Evaluating Oncologists' Practice Patterns and Decision-Making in Locally Advanced or Metastatic Urothelial Carcinoma: The US Physician PARADIGM Study.

BACKGROUND: The treatment landscape for locally advanced/metastatic urothelial carcinoma (la/mUC) has evolved. This study examined US prescribing patterns and clinical decision-making for first-line (1L) and first-line maintenance (1LM) treatment. MATERIALS AND METHODS: US-based oncologists (N = 150) completed an online survey on patient demographics, practice patterns, and important factors considered in 1L/1LM selection. Multivariable logistic regression was used to assess factors associated with more vs less frequent 1L/1LM prescribing. RESULTS: Physician reports estimated that 23% of patients with la/mUC had not received any systemic therapy in the previous 6 months; however, 46% received 1L, 32% received second-line, and 22% received subsequent-line systemic treatments. Of patients who were receiving 1L treatment, 72% were estimated to be receiving 1L platinum-based chemotherapy. Around 69% of patients eligible for 1LM received the treatment. Physicians categorized as frequent prescribers reported overall survival (OS), disease control rate (DCR), and rate of grade 3/4 adverse events (AEs) as factors associated with 1L treatment selection (all P < .05). OS, rate of grade 3/4 immune-mediated AEs, and inclusion in institutional guidelines were reported as attributes used in 1LM treatment selection (all P < .05). Multivariable analysis revealed OS, DCR, and rate of grade 3/4 AEs as important factors in oncologists' 1L treatment selection; academic practice setting and use of Response Evaluation Criteria in Solid Tumors version 1.1 were associated with 1LM use (all P < .05). CONCLUSION: OS and AEs were found to be relevant factors associated with offering 1L and 1LM treatment. Variability exists in physicians' decision-making in the real-world setting for la/mUC.

Real-world treatment satisfaction with erenumab in migraine: analysis of the US National Health and Wellness Survey.

OBJECTIVE: The treatment landscape for the prevention of migraine has rapidly evolved in recent years with the advent of calcitonin gene-related peptide therapy, including erenumab. The objective of this study was to assess patient-reported treatment satisfaction among erenumab users. METHODS: This retrospective, cross-sectional study used data from the 2019 US National Health and Wellness Survey collected during March-July 2019. Respondents self-reporting physician-diagnosed migraine and currently using erenumab were analyzed. Treatment satisfaction was measured on a seven-point Likert scale. Data were further reported by the duration of erenumab treatment. Data on respondents' socio-demographic characteristics and treatment patterns were also collected. RESULTS: Overall, 67 respondents using erenumab with or without other migraine preventives for up to 1 year were included in the analysis. The mean (standard deviation) age was 46.7 (12.9) years. Most of the respondents were women (86.6%), White (74.6%), and commercially-insured (67.2%). Notably, 40.3% had ≥1 comorbidity per the Charlson Comorbidity Index. Approximately half of the respondents were college graduates and employed (49.3% each). Among the 67 respondents, 46 received erenumab exclusively. Across both cohorts, the percentage of respondents who were satisfied with erenumab treatment was slightly higher among those with a longer treatment duration (overall erenumab cohort: 63.6%, 69.6%, and 75.8% for 0-<3, 3-<6, and 6-12 months, respectively; erenumab monotherapy cohort: 62.5%, 71.4%, and 87.5% for 0-<3, 3-<6, and 6-12 months, respectively). Treatment patterns before switching to erenumab revealed that most respondents had used ≥1 preventive treatment for migraine (80.6%; 54/67), over two-thirds (33/54) of whom had ≥2 treatment failures owing to nonresponse. CONCLUSION: Satisfaction was high among long-term erenumab users, indicating that those using erenumab for a longer duration are more satisfied. Furthermore, this study provided insights on the basic socio-demographics, disease characteristics, and health behaviors of erenumab users as well as their treatment patterns before switching to erenumab.

Understanding the Association between Red Blood Cell Transfusion Utilization and Humanistic and Economic Burden in Patients with ß-Thalassemia from the Patients' Perspective.

We assessed the humanistic and economic burden of chronic red blood cell (RBC) transfusions on patients with ß-thalassemia. This cross-sectional, US-based study included adults (≥18 years) who self-reported a ß-thalassemia physician diagnosis and had received ≥1 RBC transfusion in the past 6 months. The outcomes included the Functional Assessment of Cancer Therapy-Anemia (FACT-An), Patient Health Questionnaire-9, Generalized Anxiety Disorder-7, and ad hoc questions about treatment experience, side effects, direct/indirect costs, and psychological burden. Overall, 100 patients completed the survey, of whom 70% experienced "moderate" to "extremely high" burden due to RBC transfusions, 81% reported iron overload, 42% reported compromised social lives. The mean FACT-An score was 132 (higher score indicates better outcomes; 0-188). Mean scores were 33/52 for fatigue and 20/28 for anemia symptoms in the previous 7 days. Health-related quality of life (HRQoL) temporarily improved after RBC transfusion, although patients continued to experience mild-to-severe depression and anxiety, substantial direct costs, compromised employment, and suboptimal quality of life. Over 6 months, patients dedicated a mean of 173 h to transfusion requirements and incurred out-of-pocket costs of USD 2239 for transfusions and USD 896 for additional care costs. These findings highlight the need for new treatment options to improve patient HRQoL and economic outcomes.

