scholarly journals Febuxostat for the treatment of hyperuricaemia in people with gout: a single technology appraisal

2009 ◽  
Vol 13 (Suppl 3) ◽  
pp. 37-42
Author(s):  
M Stevenson ◽  
A Pandor
Keyword(s):  
Cost Effectiveness ◽  
Severe Disease ◽  
Treatment Algorithm ◽  
Clinical Excellence ◽  
Fixed Dose ◽  
Technology Appraisal ◽  
Tree Model ◽  
Open Label ◽  
Single Technology Appraisal ◽  
The Cost

This paper presents a summary of the evidence review group (ERG) report into the clinical effectiveness and cost-effectiveness of febuxostat for the management of hyperuricaemia in patients with gout based upon a review of the manufacturer’s submission to the National Institute for Health and Clinical Excellence (NICE) as part of the single technology appraisal (STA) process. The submission’s evidence came from two randomised controlled trials comparing the efficacy and safety of febuxostat with allopurinol. The trials were of reasonable methodological quality and measured a clinically relevant range of outcomes. A pooled clinical efficacy analysis showed that a daily dose of 80 mg or 120 mg of febuxostat was significantly more effective than fixed-dose allopurinol (300/100 mg/day) at lowering serum uric acid (sUA) levels to therapeutic targets (< 6 mg/dl); however, a large percentage of febuxostat patients did not achieve the primary end point and the fixed-dose allopurinol regimen may have introduced bias. There were no differences between treatments in more clinically important outcomes such as gout flares and tophi resolution after 52 weeks of treatment. No subgroup analyses were conducted for patients with renal impairment, non-responders to allopurinol or patients with severe disease. Supplementary data from a 2-year open-label extension study were also provided, but were difficult to interpret and poorly reported. The incidence of adverse events was similar between treatments, although more febuxostat recipients discontinued treatment prematurely. A decision tree model was developed to determine the cost-effectiveness of febuxostat. The scope was limited to the comparison of continual febuxostat treatment with continual allopurinol treatment. Switching between treatments or withdrawing treatment in patients whose sUA levels had not decreased was not permitted. The model predicted a cost-effectiveness of £16,324 [95% confidence interval (CI) £6281 to £239,928] per quality-adjusted life-year (QALY) gained for febuxostat compared with allopurinol after 2 years of treatment. The incremental cost per QALY was below £20,000 in 63% of the simulations undertaken. Changes in the time horizon did not materially affect the results. The ERG believes that the modelling structure employed was not appropriate to estimate the cost-effectiveness of febuxostat within a treatment algorithm. In addition, there were concerns about the methodology used for collecting data on key model inputs. Given these reservations the cost-effectiveness of febuxostat could not be determined. The guidance issued by NICE in August 2008 as a result of the STA states that febuxostat is recommended as an option for the management of chronic hyperuricaemia in gout only for people who are intolerant of allopurinol or for whom allopurinol is contraindicated.

scholarly journals Exploration of the difference in results of economic submissions to the National Institute of Clinical Excellence by manufacturers and assessment groups

2007 ◽  
Vol 23 (1) ◽  
pp. 96-100 ◽  
Author(s):  
Deven Chauhan ◽  
Alec H. Miners ◽  
Alastair J. Fischer
Keyword(s):  
Cost Effectiveness ◽  
Incremental Cost ◽  
Clinical Excellence ◽  
Economic Evaluations ◽  
Technology Appraisal ◽  
Single Technology Appraisal ◽  
Cost Estimates ◽  
The Difference ◽  
The Cost

