The power of hope: Views of Ovarian Cancer patients on how maintenance therapy Affects their Lives (VOCAL).

Aim: To assess maintenance preference and trade-offs for patients with advanced epithelial ovarian cancer. Methods: Patients completed a time trade-off exercise ranking five maintenance approaches. Patients' preferred approach was compared with alternatives to determine the progression-free time they would trade off to remain on their preferred approach. Results: Of 152 patients (median age 53 years, 68% White), 56% chose one of four maintenance medications, mostly to feel proactive and 44% chose active surveillance. Compared with their preferred approach, patients were willing to trade a mean progression-free time before switching of 2.3 months for once-daily oral medications, 3.2 months for twice-daily oral medications, 5.5 months for intravenous infusions every 3 weeks (iv. q3), 6.1 months for active surveillance and 7.5 months for iv. q3 and twice-daily oral. Conclusion: Findings highlight the importance of patients' awareness of all maintenance approaches and involving them in the decision-making process.

What is the article about? The VOCAL study looked at maintenance approach preferences of patients with advanced epithelial ovarian cancer. Maintenance approach refers to the methods used after a patient has completed their initial chemotherapy to prevent disease progression for as long as possible. US patients completed an online survey, ranking five different maintenance approaches: No medication (active surveillance); Once-daily oral medication (e.g. pills); Twice-daily oral medication; Medication by intravenous infusion every 3 weeks; Medication by intravenous infusion every 3 weeks and oral twice-daily. Patients were asked to assume the same time to disease progression for all five approaches and the same side effects for the four approaches involving medications (2­5). Each patient then indicated how much time to disease progression they were willing to trade off to remain on their preferred approach before switching to an alternative. What were the results? Overall, 152 patients completed the survey (median age: 53 years, 68% White). Most patients preferred a medication approach (56%, n = 85) to active surveillance (44%, n = 67). Of the 85 patients who preferred medication, 66% (n = 56) reported this was to feel proactive in preventing their cancer returning. Once-daily oral medication was the most acceptable alternative to the patients' preferred maintenance approach, given they were willing to trade the least 'disease-free' time (2.3 months) before accepting a switch. What do the results mean? Individual patient preferences vary, and healthcare professionals should work with patients to determine which approach is most appropriate for them.

Cross-walk of the Chronic Liver Disease Questionnaire for Nonalcoholic Steatohepatitis (CLDQ-NASH) and the EuroQol EQ-5D-5L in patients with NASH.

BACKGROUND: Nonalcoholic steatohepatitis (NASH) is a chronic progression of nonalcoholic fatty liver disease, which can negatively impact the health-related quality of life (HRQoL) of affected individuals. HRQoL in NASH has been assessed using the disease-specific Chronic Liver Disease Questionnaire for NASH (CLDQ-NASH) and the generic EuroQol EQ-5D-5L. As the performance of these instruments relative to each other is unknown, we performed a cross-walk analysis of CLDQ-NASH to EQ-5D-5L using data from a real-world NASH population. METHODS: Data were drawn from the Adelphi Real World 2019 NASH Disease Specific Programme, a cross-sectional survey of physicians and their patients in the United States. Patients with physician-diagnosed NASH completed a questionnaire that included the CLDQ-NASH and EQ-5D-5L. Mapping from CLDQ-NASH to EQ-5D-5L was done using tenfold cross-validation; performance was assessed using root-mean squared error as accuracy measure. Subgroup analyses compared performance of the models in obese versus non-obese patients and patients with versus without type 2 diabetes (T2D). RESULTS: Data from 347 patients were included in this analysis. Overall, 2172 models were tested for predicting EQ-5D-5L index score from CLDQ-NASH score. The best model for this mapping was a generalized linear model using Gaussian distribution and a power link. The best model for mapping from CLDQ-NASH domains to the EQ-5D-5L was a fractional logistic model. Models performed better at predicting upper versus lower values of EQ-5D-5L, for non-obese versus obese patients, and for patients without versus with T2D. CONCLUSION: We describe a scoring algorithm for cross-walking the CLDQ-NASH to the EQ-5D-5L enabling health status comparisons of HRQoL across studies.

Physician- and patient-reported symptom concordance and association with quality of life in hepatocellular carcinoma.

Aim: To investigate the association of discordance in patient- and physician-reported symptoms on health-related quality of life (HRQoL) in hepatocellular carcinoma (HCC). Patients & methods: Data were drawn from a point-in-time survey of physicians and patients conducted in Germany, Italy and Spain (October 2018 - January 2019). Physicians and their consulting patients independently reported baseline characteristics, symptoms, treatment history and satisfaction, and HRQoL derived using the Functional Assessment of Cancer Therapy-Hepatobiliary (FACT-Hep) questionnaire. Results: Of 486 patients analysed, tiredness (73.3%, 73.7%), pain/aches (67.7%, 66.9%) and weight/appetite loss (54.3%, 53.7%) were the most common and concordant patient and physician-reported symptom domains, respectively. The symptom domains showing the largest discordance were reflux/indigestion (14.6%, 5.1%), neurological (11.9%, 5.6%), dermatological (9.3%, 6.2%) symptoms and jaundice (4.7%, 10.3%). Reduced HRQoL was observed with increasing symptom-reporting discordance. Conclusion: Further studies should investigate how symptom-reporting discordance influences patient satisfaction and HRQoL.

Impact of Atopic Dermatitis Lesion Location on Quality of Life in Adult Patients in a Real-world Study.