Physicians' experience in blood supply shortages and the top factors that impact the clinical, economic, and humanistic outcomes of patients with myelodysplastic syndromes in 5 European countries.

OBJECTIVE: Blood supply shortages may create unnecessary burden, including treatment delay, worsened quality of life, or increased healthcare resource utilization in patients with myelodysplastic syndromes (MDS). This study examined physicians' experience with blood supply shortages in the MDS population. Additionally, physicians' perspectives on the factors that impact clinical, economic, and humanistic outcomes of patients with MDS were investigated. METHODS: A total of 378 physicians primarily specializing in hematology/oncology across the UK, France, Germany, Italy, and Spain completed the survey (n ≈ 75 in each country). Physicians answered questions regarding adequacy of blood supply for patients with MDS who require red blood cell (RBC) transfusions and identified factors impacting the clinical, economic, and humanistic outcomes in the MDS population. RESULTS: Over 65% of physicians reported that their patients with MDS requiring RBC transfusions encountered RBC transfusion delays due to blood supply shortage. Among physicians who reported delays, 13.8% of patients were impacted, ranging from 11.0% in Spain to 19.4% in Italy. On average, patients experienced a 4.2-day delay in receiving RBC transfusions due to blood supply shortages, and 16.7% of patients required additional healthcare provider visits. Eastern Cooperative Oncology Group performance status, threshold hemoglobin levels, and age were the top factors reported by more than two-thirds of physicians that impact outcomes of patients with MDS. CONCLUSION: Our findings support the need for new treatments in MDS that reduce transfusions and thus blood supply needs, and that would have a beneficial effect on clinical, humanistic, and economic outcomes.

Association between transfusion status and clinical and economic outcomes in patients with myelodysplastic syndromes from the physicians' perspective.

BACKGROUND: The current study investigated physicians' understanding of the impact of transfusion status (TS) on clinical and economic outcomes in patients with myelodysplastic syndromes (MDS). MATERIALS & METHODS: 378 physicians primarily specializing in hematology/oncology across five European countries completed the survey. The survey asked physicians for their perspectives on the impact of TS on risk of death, risk of progression to acute myeloid leukemia (AML), chance of leukemia-free survival, and number of significant bleeding events, infection events, hospitalizations, and emergency room (ER) visits. RESULTS: Physicians estimated that compared to transfusion-dependent (TD) patients, transfusion-independent (TI) patients had a 37.6% reduced risk of death, lower risk of progression to AML, and lower risk of non-leukemic death, for all MDS risk levels. TD patients who became TI after treatment were estimated to have 40.6% reduced risk of death and 34% reduced risk of progression to AML, compared to TD patients who remained TD. CONCLUSIONS: Compared with TD patients, physicians estimated that TI patients have fewer events of infection and significant bleeding, and experience fewer hospitalizations and ER visits per person per year. Overall, physicians reported better outcomes for TI patients. New treatment options for patients with MDS to reduce or eliminate transfusion burden are warranted.

Humanistic and economic burden associated with depression in the United States: a cross-sectional survey analysis.

BACKGROUND: Depression (major depressive disorder [MDD]) affects the functioning of patients in many facets of life. Very few large-scale studies to date have compared health and economic related outcomes of those with versus without depression, and across various depression severity groups. We aimed to evaluate humanistic and economic burden in respondents with and without depression diagnosis, and across symptom severity groups. METHODS: Data from the 2017 US National Health and Wellness Survey (NHWS) were utilized. Of the adult respondents (N = 75,004), 59,786 were < 65 years old. Respondents not meeting eligibility criteria were excluded (e.g., those self-reporting bipolar disorder or experiencing depression in past 12 months but no depression diagnosis). Overall, data from 39,331 eligible respondents (aged 18-64 years) were analyzed; and comprised respondents 'with depression diagnosis' (n = 8853; self-reporting physician diagnosis of depression and experiencing depression in past 12 months) and respondents 'without depression diagnosis' (n = 30,478; no self-reported physician diagnosis of depression and not experiencing depression). Respondents with depression were further examined across depression severity based on Patient Health Questionnaire-9 (PHQ-9). Outcome measures included health-related quality-of-life (HRQoL; Medical Outcomes Study 36-item Short Form [SF-36v2]: mental and physical component summary [MCS and PCS]; Short-Form 6 Dimensions [SF-6D]; and EuroQol 5 Dimensions [EQ-5D]), work productivity and activity impairment (WPAI), and health resource utilization (HRU). Multivariate analysis was performed to examine group differences after adjusting covariates. RESULTS: Respondents with depression diagnosis reported significantly higher rates of diagnosed anxiety and sleep problems versus those without depression (for both; P < 0.001). Adjusted MCS, PCS, SF-6D, and EQ-5D scores were significantly lower in respondents with depression versus those without depression (all P < 0.001). Consistently, respondents with depression reported higher absenteeism, presenteeism, and overall WPAI, as well as greater number of provider visits, emergency room visits, and hospitalizations compared with those without depression (all P < 0.001). Further, burden of each outcome increased with an increase in disease severity. CONCLUSIONS: Diagnosed depression was associated with lower health-related quality-of-life and work productivity, and higher healthcare utilization than those without depression, and burden increased with an increase in symptom severity. The results show the burden of depression remains high even among those experiencing minimal symptoms.

