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 been assessed as part of the STA process and was not recommended in NICE guidance issued in June 2013 (TA289). A review of TA289 was commissioned following the availability of new longer-term survival data; a price discount patient access scheme (PAS) was also introduced. The Centre for Reviews and Dissemination (CRD) and Centre for Health Economics (CHE) Technology Appraisal Group at the University of York was commissioned to act as the independent Evidence Review Group (ERG). This article provides a summary of the manufacturer or sponsor of the technology's (referred to as the company) submission, the ERG review and the resulting NICE guidance issued in March 2016. The main clinical effectiveness data were derived from two good-quality multicentre randomised controlled trials (RCTs): COMFORT-II compared ruxolitinib with best available therapy (BAT) and COMFORT-I compared ruxolitinib with placebo. Both RCTs demonstrated a statistically significant reduction in splenomegaly and its associated symptoms in intermediate-2 and high-risk myelofibrosis patients. Overall survival was statistically significantly improved with ruxolitinib compared with BAT at 3.5 years of follow-up in the COMFORT-II trial (hazard ratio 0.58, 95 % CI 0.36-0.93). Grade 3-4 adverse events were more frequent in the ruxolitinib group than in the BAT group; 42 % compared with 25 %. Evidence relating to patients with lower-risk disease or low platelet counts (50-100 × 109/L) was less robust. The company's economic model was well-presented and had an appropriate model structure. The base-case incremental cost-effectiveness ratio (ICER) was estimated to be around £45,000 per quality-adjusted life-year (QALY) gained (including the PAS discount). Extensive sensitivity and scenario analyses were presented, demonstrating that the estimated ICER was robust to a range of input values and assumptions made in the model. Alternative scenarios presented by the ERG showed only modest increases in the estimated ICER, primarily as a result of including an element of drug wastage within the model. Alternative scenarios resulted in estimated ICERs ranging from around £45,000 to £49,000 per QALY gained (including the PAS discount). At the first appraisal meeting, the NICE Appraisal Committee concluded that ruxolitinib was clinically effective and was a cost effective use of National Health Service (NHS) resources for patients with high-risk myelofibrosis who meet NICE's end-of-life criteria. Following the consultation, the company offered a revised PAS, resulting in a revised base-case ICER of £31,229 per QALY gained. The company also presented new evidence on the cost effectiveness of ruxolitinib in intermediate-2 and high-risk subgroups and a revised version of the model. The NICE Appraisal Committee considered the new evidence and recommended ruxolitinib for the treatment of patients with intermediate-2-risk disease as well as patients with high-risk disease, based on International Prognostic Scoring System (IPSS) prognostic factors.
journal_name
Pharmacoeconomicsjournal_title
PharmacoEconomicsauthors
Wade R,Hodgson R,Biswas M,Harden M,Woolacott Ndoi
10.1007/s40273-016-0447-3subject
Has Abstractpub_date
2017-02-01 00:00:00pages
203-213issue
2eissn
1170-7690issn
1179-2027pii
10.1007/s40273-016-0447-3journal_volume
35pub_type
杂志文章,评审abstract:OBJECTIVE:To explore whether the National Institute for Health and Clinical Excellence (NICE) takes account of concerns other than just incremental cost effectiveness in commissioning healthcare services. METHOD:A stated preference binary choice experiment was used to explore the preferences of members of NICE's Appra...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200725080-00006
更新日期:2007-01-01 00:00:00
abstract::The recent multinational, randomised, prospective studies Clopidogrel in Unstable Angina to Prevent Recurrent Events (CURE), Percutaneous Coronary Intervention substudy of CURE (PCI-CURE) and Clopidogrel for the Reduction of Events During Observation (CREDO) have demonstrated the clinical efficacy and safety of clopid...
journal_title:PharmacoEconomics
pub_type: 杂志文章,meta分析,多中心研究
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更新日期:2004-01-01 00:00:00
abstract:BACKGROUND:Pharmacogenetics offers the potential to improve health outcomes by identifying individuals who are at greater risk of harm from certain medicines. Routine adoption of pharmacogenetic tests requires evidence of their cost effectiveness. OBJECTIVE:The present review aims to systematically review published ec...
