Abstract:
:The National Institute for Health and Care Excellence (NICE) invited GlaxoSmithKline, the manufacturer of dabrafenib, to submit evidence for the clinical and cost effectiveness of dabrafenib for the treatment of unresectable, advanced or metastatic BRAF V600 mutation-positive melanoma in accordance with the Institute's Single Technology Appraisal (STA) process. The Liverpool Reviews and Implementation Group (LRiG) at the University of Liverpool was commissioned to act as the Evidence Review Group (ERG). This article summarizes the ERG's review of the evidence submitted by the company and provides a summary of the Appraisal Committee's (AC) final decision in October 2014. The clinical evidence for dabrafenib was derived from an ongoing phase III, randomized, double-blind, placebo-controlled, international, multicentre clinical trial (BREAK-3) involving 230 patients randomized 2:1 to receive either dabrafenib or dacarbazine. A significant improvement in median progression-free survival (PFS) but not overall survival (OS) was reported in the dabrafenib arm compared with dacarbazine. Vemurafenib is considered a more appropriate comparator than is dacarbazine. The clinical evidence for vemurafenib was derived from a completed phase III, randomized, double-blind, placebo-controlled, international, multicentre clinical trial (BRIM-3) involving 675 patients randomized 1:1 to receive either vemurafenib or dacarbazine. A significant improvement in median PFS and OS was reported in the vemurafenib arm compared with dacarbazine. As there is no direct evidence comparing dabrafenib versus vemurafenib, the company presented an indirect treatment comparison (ITC) that demonstrated no statistical differences between dabrafenib and vemurafenib for PFS or OS. The ERG expressed concerns with the ITC, mainly in relation to the validity of the assumptions underpinning the methodology; the ERG concluded this resulted in findings that are unlikely to be robust or reliable. Dabrafenib and vemurafenib are both available to patients treated by the National Health Service (NHS) in England via a Patient Access Scheme (PAS) in which the costs of the drugs are discounted. Using these discounted costs, the incremental cost-effectiveness ratios (ICERs) generated by the company were £60,980 per quality-adjusted life-year (QALY) for dabrafenib versus dacarbazine and £11,046 per QALY gained for dabrafenib versus vemurafenib. The ERG considered the economic model structure developed by the company to derive the ICERs to be overly complex and based on unsubstantiated assumptions, most importantly in relation to the projection of OS. Applying the latest OS data from BREAK-3 to a less complex model structure increased the estimated ICER for dabrafenib compared with dacarbazine from £60,980 to £112,727 per QALY gained. Since the results from the ITC were considered by the ERG to be neither reliable nor robust, the ERG also considered a cost-effectiveness comparison to be inappropriate due to a lack of meaningful or reliable data. In spite of limitations in the data, the AC took the view that dabrafenib and vemurafenib were "likely" of similar clinical effectiveness. Since the overall costs of these two drugs were similar, the AC recommended the use of dabrafenib in patients with unresectable, advanced or metastatic BRAF V600 mutation-positive melanoma.
journal_name
Pharmacoeconomicsjournal_title
PharmacoEconomicsauthors
Fleeman N,Bagust A,Beale S,Boland A,Dickson R,Dwan K,Richardson M,Dundar Y,Davis H,Banks Ldoi
10.1007/s40273-015-0276-9subject
Has Abstractpub_date
2015-09-01 00:00:00pages
893-904issue
9eissn
1170-7690issn
1179-2027journal_volume
33pub_type
杂志文章,评审abstract::As part of the National Institute for Health and Care Excellence's (NICE) Single Technology Appraisal (STA) process, ruxolitinib was assessed to determine the clinical and cost effectiveness of its use in the treatment of disease-related splenomegaly or symptoms in adults with myelofibrosis. Ruxolitinib had previously...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.1007/s40273-016-0447-3
更新日期:2017-02-01 00:00:00
abstract::Currently, the most important issue in US pharmaceutical policy is probably cost containment. Research into pharmaceutical issues should therefore concentrate on this area. Analysis of changing trends in pharmaceutical expenditures, particularly during the early 1990s, would be a useful starting point. Research into t...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-199600102-00019
更新日期:1996-01-01 00:00:00
abstract:OBJECTIVE:The study objective was to evaluate and compare quality-of-life (QOL) parameters between patients with type 2 (non-insulin-dependent) diabetes mellitus who changed therapy from an oral hypoglycaemic agent (OHA) to insulin and those who remained on an OHA. DESIGN:The World Health Organization Quality of Life ...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-199814020-00008
更新日期:1998-08-01 00:00:00
abstract::This paper analyses the 32 technology appraisals completed by the National Institute for Clinical Excellence (NICE) in the UK from its establishment to the end of January 2002. It looks at why technologies have been rejected, what has happened to products reviewed at launch, evidence of rationing on cost-effectiveness...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200220003-00010
更新日期:2002-01-01 00:00:00
abstract::The annual cost of managing migraine totals billions of US dollars. This retrospective economic analysis of a clinical trial comparing subcutaneous dihydroergotamine mesylate (DHE) with subcutaneous sumatriptan in the treatment of acute migraine is appropriate because, although each product has been shown to be effica...
