Economic evaluation of interventions for the treatment of asthma in children: A systematic review.

OBJECTIVES: This systematic review aimed to identify and critique full economic evaluations (EEs) of childhood asthma treatments with the intention to guide researchers and commissioners of pediatric asthma services toward potentially cost-effective strategies. METHODS: "MEDLINE," "Embase," "EconLit," "NHS EED," and "CEA" databases were searched to identify relevant EEs published between 2005 and May 2017. Quality of included studies was assessed with a published checklist. RESULTS: Eighteen studies were identified and comprised one cost-benefit analysis, 11 cost-effectiveness analyses, one cost-minimization analysis, and six cost-utility analyses. Treatments included pharmaceutical (n = 11) and non-pharmaceutical (n = 7) interventions. Fourteen studies identified cost-effective strategies. The quality of the studies varied and there were uncertainties due to the methods and relevance of data used. CONCLUSION: Good-quality economic evaluation studies of pediatric asthma treatments are lacking. EE of new technologies adapted to local settings is recommended and can result in cost savings.

Randomised controlled trial to assess the impact of a lifestyle intervention (ActWELL) in women invited to NHS breast screening.

INTRODUCTION: In Scotland, the incidence of breast cancer is predicted to rise significantly in the next few decades and while there are measures to support reductions in morbidity and mortality, the breast cancer community is currently exploring preventative opportunities including supporting weight management programmes in postmenopausal women. This study aims to assess the effectiveness and cost-effectiveness of a theory-based, community delivered, minimal contact, weight management (diet, physical activity and behaviour change techniques) programme (ActWELL) in women with a body mass index (BMI) >25 kg/m2 attending routine breast cancer screening appointments. METHODS AND ANALYSIS: The study will be a four-centre, 1:1 parallel group randomised controlled trial of a 12-month weight management intervention initiated in breast cancer screening centres, delivered by trained Breast Cancer Now lifestyle coaches in community settings. The intervention programme involves two intervention meetings with coaches plus (up to) nine telephone contacts over 12 months. The programme will focus on personalised diet (including alcoholic and sugary drinks) and physical activity habits. Behaviour change techniques include self-monitoring, goal setting, implementation intentions, action and coping plans. The study has a sample size of 414 women with a BMI >25 kg/m2 attending routine National Health Service breast cancer screening appointments. Measures will be taken at baseline, 12 weeks and at 12-month follow-up, complemented by qualitative interviews exploring perceived acceptability and impact on habitual behaviours. The two co-primary outcomes are mean change in measured body weight and change in physical activity between groups to 12 months. Secondary outcomes are changes in eating habits, alcohol intake, sedentary time, quality of life, waist circumference, lipid, haemoglobin A1c and insulin profiles, blood pressure and cost-effectiveness of the intervention. ETHICS AND DISSEMINATION: The protocol has been approved by East of Scotland Research Ethics Committee (17/ES/0073). All participants provide written informed consent. Dissemination will be through peer-reviewed publication and conference presentations. TRIAL REGISTRATION NUMBER: ISRCTN11057518; Pre-results.

Ruxolitinib for the treatment of myelofibrosis: a NICE single technology appraisal.

The National Institute for Health and Care Excellence (NICE) invited the manufacturer of ruxolitinib (Novartis) to submit clinical and cost-effectiveness evidence for ruxolitinib within its licensed indication (the treatment of disease-related splenomegaly or symptoms in adult patients with myelofibrosis), according to the Institute's Single Technology Appraisal process. The Centre for Reviews and Dissemination and Centre for Health Economics at the University of York were commissioned to act as the independent Evidence Review Group (ERG). This article provides a description of the company submission, the ERG review and the resulting NICE guidance TA289 issued in June 2013. The ERG critically reviewed the evidence presented in the manufacturer's submission and identified areas requiring clarification, for which the manufacturer provided additional evidence. The main clinical effectiveness data were derived from two phase III, multicentre, randomised controlled trials (RCTs): Controlled myelofibrosis study with oral JAK inhibitor treatment (COMFORT)-II compared ruxolitinib with best available therapy (BAT), and COMFORT-I compared ruxolitinib with placebo. These RCTs demonstrated that ruxolitinib confers significant benefits in terms of spleen size reduction and improvement in symptom burden. In the COMFORT-II trial, a reduction in spleen volume of ≥35 % was achieved in 28 % of ruxolitinib-treated patients compared with 0 % of patients in the BAT group (p < 0.001) at 48 weeks, and there was a mean change in spleen volume of -30.1 versus +7.3 % (p < 0.001). Ruxolitinib also provided significant improvements in myelofibrosis-associated symptoms and health-related quality-of-life compared with BAT and placebo. The ERG concluded that ruxolitinib appears to reduce splenomegaly and its associated symptoms, but that there was considerable uncertainty surrounding the manufacturer's cost-effectiveness estimates due to limitations in the manufacturer's model. The manufacturer's model did not allow for disease progression, did not accurately capture symptomatic relief, had several implausible or unjustified assumptions, and there were several parameter choices that the ERG found sub-optimal. ERG sensitivity analyses found that nearly all plausible adjustments to the model reduced the cost effectiveness of ruxolitinib. It is very likely that the base-case incremental cost-effectiveness ratio of £73,980/quality-adjusted life-year presented by the manufacturer represents a best-case scenario. The NICE Appraisal Committee concluded that ruxolitinib was clinically effective, but could not be considered a cost effective use of National Health Service (NHS) resources for treating disease-related splenomegaly or symptoms in adults with myelofibrosis. Ruxolitinib is not recommended for the treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis (also known as chronic idiopathic myelofibrosis), post-polycythaemia vera myelofibrosis and post-essential thrombocythaemia myelofibrosis in NICE TA289.

