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Background: Improved patient life engagement is a meaningful treatment goal in schizophrenia that cannot be satisfactorily measured using existing tools. This research aimed to determine whether certain items from the Positive and Negative Syndrome Scale (PANSS) can assess patient life engagement in schizophrenia., Methods: Three approaches were used to identify PANSS items that reflect patient life engagement: (1) a panel discussion with expert psychiatrists (n = 4); (2) interviews with patients with schizophrenia (n = 20); and (3) a principal component analysis to explore clustering of items (n = 954 from three randomized controlled trials). Internal consistency was assessed by Cronbach's alpha and item-total correlations. A minimal clinically important difference (MCID) was determined by anchor- and distribution-based methods., Results: Expert psychiatrists identified 11 relevant items, and patients rated 13 items as "very relevant" to patient life engagement, most of which clustered in the principal component analysis. Considering all results, a composite set of 14 PANSS items that may be relevant to patient life engagement in schizophrenia was devised: P2, N1, N2, N3, N4, N5, N6, N7, G6, G7, G11, G13, G15, G16 (Cronbach's alpha, 0.84; item-total correlations, 0.35-0.56, indicating acceptable correlation with the underlying concept; exception: G6 [depression], 0.19). An MCID of 5 points (small/moderate improvement) or 10 points (large improvement) may be appropriate., Conclusions: A subset of 14 PANSS items may be used to reflect patient life engagement in clinical practice/trials in schizophrenia, complementing the results of traditional psychiatric symptom scales with a patient-centered outcome that is relevant to real-world treatment goals., Competing Interests: Declaration of competing interest Zahinoor Ismail has received grant support from CIHR, NIH, Brain Canada, and Weston Foundation, and has served as a consultant for Eisai, Eli Lilly, Lundbeck, Novo Nordisk, Otsuka and Roche. Zahinoor Ismail is also supported by the UK National Institute for Health and Care Research Exeter Biomedical Research Centre. The views expressed are those of the author(s) and not necessarily those of the NIHR or the Department of Health and Social Care. Stine R. Meehan, Anja Farovik, and Stephane Alexandre Regnier are full-time employees of H. Lundbeck A/S. Maia Miguelez was a full-time employee of Otsuka Pharmaceutical Development & Commercialization Inc. at the time of this work. Shivani Kapadia and Zhen Zhang are full-time employees of Otsuka Pharmaceutical Development & Commercialization Inc. T. Michelle Brown and Mirline Milien are full-time employees of RTI Health Solutions, contracted to conduct this research on behalf of the sponsor. Roger S. McIntyre has received research grant support from CIHR/GACD/National Natural Science Foundation of China (NSFC) and the Milken Institute; and speaker/consultation fees from Lundbeck, Janssen, Alkermes, Neumora Therapeutics, Boehringer Ingelheim, Sage, Biogen, Mitsubishi Tanabe, Purdue, Pfizer, Otsuka, Takeda, Neurocrine, Neurawell, Sunovion, Bausch Health, Axsome, Novo Nordisk, Kris, Sanofi, Eisai, Intra-Cellular, NewBridge Pharmaceuticals, Viatris, Abbvie, and Atai Life Sciences. He is a CEO of Braxia Scientific Corp., (Copyright © 2024. Published by Elsevier B.V.)

Background: Familial chylomicronemia syndrome (FCS) is a rare metabolic disorder that impacts physical, emotional, social, and cognitive functioning. The FCS-Symptom and Impact Scale (FCS-SIS) patient-reported outcome (PRO) measure assesses common symptoms and impacts of FCS. This study was conducted to evaluate cross-sectional psychometric properties of the FCS-SIS and its scoring method., Methods: This multisite, cross-sectional, observational study of individuals with FCS was conducted in the United States and Canada. Participants completed a survey composed of 7 PRO measures, including the FCS-SIS, and questions about clinical characteristics and demographics. The structure of the FCS-SIS was evaluated using inter-item and item-scale correlations and internal consistency reliability. Construct, known-groups, and criterion validity were evaluated by examining associations between FCS-SIS item and composite scores and other measures included within the survey., Results: Most of the 33 participants were female (63.6%) and White (78.1%). On average, participants reported first noticing FCS symptoms at ~16 years, with abdominal pain the most frequently reported initial symptom (n=20). Participants reported 2.5 acute pancreatitis attacks on average over the past year. Average FCS-SIS symptom item scores ranged from 1.8 to 3.9 (on a 0-to-10 scale [none-to-worst-possible]) within the 24-hour recall period, with an average Symptom composite score of 2.7. The average impact item scores on the FCS-SIS ranged from 1.6 to 3.0 (on a 0-to-4 scale), with an average Impact composite score of 2.1. Inter-item correlations between the FCS-SIS Symptom items ranged from 0.32 to 0.78. Corrected item-total correlations were highly satisfactory for Impact items, ranging from 0.62 to 0.85. All a priori validity hypotheses were supported by observed correlations and score differences between known groups., Conclusion: The results of this study support the structure, reliability, and validity of the FCS-SIS, laying the psychometric groundwork for longitudinal evaluation of its utility in assessing treatment benefit in FCS clinical studies., Competing Interests: TMB and SF are full-time employees of RTI Health Solutions, an independent nonprofit research organization, which was retained by Ionis Pharmaceuticals to conduct the research which is the subject of this manuscript. Their compensation is unconnected to the studies on which they work. CK was an employee of RTI Health Solutions at the time the research was conducted. MVL, ASK, and AY are employees of Ionis Pharmaceuticals and hold shares and/or stock options in the company. The authors report no other conflicts of interest in this work., (© 2024 Brown et al.)

