findings from phase 2 were accepted for publication. Phase 3 recruitment began in January 2022 and is expected to conclude in September 2022. The data analysis from phase 3 is expected to be completed by November 2022. CONCLUSIONS Our findings will help differentiate the usability, CWL, and performance of the DCE and BWS within the prototypes. These findings will contribute to the optimization of the prototypes, leading to the development of an EHT that helps facilitate shared decision making. This evaluation will inform the development of EHTs to be used clinically with patients and health care workers. INTERNATIONAL REGISTERED REPORT DERR1-10.2196/39586

BACKGROUND Artificial intelligence (AI) is rapidly expanding in medicine even while lacking formal oversight. OBJECTIVE We sought to identify and describe considerations for the oversight of AI in medicine. We also explored where along the translational process (i.e., AI development, reporting, evaluation, implementation, and surveillance) these considerations were targeted. METHODS We conducted a targeted review of frameworks for the oversight of AI in medicine. The search included key topics such as ‘artificial intelligence,’ ‘machine learning’, ‘guidance as topic’, ‘translational science’, ‘medical device legislation’, and ‘evaluation study,’ and spanned the time period 2014-2021. Frameworks were included if they described translational considerations for AI. The included frameworks were summarized descriptively. Content analysis was used to identify considerations for the oversight of AI in medicine. An evaluation matrix methodology was used to map each consideration across the different translational stages for each framework. RESULTS Six frameworks were featured in the review and included peer reviewed and white papers from consortium and professional organizations. Content analysis of the frameworks revealed five overarching considerations related to the oversight of AI in medicine, including: transparency, reproducibility, ethics, effectiveness, and engagement. All frameworks included discussions regarding transparency, reproducibility, ethics, and effectiveness, while only half of frameworks discussed engagement. The evaluation matrix revealed that frameworks were most likely to report AI considerations for the translational stage of development, and least likely to report considerations for the translational stage of surveillance. CONCLUSIONS Frameworks provided broad guidance for the oversight of AI in medicine, but notably offered less input on the role engagement approaches for oversight, and regarding the translational stage of surveillance. Identifying and optimizing strategies for engagement is essential to ensure that AI can meaningfully benefit patients and other end-users.

Caregivers routinely inform medical and regulatory decision making in rare pediatric diseases. While differences in treatment preferences across caregivers and patients have been observed for Duchenne muscular dystrophy, this evidence was limited by small samples of patients and results were confounded by patient age and disease progression. We tested caregiver and patient preference concordance for treating Duchenne.Preferences and demographic/clinical information from 115 caregivers and 107 patients were collected in an international study (response = 80%) using a previously developed discrete-choice experiment consisting of 12 experimentally controlled choice tasks. Each task presented two profiles that varied across four attributes: disease progression, drug failure probability, kidney damage risk, and fracture risk. Caregivers and patients were matched 1:1 based on patient age. We tested for concordance across each task and by comparing caregivers' and patients' maximum acceptable risk of drug failure, kidney damage, and fracture for a slowing of disease progression.The final analysis included 77 caregivers and 77 patients. No differences were observed in nationality (p = 0.969), disease stage (p = 0.180), or demographic/clinical factors (p = 0.093-0.857); however, patients were more optimistic (p = 0.030). Caregivers and patients chose similarly across tasks (p = 0.101-0.993). To slow disease progression by 1 year, caregivers and patients would tolerate a 9% and 11% increase in drug failure probability, respectively (p = 0.267). Alternatively, they would accept a 3% and 4% increase in the risk of kidney damage (p = 0.719) or a 15% and 20% increase in the risk of fracture (p = 0.534).Caregivers and patients had concordant preferences for treating Duchenne. Providers and regulators can trust both caregiver and patient report of preferences to inform medical decision making.