Patient-Reported Outcome Measures in Adult Patients Diagnosed with Epilepsy Being Treated with Perampanel.

BACKGROUND: Epilepsy is a complex disorder that can affect patients' medical, psychological, and social well-being. The purpose of this study was to evaluate the patient-reported outcome (PRO) measures of health-related quality of life (HRQoL), satisfaction, and adherence in adult patients diagnosed with epilepsy treated with perampanel in the United States (US). METHODS: A US-based, multicenter, observational cross-sectional survey was completed by 61 patients taking perampanel with or without other antiseizure medications (ASMs). Respondents were ≥18 years old, had a physician-confirmed diagnosis of epilepsy, used perampanel for ≥4 months, and provided informed consent. Patients responded to questions concerning their demographic characteristics, treatment history, experiences before perampanel, experiences while taking perampanel, HRQoL, treatment satisfaction, and medication adherence. RESULTS: Patients (N=61) were 42.8 years old on average; majority were female (63.9%) and white (75.4%). Mean time on perampanel was 2.5 years, with sodium channel blockers often (55.7%) used concomitantly with perampanel. Patients reported, on average, 5.5 (standard deviation [SD]=13.2) seizures/month after initiating perampanel, whereas these same patients reported experiencing 20.4 (SD=60.0) seizures/month prior to perampanel. When comparing their experience on perampanel with their experience with previous ASMs, more patients "strongly agreed" that perampanel allowed them to live a more normal life (36.1% vs 27.5%) and worked as intended if they missed taking a dose (16.4% vs 7.8%). Average satisfaction scores were high, with ratings of 71.8 for effectiveness, 84.0 for convenience, and 71.9 for global satisfaction (0-100 scores). Perampanel use was associated with improvements in HRQoL and fewer symptoms of depression and anxiety. The majority of patients were adherent (62.3%) to perampanel. DISCUSSION: Perampanel use was associated with reductions in number of seizures, better HRQoL, and high adherence rates. These results provide initial evidence that perampanel can be an effective, tolerable, and valid option for patients with epilepsy in the real world.

Trends of self-reported non-adherence among type 2 diabetes medication users in the United States across three years using the self-reported Medication Adherence Reasons Scale.

BACKGROUND & AIMS: To determine the trends of self-reported non-adherence rates among adults taking Type 2 medicines (T2D) medicines between 2017 and 2019 and to identify the patterns for the frequently reported reasons for non-adherence in the United States. METHODS & RESULTS: Data from the National Health and Wellness Survey, a self-administered, internet-based cross-sectional survey of US adults from 2017 to 2019 was used. Non-adherence was measured using the self-reported Medication Adherence Reasons Scale (MAR-Scale). Frequencies were used to identify the reasons for non-adherence for insulin and non-insulin therapies for T2D. Data were obtained from 2983 respondents in 2017, 5416 in 2018, and 5268 in 2019. Based on the MAR-Scale, the self-reported medication non-adherence rate was 25% in 2017, 21% in 2018, and 27% in 2019. The most common reason for non-adherence across all the three years was simple forgetfulness, yet patients reported the lowest mean number of days missing medication for that reason. Though less frequently reported, non-adherence lasted longer when patient did not know how to take their medicines, cost was a reason, or had concerns about the long term effects of the medicines. CONCLUSIONS: With no significant improvement in adherence with T2D medicines over time, regardless of better awareness and extensive diabetes education, focus should be on individualized non-adherence reasons-based interventions.

Characterizing Outcomes and Unmet Needs Among Patients in the United States with Mild-to-Moderate Plaque Psoriasis Using Prescription Topicals.

