Burden of disease in pediatric narcolepsy: a claims-based analysis of health care utilization, costs, and comorbidities.

BACKGROUND: This study analyzed a privately insured pediatric population with and without narcolepsy to determine the impact of pediatric narcolepsy on comorbidities, health care utilization, and cost. Additional analyses compared narcolepsy type 1 and type 2. METHODS: This retrospective cross-sectional study identified US patients with narcolepsy <18 years of age with ≥2 claims with a diagnosis code of narcolepsy using Truven MarketScan® data 2011 to 2015. Patients were matched to controls without narcolepsy. Comorbid conditions, health care utilization, and costs were measured by calendar year. P values are nominal, and no adjustments for multiplicity or multiple comparisons were made. RESULTS: A total of 1427 pediatric patients with narcolepsy were identified and matched with 4281 controls from 2011 to 2015. Patients with narcolepsy had more comorbid conditions (mean 5.8 vs 2.4, nominal P < 0.001). Respiratory diseases and mood disorders were more common in patients with narcolepsy than controls (57% vs 32% and 56% vs 14%, respectively; both nominal P < 0.001). Compared to controls, patients with narcolepsy underwent more diagnostic tests (electroencephalogram, EEG [0.13 vs 0.0053]) and brain computed tomography, CT/magnetic resonance imaging, MRI (0.26 vs 0.022; both nominal P < 0.001). Mean annual inpatient days (0.71 vs 0.15), emergency department visits (0.51 vs 0.15), and outpatient office visits (8.6 vs 2.3) were higher for patients with narcolepsy than controls (all nominal P < 0.001). Annual mean health care costs were higher for patients with narcolepsy versus controls ($15,797 vs $2449, nominal P < 0.001). CONCLUSION: Pediatric patients with narcolepsy had greater comorbidity, higher health care utilization, and higher costs than patients without narcolepsy.

Long-term health and socioeconomic consequences of childhood and adolescent-onset of narcolepsy.

OBJECTIVES: There is limited information about the long-term consequences of childhood- and adolescent-onset narcolepsy on educational and social factors. Here, we estimate the long-term socioeconomic consequences and health care costs of narcolepsy. METHODS: The prospective cohort study included Danish individuals with narcolepsy onset in childhood or adolescence, diagnosed between 1994 and 2015. Health care costs and socioeconomic data were obtained from nationwide administrative and health registers. One hundred seventy-one patients were compared with 680 controls (mean index age, 15.2 years; SD, 3.4 years) matched for age, gender, and other sociodemographic characteristics. RESULTS: Comparing the narcolepsy patient and control groups at age 20 years we found: (1) no differences in parental educational level; (2) patients had a significantly lower educational level than controls; (3) patients had significantly lower grade-point averages; (4) patients had a lower employment rate and lower-income, even when transfer payments were considered; and (5) patients' initial health care costs were higher. Patients had a higher mortality rate than controls, although the difference was not statistically significant. CONCLUSION: Narcolepsy is associated with a significant influence on educational level, grading, social outcome, and welfare consequences. The development of narcolepsy is independent of parental social level.

Reducing the Clinical and Socioeconomic Burden of Narcolepsy by Earlier Diagnosis and Effective Treatment.

The burden of narcolepsy is likely the result of 2 main aspects: the clinical difficulties and disability incurred as a direct effect of the disorder and the socioeconomic burden. The clinical burden includes the symptoms, diagnosis, comorbidities, treatment, and even mortality that can be associated with narcolepsy. Lifelong therapy is necessary for these patients. Effective treatment results in long-term benefits from both patient and societal perspectives by improving clinical outcomes, potentially enabling improved education and increased employment and work productivity, and quality of life. Thus, reducing the time to appropriate management results in improved outcomes in these patients.

The Humanistic and Economic Burden of Narcolepsy.

