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Treatment decisions in older adults with acute myeloid leukemia (AML) are challenging, particularly for those who are not candidates for intensive chemotherapy (IC), and the trade-offs patients, their families and physicians consider when choosing a treatment option are not well understood. This qualitative research explored the value of extending survival and the treatment decision-making process from a multi-stakeholder perspective.Overall, 28 patients with AML (≥ 65 years old, unsuitable for IC), 25 of their relatives and 10 independent physicians from the US, UK and Canada took part in one-on-one, 60-minute qualitative interviews.Across all stakeholders, improved health-related quality of life (HRQoL), extended survival and relief of AML symptoms were recognized as most important in AML treatment decision-making. However, extending survival in 'good health' was more important than extending survival alone, particularly because of the extra time it gives patients and their relatives together, and allows patients to achieve important goals. Patients' limited understanding of available treatment options, paired with incorrect perceptions of treatment side effects, impacted their involvement in the treatment decision-making process. Patients and physicians perceived physicians to have the most influence in the decision-making process despite their priorities not always aligning.These findings illustrate the importance of having structured discussions which explicitly assess patients' goals and their understanding and expectations of treatments and also the need for patient friendly resources about the lived experience of AML and available treatment options. These measures will help to ensure that patients are fully involved in the shared decision-making process.

Background: Caring for an individual with Alzheimer’s disease (AD) is an allencompassing challenge that affects daily life. Assessment of the care partner experience is needed to support the development and evaluation of successful interventions for people with AD and their care partners. We developed the 27-item Zarit Caregiver Interview for Alzheimer’s Disease (ZCI-AD-27) to assess the impact of informal caregiving in the context of AD. Objective: We assessed the psychometric validity of the ZCI-AD-27 in a population of care partners for individuals with moderate AD, and established thresholds for meaningful score change. Methods: Secondary data were obtained from informal care partners of participants in a clinical trial (NCT01677754). Psychometric analyses were conducted to assess validity, reliability, and responsiveness of the ZCI-AD-27. Anchor-based and distribution-based methods were performed to determine clinically meaningful score change. Results: The ZCI-AD-27 had a 12-domain factor structure, including a second-order domain termed Humanistic impact that included four key domains (Physical, Emotional, Social, and Daily life) as confirmed by confirmatory factor analysis with the adequate fit. Internal consistency (Cronbach’s alpha ranging from 0.66 to 0.93 for domains), convergent validity, and discriminant validity indicated the good performance of the ZCI-AD-27. Known-groups validity analyses showed a greater impact on care partners with increasing disease severity. Responsiveness results demonstrated that the ZCI-AD- 27 is sensitive to change over time and meaningful change analyses indicated a range of meaningful score changes in this population. Conclusion: The ZCI-AD-27 is a comprehensive, psychometrically valid measure to assess the impact of caring for individuals with moderate AD.

Score reproducibility is an important measurement property of fit-for-purpose patient-reported outcome (PRO) measures. It is commonly assessed via test-retest reliability, and best evaluated with a stable participant sample, which can be challenging to identify in diseases with highly variable symptoms. To provide empirical evidence comparing the retrospective (patient global impression of change [PGIC]) and current state (patient global impression of severity [PGIS]) approaches to identifying a stable subgroup for test-retest analyses, 3 PRO Consortium working groups collected data using both items as anchor measures.The PGIS was completed on Day 1 and Day 8 + 3 for the depression and non-small cell lung cancer (NSCLC) studies, and daily for the asthma study and compared between Day 3 and 10. The PGIC was completed on the final day in each study. Scores were compared using an intraclass correlation coefficient (ICC) for participants who reported "no change" between timepoints for each anchor.ICCs using the PGIS "no change" group were higher for depression (0.84 vs. 0.74), nighttime asthma (0.95 vs. 0.53) and daytime asthma (0.86 vs. 0.68) compared to the PGIC "no change" group. ICCs were similar for NSCLC (PGIS: 0.87; PGIC: 0.85).When considering anchor measures to identify a stable subgroup for test-retest reliability analyses, current state anchors perform better than retrospective anchors. Researchers should carefully consider the type of anchor selected, the time period covered, and should ensure anchor content is consistent with the target measure concept, as well as inclusion of both current and retrospective anchor measures.

ObjectiveSLE and lupus nephritis (LN) have significant impacts on the health-related quality of life of patients living with the condition, which are important to capture from the patient’s perspective using patient-reported outcomes (PROs). The objectives of this study were to evaluate the content validity of PROs commonly used in SLE and LN (36-Item Short Form Health Survey (SF-36), Functional Assessment of Chronic Illness Therapy–Fatigue (FACIT-F) and Lupus Quality of Life (LupusQoL), as well as novel PRO symptom severity items measuring skin rash, joint pain, joint stiffness and swelling of the legs and/or feet, in both populations.MethodsQualitative, semi-structured, cognitive interviews were conducted with 48 participants (SLE=28, LN=20). Understanding and relevance of symptom and impact PRO concepts from existing PROs were assessed, alongside novel PRO symptom severity items with different recall periods (24 hours vs 7 days) and response scales (Numerical Rating Scale (NRS) vs Verbal Rating Scale). Interviews were conducted in multiple rounds to allow for modifications to the novel PRO items. Analysis of verbatim interview transcripts was performed.ResultsSymptom and impact concepts assessed by the SF-36, FACIT-F, and LupusQoL were well understood by both participants with SLE and LN (≥90.0%), with most considered relevant by over half of the participants asked (≥51.9%). All participants asked (100%) understood the novel PRO symptom severity items, and the majority (≥90.0%) considered the symptoms relevant. Minor modifications to the novel PRO items were made between rounds to improve clarity based on participant feedback. The selected 7-day recall period and NRS in the final iteration of the PRO items were understood and relevant. No differences in interview findings between the SLE and LN samples were identified.ConclusionsFindings provide evidence of content validity for concepts assessed by the SF-36, FACIT-F, LupusQoL and the novel PRO symptom severity items, supporting use of these PROs to comprehensively assess disease impact in future SLE and LN clinical trials.

Background The lower-risk (low and intermediate-1 risk based on IPSS) myelodysplastic syndrome (MDS) has a negative impact on patients’ health-related quality of life (HRQoL). Patient Reported Outcomes (PROs) instruments, which are used to collect patients’ HRQoL data, should have established content validity in the target population to ensure that the instrument is comprehensive and comprehensible. The present study was conducted to evaluate the content validity of the Quality of Life in Myelodysplasia Scale (QUALMS) and the Functional Assessment of Cancer Therapy-Anemia (FACT-An) PRO instruments in patients with lower-risk MDS. Methods In this cross-sectional, qualitative study, 16 patients aged ≥18 years with lower-risk MDS, who were RBC transfusion dependent, literate and fluent in US-English were interviewed. Interviews were semi-structured comprising of two parts: concept elicitation (CE) explored symptoms and impacts important to patients, and cognitive debriefing (CD) assessed understanding and relevance of the QUALMS and FACT-An. A conceptual model was developed, which was used to map the concepts that emerged during CE onto the QUALMS and FACT-An to assess concept coverage and suitability of the instruments. Results The median age of participants was 67.5 years (range: 51–91), with half being female (n = 8). Nine (56.2%) participants had intermediate-1-risk MDS and 10 (62.5%) were relapsed or refractory to erythropoiesis-stimulating agent treatment. Fatigue/tiredness (100.0%), shortness of breath (87.5%), weakness (81.2%), and low energy (75.0%) were reported most commonly and were the most bothersome symptoms as well. Of seven high-level HRQoL domains identified, activities of daily living (n = 16, 100.0%), physical functioning (n = 15, 93.8%), emotional wellbeing (n = 13, 81.3%), social functioning (n = 12, 75.0%), sleep disturbance (n = 9, 56.3%), and impact on work (n = 9, 56.3%) were the most commonly reported. For CD, the QUALMS and FACT-An were found to be mostly relevant and very well understood; response options were easy to use, and recall period was appropriate. Conclusion Both QUALMS and FACT-An demonstrated a strong face and content validity in patients with lower-risk MDS, suggesting that these instruments are appropriate for assessing HRQoL in this population.