Introduction: Discussions of weight-management strategies between patients and healthcare providers can yield positive outcomes for people with overweight or obesity. Nonetheless, people with overweight or obesity encounter communication challenges and other barriers to pursuing effective weight-management strategies with their healthcare providers. The aim of this study was to develop a new self-completed assessment tool to initiate and facilitate conversations related to weight management between patients and healthcare providers., Methods: Developing the assessment tool involved a series of steps and draft versions of the tool, based on feedback from key opinion leaders in the field of obesity (N = 4) and input from people with overweight or obesity (N = 18). Three iterative rounds of qualitative interviews were conducted in the USA. A targeted review of prior qualitative research was conducted to identify common and important impacts of obesity on patients' functioning. Standard qualitative analytical methods were used to identify concepts of importance in a concept elicitation exercise during the interviews and were evaluated for potential inclusion in the tool. Potential problems with the tool were flagged during cognitive debriefing of the draft tool., Results: During 18 individual interviews, participants referenced the impact of their weight on their lives, including health and comorbidities, physical function, emotional/mental functioning, social life, and physical appearance. Over the course of the tool's development, 24 common and important impacts of obesity on patients' functioning were reduced to a final set of eight concepts in the final tool that were deemed important and relevant to both patients and key opinion leaders., Conclusions: The assessment tool is a five-item, self-completed measure expected to foster patient self-advocacy for individuals with overweight or obesity by giving them an opportunity to define their weight-management goals and discuss these, along with various medical interventions, with a healthcare provider., (© 2022. The Author(s).)

Background: Patient-reported outcomes can measure health aspects that are meaningful to patients, such as 'life engagement' in major depressive disorder (MDD). Expert psychiatrists recently identified ten items from the Inventory of Depressive Symptomatology Self-Report (IDS-SR) that can be used to measure patient life engagement. This study aimed to explore the concept of patient life engagement and provide support for the IDS-SR 10 Life Engagement subscale from the patient perspective., Methods: Semi-structured video interviews were conducted with adults with MDD in the United States. Patients were asked if they ever felt engaged with life, and how this affected their feelings, activities, socializing, and thoughts. Then, patients discussed the ten expert-selected IDS-SR items, and rated the relevance of all 30 items to patient life engagement on a 4-point scale., Results: Patients (N = 20) understood the 'engaged with life' concept and could provide examples from their own lives, such as increased energy/motivation (100%), being more social/spending time with others (85%), being more communicative (80%), and having better mood (75%). Nineteen patients (95%) indicated that all ten IDS-SR 10 Life Engagement items were relevant to patient life engagement, and nine of the ten items had a mean score ≥ 3 (moderately relevant). Four additional items (all relating to mood) also scored ≥ 3., Conclusions: Patients found the concept of life engagement to be important and relatable, and confirmed the IDS-SR 10 captures the defining non-mood-related aspects of patient life engagement. This research supports the relevance of patient life engagement as a potential clinical outcome beyond core mood symptoms, and the use of the IDS-SR 10 Life Engagement subscale in patient-oriented research., (© 2022. The Author(s).)

Background: Though often overlooked, calming patients and increasing their life engagement are key factors in the treatment of major depressive disorder (MDD). This study aimed to test the hypothesis that adjunctive brexpiprazole increases calmness and life engagement among patients with MDD, based on clinical trial exit interviews., Methods: This was a pooled analysis of exit interview data from three exploratory, open-label studies of adjunctive brexpiprazole 1-3 mg/day. The studies enrolled 105 outpatients with MDD (DSM-IV-TR criteria), a current depressive episode, and inadequate response to antidepressant treatment during the current episode. Patients were interviewed if they completed the end-of-treatment visit (Week 6 or Week 12, depending on the study). Exit interviews took the form of semi-structured telephone interviews in which patients were asked mostly qualitative questions about their symptoms prior to the start of the study, and about improvements they had noted during treatment. Interview transcripts were reviewed and codes were assigned to calmness and life engagement vocabulary, allowing aggregation of the frequency of improvement in various domains., Results: 79.8% (83/104) of patients described improvements consistent with at least one calmness term, most commonly feeling less anxious (46.2%) or less irritable (44.2%). A four-domain concept of patient life engagement was developed in which 88.6% (93/105) of patients described improvements consistent with at least one domain, specifically, emotional (77.1%), physical (75.2%), social (41.9%), and/or cognitive (36.2%). Of the patients who described improvement in calmness, 96.4% (80/83) also described improvement in life engagement., Conclusions: Analysis of exit interview data suggests that patients were calmer and more engaged with life following treatment with adjunctive brexpiprazole. Thus, adjunctive brexpiprazole may provide a benefit on subjective patient outcomes in addition to the improvement in depressive symptoms shown by clinical rating scale data., Trial Registration: Data used in this post hoc analysis came from ClinicalTrials.gov identifiers: NCT02012218, NCT02013531, NCT02013609., (© 2021. The Author(s).)

Background: Familial chylomicronemia syndrome (FCS), a rare genetic disorder characterized by high levels of circulating triglycerides, negatively impacts multiple organs, including the liver and pancreas., Objective: The objective of this study was to develop and support the content validity of a novel patient-reported outcome (PRO) measure addressing FCS symptoms and impacts. To facilitate use in clinical trials of new treatments, evidence supporting the new measure needed to be consistent with regulatory guidance and requirements., Methods: A pool of items addressing symptoms and impacts of FCS was initially developed based on data from a large burden-of-illness study with patients with FCS as well as a review of available literature and existing PRO measures. Two rounds of qualitative interviews were conducted with patients with FCS (N = 10) to refine the draft items and support the measure's content validity. Each interview began with concept elicitation followed by cognitive debriefing of the draft FCS measure., Results: Patient-reported symptoms and impacts of FCS were generally consistent with those identified by the burden-of-illness study; abdominal pain was particularly prevalent and salient for patients. Suggested changes to the draft item pool were generally minor. Comprehensibility and ease of completion for the final instrument were confirmed during the second set of interviews., Conclusion: The content validity of the final 17-item FCS Symptoms and Impacts Scale is strongly supported by patient input gathered through both a large burden-of-illness study and qualitative research. To further support use in clinical trials, psychometric evaluation of the measure is underway., (© 2021. The Author(s).)

