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Chemotherapy; Quality of life; Symptoms Quimioterapia; Calidad de vida; Síntomas Quimioteràpia; Qualitat de vida; Símptomes Background In a phase III, randomised, active-controlled study (EMPOWER-Cervical 1/GOG-3016/ENGOT-cx9; R2810-ONC-1676; NCT03257267) and cemiplimab significantly improved survival versus investigator's choice of chemotherapy among patients with recurrent cervical cancer who had progressed on platinum-based therapy. Here we report patient-reported outcomes in this pivotal study. Methods Patients were randomised 1:1 to open-label cemiplimab (350 mg intravenously every 3 weeks) or investigator's choice of chemotherapy in 6-week cycles. Patients completed the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 during cycles 1–16. Least-squares mean changes from baseline in global health status (GHS)/quality of life (QoL) and physical functioning (PF) were secondary end-points in the statistical hierarchy. Results Of 608 patients (304/arm), 77.8% patients had squamous cell carcinoma and 22.2% patients had adenocarcinoma. Questionnaire completion rates were ∼90% throughout. In the squamous cell carcinoma population, overall between-group differences statistically significantly favoured cemiplimab in GHS/QoL (8.49; 95% confidence interval [CI]: 3.77–13.21; P = 0.0003) and PF (8.35; 95% CI: 4.08–12.62; P < 0.0001). Treatment differences favoured cemiplimab in both histologic populations by cycle 2. Overall changes from baseline in most functioning and symptom scales favoured cemiplimab, with clinically meaningful treatment differences in role functioning, appetite loss and pain in both populations. The sensitivity analyses, responder analyses and time to definitive deterioration favoured cemiplimab in both populations. Conclusions Cemiplimab conferred favourable differences in GHS/QoL and PF compared with chemotherapy among patients with recurrent cervical cancer, with benefits in PF by cycle 2, and clinically meaningful differences favouring cemiplimab in role functioning, appetite loss, and pain. This work was supported by Regeneron Pharmaceuticals, Inc., and Sanofi.

Currently, there is no patient-reported outcome (PRO) instrument specifically designed to evaluate hypertrophic cardiomyopathy (HCM).We present the development and psychometric validation of a novel PRO measure, the HCM Symptom Questionnaire version 1.0 (HCMSQv1.0).Cognitive debriefing interviews and a card-sorting task were conducted in 33 patients with HCM to support development of the HCMSQv1.0, showing the scale to be interpretable and relevant to patients' experiences. Baseline blinded data from two trials (EXPLORER-HCM and MAVERICK-HCM) were pooled (N = 299) to develop the scoring algorithm of HCMSQv1.0. Measurement properties were examined, followed by a meaningful-change analysis to interpret scores. Rasch modeling, mixed-model repeated measures, exploratory factor analysis, confirmatory factor analysis, and missing-data simulation analysis informed the number of domains and the items in each domain.The scoring algorithm for HCMSQv1.0 consists of four domains: shortness of breath, tiredness, cardiovascular symptoms, and syncope; plus a total score, with higher scores indicating more severe symptoms. Item characteristics, internal consistency, test-retest reliability, construct validity, and responsiveness were acceptable. A clinically meaningful responder definition of 1-2 points on the HCMSQv1.0 score for shortness of breath and total score, and approximately 1 point on the tiredness and cardiovascular symptom scores, was calculated based on distribution- and anchor-based methods.Our findings support the HCMSQv1.0 as a fit-for-purpose PRO instrument for assessing treatment benefit in patients with HCM. Studies in larger patient populations are ongoing to confirm responder definition and scoring approaches encompassing key HCM symptoms.

Patients experience a wide range of signs, symptoms, and impacts related to coronavirus disease 2019 (COVID-19). A patient-reported outcome (PRO) item bank that measures the most relevant patient experiences is needed to fully evaluate treatment benefit in COVID-19 clinical trials.A review of the literature and social media informed a novel PRO item bank of COVID-19 signs, symptoms, and impacts and general pandemic impacts. Twenty 1:1 concept elicitation and cognitive debriefing interviews were conducted with adults in the US who had symptomatic COVID-19. A conceptual model was developed and the PRO item bank refined following interviews.A heterogenous set of signs, symptoms, and impacts of COVID-19, as well as impacts associated with the pandemic overall, was identified. Fifty-five short-term and long-term signs and symptom items, 26 items assessing disease-related impacts, and seven items evaluating pandemic-related impacts are included in the item bank.The novel and preliminarily content-valid IQVIA COVID-19 Daily Diary Item Bank

Hypertrophic cardiomyopathy (HCM) symptoms include shortness of breath (SOB), fatigue, chest pain, palpitations, dizziness, and fainting. The HCM Symptom Questionnaire (HCMSQ), the only patient-reported outcome instrument designed to specifically measure HCM symptoms, yields four domain scores (SOB, tiredness, cardiovascular symptoms, syncope) and a total score. We evaluated the longitudinal psychometric properties of the HCMSQ using baseline to week 30 data from the phase III EXPLORER-HCM trial (NCT03470545).Test-retest reliability was assessed via intraclass correlation of patients with stable Patient Global Impression of Change (PGIC) and Patient Global Impression of Severity (PGIS) responses. Sensitivity to change was assessed via Spearman correlations with the Kansas City Cardiomyopathy Questionnaire (KCCQ-23) and the EuroQoL visual analogue scale (EQ VAS), and via one-way ANOVA comparing change groups defined on clinical (New York Heart Association [NYHA] class, left ventricular outflow tract [LVOT] gradient, peak oxygen consumption [pVOAll HCMSQ scores showed strong evidence of test-retest reliability (intraclass correlation coefficient 0.70). Sensitivity to change was demonstrated with mostly strong/moderate correlations with KCCQ-23 and EQ VAS, and significant differences (p ≤ 0.05) in PGIS, PGIC, pVOResults suggest that HCMSQ is fit for purpose in capturing HCM symptoms and may provide evidence of treatment benefit from the patients' perspectives.

Background In the EMPOWER-Lung 1 trial (, NCT03088540), cemiplimab conferred longer survival than platinum-doublet chemotherapy for advanced non-small cell lung cancer (NSCLC) with programmed cell death-ligand 1 (PD-L1) >= 50%. Patient-reported outcomes were evaluated among trial participants. Methods Adults with NSCLC and Eastern Cooperative Oncology Group performance status 0 to 1 were randomly assigned cemiplimab 350 mg every 3 weeks or platinum-doublet chemotherapy. At baseline and day 1 of each treatment cycle, patients were administered the European Organization for Research and Treatment of Cancer Quality of Life-Core 30 (QLQ-C30) and Lung Cancer Module (QLQ-LC13) questionnaires. Mixed-model repeated measures analysis estimated overall change from baseline for PD-L1 >= 50% and intention-to-treat populations. Kaplan-Meier analysis estimated time to definitive deterioration. Results In PD-L1 >= 50% patients (cemiplimab, n = 283; chemotherapy, n = 280), baseline QLQ-C30 and QLQ-LC13 scores showed moderate-to-high functioning and low symptom burden. Change from baseline favored cemiplimab on global health status/quality of life (GHS/QOL), functioning, and most symptom scales. Risk of definitive deterioration across functioning scales was reduced versus chemotherapy; hazard ratios were 0.48 (95% CI, 0.32-0.71) to 0.63 (95% CI, 0.41-0.96). Cemiplimab showed lower risk of definitive deterioration for disease-related (dyspnea, cough, pain in chest, pain in other body parts, fatigue) and treatment-related symptoms (peripheral neuropathy, alopecia, nausea/vomiting, appetite loss, constipation, diarrhea) (nominal p < .05). Results were similar in the intention-to-treat population. Conclusions Results support cemiplimab for first-line therapy of advanced NSCLC from the patient's perspective. Improved survival is accompanied by improvements versus platinum-doublet chemotherapy in GHS/QOL and functioning and reduction in symptom burden. WOS:000875663600001 Q1

Contains fulltext : 233836.pdf (Publisher’s version ) (Closed access) In order to minimize risk of infection and potential foot complications, it is recommended that people with and without diabetes check their feet regularly for problems such as cuts, sores, blisters or calluses. Hence, an understanding of how to craft effective messages to encourage people to check their feet is important. Two studies investigated the use of narrative stories to encourage foot problem detection behaviour; Study 1 in a general population sample (N = 193), and Study 2 in a sample of people with type 1 or type 2 diabetes (N = 129). In both studies participants were randomised to either (a) receive an information sheet written in first-person narrative; (b) the same in non-narrative format; or (c) no information sheet. Changes in weekly detection behaviour was the outcome of interest. In both studies, greater detection behaviour was observed in the narrative message condition vs. non-narrative condition and the non-narrative condition vs. no information condition. Our findings have implications for the design of health messages in delivering effective foot care education to people with and without diabetes, suggesting that narrative information sheets may be more effective than non-narrative information sheets. 20 p.