Objectives:A recent study showed that estimates of cost-effectiveness submitted to National Institute for Health and Clinical Excellence (NICE) by manufacturers had significantly lower incremental cost-effectiveness ratios (ICERs) than those submitted by university-based Assessment Groups. This study extends that analysis.Methods:Data were abstracted from relevant NICE documentation for thirty-two of eighty-two possible appraisals.Results:The results from the analysis showed that sources of the difference in ICERs appear to be the effectiveness estimates relating to the comparator technology and the cost estimates relating to the technology under evaluation. That is, manufacturers estimated lower average benefits for the comparator technology and lower costs relating to the technology under evaluation compared with estimates submitted by the Assessment Groups.Conclusions:These findings may be particularly important, given the introduction of the “Single Technology Appraisal.” Considerable difficulties were encountered when undertaking this study, highlighting, above all else, the complexity of explaining why results from economic evaluations purporting to answer the same question diverge.

scholarly journals Romiplostim for the treatment of chronic immune or idiopathic thrombocytopenic purpura: a single technology appraisal

2009 ◽  
Vol 13 (Suppl 2) ◽  
pp. 63-68
Author(s):  
G Mowatt ◽  
C Boachie ◽  
M Crowther ◽  
C Fraser ◽  
R Hernández ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Idiopathic Thrombocytopenic Purpura ◽  
Economic Model ◽  
Clinical Excellence ◽  
Sensitivity Analyses ◽  
Base Case ◽  
Technology Appraisal ◽  
Platelet Response ◽  
Single Technology Appraisal ◽  
Thrombocytopenic Purpura

This paper presents a summary of the evidence review group (ERG) report into the clinical and cost-effectiveness of romiplostim for the treatment of adults with chronic immune or idiopathic thrombocytopenic purpura (ITP) based upon a review of the manufacturer’s submission to the National Institute for Health and Clinical Excellence (NICE) as part of the single technology appraisal (STA) process. The submission’s evidence came from two relatively high-quality randomised controlled trials (RCTs). The ERG found no evidence that any important data were missed or that data extraction was inaccurate. In both RCTs more patients in the romiplostim than in the placebo group achieved a durable platelet response [non-splenectomised patients: romiplostim 25/41 (61%), placebo 1/21 (5%), odds ratio (OR) 24.45, 95% confidence interval (CI) 3.34 to 179.18; splenectomised patients: romiplostim 16/42 (38%), placebo 0/21 (0%), OR 8.5 (95% CI 1.15 to 372)] and an overall platelet response [non-splenectomised patients: romiplostim 36/41 (88%), placebo 3/21 (14%), OR 34.74, 95% CI 7.77 to 155.38; splenectomised patients: romiplostim 33/42 (79%), placebo 0/21 (0%), OR 16.6 (95% CI 2.37 to 706]. The difference in mean period with a platelet response was 13.9 weeks (95% CI 10.5 to 17.4) in favour of romiplostim in the RCT of non-splectomised patients and 12.1 weeks (95% CI 8.7 to 15.6) in favour of romiplostim in the RCT of splectomised patients. The manufacturer’s economic model evaluated the cost-effectiveness of romiplostim compared with standard care. The ERG had concerns about the way the decision problem was addressed in the economic model and about the non-adjustment of findings for confounding factors. In non-splenectomised patients, using romiplostim as a first option treatment, the base-case incremental cost-effectiveness ratio (ICER) was £14,840 per quality-adjusted life-year (QALY). In splenectomised patients the ICER was £14,655 per QALY. Additional sensitivity analyses performed by the ERG identified two issues of importance: whether individuals entered the model on watch and rescue or on active therapy in the comparator arm (ICER £21,674 per QALY for non-splenectomised patients, £29,771 per QALY for splenectomised patients); whether it was assumed that any unused medicine would be wasted. Combining all of the separate sensitivity analyses, and assuming that watch and rescue was not the first-line treatment, increased the ICERs further (non-splenectomised £37,290 per QALY; splenectomised £131,017 per QALY). In conclusion, the manufacturer’s submission and additional work conducted by the ERG suggest that romiplostim has short-term efficacy for the treatment of ITP, but there is no robust evidence on long-term effectiveness or cost-effectiveness of romiplostim compared with relevant comparators.

scholarly journals Docetaxel for the adjuvant treatment of early nodepositive breast cancer: a single technology appraisal