Objective: Depressive episodes and symptoms of bipolar I disorder are commonly misdiagnosed as major depressive disorder (MDD) in primary care. The novel and pragmatic Rapid Mood Screener (RMS) was developed to screen for manic symptoms and bipolar I disorder features (e.g. age of depression onset) to address this unmet clinical need., Methods: A targeted literature search was conducted to select concepts thought to differentiate bipolar I from MDD and screener tool items were drafted. Items were tested and refined in cognitive debriefing interviews with individuals with self-reported bipolar I or MDD ( n  = 12). An observational study was conducted to evaluate predictive validity. Participants with clinical interview-confirmed bipolar I or MDD diagnoses ( n  = 139) completed a draft 10-item screening tool and other questionnaires. Data were analyzed to identify the smallest possible subset of items with optimized sensitivity and specificity., Results: Adults with confirmed bipolar I ( n  = 67) or MDD ( n  = 72) participated in the observational study. Ten draft screening tool items were reduced to 6 final RMS items based on the item-level analysis. When 4 or more items of the RMS were endorsed ("yes"), sensitivity was 0.88 and specificity was 0.80; positive and negative predictive values were 0.80 and 0.88, respectively. These properties were an improvement over the Mood Disorder Questionnaire in the same analysis sample while using 60% fewer items., Conclusion: The pragmatic 6-item RMS differentiates bipolar I disorder from MDD in patients with depressive symptoms, providing real-world guidance to primary care practitioners on whether a more comprehensive assessment for bipolar I disorder is warranted.

Background: Patient-focused literature on fatigue in progressive forms of multiple sclerosis (MS) is sparse. This study aimed to explore progressive MS patients' experiences of fatigue., Methods: Adult patients in the United States with primary progressive MS (n=21) and secondary progressive MS (n=23), recruited from research panels, completed the following PRO measures: Patient Global Impression of Severity (Fatigue) (PGI-F); Fatigue Scale of Motor and Cognitive Functions (FSMC); Modified Fatigue Impact Scale (MFIS); Patient Health Questionnaire, two-item version (PHQ-2); and Patient Determined Disease Steps (PDDS). Patients subsequently participated in a 45-minute semistructured telephone interview and were asked to describe their MS symptoms and to comment on how MS affected their day-to-day lives. More detailed questions followed on the nature of their fatigue, including symptoms, impacts, frequency, and bothersomeness., Results: Patients' mean age was 52.5 years, mean time since diagnosis was 14.7 years, and 81.8% were female. 79.5% of patients were unemployed and/or receiving disability benefits. Of all spontaneously reported MS symptoms, fatigue was the most common (n=38, 86.4%), followed by ambulation problems (n=31, 70.5%) and muscle weakness (n=25, 56.8%). Patients used the words "tired," "exhausted," "wiped out," and having "little or no energy" to describe their fatigue. More patients rated fatigue as their "most troubling symptom" (n=17, 38.6%) compared with other MS-related symptoms. Half of patients reported feeling constantly fatigued, and more than 90% reported experiencing fatigue at least daily. The top three most frequently reported negative impacts of fatigue were social functioning, emotional well-being, and cognitive functioning (all >80%). Patients described themselves as "homebodies," as fatigue limited their social interactions with friends and family and impacted the types of activities they could participate in. Patients attributed their inability to think clearly or focus for long periods of time to their fatigue. Patients also reported experiencing depression and anxiety because of their fatigue, which would often have further negative effects on their relationships with friends and family. On the fatigue PRO measures, mean (standard deviation) scores were 75.2 (14.7) on the FSMC and 55.0 (15.2) on the MFIS. Most participants scored in the "high" fatigue category on the FSMC (84.1%) and above the clinically significant fatigue threshold (86.4%). MFIS and FSMC total scores correlated with PGI-F (polyserial correlations r=0.74 and r=0.62, both p<0.01) and PHQ-2 (r=0.56 and r=0.57, both p<0.01), but not with PDDS (r=0.09 and r=0.02, both p>0.05)., Conclusions: Fatigue is a common, troublesome, and disabling symptom which has a profound impact on patients' daily lives, as evidenced by qualitative analyses and high scores on established fatigue measures observed in this sample. These findings provide insights into the burden of fatigue and can inform its measurement in both clinical and research settings. Treatments that improve the symptoms of fatigue or prevent exacerbations are needed for patients with progressive MS., Competing Interests: Declaration of Competing Interest This study was performed under a research contract between RTI Health Solutions and F. Hoffmann-La Roche Ltd. and was funded by F. Hoffmann-La Roche Ltd. T. Michelle Brown, Christina Slota, and Lynda Doward are salaried employees of RTI Health Solutions. Fiona McDougall and Shibeshih Belachew are employees and shareholders of F. Hoffmann-La Roche Ltd. Laura Julian is an employee of Genentech, Inc., and a shareholder of F. Hoffmann-La Roche Ltd. Deborah Miller and Iris-Katharina Penner have served as consultants for F. Hoffman-La Roche Ltd., (Copyright © 2020. Published by Elsevier B.V.)

Introduction: Pruritus is the primary symptom of atopic dermatitis (AD). The objective of this study was to develop a patient-reported outcome (PRO) instrument for daily administration in clinical trials to measure AD-related itch in adolescents and adults that would meet the standards described in the US Food and Drug Administration's (FDA's) PRO Guidance. Materials and methods: Six focus groups were conducted with 49 patients with AD (32 adults; 17 adolescents). Three iterative rounds of cognitive debriefing interviews were conducted in 26 patients with AD (17 adults; 9 adolescents) to pretest and refine the instrument. Results: AD-related itching was considered the most bothersome AD symptom by nearly two-thirds of the focus group participants. The items in the initial version of the instrument, named the Atopic Dermatitis Itch Scale (ADIS), were developed to reflect concepts most relevant to the assessment of itching as described during the focus groups. Based on results of the cognitive debriefing interviews, an 8-item final version of the ADIS was created. Conclusion: The ADIS is a content valid PRO instrument addressing the concepts critical to the measurement of AD-related itching. To our knowledge, it is the first instrument developed to assess AD-related itch in patients as young as 12 years following the recommendations of the PRO Guidance.