Item does not contain fulltext Background: Many people with diabetes will develop foot ulcers. To reduce risk, it is recommended that the feet are protected against harm and checked daily. Spouses can help people with diabetes care for their feet. Methods: A randomized parallel arm design compared information sheets given to participants with diabetes and their spouses (dyad group; n = 64) to an information sheet given only to participants with diabetes (individual group; n = 69). The self-reported number of days that the participant with diabetes' feet were (1) checked for problems and (2) protected against problems occurring (by the person with diabetes and/or the spouse) were summed for the week after receiving the information sheet. ANCOVAs tested the effects of group. Results: Frequency of foot detection behavior (Participant + Spouse) was significantly higher in the dyad group compared with the individual group. This was not the case for foot protection behavior (Participant + Spouse). Findings revealed greater levels of spousal support (for both protection and detection behavior) in the dyad group compared to the individual group. Conclusions: Clinical recommendations and advice on foot care delivered both to people with diabetes and their spouses can encourage greater foot care than if delivered to the patient alone. 18 p.

Objectives/hypothesis The 22-item Sinonasal Outcome Test (SNOT-22) is a validated chronic rhinosinusitis health-related quality-of-life outcome (HRQoL) measure; however, SNOT-22 domains have not been validated specifically for chronic rhinosinusitis with nasal polyps (CRSwNP). Study design Validation of SNOT-22 domain structure, using data from 3 randomized, placebo-controlled, double-blinded, multicenter clinical trials of dupilumab in adults with moderate-to-severe CRSwNP. Methods Preliminary dimensional structure was derived by exploratory factor analyses of SNOT-22 data from a phase 2 trial (NCT01920893) of dupilumab for the treatment of CRSwNP. Data from 2 phase 3 clinical trials (NCT02912468 and NCT02898454) were then used for confirmatory factor analysis, and evaluated for reliability, construct validity, and responsiveness. In all three trials, the SNOT-22 was administered electronically on a tablet and trial participants were required to answer all items. Results Factor analysis supported five domains: Nasal, Ear/Facial, Sleep, Function, and Emotion. Correlations between domains were moderate to high, ranging from 0.53 (Nasal-Emotion) to 0.88 (Function-Sleep). Construct validity was mostly supported; relationships with other measures were almost always in the intended direction and magnitude. Internal consistency reliability also confirmed questionnaire structure with strong Cronbach's alpha values (all >0.80). Moderate-to-high correlations were observed between change in SNOT-22 domain scores and other study patient-reported outcome measures, along with large effect-size estimates (≥0.7), demonstrating responsiveness of the Nasal, Sleep, and Function domains. Emotion and Ear/Facial domains had small-to-moderate effect sizes. Conclusions Psychometric analyses support the validity, reliability, and responsiveness of five domains of SNOT-22 (Nasal, Ear/Facial, Sleep, Function, and Emotion) for assessing symptoms and impact on HRQoL in patients with CRSwNP. Laryngoscope, 2021.

Background Understanding the patient’s perception of their disease is vital for guiding care decisions. The current study aimed to identify the most predominant experiences in women diagnosed with, and treated for, ovarian cancer in terms of disease-related symptoms, treatment-attributed side effects and their impacts. Methods Semi-structured qualitative interviews about disease-related symptoms, treatment-attributed side effects and their impacts were conducted with women who were being treated for ovarian cancer in Europe (n = 55) or in the USA (n = 9). The women were also asked to rate the bothersomeness of the symptoms, side effects and impacts that they mentioned during the interview. Symptoms, side effects and impacts were identified from coded interview transcripts using an iterative coding framework. Results Bloating, abdominal pain, tiredness and frequent urination were the most frequently expressed symptoms, and were reported by 72%, 67%, 64% and 55% of women, respectively, which together constituted approximately 30% of all symptom expressions. The most bothersome symptoms were reported as bloating, abdominal pain, pain in the side, tiredness and fatigue. The most frequently expressed side effects were hair loss, neuropathy, tiredness and nausea, which were reported by 84%, 63%, 61% and 61% of women, respectively. The most bothersome reported side effects were constipation, nausea, diarrhoea, pain in general, fatigue, weakness, reduced sleep quality and hair loss. Feelings of anxiety, concerns about the future, physical functioning, work limitations and the adoption of coping strategies were the most frequently expressed impacts and were reported by 72–80% of women. Impacts reported as the most difficult to deal with were concerns about the future, emotional difficulties in general, physical functioning, sexual functioning, negative self-image, fatigue, sleep difficulties, financial burden and work limitations. Conclusions In our qualitative study, the most common and most bothersome experiences reported by women treated for ovarian cancer were symptoms of bloating, abdominal pain and tiredness; side effects of hair loss, nausea and tiredness/fatigue; and impacts relating to concerns about the future, physical functioning and work limitations. We suggest that clinicians measure these experiences consistently and take them into consideration when making treatment decisions.

Introduction Individualised benefit–harm assessments can help identify patient-perceived benefits and harms of a treatment, and associated trade-offs that may influence patients’ willingness to use a treatment. This research presents the first use of a patient-reported outcome measure designed to assess patient-perceived benefits and disadvantages of drugs received during clinical studies. Methods The Patient’s Qualitative Assessment of Treatment (PQAT) was developed in English and cognitively tested with US (n = 4) and Canadian (n = 3) patients with type 1 and type 2 diabetes mellitus (T2DM). The revised version of the PQAT comprises three qualitative open-ended questions focused on the benefits and disadvantages of treatment and reasons why patients would choose to continue/discontinue treatment. A final quantitative question asks patients to evaluate the balance between benefits and disadvantages using a 7-point scale. The revised version of the questionnaire was administered as an exploratory endpoint in a phase II clinical trial for a new injectable treatment for T2DM. Qualitative data were analysed using thematic analysis, and relationships between qualitative and quantitative data were identified. Results Patient-reported benefits of treatment administered during the clinical trial included clinical markers of efficacy and subjective markers. Disadvantages reported by patients were mainly related to drug adverse effects or to the mode of administration. Of the 57 patients completing the PQAT, 70.2% reported being willing to continue treatment, with 59.6% reporting that the benefits outweighed the disadvantages. The reported benefits of feeling better and improved energy levels were more likely to be associated with a more positive ratio (70% and 71.4%, respectively), while the disadvantages of fatigue, headaches, and stomach pain were associated with a negative ratio and patients not being willing to continue the treatment. Conclusions The PQAT is a unique patient-reported outcome tool designed to aid understanding patients’ real experience of benefits and disadvantages of a treatment. It combines the richness of qualitative data with quantitative data—information valuable for various stakeholders to make well-informed treatment decisions. Trial Registration ClinicalTrials.gov identifier: NCT02973321. Electronic supplementary material The online version of this article (10.1007/s40264-019-00877-4) contains supplementary material, which is available to authorized users.

