Mapping analysis to predict SF-6D utilities from health outcomes in people with focal epilepsy.

BACKGROUND: Focal-onset seizures (FOS) are commonly experienced by people with epilepsy and have a significant impact on quality of life (QoL). This study aimed to develop a mapping algorithm to predict SF-6D values in adults with FOS for use in economic evaluations of a new treatment, cenobamate. METHODS: An online survey, including questions on disease history, SF-36, and an epilepsy-specific measure (QOLIE-31-P) was administered to people with FOS in the UK, France, Italy, Germany, and Spain. A range of regression models were fitted to SF-6D scores including direct and response mapping approaches. RESULTS: 361 individuals were included in the analysis. In the previous 28 days, the mean number of FOS experienced was 3, (range 0-43) and the mean longest period of consecutive days without experiencing a seizure was 14 days (range 1-28 days or more). Mean responses on all SF-36 dimensions were lower than general population norms. Mean SF-6D and QOLIE-31-P scores were 0.584 and 45.72, respectively. The best performing model was the ordinary least squares (OLS), with root mean squared error and mean absolute error values of 0.0977 and 0.0742, respectively. Explanatory variables which best predicted SF-6D included seizure frequency, severity, freedom, and age. CONCLUSION: People with uncontrolled FOS have poor QoL. The mapping algorithm enables the prediction of SF-6D values from clinical outcomes in people with FOS. It can be applied to outcome data from clinical trials to facilitate cost-utility analysis.

The burden of epilepsy and unmet need in people with focal seizures.

BACKGROUND: Epilepsy is one of the most common neurological conditions worldwide. As a chronic condition, epilepsy imposes a significant burden on people with epilepsy and society. We aimed to assess the burden and unmet need of individuals with epilepsy and their caregivers, focusing on focal seizures, the main type of seizure in adults and children. METHODS: A targeted evidence review of the burden of epilepsy, focusing on focal seizures, was conducted to identify articles reporting: epidemiology, mortality, morbidity, quality of life (QoL), and costs. RESULTS: Focal seizures affect up to ∼61% of people with epilepsy. They are associated with an increased risk of injury and premature death than the general population. People with epilepsy also have high comorbidity, particularly depression, anxiety, and cognitive impairments. Higher seizure frequency, adverse treatment events, and employment concerns reduce QoL. A reduction in caregivers' QoL is also often reported. Epilepsy requires long-term treatment accounting for high individual costs. Hospitalizations and antiseizure medications (ASMs) are the leading cost drivers of inpatient management and indirect costs with high unemployment rates, particularly in drug-resistant populations. Despite the advent of new treatments, a high unmet need remains unaddressed; approximately 40% of people with epilepsy are drug-resistant, further increasing the risks associated with epilepsy. CONCLUSIONS: Our findings highlight a substantial burden of illness and unmet needs in individuals with focal seizures, especially those with drug-resistant epilepsy. Suboptimal treatment options negatively impact QoL and, consequently, a sizeable economic burden indicating the need for new treatments and prioritizing this condition.

Quality of life study for caregivers of people with uncontrolled focal-onset seizures.

AIM: The aim of this study was to capture and measure the impact of caregiving for an adult with uncontrolled drug-resistant focal-onset seizures (FOS) on the caregivers' quality of life (QoL), and to quantify the costs of productivity losses associated with providing informal care in this patient population. METHODS: An online survey, which included the EQ-5D-5L, CarerQol-7D and the Work Productivity and Activity Impairment: Specific-Health Problem (WPAI:SHP) questionnaires, was administered to caregivers of individuals with uncontrolled drug-resistant FOS in the United Kingdom (UK), France, Spain, Germany, Italy, and Sweden. RESULTS: The study included 345 caregivers. Most were males, aged between 25 and 34 years old whose caring responsibilities took between 15 and 24 h per week. The caregivers' mean EQ-5D-5L score was 0.6, with 95% confidence intervals (CI) of [0.58, 0.63], whilst the mean CarerQol-7D score was 72.61 [70.46, 74.76]. Caregivers' mental health was the most substantially affected aspect of their QoL. In addition, most caregivers reported deriving some or a lot of fulfilment out of their caregiving tasks. The WPAI:SHP showed that the mean percentage of work impairment due to caregiving responsibilities was 63%, [59.75, 66.26]. The mean annualised costs of productivity losses per caregiver were estimated at €14,872 [€11,908; €17,888]. LIMITATIONS: One limitation consisted in the use of an online survey instead of a face-to-face interview. However, the medical terms were clearly explained, and examples were provided to help participants to give accurate responses. Another limitation was that the respondents self-reported as caregivers. Efforts were made to mitigate this weakness by using screener questions. CONCLUSION: This study found that providing informal care for people with uncontrolled drug-resistant FOS had a negative impact on caregivers' QoL, with mental health being affected the most. However, caregivers found their role fulfilling and had support with their caring tasks.

Systematic Review with Network Meta-Analysis: Comparative Efficacy of Biologics in the Treatment of Moderately to Severely Active Ulcerative Colitis.