INTRODUCTION: Topical therapies are considered first-line treatment in the management of plaque psoriasis (PSO). However, data on patient-reported outcomes for topicals are scarce. We designed a survey to record the treatment experience of patients with mild-to-moderate PSO using prescription topicals. METHODS: An online cross-sectional survey was conducted among adult patients on prescription topicals for mild-to-moderate PSO (body surface area [BSA] ≤ 10%) in the US. Data on treatment goals, changes in PSO after current treatment, satisfaction with current treatment (assessed with the Treatment Satisfaction Questionnaire for Medication [TSQM]), and treatment adherence (how often current treatment was taken as instructed) were collected. Descriptive analysis was used to evaluate outcomes. RESULTS: Of the 175 patients with mild-to-moderate PSO who completed the survey, 67.4% were female, with a median age of 55.0 years and 10.8 years since PSO diagnosis. Patients reported (medians) the use of three topicals since diagnosis, with 5 years on the current prescription topical. The top three treatment goals for current topical treatment were improvements in visible skin, 97.1%; non-skin related symptoms, 62.9%; and social/emotional well-being, 60.0%. Overall, 43.4% of patients reported 0% BSA change and 5.7% reported ≥ 75% BSA reduction. Approximately 75.0% each reported improvement in itch and pain symptoms. Embarrassment/self-consciousness because of skin symptoms persisted in 72.6% of patients. Median TSQM scores for global satisfaction, convenience, and effectiveness ranged between 58 and 72, indicating partial treatment satisfaction, except for side effects, which was high (median: 100). Approximately half of patients (49.7%) reported not being highly adherent to treatment. CONCLUSION: Contrary to their treatment goals, patients with mild-to-moderate PSO using prescription topicals reported partial effectiveness, incomplete symptom resolution, impacted emotional and social well-being, and suboptimal global satisfaction, effectiveness, adherence, and convenience. Our findings highlight several unmet needs among topical-experienced, systemic-naïve patients with mild-to-moderate PSO using prescription topicals.

Psoriasis is a chronic skin disease caused by inflammation in the body. Raised areas, called plaques, are one of the main symptoms. These plaques may be red, flaky, itchy, and/or painful. Living with psoriasis can negatively impact a person's well-being, especially when it affects visible areas such as the head, face, hands and nails, and/or sensitive areas like the genitals. Topical treatments, such as creams and lotions, are often the first therapy a doctor prescribes for psoriasis. Since these treatments are applied directly on the plaques, they can be messy and inconvenient. We conducted an online survey of adults with mild-to-moderate psoriasis, meaning that less than 10% of their body had plaques. We wanted to learn how they felt about their disease and their prescription topical treatments. Most people had psoriasis for about 10 years and had been using their current prescription topical for about 5 years. Approximately three of four people reported embarrassment/self-consciousness because of skin symptoms. Although three of four people reported some improvement in itch and pain, almost all people desired better resolution of plaques in visible areas. People also reported that: their current prescription topical did not completely resolve their symptoms, their emotional and social well-being was still suboptimal, they did not always apply their therapy as often as instructed, and they were not completely satisfied with their treatment overall. These results highlight the need for additional treatment options for people with mild-to-moderate psoriasis, particularly options that offer improved symptom control and are more convenient to use.

Using the Medication Adherence Reasons Scale (MAR-Scale) in asthma and chronic obstructive pulmonary disease to determine the extent and identify the reasons for non-adherence.

INTRODUCTION: Adherence to medications for asthma and COPD can reduce exacerbation rates, decrease healthcare costs, and improve health-related quality of life. In spite of the advantages to treatment adherence, individuals with asthma and COPD often fail to take medicines as prescribed. The objectives of this study were to determine the extent of non-adherence with asthma and COPD medicines and to describe the reasons for non-adherence in these conditions. MATERIALS AND METHODS: Data from the National Health and Wellness Study (NHWS), a self-administered, annual, internet-based cross-sectional survey of US adults from 2018 was used. NHWS participants who self-reported taking daily prescription medication(s) to treat asthma and COPD responded to the 19 reasons for non-adherence and one global item in the Medication Adherence Reasons Scale (MAR-Scale). Frequencies were used to identify the reasons for non-adherence. RESULTS: The non-adherence rate in asthma (N = 2810) was 38.4% and 28.4% in COPD (N = 1632). For both conditions, "simply missing the medicine" was the most common cause of non-adherence. Additionally, for both conditions, there was a difference between the non-adherence reason reported by more individuals and the reason for which the medicine was missed for the most number of days. CONCLUSION: The MAR-Scale identified the most frequent reasons for non-adherence with asthma and COPD in a nationwide sample in the US. The MAR-Scale can be used as a tool in a clinic setting or at a population level to measure the extent and the reasons for non-adherence.

Reduced Hypoglycemia Risk in Type 2 Diabetes Patients Switched to/Initiating Insulin Glargine 300 vs 100 U/ml: A European Real-World Study.