Background Endometriosis is a common, chronic, impactful condition in women of reproductive age. In the absence of established sensitive and specific biomarkers, disease severity is determined by patient-reported symptoms and impacts. This article details the development of two new patient-reported outcome (PRO) measures designed to assess efficacy endpoints in clinical studies: The Endometriosis Symptom Diary (ESD) and the Endometriosis Impact Scale (EIS). Methods The ESD and EIS were developed according to best practice and scientific standards (including the Food and Drug Administration (FDA) PRO Guidance) and with extensive input from women with surgically-confirmed endometriosis. Research included: a review of published qualitative literature; concept elicitation interviews in the US, Germany and France (n = 45) to explore the experiences of women with endometriosis and to inform ESD and EIS development; and cognitive interviews in the US and Germany (n = 31) to assess relevance and understanding of the ESD and EIS and usability of administration using an electronic handheld device. The FDA and the European Medicines Agency (EMA) as well as PRO and clinical experts were consulted throughout the process. Results Pelvic pain was identified as the most frequent, severe and bothersome symptom for women with endometriosis. Pain was reported to be greatest during menstruation (dysmenorrhea) and during or after sexual intercourse (dyspareunia). Pain resulted in significant impairments in physical activities, work/study, social/leisure activities, household activities and sexual functioning. All women highlighted the emotional impact of endometriosis. Descriptions of pain and associated impacts were largely consistent across participants from the US and Europe, with the most notable differences being the words used to describe the location of pain (e.g., ‘pelvis’ vs. ‘abdomen’). Testing during cognitive interviews indicated that the ESD and EIS were well understood and consistently interpreted. Furthermore, all participants found the ePRO devices easy to use and no issues regarding visual presentation, selection of responses or navigation were identified. Conclusions Evidence from extensive qualitative research supports the content validity of the ESD and EIS as patient-reported measures of the disease-defining symptoms of endometriosis and the associated impact on women’s lives. Future research will seek to establish the measurement properties of the measures.

BACKGROUND: The Asthma Daytime Symptom Diary (ADSD) and the Asthma Nighttime Symptom Diary (ANSD) were developed to meet the need for standardized patient-reported measures of asthma symptoms to assess treatment trial outcomes in adults and adolescents. OBJECTIVE: To determine scoring and evaluate the measurement properties of the ADSD/ANSD. METHODS: Adolescents (12-17 years) and adults (18 + years) with asthma completed draft 8-item electronic versions of the ADSD/ANSD for 10 days alongside the Adult Asthma Symptom Daily Scales (AASDS) and a Patient Global Impression of Severity (PGIS). Using classical and modern psychometric methods, initial analyses evaluated the performance of ADSD/ ANSD items to inform scoring. Subsequent analyses evaluated the reliability and validity of ADSD/ANSD scores. RESULTS: A demographically and clinically diverse sample (n = 130 adolescents; n = 89 adults) was recruited. Item performance was generally strong. However, items assessing chest pressure and mucus/phlegm demonstrated redundancy and poorer performance and were removed. Principal-components analysis, confirmatory factor analysis, and item response theory supported combining items to form 6-item total ADSD/ANSD scores. Internal consistency (alpha = 0.94-0.95) and test-retest reliability (intraclass correlation coefficient = 0.86-0.95) were strong. Strong correlations (r = 0.72-0.80) were observed between ADSD scores and AASDS items assessing asthma symptom frequency, bother, and impact on activities. Significant differences (P < .001) in mean ADSD/ANSD scores were observed between groups categorized by asthma severity (PGIS), asthma control, inhaler use, nebulizer use, activity limitations, and nighttime awakenings. CONCLUSIONS: The ADSD/ANSD items and scores demonstrated strong reliability and validity. Implementation of the measures in interventional studies will enable the evaluation of responsiveness and meaningful within-patient change. (C) 2021 The Authors. Published by Elsevier Inc. on behalf of the American Academy of Allergy, Asthma & Immunology.

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.

Expectations relating to treatment and survival, and factors influencing treatment decisions are not well understood in adult patients with acute myeloid leukemia. This study analyzed combined findings from a targeted literature review with patient-reported information shared on YouTube to further understand patient perspectives in hematologic cancers and, in particular, acute myeloid leukemia. The targeted literature review included articles concerning patient (aged ≥ 18 years) experiences or perspectives in acute myeloid leukemia or other hematologic cancers. YouTube video selection criteria included patients (aged ≥ 60 years) with self-reported acute myeloid leukemia. In total, 26 articles (13 acute myeloid leukemia–specific and 14 other hematologic cancers, with one relevant to both populations) and 28 videos pertaining to ten unique patients/caregivers were identified. Key concepts reported by patients included the perceived value of survival for achieving personal and/or life milestones, the emotional/psychological distress of their diagnosis, and the uncertainties about life expectancy/prognosis. Effective therapies that could potentially delay progression and extend life were of great importance to patients; however, these were considered in terms of quality-of-life impact and disruption to daily life. Many patients expressed concerns regarding the lack of treatment options, the possibility of side effects, and how their diagnosis and treatment would affect relationships, daily lives, and ability to complete certain tasks. Both data sources yielded valuable and rich information on the patient experience and perceptions of hematologic cancers, in particular for acute myeloid leukemia, and its treatments. Further understanding of these insights could aid discussions between clinicians, patients, and their caregivers regarding treatment decisions, highlight outcomes of importance to patients in clinical studies, and ultimately, inform patient-focused drug development and evaluation. Electronic supplementary material The online version of this article (10.1007/s40271-019-00384-9) contains supplementary material, which is available to authorized users.

Background The Endometriosis Symptom Diary (ESD) and Endometriosis Impact Scale (EIS) are patient-reported outcome measures developed to evaluate efficacy in clinical trials and clinical practice. The ESD is a daily electronic diary assessing symptom severity; the EIS is a weekly electronic diary assessing symptom impact. This study explored the importance of symptoms (ESD items) and impacts (EIS domains), perspectives on scoring algorithms, and clinically important difference (CID) thresholds to inform clinical trial score interpretation. Methods Endometriosis patients in Germany (n = 8) and the US (n = 17), and expert clinicians (n = 4) in Germany, the US, Spain, and Finland participated in semi-structured qualitative interviews comprising structured tasks. Interview transcripts were analyzed using thematic analysis techniques. Results Quality and severity of endometriosis-associated pelvic pain varied considerably among patients; some experienced pelvic pain daily, others during menstrual bleeding (dysmenorrhea) only. Patients and clinicians ranked “worst pelvic pain” as the most meaningful pain concept assessed by the ESD, followed by constant and short-term pelvic pain. Preferences for summarizing daily pain scores over the 28-day menstrual cycle depended on individuals’ experience of pain: patients experiencing pain daily preferred scores summarizing data for all 28 days; patients primarily experiencing pain during selected days, and their treating clinicians preferred scores based on the most severe pain days. Initial CID exploration for the “worst pelvic pain” 0–10 numerical rating scale (0–10 NRS) revealed that, for most patients, a 2- or 3-point reduction was considered meaningful, depending on baseline severity. Patients and clinicians ranked “emotional well-being” and “limitations in physical activities” as the most important EIS domains. Conclusions This study informs the use of the ESD and EIS as clinically relevant measures of endometriosis symptoms and their impact. Findings from the ESD highlight the importance of individual-patient assessment of pain experience and identify “worst pelvic pain” as the most meaningful symptom assessed. Aggregating scores over the 28-day menstrual cycle may inform meaningful endpoints for clinical trials. Diverse EIS concepts (e.g. impact on emotional well-being and physical activities) are meaningful to patients and clinicians, emphasizing the importance of evaluating the impact on both to comprehensively assess treatment efficacy and decisions. Trial registration Not applicable. Qualitative, non-interventional study; registration not required.

