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Background:Engaging diverse populations in cancer genomics research is of critical importance and is a fundamental goal of the NCI Participant Engagement and Cancer Genome Sequencing (PE-CGS) Network. Established as part of the Cancer Moonshot, PE-CGS is a consortium of stakeholders including clinicians, scientists, genetic counselors, and representatives of potential study participants and their communities. Participant engagement is an ongoing, bidirectional, and mutually beneficial interaction between study participants and researchers. PE-CGS sought to set priorities in participant engagement for conducting the network's research.Methods:PE-CGS deliberatively engaged its stakeholders in the following four-phase process to set the network's research priorities in participant engagement: (i) a brainstorming exercise to elicit potential priorities; (ii) a 2-day virtual meeting to discuss priorities; (iii) recommendations from the PE-CGS External Advisory Panel to refine priorities; and (iv) a virtual meeting to set priorities.Results:Nearly 150 PE-CGS stakeholders engaged in the process. Five priorities were set: (i) tailor education and communication materials for participants throughout the research process; (ii) identify measures of participant engagement; (iii) identify optimal participant engagement strategies; (iv) understand cancer disparities in the context of cancer genomics research; and (v) personalize the return of genomics findings to participants.Conclusions:PE-CGS is pursuing these priorities to meaningfully engage diverse and underrepresented patients with cancer and posttreatment cancer survivors as participants in cancer genomics research and, subsequently, generate new discoveries.Impact:Data from PE-CGS will be shared with the broader scientific community in a manner consistent with participant informed consent and community agreement. more...

Introduction Limited research has explored sex differences in the relationship between partner support and smoking cessation among minority low-income population. Further, scarce attention has been given to the influence of partners who are not married. The purpose of this study is to examine the relationship between negative and positive social support provided by partners and smoking cessation among men and women smokers. Methods Data were collected as part of the Tobacco Use in Drug Environment (TIDE) study, a cross-sectional study conducted in Baltimore, Maryland, from September 2013 to May 2015. Interviews were administered with current smokers. The sample size for the current analysis was 134 men and 86 women. Results Approximately 33% of male participants (n=45) reported currently trying to quit smoking cigarettes and 29% of women were currently trying to quit. Having a sex partner who did not mind the participant’s smoking was associated with decreased odds of trying to quit among men (AOR=0.35; 95% CI: 0.13–0.91, p=0.03). Having a sex partner who expressed concern about the participant’s smoking (AOR=12.9; 95% CI: 3.49–47.0, p more...

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. more...

Context Although patients with acute myeloid leukemia (AML) experience significant toxicities and poor outcomes, few studies have quantified patients' experience. Methods A community-centered approach was used to develop an AML-specific best-worst scaling (BWS) instrument involving 13 items in four domains (psychological, physical, decision-making, and treatment delivery) to quantify patient worry. A survey of patients and caregivers was conducted using the instrument. Data were analyzed using conditional logistic regression. Results The survey was completed by 832 patients and 237 caregivers. Patients were predominantly white (88%), married/partnered (72%), and in remission (95%). The median age was 55 years (range: 19-87). Median time since diagnosis was 8 years (range: 1-40). Patients worried most about "the possibility of dying from AML" (BWS score = 15.5, confidence interval [CI] [14.2-16.7]) and "long-term side effects of treatments" (14.0, CI [12.9-15.2]). Patients found these items more than twice as worrisome as all items within the domains of care delivery and decision-making. Patients were least worried about "communicating openly with doctors" (2.50, CI [1.97-3.04]) and "having access to the best medical care" (3.90, CI [3.28-4.61]). Caregiver reports were highly correlated to patients' (Spearman's ρ = 0.89) though noted significantly more worry about the possibility of dying and spending time in the hospital. Conclusion This large convenience sample demonstrates that AML patients have two principal worries: dying from their disease and suffering long-term side effects from treatment. To better foster patient-centered care, therapeutic decision-making and drug development should reflect the importance of both potential outcomes. Further work should explore interventions to address these worries. more...

BackgroundDuchenne muscular dystrophy is a rare degenerative neuromuscular disorder with pediatric onset. Recent approvals in Duchenne have placed attention on the economic evaluation in pricing and reimbursement decisions across a range of rare conditions. We sought to assess the appropriateness of the EQ-5D, a common measure of generic health state utility, for use among patients and caregivers affected by Duchenne.MethodsAn international, cross-sectional sample of adults with Duchenne and caregivers reported patient health status using self- or proxy-reported EQ-5D-3L. Appropriateness was assessed across 6 domains of concern raised by stakeholders in Duchenne. These concerns were that the EQ-5D/EQ-VAS would not capture meaningful differences in health status, correlate with disease-specific measures, reflect real health status, exhibit face validity, be accurately interpreted, and be low burden. We evaluated these concerns by comparing EQ-5D index score and EQ-VAS scores to other condition-specific functional measures and open- and closed-ended questions.ResultsIn total, 263 participants (74% response) completed the survey, 24% of whom were adult patients. Most participants lived in the United States or United Kingdom (58%). Patient age ranged from 1 to 48 y. EQ-5D index was higher in ambulatory than nonambulatory patients (0.60 v. 0.30, P < 0.001) and was negatively correlated with upper limb impairment ( r = 0.61, P < 0.001). Three-quarters of respondents agreed that EQ-5D measured real health status (74%). Most respondents interpreted EQ-VAS anchors of best and worst imaginable health as full health (61%) and death/near death (58%). Respondents indicated the EQ-5D was easy to understand (86%) and answer (71%).ConclusionsContrary to anecdotal concerns, we found support for the appropriateness of EQ-5D to assess health status in Duchenne. While other measures may be more sensitive to specific outcomes in Duchenne, there may be some value in results using the EQ-5D measure. more...