BACKGROUND: Atopic dermatitis (AD) has a negative impact on patients’ quality of life (QoL). OBJECTIVE: To report the impact of specific AD lesion locations on QoL in adult patients with AD using real-world data. METHODS: The Adelphi US Disease Specific Programme was conducted between January–April 2018. Physicians documented patient demographics/characteristics, AD lesion locations, and body surface area; patients completed questionnaires reporting the impact of lesion locations on QoL. RESULTS: AD severity was moderate in 51.6% of patients and severe in 6.0%. Lesions were commonly identified in more than one location. All AD lesion locations impacted QoL. Visible areas were most bothersome, including head/neck (68%), hands/fingers (58%), front (30%), upper extremities (22%), and lower extremities (16%), with statistically significant associations for a number of Dermatology Life Quality Index (DLQI) items. Itch, soreness, pain, and stinging are also associated with a number of body areas but in particular with those that are most visible/accessible. Lesions on the head/neck and hands/fingers (58%) demonstrated an increased impact on the anxiety and depression dimension of the EuroQol 5-Dimension tool. CONCLUSIONS: In patients with AD, quality of life was most affected in patients with lesions in visible areas, including head/neck, hands/fingers, and upper extremities, with statistically significant associations for a number of DLQI domains. Physicians should be aware of the burden of AD lesions on QoL and consider having conversations with patients to better understand the impact of these lesions. Prior presentation: 28th Annual European Academy of Dermatology and Venereology Congress; 9–13 October 2019, Madrid, Spain. Poster number P0233.J Drugs Dermatol. 2020;19(10): 943-948. doi:10.36849/JDD.2020.5422.

Improving access to antiretrovirals in China: economic analyses of dolutegravir in HIV-1 patients.

BACKGROUND: The World Health Organisation recommended dolutegravir (DTG)-based antiretroviral therapy (ART) regimens are available but not reimbursed through the public reimbursement system in China. The objective of this analysis was to evaluate the cost-effectiveness of DTG (DTG + TDF/3TC) compared to efavirenz (EFV + TDF/3TC) in treatment-naive and ritonavir-boosted lopinavir (LPV/r + TDF/3TC) in first-line ART failure HIV-1-infected patients in China. METHODS: A dynamic Markov model comprising of 5 response states and 6 CD4+ count-based health states was used. Efficacy, estimated as probability of virologic suppression (HIV RNA < 50 copies/mL) at 48 weeks, was obtained from a published network meta-analysis for ART-naive patients and from the DAWNING study for patients failing first-line ART. Baseline cohort characteristics were informed using DTG phase 3 studies and the DAWNING study data, respectively. Health state utilities were derived from DTG phase 3 studies. A 5-year cost-effectiveness analyses was conducted using the societal perspective. Outcomes were quality-adjusted-life-years (QALYs), life-years (LYs), incremental cost per QALYs (ICER). RESULTS: The viral suppression rates for DTG + TDF/3TC were higher than EFV + TDF/3TC (75.3% vs 64.0%) in treatment-naive and LPV/r + TDF/3TC (74.8% vs 58.4%) in first-line ART failure patients. This resulted in higher QALYs for DTG + TDF/3TC in treatment-naive (4.232 vs 4.227) and first-line failure settings (4.224 vs 4.221). Total discounted cost for DTG + TDF/3TC patients (RMB 219.259 in treatment-naive and RMB 238,746 in first-line failures) were lower than comparators (EFV + TDF/3TC:RMB 221,605; LPV/r + TDF/3TC:RMB 244,364), thereby DTG dominated in both settings. Probabilistic sensitivity analyses indicated the probability of DTG + TDF/3TC being cost effective was 98.2% in treatment-naive setting and 100% in first-line failure setting at a willingness to pay threshold of RMB 100,000/QALY. CONCLUSIONS: With lower costs, higher response rates and higher QALYs, DTG + TDF/3TC can be considered as a cost-effective alternative for treatment naive and first-line failure patients in China.

Impact of nocturia on patients' health-related quality of life and healthcare resource utilisation compared with OAB and BPH: Results from an observational survey in European and American patients.

OBJECTIVE: To evaluate the impact of nocturia on patients' quality of life and healthcare resource utilisation (HRU) compared with overactive bladder (OAB) and benign prostatic hyperplasia (BPH). METHODS: Data were drawn from a multinational (France, Germany, Spain, UK and US) survey of physician and patient-reported outcomes. The patient groups of interests were those diagnosed with only nocturia, with only OAB, and with only BPH. Health-related quality of life (HRQoL) and productivity measures were derived from the EuroQoL-5D, OAB-q and the Work Productivity and Activity Impairment Questionnaire (WPAI). Measures of HRU included lower urinary tract symptoms (LUTS)-relevant surgeries, hospitalisations, current use of pads and related physician visits. Bivariate and multivariate regression analyses were used to evaluate associations between HRQoL/HRU/Productivity and nocturia status. Multivariate analysis was used to address any potential confounding factors among the groups, ie age, gender, body mass index (BMI), ethnicity and comorbidities. RESULTS: A total of 3552 patients were identified including 358 nocturia patients, 1415 OAB patients and 1779 BPH patients. The mean age of the nocturia patients was 61.2 years with a mean BMI of 27.3. About 60.6% were women, 87.2% were Caucasian, and their most common comorbidities included depression, hypertension and diabetes. In terms of impact, nocturia patients were significantly worse off than OAB patients in their HRQoL. There was no significant difference regarding HRU and productivity measurement. Nocturia patients also presented with significantly worse HRQoL and lower productivity compared with BPH patients. Nocturia patients also had more physician visits. CONCLUSIONS: Nocturia should be emphasised as a standalone LUTS disease with substantial patient impact. Compared with OAB and/or BPH, nocturia patients presented with a significant reduction on patients' quality of life, reduced work productivity and increased utilisation of healthcare resources.