Introduction Most patient-reported outcome (PRO) instruments that measure atopic dermatitis (AD) symptoms do not have sufficient documented evidence of content validity to satisfy regulatory agency guidance for inclusion in product-labelling claims in the USA or Europe. The objective of this study was to develop a PRO instrument in accordance with regulatory agency guidance to assess daily AD symptoms during the course of therapy and to establish its content validity and psychometric properties. Methods The Pruritus and Symptoms Assessment for Atopic Dermatitis (PSAAD) daily diary was developed based on qualitative interviews with US adolescents and adults with mild-to-severe AD. Content validity, test–retest reliability, internal consistency reliability, clinically important difference, clinically important responder, convergent validity, and known-group validity were evaluated using correlational and regression methods from phase 2b data from US adults with moderate-to-severe AD who were treated with abrocitinib. Results Patient interviews conducted with US adolescents and adults with mild-to-severe AD identified 11 relevant symptoms (itch, dryness, redness, flaking, discolouration, pain, bleeding, cracking, bumps, swelling, and weeping/oozing) for inclusion in the PSAAD instrument. All PSAAD psychometric parameters were acceptable based on phase 2b data from US adults with moderate-to-severe AD. Convergent validity and known-group validity were confirmed by significant correlations between PSAAD and six other PRO measures (r = 0.24–0.91, all p ≤ 0.01) and Dermatology Life Quality Index category (p ≤ 0.0001), respectively. Conclusions Evidence supports the PSAAD instrument validity, reliability, responsiveness and definitions of clinically important changes/differences for adults with moderate-to-severe AD. Supplementary Information The online version contains supplementary material available at 10.1007/s13555-020-00474-9.

Objectives The Study Participant Feedback Questionnaire (SPFQ) is a patient-completed tool designed to assess patient experiences and satisfaction with aspects associated with being involved in a clinical trial. Originally developed in oncology and among English-speaking participants, the aim of the current study was to evaluate the content and cross-cultural validity of the SPFQ in other indications and non-English-speaking countries. Methods Semi-structured qualitative telephone interviews were conducted with 80 participants across eight non-English-speaking countries (in Europe, South America and Asia) who had received an investigational medicinal product as part of a clinical trial in the past three years. Interviews comprised concept elicitation to identify concepts of importance to participants’ trial experiences, and cognitive debriefing to assess understanding and perceived importance of SPFQ instructions, items and response options. Results Concept elicitation findings supported the content validity of the SPFQ. During cognitive debriefing, SPFQ instructions and the majority of items were well understood by participants. Participants generally considered the SPFQ items important to their clinical trial experience, albeit a handful of items assessed concepts that had not been experienced by trial participants or were redundant with other SPFQ items. The instructions, response options and recall period of the SPFQ were generally well understood. No country-level differences in understanding or importance were apparent. Conclusion Study findings provide evidence for the content and cross-cultural validity of the SPFQ and support implementation of the SPFQ as a means of obtaining participant feedback across global development programmes in a variety of indications.

7016 Background: AML patients may be treated with intensive chemotherapy (IC), or non-intensive chemotherapy (NIC) or they may receive best supportive care (BSC) or hospice care. Balancing treatment efficacy and toxicities is key in treatment decision-making. IC is efficacious with extensive toxicities, while NIC has lower risk of toxicities but reduced efficacy. This study provides an international, multi-stakeholder perspective on the role of side effects in AML treatment decision-making. Methods: We conducted one-on-one, 60-minute interviews with 28 AML patients (>65 years, not receiving IC), 25 of their family members and 10 independent physicians from the US, UK and Canada. Interviews included open-ended questions to explore the treatment decision-making process. Participants also rated the importance of various factors in AML treatment decision-making from 0 (not important) to 3 (very important). Results: The sample included patients with varying treatment histories (13 no treatment, 11 on NIC, 3 discontinued NIC, 1 BSC). Side effects were rated as a ‘very important’ factor in treatment decision-making by a greater proportion of patients not on treatment (n = 9/13; 69.2%) and their relatives (n = 12/13; 92.3%) compared to those with experience of NIC (n = 5/11 who answered, 45.5%), their relatives (n = 3/11; 27.3%), and physicians (n = 4/10; 40.0%). When discussing side effects in detail, there was a disconnect between perceptions of patients not on treatment, and side effects that patients on NIC actually experienced. Many patients with no treatment experience were worried that side effects would be worse than their current symptoms (n = 6/13), referring to constant vomiting, hair loss, organ failure, or death. Fear of side effects was the primary reason for opting not to take treatment (n = 9/13), though it was not clear if these patients were distinguishing between IC and NIC. In contrast, although two patients’ experiences of side effects resulted in them discontinuing NIC (n = 2/14), a higher proportion (n = 9/14) reported that the side effects had little impact on their life. Side effects most frequently reported by patients with experience of NIC (n = 11/14) were considered mild and included fatigue, reduced appetite, generally feeling unwell, nausea and injection site irritation (all n = 3). It was most commonly reported that the worst aspect of NIC was the time commitment (n = 4/8 asked). When accounting for different treatments paths no international variation in findings was observed. Conclusions: The nature and severity of side effects of AML treatment were perceived to be worse than reality. This incorrect perception may lead to undertreatment of patients and result in worse outcomes. There is a need for more patient education and resources about the lived treatment experience, to enhance understanding and mitigate pre-conceived notions of side effects.

Objectives To qualitatively explore patient experiences of severe, recurrent, bilateral nasal polyps (NP). Methods A targeted literature review of published qualitative studies and online blogs describing patient experiences of NP was conducted. Semistructured concept elicitation interviews were conducted in the United States and Germany with participants ≥18 years with severe, recurrent, bilateral NP to explore their symptom experience and impacts on health-related quality of life (HRQoL; NCT03221192 ). A subset of 10 participants reported symptoms and impacts using a smartphone or tablet application (app) over a 10-day period. Results A paucity of qualitative evidence regarding patient experience of NP was identified from the literature or blog review. Twenty-seven participant interviews were conducted. Thirty-six symptoms were identified, including 7 primary symptoms (nasal congestion [n = 27 of 27], breathing difficulties [n = 27 of 27], postnasal drip [n = 25 of 27], runny nose [n = 24 of 27], head/facial pressure [n = 23 of 27], loss of smell [n = 23 of 27], loss of taste [n = 22 of 27]) and 29 secondary symptoms (the most common were mucus/catarrh and nose bleeds [both n = 20 of 27]). Most symptoms were reported to vary both within and between days. Sixty impacts of severe NP were reported, including impacts on sleep (n = 22 of 27), physical functioning (n = 21 of 27), activities of daily living (n = 21 of 27), emotional well-being (n = 27 of 27), treatment (n = 23 of 27), social life (n = 26 of 27), and work (n = 19 of 27). Symptoms/impacts reported using the app were consistent with interview findings, although new symptoms were identified (ear pain, throat pain, nasal scabs, and nasal burning). These results supported the development of a conceptual model outlining concepts related to symptoms, impacts, and treatment of NP. Conclusions Severe, recurrent, bilateral NP are associated with a range of symptoms that have significant detrimental impact on HRQoL.