Background Many human immunodeficiency virus (HIV) prevention technologies (pre-exposure prophylaxis, microbicides, vaccines) are available or in development. Preference elicitation methods provide insight into client preferences that may be used to optimize products and services. Given increased utilization of such methods in HIV prevention, this article identifies and reviews these methods and synthesizes their application to HIV prevention technologies. Methods In May 2020, we systematically searched peer-reviewed literature in PubMed, CINAHL, and Web of Science for studies employing quantitative preference elicitation methods to measure preferences for HIV prevention technologies among populations of any age, sex, or location. Quality assessment used an existing checklist (PREFS) and a novel adaptation of the Newcastle–Ottawa Scale (PROSPERO #CRD42018087027). Results We screened 5022 titles and abstracts, reviewed 318 full texts, and included 84 studies. Common methods employed were discrete-choice experiment (33%), conjoint analysis (25%), and willingness-to-participate/try/accept (21%). Studies were conducted in 25 countries and had a mean of 768 participants (range = 26–7176), two-thirds of them male. Common HIV prevention technologies included pre-exposure prophylaxis (23%), voluntary testing and counseling (19%), HIV self-testing (17%), vaccines (15%), and topical microbicides (9%). Most attributes focused on product design (side effects, frequency), service design (provider type, location), acceptability or willingness to accept/pay; results are summarized in these categories, by prevention type. Mean quality-adapted Newcastle–Ottawa Scale score was 4.5/8 (standard deviation = 2.1) and mean PREFS scores was 3.47/5 (standard deviation = 0.81). Conclusions This review synthesizes extant literature on quantitative measurement of preferences for HIV prevention technologies. This can enable practitioners to improve prevention products and interventions, and ultimately reduce HIV incidence. more...

Randomized clinical trials are critical for evaluating the safety and efficacy of interventions in oncology and informing regulatory decisions, practice guidelines, and health policy. Patient-reported outcomes (PROs) are increasingly used in randomized trials to reflect the impact of receiving cancer therapies from the patient perspective and can inform evaluations of interventions by providing evidence that cannot be obtained or deduced from clinicians’ reports or from other biomedical measures. This commentary focuses on how PROs add value to clinical trials by representing the patient voice. We employed 2 previously published descriptive frameworks (addressing how PROs are used in clinical trials and how PROs have an impact, respectively) and selected 9 clinical trial publications that illustrate the value of PROs according to the framework categories. These include 3 trials where PROs were a primary trial endpoint, 3 trials where PROs as secondary endpoints supported the primary endpoint, and 3 trials where PROs as secondary endpoints contrast the primary endpoint findings in clinically important ways. The 9 examples illustrate that PROs add valuable data to the care and treatment context by informing future patients about how they may feel and function on different treatments and by providing clinicians with evidence to support changes to clinical practice and shared decision making. Beyond the patient and clinician, PROs can enable administrators to consider the cost-effectiveness of implementing new interventions and contribute vital information to policy makers, health technology assessors, and regulators. These examples provide a strong case for the wider implementation of PROs in cancer trials. more...

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. more...

Background: Declines in smoking prevalence among low-income adults lag behind national samples. Understanding the influence of social context factors such as gender, and normative influence on smoking attitudes and behaviors, can inform smoking cessation interventions. Objective: This study explored how gender, smoking dependence, and cessation norms influenced the likelihood of current quit attempts among urban-dwelling, predominately African American adults. Methods: Participants answered questions about their current quit attempts, smoking dependence (heaviness of smoking index [HSI]), and cessation norms (descriptive: having friends who quit smoking; injunctive: friends disapproving of smoking) as a part of a parent study exploring social and environmental factors in tobacco use. Logistic regression stratifying by gender and adjusting for demographics examined main and interaction effects of norms and HSI on odds of having a current quit attempt. Results: Among men, having a higher smoking dependence was associated with a reduced likelihood of trying to quit (AOR = 0.30 [0.15-0.59]), but this effect was moderated by having friends who had quit smoking (AOR = 2.40 [1.20-4.78]). When accounting for the effect of friends quitting smoking, men were not influenced by friends disapproving of smoking. Among women, currently attempting to quit was predicted by neither smoking dependence nor norms. Conclusions/Importance: Cessation norms and smoking dependence influenced the likelihood of quit attempts for men, but not women, in this study. This highlights the importance of conducting gender stratification in smoking cessation research so as to better understand how social and environmental factors differently impact cessation outcomes for men and women. more...

Objective: Acute myeloid leukemia (AML) is experiencing a therapeutic renaissance due to the heightened biomedical understanding of AML and patient-focused drug development (PFDD). Many AML patient... more...

