Abstract:
INTRODUCTION:Mixture modelling is increasingly being considered where a potential cure leads to a long life. Traditional methods use relative survival models for frail populations or cure models that have improper survival functions with theoretical infinite lifespans. Additionally, much of the work uses population data with long follow-up or theoretical data for method development. OBJECTIVE:This case study uses life table data to create a proper survival function in a real-world clinical trial context. In particular, we discuss the impact of the length of trial follow-up on the accuracy of model estimation and the impact of extrapolation to capture long-term survival. METHODS:A review of recent National Institute for Health and Clinical Excellence (NICE) immuno-oncological and chimeric antigen receptor (CAR) T-cell therapy submissions was performed to assess industry uptake and NICE acceptance of survival analysis methods incorporating the potential for long-term survivorship. The case study analysed a simulated trial-based dataset investigating a curative treatment with long-term mortality based on population life tables. The analysis examined three timepoints corresponding to early trial, end-of-trial follow-up and complete follow-up. Mixture modelling approaches were considered, including both cure modelling and relative survival approaches. The curves were evaluated based on the ability to estimate cure fractions and mean life in years within the time span the models are based on and when extrapolating to capture long-term behaviour. The survival curves were fitted with Weibull distributions using non-mixture and mixture cure models. RESULTS:The performance of the cure modelling methods depended on the relative maturity of the data, indicating that care is needed when deciding when the methods should be applied. For progression-free survival, the cure fraction simulated was 15%. The cure fractions estimated using the traditional mixture cure model were 43% (95% confidence interval [CI] 30-57) at the first analysis time point (40 months), 15% (95% CI 12-20) at the end-of-study follow-up (153 months) and 0% (95% CI 0-100) at the end of follow-up. Other standard cure modelling methods produced similar results. For overall survival, we observed a similar pattern of goodness of fit, with a good fit for the end-of-study follow-up and poor fit for the other two data cuts. However, in this case, the estimate of the cure fraction was below the true value in the first analysis data. CONCLUSIONS:This case study suggests cure modelling works well with data in which the disease-specific events have had time to occur. Care is needed when extrapolating from immature data, and further information should support the estimation rather than relying on statistical estimates based on the trial alone.
journal_name
Pharmacoeconomicsjournal_title
PharmacoEconomicsauthors
Grant TS,Burns D,Kiff C,Lee Ddoi
10.1007/s40273-019-00867-5subject
Has Abstractpub_date
2020-04-01 00:00:00pages
385-395issue
4eissn
1170-7690issn
1179-2027pii
10.1007/s40273-019-00867-5journal_volume
38pub_type
杂志文章abstract::The role of economic evaluation in the efficient allocation of healthcare resources has been widely debated. Whilst economic evidence is undoubtedly useful to purchasers, it does not address the issue of affordability which is an increasing concern. Healthcare purchasers are concerned not just with maximising efficien...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-200119060-00001
更新日期:2001-01-01 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::Fluconazole (FLU) is an alternative to amphotericin B (AMB) for the treatment of candidemia in non-neutropenic patients. This agent has similar clinical efficacy but significantly reduced adverse effects compared with AMB. Using the database from a Canadian randomised multicentre comparative trial of FLU versus AMB in...
journal_title:PharmacoEconomics
pub_type: 临床试验,杂志文章,多中心研究,随机对照试验
doi:10.2165/00019053-199813050-00004
更新日期:1998-05-01 00:00:00
abstract:BACKGROUND:The symptoms of attention-deficit hyperactivity disorder (ADHD) are associated with impairment in multiple domains of health-related quality of life (HR-QOL). HR-QOL of children with ADHD has been assessed by relatively long multidimensional questionnaires. A review of the literature found no studies using t...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200523080-00004
更新日期:2005-01-01 00:00:00
abstract:BACKGROUND:Mapping Patient-Reported Outcomes Measurement Information System-Global Health (PROMIS-GH) to EuroQol 5-dimension, three-level version (EQ-5D-3L) provides a utility score for use in quality-of-life and cost-effectiveness analyses. In 2009, Revicki et al. mapped the PROMIS-GH items to EQ-5D-3L utilities using...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-017-0541-1
更新日期:2017-11-01 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:BACKGROUND:The measurement and valuation of health-related quality of life for and by young people are increasingly important, yet research on the impact of study perspective and validity of preferences obtained from young populations remains limited. OBJECTIVE:The objective of this study was to evaluate the feasibili...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-020-00884-9
更新日期:2020-05-01 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
abstract::This study evaluated the cost of sequential treatment with once-daily ofloxacin or twice-daily ciprofloxacin in 474 hospitalised patients in different countries. The patients were treated intravenously for at least 3 days, then orally for 7 to 10 days or for 3 days beyond the disappearance of infection-related symptom...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-199711040-00007
更新日期:1997-04-01 00:00:00
abstract::The objective of this study was to compare the cost effectiveness of amoxicillin/clavulanic acid with other antibacterial regimens for prophylaxis of infection after elective abdominal or gynaecological surgery. Data from 21 previously published comparative clinical trials were used to calculate statistical confidence...
journal_title:PharmacoEconomics
pub_type: 临床试验,杂志文章
doi:10.2165/00019053-199507040-00008
更新日期:1995-04-01 00:00:00
abstract::Answering the demanding questions asked of pharmacotherapy in the 21st century will require more reliance on the disciplines of pharmacoepidemiology and pharmacoeconomics. Pharmacoepidemiology can help assess patterns and appropriateness of drug utilisation, provide explanations for poor compliance, quantify the frequ...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-200422002-00009
更新日期:2004-01-01 00:00:00
abstract:BACKGROUND AND AIM:Buprenorphine offers an alternative to methadone in the treatment of heroin dependence, and has the advantage of allowing alternate-day dosing. This study is the first to examine the cost effectiveness of buprenorphine as maintenance treatment for heroin dependence in a primary care setting using eco...