Introduction Prophylactic treatment of severe hemophilia A is burdensome, requiring frequent intravenous injections. Extended half-life (EHL) factor VIII replacement therapies offer longer intervals between infusions while still meeting efficacy and safety outcomes; however, patient perspectives following long-term use of such products in the real-world remain unknown. Objective We aimed to explore the importance of infusion frequency and the potential benefits of reduced infusion frequency among patients receiving prophylactic treatment with an EHL product (BAY 94-9027). Methods Patients with severe hemophilia A participating in the PROTECT VIII extension study were invited to participate in a semi-structured, concept elicitation ‘exit’ interview to discuss their experiences. Participants were recruited from Israel, The Netherlands, and the US. Interview transcripts were translated into English and analyzed using thematic analysis methods. Results Sixteen participants (29–68 years of age) infusing with BAY 94-9027 once every 7 days, once every 5 days, or twice weekly were interviewed. Participants reported infusion frequency (alongside efficacy) as the most important treatment attribute influencing their satisfaction with therapy. Patient-reported benefits of reduced infusion frequency and longer duration of factor coverage included greater ability to participate in physical activities; better vein health; less time to schedule and administer factor VIII; reduced impact on work; and improved emotional well-being. Conclusions This study provides rich insights into the experiences of patients with EHL products and the value of reduced infusion frequency. Such data could be of value to a range of stakeholders (e.g. regulators, payers) and facilitate patient–clinician discussions to promote tailored treatment decisions.

Background Despite the widespread availability of patient-reported asthma questionnaires, instruments developed in accordance with present regulatory expectations are lacking. To address this gap, the Patient-Reported Outcome (PRO) Consortium's Asthma Working Group has developed a patient-reported asthma daily symptom diary (ADSD) for use in clinical research to assess outcomes and support medical product labeling claims in adults and adolescents with asthma. Objectives To summarize the qualitative research conducted to inform the initial development of the ADSD and to provide evidence for content validity of the instrument in accordance with the Food and Drug Administration's PRO Guidance. Methods Research informing the initial development and confirming the content validity of the ADSD is summarized. This comprised a review of published qualitative research, semi-structured concept elicitation interviews (n = 55), and cognitive interviews (n = 65) with a diverse and representative sample of adults and adolescents with a clinician-confirmed diagnosis of asthma in the United States to understand the asthma symptom experience and to assess the relevance and understanding of the newly developed ADSD. Results From the qualitative literature review and concept elicitation interviews, eight core asthma symptoms emerged. These were broadly categorized as breathing symptoms (difficulty breathing, shortness of breath, and wheezing), chest symptoms (chest tightness, chest pain, and pressure/weight on chest), and cough symptoms (cough and the presence of mucus/phlegm). Conceptual saturation was achieved and differences in the experience of participants according to socio-demographic or clinical characteristics were not observed. Subsequent testing of the ADSD confirmed participant relevance and understanding. Conclusions The ADSD is a new patient-reported asthma symptom diary developed in accordance with the Food and Drug Administration's PRO Guidance. Evidence to date supports the content validity of the instrument. Item performance, reliability, and construct validity will be assessed in future quantitative research.

By bringing data collection closer to real time and minimizing recall bias, patient diaries or event-driven logs offer substantial benefits over retrospective questionnaires for many patient-reported disease concepts. Such assessments are increasingly used to support primary and secondary endpoints in randomized controlled trials. These diaries have the potential to provide more reliable and valid assessment of patients' subjective experiences of symptoms and disease events. However, use of such diaries presents significant and unique challenges depending on the context of use. Of note, while symptom-related label claims are those most frequently granted by regulatory authorities, no guidance specific to support the development, psychometric evaluation, and interpretation of endpoints derived from patient diaries exists. This article provides an overview of key methodological, statistical, and clinical considerations for implementation of patient diaries with a regulatory perspective in mind. Approaches and solutions covered in this article include (1) techniques to establish content validity based on obtaining qualitative insights in naturalistic settings and real-life experience of diary completion, (2) demonstration of psychometric properties with respect to day-to-day variability, and (3) aggregation of data from multiple days/events to move from items to endpoints. The importance of the patients' engagement is highlighted in order to help overcome these challenges throughout all stages of diary and endpoint development and evaluation. This article can inform researchers who are developing or implementing patient diaries as clinical trial endpoints to ensure that the nuances of this mode of data collection are considered in the development of endpoints and prior to regulatory interactions.

Background:Lupus nephritis (LN) is an autoimmune disease characterized by inflammation of the kidneys as a result of systemic lupus erythematosus (SLE). Approximately 50% of SLE patients will develop LN, which is considered to be one of the most severe manifestations of SLE and the leading cause of morbidity and mortality in SLE. While there is ample existing evidence on disease experience and PROs used in extra-renal SLE, little research has been done in LN. Qualitative interviews with patients can help identify concepts that are both important and relevant to the patient. In order to effectively evaluate treatment benefit, it is critical that PRO measures used to assess such concepts and define clinical trial endpoints are fit for purpose and have strong evidence of content validity in the specific context of use.Objectives:The objective of this study was to understand the patient experience of LN and to identify and characterize the signs and symptoms of LN and their impact on health-related quality of life (HRQoL) through the development of a disease-specific conceptual model. This model was then used to evaluate the content validity of existing PRO measures available for use in LN.Methods:A structured literature search was conducted in Medline, Embase and PsycINFO to identify qualitative research articles documenting the patient experience of LN. PRO measures developed or commonly used to assess patient experiences of LN were also identified. Semi-structured concept elicitation interviews were conducted with 15 adult patients in the US with a clinician-confirmed diagnosis of LN (defined in accordance with established clinical guidelines). Supplementary qualitative data were also collected from a review of publicly available online blogs/forums. Findings were used to inform the development of a conceptual model detailing the impact of LN signs, symptoms and HRQoL and evaluate the validity of existing measures used within LN.Results:Searches revealed a paucity of qualitative research conducted with LN patients, supporting the need for prospective research in LN. Consistent with existing literature in SLE, the core signs and symptoms identified from the qualitative literature review, interviews and blog/forum review included joint pain, fatigue, joint stiffness, swelling (particularly in the extremities) and skin rashes. LN patients also reported urinary frequency, urgency, foamy urine and blood in their urine. Disease impact on physical functioning, activities of daily living, emotions, social life, work/finances and sleep were reported. PRO measures commonly used to evaluate patient experiences in LN included the SF-36, LupusQOL, LupusPRO, SLE Symptom Checklist, KDQoL and KSQ. Conceptual mapping of instruments against the newly developed conceptual model (Figure 1) highlighted that no single measure provides a comprehensive assessment of all symptoms/impact important to LN patients. Furthermore, items are largely focused on impact of symptoms with few items on symptom severity.Figure 1.Conceptual model of lupus nephritis symptoms and associated impactsConclusion:The presentation of signs and symptoms in LN patients appears similar to those reported in extra-renal SLE populations, with the addition of swelling and urinary symptoms. Qualitative research with LN patients guided the development of a comprehensive LN conceptual model outlining the disease experience from the patients’ perspective. These insights can be useful to inform PRO measurement strategies for clinical trials in LN.Acknowledgments:With thanks to Dr. Betty Diamond and Dr. David Wofsy for their collaboration and helpful insightsDisclosure of Interests:Pamela Berry Employee of: Janssen, Kate Burrows Consultant of: Adelphi Values a health outcomes research company commissioned by Janssen to conduct the research reported in this abstract, Rebecca Hall Consultant of: Adelphi Values a health outcomes research company commissioned by Janssen to conduct the research reported in this abstract., Adam Gater Consultant of: Adelphi Values a health outcomes research company commissioned by Janssen to conduct the research reported in this abstract, Helena Bradley Consultant of: Adelphi Values a health outcomes research company commissioned by Janssen to conduct the research reported in this abstract, Amy Ward Consultant of: Adelphi Values a health outcomes research company commissioned by Janssen to conduct the research reported in this abstract, Chloe Tolley Consultant of: Adelphi Values a health outcomes research company commissioned by Janssen to conduct the research reported in this abstract, Patricia Delong Employee of: Janssen, Elizabeth C Hsia Shareholder of: Johnson & Johnson, Employee of: Janssen Research & Development, LLC