Introduction The Patient-Oriented Eczema Measure (POEM) assesses patient-reported frequency of atopic dermatitis (AD) symptoms, while the Children’s Dermatology Life Quality Index (CDLQI) measures the impact of skin disease on health-related quality of life (HRQoL) in children. There is currently no threshold for clinically meaningful within-person change in POEM or CDLQI scores in adolescents. Here we empirically derive within-person thresholds of meaningful within-person change in POEM and CDLQI scores in adolescents with moderate-to-severe AD. Methods Data were used from a phase 3, randomized, double-blind, placebo-controlled trial of dupilumab in adolescents (aged ≥ 12 to

Improving symptoms is a primary treatment goal in patients with obstructive hypertrophic cardiomyopathy. Currently available pharmacological options for hypertrophic cardiomyopathy are not disease-specific and are often inadequate or poorly tolerated. We aimed to assess the effect of mavacamten, a first-in-class cardiac myosin inhibitor, on patients' health status-ie, symptoms, physical and social function, and quality of life.We did a health status analysis of EXPLORER-HCM, a phase 3, double-blind, randomised, placebo-controlled trial. The study took place at 68 clinical cardiovascular centres in 13 countries. Adult patients (≥18 years) with symptomatic obstructive hypertrophic cardiomyopathy (gradient ≥50 mm Hg and New York Heart Association class II-III) were randomly assigned (1:1) to mavacamten or placebo for 30 weeks, followed by an 8-week washout period. Both patients and staff were masked to study treatment. The primary outcome for this secondary analysis was the Kansas City Cardiomyopathy Questionnaire (KCCQ), a well validated disease-specific measure of patients' health status. It was administered at baseline and weeks 6, 12, 18, 30 (end of treatment), and 38 (end of study). Changes from baseline to week 30 in KCCQ overall summary (OS) score and all subscales were analysed using mixed model repeated measures. This study is registered with ClinicalTrials.gov, NCT03470545.Between May 30, 2018, and July 12, 2019, 429 adults were assessed for eligibility, of whom 251 (59%) were enrolled and randomly assigned. Of 123 patients randomly assigned to mavacamten, 92 (75%) completed the KCCQ at baseline and week 30 and of the 128 patients randomly assigned to placebo 88 (69%) completed the KCCQ at baseline and week 30. At 30 weeks, the change in KCCQ-OS score was greater with mavacamten than placebo (mean score 14·9 [SD 15·8] vs 5·4 [13·7]; difference +9·1 [95% CI 5·5-12·8]; p0·0001), with similar benefits across all KCCQ subscales. The proportion of patients with a very large change (KCCQ-OS ≥20 points) was 36% (33 of 92) in the mavacamten group versus 15% (13 of 88) in the placebo group, with an estimated absolute difference of 21% (95% CI 8·8-33·4) and number needed to treat of five (95% CI 3-11). These gains returned to baseline after treatment was stopped.Mavacamten markedly improved the health status of patients with symptomatic obstructive hypertrophic cardiomyopathy compared with placebo, with a low number needed to treat for marked improvement. Given that the primary goals of treatment are to improve symptoms, physical and social function, and quality of life, mavacamten represents a new potential strategy for achieving these goals.MyoKardia, a Bristol Myers Squibb company.

Asif H Khan,1 Adeline Abbe,1 Bruno Falissard,2 Paulo Carita,1 Claus Bachert,3,4 Joaquim Mullol,5 Matthew Reaney,6 Jingdong Chao,7 Leda P Mannent,1 Nikhil Amin,7 Puneet Mahajan,8 Gianluca Pirozzi,8 Laurent Eckert1 1Sanofi, Chilly-Mazarin, France; 2Centre de recherche en epidémiologie et santé des populations (CESP), INSERM U1018, Paris, France; 3Upper Airways Research Laboratory, Ghent University, Ghent, Belgium; 4CLINTEC, Karolinska Institutet, Stockholm, Sweden; 5Rhinology Unit & Smell Clinic, ENT Department, Hospital Clínic, Universitat de Barcelona; Clinical and Experimental Respiratory Immunoallergy, IDIBAPS; and CIBERES, Barcelona, Catalonia, Spain; 6IQVIA, Reading, UK; 7Regeneron Pharmaceuticals, Inc., Tarrytown, NY, USA; 8Sanofi, Bridgewater, NJ, USACorrespondence: Asif H KhanSanofi, 1 Avenue Pierre Brossolette, Chilly-Mazarin, 91380, FranceTel +33 1 60 49 77 77Email asif.khan@sanofi.comPurpose: Patient perspective is an important and increasingly sought-after complement to clinical assessment. The aim of this study was to transcribe individual patients’ experience of treatment in a dupilumab clinical trial through free-text responses with analysis using natural language processing (NLP) to obtain the unique perspective of patients on disease impact and unmet needs with existing treatment to inform future trial design.Patients and Methods: Patients with chronic rhinosinusitis with nasal polyps (CRSwNP) who were enrolled in a Phase IIa randomized controlled trial comparing dupilumab with placebo (NCT01920893) were invited to complete a self-assessment of treatment (SAT) tool at the end of treatment, asking, “What is your opinion on the treatment you had during the trial? What did you like or dislike about the treatment?” Free-text responses were analyzed for the overall cohort and according to treatment assignment using natural language processing including sentiment scoring. In a mixed-methods approach, quantitative patient-reported outcome (PRO) results were utilized to complement the qualitative analysis of free-text responses.Results: Of 60 patients enrolled in the study, 43 (71.6%) completed the SAT and responses from 37 patients were analyzed (placebo, n = 16; dupilumab, n = 21). Word analyses showed that the most common words were “smell,” “improve,” “staff,” “great,” “time,” and “good.” Across the whole cohort, “smell” was the most common symptom-related word. The words “smell” and “experience” were more likely to occur in patients treated with dupilumab. Patients treated with dupilumab also had more positive sentiment in their SAT responses than those who received placebo. The results from this qualitative analysis were reflected in quantitative PRO results.Conclusion: “Smell” was important to patients with CRSwNP, highlighting its importance as a patient-centric efficacy outcome measure in the context of clinical trials in CRSwNP.Trial Registration: ClinicalTrials.gov, NCT01920893. Registered 12 August 2013, https://www.clinicaltrials.gov/ct2/show/NCT01920893.Keywords: CRSwNP, free-text data mining, patient perspective, self-assessment, sense of smell

Patient-reported outcome (PRO) research is conducted to gather information on an individual’s disease or treatment experiences. Patient preference (PP) research is conducted to gather an individual’s evaluation of alternatives or choices among outcomes or other attributes. The fields of PRO and PP research have largely been kept separate, partly because the use and audience have been different; although both can influence health care choices. Recent initiatives on the generation of ‘patient experience information’ provide a regulatory framework for simultaneous consideration of PRO and PP data. This should act as an impetus for PRO and PP researchers to collaborate to generate scientifically robust information on patients’ experiences, perspectives, needs, and priorities. This article discusses the similarities between PRO and PP research and proposes a streamlined and harmonized approach from which sponsors can provide regulators (and payers, clinicians, caregivers, and patients) with information derived using psychometrically robust and interpretable PRO and PP methodologies.

The era of patient-focused drug development (PFDD) brings with it a greater use of patient-reported outcome measures (PROMs) in clinical trials. This is facilitated through electronic technology designed to capture PROM data. However, PFDD goes beyond just PROMs, and technology has a key role in capturing timely and patient-relevant information through active and passive means to inform study endpoints. This brief paper aims to highlight four trends the authors have observed across the pharmaceutical industry in using technology to enhance PFDD: (1) capturing qualitative data from patients; (2) using digital health technology tools (DHTTs); (3) employing reactive technology-enabled clinical outcome assessments TeCOA; and (4) generating passive patient experience data. Opportunities and challenges associated with these trends are discussed, and a ‘call to action’ is made to consolidate learning and understanding across science, medical and technology disciplines, and to conduct collaborative research to improve the opportunities and minimize the challenges.