Direct medical costs of severe hypoglycaemic events in patients with type 2 diabetes in England: A retrospective database study.

AIMS: Hypoglycaemia in patients with diabetes can be induced by insulins and sulfonylureas. We assessed the real-world impact of specific monotherapy and combination regimens on hypoglycaemic events requiring hospitalisation and related secondary costs to the English healthcare system. METHODS: This retrospective observational study used the Clinical Practice Research Datalink with linked hospital admission data during 2008-2012. Patients with type 2 diabetes mellitus (T2DM) using antihyperglycaemic agents (AHAs) were assigned to mutually exclusive subgroups (insulin- and non-insulin-containing regimens; treatment groups of interest; age group) based on treatment at index date (date of first AHA prescription). Outcomes were number and cost of hospital admissions with hypoglycaemic event-related diagnosis codes. RESULTS: We identified 110 206 patients with T2DM (mean age 64.9 years, time since diagnosis 5.4 years, HbA1c at index 7.4%), with 439 hypoglycaemic events requiring inpatient hospitalisation (mean length of stay 6.3 days, mean cost/stay £1351). Event rates and cost of stay were highest in patients treated with sulfonylurea- or insulin-based regimens. Event rates, duration and cost of stay were higher in older patients. CONCLUSION: Rates of severe hypoglycaemic events varied substantially between T2DM regimens. In this study of patients treated in clinical practice in England, sulfonylurea- and insulin-based regimens were associated with the highest event rates and costs associated with hospitalisation for severe hypoglycaemic events; hospitalisation for severe hypoglycaemic events was not observed with dipeptidyl peptidase-4 inhibitor monotherapy or with metformin.

Impact of urinary incontinence on healthcare resource utilization, health-related quality of life and productivity in patients with overactive bladder.

OBJECTIVE: To evaluate the impact of urinary incontinence (UI) on healthcare resource utilization (HRU), health-related quality of life (HRQoL) and productivity measures in patients with overactive bladder (OAB). PATIENTS AND METHODS: This retrospective, cross-sectional study used data from the Adelphi OAB/UI Disease Specific Programme, a multinational survey of patient- and physician-reported data, fielded between November 2010 and February 2011. The primary patient groups of interest were those with OAB, both with and without UI. Health-related quality of life and productivity measures were derived from the EuroQoL-5D, the Incontinence Quality of Life questionnaire, the Overactive Bladder Questionnaire, and the Work Productivity and Activity Impairment Questionnaire. Measures of HRU included OAB-related surgeries, OAB-related hospitalizations, incontinence pads, anticholinergic use and physician visits. Multivariate linear regression models and literature-based minimal clinically important differences were used to assess statistically significant and clinically meaningful differences in HRQoL and productivity measures between patients with OAB with UI and those without UI. RESULTS: A total of 1 730 patients were identified, with a mean age of 60.7 years, and 77.0% of them were women, 84.2% were non-Hispanic whites, and 71% were incontinent. Bivariate analyses showed that HRU was significantly higher among patients with OAB with UI than among those without UI in all categories except for the number of OAB-related physician visits. In both bivariate and multivariate analyses, incontinent patients presented with clinically and statistically significantly lower HRQoL and productivity measures with respect to all study endpoints, except for percentage of work time missed due to their OAB/UI. CONCLUSIONS: Urinary incontinence was associated with significantly higher HRU and lower HRQoL and productivity in this population of patients with OAB from five different countries. In addition to clinical considerations, the economic and humanistic impact of UI should be taken into account when evaluating treatment options for patients with OAB.

Impact of urinary incontinence on health-related quality of life, daily activities, and healthcare resource utilization in patients with neurogenic detrusor overactivity.

BACKGROUND: Neurogenic detrusor overactivity (NDO) leads to impaired health-related quality of life (HRQoL), productivity, and greater healthcare resource burden. The humanistic and economic burden may be more apparent in NDO patients with urinary incontinence (UI). The objective of this study was to compare the HRQoL, productivity, and health resource use (HRU) between continent and incontinent NDO patients. METHODS: A retrospective database analysis was conducted using the Adelphi Overactive Bladder (OAB)/UI Disease Specific Programme, a multi-national, cross-sectional survey reported from both patients' and physicians' perspectives. The population for this analysis included NDO patients with or without UI. General and disease-specific HRQoL were assessed using the EuroQoL-5D (EQ-5D), Incontinence Quality of Life questionnaire (I-QOL), and the Overactive Bladder Questionnaire (OAB-q). Productivity and daily activity impairment were measured using the Work Productivity and Activity Impairment (WPAI) questionnaire. HRU indicators included OAB-related surgery, OAB-related hospitalizations, incontinence pad usage, switching anticholinergics used for OAB due to inadequate response or adverse effects, and OAB-related physician visits. Bivariate analyses, multivariate ordinary least squares (OLS) regression analyses and published minimal clinically important differences (MCID) were used to assess relationships between incontinent status and the aforementioned outcome measures. RESULTS: A total of 324 NDO patients with or without urinary incontinence were included, averaging 54 years of age (SD 16), of whom 43.8 percent were male. Bivariate analyses detected no significant relationship between incontinent status and HRU variables. Regression analyses revealed that incontinent patients had clinically and statistically lower disease-specific HRQoL and greater impairment in daily activities as compared to continent patients. On average, incontinent patients scored 10 points lower on the I-QOL total score, 9 points lower on the OAB-q HRQoL score, 15 points higher on OAB-q symptom severity, and experienced 8.2 percent higher activity impairment due to their bladder condition (all p < 0.001). CONCLUSIONS: Incontinent NDO patients experience significantly lower HRQoL and activity impairment as compared to continent NDO patients.