7520 Background: Current treatment options for acute myeloid leukemia (AML) are diverse, including intensive chemotherapy (IC), low intensity therapy, best supportive care (BSC), and hospice care. Despite continued development of new therapies, recent data suggest that approximately 60% of older US patients remain untreated, but reasons for this are not well understood. By gathering insights from physicians, patients, and their family members, this study aims to better understand the factors that influence treatment decisions for adults with AML. Methods: Physicians in the US (n=4), UK (n=3) and Canada (n=3), and 15 US AML patient-family member dyads took part in one-on-one, 60-minute semi-structured interviews. Each participant rated a series of factors on a scale from 0 (not at all important) to 3 (very important) to determine their importance in treatment decision-making. Among the 15 adults with AML (>65 years, not taking IC) interviewed thus far, 13 had not received any treatment. Additional interviews are scheduled in the UK and Canada with patients having varied treatment experiences (data will be available for presentation). Results: To date, findings highlight the key role perceptions of side effects and patient health play in treatment decision making. A fear of treatment side effects was the primary reason patients (n=9/13) opted not to receive treatment. For the 2/15 study patients who had received treatment, side effects were considered the worst part of their treatment experience. Physicians also stated patients on BSC would be more willing to take low intensity treatments if risks (e.g., side effects) were minimized. Patients (n=11/15), their family members (n=11/15), and physicians (n=10/10) agreed that patients’ health (including age and comorbidities) influenced if treatment was pursued. Additionally, US physicians suggested that some patients have little desire to pursue treatment, with patients’ perception of low intensity therapy having poor efficacy and proximity of care influencing their choice for BSC or hospice care. Further analysis will explore other factors influencing patients’ treatment decisions and differences among patients who receive treatment versus those who do not. Conclusions: The treatment decision-making process for older adults with AML is complex and multifactorial. Understanding factors that influence treatment decisions is important if drug developers and prescribers are to ensure the availability of therapies that better align with individual patients’ needs.

Introduction Recent studies suggest that docetaxel plus androgen deprivation therapy can prolong survival among men with metastatic hormone-sensitive prostate cancer (mHSPC). However, as a cytotoxic therapy, there is a need to understand the experiences of men with mHSPC receiving docetaxel and their carers in a real-world setting. Methods During phase 1, semi-structured qualitative interviews were conducted with men with mHSPC (n = 31) and their carers (n = 14) in Europe to elicit in-depth data concerning their experiences with docetaxel. Eighteen men were also asked to record their experiences in a diary for 7 days. During phase 2, men with mHSPC (n = 161) and carers of men with mHSPC (n = 135) completed an online survey comprising self-report questionnaires including the Cancer Therapy Satisfaction Questionnaire, Brief Fatigue Inventory, Functional Assessment of Cancer Therapy-Prostate, EuroQol-5-Dimensions and the Burden Scale for Family Caregivers (carers only). Results At the outset of therapy, men reported a willingness to take docetaxel to prolong their life, despite being fearful of the potential side effects and impacts on their daily lives. Patient and carer experiences were generally consistent with pre-treatment expectations. However, variations in individual experiences and their ability to tolerate side effects were evident. Fatigue emerged as a prominent symptom with the majority (n = 98, 60.9%) of men reporting experiencing moderate-severe fatigue in the past 24 h. Participant ratings of fatigue were strongly correlated with health-related quality of life (r = − 0.82). Nausea, diarrhoea and sore mouth were also among the most bothersome symptoms for participants. Conclusions Findings from this study highlight that real-world experience of docetaxel may differ from that observed in clinical trials and that care must be taken to ensure that treatment options are tailored to the needs of individual patients to promote not only how long patients survive but also the quality of that survival. Funding Janssen

Introduction: Tyrosine kinase inhibitors (TKIs) represent the standard of care for patients with chronic-phase chronic myeloid leukemia (CP-CML). The emergence and availability of second-generation TKIs (dasatinib, nilotinib and bosutinib) has led to increased choice for patients and physicians, with data from randomized clinical trials indicating improved efficacy of such therapies compared to the first-generation TKI (imatinib). Differences in the efficacy of second-generation TKIs are negligible; however, all have distinct dosing instructions and safety profiles. Understanding the patient perspective is invaluable for optimizing healthcare decisions, with interest in information regarding patient preferences growing among a wide variety of stakeholders, including regulatory agencies, reimbursement bodies and prescribers. However, evidence is lacking regarding treatment preferences and priorities for patients with CP-CML. The aim of this study was to explore attributes of currently-licensed TKI therapies that are relevant and important to patients with CP-CML. This research represents the first step in the conduct of a discrete choice experiment (DCE), or conjoint analysis, to explore treatment preferences more broadly among CP-CML patients. Patient-guided design is crucial to ensuring attributes of investigation are meaningful. Hence the qualitative insights will advise and validate attribute and level selection for inclusion in the forthcoming DCE. Methods: In-depth qualitative interviews were conducted with 12 US-based patients with a clinician-confirmed diagnosis of CP-CML. The sample included 10 females and 2 males (median age 46 years) who were diagnosed 2 years (n=3), 4-7 years (n=5), 10-12 years (n=3) and 16.5 years (n=1) prior to interview. Ten patients had received a second-generation TKI. Current treatments were dasatinib (n=8), bosutinib (n=1) and imatinib (n=3). Previous treatments included imatinib (n=6), nilotinib (n=3) and dasatinib (n=1). The interviews utilized concept elicitation techniques to capture attributes of importance in both TKI treatment experience and TKI treatment selection. Transcripts were thematically analyzed using Atlas.ti. Results: Open-ended questioning elicited an array of attributes related to TKI treatments. It was important to all patients to ensure that a CP-CML treatment was effective. Treatment efficacy was conveyed with multiple descriptors; most commonly "BCR-ABL" (n=7) or major molecular response "MMR", polymerase chain reaction "PCR" and "remission" (each n=3). Eight patients discussed the importance of dose frequency, specifically the burden of scheduling self-administration of oral tablets (n=5). Seven patients conveyed the difficulty of taking a TKI with specific meal requirements, most notably when fasting is required prior to and post-administration (n=4). A multitude of important treatment side effects were elicited at investigation into the patient TKI treatment experience. Fatigue was reported by 11 patients (n=10 spontaneous) and considered by most patients (n=8/11) to be directly attributable to treatment versus the CML itself. Four patients considered fatigue an inevitable component of TKI treatment. Nausea was elicited by 7 patients (n=5 spontaneous) and some patients (n=5/9) considered it to be influential in treatment selection. Muscle pain was reported by 6 patients (n=4 spontaneous), while skin rash was reported by 5 patients and bone pain by 4 patients (all spontaneous). Diarrhea was reported by 4 patients (n=3 spontaneous). Additional symptoms reported by ≤2 patients are presented in Figure 1. Spontaneously elicited concepts were ranked by importance. All patients considered efficacy within the top three most important attributes. Six patients considered fatigue to be either of second or third most-importance. The rankings in Figure 2 serve to portray the varied perspectives and experiences of patients on TKI treatments. Conclusions: The TKI treatment experience for CP-CML patients is multifaceted. This type of study - the first that we know of - supplies valuable insight into the patient perspectives of TKI treatment to establish a foundation to better inform relevant decisions in the field. The qualitative findings will provide the input necessary to inform attribute and level selection to characterize treatment profiles ahead of an upcoming patient-preference DCE. Disclosures Mason: Adelphi Values: Employment; Pfizer: Consultancy. Mamolo:Pfizer: Employment, Equity Ownership. Johnson:Pfizer Inc: Consultancy; Adelphi Values Ltd: Employment. Viqueira:Pfizer Inc: Employment, Equity Ownership. Gater:Pfizer Inc: Consultancy; Adelphi Values Ltd: Employment. Russell-Smith:Pfizer: Employment, Equity Ownership. Tatlock:Adelphi Values: Employment. Shah:Pfizer: Employment, Equity Ownership. Cappelleri:Pfizer: Employment, Equity Ownership.

