Abstract:
BACKGROUND:Pharmaceuticals are usually granted a marketing authorisation on the basis of randomised controlled trials (RCTs). Occasionally the efficacy of a treatment is assessed without a randomised comparator group (either active or placebo). OBJECTIVE:To identify and develop a taxonomic account of economic modelling approaches for pharmaceuticals licensed without RCT data. METHODS:We searched PubMed, the websites of UK health technology assessment bodies and the International Society for Pharmacoeconomics and Outcomes Research Scientific Presentations Database for assessments of treatments granted a marketing authorisation by the US Food and Drug Administration or European Medicines Agency from January 1999 to May 2014 without RCT data (74 indications). The outcome of interest was the approach to modelling efficacy data. RESULTS:Fifty-one unique models were identified in 29 peer-reviewed articles, 30 health technology appraisals, and 15 International Society for Pharmacoeconomics and Outcomes Research abstracts concerning 30 indications (44 indications had not been modelled). We noted the high rate of non-submission to health technology assessment agencies (28/98). The majority of models (43/51) were based on 'historical controls'-comparisons to previous meta-analysis or pooling of trials (5), individual trials (16), registries/case series (15), or expert opinion (7). Other approaches used the patient as their own control, performed threshold analysis, assumed time on treatment was added to overall survival, or performed cost-minimisation analysis. CONCLUSIONS:There is considerable variation in the quality and approach of models constructed for drugs granted a marketing authorisation without a RCT. The most common approach is of a naive comparison to historical data (using other trials/registry data as a control group), which has considerable scope for bias.
journal_name
Pharmacoeconomicsjournal_title
PharmacoEconomicsauthors
Hatswell AJ,Freemantle N,Baio Gdoi
10.1007/s40273-016-0460-6subject
Has Abstractpub_date
2017-02-01 00:00:00pages
163-176issue
2eissn
1170-7690issn
1179-2027pii
10.1007/s40273-016-0460-6journal_volume
35pub_type
杂志文章,评审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::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:BACKGROUND:Recent randomised trials have demonstrated a statistically significant effect of trastuzumab on disease-free survival when used as adjuvant therapy for human epidermal growth factor receptor 2 protein (HER2)-positive resectable early stage breast cancer, with a treatment course lasting either 9 or 52 weeks. ...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200725050-00006
更新日期:2007-01-01 00:00:00
abstract::Total hip and knee replacements (THR and TKR) are high-risk settings for venous thromboembolism (VTE). This review summarizes the cost effectiveness of VTE prophylaxis regimens for THR and TKR. We searched MEDLINE (January 1997 to October 2009), EMBASE (January 1997 to June 2009) and the UK NHS Economic Evaluation Dat...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/11535210-000000000-00000
更新日期:2010-01-01 00:00:00
abstract:INTRODUCTION:Recent clinical trial results have demonstrated that, in patients with type 2 diabetes, second-line treatment of rosiglitazone in combination with metformin can lead to significant improvements in the control of fasting plasma glucose/ glycosylated haemoglobin A1c (HbA1c) after the failure of metformin mon...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-200624001-00003
更新日期:2006-01-01 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 prospective sequential multinational (5 countries) study was to concurrently evaluate the effects of subcutaneous sumatriptan on clinical parameters, health-related quality-of-life (HRQOL) measures, workplace productivity and patient satisfaction. This report presents the HRQOL results. 582 patients (a...
journal_title:PharmacoEconomics
pub_type: 临床试验,杂志文章,多中心研究
doi:10.2165/00019053-199700111-00005
更新日期:1997-01-01 00:00:00
abstract::In this article, we model the behavior of a pharmaceutical firm that has marketing authorization for a new therapy believed to be a candidate for personalized use in a subset of patients, but that lacks information as to why a response is seen only in some patients. We characterize the optimal outcome-based reimbursem...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-018-0619-4
更新日期:2018-07-01 00:00:00
abstract:BACKGROUND:In Australia, many patients who are initiated on asthma controller inhalers receive combination inhaled corticosteroid/long-acting beta2-agonist (ICS/LABA) despite having asthma of sufficiently low severity that ICS-alone would be equally effective and less costly for the government. METHODS:We conducted a ...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-018-0731-5
更新日期:2019-02-01 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::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:BACKGROUND:Candidaemia and other forms of invasive candidiasis (C/IC) are serious and costly events for hospitalized patients, particularly those in the ICU. Both fluconazole and the echinocandins are recommended as first-line therapy for C/IC. Resource use and cost considerations are important in selecting appropriate...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/11584810-000000000-00000
更新日期:2011-08-01 00:00:00
abstract:OBJECTIVE:Cost is an issue when prescribing two drugs with equivalent efficacy. We compared the direct medical costs of topical brinzolamide 1% (twice a day or three times daily) with topical dorzolamide 2% (twice a day or three times daily) in France, Italy, Portugal and Spain in patients with ocular hypertension or p...