Duchenne muscular dystrophy has been a launching pad for patient-focused drug development (PFDD). Yet, PFDD efforts have largely neglected non-ambulatory patients. To support PFDD efforts in this population, we primarily sought to understand the needs of non-ambulatory Duchenne patients and, secondarily, to examine these needs in the context of the PUL-PROM-a validated patient-reported outcome measure of upper limb functioning.Non-ambulatory Duchenne patients or their caregivers from eight countries answered open-ended survey questions about patients' needs related to their most significant symptoms and important benefits of new treatments. The PUL-PROM was used to evaluate patients' upper limb functioning and was compared to data collected on non-ambulatory stage and quality of life. We thematically analyzed open-ended data, descriptively analyzed close-ended data, and compared themes by non-ambulatory stage.The study included 275 participants. Mean patient age was 24. Most patients were early-stage non-ambulatory (67%). Thematic analysis identified three congruent themes between significant symptoms and important benefits of new treatments: muscle functioning, especially upper limb function; body system functioning; and quality of life. Muscle functioning and body system functioning were endorsed more frequently in responses from early- and late-stage patients, respectively. Mean PUL-PROM total score was 22 with higher scores in early-stage patients (p ≤ 0.001). Upper limb function positively correlated with quality of life (r = 0.42, p ≤ 0.001).Non-ambulatory Duchenne patients want new treatments that improve upper limb functioning and body system functioning, and not exclusively regaining ambulation. The PUL-PROM can be used as a patient-centric measure that accounts for the needs of later-stage Duchenne patients.

Introduction: Electronic cigarettes are the most popular tobacco product among U.S. youth, and over 80% of current youth users of e-cigarettes use flavored e-cigarettes, with fruit, mint/menthol, and candy/sweets being the most popular flavors. A number of new e-liquid flavors are currently emerging in the online e-cigarette market. Menthol and other flavored e-cigarettes could incentivize combustible tobacco smokers to transition to e-cigarette use. Methods: From February to May 2021, we scraped data of over 14,000 e-liquid products, including detailed descriptions of their flavors, from five national online vape shops. Building upon the existing e-liquid flavor wheel, we expanded the semantic databases (i.e., key terms) to identify flavors using WordNet—a major database for keyword matching and group discussion. Using the enriched databases, we classified 14,000+ e-liquid products into the following 11 main flavor categories: “fruit”, “dessert/candy/sweets”, “coffee/tea”, “alcohol”, “other beverages”, “tobacco”, “mint/menthol”, “nuts”, “spices/pepper”, “other flavors”, and “unspecified flavor”. Results: We find that the most prominent flavor sold in the five online vape shop in 2021 was fruit flavored products, followed by dessert/candy/other sweets. Online vendors often label a product with several flavor profiles, such as fruit and menthol. Conclusions: Given that online stores market products with multiple flavor profiles and most of their products contain fruit flavor, the FDA may have issued marketing denial orders to some of these products. It is important to further examine how online stores respond to the FDA flavor restrictions (e.g., compliance or non-compliance).

IMPORTANCE: Research into the genetic and genomic (“genomics”) foundations of disease is central to our understanding of disease prevention, early detection, diagnostic accuracy, and therapeutic intervention. Inequitable participation in genomics research by historically excluded populations limits the ability to translate genomic knowledge to achieve health equity and ensure that findings are generalizable to diverse populations. OBSERVATIONS: We propose a novel framework for promoting diversity, equity, and inclusion in genomics research. Building on principles of community-based participatory research and collective impact frameworks, the framework can guide our understanding of the social, cultural, health system, policy, community, and individual contexts in which engagement and genomics research are being done. Our framework highlights the involvement of a multistakeholder team, including the participants and communities to be engaged, to ensure robust methods for recruitment, retention, return of genomic results, quality of engagement, follow-up, and monitoring of participants. CONCLUSIONS AND RELEVANCE: The proposed engagement framework will guide investigators in optimizing equitable representation in research and enhancing the rigor of genomics investigation.

Neoadjuvant therapy (NT) is increasingly being offered to patients with pancreatic ductal adenocarcinoma (PDAC) prior to surgical resection. However, the experience and quality of life (QOL) of patients undergoing NT are poorly understood.A systematic review of the Cinahl, Embase, Medline, Pubmed, Scopus, and Web of Science databases was conducted to evaluate the available literature pertaining to the experience and QOL of patient's undergoing NT for PDAC.Among 6041 articles screened, only six met criteria for full-text review including three prospective clinical trials of NT with QOL secondary endpoints. Overall, global QOL during or following NT did not significantly change from baseline. Pain scores seemed to improve during NT while the impact of NT on physical functioning varied across studies. No studies were identified evaluating other aspects of the patient experience.Although NT appears to have a minor impact on the QOL of patients with PDAC, this systematic review identified significant evidence gaps in the literature. A protocol of a prospective observational cohort study utilizing a digital smartphone app that aims to evaluate the patient experience and longitudinal QOL of patients with PDAC undergoing NT is presented.