INTRODUCTION: Acute myeloid leukemia (AML) is a rare hematologic cancer primarily affecting older people, with a median age at diagnosis of 67 years old (Almeida & Ramos, 2016). Heterogeneity in the presentation, functional status and presence of comorbidities among AML patients presents unique challenges for treatment. Intensive chemotherapy (IC), although the best option for prolonging survival, carries a risk of early death and other trade-offs, including significant time spent in the hospital. The toxicity of IC treatment and the requirement for prolonged hospitalizations may negatively affect patients' physical functioning and health-related quality of life (HRQoL). Novel, low-intensity treatments can be administered in the clinic and may pose less risk of immediate toxicities but may be associated with reduced efficacy. Each patient and family will approach these trade-offs differently, yet few studies examine the process of shared decision-making in AML. We aimed to better understand this process by eliciting patient/family and physician narratives about expectations and attitudes towards AML treatments. METHODS: Ten physicians in the US (n=4), UK (n=3) and Canada (n=3) and 12 AML patients (all US) and a member of their family took part in an individual, 60-minute qualitative telephone interview. Further interviews are scheduled and any additional data at the time of presentation will also be reported. The interviews followed a semi-structured guide comprising open-ended questions. The overarching aim of the interviews was to understand the value of living longer for AML patients who are not candidates for standard IC and to explore the treatment decision process from the patient, family and physician perspective. During the interviews, each participant (patient, family member or physician) completed a rating exercise in which they were asked to rate a list of pre-defined factors (9-10 factors) on a scale of 0 (not at all important) to 3 (very important) to determine their importance in AML treatment decisions. As well as providing a numerical rating for each factor, each participant was asked why they selected their rating and which three factors they would consider the most important. RESULTS: Across all three groups, relief in AML symptoms (namely fatigue and pain), longer survival and better QoL were equally considered the three most important factors when making a treatment decision. All three groups described the interaction between QoL and longer survival, explaining that any increase in survival would be important, but only if QoL (time spent with family, maintaining hobbies/interests) was maintained or improved. Physician's advice was also important to AML patients when making decisions regarding treatment. Other treatment-related factors within the rating task were rated as very important for at least a subset of patients, with no factors widely considered to be of limited importance. All participants noted that while relief of AML symptoms, longer survival and quality of life were all important, these had to be considered in light of treatment side effects and risk of infection. Likelihood of being hospitalized was important to family members, as they wanted to spend quality time at home with their loved ones, while patients and physicians considered hospitalization as an inevitable consequence of the disease and treatment. All participants reported that AML patients would consider taking any form of treatment if suitable and effective, regardless of the mode of administration. However, it was acknowledged that oral treatments would be more convenient and less invasive. The relative importance of location of care/treatment delivery was influenced by proximity to resources and whether the participant was based in an urban or rural setting. The ability to receive treatment at home was considered beneficial. CONCLUSIONS: The results from this rating exercise and qualitative interviews showed convergence across all stakeholders, indicating that AML patients, family and physicians have similar priorities regarding treatment decisions, prioritizing symptom relief, survival, and quality of life. The predominant treatment pathways in AML each require trade-offs in these factors, demonstrating the importance of shared decision making in ensuring the most appropriate treatment is selected for a given individual, in accordance with their values, goals, and preferences. Figure Disclosures LeBlanc: Jazz Pharmaceuticals: Research Funding; Daiichi-Sankyo: Membership on an entity's Board of Directors or advisory committees; Otsuka: Consultancy, Membership on an entity's Board of Directors or advisory committees; Heron: Membership on an entity's Board of Directors or advisory committees; Helsinn: Consultancy; Flatiron: Consultancy; NINR/NIH: Research Funding; Duke University: Research Funding; Astra Zeneca: Consultancy, Research Funding; Amgen: Membership on an entity's Board of Directors or advisory committees; Medtronic: Membership on an entity's Board of Directors or advisory committees; Seattle Genetics: Consultancy, Research Funding; Celgene: Honoraria; AbbVie: Membership on an entity's Board of Directors or advisory committees; Pfizer Inc: Consultancy; American Cancer Society: Research Funding; Agios: Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; CareVive: Consultancy. Walter:Seattle Genetics: Research Funding; Agios: Consultancy; Amgen: Consultancy; Amphivena Therapeutics: Consultancy, Equity Ownership; Aptevo Therapeutics: Consultancy, Research Funding; Argenx BVBA: Consultancy; Astellas: Consultancy; BioLineRx: Consultancy; BiVictriX: Consultancy; Boehringer Ingelheim: Consultancy; Boston Biomedical: Consultancy; Covagen: Consultancy; Daiichi Sankyo: Consultancy; Jazz Pharmaceuticals: Consultancy; Kite Pharma: Consultancy; New Link Genetics: Consultancy; Pfizer: Consultancy, Research Funding; Race Oncology: Consultancy. Hernandez-Aldama:Pfizer Inc: Consultancy. Sully:Pfizer Inc: Consultancy; Adelphi Values Ltd: Employment. Bell:Pfizer Inc.: Employment, Equity Ownership. Johnson:Pfizer Inc: Consultancy; Adelphi Values Ltd: Employment. Peloquin:Pfizer Inc: Employment, Equity Ownership. Gater:Pfizer Inc: Consultancy; Adelphi Values Ltd: Employment. Welch:Pfizer Inc: Employment, Equity Ownership. O'Hara:Adelphi Values Ltd: Employment; Pfizer Inc: Consultancy. Russell:Astellas: Consultancy, Honoraria, Speakers Bureau; Pfizer Inc: Consultancy, Honoraria, Speakers Bureau; Jazz: Consultancy, Honoraria, Speakers Bureau; DSI: Consultancy, Honoraria, Speakers Bureau. Horikoshi:Pfizer Inc: Consultancy. Maze:Pfizer Inc: Consultancy; Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees.