Background Hypertrophic cardiomyopathy (HCM) is a primary myocardial disorder defined by left ventricular hypertrophy that cannot be explained by another cardiac or systemic disease. There is a general lack of knowledge about patients’ perspectives on the symptoms and day-to-day limitations they experience as a result of HCM. We therefore sought an in-depth understanding of patients’ experiences of obstructive (oHCM) and nonobstructive (nHCM) forms of the disease, including symptoms and their quality of life impacts, and to develop a conceptual model to capture them. Methods Development of the HCM conceptual model involved a web-based survey to capture patients’ insights, a targeted literature review (which included relevant guidelines and patient advocacy websites), one-to-one interviews with clinical experts, and one-to-one qualitative concept elicitation interviews with patients. Key symptoms and their impacts most important to patients’ experiences were identified and used to develop a conceptual model of the patient experience with HCM. Results The HCM symptoms reported by patient interviewees (n = 27) were largely consistent with findings from the patient web survey (n = 444), literature review, and interviews with three expert clinicians. The symptoms most commonly reported in patient interviews included tiredness (89%), shortness of breath (89%), shortness of breath with physical activity (89%), and dizziness/light-headedness (89%). Other symptoms commonly reported included chest pain (angina) (70%), chest pain (angina) with physical exertion (70%), and palpitations (fluttering or rapid heartbeat) (81%). The most commonly reported impacts of HCM symptoms on patients’ lives included limitations to physical activities (78%), emotional impacts, including feeling anxious or depressed (78%), and impacts on work (63%). Symptoms and impacts were similar for both oHCM and nHCM. Conclusions A conceptual model was developed, which identifies the core symptoms that patients with oHCM and nHCM reported as most frequent and most important: shortness of breath, palpitations, fatigue/tiredness, dizziness/light-headedness, and chest pain, as well as the impacts those symptoms have on patients’ lives. This HCM conceptual model reflecting patients’ experiences and perspectives was used in the development of a patient-reported outcomes instrument for use in clinical trials and it may also help inform the clinical management of HCM.

Background Hypertrophic cardiomyopathy (HCM) is a primary myocardial disorder defined by left ventricular hypertrophy that cannot be explained by another cardiac or systemic disease. There is a general lack of knowledge about patients’ perspectives on the symptoms and day-to-day limitations they experience as a result of HCM. We therefore sought an in-depth understanding of patients’ experiences of obstructive (oHCM) and nonobstructive (nHCM) forms of the disease, including symptoms and their quality of life impacts, and to develop a conceptual model to capture them. Methods Development of the HCM conceptual model involved a web-based survey to capture patients’ insights, a targeted literature review (which included relevant guidelines and patient advocacy websites), one-to-one interviews with clinical experts, and one-to-one qualitative concept elicitation interviews with patients. Key symptoms and their impacts most important to patients’ experiences were identified and used to develop a conceptual model of the patient experience with HCM. Results The HCM symptoms reported by patient interviewees (n = 27) were largely consistent with findings from the patient web survey (n = 444), literature review, and interviews with three expert clinicians. The symptoms most commonly reported in patient interviews included tiredness (89%), shortness of breath (89%), shortness of breath with physical activity (89%), and dizziness/light-headedness (89%). Other symptoms commonly reported included chest pain (angina) (70%), chest pain (angina) with physical exertion (70%), and palpitations (fluttering or rapid heartbeat) (81%). The most commonly reported impacts of HCM symptoms on patients’ lives included limitations to physical activities (78%), emotional impacts, including feeling anxious or depressed (78%), and impacts on work (63%). Symptoms and impacts were similar for both oHCM and nHCM. Conclusions A conceptual model was developed, which identifies the core symptoms that patients with oHCM and nHCM reported as most frequent and most important: shortness of breath, palpitations, fatigue/tiredness, dizziness/light-headedness, and chest pain, as well as the impacts those symptoms have on patients’ lives. This HCM conceptual model reflecting patients’ experiences and perspectives was used in the development of a patient-reported outcomes instrument for use in clinical trials and it may also help inform the clinical management of HCM.

Objectives: People with type 2 diabetes (T2DM) should check their feet and\ud protect them against harm, but few do. Living with a spouse contributes to\ud good foot care behaviour. This study explores awareness, perceived susceptibility\ud of, and concern about, foot problems, and reported foot care behaviour,\ud and ways in which a spouse may or may not contribute to foot care in\ud T2DM.\ud Methods: 1:1 interviews were conducted with 6 individuals with T2DM. Half\ud had a spouse half did not. There was one person at low, medium and high\ud risk in each sample. Each spouse participated in a separate interview, and the\ud dyads were interviewed together. Interviews were analysed using Applied\ud Thematic Analysis.\ud Results: All participants knew that diabetes was associated with foot problems.\ud Not all people with T2DM thought that they were susceptible; spouses\ud perceived greater susceptibility for the patient. This was unrelated to risk\ud level. Most people with T2DM and all spouses engaged in behaviour to identify\ud problems or protect feet, but rarely both. Spouses’ attitude and behaviour\ud did influence the patients’ own behaviour. At times spouse support was perceived\ud positively, and at times negatively.\ud Conclusion: Engaging spouses in foot care education may improve foot care\ud behaviour.

Background Endometriosis is a common gynecological disorder that causes inflammation and pelvic pain. Endometriosis-related pain is best captured with patient-reported outcome (PRO) measures, however, assessment of endometriosis-related pain in clinical trials has been difficult in the absence of a reliable and valid PRO instrument. We describe the development of the Endometriosis Pain Daily Diary (EPDD), an electronic PRO developed as a survey instrument to assess endometriosis-related pain and its impact on patients’ lives. Methods The EPDD was initially developed on the basis of an existing Endometriosis Pain and Bleeding Diary, a targeted review of relevant literature, clinical expert interviews, and open-ended (concept elicitation) patient interviews in the United States (US) and Japan which captured patients’ experience with endometriosis. Cognitive interviews of patients with endometriosis were conducted to evaluate patient comprehension of the EPDD items. A conceptual model of endometriosis was developed, and meetings with US and European regulatory authorities provided feedback for validating the EPDD in the context of clinical trials. Translatability assessments of the EPDD were conducted to confirm its appropriate interpretation and ease of completion across 17 languages. Results The iterative development progressed through three versions of the instrument. The EPDDv1 included 18 items relating to dysmenorrhea/pelvic pain, dyspareunia and sexual activity, bleeding, hot flashes, daily activities, and use of rescue medication. The EPDDv2 was a larger 43-item survey tested in cognitive interviews and subsequently revised to yield the current 11-item EPDDv3, consisting of five core items relating to dysmenorrhea, non-menstrual pelvic pain, and dyspareunia, and six extension items relating to sexual activity, daily activities, and use of rescue medication. Conclusions The EPDD is a PRO for the evaluation of endometriosis-related pain and its associated impacts on patients’ lives. The EPDD represents an important step in providing a PRO that is relevant to patients with endometriosis-related pain in the context of a clinical study setting (ie, fit-for-purpose), designed to evaluate pain associated with endometriosis, including regulatory agency support for its further exploration in clinical trials.