STUDY OBJECTIVES: To evaluate the burden of narcolepsy--with respect to psychiatric comorbidities, Health-Related Quality of Life (HRQoL), direct costs for healthcare resource utilization, and indirect costs for reported work loss-through comparison of patients to matched controls. METHODS: This analysis was conducted on data from the 2011, 2012, and 2013 US National Health and Wellness Survey (NHWS; 2011 NHWS n = 75,000, 2012 NHWS n = 71,157, and 2013 NHWS n = 75,000). Patients who reported a narcolepsy diagnosis (n = 437) were matched 1:2 with controls (n = 874) on age, sex, race/ethnicity, marital status, education, household income, body mass index, smoking status, alcohol use, exercise, and physical comorbidity. Chi-square tests and one-way analyses of variance were used to assess whether the narcolepsy and control groups differed on psychiatric comorbidities, HRQoL, labor force participation, work productivity, and healthcare resource utilization. RESULTS: Patients with narcolepsy, in comparison to matched controls, reported substantially (two to four times) greater psychiatric comorbidity, HRQoL impairment, prevalence of long-term disability, absenteeism, and presenteeism, and greater resource use in the past 6 mo as indicated by higher mean number of hospitalizations, emergency department visits, traditional healthcare professional visits, neurologist visits, and psychiatrist visits (each p < 0.05). CONCLUSIONS: These population-based data suggest that a narcolepsy diagnosis is associated with substantial adverse impact on mental health, HRQoL, and key economic burdens that include work impairment, resource use, and both direct and indirect costs. Although this study is cross-sectional, the results highlight the magnitude of the potential opportunity to improve mental health, lower costs, and augment work-related productivity through effective assessment and treatment of narcolepsy.

The Burden of Narcolepsy Disease (BOND) study: health-care utilization and cost findings.

OBJECTIVES: The aim of this study was to characterize health-care utilization, costs, and productivity in a large population of patients diagnosed with narcolepsy in the United States. METHODS: This retrospective, observational study using data from the Truven Health Analytics MarketScan Research Databases assessed 5 years of claims data (2006-2010) to compare health-care utilization patterns, productivity, and associated costs among narcolepsy patients (identified by International Classification of Diseases, Ninth Revision (ICD9) narcolepsy diagnosis codes) versus matched controls. A total of 9312 narcolepsy patients (>18 years of age, continuously insured between 2006 and 2010) and 46,559 matched controls were identified. RESULTS: Compared with controls, narcolepsy subjects had approximately twofold higher annual rates of inpatient admissions (0.15 vs. 0.08), emergency department (ED) visits w/o admission (0.34 vs. 0.17), hospital outpatient (OP) visits (2.8 vs. 1.4), other OP services (7.0 vs. 3.2), and physician visits (11.1 vs. 5.6; all p<0.0001). The rate of total annual drug transactions was doubled in narcolepsy versus controls (26.4 vs. 13.3; p<0.0001), including a 337% and 72% higher usage rate of narcolepsy drugs and non-narcolepsy drugs, respectively (both p<0.0001). Mean yearly costs were significantly higher in narcolepsy compared with controls for medical services ($8346 vs. $4147; p<0.0001) and drugs ($3356 vs. $1114; p<0.0001). CONCLUSIONS: Narcolepsy was found to be associated with substantial personal and economic burdens, as indicated by significantly higher rates of health-care utilization and medical costs in this large US group of narcolepsy patients.

The burden of narcolepsy with cataplexy: how disease history and clinical features influence socio-economic outcomes.

OBJECTIVE: To investigate in narcolepsy with cataplexy (NC) patients of working age (18-65 years) the influence of age at onset, age at diagnosis and clinical features on socio-occupational conditions, disease-related economic burden, and quality of life. METHODS: One hundred consecutive patients underwent a semistructured interview on socio-occupational aspects, NC-related direct and indirect costs, and NC history. Questionnaires were used to evaluate excessive daytime sleepiness, cataplexy, depressive symptoms, and quality of life. RESULTS: NC patients (51 males, mean age 37±11.5 years) had educational and occupational levels similar to those of the Italian population of the same age range, but married less often, especially if NC onset occurred at a young age. Total annual NC-related costs were €9814 ± 10,372 per patient. Multivariate analyses showed that patients with NC onset before the age of 30 years had a higher educational level, married less frequently, and were less frequently unemployed or inactive (retired, housewife). Patients diagnosed before the age of 30 years were less frequently unemployed or inactive, had fewer work changes, and had a better general health perception. Irresistible sleepiness was associated with work absences and higher indirect costs. Depressive symptoms were strongly associated with lower quality-of-life scores. CONCLUSIONS: NC age at onset and at diagnosis modulates the disease-related burden. A diagnosis at a young age could improve patients' occupational prognosis, and their general health perception.

