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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.

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.

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

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.

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.

Background. Prader-Willi syndrome (PWS) is a rare neurodevelopmental disorder causing quality of life impairments such as insatiable hunger (hyperphagia) and obesity. We explored caregivers’ willingness to assume treatment risk in exchange for reduced hyperphagia according to a PWS-validated observer-reported outcome measure. Methods. We partnered with PWS patient organizations to develop a discrete-choice experiment exploring caregivers’ benefit-risk tradeoffs for emerging PWS treatments. The treatment benefit was a reduction in hyperphagia (as measured by a 0-, 5-, or 10-point change on the Hyperphagia Questionnaire for Clinical Trials [HQ-CT]). Treatment risks included weight gain (none, 5%, 10%), added risk of skin rash (none, 10%, 20%), and risk of liver damage (none, 1 in 1000, 10 in 1000). Preference models were estimated using mixed logistic regression and maximum acceptable risk. We explored differences in preferences across familial caregivers of patients with and without hyperphagia. Results. Four hundred sixty-eight caregivers completed the online survey. The majority of caregivers reported that patients experienced hyperphagia (68%) and half of patients experienced obesity (52%). Caregivers of patients without hyperphagia were willing to accept greater weight gain (16.4% v. 8.1%, P = 0.004) and a higher risk of skin rash (11.7% v. 6.2% P = 0.008) as compared to caregivers of patients with hyperphagia. Caregivers of patients with hyperphagia would accept a higher risk of liver damage as compared to caregivers of patients without hyperphagia (11.9 out of 1000 v. 6.4 out of 1000, P = 0.04). Conclusions. This research demonstrates that caregivers are willing to accept risk in exchange for a five-point improvement on the HQ-CT, a smaller marginal improvement than had been previously classified as meaningful. Patient experience with hyperphagia is a modifier in how much risk caregivers will accept.

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.

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.

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.