Introduction Immune thrombocytopenia (ITP) is a rare autoimmune disorder in which there is an increase in platelet destruction and a decrease in platelet production, resulting in low platelet count, high bleeding risk and impact on quality of life (QoL). A critical appraisal of the literature was conducted to understand the patient experience with ITP and its treatment, and to assess which clinical outcome assessment (COA) instruments can best capture the ITP patient experience and changes due to disease management. Methods A focused PubMed search was performed for qualitative studies describing patient experience with ITP published prior to June 2019. The search paired population terms (ITP) with qualitative study design terms ("qualitative", "focus group/s", "interview/s", "blog"). Grey literature (congresses, patient advocacy group websites) was also consulted. Key patient-reported concepts of interest for measurement in clinical trials were identified. A second review was conducted to identify COA instruments used in ITP to measure the key concepts using PROQOLID (no limits; prior to November 2019), Clinicaltrials.gov (Phase 2 and 3; November 2009-2019), and PubMed (October 2009-2019). COA instruments were critically appraised based on their published psychometric properties and use in regulatory labels and health technology assessment (HTA) appraisal decisions. Results Of the 52 articles screened in the initial search, 2 relevant articles were identified. One reported the development of ITP-patient assessment questionnaire (ITP-PAQ), a disease-specific measure of health-related QoL (HRQoL) in adults with ITP (Mathias et al. Health and Quality of Life Outcomes 2007); the second introduced a conceptual model informed by literature, clinical expertise and qualitative research to describe the impact of ITP and its treatment on patients' HRQoL (Mathias et al. Health and Quality of Life Outcomes 2008). This conceptual model proposed 1 biological variable (low platelet count), 2 main determinants of HRQoL (symptoms of ITP, treatment side-effects) and 5 conceptual domains of HRQoL (emotional health, functional health, social and leisure, reproductive health, work). Fatigue and bleeding/bruising symptoms were considered the main determinants of HRQoL change for patients with ITP. However, the literature supporting this conceptual model was over 15 years old, from one country (US) and conducted in one clinical setting (tertiary care). A third recent publication reporting an interim analysis from the ITP World Impact Survey in 1,400 patients was identified from grey literature (Cooper et al. EHA Library 2018; PF654). Fatigue and bleeding (including petechiae) were the most frequently reported symptoms, at diagnosis and at time of survey completion, and fatigue was reported as one of the most severe symptoms. Twenty-two COA instruments used in ITP were identified for assessments of bleeding/bruising, fatigue/tiredness, anxiety/fear, and/or avoidance of physical activity. From this search, 8 instruments were analysed in depth: ITP-PAQ; Functional Assessment of Cancer Therapy - Thrombocytopenia 6 (FACT-Th6); FACT-Th11/18; Functional Assessment of Chronic Illness - Fatigue (FACIT-F); WHO Bleeding Score; ITP Bleeding Score; ITP-specific bleeding assessment tool (ITP-BAT); Short Form 36 (SF-36). The WHO Bleeding Scale is widely used in ITP and mentioned in at least one product label for ITP treatments (eltrombopag). The FACIT-F is the most widely-used fatigue instrument and was utilized in non-ITP related regulatory labels in the US and EU. It has also been considered by HTA agencies for ITP and its psychometric properties were validated in ITP. The ITP-PAQ was developed and validated in ITP for HRQoL evaluation; it was used for clinical development of romiplostim and was well considered by the National Institute for Health and Care Excellence (NICE). Conclusion The studies identified in these literature searches indicate that fatigue and bleeding are key determinants of HRQoL in patients with ITP. Three COA instruments (WHO Bleeding scale, FACIT-F and ITP-PAQ) were identified using evidence from the existing literature as potential valid tools to measure these concepts of interest in future ITP clinical trials. Disclosures Joly: Sanofi: Current Employment, Current equity holder in publicly-traded company. Reaney:IQVIA: Current Employment; Sanofi: Research Funding. Daak:Sanofi: Current Employment, Current equity holder in publicly-traded company. Venerus:Sanofi: Research Funding; IQVIA: Current Employment. Roborel de Climens:Sanofi: Current Employment, Current equity holder in publicly-traded company.

Objectives: The Atopic Dermatitis Control Tool (ADCT) was designed to evaluate patient-perceived AD control and facilitate patient–physician discussion on long-term disease control. Methods: The study was performed in adult patients with AD. Development of the ADCT followed US Food and Drug Administration (FDA) guidelines on patient-reported outcome measures (PROMs). Qualitative research, including targeted literature review, interviews with clinical experts, and combined concept elicitation/cognitive debriefing with patients with AD, was conducted to provide a list of comprehensive concepts capturing AD control per physician and patient perspectives. Quantitative methods assessed psychometric properties of the instrument and defined the threshold for AD control. Results: The resulting pilot six-item ADCT, reflecting key concepts related to AD control, had 7-day recall and assessed symptoms and impacts on patients’ everyday lives by severity and/or frequency. The ADCT showed good content validity (well understood by adult patients with AD), and quick completion time (r ≥ 0.80) inter-item correlations for the six ADCT items, robust construct validity (r > 0.50), and item-level discriminating ability (p 0.80). A score ≥7 points was identified as an optimum threshold to identify patients whose AD is “not in control.” Conclusions: No single validated instrument has been available to holistically evaluate patient-perceived AD control. The newly developed ADCT displays good-to-excellent content validity, construct validity, internal consistency, reliability, and discriminating ability.

Background Evolving regulatory guidelines recommend routine assessment of the acceptability of pediatric oral medicines throughout clinical development processes. However, such assessment is problematic owing to a lack of standard methods or criteria that define acceptability for children and their caregivers. This research aimed to identify the attributes of acceptability for targeted oral formulation types that are important to children, and to develop content-valid patient- and caregiver-reported outcome acceptability measures for use in the context of clinical drug development. Methods A concept-focused literature review and two advisory panel meetings involving researchers, clinicians, and measurement scientists were conducted to identify acceptability attributes that may be relevant to children taking targeted oral medicine formulations. The Pediatric Oral Medicines Acceptability Questionnaires (P-OMAQs), including patient (P-OMAQ-P) and caregiver (P-OMAQ-C) versions, were drafted to assess these attributes. Qualitative concept elicitation (CE) and cognitive debriefing (CD) patient and caregiver interviews were conducted to confirm key acceptability attribute concepts for measurement and to evaluate patient and caregiver ability to understand and respond to the questions. Results A full-text review of 40 articles identified 24 acceptability attributes that were categorized into 10 overarching domains and organized into a preliminary conceptual model. Feedback from the advisory panel refined the preliminary model. In total, 14 attributes were reported during the CE phase of the interviews (n = 23 pediatric patients, n = 13 caregivers); six attributes were included in the final model. The draft P-OMAQ was refined over four waves of CD interviews (n = 31 pediatric patients, n = 48 caregivers). The final version of the P-OMAQ-P is a 12-item questionnaire designed for young people aged 8–17 years. The P-OMAQ-C is a 19-item questionnaire designed for adult caregivers of young people aged 6 months to 17 years. There are two versions of each questionnaire: one with a 24-h recall period and one with a 7-day recall period. All items are answered on a 5-point numerical rating scale. Conclusions This research supports the content validity of the patient and caregiver versions of the P-OMAQ. Both questionnaires appropriately assess the acceptability of oral medicine formulations from the perspective of pediatric patients and their caregivers.

Purpose The purpose of this study was to conduct qualitative participant interviews to provide context to the meaningfulness of improvements in end points seen in 2 large-scale Phase III sotagliflozin trials in participants with type 1 diabetes. Methods Participants were eligible for an interview if they had exited one of the clinical trials within the previous 12 months. Participants were recruited by investigators at the clinical trial sites, and interviews were conducted by independent interviewers by telephone in accordance with a semistructured interview guide. Both interviewers and participants were blinded to treatment assignment. Qualitative analysis was conducted using ATLAS-ti version 7.5, and descriptive statistics were computed and summarized. Findings Across 3 countries, 41 participants were interviewed. Difficulty maintaining blood glucose within a desired range, described by participants as lack of blood glucose “stability,” was the most concerning symptom that they reported, wanting to see it improved during the clinical trial because it negatively impacted their physical, mental, and emotional lives. Participants who reported symptom improvement also reported a positive psychosocial impact while taking the clinical trial medication. All participants who monitored ketones described themselves as being “pretty confident” to “very confident” that they could avoid diabetic ketoacidosis by monitoring both ketone levels and understanding the physical signs and symptoms of hyperglycemia. Implications Improvements in glucose stability and control were important to participants with type 1 diabetes, as these improvements were correlated with improvements in the participants' lives. ClinicalTrials.gov identifiers: NCT02384941; NCT02421510.