Degree of Alignment Between Japanese Patients and Physicians on Alopecia Areata Disease Severity and Treatment Satisfaction: A Real-World Survey.

INTRODUCTION: Alopecia areata (AA) is characterized by non-scarring scalp and/or body hair loss and can negatively impact patient mental health. Data are limited on the alignment of patient and physician perceptions of AA severity with each other and with Japanese Dermatological Association (JDA) guideline criteria, and of patient-physician alignment on treatment satisfaction. Therefore, we performed analyses to compare JDA severity groupings with patient-physician alignment on disease severity and to explore treatment satisfaction in AA in Japan. METHODS: Data were drawn from the Adelphi AA Disease Specific Programme (DSP)™, a real-world survey of physicians and patients with AA in Japan conducted January-March 2021. Patients and physicians reported patient AA severity as mild, moderate or severe based on their subjective judgement. Patients were then categorized into five hair loss severity groups according to JDA criteria (S1-5), and patient-physician pairs were matched to assess alignment on severity and treatment satisfaction. RESULTS: Subjective patient- and physician-reported disease severity generally followed JDA severity groupings. The percentage of patient-physician alignment on severity recognition was 76.3% in the overall population. In misaligned pairs, 20.2%, 14.5%, 7.3%, 25.0% and 0.0% of physicians rated disease as more severe than patients in S1, S2, S3, S4 and S5, respectively. Regarding treatment satisfaction, patient-physician alignment was 57.6% in the overall population. In S5, 46.2% of physicians reported being less satisfied than patients. Both physicians and patients cited lack of efficacy as the main reason for dissatisfaction. Of 221 patients, 39.8% and 29.9% were categorized as borderline-abnormal cases for anxiety and depression, respectively. CONCLUSIONS: This study highlights previously unreported patient-physician misalignment on disease severity, level of treatment dissatisfaction and unmet needs due to the lack of effective treatment. Further study on how improvement of the misalignment between physicians and patients could increase both patient and physician satisfaction with treatment and improve the quality of life for patients with AA.

Patient Burden of Nonsegmental Vitiligo: A US Real-World Survey of Dermatologists and Their Patients.

INTRODUCTION: Vitiligo is a chronic autoimmune disease characterized by destruction of melanocytes, leading to skin depigmentation. Vitiligo can have a high quality-of-life burden and profound impact on psychosocial well-being. The objectives of this study were to describe the self-reported patient burden among patients with nonsegmental vitiligo with ≤ 10% affected body surface area, summarize the physician-reported psychosocial and psychological impact of vitiligo on patient lives, and describe disease characteristics and treatment history, goals, and satisfaction. METHODS: Data were drawn from the Adelphi Vitiligo Disease Specific Programme™, a real-world, cross-sectional survey with retrospective data collection of physicians and patients with vitiligo, collected in the United States between October 2021 and April 2022. Separate surveys for dermatologists and patients contained questions on clinical and demographic characteristics of patients with vitiligo and burden of vitiligo. Treatment history, goals, and satisfaction were assessed together with the impact of vitiligo on quality of life. RESULTS: Sixty-one dermatologists provided data for 326 patients with ≤ 10% affected body surface area (adults, n = 221; adolescents, n = 105); 90 of those patients also responded to the survey. The most common treatments were topical corticosteroids, topical calcineurin inhibitors, and narrow-band ultraviolet-B phototherapy, with the main treatment goal being repigmentation. Physician-reported treatment satisfaction was 56%; 25% of patients reported frustration with treatment options. Physicians reported impact of vitiligo on everyday life in 46% of patients. Patients reported 12.7% overall work impairment; mean scores for Hospital Anxiety and Depression Scale anxiety and depression domains were 3.5 and 2.2, respectively, and mean Vitiligo-specific Quality of Life index score was 26.9. Patients with facial involvement experienced higher burden than those without. CONCLUSION: A high patient burden was reported by dermatologists and their patients with vitiligo who had ≤ 10% affected body surface area, including psychosocial and psychological consequences. These findings highlight an unmet need in the treatment of vitiligo.

Vitiligo is a chronic disease in which cells that produce the skin pigment melanin are attacked, causing patches of skin to lose color and become pale. Vitiligo can have emotional impacts such as social or psychological distress that can affect the day-to-day well-being of individuals. However, there is a lack of studies that assess the ways that vitiligo affects the everyday lives of people with the condition in the United States. Dermatologists and people with vitiligo answered survey questions on treatment goals, any vitiligo treatments currently and previously used, and how satisfied they were with the results of treatment. The surveys also contained questions that assessed the impact of vitiligo on everyday life. Sixty-one dermatologists answered questions about 326 patients and 90 of those patients also provided their own answers to the survey questions. Both dermatologists and patients reported that restoring color to patches of pale skin was their goal in treating vitiligo. However, dermatologists and patients both reported that they were dissatisfied with the results of available treatments. Dermatologists and patients both reported that vitiligo impacted aspects of everyday life. Emotional and psychological impacts such as anxiety and depression were reported, as well as negative effects on patients' work and social lives due to vitiligo. These results confirm that vitiligo impacts the day-to-day well-being of patients. Furthermore, this study highlights that there is a need for improvements in the treatment of vitiligo.