Objective: Qualitative research exploring patient preferences regarding the mode of treatment administration for psoriatic arthritis (PsA) is limited. We report patient preferences and their reasons across PsA treatment modes., Methods: In this global, cross-sectional, qualitative study, interviews were conducted with adult patients with PsA in Brazil, France, Germany, Italy, Spain, the UK, and the US. Patients were currently taking a disease-modifying antirheumatic drug (DMARD). Patients indicated the order and strength of preference (0-100; 100 = strongest) across four modes of treatment administration: oral (once daily), self-injection (weekly), clinic injection (weekly), and infusion (monthly); reasons for preferences were qualitatively assessed. Descriptive statistics were reported. Fisher's exact tests and t- tests were conducted for treatment mode outcomes., Results: Overall, 85 patients were interviewed (female, 60.0%; mean age, 49.8 years). First-choice ranking (%) and mean [standard deviation] preference points were: oral (49.4%; 43.9 [31.9]); self-injection (34.1%; 32.4 [24.8]); infusion (15.3%; 14.5 [20.0]); clinic injection (1.2%; 9.2 [10.0]). Of 48 (56.5%) patients with a strong first-choice preference (ie point allocation ≥60), 66.7% chose oral administration. Self-injection was most often selected as second choice (51.8%), clinic injection as third (49.4%), and infusion as fourth (47.1%). Oral administration was the first-choice preference in the US (88.0% vs 38.0% in Europe). The most commonly reported reason for oral administration as the first choice was speed and ease of administration (76.2%); for self-injection, this was convenience (75.9%). The most commonly reported reason for avoiding oral administration was concern about possible drug interactions (63.6%); for self-injection, this was a dislike of needles or the injection process (66.7%)., Conclusion: Patients with PsA preferred oral treatment administration, followed by self-injection; convenience factors were common reasons for these preferences. Overall, 43.5% of patients did not feel strongly about their first-choice preference and may benefit from discussions with healthcare professionals about PsA treatment administration options., Competing Interests: DA has received grant/research support from AbbVie, Bristol-Myers Squibb, and MSD; is a consultant for AbbVie, Bristol-Myers Squibb, Eli Lilly, Janssen, Medac, Merck, MSD, Pfizer Inc, Roche, and UCB; and is a member of speakers’ bureaus for AbbVie, Bristol-Myers Squibb, Eli Lilly, Janssen, Medac, Merck, MSD, Pfizer Inc, Roche, and UCB. MEH is a consultant for and reports personal fees from AbbVie, Amgen, Eli Lilly, Janssen, Novartis, Pfizer Inc, and UCB. JFM is a consultant and/or investigator for and reports personal fees from AbbVie, Aclaris, Almirall, Arena, Avotres, Biogen, Celgene, Dermavant, Eli Lilly, GSK, Incyte, Janssen, Leo Pharma, Merck, Novartis, Pfizer Inc, EMD Serono, Samumed, Sanofi Regeneron, Sun Pharma, and UCB. RR is a consultant for AbbVie, Janssen, Novartis, and Pfizer Inc; and is a member of speakers’ bureaus for AbbVie, Janssen, Novartis, and Pfizer Inc. HB is a consultant for Pfizer Inc. RL was an employee and shareholder of Pfizer Inc at the time of analysis. RL reports personal fees from Pfizer Pharma GmbH, during the conduct of the study; personal fees from AbbVie Inc and Pfizer Pharma GmbH, outside the submitted work. TMB and CE are employees of RTI Health Solutions (non-profit independent research institute), which receives funding from pharmaceutical companies for research consulting services. TMB and CE receive no compensation from the pharmaceutical companies, and their RTI salary is not connected to the projects they work on or the clients for whom research is conducted. PMY, JCC, M-AH, and LF are employees of, and hold stock/stock options in, Pfizer Inc. The authors report no other conflicts of interest in this work., (© 2020 Aletaha et al.)

Background: Hereditary angioedema (HAE) with C1-inhibitor deficiency is associated with painful, potentially fatal attacks affecting subcutaneous or submucosal tissues., Objective: To evaluate HAE burden from the patients' perspective., Methods: This was a noninterventional survey of patients with HAE in the United States, conducted from March 17 to April 28, 2017. Patients were recruited through the US Hereditary Angioedema Association. Key eligibility criteria included the following: (1) aged 18 years and older, (2) self-reported physician diagnosis of HAE type I or II, (3) 1 or more HAE attacks or prodromal symptoms within the last year, and (4) receipt of HAE medication for an attack within the last 2 years. Descriptive analyses were conducted., Results: A total of 445 patients completed the survey. Most patients (92.8%) were aged 18 to 64 years with HAE type I (78.4%) and had a positive family history (78.4%). Mean (SD) ages at symptom onset and diagnosis were 12.5 (9.1) and 20.1 (13.7) years, respectively. Most patients (78.7%) experienced an attack within the past month. The abdomen (58.0%) and extremities (46.1%) were commonly affected sites; pain (73.9%) and abdominal (57.0%) and nonabdominal (55.1%) swelling were frequently reported symptoms. Most patients (68.5%) had received or were currently receiving long-term prophylaxis. Most patients (88.8%) reported visiting allergists or immunologists, whereas 9.2% visited emergency departments or urgent care clinics. Per the Hospital Anxiety and Depression Scale, 49.9% and 24.0% of respondents had anxiety and depression, respectively. Mean Hereditary Angioedema-Quality of Life scores were generally lower with higher attack frequency. General health was "poor" or "fair" for 24.8% of patients. Mean (SD) percentage impairments were 5.9% (14.1%) for absenteeism, 23.0% (25.8%) for presenteeism, 25.4% (28.1%) for work productivity loss, and 31.8% (29.7%) for activity impairment., Conclusion: Despite treatment advances, patients with HAE in the United States continue to have a high burden of illness., (Copyright © 2020 The author. Published by Elsevier Inc. All rights reserved.)