9566 Background: Cemiplimab-rwlc is the first immunotherapy to receive approval in the US, fully for pts with laBCC and accelerated for metastatic BCC, post hedgehog inhibitors or for whom hedgehog inhibitors are not appropriate. Cemiplimab resulted in clinically meaningful anti-tumor activity in pts with laBCC who progressed on or were intolerant to hedgehog inhibitor therapy (NCT03132636). This analysis evaluated HRQoL in these pts. Methods: Adults with laBCC and ECOG performance status ≤1 (n=84) received IV cemiplimab 350 mg Q3W for up to 9 treatment cycles. At baseline (BL) and day 1 of each cycle (C), pts completed EORTC QLQ-C30 and SKINDEX-16 questionnaires that assess Global Health Status (GHS)/QoL, functioning, and BCC-related symptoms. Mixed-effects repeated measures (MMRM) models were used to estimate least squares (LS) mean (standard error [SE]) change from BL during treatment (i.e., across C2 to C9); changes ≥|10| points were considered clinically meaningful. Responder analyses were conducted in pts with non-missing data from BL to determine the proportions with clinically meaningful improvement or deterioration, or stability on QLQ-C30 and SKINDEX-16 at C2 and C9; a 10-point threshold was considered meaningful for both instruments. Results: BL scores showed moderate to high levels of functioning and low symptom burden. In MMRM models, overall changes from BL on QLQ-C30 indicated stability for GHS/QoL and all scales except for clinically meaningful worsening of fatigue (LS mean [SE] change 12.5 [3.9]; P

9078 Background: Cemiplimab, a PD-1 inhibitor, improved survival and progression-free survival vs platinum doublet chemotherapy (chemo) in patients (pts) with advanced NSCLC and PD-ligand(L)1 expression ≥50% in the EMPOWER-Lung 1 Phase 3 study (NCT03088540). Since pts with advanced NSCLC have a high symptom burden that adversely impacts QoL and functioning, these outcomes were evaluated as secondary endpoints in the clinical trial. Methods: Pts with advanced NSCLC with PD-L1 expression ≥50% and ECOG performance status ≤1 were randomized to IV cemiplimab 350 mg Q3W (n=356) or platinum doublet chemo (n=354). At baseline (BL) and day 1 of each treatment cycle (C) to C15, pts were administered the EORTC core questionnaire (QLQ-C30) and its lung cancer specific module (QLQ-LC13) to assess symptoms, functioning, and Global Health Status (GHS)/QoL. In the intent-to-treat population, mixed-effects repeated measures models were used to estimate least squares (LS) mean change from BL on all scales. Kaplan–Meier analysis estimated time to definitive deterioration, defined as worsening ≥10 points from BL observed at all subsequent time points or patient withdrawal after worsening; hazard ratios (HR) with 95% CIs estimated the likelihood of definitive deterioration. Results: BL scores showed moderate to high levels of functioning and low symptom burden. Cemiplimab-treated pts had lower likelihood of definitive deterioration vs chemo on key symptoms of dyspnea, cough, pain in chest, pain in other body parts, fatigue, nausea/vomiting, appetite loss, constipation, and diarrhea vs chemo (all P

The single-item Psoriasis Itch VAS was developed to measure itch intensity within the last 24 hours in psoriasis vulgaris to assess treatment benefit. Its psychometric properties were explored in two trials.Data from two randomized, parallel-group phase 3 trials with subjects suffering from psoriasis vulgaris on the body (n = 426, 463) were analyzed. Cross-sectional distributional properties and construct validity of the Psoriasis Itch VAS as well as longitudinal test-retest reliability and sensitivity to change of the Psoriasis Itch VAS were investigated. All statistical tests were two-tailed.Across both trials, acceptable distributional properties were observed. Convergent-validity correlations between the Psoriasis Itch VAS and other patient-reported and clinician-reported outcomes provided strong endorsement for construct validity as did tests of known-groups validity. Longitudinal measurement properties, involving test-retest reliability and sensitivity to change, also offered evidence for the measurement integrity of the Psoriasis Itch VAS.Results from the assessment of validity, reliability, and sensitivity to change support the use of the Psoriasis Itch VAS to measure itch intensity in psoriasis vulgaris. Data from two trials provided evidence that the Psoriasis Itch VAS is well-defined and reliable for measuring itch in psoriasis vulgaris to assess treatment benefit (i.e. therapeutic response).

Aims Many treatment options are available for people with Type 2 Diabetes (T2D). While the goal of treatment is to reach target HbA1c levels, the psychological experience may be more tangible and meaningful for the patient. Together with biomarkers, Patient-Reported Outcome (PRO) data provide a holistic understanding of the clinical and psychosocial impact of T2D and its treatment. Methods A literature review was conducted in multiple databases to identify PRO endpoints used in Phase 3 trials of newer classes of drugs for the treatment of T2D. Results This review identified five PRO concepts which were evaluated as endpoints in Phase 3 trials of GLP-1 receptor agonists, novel insulins, SGLT-2 inhibitors, and DPP-4 inhibitors; symptoms, health-related quality of life, psychological well-being, satisfaction with treatment/health and impact of weight. Twenty PRO questionnaires were used to measure these concepts/endpoints directly from patients. The relevance of, and scientific basis for, these concepts as endpoints are discussed, the similarities and differences between questionnaires targeting the same concepts/endpoints are explored, and the sensitivity of the questionnaires to the experimental intervention is summarized. In addition, factors that should be considered when choosing PROs for future T2D trials are discussed. Conclusions The information gained from PROs in clinical trial research is important in defining treatment benefit within the context of the trial, and the potential benefit (i.e. better adherence) in clinical practice. However, variable results have been observed in recent trials and careful and systematic consideration should be given to PRO selection for future studies of T2D.

From humanistic, clinical, and economic perspectives, it is important to understand patients’ health care attitudes and behavior. Of particular interest in defining the value of a pharmacologic therapy is medication adherence. A DIA workstream was convened to define medication adherence in a drug-development context, explore the relevance of medication nonadherence from various stakeholder perspectives, examine methods of collecting medication adherence data in, or alongside, drug-development programs, and propose a robust approach to predicting medication adherence in routine clinical practice from data derived in, or alongside, drug-development programs. This article summarizes the workstream findings and guidance as it pertains to these objectives. The proposed approach to predicting medication adherence involves a patient-reported outcome (PRO) questionnaire that contains a series of standardized questions for patient self-completion that asks about likelihood of medication adherence in clinical practice, and that queries about perceived benefits and barriers to adherence on exit from an efficacy trial. This methodology can be used to both gain a better understanding of the experimental medication and compare the experimental treatment to comparator therapy as warranted. This approach may assist regulators and payers in making meaningful treatment comparisons and facilitate manufacturers in developing empirically based patient-support programs. This workstream will now turn to the challenge of developing the PRO questionnaire in line with this proposal.

This paper outlines the creation of an intuitive, personalized evaluation framework for Patient-Reported Outcomes, using the EQ-5D visual analog scale (VAS) as an illustration. A draft framework asked patients to divide and label the EQ-5D-VAS into different levels or categories of health. Comprehension of the framework and patient-defined health level labels, and how they map onto the EQ-5D-VAS, were tested through in-person, semi-structured interviews with individuals self-reporting cardiovascular disease. Interviews were conducted in three waves, with the framework revised between waves. Analyses included 14 participants. Eight participants (57.1%) felt that four levels of health were appropriate and there was general agreement on the labels; Poor, Fair, Good, and Excellent. There was substantial variability in where patients drew lines to indicate the level boundaries; Poor ranged between 0 and 50; Fair 10-75; Good 40-91; Excellent 60-100. In wave 3, all participants demonstrated appropriate comprehension of the framework. The framework was well understood. The wide range of margins and the extent of overlap between the levels provide strong evidence for the relevance of the personalized evaluation framework approach, and specifically a personalized EQ-5D-VAS evaluation framework, to better understand and interpret each individual's response to the item.