Health-Related Quality of Life Burden Among Adults with Vitiligo: Relationship to Disease Severity and Disease Location.

INTRODUCTION: Vitiligo was historically regarded as a cosmetic disorder; however, it is an autoimmune disease. As a visible condition, it affects patient well-being. We assessed the impact of disease severity, lesion location, and body surface area (BSA) affected on patient health-related quality of life (HRQoL). METHODS: Retrospective data were from the Adelphi Real World Vitiligo Disease Specific Programme: a cross-sectional survey of physicians and their patients with vitiligo (10/2021-07/2022). Patient-reported outcomes were assessed by the Vitiligo-Specific Quality of Life Instrument (VitiQoL), Hospital Anxiety and Depression Scale (HADS), and EQ-5D-5L. The Work Productivity and Impairment Questionnaire (WPAI) questionnaire was used to assess disease-related impairment of daily activities. Data were stratified by physician-reported disease severity, presence/absence of vitiligo on the face, and BSA percentage affected. RESULTS: In total, 1388 patients were included. Mean (SD) VitiQoL, HADS depression, and anxiety scores were higher for those with severe disease [40.5 (26.1), 5.2 (4.4), and 6.8 (4.7)] than those with mild [24.8 (18.8), 3.6 (3.8), 4.2 (3.8)] or moderate [27.1 (22.6), 3.8 (4.5), 4.3 (4.4)] disease. Patients with face affected reported higher VitiQoL [30.0 (22.3) versus 23.2 (19.3)], and HADS scores [depression, 4.3 (4.3) versus 3.2 (3.9); anxiety, 5.0 (4.3) versus 3.8 (3.9)] than those without. Patients with ≥ 5% BSA affected had higher VitiQoL, depression and anxiety scores [27.9 (21.8), 4.0 (4.4), and 4.5 (4.2)] than those with 0-5% [24.6 (19.7), 3.4 (3.7), and 4.3 (4.1)]. Patients with severe vitiligo, facial lesions, or ≥ 5% BSA reported higher activity impairment. Mean EQ-5D-5L-utility score was approximately 0.9 regardless of disease severity or total BSA affected. CONCLUSIONS: These data demonstrate the impact disease severity can have on HRQoL and daily activities for patients with vitiligo. Lesions that are more severe, on the face, or covering a greater BSA are more often associated with poorer outcomes and activity impairment. These data also highlight the potential insensitivity of commonly used HRQoL measures and a need for more sensitive disease-specific measures.

Vitiligo is a disorder that causes patches of skin to lose pigment. In this study, we examined medical records of patients who have non-segmental vitiligo (the most common type of vitiligo) to better understand how the following factors affect the quality of life of patients with non-segmental vitiligo: (a) disease severity, (b) whether the face was affected, and (c) how much of the body was affected. Using a variety of measures, we found that patients with non-segmental vitiligo had lower quality of life, more symptoms of anxiety and depression, and higher activity impairment than those who did not. Our research highlights the differences in the measures used to assess the quality of life of patients, as well as the need for new therapies for non-segmental vitiligo.

Disease Course, Treatment Patterns and Goals Among Patients with Non-segmental Vitiligo Across Europe and the United States.

INTRODUCTION: There is currently a lack of research regarding disease course and burden as well as treatment patterns and goals in patients with non-segmental vitiligo (NSV). The aim of this analysis was to evaluate disease course, treatment patterns and goals in patients with NSV. METHODS: This analysis used secondary data from the Adelphi Real World Vitiligo Disease Specific Programme™ 2021, specifically, a survey of physicians and their adult and adolescent patients with NSV. Physicians categorized patients by the extent of NSV at time of survey completion as mild, moderate or severe/very severe. Physician-reported patient information included demographics, current/previously prescribed NSV therapies, treatment satisfaction and the Vitiligo Noticeability Scale (VNS). Patients completed a survey on treatment satisfaction and the VNS. Treatment pattern data were stratified by disease extent and Fitzpatrick skin type. RESULTS: At survey completion, physicians reported that 38, 50 and 12% of patients (N = 1865) had improving, stable and deteriorating/progressing disease, respectively. Most patients (96%) with mild disease at treatment initiation still had mild disease at the time of survey completion. More than half of patients with moderate disease (62%) or severe/very severe disease (57%) at treatment initiation still had moderate or severe/very severe disease at survey completion. Topical calcineurin inhibitors (TCIs) were the most common treatment in 40% of patients followed by phototherapy in 30%. Patients hoped for re-pigmentation (mild 56%, moderate 62%, severe/very severe 66%), reduction (mild 50%, moderate 56%, severe/very severe 49%) or cessation of affected areas with vitiligo (mild 48%, moderate 54%, severe/very severe 43%). CONCLUSION: The study findings indicate that a significant proportion of patients with NSV are not improving on current treatments, most commonly TCIs and phototherapy. The results highlight the unmet need for novel and effective therapies to substantially improve re-pigmentation, an important treatment goal for patients with NSV.

Satisfaction with Control of Mild to Moderate Atopic Dermatitis with Ruxolitinib Cream: US Physician and Patient Perspectives.