BACKGROUND: Biological therapies are increasingly used to treat ulcerative colitis (UC). AIM: To compare the efficacy of biologics in adults with moderately-to-severely active UC, stratified by prior exposure to anti-tumour necrosis factor (anti-TNF) therapy. METHODS: A systematic literature review was undertaken to identify studies of biologics approved for UC. Network meta-analysis was conducted for endpoints at induction and maintenance. RESULTS: Seven studies were included in the meta-analysis of induction treatment for anti-TNF therapy-naïve patients. All biologics were more effective than placebo in inducing clinical response, clinical remission, and mucosal healing. Infliximab demonstrated a statistically significant improvement over adalimumab in clinical response (odds ratio [OR] [95% credible interval (CrI)]: 2.19 [1.35-3.55]), clinical remission (OR [95% CrI]: 2.81 [1.49-5.49]), and mucosal healing (OR [95% CrI]: 2.23 [1.21-4.14]); there were no other significant differences between biologics for induction efficacy. Five studies were included in the meta-analysis of maintenance treatment, two studies rerandomised responder patients at end of induction, and three followed the same patients 'straight through'. To account for design differences, the number of responders at end of induction was assumed to be equivalent to the number rerandomised. Vedolizumab showed significantly different durable clinical response from comparators (OR [95% CrI] infliximab 3.18 [1.14-9.20], golimumab 2.33 [1.04-5.41], and adalimumab 3.96 [1.67-9.84]). In anti-TNF therapy-experienced patients, only vedolizumab and adalimumab could be compared. At induction, no significant differences in efficacy were seen. During maintenance, vedolizumab showed significantly improved rates of mucosal healing versus adalimumab (OR [95% CrI]: 6.72 [1.36-41.0]). CONCLUSIONS: This study expands the understanding of comparative efficacies of biologic treatments for UC, encompassing outcomes and populations not previously studied. All biologic treatments were effective for UC during induction. Vedolizumab demonstrated possible clinical benefits in the maintenance setting versus all comparators, irrespective of prior anti-TNF exposure and after adjusting for differences in study design.

Direct economic burden of chronic hepatitis C virus in a United States managed care population.

GOALS AND BACKGROUND: To estimate all-cause and disease-related resource utilization and costs among managed care enrollees with chronic hepatitis C virus (HCV). STUDY: A large United States claims database was analyzed (1/1/2002 to 12/31/2006). Inclusion criteria were: diagnosis of chronic HCV; no hepatitis B diagnoses; ≥6 and ≥12 months of continuous plan enrollment prediagnosis and postdiagnosis, respectively. Use and costs of medical services and prescription drugs over a 12-month period postdiagnosis were evaluated. Outcomes were assessed in controls without HCV matched (1:1) on age, sex, and plan enrollment. All cost estimates were generated using multivariate generalized linear models to adjust for additional covariates and skewness common in health care cost data. RESULTS: Of the 20,662 patients who met all inclusion criteria, mean age was 49 years; 61% were male. Adjusted all-cause costs were $20,961 per HCV patient, compared with $5451 per control (P<0.0001). Hospitalization occurred in 24% of HCV patients compared with 7% of controls (P<0.0001). Mean inpatient costs were $5892 and $1159 per patient, respectively (P<0.0001). Patients with HCV had higher prescription costs compared with controls ($6191 vs. $1315; P<0.0001). At $6864 per patient, disease-related costs were nearly one-third of all costs in patients with HCV, which exceeded all-cause costs among controls by 26% (P<0.0001). CONCLUSIONS: Chronic HCV is a costly disease to managed care organizations. Disease-related costs in HCV exceed all-cause costs in demographically matched controls. Increased efforts in HCV screening and early treatment, particularly before progression to liver cirrhosis, may lead to long-term cost savings in HCV management for managed care systems.

Treatment patterns and adherence among patients with chronic hepatitis C virus in a US managed care population.

OBJECTIVE: The purpose of this study was to document real-world treatment patterns, medication adherence, and the impact of adherence on disease-specific and all-cause health-care costs among chronic hepatitis C virus (HCV) patients in a US managed care population. METHODS: Commercial insurance claims data between January 1, 2002 and December 31, 2006 from the Ingenix Impact (formerly Integrated Health Care Information Services) database were retrospectively analyzed. Chronic HCV patients with one or more prescriptions for an HCV-specific treatment within 6 months before or at any time after their first observed diagnosis of chronic HCV were selected. Prescribing patterns, treatment cost, and duration of treatment were assessed over the entire therapy period. Medication adherence rates and the relationship between adherence and health-care costs were assessed over the 24-week period after treatment initiation. The results were stratified by key clinical characteristics such as genotype, sustained virologic attainment, and disease severity. RESULTS: Results showed that peginterferon and ribavirin combination regimens were the most common treatments for chronic HCV. The patients underwent treatment for approximately 30-32 weeks on average, and treatment costs were over $20,000 per patient. Adherence to medication was suboptimal, especially among patients with severe disease. Adherent patients had higher pharmacy costs but significantly lower total costs when pharmacy was excluded. CONCLUSIONS: New and improved treatments that promote better adherence and impose a lower cost burden on patients and payers are needed.