Abstract:
:The National Institute for Health and Care Excellence (NICE) invited Pfizer, the manufacturer of inotuzumab ozogamicin (henceforth inotuzumab), to submit clinical- and cost-effectiveness evidence for inotuzumab as part of NICE's single technology appraisal process. The Centre for Reviews and Dissemination and the Centre for Health Economics, both at the University of York, were commissioned as the independent evidence review group (ERG). The clinical-effectiveness data were from a multicentre randomised controlled trial that compared inotuzumab with standard of care (SoC), where SoC was the investigator's choice of chemotherapy. Inotuzumab demonstrated statistically significant improvements in response rates or in the proportion of patients progressing to haematopoietic stem cell transplant (HSCT) but failed to meet the second primary objective of longer overall survival. Treatment-emergent adverse events were more frequent in the SoC arm, except veno-occlusive disease, which was more frequent in the inotuzumab arm. The company's economic model split patients into three post-hoc subgroups and used a partitioned survival approach within each group, with a cure assumption 3 years after receiving HSCT. In contrast with the trial results, the economic model estimated substantial improvement in survival with inotuzumab compared with SoC, providing an additional 5.2 life-years and 2.2 quality-adjusted life-years (QALYs) using a discount rate of 1.5% per annum. The ERG's critique highlighted a number of concerns, including the use of a post-hoc post-randomisation patient subset for extrapolation, the choice of a 1.5% discount rate, the complexity of the parametric modelling, the assumption of further treatment benefit post-HSCT, the nature of the cure assumption, and the length of inpatient stay while receiving treatment. The combination of the ERG's adjustments resulted in an incremental cost-effectiveness ratio (ICER) of £122,174 per QALY gained using Kaplan-Meier survival estimates and £114,078 per QALY gained with parametric survival models fit to the trial data. The final determination of the appraisal followed four NICE Appraisal Committee meetings, an appeal by the company and other stakeholders, two patient access schemes, and a company response to each appraisal consultation. The final ICER post-consultation was between £33,749 and £37,497 per QALY gained compared with SoC (excluding the confidential discount for blinatumomab received as subsequent therapy). The Appraisal Committee concluded that the ICER for inotuzumab was within the range usually considered cost effective for end-of-life care and recommended inotuzumab within its licensed indication.
journal_name
Pharmacoeconomicsjournal_title
PharmacoEconomicsauthors
Cox E,Wade R,Peron M,Dietz KC,Eastwood A,Palmer S,Griffin Sdoi
10.1007/s40273-019-00779-4subject
Has Abstractpub_date
2019-09-01 00:00:00pages
1081-1091issue
9eissn
1170-7690issn
1179-2027pii
10.1007/s40273-019-00779-4journal_volume
37pub_type
杂志文章,评审abstract::Scientific reviewing methods were applied to economic studies of influenza vaccination, and an economic model of influenza vaccination was developed from these primary sources. Issues arising from the secondary literature review include the quality of evidence on the effectiveness of the vaccines, the absence of a tra...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-199600093-00015
更新日期:1996-01-01 00:00:00
abstract:BACKGROUND:Results from the PROVE IT trial suggest that patients with acute coronary syndrome (ACS) treated with atorvastatin 80 mg/day (A80) have significantly lower rates of cardiovascular events compared with patients treated with pravastatin 40 mg/day (P40). In a genetic post hoc substudy of the PROVE IT trial, the...
journal_title:PharmacoEconomics
pub_type: 杂志文章,随机对照试验
doi:10.1007/s40273-013-0054-5
更新日期:2013-06-01 00:00:00
abstract::Cefotaxime is a parenterally administered third generation cephalosporin with a broad spectrum of antimicrobial activity. After more than a decade of use, cefotaxime continues to play an important role in the treatment of patients with serious infections, particularly those caused by Gram-negative bacteria. Clinical t...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-199813010-00009
更新日期:1998-01-01 00:00:00
abstract:BACKGROUND:The process of "mapping" is increasingly being used to predict health utilities, for application within health economic evaluations, using data on other indicators or measures of health. Guidance for the reporting of mapping studies is currently lacking. OBJECTIVE:The overall objective of this research was ...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-015-0312-9
更新日期:2015-10-01 00:00:00
abstract:BACKGROUND:A wide variety of oral antiplatelet trials have been carried out, and a large number of cost-effectiveness estimates based on them have been published. OBJECTIVE:To assess the cost effectiveness of oral antiplatelet treatments in the prevention of cardiovascular events. METHODS:A comprehensive literature s...