Purpose: Current knowledge of the reasons for patients' preference for rheumatoid arthritis (RA) treatment modes is limited. This study was designed to identify preferences for four treatment modes, and to obtain in-depth information on the reasons for these preferences., Patients and Methods: In this multi-national, cross-sectional, qualitative study, in-depth interviews were conducted with adult patients with RA in the United States, France, Germany, Italy, Spain, Switzerland, the United Kingdom, and Brazil. Patients' strength of preference was evaluated using a 100-point allocation task (0-100; 100=strongest) across four treatment modes: oral, self-injection, clinic-injection, and infusion. Qualitative descriptive analysis methods were used to identify, characterize, and summarize patterns found in the interview data relating to reasons for these preferences., Results: 100 patients were interviewed (female, 75.0%; mean age, 53.9 years; mean 11.6 years since diagnosis). Among the four treatment modes, oral administration was allocated the highest mean (standard deviation) preference points (47.3 [33.1]) and was ranked first choice by the greatest percentage of patients (57.0%), followed by self-injection (29.7 [27.7]; 29.0%), infusion (15.4 [24.6]; 16.0%), and clinic-injection (7.5 [14.1]; 2.0%). Overall, 56.0% of patients had a "strong" first-choice preference (ie, point allocation ≥70); most of these patients chose oral (62.5%) vs self-injection (23.2%), infusion (10.7%), or clinic-injection (3.6%). Speed and/or ease of administration were the most commonly reported reasons for patients choosing oral (52.6%) or self-injection (55.2%). The most common reasons for patients not choosing oral or self-injection were not wanting to take another pill (37.2%) and avoiding pain due to needles (46.5%), respectively., Conclusion: These data report factors important to patients regarding preferences for RA treatment modes. Patients may benefit from discussions with their healthcare professionals and/or patient support groups, regarding RA treatment modes, to facilitate shared decision-making., Competing Interests: PCT has received research grants from Eli Lilly, Galapagos, and UCB; and is a consultant for AbbVie, Eli Lilly, Galapagos, Gilead, and Pfizer Inc. NB is a consultant for Amgen, Eli Lilly, Grunenthal, Pfizer Inc, Roche, and Sanofi; reports personal fees from GSK and from Global Alliance for Patient Access, and is International Liaison Officer for EULAR. TMB, DW, and OO-A are employees of RTI Health Solutions, who were paid consultants to Pfizer Inc in connection with the development of this manuscript. JW, CC, PD, DPdL, DG, and LF are employees and shareholders of Pfizer Inc. AJK is a shareholder of Novartis; a consultant for AbbVie, Boehringer Ingelheim, Flexion, Genzyme, Janssen, Pfizer Inc, Regeneron, Sanofi, SUN Pharma, and UCB; a member of speakers’ bureaus for Celgene, Flexion, Genentech, Genzyme, Horizon, Ironwood, Merck, Novartis, Pfizer Inc, Regeneron, and Sanofi; and President of Altoona Center for Clinical Research. He also reports personal fees from Amgen, personal fees from Gilead, personal fees from GSK, outside the submitted work. CZ has received research grants from Amgen, Biogen, Eli Lilly, Merck, Novartis, Pfizer Inc, and Sanofi; and is a member of speakers’ bureaus for Amgen, Eli Lilly, Pfizer Inc, and Sanofi. The authors report no other conflicts of interest in this work., (© 2020 Taylor et al.)

Objective: To evaluate physician knowledge of and attitudes about binge-eating disorder (BED) and the value and ease-of-use of the 7-item Binge Eating Disorder Screener (BEDS-7) in clinical practice., Methods: Two internet surveys (wave 1: April 15-May 6, 2015; wave 2: August 19-25, 2015) were administered to primary care physicians serving adults (PCPs-adults) and psychiatrists. Wave 1 invitees were US-based physicians spending ≥ 50% of their time in direct patient care and reporting "no" to "some to average" experience with eating-disorder patients. Respondents completing wave 1 qualified for wave 2., Results: Among the 1,047 physicians who responded, 313 did not meet at least 1 of the screening criteria, including 3.15% of respondents who spent < 50% of their time in direct patient care. Overall, 122 PCPs-adults and 123 psychiatrists completed both waves. Physician groups spent similar mean ± SD amounts of time providing direct patient care (PCPs-adults: 94.66% ± 8.4%, psychiatrists: 91.15% ± 12.2%). Based on composite scores, BED knowledge increased from wave 1 to wave 2 in PCPs-adults (P < .001) and psychiatrists (P < .05). Composite scores pertaining to knowledge of and comfort with diagnosing and treating BED were lower for PCPs-adults than psychiatrists in both waves (all P < .001). Based on wave 2 responses, the BEDS-7 was used by 32.0% of PCPs-adults and 26.8% of psychiatrists. All BEDS-7 users (100%) indicated the screener was "very" or "somewhat" valuable, and nearly all users (psychiatrists: 100%, PCPs-adults: 97.4%) reported it was "very" or "reasonably" easy to use. BEDS-7 users reported that important uses of the screener included assisting clinicians in identifying BED patients and encouraging/initiating doctor-patient discussions about BED., Conclusions: These results support the utility of the BEDS-7 in clinical practice, with BEDS-7 users reporting that it is a highly valued and easy-to-use screener. Furthermore, both PCPs-adults and psychiatrists acknowledged the importance of being knowledgeable about BED., (© Copyright 2017 Physicians Postgraduate Press, Inc.)