2009 ◽  
Vol 13 (Suppl 1) ◽  
pp. 7-13
Author(s):  
J Chilcott ◽  
M Lloyd Jones ◽  
A Wilkinson
Keyword(s):  
Breast Cancer ◽  
Overall Survival ◽  
Cost Effectiveness ◽  
Adverse Events ◽  
Adjuvant Treatment ◽  
Disease Free Survival ◽  
Technology Appraisal ◽  
Single Technology Appraisal ◽  
The Cost ◽  
Disease Free

This paper presents a summary of the evidence review group (ERG) report into the clinical and cost-effectiveness of docetaxel for the adjuvant treatment of early node-positive breast cancer based upon the manufacturer’s submission to the National Institute for Health and Clinical Excellence (NICE) as part of the single technology appraisal (STA) process. The manufacturer’s scope restricts the intervention to docetaxel in combination with doxorubicin and cyclophosphamide (TAC), and the comparator to anthracycline-based chemotherapy. Based on the BCIRG 001 trial, the submitted evidence shows that TAC is associated with superior disease-free and overall survival at 5 years compared with the anthracycline-based regimen FAC. The absolute risk reduction in patients treated with TAC compared with those treated with FAC was 7% for disease-free survival and 6% for overall survival. However, TAC was associated with significantly greater toxicity than FAC. There is also evidence that docetaxel, in an unlicensed sequential regimen FEC100-T, is associated with superior diseasefree and overall survival at 5 years compared with FEC100. An economic model was developed by the manufacturer based on the BCIRG 001 trial. This generated central estimates of the cost per life-year gained and cost per quality-adjusted lifeyear (QALY) gained of TAC compared with FAC of £7900 and £9800 respectively. The manufacturer’s submission predicts a cost-effectiveness of £15,000–£20,000 per QALY gained for TAC compared with E-CMF (epirubicin in sequential therapy with cyclophosphamide, methotrexate, and fluorouracil), and estimates the cost-effectiveness of FEC100-T to be £8200 per QALY compared with FEC100. Taking into account a number of issues identified by the ERG this may generate higher estimates of cost-effectiveness, but these are unlikely to exceed £35,000 per QALY gained. Importantly, FAC is not commonly used in clinical practice in the UK and, therefore, the submitted evidence does not indicate whether TAC is superior to the anthracycline-based regimens that are in common use (FEC or E-CMF). The indirect comparisons presented suggest that the economic case for TAC in comparison to current UK practice may not be proven. The manufacturer’s submission failed to record evidence of three serious adverse events in patients receiving docetaxel with doxorubicin or to mention the concern of the European Medicines Agency regarding TAC’s long-term adverse events. The guidance issued by NICE in June 2006 as a result of the STA states that docetaxel, when given concurrently with doxorubicin and cyclophosphamide (the TAC regimen), is recommended as an option for the adjuvant treatment of women with early nodepositive breast cancer.

P14.41 Cost-effectiveness of FET PET for early treatment response assessment in glioma patients following adjuvant temozolomide chemotherapy

2021 ◽  
Vol 23 (Supplement_2) ◽  
pp. ii46-ii46
Author(s):  
J Rosen ◽  
G Ceccon ◽  
E K Bauer ◽  
J M Werner ◽  
C Kabbasch ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Cost Effective ◽  
Response Assessment ◽  
Sensitivity Analyses ◽  
Tree Model ◽  
Fet Pet ◽  
Conventional Mri ◽  
Adjuvant Temozolomide ◽  
The Cost ◽  
Temozolomide Chemotherapy