journal_title:PharmacoEconomics
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.2165/00019053-200523010-00007
更新日期:2005-01-01 00:00:00
abstract::There is a global perspective to the economic consequences of diabetes, which is illustrated in the many publications now being produced on this subject. Because of the higher prevalence of type II diabetes, its impact is greater than that of type I diabetes in both developed and developing countries (but particularly...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-199500081-00017
更新日期:1995-01-01 00:00:00
abstract::There is an inevitable tension between robust reimbursement processes and providing speedy access to new and novel technologies, given uncertainties about key pieces of evidence and subsequent concerns regarding their overall efficiency. The public perception of these treatments as 'breakthrough', combined with substa...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/11530850-000000000-00000
更新日期:2010-01-01 00:00:00
abstract:BACKGROUND:Dengue disease poses a great economic burden in Malaysia. METHODS:This study evaluated the cost effectiveness and impact of dengue vaccination in Malaysia from both provider and societal perspectives using a dynamic transmission mathematical model. The model incorporated sensitivity analyses, Malaysia-speci...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-017-0487-3
更新日期:2017-05-01 00:00:00
abstract::The aim of this article was to perform a detailed methodological review of models used to estimate the cost effectiveness of drug treatment regimens for HIV infection in Europe and North America and assess the relationship between the different modeling approaches or key structural assumptions and the results. Electro...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.1007/s40273-013-0098-6
更新日期:2013-11-01 00:00:00
abstract::Our objective was to assess the quality of reporting of original economic research articles in PharmacoEconomics from inception to the end of 1995, in order to identify areas of strength and weakness, and analyse trends over time. Each regular issue of the journal was examined for original economic evaluations. Accept...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-199712060-00008
更新日期:1997-12-01 00:00:00
abstract:OBJECTIVE:Our objective was to identify, among the information routinely collected on patients in intensive care units (ICUs), data that determine the total cost for a given patient. DESIGN:We developed a model that could help physicians in medical ICUs to estimate the cost of care for their patients when no cost data...
journal_title:PharmacoEconomics
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.2165/00019053-199915060-00005
更新日期:1999-06-01 00:00:00
abstract:BACKGROUND:Carcinoid syndrome, a rare condition in patients with neuroendocrine tumours, characterised by flushing and diarrhoea, severely affects patients' quality of life. The current carcinoid syndrome standard of care includes somatostatin analogues, but some patients experience uncontrolled symptoms despite somato...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-020-00896-5
更新日期:2020-06-01 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::Irritable bowel syndrome (IBS) is one of the most common functional gastrointestinal disorders, with an estimated prevalence rate in the general population of 10-15% in industrialised countries. Although IBS is not a life-threatening disease, it contributes significantly to a large segment of healthcare resource consu...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-200624010-00002
更新日期:2006-01-01 00:00:00
abstract::Open-heart surgery (OHS) is performed to bypass occluded arteries, replace malfunctioning cardiac valves or correct congenital abnormalities. The average cost of OHS varies from $US25 057-$US79 795 (1997 values). The objective of this paper was to review economic studies of pharmacological strategies in open-heart sur...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-200321040-00003
更新日期:2003-01-01 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 National Institute for Health and Clinical Excellence (NICE) invited the manufacturer of denosumab (Amgen Inc., UK) to submit evidence for the clinical and cost effectiveness of denosumab for the prevention of fragility fractures in post-menopausal women, as part of the Institute's single technology appraisal (STA...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/11589310-000000000-00000
更新日期:2011-11-01 00:00:00
abstract:BACKGROUND:Several vaccine and antibody candidates are currently in development for the prevention of lower respiratory tract infections caused by the respiratory syncytial virus (RSV). METHODS:We searched MEDLINE, Embase, and SCOPUS and included model-based evaluations of RSV vaccinations. Two reviewers performed the...
journal_title:PharmacoEconomics
pub_type:
doi:10.1007/s40273-020-00991-7
更新日期:2021-01-19 00:00:00
abstract:BACKGROUND:Of 1 346 700 total deaths each year in the US, an estimated 440 100 are smoking related, making it the leading preventable cause of premature death in the US. Despite the health and economic benefits of smoking cessation being well documented, reimbursement coverage for smoking cessation therapies is general...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200826060-00004
更新日期:2008-01-01 00:00:00
abstract::Which costs and benefits to consider in economic evaluations of healthcare interventions remains an area of much controversy. Unrelated medical costs in life-years gained is an important cost category that is normally ignored in economic evaluations, irrespective of the perspective chosen for the analysis. National gu...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-200826100-00003
更新日期:2008-01-01 00:00:00
abstract:OBJECTIVE:The objective of this study was to assess the cost effectiveness of using epoetin-alpha (erythropoietin) to augment preoperative autologous donation (PAD) of blood prior to elective cardiac surgery. DESIGN AND SETTING:We designed a decision-analytic model incorporating the risk of receiving allogeneic blood,...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200018020-00006
更新日期:2000-08-01 00:00:00
abstract::Epidural analgesia and intravenous analgesia with opioids are two techniques for providing pain relief for women in labour. Labour pain is comparable to surgical pain in its severity, and epidural analgesia provides better relief from this pain than intravenous analgesia; a meta-analysis quantified this improvement to...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-200220050-00002
更新日期:2002-01-01 00:00:00