256 Background: Numerous studies have demonstrated that physician perceptions of patient priorities and patients’ stated preferences differ substantially. Use of preference clarification using discrete choice experiments (DCEs) has been shown to improve preference-concordance for patients with solid tumors. Previously, we developed a DCE to describe preferences of survivors of acute myeloid leukemia (AML). We sought to evaluate this DCE among newly diagnosed patients with AML to inform shared decision-making. Methods: We used a sequential explanatory (quantitative to qualitative) mixed methods design to assess acceptability, feasibility, and content validity of the DCE to elicit individual preferences. Newly diagnosed older (≥ 60 years) adults with AML completed the DCE at the time of treatment decision. Patients and caregivers then completed semi-structured interviews to expand on quantitative findings. Based on initial results, healthy volunteers completed think-aloud sessions while completing the DCE and semi-structured interviews to further evaluate comprehensibility. Results: 47 participants were enrolled (18 patients [8 female, 10 male; aged 60-87], 16 caregivers, 15 healthy volunteers). Patients received best supportive care (n = 1), hypomethylating agents (n = 9), and high-intensity chemotherapy (n = 8). Feasibility/Acceptability: All patients completed the DCE (100% feasibility), and all reported that they answered according to their preferences (100% acceptability). Content Validity: 1) Relevance: 17/18 (94%) felt the DCE was relevant. 2) Comprehensiveness: In addition to the attributes in the DCE, provider recommendations and family considerations were noted to be critical in treatment decision-making. 3) Comprehensibility: Only 13/18 (72%) felt the DCE was easy to understand, and 14/18 (78%) felt it was easy to answer. Explanatory Interviews: Patients reported being in “shock,” “devastated” from receiving the diagnosis, feeling overwhelmed (“I was floored”) and not being able to reliably concentrate on the DCE due to these factors. Caregivers corroborated patients’ reports. Healthy volunteers also reported “information overload” and that the DCE was “technical” and the included outcome levels were “confusing.”. Conclusions: This DCE designed for older adults with AML was relevant, feasible, and acceptable. However, nearly 30% of patients reported difficulty understanding the DCE. Some reported not being able to attend to the complex tasks in the DCE, thereby compromising content validity of the measure. We suspect this was due to the distress caused by the diagnosis. This study demonstrates the challenge of developing valid preference elicitation instruments for newly diagnosed patients and highlights the need for extensive evaluation prior to clinical implementation. more...

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. more...

Background Artificial intelligence (AI) is rapidly expanding in medicine despite a lack of consensus on its application and evaluation. Objective We sought to identify current frameworks guiding the application and evaluation of AI for predictive analytics in medicine and to describe the content of these frameworks. We also assessed what stages along the AI translational spectrum (ie, AI development, reporting, evaluation, implementation, and surveillance) the content of each framework has been discussed. Methods We performed a literature review of frameworks regarding the oversight of AI in medicine. The search included key topics such as “artificial intelligence,” “machine learning,” “guidance as topic,” and “translational science,” and spanned the time period 2014-2022. Documents were included if they provided generalizable guidance regarding the use or evaluation of AI in medicine. Included frameworks are summarized descriptively and were subjected to content analysis. A novel evaluation matrix was developed and applied to appraise the frameworks’ coverage of content areas across translational stages. Results Fourteen frameworks are featured in the review, including six frameworks that provide descriptive guidance and eight that provide reporting checklists for medical applications of AI. Content analysis revealed five considerations related to the oversight of AI in medicine across frameworks: transparency, reproducibility, ethics, effectiveness, and engagement. All frameworks include discussions regarding transparency, reproducibility, ethics, and effectiveness, while only half of the frameworks discuss engagement. The evaluation matrix revealed that frameworks were most likely to report AI considerations for the translational stage of development and were least likely to report considerations for the translational stage of surveillance. Conclusions Existing frameworks for the application and evaluation of AI in medicine notably offer less input on the role of engagement in oversight and regarding the translational stage of surveillance. Identifying and optimizing strategies for engagement are essential to ensure that AI can meaningfully benefit patients and other end users. more...

Healthcare treatments and interventions are traditionally evaluated from the societal perspective, but a more patient-centric perspective has been proposed in recent years. We sought to compare preferences of patients and the general public for treatment outcomes of type 2 diabetes using both best–worst scaling (BWS) and rating approaches. A survey evaluating the treatment priorities for type 2 diabetes was conducted in the United States. Members of the general public and patients with type 2 diabetes were recruited from a nationally sampled panel. Participants indicated the importance of seven potential treatment outcomes (hypoglycemic events, glycated hemoglobin [A1c], weight loss, mental health, functioning, glycemic stability, and cardiovascular health) using (1) BWS case 1 and (2) a rating task. Preference differences from BWS prioritizations were explored using mixed logistic regression (BWS preference weights were probability re-scaled so that the weightings of the seven items collectively summed to 100). The consistency of scale between samples was explored using heteroskedastic conditional logistic regression of BWS data. Spearman rank correlation was used to compare standardized BWS preference weights and rating scores for each group. Both groups evaluated the BWS and rating activities using debriefing questions. The public and patient samples included 314 and 313 respondents, respectively. The public was on average 16 years younger than patients (48 vs 64 years, P more...