BACKGROUND We investigated the rate of severe hypoglycemic events and confounding factors in patients with type-2-diabetes treated with sulfonylurea (SU) at specialized diabetes centers, documented in the German/Austrian DPV-Wiss-database. METHODS Data from 29,485 SU-treated patients were analyzed (median[IQR] age 70.8[62.2-77.8]yrs, diabetes-duration 8.2[4.3-12.8]yrs). The primary objective was to estimate the event-rate of severe hypoglycemia (requiring external help, causing unconsciousness/coma/convulsion and/or emergency.hospitalization). Secondary objectives included exploration of confounding risk-factors through group-comparison and Poisson-regression. RESULTS Severe hypoglycemic events were reported in 826(2.8%) of all patients during their most recent year of SU-treatment. Of these, n = 531(1.8%) had coma, n = 501(1.7%) were hospitalized at least once. The adjusted event-rate of severe hypoglycemia [95%CI] was 3.9[3.7-4.2] events/100 patient-years (coma: 1.9[1.8-2.1]; hospitalization: 1.6[1.5-1.8]). Adjusted event-rates by diabetes-treatment were 6.7 (SU + insulin), 4.9 (SU + insulin + other OAD), 3.1 (SU + other OAD), and 3.8 (SU only). Patients with ≥1 severe event were older (p 60 mL/min: 3.9). CONCLUSIONS These real-life data showed a rate of severe hypoglycemia of 3.9/100 patient-years in SU-treated patients from specialized diabetes centers. Higher risk was associated with known risk-factors including lack of diabetes-education, older age, and decreased eGFR, but also with lower BMI and lower triglyceride-levels, suggesting that SU-treatment in those patients should be considered with caution. This article is protected by copyright. All rights reserved.

For a new intervention to achieve commercial success, regulators (to provide marketing authorisation), payers (for reimbursement and formulary placement), healthcare professionals (HCPs; to prescribe) and patients (to adhere and persist) must all consider the intervention to be valuable. These stakeholders are increasingly using the patient’s perspective on a condition and its treatment—measured through patient-reported outcome (PRO) instruments—to define the value of a treatment. However, there is common confusion about the most appropriate way to incorporate the patient’s perspective into the clinical development of pharmaceutical interventions in a way that will resonate with these four key stakeholder groups. This article briefly summarises the perspectives of regulators, payers, HCPs and patients regarding PRO endpoints, and examines how a robust, comprehensive and systematic PRO endpoint strategy can be developed to meet the needs of all stakeholders in a single development programme. Such an endpoint strategy may include PRO assessments of first-order signs and symptoms using a disease-specific measure, health-related quality of life using both a disease-specific measure and generic utility measure, and general quality of life. Other PRO endpoints (e.g. preference/satisfaction, ease of use, HCP contact time, absenteeism) may be useful with some stakeholders to provide further differentiation between interventions.

Background and aim This paper presents results from the Spanish subpopulation of a study comparing Conversation Maps™ (CM)-based education with regular care (RC) in type 2 diabetes mellitus (T2DM). Patients and methods Adult patients with T2DM who were considered as not demonstrating ideal disease management were randomly assigned to CM or RC with assessments following (Visit 2), and at follow-up 6 months after (Visit 3), the final CM session. The primary endpoint was diabetes knowledge at Visit 3. Results Three hundred and ten patients were randomized to receive CM education (n=148) or RC (n=162). Median knowledge scores were ranked significantly higher in the CM group than the RC group at Visit 2 and Visit 3 ( p p p =0.055, Visit 3) and perception of goal attainment ( p p =0.046, respectively) that were both higher in the CM group. Conclusions In these patients from Spain, CM was superior to RC in terms of diabetes knowledge 6 months after education was completed, suggesting that CM should be considered for use in patients requiring diabetes education.

Resumen Antecedentes y objetivo Este articulo presenta los resultados de la subpoblacion espanola de un estudio que compara una formacion usando Mapas de Conversaciones™ (MC) con la atencion habitual (AH) en pacientes con diabetes mellitus tipo 2. Pacientes y metodos A pacientes adultos con diabetes mellitus tipo 2 que se considero que no tenian un manejo ideal de su enfermedad se les asigno aleatoriamente a recibir MC o AH, realizando una evaluacion inmediatamente despues (visita 2) y otra a los 6 meses (visita 3) de la sesion final de los MC. La variable principal de valoracion fue el conocimiento adquirido sobre la diabetes en la visita 3. Resultados Participaron 310 pacientes a los que se asigno aleatoriamente a recibir una formacion con MC (n = 148) o AH (n = 162). La mediana de la puntuacion de conocimiento fue significativamente mas elevada en el grupo MC que en el grupo AH tanto en la visita 2 como en la visita 3. No se identificaron diferencias significativas en las variables clinicas o de otra indole entre intervenciones, excepto en la satisfaccion con el cuidado (visita 2, p Conclusiones En estos pacientes espanoles, los MC fueron superiores a la AH en terminos del conocimiento sobre la diabetes 6 meses despues de completar la formacion, por lo que los MC podrian ser una herramienta a considerar en pacientes que requieran una educacion diabetologica.

To understand the true impact of treatment strategies in diabetes, it is important to look beyond just biomedical efficacy. The primary goal of diabetes treatment is to achieve near-normal blood glucose levels over the long term.1 However, achieving good metabolic control requires full understanding, engagement, and commitment of the patient to the prescribed treatment strategy. Patients' experience can be adversely affected because of the direct effects of diabetes or its treatment, which can, in turn, affect clinical outcomes through such mediating factors as medication-taking behavior and adherence to lifestyle changes. Thus, treatment strategies for diabetes must seek to balance glycemic control against negative patient experiences, such as hypoglycemia symptoms and other effects that may adversely affect overall quality of life (QoL).2 Although treatment efficacy is measured with biomarkers, examining patients' experiences requires health care providers to ask them directly about those experiences.3 Patients' experiences can be measured in a quantifiable and standardized manner in clinical research or practice by the administration and completion of patient-reported outcome measures (PROs), which encompass measurement of any aspect of a patient's health status that comes directly from the patient and is based on the patient's perception of a disease and its treatment(s).3 There are numerous PROs measuring a range of concepts, including perceived symptoms of high and low blood glucose, treatment satisfaction, health-related QoL (HRQL), health status, and weight changes. Each concept represents a different aspect of an individual's thoughts and feelings about diabetes or its treatment. PROs may be …

Introduction Although the largest improvement in glycemic control occurs within the first 90 days of insulin therapy, little is known about early persistence on insulin therapy. This research aimed to identify predictors of early discontinuation and of subsequent restart of basal or mixture insulin among patients with type 2 diabetes mellitus (T2DM) and to assess the economic cost associated with such behaviors over a 1-year period. Methods Truven’s Health Analytics Commercial Claims and Encounters database was utilized for the study. Logistic regressions were used to examine factors associated with early discontinuation of insulin (basal or mixture) and, among patients who discontinued early, the factors associated with restarting. Cost regressions were estimated using generalized linear models with a gamma distribution and logistic link. Kaplan–Meier survival curves were used to examine time to discontinuation and time to restart among those who discontinued. Results Multivariate analyses revealed that patient characteristics, prior healthcare resource utilization, comorbid diagnoses, and type of initiated insulin were associated with early discontinuation of insulin and of restarting among patients who discontinued early. Acute care (hospitalization and emergency room) costs were 9.6% higher among patients who discontinued early (P