Background Advance care planning (ACP) aids can help prepare patients, family members, and physicians for in-the-moment medical decision-making. We wished to describe the content and approach of paper-based ACP aids in order to characterize existing aids and inform the development of a new ACP aid. Methods Paper-based ACP aids were identified through an environmental scan and screened for eligibility. ACP conceptual frameworks and data were gathered via stakeholder engagement and used to inform the coding framework that two investigators used to independently code each aid. A directed content analysis was conducted on these eligible aids. Aids were categorized through a deliberative process with an investigator abstracting general information for each aid. Results Fifteen aids met the eligibility criteria. They ranged in length from 6 to 78 pages with the average aid written at an eighth-grade reading level. The content analysis revealed that many aids encouraged choosing a surrogate decision maker and informed users about legal medical documents. Fewer than half of the aids facilitated patient clarification of values regarding quality of life issues. The authors identified and termed the following three categories of aids: informative; semi-action oriented; and action-oriented. It was often unclear whether patients contributed to the development or testing of the ACP aids reviewed. Conclusions Most existing paper-based ACP aids address legal matters such as completing an advance directive. Only a minority elicited patient values and it was unclear whether any were developed in partnership with patients. Future development of ACP aids should account for patient preferences with a goal of supporting in-the-moment medical decision-making.

The Johns Hopkins University School of Medicine organized 2 multistakeholder symposia on February 2, 2018 and January 11, 2019 to address the problem of high graft failure in adolescent and young adult (AYA) solid organ transplant (SOT) recipients. Participants included international experts in transplantation, behavioral psychology, patient/parent advocacy, and technology. The objectives of the symposia were as follows: (1) to identify and discuss the barriers to and facilitators of effective transfer of care for AYA SOT recipients; (2) to actively explore strategies and digital solutions to promote their successful transfer of care; and (3) to develop meaningful partnerships for the successful development, evaluation, implementation, and dissemination of these digital solutions. Additionally, data were collected from 152 AYA SOT recipients demonstrating a substantial gap in how this population uses technologies for health-related activities, alongside an increased interest in an app to help them manage their transplant.

This systematic review identifies possible decision aids that promote perioperative advance care planning (ACP) and synthesizes the available evidence regarding their use. Using PubMed, EMBASE, Cochrane, SCOPUS, Web of Science, CINAHL, PsycINFO and Sociological Abstracts, researchers identified and screened articles for eligibility. Data were abstracted and risk of bias assessed for included articles. Thirty-nine of 5327 articles satisfied the eligibility criteria. Primarily completed in outpatient ambulatory populations, studies evaluated a variety of ACP decision aids. None were evaluated in a perioperative population. Fifty unique outcomes were reported with no head-to-head comparisons conducted. Findings are likely generalizable to a perioperative population and can inform development of a perioperative ACP decision aid. Future studies should compare the effectiveness of ACP decision aids.

Objective To identify videos on YouTube concerning advance care planning (ACP) and synthesise existing video content and style elements. Methods Informed by stakeholder engagement, two researchers searched YouTube for ACP videos using predefined search terms and snowballing techniques. Videos identified were reviewed and deemed ineligible for analysis if they: targeted healthcare professionals; contained irrelevant content; focused on viewers under the age of 18; were longer than 7 min in duration; received fewer than 150 views; were in a language other than English; or were a duplicate version. For each video, two investigators independently extracted general information as well as video content and stylistic characteristics. Results The YouTube search identified 23 100 videos with 213 retrieved for assessment and 42 meeting eligibility criteria. The majority of videos had been posted to YouTube since 2010 and produced by organisations in the USA (71%). Viewership ranged from 171 to 10 642. Most videos used a documentary style and featured healthcare providers (60%) rather than patients (19%) or families (45%). A minority of videos (29%) used upbeat or hopeful music. The videos frequently focused on completing legal medical documents (86%). Conclusions None of the ACP videos on YouTube went viral and a relatively small number of them contained elements endorsed by stakeholders. In emphasising the completion of legal medical documents, videos may have failed to support more meaningful ACP. Further research is needed to understand the features of videos that will engage patients and the wider community with ACP and palliative and end-of-life care conversations.