INTRODUCTION: Randomized controlled trials and real-world data from the USA have shown similar glycemic control with insulin glargine 300 U/ml (Gla-300) and insulin glargine 100 U/ml (Gla-100) and reduced hypoglycemia risk with Gla-300. This real-world study describes the efficacy and safety of Gla-300 and Gla-100 in patients with type 2 diabetes (T2D) in France, Spain, and Germany. METHODS: This retrospective chart review analysis used anonymized data for adults with T2D switching basal insulin analog (BIA) therapy to Gla-300 or Gla-100, or insulin-naïve patients initiating Gla-300 or Gla-100. Outcomes included change from baseline to 6-month follow-up in glycated hemoglobin A1c (A1C), total and severe hypoglycemia incidences and events, insulin dose, and reasons for BIA choice. RESULTS: Six hundred sixty-five physicians (33.8% Spain, 31.7% France, 34.4% Germany) provided chart data for patients switching to Gla-300 (n = 679) or Gla-100 (n = 429) or initiating Gla-300 (n = 719) or Gla-100 (n = 711). After adjustment for baseline characteristics, A1C reductions from baseline were similar for patients switching to Gla-300 or Gla-100 (- 0.87% vs. - 0.93%; p = 0.326) while those switched to Gla-300 vs. Gla-100 had a significantly greater mean reduction in hypoglycemic events (- 1.29 vs. - 0.81 events during 6 months; p = 0.012). Mean insulin doses after titration were 0.43 ± 0.36 and 0.40 ± 0.28 U/kg in Gla-300 and Gla-100 switchers, respectively. Factors that significantly influenced BIA choice included a lower risk of hypoglycemia (for Gla-300) and physician familiarity (for Gla-100). Outcomes for insulin-naïve patients were broadly similar to those of switchers. CONCLUSIONS: In this real-world European study, patients with T2D who switched therapy to Gla-300 or Gla-100 had improved glycemic control and reduced hypoglycemia at 6 months, with significant hypoglycemia advantages with Gla-300.

The Humanistic and Economic Burden of Migraine in Europe: A Cross-Sectional Survey in Five Countries.

INTRODUCTION: Prior studies have estimated the burden of migraine in patients suffering from ≥ 4 monthly headache days (MHDs), but the burden experienced by migraineurs suffering from one to three (1-3) MHDs is unknown. The aim of this study was to examine the incremental burden of migraine in terms of health-related quality of life (HRQoL), impairments to work and daily activities, and healthcare resource utilization (HRU) in five European countries (France, Germany, Italy, Spain, and the UK (EU5]), by comparing migraineurs with ≥ 4 MHDs and migraineurs with 1-3 MHDs. METHODS: The sample for this retrospective cross-sectional study was collected from the 2017 National Health and Wellness Survey (N = 62,000). The Short-Form 12-Item Health Survey Instrument, version-2 physical and mental component summary (PCS and MCS) scores, Short-Form 6-dimensions (SF-6D), EuroQoL 5-dimensions (EQ-5D) and EuroQoL visual analog scale (VAS) scores, impairments to work productivity and daily activities (Work Productivity and Activity Impairment [WPAI] Questionnaire) scores, and HRU were compared between migraineur groups with ≥ 4 MHDs (4-7, intermediate-frequency episodic migraine; 8-14, high-frequency episodic migraine; ≥ 15 chronic migraine) and the migraineur subgroup with 1-3 MHDs (low-frequency episodic migraine) using generalized linear modeling after adjusting for covariates. RESULTS: Data from a total of 62,000 survey respondents were examined, of whom 1323 and 1569 were considered to have 1-3 MHDs and ≥ 4 MHDs (4-7 MHDs [n = 783]; 8-14 MHDs [n = 429]; ≥ 15 MHDs [n = 357]), respectively. The adjusted HRQoL was significantly lower in the 4-7 MHDs (for MCS and SF-6D scores; p < 0.0001) and 8-14 MHDs subgroups (for MCS, SF-6D, EQ-5D, and EuroQoL VAS scores, p < 0.0001; for PCS scores, p = 0.0007) than in the 1-3 MHDs subgroup. Migraineurs with 4-7 and 8-14 MHDs reported higher activity impairment and more frequent visits to healthcare providers (all p < 0.0001) and neurologists (p = 0.0006 and p < 0.0001, respectively) compared to the 1-3 MHDs subgroup. Migraineurs with ≥ 15 MHDs had significantly lower HRQoL and increased WPAI scores and HRU than the 1-3 MHDs subgroup. CONCLUSIONS: This study provides evidence supporting the incremental burden of migraine, characterized by poorer HRQoL and increased WPAI scores and greater HRU, among migraineurs experiencing ≥ 4 MHDs compared with migraineurs experiencing 1-3 MHDs in the EU5.

The economic burden of caregiving in epilepsy: An estimate based on a survey of US caregivers.

OBJECTIVE: The burden of caregiving for persons with epilepsy (PWEs) has not been examined previously in the United States. We assessed the clinical impact and direct and indirect economic costs for caregivers of PWEs. METHODS: An internet survey of 500 caregivers of PWEs was conducted from May to July 2015 using a combination of validated instruments and questions designed specifically for this survey. Caregivers were stratified by PWE age (adult/child) and disease severity (low: 0 vs high: 1 + seizures in the prior month). Annual self-reported direct and indirect costs were reported per caregiver and extrapolated to all US caregivers. The economic burden of caregiving for PWEs was defined as the difference between costs for caregivers and the general population. RESULTS: Caregivers reported that PWEs averaged 11.4 seizures in the prior month. Eighty percent of respondents were female and the average age was 44.3. Since becoming a caregiver, many reported anxiety (52.8%), depression (41.0%), and insomnia (30.8%). Annual mean direct medical costs for caregivers of children with low vs high seizure frequency were $4344 and $10 162, respectively. Costs for caregivers of adult PWEs were $4936 and $8518. Mean indirect costs associated with caregiving for a child with low vs high seizure frequency were $20 529 and $40 137; those for caregivers of an adult were $13 981 and $28 410. The cost estimates are higher vs the general US population; annual per-person healthcare utilization costs were $2740 and productivity loss costs were $5015. When extrapolating to the US population of PWE caregivers, annual costs exceeded $62 billion vs $14 billion for the general population, resulting in a caregiver burden of nearly $48 billion. SIGNIFICANCE: The clinical and economic burden of caregivers for PWE were substantial, and greatest for those caring for children with frequent seizures. The impact on caregivers should be considered when estimating the value of interventions that control epilepsy.