Abstract BACKGROUND In light of increasing healthcare costs, higher medical expenses should be justified socio-economically. Therefore, we calculated the effectiveness and cost-effectiveness of PET using the radiolabeled amino acid O-(2-[18F]-fluoroethyl)-L-tyrosine (FET) compared to conventional MRI for early identification of responders to adjuvant temozolomide chemotherapy. A recent study in IDH-wildtype glioma patients suggested that after two cycles, FET-PET parameter changes predicted a significantly longer survival while MRI changes were not significant. MATERIALS AND METHODS To determine the effectiveness and cost-effectiveness of serial FET-PET imaging, we analyzed published clinical data and calculated the associated costs in the context of the German healthcare system.Based on a decision-tree model, FET-PET and MRI’s effectiveness was calculated, i.e., the probability to correctly identify a responder as defined by an overall survival ≥15 months. To determine the cost-effectiveness, the incremental cost-effectiveness ratio (ICER) was calculated, i.e., the cost for each additionally identified responder by FET-PET who would have remained undetected by MRI. The robustness of the results was tested by deterministic and probabilistic (Monte Carlo simulation) sensitivity analyses. RESULTS Compared to MRI, FET-PET increases the rate of correctly identified responders to chemotherapy by 26%; thus, four patients need to be examined by FET-PET to identify one additional responder. Considering the respective cost for serial FET-PET and MRI, the ICER resulted in €4,396.83 for each additional correctly identified responder by FET-PET. The sensitivity analyses confirmed the robustness of the results. CONCLUSION In contrast to conventional MRI, the model suggests that FET PET is cost-effective in terms of ICER values. Concerning the high cost of temozolomide, the integration of FET-PET has the potential to avoid premature chemotherapy discontinuation at a reasonable cost.

scholarly journals A Cost-Effectiveness Analysis of Newborn Screening for Severe Combined Immunodeficiency in the UK

2019 ◽  
Vol 5 (3) ◽  
pp. 28 ◽  
Author(s):  
Alice Bessey ◽  
James Chilcott ◽  
Joanna Leaviss ◽  
Carmen de la Cruz ◽  
Ruth Wong
Keyword(s):  
Cost Effectiveness ◽  
Severe Combined Immunodeficiency ◽  
Cost Effective ◽  
Sensitivity Analyses ◽  
Combined Immunodeficiency ◽  
Tree Model ◽  
Life Years ◽  
The Uk ◽  
The Cost ◽  
The Impact

Severe combined immunodeficiency (SCID) can be detected through newborn bloodspot screening. In the UK, the National Screening Committee (NSC) requires screening programmes to be cost-effective at standard UK thresholds. To assess the cost-effectiveness of SCID screening for the NSC, a decision-tree model with lifetable estimates of outcomes was built. Model structure and parameterisation were informed by systematic review and expert clinical judgment. A public service perspective was used and lifetime costs and quality-adjusted life years (QALYs) were discounted at 3.5%. Probabilistic, one-way sensitivity analyses and an exploratory disbenefit analysis for the identification of non-SCID patients were conducted. Screening for SCID was estimated to result in an incremental cost-effectiveness ratio (ICER) of £18,222 with a reduction in SCID mortality from 8.1 (5–12) to 1.7 (0.6–4.0) cases per year of screening. Results were sensitive to a number of parameters, including the cost of the screening test, the incidence of SCID and the disbenefit to the healthy at birth and false-positive cases. Screening for SCID is likely to be cost-effective at £20,000 per QALY, key uncertainties relate to the impact on false positives and the impact on the identification of children with non-SCID T Cell lymphopenia.

P0434COST-EFFECTIVENESS OF MAINTANCE THERAPY WITH AZATHIOPRIME VERSUS RITUXIMAB (TAILORED OR FIXED-SCHEDULE) IN ADULTS WITH GENERALIZED ANCA VASCULITIS IN COLOMBIA

2020 ◽  
Vol 35 (Supplement_3) ◽  
Author(s):  
Kateir Mariel Contreras ◽  
Viviana Orozco Ortiz ◽  
Eduardo José Puche ◽  
Paola Karina Garcia ◽  
Camilo Alberto Gonzalez ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Lower Cost ◽  
Transition Probabilities ◽  
Cost Effective ◽  
Sensitivity Analyses ◽  
Fixed Dose ◽  
National Guidelines ◽  
Anca Vasculitis ◽  
Life Years ◽  
The Cost