Patient-centered outcomes research (PCOR) methods and social learning theory (SLT) require intensive interaction between researchers and stakeholders. Advance care planning (ACP) is valuable before major surgery, but a systematic review found no extant perioperative ACP tools. Consequently, PCOR methods and SLT can inform the development of an ACP educational video for patients and families preparing for major surgery.The objective is to develop and test acceptability of an ACP video storyline.The design is a stakeholder-guided development of the ACP video storyline. Design-thinking methods explored and prioritized stakeholder perspectives. Patients and family members evaluated storyboards containing the proposed storyline.The study was conducted at hospital outpatient surgical clinics, in-person stakeholder summit, and the 2014 Maryland State Fair.Measurements are done through stakeholder engagement and deidentified survey.Stakeholders evaluated and prioritized evidence from an environmental scan. A surgeon, family member, and palliative care physician team iteratively developed a script featuring 12 core themes and worked with a medical graphic designer to translate the script into storyboards. For 10 days, 359 attendees of the 2014 Maryland State Fair evaluated the storyboards and 87% noted that they would be "very comfortable" or "comfortable" seeing the storyboard before major surgery, 89% considered the storyboards "very helpful" or "helpful," and 89% would "definitely recommend" or "recommend" this story to others preparing for major surgery.Through an iterative process utilizing diverse PCOR engagement methods and informed by SLT, storyboards were developed for an ACP video. Field testing revealed the storyline to be highly meaningful for surgery patients and family members.

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.A best-worst scaling survey (BWS) was administered to caregivers and patients. Across 9 profiles, respondents selected the best and worst attributes. Utility scores were estimated using mixed logistic regression.Respondents indicated greatest preference for therapies that maintain their current level of cough strength for 10 years or for 2 years. Preference scores for risks were low: 50% chance of diarrhea and 4 additional blood draws per year.There is a strong preference for pulmonary benefit and willingness to trade off risks and burden to achieve these benefits. In exchange for maintaining cough strength for 10 years, respondents were willing to tolerate high probabilities of diarrhea and additional blood draws. Muscle Nerve 55: 626-634, 2017.

Objectives:Schizophrenia imposes a great burden on society, and while evaluation should play an important role in informing society's efforts to alleviate these burdens, it is unclear what “endpoints” should be chosen as the objective of such analyses. The objectives of the study were to elicit endpoints directly from patients with schizophrenia, to ascertain whether patients are sufficiently cognoscente to express what endpoints are and are not important to them and to rank the relevant endpoints.Methods:We applied principles of patient-centered health technology assessment to identify and value endpoints from the patient's perspective. Focus groups were conducted to elicit endpoints, using interpretive phenomalogical analysis (IPA) to guide the collection, analysis and interpretation of data. Patient interviews were subsequently used to elicit patient preference over endpoints. Respondents were presented with cards outlining the endpoints and asked to remove irrelevant cards. They where then asked to identify and rank their five most relevant endpoints in order of importance. Interviews were recorded for the purposed of triangulation, and data was analyzed using descriptive statistics. Patients were recruited from five geographically diverse cities in Germany. Eligibility required a diagnosis of schizophrenia by a physician and treatment with an antipsychotic medication for at least one year. Respondents were excluded if they were experiencing an acute episode.Results:Thirteen endpoints emerged as important from the focus groups spanning side-effects, functional status, processes of care and clinical outcomes. Respondents could clearly identify relevant and irrelevant endpoints, and rank which factors were important to them. Triangulation between field notes of the ranking exercise and recordings confirmed that rankings were not arbitrary, but justified from the respondents' point of view.Conclusions:Patients with schizophrenia can express preferences over endpoints. Our results show that qualitative methods such as IPA can be used to identify factors, but ranking exercises provide a more robust method for ranking the importance of endpoints. Future research involving patients with schizophrenia ranking outcomes is needed to identify variations across patients and methods such as conjoint analysis could prove beneficial in identifying acceptable tradeoffs across endpoints.