Patient-reported outcomes (PROs) are increasingly used in clinical trials to provide patients’ perspectives regarding symptoms, health-related quality of life, and satisfaction with treatments. A range of guidance documents exist for the selection of patient-reported outcome measures (PROMs) in clinical trials, and it is unclear to what extent these documents present consistent recommendations. We conducted a targeted review of publications and regulatory guidance documents that advise on the selection of PROMs for use in clinical trials. A total of seven guidance documents from the US Food and Drug Administration, European Medicines Agency, and scientific consortia from professional societies were included in the final review. Guidance documents were analyzed using a content analysis approach comparing them with minimum standards recommended by the International Society for Quality of Life Research. Overall there was substantial agreement between guidance regarding the appropriate considerations for PROM selection within a clinical trial. Variations among the guidance primarily related to differences in their format and differences in the perspectives and mandates of their respective organizations. Whereas scientific consortia tended to produce checklist or rating-type guidance, regulatory groups tended to use more narrative-based approaches sometimes supplemented with lists of criteria. The consistency in recommendations suggests an emerging consensus in the field and supports use of any of the major guidance documents available to guide PROM selection for clinical trials without concern of conflicting recommendations. This work represents an important first step in the international PROTEUS Consortium’s ongoing efforts to optimize the use of PROs in clinical trials. more...

Background: Limited research has explored sex differences in the relationship between partner behavior and attitudes and smoking cessation. Methods: The study examined sex partner attitudes about smoking among men and women who are currently trying to quit smoking cigarettes. Data were collected as part of the Tobacco Use in Drug Environment (TIDE) study, a cross-sectional study conducted in Baltimore, MD, USA from September 2013-May 2015. Interviews were administered with current smokers. The sample size for the current analysis was 134 men and 86 women. Results: Approximately thirty-three percent of male participants (n=45) reported currently trying to quit smoking cigarettes. Twenty-nine percent of women were currently trying to quit. Having a sex partner who did not mind the participant’s smoking was associated with decreased odds of trying to quit among men (AOR=0.35, p=0.03, 95% CI: 0.13, 0.91). Having a sex partner who expressed concern about the participant’s smoking (AOR=12.9, pConclusions: The relationship between partner support for cessation and current quit attempt was stronger for women than men in this population. Understanding sex-partner attitudes regarding smoking and their relationship to smoking cessation activities may provide insights for future tailored cessation interventions. more...

The current review highlights the growing number of available methods used to measure patient preferences and discusses how this impacts preference research in rheumatology. Spurred by the growing role of preferences in regulatory decisions and drug development, researchers have begun applying preference methods to study questions beyond the clinical context. We explore these trends, provide case studies highlighting changes in measuring patient preferences, compare strengths and weaknesses of common stated-preference methods, and discuss considerations for the future use of these methods.Early literature on patient preferences often mimicked clinical practice, asking whether treatment A is better or worse than treatment B for a patient. Early applications of patient preference methods in rheumatology aimed to value different attributes of treatments, but remained focused on informing clinical questions. Spurred by interest in preferences by regulatory agencies and patient-centeredness throughout the product lifecycle, there are now a wide array of methods available to measure preference.Although these different preference methods have strengths and weaknesses, they serve to highlight the broad number of questions that could help rheumatology beyond the clinical context. Researchers in rheumatology now have the opportunity to better serve diverse stakeholders by considering how these methods could aid in clinical trial design, regulatory policy, and other elements of the medical product life cycle. more...

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. more...

Best–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 would accept a discrete-choice experiment (DCE) that incorporates uncertainty regarding individual-level benefit. A 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 uncertainty attribute. The DCE assessed preferences across a primary outcome (muscle strength) and several risks (uncertainty regarding treatment benefit, kidney damage risk, and fracture risk). The single instrument was tested among adult patients, caregivers, and professionals at the national Parent Project Muscular Dystrophy annual meeting. The DCE was analyzed using conditional logit. Instrument acceptability was evaluated using a previously developed set of questions assessing ease of understanding and answering, and if answers reflected the respondents’ real preferences. We proposed a 75% agreement rate as a threshold of acceptability, and used a Z score to assess if this was met, exceeded, or rejected. A total of 161 people completed the survey including 9 patients, 87 caregivers, and 65 professionals. Patients reported high acceptability across all evaluation items (p values > 0.21). Caregivers and professionals exceeded the benchmark of acceptability on understanding and reflecting real preferences (p more...

Background Post-traumatic stress disorder (PTSD) prevalence is high, but not well-understood, among women living in urban, impoverished areas. Although previous studies have established social support as an important factor in PTSD development and maintenance, little is known about how perceptions of neighborhood are linked to PTSD. This study examined the relationship between PTSD and social network and neighborhood factors among women with a low socioeconomic status. Methods We analyzed cross-sectional data collected from a human immunodeficiency virus/sexually transmitted infection peer network study in Baltimore, Maryland (n = 438). We used bivariate analyses to examine the associations between PTSD and social network characteristics and time in neighborhood and satisfaction. We then constructed multivariable regression models that controlled for the following with PTSD: homelessness, cocaine/heroin use, and unemployment. Main Findings Overall, 30% of women had PTSD symptom severity consistent with a clinical diagnosis. In the multivariable model, dissatisfaction with neighborhood block (odds ratio [OR], 1.80; p = .03) and living in one's neighborhood for more than 5 years (OR, 1.69; p = .03) were associated with PTSD. Social network factors that were significantly associated with PTSD included a higher number of network members in conflict with the participant (OR, 1.28; p = .02), presence of a network member who would let the participant stay with them (OR, 0.4; p = .004), and the number of network members with whom the participant socialized (OR, 0.6; p = .04). Conclusions In this sample of impoverished urban women with a high prevalence of PTSD, duration of residency, satisfaction with neighborhood, and network characteristics were found to be strongly associated with PTSD symptom severity. more...