INTRODUCTION: The 2021 US approval of ruxolitinib cream for treatment of atopic dermatitis (AD) in patients aged ≥ 12 years was based on the results of two pivotal phase 3 studies. Currently, real-world data to describe effectiveness of ruxolitinib cream and physician satisfaction with treatment remain limited. Our objective is to describe disease control among adults with mild to moderate AD prescribed ruxolitinib cream and physician satisfaction with treatment. METHODS: Data were from the Adelphi AD Disease Specific Programme™, a US real-world, cross-sectional survey of physician-reported data, undertaken between August 2022 and March 2023. For patients aged ≥ 18 years, physicians reported patient demographics, clinical characteristics, treatment patterns, and physician satisfaction with disease control. Descriptive analysis of data for patients with mild to moderate AD prior to the initiation of ruxolitinib cream and treated with ruxolitinib cream for ≥ 1 month was undertaken. RESULTS: Among physician-reported data from 1360 patients with AD, 149 patients had received ruxolitinib cream (in combination or as monotherapy) for ≥ 1 month, including 59 patients receiving monotherapy. Prior to treatment with ruxolitinib cream, 84.6% of patients had moderate AD (Investigator's Global Assessment, IGA of 3), whereas after treatment (median duration, 26 weeks), only 21.5% had an IGA of 3, with 48.3% of patients having clear or almost clear skin (IGA of 0/1). For these patients, 81.2% were not currently experiencing a flare, and physicians were satisfied with disease control for 87.3%. Results were similar in patients receiving monotherapy. The most frequent physician-reported reasons for prescribing ruxolitinib cream included relieving itch, improving lesion redness/thickness, achieving disease control, and reducing/controlling flares. CONCLUSIONS: These real-world findings demonstrate effective disease control and physician satisfaction with ruxolitinib cream for the treatment of AD in adults in a clinical practice setting. Outcomes were similar whether ruxolitinib cream was prescribed as monotherapy or in combination regimens, suggesting a role for ruxolitinib cream across the spectrum of disease.

Atopic dermatitis (AD) is a disease in which skin can be itchy, inflamed, and cracked. Traditional therapies for mild to moderate AD can be limited by side effects and long-term safety issues. After US approval of ruxolitinib cream for the treatment of mild to moderate AD in 2021, the goal of this study was to describe disease control and doctor satisfaction with ruxolitinib cream in a real-world setting. The Adelphi AD Disease Specific Programme™ surveyed 159 doctors who treated people with AD between August 2022 and March 2023. Doctors reported records from 1360 patients with mild to moderate AD. In these patients, ruxolitinib cream was used for at least 1 month in 149 patients and was used alone in 59 patients. Before the use of ruxolitinib cream, nearly 85% of the 149 patients had moderate AD. After the use of ruxolitinib cream, about 20% had moderate AD, with half having clear or almost clear skin. About 80% were not currently experiencing flares. Doctors were satisfied with disease control in more than 85% of patients. Patients applying ruxolitinib cream alone had similar results. Doctors most often prescribed ruxolitinib cream for itch relief, disease control, and to reduce or control flares. In summary, when ruxolitinib cream was used by patients, it provided good disease control, and doctors were satisfied with results. Outcomes were similar in patients who applied ruxolitinib cream alone or with another treatment. This suggests that ruxolitinib cream may be useful for patients with AD of differing levels of severity.

Real-World Experience of Adalimumab Biosimilar (ABP 501) Use in Patients with Inflammatory Bowel Disease in Europe.

INTRODUCTION: Approval of adalimumab biosimilar ABP 501 (Amgevita®) for inflammatory bowel disease (IBD) was based upon the principle of extrapolation. Real-world experience of ABP 501 utilization in IBD can provide useful information to healthcare providers and patients. METHODS: Data were drawn from the 2020-2021 Adelphi IBD Disease Specific Programme™ conducted in five major European countries. Participating gastroenterologists completed a point-in-time survey to provide patient medical record data, and patients voluntarily completed questionnaires to report health-related quality of life (HRQoL). Descriptive analyses were conducted for "ABP 501 initiators" (received ABP 501 as first advanced therapy) and "RP-ABP 501 switchers" (switched to ABP 501 from reference product [RP; Humira®] as first advanced therapy). RESULTS: This analysis included 239 ABP 501 initiators and 136 RP-ABP 501 switchers. At consultation, initiators had been on ABP 501 treatment for a median of 7.5 months and switchers had received ABP 501 for a median of 7.7 months following the switch from a median of 14.0 months treatment with RP. About 74% of initiators and 89% of switchers were reported by their treating physicians as being in clinical remission. Physicians and patients reported satisfaction with ABP 501 in the range of 92-99% across both groups. Patient self-assessment, including EuroQol visual analogue scale, Short IBD Questionnaire, and Work Productivity and Activity Impairment scores, suggested minimal impairment of HRQoL while on ABP 501. The most common reason for RP to ABP 501 switch was lower healthcare costs. CONCLUSION: Both patients with IBD and treating physicians reported high levels of satisfaction with ABP 501 among initiators and switchers.

ABP 501 (Amgevita^®) is the first approved biosimilar to adalimumab originator (Humira^®), referred to here as the reference product. A biosimilar is a biological product that is highly similar to its reference product in terms of safety, purity, and effectiveness. ABP 501 has been approved for the treatment of certain chronic inflammatory diseases. The approval of ABP 501 for inflammatory disease is based upon the principle of extrapolation, with no clinical trial being conducted in patients with inflammatory bowel disease. Therefore, in this current study, we evaluated the utilization experience of biosimilar ABP 501 in the real-world setting from both physicians' and patients' perspectives. We reported that patients with inflammatory bowel disease who initiated ABP 501 as the first advanced therapy as well as patients who continued therapy on ABP 501 after a switch from the adalimumab reference product both had a high level of satisfaction when using the biosimilar ABP 501. Treating physicians also reported that most of their patients were in clinical remission while on treatment with ABP 501, and patients themselves reported minimal impairment of health-related quality of life.

