Cost of illness associated with Niemann-Pick disease type C in the UK.

OBJECTIVE: Niemann-Pick disease type C (NP-C) is a rare and devastating genetic disorder characterised by a range of progressive neurological symptoms, which imposes a burden on patients, family members, the healthcare system and society overall. The objective of this study was to assess direct and indirect costs associated with NP-C in the UK. METHODS: This was a non-interventional, retrospective, cross-sectional cohort study based on responses from patients and/or their carers/guardians recruited from a UK NP-C database. Resource use and direct medical, direct non-medical and indirect costs were evaluated using data collected via postal survey in October 2007, which included a Medical Resource Use questionnaire. Total annual costs per patient were estimated. RESULTS: In total, 18 Medical Resource Use questionnaires (29% response rate) were received and analysed. The mean total annual cost (SD) of NP-C per patient was 39,168 pounds (50,315 pounds); 46% were direct medical costs, to which home visits and residential care contributed 68% and 15%, respectively. Direct non-medical costs accounted for 24% of the average annual cost per patient, mainly due to specialist education, and indirect costs 30%. If only direct medical costs were considered, the mean annual cost (SD) per patient was reduced to 18,012 pounds (46,536 pounds). CONCLUSIONS: The direct annual per-patient cost of NP-C illness in 2007 appears moderate when compared with other rare and severely disabling diseases. However, cost estimates may be conservative, since findings are limited by a small sample size, low survey response rate and potential recall bias. As demonstrated by this study, a substantial proportion of the cost is shifted from the healthcare system to the patient, family and non-medical providers. These findings highlight the need for treatments that can slow or stop disease progression in NP-C.

Self-reported healthcare decision-makers' attitudes towards economic evaluations of medical technologies.

BACKGROUND: Increasing costs have generated concern among governments and healthcare providers who have realized the need for cost containment measures and more efficient resource utilization. Health economics is one potential source of information that can make healthcare more efficient. SCOPE: This review article summarizes the published literature on self-reported attitudes of healthcare decision-makers towards economic evaluations of medical technologies and examines the extent to which economic evaluations are used in health policy decisions. METHODS: A systematic literature review of published English language studies was conducted using MEDLINE, EMBASE, and HEED from January 1995 to December 2007. FINDINGS: Fifty-five articles investigated the use of economic evaluations on three levels of decision-making: central, local, and physician level. Results indicate the use of economic evaluation information increased from limited/minor to moderate use. The influence of economic evaluations increased with the level of centralization of healthcare system. Barriers to use health economics research varied across levels and included health economics research-related barriers such as timely availability, lack of credibility, insufficient methodological quality and decision-context-related barriers including limited decision makers' knowledge, inflexibility in healthcare budgets and variability among healthcare organizations. CONCLUSIONS: For consistent policy-making it is important that similar recommendations for cost-effective interventions and programs are developed at all levels and that implementation is promoted by incorporating the appropriate incentives in healthcare provision.

Uncertainty in decision-making: value of additional information in the cost-effectiveness of lifestyle intervention in overweight and obese people.

OBJECTIVES: To quantify the uncertainty in the cost-effectiveness of lifestyle intervention versus standard care in overweight and obese people in Switzerland and to determine if further research is necessary based on current information. There has been an increasing interest in using value of information analysis in medical decision-making to quantify the uncertainty in decision-making, and to identify the need for further research. METHODS: Value of information analysis was applied on a probabilistic cost-effectiveness model to evaluate the uncertainty by calculating the patient expected value of perfect information (EVPI), population EVPI, and partial EVPI. The costs were expressed in Swiss Francs (CHF), price year 2006. RESULTS: Overall, the EVPI was higher in overweight than in obese people. The maximum population EVPI was CHF 6.8 million in overweight people and CHF 3.2 million in moderate obese people, representing the upper limit on costs associated with decision uncertainty. The partial EVPI estimated a higher uncertainty in the model parameters such as utilities, body mass index, cardiovascular risk factors, and systolic blood pressure in overweight and moderate obese subjects. CONCLUSIONS: The EVPI analysis indicates that there is some uncertainty regarding the choice between lifestyle intervention and standard care. The parameter EVPI suggests that if further research is commissioned, this should focus on the effectiveness of lifestyle intervention on cardiovascular risk factors and utilities.