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更新日期:2016-08-01 00:00:00
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journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-199405050-00005
更新日期:1994-05-01 00:00:00
abstract:BACKGROUND:Assessment of health state and health-related quality of life (HR-QOL) are limited by a child's age and cognitive ability. Parent-proxy reports are known to differ from children's reports. Simultaneous assessment using a parent-child dyad is an alternative approach. OBJECTIVE:Our objective was to assess the...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/11597890-000000000-00000
更新日期:2012-08-01 00:00:00
abstract::Several randomised comparative trials have shown that granulocyte colony-stimulating factor (G-CSF) reduces the duration of neutropenia, hospitalisation and intravenous antibacterial use in patients with cancer who are receiving high-dosage antineoplastic therapy. However, one area that has received less attention is ...
journal_title:PharmacoEconomics
pub_type: 临床试验,杂志文章
doi:10.2165/00019053-199711060-00005
更新日期:1997-06-01 00:00:00
abstract:OBJECTIVE:To examine the relative cost effectiveness of topical calcipotriol and short-contact dithranol in the treatment of mild to moderate plaque psoriasis. DESIGN AND SETTING:This was a modelling study from the perspective of the UK National Health Service as payer. METHODS:The interventions were compared using 2...
journal_title:PharmacoEconomics
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doi:10.1007/s40273-020-00954-y
更新日期:2020-12-01 00:00:00
abstract::Barrett's oesophagus is a premalignant complication that occurs in approximately 10% of patients with gastro-oesophageal reflux disease (GORD). In patients with Barrett's oesophagus, the risk of adenocarcinoma of the oesophagus approaches 0.5% per patient-years. Therefore, practice guidelines have been developed that ...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-200119100-00003
更新日期:2001-01-01 00:00:00
abstract::Total hip arthroplasty (THA) is a major orthopaedic procedure with a high risk of postoperative thromboembolism. Increasing demand for this type of surgery, together with its high cost, has led to examination of means by which the cost of THA may be minimised. Current clinical opinion favours the use of suitable pharm...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-199610020-00009
更新日期:1996-08-01 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...
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doi:10.1007/s40273-017-0584-3
更新日期:2018-03-01 00:00:00
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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::As part of its Single Technology Appraisal (STA) process, the National Institute for Health and Care Excellence (NICE) invited the manufacturer of evolocumab (Amgen) to submit evidence on the clinical and cost effectiveness of evolocumab. The appraisal assessed evolocumab as monotherapy or in combination with a statin...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
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更新日期:2016-11-16 00:00:00
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doi:10.1007/s40273-020-00991-7
更新日期:2021-01-19 00:00:00
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doi:10.2165/00019053-200321150-00005
更新日期:2003-01-01 00:00:00
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更新日期:2015-11-01 00:00:00
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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
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pub_type: 杂志文章,评审
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更新日期:2018-04-01 00:00:00
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journal_title:PharmacoEconomics
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更新日期:2009-01-01 00:00:00
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journal_title:PharmacoEconomics
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更新日期:1995-01-01 00:00:00
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更新日期:2000-09-01 00:00:00
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更新日期:2019-02-01 00:00:00
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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
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pub_type: 杂志文章,评审
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更新日期:2013-11-01 00:00:00
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journal_title:PharmacoEconomics
pub_type: 杂志文章
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更新日期:1996-01-01 00:00:00
abstract:OBJECTIVE:The aim of this study was to develop a generic treatment algorithm for influenza and influenza-like illness (ILI) that could be used to estimate the costs and outcomes of current and new treatments for influenza in different countries for different patient subgroups. METHODS:A series of possible treatment pa...
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journal_title:PharmacoEconomics
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更新日期:1993-05-01 00:00:00
abstract:OBJECTIVES:To obtain utility estimates suitable for use in economic models for chronic spontaneous (idiopathic) urticaria (CSU). METHODS:Patient-level data from three randomized clinical trials-ASTERIA I, ASTERIA II and GLACIAL-were analysed. Health states were derived from the Urticaria Activity Score over 7 days (UA...
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pub_type: 杂志文章
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更新日期:2016-05-01 00:00:00