Refractory overactive bladder (OAB) treatments, including sacral neuromodulation (SNM), onabotulinumtoxin A (OnabotA), and percutaneous tibial nerve stimulation (PTNS), differ considerably. Best-Worst Scaling (BWS) was used to assess patient preferences for these treatments.A cross-sectional Web survey, based on findings from qualitative interviews with 23 OAB patients and 7 clinical experts, was conducted with idiopathic OAB patients in the US and UK. The BWS exercise involved prioritizing subsets of 13 attributes across 13 choice tasks, where patients identified the attribute they considered as best and as worst in each task. Attribute BWS scores, ranging from -1.0 (worst) to 1.0 (best) were calculated based on the rates that each attribute was chosen. Attitudes toward the attributes also were assessed via like/dislike Likert scales, and questions regarding the percentage likelihood (0-100%) of trying each treatment, based on standardized treatment descriptions.The sample included 245 patients (118 US, 127 UK); 79% female, mean age 50 ± 7.8 years. All 13 attribute BWS scores were significantly positive or negative. "Lasting improvement" (0.82), "minimal side effects" (0.67), and "sends signals between bladder and brain" (0.35)' were rated most favorably, and "complications with implant" (-0.65), and "be willing to self-catheterize" (-0.53) were rated worst. All but one of the attribute BWS scores were significantly correlated with the respective like/dislike scores and the percentage likelihood estimates for trying one of the three treatments.BWS was successful in assessing the magnitude of patient preferences for key attributes associated with substantially different refractory OAB therapies. Neurourol. Urodynam. 35:1028-1033, 2016. © 2015 Wiley Periodicals, Inc.

Internationally, there has been a movement in medicine to better involve patients in decision making, whether it be at the individual or aggregate level. However, the German health sector has been slow to understand and accommodate the preferences of patients. This paper discusses the reasons why and attempts to highlight some of the consequences of not involving patients in healthcare decision making within the German context. We argue that a fundamental paradigm shift at all levels of the German health system, including better doctor/patient communication, is necessary for more choices to be available to patients and to better reflect patient preferences in centralised healthcare decision making. The newly created German health technology assessment agency (IQWiG; Institute for Quality and Economic Efficiency in the Health Care Sector) has indicated that they are interested in focusing on patient preferences, but there is a need for them to clarify what methods they deem suitable for doing so.

Introduction Despite positive health outcomes associated with advance care planning (ACP), little research has investigated the impact of ACP in surgical populations. Our goal is to evaluate how an ACP intervention video impacts the patient centredness and ACP of the patient-surgeon conversation during the presurgical consent visit. We hypothesise that patients who view the intervention will engage in a more patient-centred communication with their surgeons compared with patients who view a control video. Methods and analysis Randomised controlled superiority trial of an ACP video with two study arms (intervention ACP video and control video) and four visits (baseline, presurgical consent, postoperative 1 week and postoperative 1 month). Surgeons, patients, principal investigator and analysts are blinded to the randomisation assignment. Setting Single, academic, inner city and tertiary care hospital. Data collection began July 16, 2015 and continues to March 2017. Participants Patients recruited from nine surgical oncology clinics who are undergoing major cancer surgery. Interventions In the intervention arm, patients view a patient preparedness video developed through extensive engagement with patients, surgeons and other stakeholders. Patients randomised to the control arm viewed an informational video about the hospital surgical programme. Main outcomes and measures Primary Outcome: Patient centredness and ACP of patient-surgeon conversations during the presurgical consent visit as measured through the Roter Interaction Analysis System. Secondary outcomes: patient Hospital Anxiety and Depression Scale score; patient goals of care; patient, companion and surgeon satisfaction; video helpfulness; medical decision maker designation; and the frequency patients watch the video. Intent-to-treat analysis will be used to assess the impact of video assignment on outcomes. Sensitivity analyses will assess whether there are differential effects contingent on patient or surgeon characteristics. Ethics and dissemination This study has been approved by the Johns Hopkins School of Medicine institutional review board and is registered on clinicaltrials.gov (NCT02489799, First received: July 1, 2015). Trial registration number clinicaltrials.gov, NCT02489799.