Background: Treatment of schizophrenia and major depressive disorder (MDD) with atypical antipsychotics (AAPs) show improved efficacy and reduced side effect burden compared with older antipsychotic medications. However, a risk of treatment-emergent adverse events (TEAEs) remains. TEAEs are hard to quantify and perspectives on the importance of TEAEs differ across patients and between patients and physicians. The current study is a qualitative assessment that investigates TEAEs of AAPs from both patient and physician perspectives to provide better understanding of the occurrence and burden of TEAEs associated with these medications., Methods: Focus groups comprised of patients with MDD and interviews with patients with schizophrenia were conducted at two qualitative research facilities, along with a physician focus group at one of the facilities. Information collected from patients included an exhaustive list of TEAEs experienced, and the frequency and level of bother of each TEAE; from psychiatrists, information included an exhaustive list of TEAEs based on personal observations and patient report, frequency of TEAEs, clinically important TEAEs, and levels of patient-perceived bother. Standard qualitative analysis methods were used to identify, quantify, characterize, and summarize patterns found in the data collected., Results: A total of 42 patients (25 with MDD and 17 with schizophrenia) and 4 psychiatrists participated in the study. TEAEs reported as bothersome across both patients groups included cognitive issues, weight gain and/or increased appetite, low energy, extrapyramidal symptoms (EPS), and need to sleep/excessive sleep/excessive sleepiness. TEAEs considered more bothersome by patients with schizophrenia were weight gain, low energy, EPS, mental anxiety, and increased positive symptoms; those considered more bothersome by patients with MDD were cognitive issues, somnolence/sedation, and flat/restricted affect. TEAEs considered most clinically important by psychiatrists included metabolic syndrome, weight gain, neutropenia, hyperglycemia, and QT prolongation; those TEAEs considered most bothersome to patients from physicians' perspectives included weight gain, reduced sexual desire or performance, EPS, akathisia, and hormonal issues., Conclusions: The wide range of TEAEs that are both frequent and bothersome and the variation in perceived burden according to diagnosis highlight the need for a tailored TEAE-awareness approach when choosing an AAP.

Background: Metachromatic leukodystrophy (MLD) is a rare disease with three forms based on the age at onset of signs and symptoms. The objective of this study was to develop a caregiver-reported clinical outcome assessment that measures impairments in physical functioning related to activities of daily living in patients with juvenile MLD., Methods: A targeted literature review and exploration of proprietary research, including a conceptual model, were conducted. Concept elicitation interviews were conducted to elicit additional concepts related to impairments in patients' physical functioning with caregivers of five individuals with juvenile MLD. Based on the research review and concept elicitation interviews, the conceptual model was updated and the Impact of Juvenile Metachromatic Leukodystrophy on Physical Activities (IMPA) scale draft items were created. Cognitive debriefing interviews were conducted with six additional caregivers to finalize the conceptual model and to refine the IMPA scale., Results: Initially, 17 potentially important concepts were identified and addressed in the draft IMPA scale. Following the cognitive debriefing interviews, 15 activities/items remained: brush teeth, comb/brush hair, bathe/shower, dress self, eat, drink, use pencil/crayon, sit upright, use toilet, get on/off toilet, walk, use stairs, get in/out of bed, get in/out of chair/wheelchair, and get in/out of vehicle. Items that did not uniquely contribute to the purpose of the instrument were removed., Conclusion: The IMPA scale, developed according to regulatory standards, provides a means of detecting changes in activities of daily living in individuals with juvenile MLD and can hence be used in future studies to measure benefits of therapeutic interventions., Competing Interests: This study was conducted in accordance with the ethical principles outlined in the Declaration of Helsinki 2008 and reviewed and approved by the RTI International Institutional Review Board. Informed consent was obtained before participation.All authors approved this manuscript and provided consent for publication.The authors declare the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article. This study was conducted by RTI Health Solutions under the direction and funding of Shire Development LLC. RTI Health Solutions was contracted by Shire to design and implement this project. T.M.B., S.M., and S.E.F. are employees of RTI Health Solutions. L.S.D. is currently at Pfizer, Inc. but was employed at Shire Development LLC during the conduct of this project and is currently a shareholder of Shire stock. Shire develops and manufactures treatments for rare diseases, including metachromatic leukodystrophy. The authors have no additional financial relationships or otherwise to declare.Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.

Objective: Develop a brief, patient-reported screening tool designed to identify individuals with probable binge-eating disorder (BED) for further evaluation or referral to specialists., Methods: Items were developed on the basis of the DSM-5 diagnostic criteria, existing tools, and input from 3 clinical experts (January 2014). Items were then refined in cognitive debriefing interviews with participants self-reporting BED characteristics (March 2014) and piloted in a multisite, cross-sectional, prospective, noninterventional study consisting of a semistructured diagnostic interview (to diagnose BED) and administration of the pilot Binge-Eating Disorder Screener (BEDS), Binge Eating Scale (BES), and RAND 36-Item Short-Form Health Survey (RAND-36) (June 2014-July 2014). The sensitivity and specificity of classification algorithms (formed from the pilot BEDS item-level responses) in predicting BED diagnosis were evaluated. The final algorithm was selected to minimize false negatives and false positives, while utilizing the fewest number of BEDS items., Results: Starting with the initial BEDS item pool (20 items), the 13-item pilot BEDS resulted from the cognitive debriefing interviews (n = 13). Of the 97 participants in the noninterventional study, 16 were diagnosed with BED (10/62 female, 16%; 6/35 male, 17%). Seven BEDS items (BEDS-7) yielded 100% sensitivity and 38.7% specificity. Participants correctly identified (true positives) had poorer BES scores and RAND-36 scores than participants identified as true negatives., Conclusions: Implementation of the brief, patient-reported BEDS-7 in real-world clinical practice is expected to promote better understanding of BED characteristics and help physicians identify patients who may have BED.

Background: Moderate to severe plaque psoriasis (with or without psoriatic arthritis) places significant burden on patients' lives., Objective: Explore and document patients' experiences of living with psoriasis, including symptoms, treatments, impact on daily lives and patient-reported functioning., Methods: In a US-based, non-interventional study, narrative interviews were conducted at baseline and again within 16 weeks. In interviews, patients with moderate to severe psoriasis indicated symptoms, ranked symptoms according to level of bother and indicated areas of their lives affected by psoriasis. Transcripts of interviews were coded for themes. Measurements of psoriasis severity including BSA, PGA and PASI were recorded., Results: Symptoms reported most frequently included flaking/scaling (non-scalp areas), itching/scratching and rash, while the most bothersome symptoms were itching/scratching, flaking/scaling (non-scalp areas) and skin pain. Frequently reported impact areas were social and emotional., Conclusion: Broad-reaching interviews with patients with psoriasis show that these patients suffer in many aspects of their lives and in ways not indicated by typical psoriasis severity measures. Patients with psoriatic arthritis reported symptoms and disease-related complications at higher rates than those without arthritis. Physicians' explorations of the effect of psoriasis on patients' life events could aid in managing these patients.