ObjectiveVideo-based advanc care planning (ACP) tools have been studied in varied medical contexts; however, none have been developed for patients undergoing major surgery. Using a patient- and family-centredness approach, our objective was to implement human-centred design (HCD) to develop an ACP decision support video for patients and their family members when preparing for major surgery.Design and methodsThe study investigators partnered with surgical patients and their family members, surgeons and other health professionals to design an ACP decision support video using key HCD principles. Adapting Maguire’s HCD stages from computer science to the surgical context, while also incorporating Elwyn et al’s specifications for patient-oriented decision support tool development, we used a six-stage HCD process to develop the video: (1) plan HCD process; (2) specify where video will be used; (3) specify user and organisational requirements; (4) produce and test prototypes; (5) carry out user-based assessment; (6) field test with end users.ResultsOver 450 stakeholders were engaged in the development process contributing to setting objectives, applying for funding, providing feedback on the storyboard and iterations of the decision tool video. Throughout the HCD process, stakeholders’ opinions were compiled and conflicting approaches negotiated resulting in a tool that addressed stakeholders’ concerns.ConclusionsOur patient- and family-centred approach using HCD facilitated discussion and the ability to elicit and balance sometimes competing viewpoints. The early engagement of users and stakeholders throughout the development process may help to ensure tools address the stated needs of these individuals.Trial registration numberNCT02489799. more...

John FP Bridges,1,2 Norah L Crossnohere,2 Anne L Schuster,1 Judith A Miller,3 Carolyn Pastorini,3,† Rebecca A Aslakson2,4,5 1Department of Health Policy and Management, The Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, 2Department of Health, Behavior, and Society, The Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, 3Patient-Centered Outcomes Research Institute (PCORI) Project, Baltimore, MD, 4Department of Anesthesiology and Critical Care Medicine, The Johns Hopkins School of Medicine, Baltimore, MD, 5Armstrong Institute for Patient Safety and Quality, The Johns Hopkins School of Medicine, Baltimore, MD, USA †Carolyn Pastorini passed away on August 24, 2015 Background: Despite a movement toward patient-centered outcomes, best practices on how to gather and refine patients’ perspectives on research endpoints are limited. Advanced care planning (ACP) is inherently patient centered and would benefit from patient prioritization of endpoints for ACP-related tools and studies.Objective: This investigation sought to prioritize patient-centered endpoints for the content and evaluation of an ACP video being developed for patients undergoing major surgery. We also sought to highlight an approach using complementary engagement and research strategies to document priorities and preferences of patients and other stakeholders.Materials and methods: Endpoints identified from a previously published environmental scan were operationalized following rating by a caregiver co-investigator, refinement by a patient co-investigator, review by a stakeholder committee, and validation by patients and family members. Finalized endpoints were taken to a state fair where members of the public who indicated that they or a loved one had undergone major surgery prioritized their most relevant endpoints and provided comments.Results: Of the initial 50 ACP endpoints identified from the review, 12 endpoints were selected for public prioritization. At the state fair, 359 individuals prioritized the endpoints, the highest ranking of which was having a meaningful conversation with a physician before surgery (57%).Conclusion: Using a novel combination of engagement and research techniques provided the opportunity to understand which endpoints were most important to patients and families and fostered framework development clarifying the differential contributions of engagement and research. Results from this study ultimately changed the content and evaluation of the ACP video. Keywords: patient preference, patient participation, community participation, research design&nbsp more...

Introduction:Emerging evidence suggests a heightened interest in healthy behaviour changes, including smoking cessation, at the beginning of the week. Evidence from Google searches, quitlines, and cessation websites show greater information-seeking and interest in early week quitting.Aims:This pilot assesses the comparative effectiveness of a smoking cessation intervention that encourages participants to use Mondays as a day to quit or recommit to quitting smoking.Methods:We partnered with existing smoking cessation group programs to conduct a quasi-experimental, pre–post study. Both comparison and intervention groups received the same standard-care curriculum from program instructors. Intervention group participants received Monday materials including a wallet card and a mantra card during enrolment. On Mondays, intervention participants received an emailed tip-of-the-week and were encouraged to quit or recommit to quitting. Quit buddies were recommended in both groups, but intervention participants were encouraged to check-in with quit buddies on Mondays. The outcomes of smoking abstinence, number and length of quit attempts, and self-efficacy were assessed at the final program session and three months later.Results:At the last session, intervention group participants who were still smoking had a higher self-efficacy of quitting in the future, rated their programs as more helpful in quitting smoking, and were more likely to rate quit buddies as very helpful. Differences in self-efficacy were no longer observed at the second follow-up. No differences were observed between intervention and standard group participants in abstinence, number of quits, length of quits, or self-efficacy of staying quit at either follow-up.Conclusions:Encouraging results from this pilot study indicate that further research is needed to explore how Monday messaging may improve smoking cessation programs. more...