Improving Assessment of Adherence Behaviors and Drivers: Targeted Literature Review and Concept Elicitation Interviews in Multiple Countries and Disease Populations.

Purpose: Medication adherence is crucial for achieving clinical goals. Medication adherence drivers and behaviors were explored across multiple conditions, countries, and medication schedules/modalities to develop a conceptual model of medication adherence, which could later be used to support development of a patient-reported outcome (PRO) measure of adherence. Patients and Methods: Targeted review of qualitative literature identified important medication adherence concepts. Fifty-seven qualitative concept elicitation interviews were conducted (USA n=21, Spain n=18, Germany n=18). Participants were prescribed medication for: hypertension (n=9), asthma (n=8), multiple myeloma (n=8), psoriasis (n=8), diabetes (n=7), depression (n=7), multiple sclerosis (n=7), and/or schizophrenia (n=6). Thematic analysis of verbatim transcripts was performed. Expert clinicians (n=3) provided input throughout. Results: Nine qualitative articles were selected for review from 2168 screened abstracts. Forty-two medication adherence concepts were reported and grouped into 10 domains. Eight forms of medication adherence were reported during interviews, along with 27 drivers of non-adherence, all of which were incorporated into a conceptual model. Participants reported skipping medication doses (n=36/57; 63.2%) or taking medication later in the day than prescribed (n=29/57; 50.9%). Common drivers of non-adherence included forgetfulness (n=35/57; 61.4%), being out of the usual routine (n=31/57; 54.4%) and being busy (n=22/57; 38.6%). US participants were more likely to report non-adherence due to low perceived efficacy (n=6/21, 28.6%) and cost (n=5/21, 23.8%) than German (n=1/18, 5.6%; n=0/18, 0.0%) or Spanish (n=2/18, 11.1%; n=1/18, 5.6%) participants. Conclusion: Findings highlight the diverse forms and drivers of medication non-adherence, informing the development of a comprehensive conceptual model of medication adherence. The conceptual model builds on and advances previous models of medication adherence and can be used by healthcare professionals to understand and interpret barriers to medication adherence and how best to support patients in taking their medication as intended.

Medication adherence is the extent to which a patient takes their medication as prescribed. This paper describes a literature review and concept elicitation interviews to identify forms and drivers of medication adherence across a diverse sample of participants. Forms of non-adherence identified included: deviating from the prescription, skipping a dose, taking a different amount, and taking medication at a different time. Behaviours and drivers can vary by condition, treatment modality, and dosing schedule.This research highlights the variation in the prevalence of medication non-adherence, and the different forms and drivers of non-adherence, based on individuals' demographic and clinical characteristics. The conceptual model developed advances previous models of medication adherence and may support healthcare professionals in the management of patients and how they can be supported to take medication as intended. The research ultimately informed the development of the Adelphi Adherence Questionnaire (ADAQ^©), a novel generic patient-reported outcome measure.

Patient and family burden in pediatric atopic dermatitis and its treatment pattern in Japan.

BACKGROUND: This study evaluated the level of burden in pediatric and adolescent atopic dermatitis (AD) patients in Japan, the associated burden on caregivers/families, and whether this burden varied with age. METHODS: Data were drawn from the Adelphi Pediatric AD Disease Specific Programme (DSP)™, a cross-sectional survey of physicians and their patients conducted in Japan between July and December 2022. Physicians reported patient demographics, clinical characteristics, disease burden, and current/previous therapies. Patients and/or caregivers reported perceived disease severity and impact of AD, including the Children's Dermatology Life Quality Index (CDLQI) and Dermatitis Family Impact questionnaire (DFI). RESULTS: Overall, 55 physicians provided data for 537 AD patients aged ≤17. Mean (SD) overall scores for CDLQI, POEM, and DFI were 9.3 (6.3), 8.3 (6.8), and 11.7 (7.2), respectively. Age was associated with higher patient and/or caregiver-reported CDLQI scores, which increased by 0.543 points per year of age (P = 0.01). Patients with severe disease reported a more significant impact on quality of life factors compared with mild patients (P < 0.001). Age was associated with higher caregiver-reported burden, with DFI scores increasing by 0.325 per year (P = 0.01). Physician-reported impact on caregivers showed that age was significantly associated with increased burden on sleep, daily activities, work, and mood (P < 0.05), with disease severity associated with impact across all factors (P < 0.01). CONCLUSIONS: Both increasing age and disease severity were associated with the increased impact of AD on patients and their caregivers. Disease control/modification through appropriate therapeutic intervention at a younger age may relieve the burden of pediatric AD on patients and their families.

Impact of rheumatoid arthritis on quality of life, work productivity and resource utilisation: an observational, cross-sectional study in Brazil.

OBJECTIVES: Increasing severity in rheumatoid arthritis (RA) may result in poorer health-related quality of life (HRQoL), reduced work productivity and increased resource utilisation. This study investigated the impact of RA severity on HRQoL and healthcare resource utilisation among RA patients in Brazil. METHODS: Data were drawn from an observational cross-sectional study of consulting RA patients undertaken in November-December 2007. Rheumatologists (n=55) provided information for 526 RA patients, 521 of whom also completed patient self-completion (PSC) questionnaires. Physicians subjectively rated each patient's RA as mild, moderate or severe. The PSC included the Work Productivity and Activity Impairment questionnaire (WPAI), Health Assessment Questionnaire Disability Index (HAQ-DI) and HAQ-Pain score, EuroQoL-5D (EQ-5D) and Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue. Data on the number of hospitalisations and consultations to healthcare professionals in the past 12 months and RA drug therapy class received were also collected. RESULTS: Patients with severe RA had significantly impaired health and work status compared to those with mild/moderate disease. Overall work and activity impairment rose with increasing disease severity. Health status deteriorated as disease severity increased with worsening disability, pain, fatigue, quality of life and perceived general health status. Hospitalisation rate and frequency of physician consultations were also significantly greater among those with severe RA. CONCLUSIONS: In Brazil, moderate to severe RA is associated with significant functional disability and morbidity. Disease severity should be considered when treating patients with RA. More aggressive treatment strategies may be needed to effectively manage patients with moderate to severe RA.