Objective: Develop and validate the ADHD Benefits of Coaching Scale (ABCS), a self-report instrument designed to measure benefits of ADHD coaching in college students., Method: Concepts were elicited from interviews with ADHD coaches and college students receiving ADHD coaching. Draft items were tested in cognitive interviews among ADHD students. Item and scale reliability and validity of ABCS were evaluated using web-survey data from students reporting ADHD. Mean ABCS scores for coached and noncoached groups were compared., Results: Five coaches and 14 students participated in concept elicitation interviews; 10 students participated in the cognitive interviews; 209 students completed the web-survey. The 10-item ABCS showed satisfactory internal consistency reliability and validity and differentiated between coached (n = 17) and noncoached groups (n = 192; p < .01)., Conclusion: To assess benefits of ADHD coaching in college students, the ABCS was developed and psychometrically validated in accordance with Food and Drug Administration's (FDA) patient-reported outcome guidance., (© 2014 SAGE Publications.)

Hospital management and leadership systems are associated with organizational success and quality care. The Strategy and Leadership Systems Capability Evaluation (CE) survey was developed by GE Healthcare to assess management and leadership systems at health care institutions, serve as a benchmark for improvement, and measure progress. To assess the psychometric properties of the 29-item CE survey, including the factor structure, scoring algorithm, reliability, and discriminant validity, an online survey was completed by 3450 employees at 15 US hospitals. Of these employees, 609 worked at a hospital where a leadership and management intervention occurred after the initial survey administration. Data were also collected on job level, number of hospital beds, hospital ownership, location, community type, and the implementation of hospital interventions. Item response frequencies showed no floor or ceiling effects and limited missing data. Interitem correlations were strong without obvious redundancies, and factor analysis suggested a unidimensional scale. The resulting scale had strong internal consistency and was able to discriminate among known groups. The CE survey was developed to evaluate management and leadership systems at health care institutions. This study provides psychometric evidence in support of the reliability, validity, and scoring structure of this survey.

Objective: The office time required for primary care physicians (PCPs) to diagnose, treat and manage fibromyalgia (FM) patients can be extensive. The study objective was to determine if PCPs can positively impact practice economics by requiring fewer patient visits and less office time, while still achieving an acceptable quality of life, as reported by the physician., Study Design: Survey of PCPs who diagnose, manage and treat FM patients., Methods: Surveys were administered to US private practice PCPs, obtaining information on the number of office visits, and time spent with FM patients. PCPs were allotted into two groups: FM-efficient (FME; n = 40) and FM usual care (FMUC; n = 54), based on their reported ability to achieve an acceptable quality of life for ≥50% of their FM patients in less than four office visits post FM diagnosis. An economic model estimated the monetary value of each PCP cohorts' time spent with a newly diagnosed FM patient over a 2-year timeframe., Results: Significant office time cost differences across 2 years exist between FME PCPs and FMUC PCPs ($840 vs. $1117, P < 0.05). FME PCPs had a significantly lower cost of scheduled time to confirm diagnosis ($243 vs. $339, P < 0.05) and time to find right treatment ($264 vs. $365, P < 0.05) than FMUC PCPs. Both groups incurred costs related to excess visit time, but it was less for FME PCPs ($119, 29 minutes) than FMUC PCPs ($182, 44 minutes, P < 0.01), driven by quicker diagnosis confirmation (P < 0.01) and treatment initiation (P < 0.01)., Conclusions: Research suggests that efficient FM care delivery during diagnosis and treatment can be associated with improved practice economics., (© 2011 Blackwell Publishing Ltd.)

Background: Oral anticoagulant (OA) medication is the recommended therapy for reducing the risk of thromboembolic complications in patients with atrial fibrillation (AF), and warfarin is the medication most frequently used. However, nonadherence associated with OA medications may lead to considerable health risks. A conceptual model of OA medication adherence in patients with AF could clarify factors affecting adherence, thereby assisting in the development and structuring of adherence-promotion programs. To our knowledge, such a model, driven by information obtained directly from patients, has never been developed., Objective: To develop a conceptual model of adherence to OA medication based on a literature review and patient feedback via qualitative research among patients with AF., Methods: A literature search was conducted of English-language articles published between the years 2005 and 2010 that related to factors affecting OA medication adherence, excluding articles pertaining to AF associated with mechanical heart valve replacement. To expand on the literature review findings, 4 focus groups totaling 38 participants aged 60 years or older, diagnosed with nonvalvular AF, and currently taking any OA medication were conducted in 2011. Participants completed the Modified Morisky Scale (MMS), with subscales measuring motivation and knowledge, and were asked about daily processes and behaviors related to taking OA medication. The identification of focus group themes was based on the frequency of participant report and endorsement; themes were spontaneously mentioned or supported by at least 2 people in each of at least 3 focus groups. Model concepts, based on focus group themes and factors identified in the literature review, were determined by the consensus of 3 authors., Results: 181 publications were identified; 30 were selected for full-text review. The focus group participants had a mean age of 69.9 years. Most participants reported a diagnosis of hypertension (86.8%, n=33), high cholesterol (50.0%, n=19), heart disease or chronic heart failure (31.6%, n=12), or diabetes (28.9%, n=11). Most (89.5%, n=34) were taking warfarin. About one-half (52.6%, n=20) had been taking an OA medication for less than 5 years. On the MMS, 78.9% of participants reported high levels of motivation, and 100% reported high levels of knowledge. Four concepts emerged from the focus groups and were supported by the literature for inclusion in the model: (a) knowledge base of the disease and continued reinforcement (i.e., health care professional reinforcement); (b) short-term and long-term motivation (e.g., avoidance of negative health consequences); (c) personalized system, habit formation, and system adaptation (e.g., developing a routine or external reminders); and (d) self-efficacy loop (i.e., the personalized system and its adaptability are reinforced as patients become more consistent, confident, and adherent). The literature review also suggested other factors that may also affect patient adherence (e.g., demographic, psychosocial, cognitive)., Conclusion: Adherence in patients with AF is complex and involves multiple factors, some specific to each individual and others more general. This model identifies an adherence process that can guide opportunities for effective interventions, such as educational and behavioral programs targeted at these processes, to improve patient adherence to OA medication.