The health and economic burden from liver disease in the United States is substantial and rising. The objective of this study was to characterize temporal trends in mortality from chronic liver disease and liver cancer and the incidence of associated risk factors using population-based data over the past 30 years.Population-based mortality data were obtained from the National Vital Statistics System, and population estimates were derived from the national census for US adults (aged45 years). Crude death rates (CDRs), age-adjusted death rates (ADRs), and average annual percentage change (AAPC) statistics were calculated.In total, 690,414 deaths (1.1%) were attributable to chronic liver disease, whereas 331,393 deaths (0.5%) were attributable to liver cancer between 1981 and 2010. The incidence of liver cancer was estimated at 7.1 cases per 100,000 population. Mortality rates from chronic liver disease and liver cancer increased substantially over the past 3 decades, with ADRs of 23.7 and 16.6 per 100,000 population in 2010, respectively. The AAPC from 2006 to 2010 demonstrated an increased ADR for chronic liver disease (AAPC, 1.5%; 95% confidence interval, 0.3%-2.8%) and liver cancer (AAPC, 2.6%; 95% confidence interval, 2.4%-2.7%).A comprehensive approach that involves primary and secondary prevention, increased access to treatment, and more funding for liver-related research is needed to address the high death rates associated with chronic liver disease and liver cancer in the United States.

Hepatocellular carcinoma (HCC) is particularly burdensome in the Asia-Pacific region, however, cross-country comparisons have been limited to somewhat unreliable epidemiological measures. We conducted a comparative needs assessment for HCC control policy to inform HCC control efforts in the Asia-Pacific region. The aims were to identify regional needs, to compare overall competence across the region, and to identify which needs were concordant across the region.Using the self-explicated method, a stated-preference approach, clinical experts from Australia, China, Japan, Korea, Taiwan, and the United States valued ten previously identified dimensions of HCC control: clinical education; risk assessment; HBV strategy; HCV strategy; life-style risk factors; national statistics; funding for screening; funding for treatment; political awareness; and public awareness. Results were normalized and analyzed using Z-scores and ANOVA, with concordance of need across the region tested via the F-test.Seventy-two respondents, equally drawn from the study sites, completed the survey (response rate: 36%). Respondents were hepatologists (39%), oncologists (21%), radiologists (17%), surgeons (17%), and other specialists (7%) who were involved in liver cancer control at local/regional (35%) national (44%) or international (21%) levels. In aggregate, the most significant needs were political awareness, public awareness, and life-style risk factors (all p0.001). Significant differences in aggregate competence were observed across the region (p0.001), with better than expected competence reported by respondents from Taiwan (p0.001), Japan (p=0.006), and Korea (0.041), and close to expected competence reported by respondents from Australia, China, and USA (all p0.05). There were differences in the extent of needs across the region (p0.05) on all dimensions except funding for screening, clinical education and life style risk factors.As the first comparative needs assessment for HCC for the Asia-Pacific region, our results can inform national and cross-national priorities for intervention and facilitate the identification of best practices. Regional efforts to control HCC should adopt as objectives the needs for greater political and public awareness and improved management of lifestyle risk factors because these are the most significant needs, and are shared concerns across the region.

Clinical and healthcare decision makers have repeatedly endorsed patient-centered care as a goal of the health system. However, traditional methods of evaluation reinforce societal views, and research focusing on views of patients is often referred to as 'soft science.' Conjoint analysis presents a scientifically rigorous research tool that can be used to understand patient preferences and inform decision making. This paper documents applications of conjoint analysis in medicine and systematically reviews this literature in order to identify publication trends and the range of topics to which conjoint analysis has been applied. In addition, we document important methodological aspects such as sample size, experimental design, and method of analysis.Publications were identified through a MEDLINE search using multiple search terms for identification. We classified each article into one of three categories: clinical applications (n = 122); methodological contributions (n = 56); and health system applications (n = 47). Articles that did not use or adequately discuss conjoint analysis methods (n = 164) were discarded. We identified a near exponential increase in the application of conjoint analyses over the last 10 years of the study period (1997-2007). Over this period, the proportion of applications on clinical topics increased from 40% of articles published in MEDLINE from 1998 to 2002, to 64% of articles published from 2003 to 2007 (p = 0.002).The average sample size among articles focusing on health system applications (n = 556) was significantly higher than clinical applications (n = 277) [p = 0.001], although this 2-fold difference was primarily due to a number of outliers reporting sample sizes in the thousands. The vast majority of papers claimed to use orthogonal factorial designs, although over a quarter of papers did not report their design properties. In terms of types of analysis, logistic regression was favored among clinical applications (28%), while probit was most commonly used among health systems applications (38%). However, 25% of clinical applications and 33% of health systems articles failed to report what regression methods were used. We used the International Classification of Diseases - version 9 (ICD-9) coding system to categorize clinical applications, with approximately 26% of publications focusing on neoplasm. Program planning and evaluation applications accounted for 22% of the health system articles.While interest in conjoint analysis in health is likely to continue, better guidelines for conducting and reporting conjoint analyses are needed.