Modeling the impact of screening policy and screening compliance on incidence and mortality of cervical cancer in the post-HPV vaccination era.

BACKGROUND: In Norway, pap smear screening target women aged 25-69 years on a triennial basis. The introduction of human papillomavirus (HPV) mass immunization in 2009 raises questions regarding the cost-saving future changes to current screening strategies. METHODS: We calibrated a dynamic HPV transmission model to Norwegian data and assessed the impact of changing screening 20 or 30 years after vaccine introduction, assuming 60 or 90% vaccination coverage. Screening compliance among vaccinated women was assumed at 80 or 50%. Strategies considered: (i) 5-yearly screening of women of 25-69 years, (ii) 3-yearly screening of women of 30-69 years and (iii) 3-yearly screening of women of 25-59 years. RESULTS: Greatest health gains were accomplished by ensuring a high vaccine uptake. In 2060, cervical cancer incidence was reduced by an estimated 36-57% compared with that of no vaccination. Stopping screening at the age of 60 years, excluding opportunistic screening, increased cervical cancer incidence by 3% (2060) compared with maintaining the current screening strategy, resulting in 1.0-2.4% extra cancers (2010-2060). The 5-yearly screening strategy elevated cervical cancer incidence by 30% resulting in 4.7-11.3% additional cancers. CONCLUSION: High vaccine uptake in the years to come is of primary concern. Screening of young women <30 years remains important, even under the conditions of high vaccine coverage.

Cost comparison of insulin glargine with insulin detemir in a basal-bolus regime with mealtime insulin aspart in type 2 diabetes in Germany.

OBJECTIVE: To compare the treatment costs of insulin glargine (IG; Lantus) to detemir (ID; Levemir), both combined with bolus insulin aspart (NovoRapid) in type 2 diabetes (T2D) in Germany. METHODS: Cost comparison was based on data of a 1-year randomised controlled trial. IG was administered once daily and ID once (57% of patients) or twice daily (43%) according to treatment response. At the end of the trial, mean daily basal insulin doses were 0.59 U/kg (IG) and 0.82 U/kg (ID). Aspart doses were 0.32 U/kg (IG) and 0.36 U/kg (ID). Costs were calculated from the German statutory health insurance (SHI) perspective using official 2008 prices. Sensitivity analyses were performed to test robustness of the results. RESULTS: Annual basal and bolus insulin costs per patient were euro 1,473 (IG) and euro 1,940 (ID). The cost of lancets and blood glucose test strips were euro 1,125 (IG) and euro 1,286 (ID). Annual costs for needles were euro 393 (IG) and euro 449 (ID). The total annual cost per patient of administering IG was euro 2,991 compared with euro 3,675 for ID, translating into a 19% annual cost difference of euro 684/patient. Base case results were robust to varying assumptions for insulin dose, insulin price, change in weight and proportion of ID once daily administrations. CONCLUSION: IG and ID basal-bolus regimes have comparative safety and efficacy, based on the Hollander study, IG however may represent a significantly more cost saving option for T2D patients in Germany requiring basal-bolus insulin analogue therapy with potential annual cost savings of euro 684/patient compared to ID.

Cost-effectiveness analysis of human papillomavirus-vaccination programs to prevent cervical cancer in Austria.

OBJECTIVE: The study predicts long-term cervical cancer related population health and economic effects of introducing the HPV-vaccination for 12-year-old girls (and boys) in addition to current screening compared with screening only. METHOD: Health effects are predicted by a dynamic transmission model. Model results are used to calculate incremental cost-effectiveness ratios (ICER) in euro per life year gained (LYG) for a time-horizon between 2008 and 2060 from a public payer and a societal perspective. RESULTS: Vaccination of girls results a discounted ICER of euro 64,000/LYG and euro 50,000/LYG from a payer's and societal perspectives respectively. The additional vaccination of boys increases the ICER to euro 311,000 and euro 299,000/LYG respectively. Results were most sensitive to vaccination price, discount rate and time-horizon. CONCLUSION: HPV-vaccination for girls should be cost-effective when adopting a longer time-horizon and a societal perspective. Applying a shorter time frame and a payer's perspective or vaccinating boys may not be cost-effective without reducing the vaccine price.

Comparison of treatment costs in inadequately controlled type 2 diabetes in Germany based on the APOLLO trial with insulin glargine.

OBJECTIVE: A cost analysis of once-daily insulin glargine versus three-times daily insulin lispro in combination with oral antidiabetic drugs (OADs) for insulin-naive type 2 diabetes patients in Germany based on the APOLLO trial (A Parallel design comparing an Oral antidiabetic drug combination therapy with either Lantus once daily or Lispro at mealtime in type 2 diabetes patients failing Oral treatment). METHODS: Annual direct treatment costs were estimated from the perspective of the German statutory health insurance (SHI). Costs accounted for included insulin medication, disposable pens and consumable items (needles, blood glucose test strips and lancets). Sensitivity analyses (on resource use and unit costs) were performed to reflect current German practice. RESULTS: Average treatment costs per patient per year in the base case were 1,073 euro for glargine and 1,794 euro for lispro. Insulin costs represented 65% vs. 37% of total costs respectively. Acquisition costs of glargine were offset by the lower costs of consumable items (380 euro vs. 1,139 euro). Sensitivity analyses confirmed the robustness of the results in favour of glargine. All scenarios yielded cost savings in total treatment costs ranging from 84 euro to 727 euro. CONCLUSIONS: Combination therapy of once-daily insulin glargine versus three-times daily insulin lispro both with OADs, in the management of insulin-dependent type 2 diabetes offers the potential for substantial cost savings from the German SHI perspective.