Background: Chronic immune thrombocytopenia (ITP) is a debilitating autoimmune disorder that causes a reduction in blood platelets and increased risk of bleeding. ITP is currently managed with various pharmacologic therapies and splenectomy.This study was conducted to assess patient perceived and reported treatment side effects, as well as the perceived burden or bother, and need to reduce or stop treatment, associated with these side effects among adult patients with chronic ITP., Methods: A Web-enabled survey was administered to members of a US-based ITP patient support group. Patients reported demographic and clinical characteristics, ITP treatments' side effects for treatments received since diagnosed, level of bother (or distress), and need to reduce or stop treatment, associated with side effects. Current and past exposure was assessed for five specific treatment types: corticosteroids (CS), intravenous immunoglobulin (IVIg), anti-D immunoglobulin (anti-D), rituximab (RT), and splenectomy (SPL), as well as for other patient-referenced therapies (captured as "other")., Results: The survey was completed by 589 patients; 78% female, 89% white, mean age 48 years (SD = 14.71), and 68% reported a typical low platelet count of < 50,000/μL. Current or past treatment with CS was reported by 92% (n = 542) of patients, 56% (n = 322) for IVIg, 36% (n = 209) for anti-D, 36% (n = 213) for RT, and 39% (n = 227) for SPL. A substantial proportion of CS-treated patients reported side effects (98%, P < 0.05), were highly bothered by their side effects (53.1%, P < 0.05), and reported the need to stop or reduce treatment due to side effects (37.8%, P < 0.05). Among patients reporting side effects of treatment, significant associations were noted for the number of side effects, aggregate bother of reported side effects, and the need to stop or reduce treatment (all P < 0.05)., Conclusions: Current ITP treatments, particularly corticosteroids, are associated with multiple bothersome side effects that may lead to patients stopping or reducing therapy. Open, informed and complete communication between clinician and patient regarding both the benefits and the side effects of ITP treatment may better prepare patients for their prescribed regimens.

Objectives: Ovarian cancer (OC) typically is diagnosed at advanced stages, in which the primary goal of therapy is to prolong progression-free survival (PFS) and overall survival (OS). In recent years, maintenance therapy has been tested for this purpose in advanced OC (AOC). Literature on maintenance therapy in AOC was systematically reviewed to assess current knowledge regarding the impact of this therapeutic approach., Methods: A MEDLINE search was performed 2/2009 for articles published 1/2001-1/2009 pertaining to OC maintenance therapy guidelines, patterns, and outcomes. A second search used keywords specific to maintenance and included primary studies published in the last 10 years. Of 406 sources identified, 36 primary studies and 16 review articles were included in this systematic review. A third search used the keyword "consolidation" to find maintenance articles not identified through other searches; of 48 additional sources, 13 primary studies and 6 reviews were included. A fourth search of non-MEDLINE-indexed sources yielded 14 additional relevant publications from the same time period., Results: Among practice guidelines identified, only the National Comprehensive Cancer Network (NCCN) 2008 guidelines provide recommendations regarding maintenance therapy, assigning it a category 2B recommendation. No studies were identified that reported current treatment patterns or economic outcomes in maintenance therapy; quality of life data were reported in one study. A variety of agents have been tested for maintenance, with paclitaxel the most commonly evaluated. The Southwest Oncology Group-Gynecologic Oncology Group 178 trial has found that 12 cycles of paclitaxel extend PFS (by 7 months) compared to 3 months paclitaxel, but could not adequately evaluate OS., Conclusions: Maintenance therapy may improve clinical outcomes in AOC, but additional research is needed to demonstrate an OS advantage. Future studies should investigate the long-term clinical benefit of maintenance treatment and its impact on resource utilization and health-related quality of life.

Objective: To assess the economic impact of a hypertension educational and awareness program ("BP Downshift") on improvement in blood pressure among commercial driver license (CDL) employees in a large southeastern U.S. electric utility company., Methods: An economic simulation model was developed to evaluate the costs/cost savings the company realized from implementation of the BP DownShift Program in terms of changes in work productivity, CDL certification status, hypertension treatment, cardiovascular disease events, and diabetes care., Results: Model results showed a 16.3% (more than $540,000) reduction in costs for a sample of 499 CDL employees over 2 years. On a per-employee basis, 2-year cost savings were estimated to be $1084, or $542 annually., Conclusions: Study results will interest employers who are considering using disease management and health promotion programs to control costs of hypertension and other chronic illnesses.

Objective: Federal regulations governing transportation safety disqualify commercial drivers with persistent uncontrolled hypertension. We sought to determine whether a hypertension management and health promotion program designed for commercial drivers improved blood pressure (BP) outcomes among drivers employed by a self-insured utility company., Methods: This retrospective study examined the employment-related medical examinations of 501 randomly selected commercial drivers for measurements of BP, height, and weight taken before and after the intervention., Results: After the program, significantly fewer employees had uncontrolled hypertension according to the Department of Transportation hypertension guidelines (17.2% vs 26.1%, P < 0.01). This improvement was consistent across subgroups defined by diabetes, obesity, and use of antihypertensive medication., Conclusions: An education program improved control of BP among commercial drivers, improving their health and safety, and reducing the number at high risk of medical disqualification.