Introduction: There is a groundswell of interest from patient, industry, and regulatory groups to rigorously and transparently integrate patient-voice into regulatory decision-making. Patient-focused drug development (PFDD) is an approach established by the US Food and Drug Administration to systematically incorporate patient experiences into drug development and evaluation. It has created a demand for scientific advancement to measure and integrate patient-voice into decision-making.Areas covered: This narrative review describes the evolving nature of advocacy-regulatory relations preceding PFDD, characterizes current PFDD and other patient-engagement activities, and explores future opportunities for patient participation along the drug development pipeline. We present Duchenne muscular dystrophy as a case study to illustrate how PFDD is being operationalized by patient groups and regulators using both verbal and written data sources.Expert opinion: PFDD represents the most widespread approach yet to integrate the patient voice as a source of evidence to inform regulatory decision-making. Regulatory approvals are just one frontier in drug development. On the horizon remain uncertainties in how patient experience can inform post-marketing surveillance, pricing, reimbursement, and health technology assessment. Patient-input may be particularly crucial to demonstrate the value of expensive first-generation rare disease treatments that confer meaningful benefits but do not meet traditional thresholds for cost-effectiveness. more...

Background: The recent expansion of treatment options in acute myeloid leukemia (AML) has necessitated a greater understanding of patient preferences for treatment benefits, about which little is known. Methods: We sought to quantify and assess heterogeneity of the preferences of AML patients for treatment outcomes. An AML-specific discrete choice experiment (DCE) was developed involving multiple stakeholders. Attributes included in the DCE were event-free survival (EFS), complete remission (CR), time in the hospital, short-term side effects, and long-term side effects. Continuously coded conditional, stratified, and latent-class logistic regressions were used to model preferences of 294 patients with AML. Results: Most patients were white (89.4%) and in remission (95.0%). A 10% improvement in the chance of CR was the most meaningful offered benefit (P < 0.001). Patients were willing to trade up to 22 months of EFS or endure 8.7 months in the hospital or a two-step increase in long-term side effects to gain a 10% increase in chance of CR. Patients diagnosed at 60 years or older (21.6%) more strongly preferred to avoid short-term side effects (P = 0.03). Latent class analysis showed significant differences of preferences across gender and insurance status. Conclusions: In this national sample of mostly AML survivors, patients preferred treatments that maximized chance at remission; however, significant preference heterogeneity for outcomes was identified. Age and gender may affect patients' preferences. Impact: Survivor preferences for outcomes can inform patient-focused drug development and shared decision-making. Further studies are necessary to investigate the use of DCEs to guide treatment for individual patients. more...

Importance Diabetes guidelines recommend considering specific factors, such as diabetes duration and life expectancy, to individualize treatment in older adults. These individualized glycemic targets inform decisions on whether to intensify or deintensify medication treatment plans. How older adults with diabetes perceive these factors used to individualize glycemic targets is unknown. Objectives To examine how older adults perceive factors used in diabetes guidelines for individualizing glycemic targets. Design, Setting, and Participants A cross-sectional national survey was conducted from December 13, 2018, to January 3, 2019, of a nationally representative, probability-based online survey panel (KnowledgePanel). A total of 1364 KnowledgePanel members who were 65 years or older and had type 2 diabetes were invited to participate in the survey; 836 (61.3%) responded, and 818 (60.0%) completed the survey. Main Outcomes and Measures The study randomized participants to 2 vignettes: one about adding and the other about removing diabetes medications from treatment plans. Participants rated the importance of 7 factors (diabetes duration, established diabetes complications, other health conditions, life expectancy, risk of adverse effects, cost, and treatment effort) in these treatment decisions using binary (yes/no) responses and the best-worst scaling method to quantify the factors’ relative importance. All participants then answered questions on how different levels of each factor were associated with aggressiveness of diabetes treatment. Results The sample included 818 participants (mean [SD] age, 74.0 [6.8] years; 469 [53.7%] male; and 668 [67.7%] white). A total of 410 participants answered questions about adding medicine, whereas 408 participants answered questions about stopping medicine. Of the 7 factors to consider for adding a diabetes medication to the treatment plan, the number who deemed each factor important ranged from 197 (45.6%) to 263 (62.8%). In contrast, these same factors were considered important by only 29 (8.4%) to 146 (37.7%) of participants when deciding to stop use of a diabetes medication. In both decisions, participants perceived the risk of adverse effects as the most important factor (relative importance was 22.8 for adding a medicine and 25.0 for stopping a medicine on a ratio scale in which, for each decision, the relative importance of the 7 factors adds up to 100, with 0 indicating complete indifference and 100 complete priority). In contrast to current guideline recommendations, most participants believed that patients with longer disease duration (498 [60.1%]), more established complications (632 [75.6%]), and greater number of other health conditions (545 [67.5%]) should receive more aggressive diabetes treatment. Conclusions and Relevance Many older adults do not place high importance on factors recommended by guidelines to individualize diabetes treatment, especially when deciding to stop use of diabetes medications. Moreover, when considering treatment aggressiveness, many older adults weighted several factors in the opposite direction than suggested by the guidelines. Individualizing diabetes care in older adults will require effective communication regarding the benefits and consequences of making changes to treatment plans. more...