Prevention and treatment of obesity with lifestyle interventions: review and meta-analysis.

OBJECTIVE: To assess the mid- to long-term effectiveness of lifestyle interventions in the prevention and treatment of obesity. METHODS: A systematic literature review with meta-analysis was performed. Electronic databases, reference lists, books and reports covering topic of obesity were searched. The included studies were randomized clinical trials of lifestyle interventions in overweight and obese subjects that had a minimum observation period of one year. Outcomes evaluated were measurements of body weight, body mass index, waist circumference, systolic and diastolic blood pressure, blood lipids: total cholesterol, low density lipoprotein, high density lipoprotein, triglyceride, blood glucose control: two-hour plasma glucose, fasting plasma glucose, and glycosylated haemoglobin. RESULTS: Thirteen studies have been selected in the prevention and seventeen in the treatment of obesity. Compared with standard care, lifestyle intervention reduced significantly body weight, body mass index, waist circumference, blood pressure, blood lipids and blood glucose in overweight and obese people. The favorable effects were maintained up to three years. CONCLUSIONS: Lifestyle interventions were efficacious in the mid- to long-term prevention and treatment of obesity leading to a significant reduction in body weight and cardiovascular risk factors.

Modelling the lifetime costs and health effects of lifestyle intervention in the prevention and treatment of obesity in Switzerland.

AIM: To quantify the lifetime health and economic consequences of preventing and treating obesity with lifestyle intervention in Switzerland. METHODS: A Markov model was developed comparing lifestyle intervention and standard care in overweight and obese people. Changes in weight and cardiovascular risk factors over time were modeled from reduction in body mass index, systolic blood pressure, total cholesterol and high density lipoprotein in three-year active treatment period. A probabilistic sensitivity analysis was performed. Three groups of people were followed in the analysis: overweight, borderline and moderate obese. The cost-effectiveness of interventions was compared using incremental cost-effectiveness ratio. RESULTS: Lifestyle intervention resulted in increased survival duration and quality of life over lifetime. Compared with standard care, the average incremental cost of lifestyle intervention was lower in borderline and obese and higher in overweight. Lifestyle intervention dominated standard care in borderline female age 35 to 55 years, borderline male age 25 to 60 years, obese female age 45 years and obese male age 55 years. CONCLUSION: Our economic analysis suggests that lifestyle intervention is cost-effective in the long-term prevention and treatment of obesity.

Validation of electronic data capture of the Irritable Bowel Syndrome--Quality of Life Measure, the Work Productivity and Activity Impairment Questionnaire for Irritable Bowel Syndrome and the EuroQol.

OBJECTIVES: To assess the comparability, reliability, and subject acceptability of electronic data capture (EDC) versions of Irritable Bowel Syndrome-Quality of Life (IBS-QOL), EuroQoL (EQ-5D) and Work Productivity and Activity Impairment (WPAI:IBS) instruments. METHODS: Comparability of EDC and paper questionnaires was evaluated in 72 subjects with IBS who completed a baseline EDC or paper questionnaire, a crossover questionnaire 24 hours later, and a retest of the crossover version at 1 week. The EDC version was presented on a hand-held device. Comparability was assessed using paired t-test statistics, intraclass correlation coefficients (ICC) and tests for internal consistency (Cronbach's alpha). RESULTS: No significant differences were found between scores obtained by paper questionnaire and EDC at the baseline and crossover assessments. ICCs between baseline and crossover assessments ranged from 0.83 to 0.96 for the IBS-QOL scores, 0.82 to 0.96 for the WPAI:IBS scores, and 0.77 to 0.82 for the EQ-5D. Internal consistency was comparable for the two data collection methods for the IBS-QOL overall score (0.96) and subscales and the EQ-5D Index (0.70 vs. 0.74). Retest statistics (ICC) were generally comparable between the EDC and paper versions for all scores. Ease of use was comparable for the two modes of administration, but more patients preferred EDC (47.2%) than the paper questionnaire (23.6%). CONCLUSIONS: EDC versions of the IBS-QOL, EQ-5D, and WPAI:IBS are comparable to paper questionnaires in internal consistency and test-retest reliability, and have greater patient acceptability.