Background Asthma-chronic obstructive pulmonary disease overlap (ACO) differs from asthma and chronic obstructive pulmonary disease (COPD) in demographics, phenotypic characteristics and outcomes, yet the patient experience of ACO is poorly characterized. We aimed to understand and compare the patient experience of symptoms and domains of impact in ACO relative to COPD, and assess the content validity of existing patient-reported outcome (PRO) instruments in ACO. Methods This US qualitative, interview study included patients who met American Thoracic Society/European Respiratory Society spirometric criteria for COPD. Additionally, patients with ACO demonstrated reversibility (forced expiratory volume in 1 s [FEV1] increase ≥ 12% and ≥ 200 mL) to albuterol/salbutamol and an FEV1/forced vital capacity ratio

Background: Improved cancer care means that more patients are surviving longer, but there is a need to examine how well patients survive. We conducted an exploratory analysis of a new conceptual framework termed ‘quality of survival’ (QoS) that delineates the quality of patients’ experience. Methods: This project included an electronic database search to investigate the survivorship landscape and to create a visual QoS map and semi-structured interviews with patients (n = 35), clinicians (n = 40), and payers (n = 7) to support the QoS map. QoS was discussed in the context of two tumor types, metastatic non-small cell lung cancer and metastatic melanoma. Results: Despite increased long-term survival, no specific definition of QoS exists. Patients reported many impacts that affect QoS, clinicians viewed QoS as relevant to treatment decisions, and payers felt it could help communicate different aspects relevant to the patient. Four interconnected QoS dimensions were developed (quality of life, survival, side effects, and economic impact), which vary in importance along the care continuum. Conclusion: QoS is a patient-centric concept that could help decision-making and patient communication. The QoS map could provide a framework to monitor patient experience and help patients frame what treatment attribute is most important to them at any point in the cancer continuum.

Objectives. As a disease typified by early onset and chronic disease course, caring for a person with schizophrenia may have a significant impact on caregivers’ lives. This study aimed to investigate the subjective experiences of caregivers of people with schizophrenia as a means of understanding “caregiver burden” in this population.Methods. Face-to-face qualitative interviews were conducted with a diverse sample of 19 US-English speaking caregivers of people with schizophrenia (who were at least moderately ill). Interview transcripts were analyzed using grounded theory methods and findings used to inform the development of a preliminary conceptual model outlining caregivers’ experiences.Results. Findings support assertions that people with schizophrenia were largely dependent upon caregivers for the provision of care and caregivers subsequently reported lacking time for themselves and their other responsibilities (e.g., family and work). Caregiver burden frequently manifested as detriments in physical (e.g., fatigue, sickness) and emotional well-being (e.g., depression and anxiety).Conclusions. Caring for a person with schizophrenia has a significant impact on the lives of informal (unpaid) caregivers and alleviating caregiver burden is critical for managing individual and societal costs. Future research should concentrate on establishing reliable and valid means of assessing burden among caregivers of persons with schizophrenia to inform the development and evaluation of interventions for reducing this burden.

INTRODUCTION: Patient experience data provides an opportunity to explore patient's perspectives on current and potential treatments. Gaining patient narratives on the expectation, tolerance and attitudes towards treatments can enhance clinical management and inform discussions with payers and regulatory agencies. Patient experience data is particularly beneficial in conditions where there is no clear, prescribed treatment pathway as it can contextualize joint treatment decisions made by patients and their treating physicians. Life expectancy among elderly patients with hematogical cancers such as acute myeloid leukemia (AML) is short. Patients with these conditions may be eligible for intensive chemotherapy (IC) or may face a decision between non-intensive chemotherapy (NIC) or palliative care. Further, eligible patients may not wish to undergo the taxing treatment regimens associated with IC. A targeted literature review was conducted to understand the trade-offs associated with treatment decision making and to gain patient perspectives on the value of extended overall survival (OS) in hematological cancers. METHODS: Searches of computerized bibliographic databases including PubMed (Medline), EMBASE and PsycINFO were conducted using the OVID platform. Searches were conducted using a combination of keywords and Medical Subject Headings (MeSH) terms. To be selected for review, articles must have contained keywords in the title and/or abstract and include information regarding patient experience or perspectives. Articles focussed on children ( RESULTS: A total of 26 articles were selected for full text review (12 AML-specific, 13 other hematological cancers and one article with findings relevant to both populations). Most articles selected for inclusion were qualitative research studies (n=23), providing rich detail regarding the patient perspective. AML patients discussed the lack of a clear, defined treatment pathway, unlike other cancers where there is a prescribed course of action. These patients described feeling trapped between two undesirable treatment options (IC versus palliative care) and described this treatment decision as "do or die". Similarly, in other hematological cancers patients reported choosing IC to extend their life, while acknowledging that the treatment itself can be distressing. It appeared that the idea of living longer dominated this trade-off. Some patients expressed regret in choosing IC after experiencing the associated side effects. These findings suggest that patients may not fully understand the treatment options available to them, which highlights the importance of the patient-clinician relationship. Findings from studies also highlighted quality of life as an important consideration for patients in addition to the quantity or length of life. Specifically, patients emphasized their need to maintain a 'normal life' following their diagnosis and to be able to engage in their hobbies and daily activities. There is a paucity of research into important life milestones for AML patients. However, patients and caregivers with other hematological cancers described their desire to be alive for family occasions, such as births or weddings, as well as a sense of appreciation and gratitude for the time remaining with their loved one. This was particularly evident for those patients who were parents of younger children, who felt unprepared for the untimely passing of their kin. CONCLUSIONS: Heterogeneity in the presentation, functional status and presence of comorbidities among patients with AML and other hematological cancers presents unique challenges for treatment. Nevertheless, understanding of aetiology and epidemiology of these conditions continues to evolve. In response, medical product development/evaluation, policy and treatment decisions in clinical practice need to reflect patient perspectives of treatment in terms of expectations, goals, experiences and preferences to ensure selection of the most appropriate treatment for a given individual. Existing evidence provides some insight as to factors that are important to patients and may influence treatment decisions. However, further research to understand patient perspectives, particularly among those who may not be eligible or willing to receive IC, is required. Disclosures Johnson: Adelphi Values Ltd: Consultancy. Bell:Pfizer: Employment, Equity Ownership. Sully:Adelphi Values Ltd: Employment; Amgen: Consultancy. Welch:Pfizer: Employment, Equity Ownership. Peloquin:Pfizer: Employment, Equity Ownership. Gater:Adelphi Values Ltd: Consultancy.

Clinical studies have demonstrated the benefits of abiraterone acetate + prednisone (AAP) and enzalutamide (ENZ) in significantly improving survival among metastatic castration-resistant prostate cancer (mCRPC) patients. However, evidence regarding patient's real-world experience, particularly with respect to fatigue, treatment satisfaction and health-related quality of life (HRQoL) is limited. Interviews were initially conducted with patients (n = 38) and carers (n = 12) to elicit qualitative data regarding their experiences. Findings informed the design of a quantitative, multinational online survey of mCRPC patients (n = 152) receiving AAP or ENZ. Participants completed validated questionnaires assessing fatigue (Brief Fatigue Inventory), treatment satisfaction (Cancer Therapy Satisfaction Questionnaire) and HRQoL (EuroQol-5-Dimensions). Results indicated that patients were generally satisfied with these therapies, more specifically with reductions in prostate-specific antigen levels and extended survival. Fatigue was commonly linked to poor HRQoL and responses indicated that significantly fewer patients in the AAP group reported feeling usually tired or fatigued in the last week compared to the ENZ group (33% vs. 55%, p = 0.006 respectively). Findings highlight the benefit of AAP and ENZ in promoting the "quality" of extended survival. That fatigue was lower among patients receiving AAP may be important for informing treatment decisions. Further research is needed to gain deeper insights.

Ensuring adherence to therapy is a challenge in chronic diseases, particularly in cancers such as chronic myeloid leukemia (CML), where there has been increased availability and use of oral formulations. A conceptual model of adherence was developed based on findings from a comprehensive literature review, to inform strategies for improving adherence to oral CML therapies. A complex interplay of factors (including clinical, psychological and behavioural) influence adherence to such therapies. Healthcare professionals have a key role in promoting and facilitating adherence and future strategies should place greater emphasis on understanding patient-level experiences in order to create personalized solutions.