Associations Between Headache-Free Days and Patient-Reported Outcomes Among Migraine Patients: A Cross-Sectional Analysis of Survey Data in Europe.

INTRODUCTION: Migraine imposes a substantial burden on patients, society, and healthcare systems. This study aimed to assess the associations between the number of headache-free days (HFDs) and health-related quality of life (HRQoL), work productivity and activity impairment (WPAI), and healthcare resource utilization (HRU) in patients with migraine in the EU5 (France, Germany, Italy, Spain, and the United Kingdom). METHODS: This retrospective cross-sectional study collected survey responses from adults aged ≥ 18 years from the 2017 National Health and Wellness Survey (N = 62,000). Migraine patients with a physician's diagnosis of migraine and who had experienced at least 4 monthly headache days in the prior month were included (N = 1569). Number of HFDs was compared with Short-Form 12-Item Survey Instrument, version 2 (SF-12v2) physical and mental component summary scores (PCS and MCS), Short-Form-6D (SF-6D) and EuroQoL-5D (EQ-5D) scores, WPAI, and HRU in migraine patients. Correlation analyses were conducted to test the bivariate associations, whereas generalized linear models were used for multivariable analyses. RESULTS: An increase of 1 HFD was associated with average increases of 0.171, 0.306, 0.003, and 0.008 points for MCS, PCS, SF-6D utility score, and EQ-5D index score, respectively (p < 0.001 for all). Extrapolating the results to a tenfold increase in monthly HFDs corresponded to clinically meaningful increases in PCS and EQ-5D scores. An increase of 1 HFD was associated with expected average decreases in absenteeism of 3.9% and presenteeism of 2.1%. Further, an increase of 1 HFD was associated with expected decreases in HCP visits and neurologist visits of 1.0% and 4.7%, respectively. CONCLUSION: An increase in the number of HFDs was associated with better health-related outcomes in patients suffering from migraine. Further, the results demonstrate a need for more effective treatments that can reduce migraine frequency and thus improve HRQoL, increase work productivity, and reduce both activity impairment and HRU. FUNDING: Novartis Pharma AG, Switzerland.

Burden of illness in adults with atopic dermatitis: Analysis of National Health and Wellness Survey data from France, Germany, Italy, Spain, and the United Kingdom.

BACKGROUND: The disease burden of atopic dermatitis (AD) in European populations is not well known. OBJECTIVE: To establish the disease burden in European adult patients with AD. METHODS: Data were from the 2016 National Health and Wellness Survey conducted in France, Germany, Italy, Spain, and the United Kingdom. Bivariate analyses were conducted on outcomes between controls without AD matched to patients with self-reported AD (both n = 1860). RESULTS: Patients with AD and a subset of patients with inadequately controlled AD (IC-AD) versus controls without AD, respectively, reported significantly higher (P < .001) 36-Item Short Form Health Survey Physical and Mental Component Summaries (PCS, MCS), and anxiety (31.9% and 51.7% vs 14.4%), depression (25.8% and 36.2% vs 12.9%), and sleep disorder (22.7% and 39.7% vs 12.6%) prevalences. Patients with IC-AD versus controls without AD reported significantly greater (P < .001) overall work (57.1% vs 23.7%) and activity impairment (51.7% vs 26.5%). In addition, 21.6% of patients with AD and 37.9% of patients with IC-AD reported ≥1 emergency department visit in the previous 6 months versus 16.5% of controls without AD, and 93.1% of patients with AD versus 84.2% of those without AD had ≥1 clinician visit (both P < .001). Of these, patients with IC-AD showed greater burden on most outcomes than patients with controlled AD. LIMITATIONS: Low response rate, possible selection bias due to survey technology availability, and patient-reported data susceptible to recall bias. CONCLUSION: Patients with AD reported significant burden on health, health-related quality of life, productivity, activities, and health care.

Respira project: Humanistic and economic burden of asthma in Brazil.