Health, social, and economic consequences of narcolepsy: a controlled national study evaluating the societal effect on patients and their partners.

OBJECTIVE: Despite the fact that narcolepsy is a chronic disorder affecting younger people, there is insufficient information about its societal burden, time course, and familiar effect. We aimed to estimate the factual direct and indirect costs of narcolepsy patients and their families in a national sample using a controlled study design. PATIENTS/METHODS: Using records from the Danish National Patient Registry (1997-2009), all 816 narcolepsy patients and their partners were identified and compared with 3254 randomly chosen controls matched for age, gender, geographic area, and civil status. Direct and indirect costs, including frequencies of primary and secondary sector contacts' and procedures, medication, labor supply, and social transfer payments were extracted from the national databases. RESULTS: Fewer patients (46.7%) than controls (51.4%) were married or cohabiting. Patients with narcolepsy had significantly higher rates of health-related contact, medication use, and a higher socioeconomic cost. Furthermore, they had lower employment rates, and those in employment had a lower income level than control subjects. Partners presented higher public health insurance and public transfers and lower income from employment. In all, the annual mean excess health-related cost, including social transfers, was €9572 for patients with narcolepsy and €3606 for their partners (both p<0.001). Patient consequences could be identified up to 11 years before first diagnosis and became more pronounced as the disease advanced. CONCLUSION: Narcolepsy causes socioeconomic consequences, not only for patients, but also for their partners, which is present years prior to disease diagnosis, confirming a diagnostic delay.

The economic consequences of narcolepsy.

BACKGROUND: Narcolepsy is a chronic neurodegenerative disorder with a typical onset in childhood or early adulthood. Narcolepsy may have serious negative effects on health-, social-, education-, and work-related issues for people with narcolepsy and for their families. The disease may, thus, present a significant socioeconomic burden, but no studies to date have addressed the indirect and direct costs of narcolepsy. METHODS: Using records from the Danish National Patient Registry (1998-2005), we identified 459 Danish patients with the diagnosis of narcolepsy. Using a ratio of 1 patient record to 4 control subjects' records, we then compared the information of patients with narcolepsy with that of 1836 records from age- and sex-matched, randomly chosen citizens in the Danish Civil Registration System Statistics. We calculated the annual direct and indirect health costs, including labor supply and social transfer payments (which include income derived from state coffers, such as subsistence allowances, pensions, social security, social assistance, public personal support for education, etc.). Direct costs included frequencies and costs of hospitalizations and weighted outpatient use, according to diagnosis-related groups, and specific outpatient costs based on data from The Danish Ministry of Health. The use of and costs of drugs were based on data from the Danish Medicines Agency. The frequencies and costs from primary sectors were based on data from The National Health Security. Indirect costs were based on income data derived from data from the Coherent Social Statistics. RESULTS: Patients with narcolepsy had significantly higher rates of health-related contact and medication use and higher expenses, as compared with control subjects. They also had higher unemployment rates. The income level of patients with narcolepsy who were employed was lower than that of employed control subjects. The annual total direct and indirect costs were euro 11,654 (euro = Eurodollars) for patients with narcolepsy and euro 1430 for control subjects (p < 0.001), corresponding to an annual mean excess health-related cost of euro 10,223 for each patient with narcolepsy. In addition, the patients with narcolepsy received an annual social transfer income of euro 2588. CONCLUSION: The study confirms that narcolepsy has major socioeconomic consequences for the individual patient and for society. Early diagnosis and treatment could potentially reduce disease burden, which would have a significant socioeconomic impact.

The socioeconomic impact of narcolepsy.