Background Liver cancer is both common and burdensome in Asia. Effective liver cancer control, however, is hindered by a complex etiology and a lack of coordination across clinical disciplines. We sought to identify strategies for inclusion in a comprehensive liver cancer control for Asia and to compare qualitative and quantitative methods for prioritization. Methods Qualitative interviews (N = 20) with international liver cancer experts were used to identify strategies using Interpretative Phenomenological Analysis and to formulate an initial prioritization through frequency analysis. Conjoint analysis, a quantitative stated-preference method, was then applied among Asian liver cancer experts (N = 20) who completed 12 choice tasks that divided these strategies into two mutually exclusive and exhaustive subsets. Respondents' preferred plan was the primary outcome in a choice model, estimated using ordinary least squares (OLS) and logistic regression. Priorities were then compared using Spearman's Rho. Results Eleven strategies were identified: Access to treatments; Centers of excellence; Clinical education; Measuring social burden; Monitoring of at-risk populations; Multidisciplinary management; National guidelines; Public awareness; Research infrastructure; Risk-assessment and referral; and Transplantation infrastructure. Qualitative frequency analysis indicated that Risk-assessment and referral (85%), National guidelines (80%) and Monitoring of at-risk populations (80%) received the highest priority, while conjoint analysis pointed to Monitoring of at-risk populations (p < 0.001), Centers of excellence (p = 0.002), and Access to treatments (p = 0.004) as priorities, while Risk-assessment and referral was the lowest priority (p = 0.645). We find moderate concordance between the qualitative and quantitative methods (rho = 0.20), albeit insignificant (p = 0.554), and a strong concordance between the OLS and logistic regressions (rho = 0.979; p < 0.0001). Conclusions Identified strategies can be conceptualized as the ABCs of comprehensive liver cancer control as they focus on Antecedents, Better care and Connections within a national strategy. Some concordance was found between the qualitative and quantitative methods (e.g. Monitoring of at-risk populations), but substantial differences were also identified (e.g. qualitative methods gave highest priority to risk-assessment and referral, but it was the lowest for the quantitative methods), which may be attributed to differences between the methods and study populations, and potential framing effects in choice tasks. Continued research will provide more generalizable estimates of priorities and account for variation across stakeholders and countries.

While much discussion has been placed on the problem of poor compliance in the treatment of schizophrenia, there has been little discussion on the concordance between patients and psychiatrists, an important contributing factor to patient-centred care.To estimate the concordance between patients' and psychiatrists' (ordinal and cardinal) valuations of multiple goals for schizophrenia treatment and to illustrate the utility of the self-explicated method in valuing a large number of treatment goals.Twenty treatment goals were identified during focus groups and literature review and were presented to patients and psychiatrists during structured interviews. Respondents were asked to rank the multiple treatment goals and rate them on a 5-point Likert scale. Three scores were calculated based on the ranking (1-20), rating (Likert scale) (1-5) and a self-explicated method estimated as the product of rating and ranking score (1-100). Concordance was tested using Spearman's rho for overall ordinal rankings and via anova and F-test for the cardinal values assigned to a specific treatment goal.A total of 105 outpatients diagnosed with schizophrenia and 160 psychiatrists in Germany.Patient and psychiatrist values were concordant when the ordinal properties of their valuations were assessed by rating (ρ = 0.63; P = 0.002), ranking (ρ = 0.51; P = 0.02) and self-explicated methods (ρ = 0.54; P = 0.01). Significant discordances were found when comparing the cardinal value placed on any given treatment goal using all three approaches, but the self-explicated method produced a more discerning statistic. Relative to patients, psychiatrists significantly (P0.05) overvalued reduced lack of emotion, improved sexual pleasure and improved communication while undervaluing reuptake of activities of daily living, improved satisfaction and recovered capacity for work.While there is an overall concordance between patients' and psychiatrists' valuation, significantly different valuations on specific goals can be identified. Here, psychiatrists tend to focus on 'textbook' outcomes, while patients are more concerned with functioning and living a normal life. This study also demonstrates the importance of comparing the concordance in treatment goals and the importance of preference-based methods, such as the self-explicated method, in the study of concordance.