journal_title:PharmacoEconomics
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.2165/00019053-200321030-00005
更新日期:2003-01-01 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: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:OBJECTIVE:To assess the economic efficiency of meloxicam, a cyclo-oxygenase (COX)-2 selective inhibitor, versus diclofenac and piroxicam in the UK for the treatment of patients with osteoarthritis and the impact on the NHS budget of substituting nonselective NSAIDs with meloxicam. Methods and perspective: A decision an...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200321060-00007
更新日期:2003-01-01 00:00:00
abstract::For practical reasons, in order to carry out economic evaluations of collective decisions, total costs will generally be compared with total benefits; hence, individuals' willingness to pay (WTP) or quality-adjusted life-years (QALYs) have to be estimated at an aggregate level. So far, aggregation has usually been don...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-200119040-00001
更新日期:2001-01-01 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::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:Economic evaluations of new diabetes therapies rely heavily upon the UK Prospective Diabetes Study (UKPDS) equations for prediction of cardiovascular events; however, concerns persist regarding their relevance to current clinical practice and appropriate use in populations other than newly diagnosed patients....
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-014-0225-z
更新日期:2015-02-01 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: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::In the current climate of cost containment and quality control, nosocomial infection is a worrisome adverse event in hospital care. Hospitalised patients require care for increasingly severe illnesses, and are therefore more susceptible to infection, especially by opportunistic micro-organisms. It is thus necessary to...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-199507020-00005
更新日期:1995-02-01 00:00:00
abstract::Until a few years ago, griseofulvin and ketoconazole were the only 2 oral agents available for the treatment of dermatophyte onychomycosis of the toenails. With the availability of the newer antifungal agents, such as itraconazole, terbinafine and fluconazole, the armamentarium of drugs available to treat onychomycosi...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-199813020-00007
更新日期:1998-02-01 00:00:00
abstract::Non-melanoma skin cancer (NMSC) and actinic keratosis are becoming an increasingly important healthcare problem. There are approximately 1 million cases of NMSC in the US each year, primarily basal cell carcinomas, and the incidence is increasing. Although NMSC is significant in terms of both health risk and the resou...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-200422020-00002
更新日期:2004-01-01 00:00:00
abstract:BACKGROUND:Given the association between CD4 cell counts and HIV-related morbidity/mortality, new antiretroviral therapies could potentially lower the direct costs of HIV care by raising CD4 cell counts. OBJECTIVES:To predict the effects of the ritonavir-boosted, HIV protease inhibitor (PI) darunavir on the direct cos...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/11587510-000000000-00000
更新日期:2010-01-01 00:00:00
abstract::The cost of the development of a new drug product, from concept and synthesis of a new chemical entity to safety and efficacy testing, to the approval process and postmarketing surveillance, has more than quadrupled in the last 20 years. To amortise these costs, the pharmaceutical industry has taken on international ...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-199202050-00005
更新日期:1992-11-01 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:OBJECTIVE:The aim of the present study is to describe a refinement of a previously presented method, based on the concept of point sensitivity, to deal with uncertainty in economic studies. DESIGN:The original method was refined by the incorporation of probability distributions which allow a more accurate assessment o...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-199916010-00004
更新日期:1999-07-01 00:00:00
abstract:OBJECTIVE:To develop incidence-based estimates of the cost of several diabetes-related complications. DESIGN AND SETTING:This was a retrospective cohort study in a large health maintenance organisation. A total of 8905 patients with type 1 (insulin-dependent) and type 2 (non-insulin-dependent) diabetes mellitus and 36...
journal_title:PharmacoEconomics
pub_type: 临床试验,杂志文章
doi:10.2165/00019053-199916030-00005
更新日期:1999-09-01 00:00:00