Adhering to prescribed medication is often a problem for patients with diabetes yet there is no consensus on how best to measure adherence in this patient population. This systematic literature review critically reviewed and summarized the methods used to measure medication adherence in patients with diabetes (on oral hypoglycemic agents [OHAs] and/or insulin) in original research published between 2007-2013.Literature review.A systematic search for methods to assess medication adherence in patients with type I or type II diabetes was conducted using PubMed, EMBASE, PsychInfo, and Cochrane databases. Two researchers independently screened abstracts for initial eligibility and then applied the inclusion/exclusion criteria to the relevant full-text articles.Fifty-nine articles met the criteria for inclusion. Subjective assessment (observer-reported and patient-reported), pill counts, Medication Event Monitoring System (MEMS), cell-phone real-time assessment, and logbooks were used in prospective studies. In pharmacy claims databases, medication possession ratios (MPRs), or some derivation thereof, were utilized. Each method has strengths and weaknesses, but few approaches specifically addressed issues unique to assessing insulin adherence. Three novel approaches (using cell-phone real-time assessment, computerized logbooks, and a questionnaire about different dosing irregularities) provided insight on timing and dosing issues that could be useful for highlighting interventions to improve insulin adherence.No gold standard exists for measuring medication adherence in patients with diabetes. The plethora of adherence methods precludes the comparison of adherence rates across studies. Greater consistency is therefore needed in adherence measurement, including question content, recall period, and response options for self-report measures. Novel methods for understanding adherence to variable-dosed insulin require further research. Researchers should select a methodology that best fits their research question, study design, patient population and resources.

As of the start of 2015, there were 12 classes of drug therapy approved in Europe to treat hyperglycemia in type 2 diabetes, with each class containing multiple medications. Despite this array of medication, adherence and persistence remain poor, with estimates of nonadherence ranging from 7 to 64%, depending on the population and therapy studied (1–3). Poor medication adherence results in the suboptimal clinical benefit of therapy (4), with poor metabolic outcomes, earlier onset and progression of severe microvascular complications, hospitalizations, emergency department visits, and increased mortality associated with lower adherence rates (1,5–7). Nonadherence also poses an immense economic burden on the health care system (6). One reason for poor adherence to and persistence in taking type 2 diabetes medications may be the continued glucocentric model of diabetes care, in which a steadfast (and at times exclusive) pursuit of optimum A1C levels remains at the forefront of prescribers’ minds. However, there is increasing recognition that how patients perceive their condition and associated treatment is an important consideration when making treatment decisions and that achieving optimum A1C levels requires health care professionals (HCPs) and patients to address the clinical and psychosocial aspects of type 2 diabetes together (8,9). Indeed, providing care that is respectful of and responsive to individual patients’ preferences, needs, and values and ensuring that patients’ values guide all clinical decisions, as well as making treatment decisions using evidence-based guidelines that are tailored to individual patients’ …

Objective. This study compared the effectiveness of Conversation Map™ (CM) education tools versus regular care of adults with type 2 diabetes. Methods. Participants from Spain and Germany were randomized to either four CM education sessions or regular care. Data were collected at baseline (visit 1), immediately post-education (visit 2), and 6 months later (visit 3). The primary objective was to compare diabetes knowledge between education groups at visit 3, using the Audit of Diabetes Knowledge (ADKnowl) questionnaire (score range 0–100). Results. Of 681 participants randomized (CM 330, regular care 351), 91.8% completed visit 3. Median ADKnowl scores increased significantly (P < 0.001) in both groups (CM visit 1, 60.3; visit 2, 73.5; and visit 3, 72.5; regular care visit 1, 63.3; visit 2, 73.5; and visit 3, 73.6). At visit 3, scores were not ranked significantly differently between education groups (P = 0.284). In Spain (n = 310), visit 3 scores were ranked higher for CM than for regular care (P < 0.001); in Germany (n = 371), the opposite was true (P < 0.001). Of participants receiving regular care, 78.3% of those in Germany and 13.5% of those in Spain had non-CM structured education between visits 1 and 2. In Spain, CM patients had higher satisfaction with care and goal attainment than regular-care patients at visit 2 (visit 2 P < 0.001, visit 3 P = 0.055). In Germany, CM patients had less emotional distress than regular-care patients (visit 2 P = 0.025, visit 3 P = 0.003). Median A1C decreased similarly in both groups and countries. Conclusion. Diabetes knowledge and clinical outcomes improved in both education groups. At visit 3, diabetes knowledge in the CM group was not ranked higher than in the regular-care group. In Germany, where regular care frequently involves structured education programs, diabetes knowledge was ranked higher with regular care than with CM. In Spain, where no structured national diabetes education and awareness programs are in place, scores were ranked higher with CM than with regular care.

To describe the changes in resource utilization in seven European countries (Germany, Greece, Portugal, Romania, Sweden, Spain, and Turkey) and direct costs in four European countries (Germany, Spain, Sweden, and Greece) over the first 12 months of insulin treatment in patients with type 2 diabetes mellitus (T2DM).INSTIGATE and TREAT (2005-2010) were non-interventional, prospective, observational studies in patients with T2DM and initiating insulin for the first time. A 6-month retrospective data capture was conducted at baseline (insulin initiation) followed by prospective data collections at ∼3, 6, and 12 months. Statistical analyses were descriptive; estimated costs are presented as nominal values.This study presents data for 1450 patients. Overall, in the first 6 months after insulin initiation, the use and cost of blood glucose monitoring and insulin increased, while the cost of oral diabetic medication decreased. Contributors to total direct costs differed between countries. Ranges of total mean direct costs over the 6-month period before insulin initiation were €489.10-€658.50 (Greece-Spain); 0-6 months after insulin initiation, €573.40-€1084.70 (Greece-Spain); and 6-12 months after insulin initiation, €495.80-€859.30 (Greece-Germany). Thus, the mean cost of treatment increased in all countries in the first 6 months after insulin initiation and then returned to baseline except in Germany.Overall, 15% of patients were lost to follow-up over 12 months. Costs were not pro-rated to account for variation of visits. Participating centres may not have been fully representative of all levels of care.Contributors to total cost differed between countries, potentially reflecting local clinical practice patterns and insulin regimens. In each country, mean direct total costs of T2DM care increased during the first 6 months after insulin initiation and decreased thereafter.

Although many patients with type 2 diabetes are initially managed through lifestyle modification, most eventually require insulin therapy. However, insulin initiation is often delayed because of factors such as patients' resistance to insulin therapy and worries about injections. Such delays affect glycemic control, have a direct effect on patient encounters, and may affect medication adherence. Insulin pen delivery systems may address some of these concerns. This study had two phases. First, semi-structured qualitative interviews were conducted to identify the most important features of insulin delivery devices for prandial use from the perspective of patients (n = 8) and health care professionals (HCPs; n = 10). From phase 1, a 26-item questionnaire was developed. In phase 2, patients (n = 33 insulin naive, n = 78 pen users) and HCPs (n = 151) were asked to indicate the most important features to them in insulin delivery devices. Patients then simulated injection using three different pens (SoloSTAR, KwikPen, and FlexPen) and ranked them based on the same features. The most important features were knowing that the entire dose has been injected, ease of reading the dose correctly, and ease of correcting if the dose is over-dialed. In the simulation study, KwikPen and SoloSTAR scored significantly higher (paired t test, P < 0.05) than FlexPen on “knowing if you have injected the entire dose” (mean score out of 10: KwikPen, 8.9; SoloSTAR, 8.6; and FlexPen, 8.4). No other significant differences among the pens were noted in usability or design, and the mean ranking (from 1 to 3) of the pens was similar (KwikPen, 2.0; FlexPen, 2.1; and SoloSTAR, 1.9). By identifying which insulin delivery pens offer these features, HCPs can choose the most appropriate delivery device for patients, which may lead to earlier insulin initiation, greater patient adherence, and better clinical outcomes.

This qualitative interview study was designed to highlight the symptoms and functional limitations experienced by patients in the year following a myocardial infarction (MI). This information can support the use or development of patient-reported outcome (PRO) instruments in the post-MI population. Individual face-to-face interviews were conducted with 38 participants who had experienced an MI (with or without ST segment elevation) within the past month to