journal_title:PharmacoEconomics
pub_type: 杂志文章,meta分析,评审
doi:10.2165/00019053-200725120-00007
更新日期:2007-01-01 00:00:00
abstract:BACKGROUND:National EQ-5D value sets are developed because preferences for health may vary in different populations. UK values are lower than US values for most of the 243 possible EQ-5D health states. Although similar protocols were used for data collection, analytic choices regarding how to model values from the coll...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/11595420-000000000-00000
更新日期:2012-12-01 00:00:00
abstract:BACKGROUND:International phase III studies (CHIB 201 and 352) showed that basiliximab, a high affinity chimeric monoclonal antibody interleukin-2 receptor antagonist, is highly effective in preventing acute rejection when used as immunoprophylaxis in patients receiving cyclosporin (Neoral). We conducted a cost evaluati...
journal_title:PharmacoEconomics
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.2165/00019053-200321110-00003
更新日期:2003-01-01 00:00:00
abstract:OBJECTIVE:To estimate the cost effectiveness (from the UK NHS and personal social services perspective) of the cholinesterase inhibitors donepezil, rivastigmine and galantamine compared with usual care in the treatment of mild to moderately severe Alzheimer's disease. Patients had a mean age of 74 years, a mean disease...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200523120-00010
更新日期:2005-01-01 00:00:00
abstract::Mapping functions are estimated using regression analyses and are frequently used to predict health state utility values (HSUVs) in decision analytic models. Mapping functions are used when evidence on the required preference-based measure (PBM) is not available, or where modelled values are required for a decision an...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.1007/s40273-017-0548-7
更新日期:2017-12-01 00:00:00
abstract:BACKGROUND AND OBJECTIVE:Accurate prediction of relevant outcomes is important for targeting therapies and to support health economic evaluations of healthcare interventions in patients with diabetes. The United Kingdom Prospective Diabetes Study (UKPDS) risk equations are some of the most frequently used risk equation...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-019-00822-4
更新日期:2019-12-01 00:00:00
abstract::Due to a single error in the annual cost of sarilumab the following needs to be corrected in the article. ...
journal_title:PharmacoEconomics
pub_type: 已发布勘误
doi:10.1007/s40273-019-00857-7
更新日期:2020-01-01 00:00:00
abstract::Published estimates for the treatment costs of breast cancer vary widely in methodology, perspective, patient populations and time horizon. We systematically summarized and analysed the published literature on per-patient costs of breast cancer, and highlight the perspectives, populations studied, time horizons and fu...