STUDY OBJECTIVE: To evaluate the economic burden of patients suffering from narcolepsy. DESIGN: Cross-sectional study. PARTICIPANTS: 75 narcoleptic patients diagnosed at the Hephata Klinik, Germany, who met the International Classification of Sleep Disorders criteria for narcolepsy. MEASUREMENTS: A standardized telephone interview was used to inquire about the disease and its burden. In addition, health-related quality of life scales (SF-36 and EQ-5D) were mailed to the patients. Complete data were available for 75 patients. Direct and indirect costs were calculated from the societal perspective. All costs (mean +/- SD) were calculated in 2002 Euros (Euros). RESULTS: Total annual costs were Euros 14,790 +/- 16,180 (US-dollars 15,410) per patient. Direct costs were Euros 3,180 +/- 3,540 (US-dollars 3,310), which consisted of hospital costs (Euros 1,210 +/- 2,210; US-dollars 1,260), drug costs (narcolepsy medication: Euros 1,020 +/- 1,420; US-dollars 1,060), ambulatory care (Euros 90 +/- 90; US-dollars 40), and ambulatory diagnostics (Euros 20 +/- 30; US-dollars 20). More than 50% of the drug costs were due to the newer wake-promoting drugs. Total annual indirect costs amounted to Euros 11,390 +/- 15,740 (US-dollars 11,860) per patient and were mainly attributable to early retirement due to the disease. Narcolepsy was listed as being the cause of unemployment by 32 patients. In patients with sleep attacks, there was a significant correlation between early retirement and higher indirect costs compared to patients without these attacks. CONCLUSIONS: Narcolepsy causes a high socioeconomic burden, which is comparable to other chronic neurologic diseases, Indirect costs are considerably higher than direct costs. Measures should be taken to increase public awareness of this disease. Appropriate treatment must be provided in order to increase quality of life and to assist patients continuing in working life.

[Excessive daytime somnolence and its psychosocial sequelae]. / Nadmerná denní spavost a její psychosociální dusledky.

Excessive daytime sleepiness arises frequently as a consequence of insufficient or shortened nocturnal sleep, or as a result of poor sleeps hygiene. One of the most common dyssomnias with imperative sleepiness is narcolepsy. Narcolepsy is a chronic disease that greatly affects quality of life. The present study of 57 patients shows that the negative assessment is partly due to the subjective views exhibited by the narcoleptic personality prone to underestimation, negative self-image and depressive disposition. The personality traits may be due to an adaptation reaction to the disease but also to the biological predisposition. Comparison with population data for our Republic brought no evidence of progress at education or family life being adversely affected by narcolepsy, however, decreased assertion in employment and limitation in self-realization in free time was an explicit consequence of the disease. All 57 patients reported excessive daytime sleepiness as the most discomforting symptom that in 40% of them was responsible for career curtailment in a productive age and for living on partial or full disability.

Benefits and risks of pharmacotherapy for narcolepsy.

Narcolepsy is a life-long central nervous system (CNS) syndrome characterised by excessive sleepiness, cataplexy, sleep paralysis, hypnagogic hallucinations and disturbed night-time sleep. Unsuccessfully treated narcolepsy confers increased risks on patients and on society due to the patient's increased chance of becoming involved in vehicle crashes and workplace mishaps. The syndrome may be diagnosed by a clinical history positive for cataplexy and excessive daytime sleepiness and negative for other more common sleep disorders such as sleep apnoea and sleep deprivation. Night-time polysomnography and multiple sleep latency testing are helpful in differentiating narcolepsy from other sleep problems. Recent data from canine, murine, and human forms of narcolepsy indicate that genetically or developmentally mediated deficits in the hypocretin neurotransmitter system may cause some, but not all, forms of narcolepsy. Pharmacotherapy for narcolepsy is required to control symptoms and involves the use of CNS stimulants or modafinil to control sleepiness and antidepressant medications or sodium oxybate to control cataplexy. Modafinil and sodium oxybate have been developed and approved specifically for the indication of narcolepsy based on large, double-blind, placebo-controlled, parallel group efficacy and safety studies. The efficacy of drugs in the treatment of narcolepsy is variable from patient to patient and usually associated with adverse effects that can limit patient compliance and, therefore, symptom control. Nevertheless, the benefits of pharmacotherapy are judged to outweigh the risks to the patient. The favourable benefit-risk ratio of pharmacotherapy is greater if one considers the reduced risk to society of vehicle crashes and workplace mishaps that might be precipitated by attentional lapses or sleep attacks in the untreated or under-treated patient with narcolepsy.