journal_title:PharmacoEconomics
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.2165/00019053-199610010-00006
更新日期:1996-07-01 00:00:00
abstract:INTRODUCTION:This analysis compared the cost effectiveness of adding ezetimibe to atorvastatin therapy versus atorvastatin titration or adding cholestyramine (a resin) for patients at high risk of a coronary artery disease (CAD) event who did not reach target cholesterol levels on their current atorvastatin dosage. The...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200624080-00007
更新日期:2006-01-01 00:00:00
abstract::Drug utilisation in term and preterm neonates (i.e. less than 28 days of age) has been investigated prospectively in 4 clinical studies during the past 10 years. 3880 neonates with a mean gestational age of 34.5 weeks (corresponding birthweight 2280g) were enrolled in these studies. An overview indicates a high prev...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-199304060-00005
更新日期:1993-12-01 00:00:00
abstract::Rheumatoid arthritis (RA) is a chronic, generally progressive autoimmune disease that causes functional disability, significant pain and joint destruction, and leads to premature mortality. It is estimated to affect between 0.5 and 1.0% of the adult population worldwide, increases in prevalence with age and affects mo...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-200422001-00002
更新日期:2004-01-01 00:00:00
abstract:CONTEXT:This study assesses if, and how, existing methods for economic evaluation are applicable to the evaluation of personalized medicine (PM) and, if not, where extension to methods may be required. METHODS:A structured workshop was held with a predefined group of experts (n = 47), and was run using a modified nomi...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-014-0211-5
更新日期:2015-01-01 00:00:00
abstract:OBJECTIVE:A cost-effectiveness analysis of oral capecitabine versus intravenous bolus 5-fluorouracil/l-leucovorin (FU/LV) as adjuvant therapy in patients with stage 3 colon cancer was performed from a Japanese healthcare payer perspective. METHODS:Adjuvant therapy comprised 24 weeks of treatment with either oral capec...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/11310110-000000000-00000
更新日期:2009-01-01 00:00:00
abstract:BACKGROUND:The use of granulocyte colony-stimulating factor (G-CSF) can enable dose intensification of chemotherapy in small-cell lung cancer (SCLC). However, given its acquisition cost, it is important to assess its cost effectiveness within a resource-constrained health service. OBJECTIVE:To assess the cost effectiv...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200624050-00003
更新日期:2006-01-01 00:00:00
abstract::This study evaluated the cost utility of adding interferon-alpha 2b to conventional treatment in patients with multiple myeloma. It also provides a methodology for transforming complex quality-of-life profiles into a single index value on the conventional 0 to 1 quality-adjusted life-year scale (QALY). From 1990 to 19...