Background: Preoperative advance care planning (ACP) may benefit patients undergoing major surgery. Objective: To evaluate feasibility, safety, and early effectiveness of video-based ACP i...

Background: Advances in patient-facing health information technology (HIT) promise to improve health care delivery and patient outcomes. Low utilization of HIT suggests that the preferences of patients may not be adequately guiding the development of these technologies. This systematic review offers an assessment of published evidence regarding patient preferences for HIT. Methods: Articles addressing preferences for HIT from patient and other end-user groups published up through 2020 were identified from PubMed, Web of Science, Scopus and via hand searching. Articles that used quantitative stated-preference methods to explore preferences for HIT were eligible for inclusion. Studies that explored attitudes towards HIT without eliciting trade-offs were excluded. Critical appraisal of study quality was conducted using the PREFS checklist and quality criteria identified by the US Food and Drug Administration including heterogeneity analysis and patient engagement in study design. We conducted thematic analysis of the main preference findings from each study to synthesize patient and enduser preferences for HIT. The review was not registered and authors received no funding to conduct the review. Results: The search yielded 7,299 unique articles, 59 of which were ultimately included in the review. Studies explored preferences for telemedicine (n=30), patient portals (n=12), mHealth (n=9) or multiple HITs (n=8). Preference elicitation methods included direct elicitation (n=26), discrete-choice experiments (n=13), conjoint analysis (n=6), contingent valuation (n=5), and ranking exercises (n=9). Studies had a mean PREFS score of 3.51 out of 5. Forty-two studies conducted preference heterogeneity analysis and only 20 included patients in study design. Thematic meta-analysis indicated that patients prefer HIT that is convenient and lower cost, but does not sacrifice quality, and preferences varied by demographic features such as age as well as depending on the type of health information being communicated. Conclusions: Patient and end-users have distinct preferences for the use of HIT in their medical care. It is timely that researchers and healthcare administrators consider these preferences for HIT given its rapid uptake amidst the COVID-19 pandemic. Although this literature demonstrates that patients can be engaged as participants in preference studies to identify meaningful aspects of HIT, the field was limited in its inclusion of patients in the design of such studies. Future development of HIT should be guided by high-quality preference research that integrates patients in all stages in the design and implementation of HIT. © Journal of Hospital Management and Health Policy. All rights reserved. more...

Prader-Willi syndrome (PWS) is a rare disease associated with cognitive impairment, hypotonia, hyperphagia (an insatiable hunger), and obesity. Therapies that target hyperphagia are in development, but understanding the value of these therapies to inform patient-focused drug development (PFDD) requires valid data on disease burden. We estimated disease burden by measuring and comparing quality-adjusted life-years (QALYs) for 3 PWS health states relevant to current PFDD initiatives.Time trade-off (TTO) and a visual analog scale (VAS) were used to elicit PWS caregivers' values for 3 fixed health states for a standardized patient described with (1) untreated PWS, (2) PWS with controlled obesity, and (3) PWS with controlled obesity and hyperphagia. We excluded participants who left at least 1 TTO or VAS question blank or incomplete (noncompleters) and respondents who reported the same answer for all TTO scenarios (nontraders). The remaining group of respondents (traders) were used for all primary analyses. We assessed validity and bias of QALY estimates by comparing differences in health state valuations, treatment priorities, and characteristics among respondents who did and did not complete the TTO.A total of 458 respondents completed the survey, including 226 traders, 93 nontraders, and 139 noncompleters. Traders valued untreated PWS at 0.69 QALYs, PWS with controlled obesity at 0.79 QALYs, and controlled hyperphagia/obesity at 0.91 QALY (P0.01 for differences among health state values). Reported VAS ratings were similar for traders versus nontraders for untreated PWS (38.64 vs 38.95, P = 0.89) and PWS with controlled obesity (57.36 vs 55.14, P = 0.35) but varied for PWS with controlled obesity and hyperphagia (70.70 vs 64.46, P = 0.02). Exclusion of noncompleters did not introduce obvious bias because traders and noncompleters were similar in treatment priorities and characteristics. The exclusion of nontraders did not meaningfully alter mean or distribution of valuations.This study found that avoiding hyperphagia decreases the burden of PWS and that these results are robust, even once imposing strict inclusion criteria. Use of fixed health states to estimate QALYs addresses many of the complexities of measuring disease burden in rare and pediatric conditions, indicating the potential value of this approach to inform premarket decision makers in identifying outcome importance. more...

For instance, experience-based time trade-off and visual analog scale value sets for the EQ-5D-3L were recently published [16].