OBJECTIVE: To determine the association of asthma on health-related quality of life (QoL), productivity, and use of healthcare resources among adults in Brazil. METHODS: Data were analyzed from the 2015 Brazil National Health and Wellness Survey, a cross-sectional survey with 12,000 adult respondents. Asthma and control groups were compared with respect to health-related QoL, asthma control, work productivity, and adherence. Generalized linear models were developed to compare asthma-related associations controlling for potential confounding factors. RESULTS: Among respondents, 4.1% (n = 494) reported an asthma diagnosis; those without asthma symptoms were used as the control group (n = 11,487). Regarding asthma control, 51.2% of patients had uncontrolled asthma, 36.4% partially controlled asthma, and 12.3% were fully controlled. Short-acting ß2 agonists were the most commonly used class of drugs (38.5%). Approximately 32.4% of asthma patients were considered fully adherent to their treatment. In multivariable analyses, asthma patients presented lower health-related QoL and had more frequent visits with medical healthcare providers (6.1 versus 4.2) emergency room visits (1.0 versus 0.5), and more hospitalizations (0.4 versus 0.2), than control respondents six months prior to the study (p < 0.05). Rates of absenteeism and presenteeism varied between 11.5% and 7.4% (p < 0.05) and 30.4% and 20.9% (p < 0.001) between asthma patients and controls, respectively. CONCLUSIONS: Asthma had a negative association on health-related QoL, work productivity, and use of healthcare resources. Excessive use of short-acting ß2 agonists and poor treatment adherence reflect poor asthma control and suggest the need to implement new strategies for asthma treatment in Brazil.

Patients' perspective on the burden of migraine in Europe: a cross-sectional analysis of survey data in France, Germany, Italy, Spain, and the United Kingdom.

BACKGROUND: Migraine is a distinct neurological disease that imposes a significant burden on patients, society, and the healthcare system. This study aimed to characterize the incremental burden of migraine in individuals who suffer from ≥4 monthly headache days (MHDs) by examining health-related quality of life (HRQoL), impairments to work productivity and daily activities, and healthcare resource utilization (HRU) in the EU5 (France, Germany, Italy, Spain, United Kingdom). METHODS: This retrospective cross-sectional study used data from the 2016 National Health and Wellness Survey (NHWS; N = 80,600). Short-Form 36-Item Health Survey, version 2 (SF-36v2) physical and mental component summary scores (PCS and MCS), Short-form-6D (SF-6D), and EuroQoL (EQ-5D), impairments to work productivity and daily activities (Work Productivity and Activity Impairment Questionnaire (WPAI), and HRU were compared between migraine respondents suffering from ≥4 MHDs (n = 218) and non-migraine controls (n = 218) by propensity score matching using sociodemographic characteristics. Chi-square, T-tests, and Mann-Whitney tests were performed to determine significant differences between the groups after propensity score matching. RESULTS: HRQoL was lower in migraine individuals suffering from ≥4 MHDs compared with non-migraine controls, with reduced SF-36v2 PCS (46.00 vs 50.51) and MCS (37.69 vs 44.82), SF-6D health state utility score (0.62 vs 0.71), and EQ-5D score (0.68 vs 0.81) (for all, p < 0.001). Respondents with migraine suffering from ≥4 MHDs also reported higher levels of absenteeism from work (14.43% vs 9.46%; p = 0.001), presenteeism (35.52% vs 20.97%), overall work impairment (38.70% vs 23.27%), and activity impairment (44.17% vs 27.75%) than non-migraine controls (for all, p < 0.001). Additionally, HRU was significantly higher for individuals with ≥4 MHDs compared to their matched controls. Consistently, migraine subgroups (4-7 MHDs, 8-14 MHDs and CM) had lower HRQoL, greater overall work and activity impairment, and higher HRU compared to non-migraine controls. CONCLUSIONS: Migraine of ≥4 MHDs was associated with poorer HRQoL, greater work productivity loss, and higher HRU compared with non-migraine controls. The findings of the study suggest that an unmet need exists among individuals suffering from ≥4 MHDs in the EU5 suggesting the need for effective prophylactic treatments to lessen the humanistic and economic burden of migraine.

Assessing the Effect of Adherence on Patient-reported Outcomes and Out of Pocket Costs Among Patients With Multiple Myeloma.

BACKGROUND: The present study characterized the effect of multiple myeloma (MM) on work productivity, health care resource usage, and out of pocket costs (OOPCs) and examined the association of adherence with quality of life (QoL) and productivity loss. MATERIALS AND METHODS: The present cross-sectional study included 162 patients categorized by their 4-item Morisky Medication Adherence Scale (MMAS-4) score (4 vs. ≤ 3). Online surveys included the Work Productivity and Activity Impairment questionnaire, Functional Assessment of Cancer Therapy-Multiple Myeloma (FACT-MM), and MM-specific questions. RESULTS: On average, patients reported FACT-MM scores of 98.5 ± 29.3, absenteeism of 18.3% ± 17.8%, presenteeism of 51.8% ± 30.2%, overall work productivity impairment of 57.3% ± 31.7%, and activity impairment of 49.9% ± 29.5% in the previous 7 days. During the previous 3 months, the mean OOPCs were $709 ± $1307; prescription medications accounted for 55% of these costs. Patients attended 4.1 ± 4.6 visits to oncologists or hematologists during that time, which accounted for 45% of the OOPCs. Patients spent an average of 6.8 ± 8.3 hours at MM-related monthly appointments, and 35.2% reported frustration while at the doctor's office. Patients with an MMAS-4 score of 4 reported higher FACT-MM scores (106.9 vs. 89.2; P < .001). Patients with an MMAS-4 score of ≤ 3 reported greater activity impairment (56.5% vs. 39.8%; P = .015) and feeling overwhelmed or frustrated with rescheduling MM appointments (64.0% vs. 26.0%; P = .002). CONCLUSION: MM was associated with significant workplace and functional impairment, high OOPCs, and frequent office visits. High medication adherence was associated with better outcomes across these domains. As survival for patients with MM improves, patient QoL should be considered to enhance these outcomes.