Suboptimal Clinical and Quality of Life Outcomes in Patients with Psoriasis Undertreated with Oral Therapies: International Physician and Patient Survey.

INTRODUCTION: Psoriasis is a chronic, inflammatory, immune-mediated disease. This study assessed the time at which patients switched from a conventional oral systemic treatment to a biologic therapy; patient clinical and quality of life (QoL) outcomes associated with oral systemic treatments; and the proportion of patients who persisted on oral therapy (nonswitchers), despite reported suboptimal clinical and QoL outcomes. METHODS: This data analysis used the Adelphi Real World Psoriasis Disease Specific Programme, a non-interventional, retrospective, cross-sectional survey conducted in the USA, France, Germany, and United Kingdom (August 2018-April 2019). Kaplan-Meier (KM) analysis assessed switching from oral to biologic therapy in patients treated ≥ 3 years at survey completion (n = 597). The severity of psoriasis was reported by physicians as the percentage of body surface area (BSA) affected by psoriasis. Dermatology Life Quality Index (DLQI) scores were calculated for three groups: nonswitchers who met treatment failure criteria, nonswitchers who did not meet failure criteria, and switchers to a biologic therapy. RESULTS: In KM analysis, approximately 50% of the patient population switched by 24 months. A substantial portion of nonswitchers continued to have moderate-to-severe psoriasis. Among nonswitchers, 57-77% had BSA ≥ 3% and 16-24% had BSA ≥ 10% at the time of the survey compared with 37% of switchers who had BSA of ≥ 3% and 9% who had BSA of ≥ 10%. QoL was poor among nonswitchers. The mean [standard deviation (SD)] DLQI scores for nonswitchers meeting treatment failure criteria, nonswitchers not meeting failure criteria, and switchers were 6.11 (4.55), 2.62 (3.29), and 2.25 (4.23), respectively. CONCLUSION: There is a clear unmet need for more effective oral therapies, and further research into the reasons for patients remaining undertreated, which may include patient preference for oral treatments (despite lack of response), contraindications, or insurance/formulary-related barriers to access, are needed.

Psoriasis is a common skin disease that causes itchy, painful, scaly sores. Patients may feel stigmatized, which can impact their quality of life and productivity. About 1 in 5 patients have severe psoriasis, which is harder to treat and may require pills or shots. Both shots and pills are effective at treating psoriasis; however, many patients choose to continue taking pills, even if their psoriasis worsens, for reasons including a desire to avoid needles. We described patients with moderate-to-severe psoriasis who started on pills and either remained on pills or switched to shots by the time of the survey. We looked at the reasons they gave for switching, as well as at quality of life measures reported by the patients. To do this, we analyzed data from a survey called the Adelphi Real World Psoriasis Disease Specific Programme. This survey was conducted among doctors who treat skin diseases and their patients. Participating doctors and patients from the USA, France, Germany, and the United Kingdom were asked questions about the patients' health and how psoriasis affects their lives. Survey results showed that nearly half of patients switched to shots by 24 months, and most who switched cited treatment failure as the reason. Those who continued to take their pills despite having more severe psoriasis symptoms had more itching and pain and had lower quality of life than those who switched to shots. This suggests that there is a need for more effective oral treatments for patients with psoriasis.

Real-world evidence generation from patients, their caregivers and physicians supporting clinical, regulatory and guideline decisions: an update on Disease Specific Programmes.

OBJECTIVE: To update on and describe the role of Disease Specific Programmes (DSPs), a multi-perspective real-world data (RWD) source, in the context of the evolution of the value and acceptance of real-world evidence (RWE) in clinical, regulatory and guideline decision-making. METHODS: DSPs are multi-national, multi-subscriber, multi-therapy cross-sectional surveys incorporating retrospective data collection from patient, caregiver and physician perspectives. Information collected covers the patient journey, including treatment/prescribing patterns and rationale, patient-reported outcomes, impact on work and everyday activities, attitudes towards and perceptions of the condition, adherence to treatment and burden of illness. Published peer-reviewed DSP papers were aligned with current key RWE themes identified in the literature, alongside their contribution to RWE. RESULTS: RWE themes examined were: using RWE to inform clinical practice, patient and caregiver engagement, RWE role in supporting health technology assessments and regulatory submissions, informing value-driven healthcare decisions, real-world patient subgroup differences and therapeutic inertia/unmet needs; highlighting patients' and caregivers' experience of living with a disease, disconnect from their physicians, unmet needs and educational gaps. CONCLUSIONS: DSPs provide a wealth of RWD in addition to evidence generated by registries, clinical trials and observational research, with wide use for the pharmaceutical industry, government, funding/regulatory bodies, clinical practice guideline insights and, most importantly, informing improvements in people's lives. The depth, breadth and heritage of information collected via DSPs since 1995 is unparalleled, extending understanding of how diseases are managed by physicians in routine clinical practice and why treatment choices are made, patients' perceptions of their disease management, and caregiver burden.