BACKGROUND/OBJECTIVES: Many older adults receive unnecessary screening colonoscopies. We previously conducted a survey using a national online panel to assess older adults’ preferences for how clinicians can discuss stopping screening colonoscopies. We sought to assess the generalizability of those results by comparing them to a sample of older adults with low health literacy. DESIGN: Cross-sectional survey. SETTING: Baltimore metropolitan area (low health literacy sample) and a national, probability-based online panel—KnowledgePanel (national sample). PARTICIPANTS: Adults 65+ with low health literacy measured using a single-question screen (low health literacy sample, n = 113) and KnowledgePanel members 65+ who completed survey about colorectal cancer screening (national sample, n = 441). MEASUREMENTS: The same survey was administered to both groups. Using the best-worst scaling method, we assessed relative preferences for 13 different ways to explain stopping screening colonoscopies. We used conditional logistic regression to quantify the relative preference for each explanation, where a higher preference weight indicates stronger preference. We analyzed each sample separately, then compared the two samples using Spearman’s correlation coefficient, the likelihood ratio test to assess for overall differences between the two sets of preference weights, and the Wald test to assess differences in preference weights for each individual phrases. RESULTS: The responses from the two samples were highly correlated (Spearman’s coefficient 0.92, p more...

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. more...

Introduction: Symptom management is considered a top priority of cancer care. Monitoring patient-reported outcomes (PROs) such as side effects of treatment has been shown to improve quality of life and even prolong survival among cancer patients. For regulators, PROs collected from patients and the caregivers who speak for patients when they are too ill or unable to speak for themselves informs patient-focused drug development (PFDD). PFDD is a key-component of the 21st Century Cures Act that aligns FDA, industry, advocacy and academic partners to accelerate medical product development for those who need it most. We sought to understand the prevalence of side effects experienced by people with acute myeloid leukemia (AML) and to assess differences in the report of side effects from patients across the disease trajectory, including those who were deceased or too ill to directly self-report. Methods : We assessed patient reported prevalence of severe short- and long-term side effects from AML treatments as a part of a national survey. Caregiver reports were included so as to capture the experiences of patients who had either passed away from AML or who were too ill to participate. Participants rated their experience with short-term side effects (nausea, infection, mouth sores, diarrhea, hair loss) and long-term side effects (organ dysfunction, neuropathy, fatigue, chemo brain) as 'none,' 'mild,' 'moderate,' or 'severe.' Prevalence of severe side effects was assessed using descriptive statistics, and differences in likelihood of reporting severe side effects were assessed using logistic regression. Results : A total of 1182 eligible participants completed the survey, including 901 patients and 281 caregivers. Participants were an average of 55 years old (SD=13.0), and 12% were non-white minorities. Patient participants had been diagnosed with AML for 7.7 years (SD=4.7), while patients reported on by caregiver participants had been diagnosed for 5.7 years (SD=4.1). Almost all participants received chemotherapy (98%), and one-fifth received radiation (21%). Hair loss was the most common severe short-term side effect among caregivers (68%) as well as patients (79%, p=0.01). Conversely, patients were less likely than caregivers to report a severe infection (29% vs 36%, p Conclusions: AML patients of diverse disease stages experience side effects as well as long-lasting collateral damage from treatments. PRO measures can collect important prevalence data for these events. As compared to their healthier patient counterparts, caregivers reporting on deceased or ill patients demonstrated similar rates of severe short-term side effects, but higher rates of long-term, collateral damages from their treatment. Despite the difficulty in objective quantification, that over 10% of the sample reported severe 'chemo brain' validates the disease community's continued interest in better understanding this experience through PFDD. Disclosures Bridges: The Leukemia & Lymphoma Society: Research Funding. more...

5 Background: The feasibility and impact of integrating advance care planning (ACP) in surgical oncologic populations is unknown. We hypothesized that an ACP video could be integrated into surgical oncologic care and that patients who preoperatively viewed the video would engage in more ACP and patient-centered communication with their surgeon. Methods: Randomized controlled, phase II trial with two study arms: intervention ACP video and control (no ACP) video; and four data collection periods: enrollment, presurgical consent, postoperative one week, and postoperative one month. Participants included patients undergoing major cancer surgery recruited from nine surgical oncology clinics at a single, academic, inner city, tertiary care hospital. Main Outcomes and Measures: Primary Outcome - Content of ACP and patient-centeredness during the presurgical consent visit as measured through the Roter Interaction Analysis System (RIAS). Secondary patient outcomes – mood; goals of care; and video helpfulness. Results: Ninety-two patients (target enrollment of ninety) were enrolled over 15 months. The ACP video was successfully integrated into the intervention arm and no study-related harms were noted. Patient-centeredness was unchanged between the study arms (IRR=1.06, CI [0.87,1.3], p=0.545) and there was a trend of increased ACP content in the intervention arm (23% intervention vs. 10% control, p=0.182). There were no differences in secondary outcomes other than patients found the intervention video more “helpful” (p=0.007). There was a statistically significant increase in mood symptoms in both groups one week post-operative as compared to enrollment (p=0.01). Patients in both groups most frequently noted cure-related goals of care. Conclusions: The ACP video was integrated successfully into surgical care, well-tolerated, and thought helpful by patients, but did not significantly change the nature or ACP content in patient-surgeon pre-operative conversation. Future studies could increase the “dose” of ACP through modifying video content and/or how or who presents that content prior to surgery. Clinical trial information: NCT02489799. more...