Abstract Background and Aims Azathioprine has been for decades the drug of choice for maintenance therapy in patients with generalized ANCA vasculitis in remission. However, recent studies show that rituximab, a high-cost biological agent, which can be administrated in two different schedules, might be more effective, so it is necessary to know the cost- effectiveness. Our goal was to compare the cost-effectiveness of the 3 maintenance schemes: standard therapy with azathioprine; fixed-dose rituximab and rituximab tailored according to CD19 lymphocyte level and ANCA titres, from the perspective of the Colombian healthcare system. Method We designed a 5-year annual cycle Markov model with the following stages: remission, minor relapse, mayor relapse and death. Transition probabilities were obtained from a systematic review of the literature (Scopus and Pubmed). Following national guidelines for economic studies, costs (in 2018, 1 euro = 3489 Colombian pesos) were estimated based on national drug registries, and official tariff manuals for procedures and other resources. Main outcome was quality-adjusted life years (QALY), using lupus nephropathy as a proxy; values were obtained from Tufts CEA Registry and validated by local expert panel through a modified Delphi technique. Cost-effectiveness threshold was three-times per capita GDP (16.872 euros). Discount rate was 5%. Univariate and probabilistic sensitivity analyses were performed. Results Overall discounted 5-years costs were € 1149 for azathioprine; € 4025 for tailored rituximab and € 5221 for fixed rituximab. QALY gains were 2.94, 3.63 and 3.64, respectively. Both tailored and fixed rituximab were cost-effective (cost per QALY gained: € 4168 and € 5817 respectively), but tailored dosing was preferable due to its lower cost. Sensitivity analyses did not modify these results significantly. Conclusion To our knowledge this is the first economic evaluation that compare azathioprine with tailored and fixed rituximab regimens as a vasculitis maintenance treatment in adults with ANCA generalized. Due to its lower effectiveness azathioprine should not be the first line of treatment. Tailored rituximab should be a better option than fixed schedule due to its lower cost with similar effectiveness.

scholarly journals Diabetes Screening Through Community Pharmacies in England: A Cost-Effectiveness Study

Pharmacy ◽  
2019 ◽  
Vol 7 (1) ◽  
pp. 30 ◽  
Author(s):  
David Wright ◽  
Richard Little ◽  
David Turner ◽  
Tracey Thornley
Keyword(s):  
Cost Effectiveness ◽  
Social Deprivation ◽  
Geographical Location ◽  
Community Pharmacies ◽  
Diabetes Screening ◽  
Tree Model ◽  
Patients With Diabetes ◽  
One Year ◽  
The Cost ◽  
Mixed Ethnicity

Community pharmacies are commonly used to screen for patients with diabetes. The aim of this paper is to estimate the cost per test and cost per appropriately referred patient from a pharmacy perspective using a one-year decision tree model. One-way sensitivity analysis was performed to estimate the effect of geographical location and patient self-referral rate. Data was used from 164 patients screened and located in an area with average social deprivation and largely white European inhabitants and 172 patients in an area with higher social deprivation (lower than average ability to access society’s resources) and a mixed ethnicity population in England. The diabetes screening consisted of initial risk assessment via questionnaire followed by HbA1c test for those identified as high risk. The cost per person screened was estimated as £28.65. The cost per appropriately referred patient with type 2 diabetes was estimated to range from £7638 to £11,297 in deprived mixed ethnicity and non-deprived areas respectively. This increased to £12,730 and £18,828, respectively, if only 60% of patients referred chose to inform their general practitioner (GP). The cost per test and identification rates through community pharmacies was similar to that reported through medical practices. Locating services in areas of suspected greater diabetes prevalence and increasing the proportion of patients who follow pharmacist advice to attend their medical practice improves cost-effectiveness.

scholarly journals The Cost-effectiveness of Biological Therapy Cycles in the Management of Crohn’s Disease