Burden of atopic dermatitis in Japanese adults: Analysis of data from the 2013 National Health and Wellness Survey.

Atopic dermatitis is a chronic inflammatory skin disease. The objective of this study was to characterize the burden of atopic dermatitis in Japanese adult patients relative to the general population. Japanese adults (≥18 years) with a self-reported diagnosis of atopic dermatitis and adult controls without atopic dermatitis/eczema/dermatitis were identified from the 2013 Japan National Health and Wellness Survey. Atopic dermatitis patients were propensity-score matched with non-atopic dermatitis controls (1:2 ratio) on demographic variables. Patient-reported outcome data on comorbidities, mood and sleep disorders, health-related quality of life, work productivity and activity impairment, and health-care resource utilization were analyzed in atopic dermatitis patients and matched controls. A total of 638 Japanese adult patients with atopic dermatitis were identified, of whom 290 (45.5%) rated their disease as "moderate/severe" and 348 (54.5%) as "mild". The analysis cohort comprised 634 atopic dermatitis patients and 1268 matched controls. Atopic dermatitis patients reported a significantly higher prevalence of arthritis, asthma, nasal allergies/hay fever, anxiety, depression and sleep disorders compared with controls (all P < 0.001). Atopic dermatitis patients also reported a significantly poorer health-related quality of life, higher overall work and activity impairment, and higher health-care resource utilization (all P < 0.001). Self-rated disease severity was not associated with disease burden, except for a significantly higher overall work and activity impairment. In conclusion, Japanese adult patients with atopic dermatitis reported a substantial disease burden relative to adults without atopic dermatitis, suggesting an unmet need for effective strategies targeting disease management.

Treatment Dosing Patterns and Clinical Outcomes for Patients with Type 2 Diabetes Starting or Switching to Treatment with Insulin Glargine (300 Units per Milliliter) in a Real-World Setting: A Retrospective Observational Study.

INTRODUCTION: Usage patterns and effectiveness of a longer-acting formulation of insulin glargine at a strength of 300 units per milliliter (Gla-300) have not been studied in real-world clinical practice. This study evaluated differences in dosing and clinical outcomes before and after Gla-300 treatment initiation in patients with type 2 diabetes starting or switching to treatment with Gla-300 to assess whether the benefits observed in clinical trials translate into real-world settings. METHODS: This was a retrospective observational study using medical record data obtained by physician survey for patients starting treatment with insulin glargine at a strength of 100 units per milliliter (Gla-100) or Gla-300, or switching to treatment with Gla-300 from treatment with another basal insulin (BI). Differences in dosing and clinical outcomes before versus after treatment initiation or switching were examined by generalized linear mixed-effects models. RESULTS: Among insulin-naive patients starting BI treatment, no difference in the final titrated dose was observed in patients starting Gla-300 treatment versus those starting Gla-100 treatment [least-squares (LS) mean 0.43 units per kilogram vs 0.44 units per kilogram; P = 0.77]. Both groups had significant hemoglobin A1c level reductions (LS mean 1.21 percentage points for Gla-300 and 1.12 percentage points for Gla-100 ; both P < 0.001). The relative risk of hypoglycemic events after Gla-300 treatment initiation was lower than that after Gla-100 treatment initiation [0.31, 95% confidence interval (CI) 0.12-0.81; P = 0.018] at similar daily doses. The daily dose of BI was significantly lower after switching to treatment with Gla-300 from treatment with another BI (0.73 units per kilogram before switch vs 0.58 units per kilogram after switch; P = 0.02). The mean hemoglobin A1c level was significantly lower after switching than before switching (adjusted difference - 0.95 percentage points, 95% CI - 1.13 to - 0.78 percentage points ; P < 0.0001). Hypoglycemic events per patient-year were significantly lower (relative risk 0.17, 95% CI 0.11-0.26; P < 0.0001). CONCLUSIONS: Insulin-naive patients starting Gla-300 treatment had fewer hypoglycemic events, a similar hemoglobin A1c level reduction, and no difference in insulin dose versus patients starting Gla-100 treatment. Patients switching to Gla-300 treatment from treatment with other BIs had significantly lower daily doses of BI, with fewer hypoglycemic events, without compromise of hemoglobin A1c level reduction. These findings suggest Gla-300 in a real-world setting provides benefits in terms of dosing, with improved hemoglobin A1c level and hypoglycemia rates. FUNDING: Sanofi US Inc. (Bridgewater, NJ, USA).