journal_title:PharmacoEconomics
pub_type: 杂志文章,meta分析,评审
doi:10.2165/00019053-200927030-00003
更新日期:2009-01-01 00:00:00
abstract::The symptoms of onychomycosis and their impact on personal appearance are important determinants of patients' perceptions of their own health. The effect of onychomycosis is greater on psychosocial than physical functioning and is directly related to the extent of nail involvement. This review identified a number of d...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-200220010-00003
更新日期:2002-01-01 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 vedolizumab (Takeda UK) to submit evidence of the clinical effectiveness and cost effectiveness of vedolizumab for the treatment of patients with moderate-to-severe active ...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-015-0334-3
更新日期:2016-03-01 00:00:00
abstract:BACKGROUND:Like other developed countries with aging populations, Sweden is expecting large increases in the prevalence of Alzheimer's disease and corresponding escalations in the cost of care for patients with this disease. Galantamine, a new acetylcholinesterase inhibitor and nicotinic modulator, has proved effective...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200220090-00006
更新日期:2002-01-01 00:00:00
abstract::In this review, the economic aspects of pneumococcal pneumonia are analysed, including the costs, cost effectiveness and cost benefit of treatment and prevention. We identified eight cost-of-illness studies, 15 analyses comparing the costs of different treatment options and 15 economic evaluations of prevention that m...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-200422110-00003
更新日期:2004-01-01 00:00:00
abstract:BACKGROUND:While drotrecogin alfa (activated) was shown to decrease absolute 28-day mortality by 6.1% in patients with severe sepsis in the Recombinant Human Protein C Worldwide Evaluation in Severe Sepsis (PROWESS) study, no mortality benefit was observed in the subset of patients with only one organ system failure. C...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/BF03262331
更新日期:2003-01-01 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::Epilepsy is a chronic condition with numerous social and psychological consequences. This work aimed to review available data on epilepsy and the impact of surgical and pharmaceutical treatments on the quality of life in adults and children. Research on quality of life in epilepsy is characterised by a wide and fragme...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-200220150-00002
更新日期:2002-01-01 00:00:00
abstract:OBJECTIVES:Discrete choice experiments (DCEs) are increasingly advocated as a way to quantify preferences for health. However, increasing support does not necessarily result in increasing quality. Although specific reviews have been conducted in certain contexts, there exists no recent description of the general state ...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-018-0734-2
更新日期:2019-02-01 00:00:00
abstract:BACKGROUND:Assessing the societal perspective in economic evaluations of new interventions requires estimates of indirect non-medical costs caused by the disease. Different methods exist for measuring the labor input function as a surrogate for these costs. They rarely specify the effect of health on labor and who gain...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-020-00978-4
更新日期:2020-11-25 00:00:00
abstract:BACKGROUND:Economic models are considered to be important, as they help evaluate the long-term impact of diabetes treatment. To date, it appears that no article has reviewed and critically appraised the cost-effectiveness models developed to evaluate new oral treatments [glucagon-like peptide-1 (GLP-1) receptor agonist...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.1007/s40273-013-0117-7
更新日期:2014-01-01 00:00:00
abstract::A number of health-related QOL (HR-QOL) measures specifically designed for people with diabetes mellitus have appeared in the literature. This article provides a selective review of 12 measures that address this important construct. For each included study, a description of the measure and its development phase is pro...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-200422170-00002
更新日期:2004-01-01 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::Diseases of the prostate currently represent a major health problem worldwide. As the age of the male population increases in the future, so will the number of patients suffering from these disorders and the cost for treatment increase. Currently, benign prostatic hyperplasia (BPH) and prostate cancer are common in ...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-199201050-00007
更新日期:1992-05-01 00:00:00
abstract:OBJECTIVE:Pertussis is a highly contagious infection affecting mainly children. Acellular pertussis vaccines were recently introduced in Canada based on evidence of improved safety and efficacy over whole cell vaccines, the current standard of care. The following study reports the economic impact of replacing the whole...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200119050-00009
更新日期:2001-01-01 00:00:00
abstract:INTRODUCTION:The objective of this study was to evaluate the potential economic implications of using etoricoxib versus non-selective NSAID alternatives in the treatment of patients with osteoarthritis (OA) or rheumatoid arthritis (RA) in the UK. STUDY DESIGN:Decision-analytical modelling was used to calculate the exp...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200422100-00003
更新日期:2004-01-01 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
doi:10.2165/00019053-200422004-00006
更新日期:2004-01-01 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 (Sanofi Genzyme) of sarilumab (SAR; Kevzara®) to submit evidence of its clinical effectiveness and cost-effectiveness for previously treated moderate or severe rheumatoid arth...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.1007/s40273-018-0677-7
更新日期:2018-12-01 00:00:00
abstract::Rufinamide (Inovelon®), a triazole derivative, is an oral antiepileptic drug approved in the EU as adjunctive therapy in the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) in patients aged ≥4 years. The efficacy of oral rufinamide as adjunctive therapy in patients with LGS uncontrolled on one to t...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/11208630-000000000-00000
更新日期:2012-03-01 00:00:00