2019 ◽  
Vol 13 (10) ◽  
pp. 1323-1333 ◽  
Author(s):  
Kristian Bolin ◽  
Erik Hertervig ◽  
Edouard Louis
Keyword(s):  
Crohn’S Disease ◽  
Cost Effectiveness ◽  
Crohn's Disease ◽  
Combination Therapy ◽  
Willingness To Pay ◽  
Cost Effective ◽  
Treatment Intensification ◽  
Tree Model ◽  
Necrosis Factor Alpha ◽  
The Cost

Abstract Objectives To examine the cost-effectiveness of continued treatment for patients with moderate-severe Crohn’s disease in clinical remission, with a combination of anti-tumour necrosis factor alpha [anti-TNFα] [infliximab] and immunomodulator therapy compared with two different withdrawal strategies: [1] withdrawal of the anti-TNFα therapy; and [2] withdrawal of the immunomodulator therapy, respectively. Methods A decision-tree model was constructed mimicking three treatment arms: [1] continued combination therapy with infliximab and immunomodulator; [2] withdrawal of infliximab; or [3] withdrawal of the immunomodulator. Relapses in each arm are managed with treatment intensification and re-institution of the de-escalated drug according to a prespecified algorithm. State-dependent relapse risks, remission probabilities, and quality of life weights were collected from previous published studies. Results Combination therapy was less costly and more efficient than the withdrawal of the immunomodulator, and more costly and more efficient than withdrawal of infliximab. Whether or not combination therapy is cost-effective, compared with the alternatives, depends primarily on current pharmaceutical prices and the willingness-to-pay per additional quality-adjusted life-year [QALY]. Conclusions Combination therapy using a combination of anti-TNFα [infliximab] and an immunomodulator is cost-effective in the treatment of Crohn’s disease compared with treatment cycles in which the immunomodulator is withdrawn. Combination treatment is cost-effective compared with treatment cycles in which infliximab is withdrawn, at prices of infliximab below€192/100 mg, given a willingness-to-pay threshold at€49 020 [Sweden] per additional QALY.

Cost-Effectiveness Analysis: Discount the Placebo at Your Peril

2010 ◽  
Vol 30 (5) ◽  
pp. 536-543 ◽  
Author(s):  
Neil Hawkins ◽  
David A. Scott
Keyword(s):  
Clinical Practice ◽  
Cost Effectiveness ◽  
Placebo Effect ◽  
Severe Disease ◽  
Quality Adjusted Life Year ◽  
Routine Clinical Practice ◽  
Regression To The Mean ◽  
Expectancy Effect ◽  
The Cost ◽  
Selection Of

Introduction: The authors consider alternative mechanisms that might explain placebo responses and their implications for cost-effectiveness modeling. Three alternative placebo mechanisms are examined: a ‘‘regression to the mean’’ effect arising from natural variation and the preferential selection of patients with acutely severe disease into clinical trials, a patient expectancy effect specific to the clinical trial setting (Hawthorne effect), and a patient expectancy effect generalizable to routine clinical practice (true placebo effect). Methods: To estimate cost-effectiveness, the authors needed to generalize from trial data to estimate responses to treatment that they would see in routine clinical practice. They use an example analysis of the cost-effectiveness of adjunct epilepsy treatments to illustrate the potential effects of these different placebo mechanisms on this generalization and subsequent cost-effectiveness estimates and adoption decisions. Results: If an acceptable willingness-to-pay threshold of 30,000 per quality-adjusted life year (QALY) is assumed, then each of the placebo effect scenarios identifies a different treatment alternative as being optimum. Discussion: Estimated cost-effectiveness ratios and associated policy decisions may be sensitive to assumptions regarding the mechanism underlying placebo responses. These assumptions should, if possible, be investigated through analysis of trial or observational data and, in the absence of other evidence, sensitivity analysis.

Sign in / Sign up

Export Citation Format

Share Document