The primary objective of this review is to develop a conceptual model for Crohn's disease (CD) outlining the disease burden for patients, healthcare systems and wider society, as reported in the scientific literature. A search was conducted using MEDLINE, PsycINFO, EconLit, Health Economic Evaluation Database and Centre for Reviews and Dissemination databases. Patient-reported outcome (PRO) measures widely used in CD were reviewed according to the US FDA PRO Guidance for Industry. The resulting conceptual model highlights the characterization of CD by gastrointestinal disturbances, extra-intestinal and systemic symptoms. These symptoms impact physical functioning, ability to complete daily activities, emotional wellbeing, social functioning, sexual functioning and ability to work. Gaps in conceptual coverage and evidence of reliability and validity for some PRO measures were noted. Review findings also highlight the substantial direct and indirect costs associated with CD. Evidence from the literature confirms the substantial burden of CD to patients and wider society; however, future research is still needed to further understand burden from the perspective of patients and to accurately understand the economic burden of disease. Challenges with existing PRO measures also suggest the need for future research to refine or develop new measures.

Chloe Tolley,1Diana Rofail,2 Adam Gater,1 Justine K Lalonde31Adelphi Values Ltd, Bollington, UK; 2Roche Products Ltd, Welwyn Garden City, UK; 3Roche S.A.S, Paris, France Abstract: Many clinical outcome assessments (COAs) were originally developed for completion via pen and paper. However, in recent years there have been movements toward electronic capture of such data in an effort to reduce missing data, provide time-stamped records, minimize administrative burden, and avoid secondary data entry errors. Although established in many patient populations, the implications of using electronic COAs in schizophrenia are unknown. In accordance with International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Task Force recommendations, in-depth cognitive debriefing and usability interviews were conducted with people with schizophrenia (n=12), their informal (unpaid) caregivers (n=12), and research support staff (n=6) to assess the suitability of administration of various electronic COA measures using an electronic tablet device. Minimal issues were encountered by participants when completing or administering the COAs in electronic format, with many finding it easier to complete instruments in this mode than by pen and paper. The majority of issues reported were specific to the device functionality rather than the electronic mode of administration. Findings support data collection via electronic tablet in people with schizophrenia and their caregivers. The appropriateness of other forms of electronic data capture (eg, smartphones, interactive voice response systems, etc) is a topic for future investigation. Keywords: ePRO, eCOA, mode of administration, electronic data capture, usability&nbsp

Background Patient-reported outcome (PRO) instruments are important tools for monitoring disease activity and response to treatment in clinical trials and clinical practice. In recent years, there have been movements away from traditional pen-and-paper PROs towards electronic administration. When using electronic PROs (ePROs), evidence that respondents complete ePROs in a similar way to their paper counterparts provides assurance that the two modes of administration are comparable or equivalent. The European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30 item (EORTC QLQ-C30) and associated disease-specific modules are among the most widely used PROs in oncology. Although studies have evaluated the comparability and equivalence of electronic and original paper versions of the EORTC QLQ-C30, no such studies have been conducted to date for the head and neck cancer specific module (EORTC QLQ-H&N35). Objective This study aimed to qualitatively assess the comparability of paper and electronic versions of the EORTC QLQ-H&N35. Methods Ten head and neck cancer patients in the United States underwent structured cognitive debriefing and usability interviews. An open randomized crossover design was used in which participants completed the two modes of administration allocated in a randomized order. Using a “think-aloud” process, participants were asked to speak their thoughts aloud while completing the EORTC QLQ-H&N35. They were thoroughly debriefed on their responses to determine consistency in interpretation and cognitive process when completing the instrument in both paper and electronic format. Results Participants reported that the EORTC QLQ-H&N35 demonstrated excellent qualitative comparability between modes of administration. The proportion of noncomparable responses (ie, where the thought process used by participants for selecting responses appeared to be different) observed in the study was low (11/350 response pairs [35 items x 10 participants]; 3.1%). Evidence of noncomparability was observed for 9 of the 35 items of the EORTC QLQ-H&N35 and in no more than 2 participants per item. In addition, there were no apparent differences in level of comparability between individual participants or between modes of administration. Conclusions Mode of administration does not affect participants’ response to, or interpretation of, items in the EORTC QLQ-H&N35. The findings from this study add to the existing evidence supporting the use of electronic versions of the EORTC instruments when migrated to electronic platforms according to best practice guidelines.

Background Herpes zoster (HZ) or “shingles” is common in persons aged 50 years or over. HZ is characterised by a painful dermatological rash which typically resolves in approximately one month. Persistent pain for months or years after rash onset, however, is a common complication of HZ; referred to as post-herpetic neuralgia (PHN). Both HZ and PHN have a significant impact on patients’ lives, with considerable implications for healthcare systems and wider society. The aim of the present review is to provide comprehensive documentation and critical appraisal of published data concerning the humanistic, economic and societal burden of HZ in Europe. Methods Systematic literature searches were conducted in Medline, EMBASE, PsycINFO, EconLit, HEED and CRD databases. Searches were conducted in July 2014 and restricted to articles published in the past 20 years. Articles were selected for full review by two independent researchers in accordance with predefined eligibility criteria. Results From a review of 1619 abstracts, 53 eligible articles, were identified which reported data concerning healthcare resource use (n = 38), direct costs (n = 20), indirect costs (n = 16), total costs (n = 10) and impact on health-related quality of life (HRQoL) (n = 21). Findings highlight that PHN is associated with greater impairments in HRQoL and higher costs of management than HZ. For both HZ and PHN, pain severity is a significant predictor of impact on individuals, healthcare systems and society. While the incidence of HZ and PHN increase with age, age does not appear to be a key driver of overall costs for HZ and PHN. Specifically, while direct costs (e.g. GP, specialists, medications, hospitalisations) tend to be higher for older patients, indirect costs (e.g. work time missed) are higher for younger patients. Conclusions Available evidence highlights that HZ and PHN result in significant humanistic and economic burden for patients, healthcare systems and wider societies. A tendency to focus upon healthcare resource use and the direct costs of management at the expense of other impacts (e.g. informal caregivers and formal social care) may result in an underestimation of the true burden of HZ and PHN.

Background Herpes zoster (HZ) is a painful condition that can have a substantial negative impact on patients’ lives. However, UK-specific data on the debilitating impact of HZ, in terms of patients’ experience of pain and impairments in Health-Related Quality of Life (HRQoL) are limited. The Zoster Quality of Life (ZQOL) study, a large-scale UK cross-sectional study, was conducted to quantify the burden of HZ in UK patients. Methods A total of 229 HZ patients aged 50 years or over were recruited from primary and secondary/tertiary care centres throughout the UK. Patients completed a battery of validated questionnaires, including the Zoster Brief Pain Inventory (ZBPI), the Medical Outcomes Study Short-Form 36 (SF-36) and the EuroQol-5 Dimensions (EQ-5D) on initial presentation to the doctor and again 7–14 days later. At follow-up patients also completed the Treatment Satisfaction with Medication (TSQM) questionnaire. Where available, mean questionnaire scores in the HZ population were compared to scores for age-matched norms to investigate the burden associated with HZ. Results Pain was prominent among patients, with 57.9% at the initial study visit reporting pain in the preceding 24 hours at levels typically considered to have a significant impact on HRQoL (i.e. ZBPI worst pain ≥ 5). This was reflected in SF-36 and EQ-5D scores that were significantly lower for patients when compared to age-matched norms (p