Although the US has lagged behind international developments in health technology assessment (HTA), renewed interest in HTA in the US has been fueled by the appropriation of $1.1 billion comparative effectiveness research (CER) in 2009 and the debate over health care reform.To inform CER practices in the US, we present case studies of HTA from England/Wales and Germany: contrasting methods; relevance to the US; and impact on innovation.The National Institute of Health and Clinical Excellence (NICE) was established in 1999 to inform trusts within the National Health Service of England and Wales. It uses cost-effectiveness analysis to guide the allocation resource across preventative and curative interventions. In Germany, the Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen (IQWiG) was established in 2004 to inform reimbursement and pricing policies for the statutory sickness funds set by the Gemeinsamer Bundesaursschuss (G-BA). IQWiG evaluates competing technologies within specific therapeutic areas, placing more weight on clinical evidence and the relative efficiency of competing therapies.Although having deep political and cultural antecedents, differences between NICE and IQWiG can be explained by perspective: the former guiding resource allocation across an entire system (macro-evaluation), the latter focusing on efficiency within the bounds of a particular therapeutic area (micro-evaluation). Given the decentralized nature of the US health care system, and the relative powers of different medical specialties, the IQWiG model presents a more suitable case study to guided CER efforts in the US.

One focus of health economics is the trade-off between limited resources and the (health) needs of a community. Cost-effectiveness analysis (CEA), while being one of the most accepted evaluation methodologies in health economics, does not account for many important costs and benefits of health care interventions. Some health economists have attempted to modify CEA to account for these deficiencies, while others have been working on alternative methodologies. One group of alternative methodologies can be described as stated preference techniques. These aim to measure both health and non-health outcomes (ie costs and benefits), and include qualitative analysis, conjoint analysis (often referred to as discrete choice analysis/modelling) and willingness to pay (or contingent valuation). This paper provides an overview of stated preference techniques in health economics, with particular focus on their strengths as compared with traditional evaluation methods in health care. The limitations and policy implications of these methods are also discussed.

Background Breast cancer is the most frequent cause of cancer death in women worldwide, but global disparities in breast cancer control persist, due to a lack of a comprehensive breast cancer control strategy in many countries. Objectives To identify and compare the need for breast cancer control strategies in Asia, Latin America and the Middle East/North Africa and to develop a common framework to guide the development of national breast cancer control strategies. Methods Data were derived from open-ended, semi-structured interviews conducted in 2007 with 221 clinicians, policy makers, and patient advocates; stratified across Asia (n = 97), Latin America (n = 46), the Middle East/North Africa (ME/NA) (n = 39) and Australia and Canada (n = 39). Respondents were identified using purposive and snowballing sampling. Interpretation of the data utilized interpretive phenomenological analysis where transcripts and field notes were coded and analyzed and common themes were identified. Analysis of regional variation was conducted based on the frequency of discussion and the writing of the manuscript followed the RATS guidelines. Results Analysis revealed four major themes that form the foundation for developing national breast cancer control strategies: 1) building capacity; 2) developing evidence; 3) removing barriers; and 4) promoting advocacy - each specified across five sub-ordinate dimensions. The propensity to discuss most dimensions was similar across regions, but managing advocacy was discussed more frequently (p = 0.004) and organized advocacy was discussed less frequently (p < 0.001) in Australia and Canada. Conclusions This unique research identified common themes for the development of breast cancer control strategies, grounded in the experience of local practitioners, policy makers and advocacy leaders across diverse regions. Future research should be aimed at gathering a wider array of experiences, including those of patients.