journal_title:PharmacoEconomics
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.2165/00019053-199712010-00009
更新日期:1997-07-01 00:00:00
abstract::The application of cost-effectiveness methodology is particularly important in widespread diseases such as hypertension. However, because prospective cost-effectiveness analyses comparing different antihypertensive drugs are not currently available, differences in the cost effectiveness of these drugs can only be esti...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-199508060-00006
更新日期:1995-12-01 00:00:00
abstract::Vaccination is an underutilised, low cost and effective method of preventing illness. Cost-effectiveness analysis discloses a beneficial role of influenza vaccination in preventing illness especially in patients over 65 years of age and in high risk patients. In one large study, when 150 million doses of influenza v...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-199202030-00003
更新日期:1992-09-01 00:00:00
abstract::Asian healthcare systems are very diverse, representing cultures, political systems and economies from more than 30 countries with varying histories. Despite the diversity in the region, there has been enormous growth in health economics and outcomes research since the beginning of the 21st century. Whilst Japan has s...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/11538360-000000000-00000
更新日期:2010-01-01 00:00:00
abstract::There are insufficient resources in the UK to fund all new technologies and new indications approved by the National Institute for Health and Clinical Excellence (NICE). Diverting funding from existing sources will have a detrimental effect on the provision of other priority services. The UK Office of Fair Trading (OF...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-200826020-00001
更新日期:2008-01-01 00:00:00
abstract::Decision-analytic models play a key role in informing healthcare resource allocation decisions. However, there are ongoing concerns with the credibility of models. Modelling methods guidance can encourage good practice within model development, but its value is dependent on its ability to address the areas that modell...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-017-0499-z
更新日期:2017-06-01 00:00:00
abstract::Uncertainty in the decision-making process for reimbursement of health technologies could be reduced if additional information were available. Although methods to evaluate the monetary value of the uncertainty have been previously described, an economic evaluation of alternative methods to acquire additional informati...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-200725110-00002
更新日期:2007-01-01 00:00:00
abstract::We review regulation of two important parameters for third-party payers and manufacturers of prescription drugs: regulation of reimbursement and pricing. We find that centralised regulation of reimbursement and pricing prevails in the 15 original EU member countries (EU-15) and in European Free Trade Association (EFTA...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200725060-00001
更新日期:2007-01-01 00:00:00
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journal_title:PharmacoEconomics
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doi:10.1007/s40273-017-0554-9
更新日期:2017-12-01 00:00:00
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journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-018-0755-x
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abstract:BACKGROUND:Within the framework of economic evaluations, the transferability of utility scores between jurisdictions remains unclear. The EQ-5D is a generic instrument for measuring health-related quality of life in economic evaluations, which can be used for comparing utility scores across countries. At present, the E...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/11314120-000000000-00000
更新日期:2009-01-01 00:00:00
abstract::In this paper we consider the evolving American healthcare landscape and what it means for the use of economic evaluation of health interventions. We emphasise that use of economic evaluation in the US is unlikely to follow the European, Canadian or Australian models, which use cost effectiveness openly and explicitly...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-200624110-00012
更新日期:2006-01-01 00:00:00
abstract::Health economic evaluation is a framework for the comparative analysis of the incremental health gains and costs associated with competing decision alternatives. The process of developing health economic models is usually complex, financially expensive and time-consuming. For these reasons, model development is someti...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-017-0553-x
更新日期:2017-11-01 00:00:00
abstract::The progressive disability of Parkinson's disease results in substantial burdens for patients, their families and society in terms of increased health resource use, poorer quality of life, caregiver burden, disrupted family relationships, decreases in social and leisure activities, deteriorating emotional well-being, ...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-200119070-00003
更新日期:2001-01-01 00:00:00
abstract::Advanced controlled release (CR) dosage forms are relative newcomers to pharmaceutical markets, and few studies relate their efficacy, safety or compliance benefits to economic value. This literature review was undertaken to assess the cost effectiveness of CR dosage forms using such measures as purchase costs, total ...
journal_title:PharmacoEconomics
pub_type: 杂志文章,meta分析
doi:10.2165/00019053-199405060-00005
更新日期:1994-06-01 00:00:00
abstract:BACKGROUND:Probabilistic sensitivity analysis (PSA) in cost-effectiveness analysis involves sampling a large number of realisations of an economic model. For some parameters, we may be uncertain around the true mean values of the variables, but the ordering of the values is known. Typical sampling approaches lack eithe...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-017-0584-3
更新日期:2018-03-01 00:00:00
abstract:BACKGROUND:Swedish formulary committees are expected to influence prescribing practice by establishing and issuing drug lists and clinical practice guidelines, particularly now that financial responsibility for prescription drugs has been transferred from the national to the county council level. OBJECTIVE:The purpose...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200017020-00006
更新日期:2000-02-01 00:00:00
abstract:AIM:To estimate the direct and indirect costs of morbidity and mortality attributable to alcohol consumption in Germany from a societal perspective in 2002. METHODS:Using the concept of attributable risks and the prevalence-based approach, age- and gender-specific alcohol-attributable fractions for morbidity and morta...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200725070-00006
更新日期:2007-01-01 00:00:00
abstract::Disease management is a systematic approach to a health condition or a healthcare intervention that organises preventative, interventional and care approaches throughout the continuum of care and which measures outcomes in terms of populations, not individuals. Disease management's advantage over the current component...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-199814010-00002
更新日期:1998-07-01 00:00:00