Abstract:
BACKGROUND AND OBJECTIVE:Patient heterogeneity is the part of variability that can be explained by certain patient characteristics (e.g. age, disease stage). Population reimbursement decisions that acknowledge patient heterogeneity could potentially save money and increase population health. To date, however, economic evaluations pay only limited attention to patient heterogeneity. The objective of the present paper is to provide a comprehensive overview of the current knowledge regarding patient heterogeneity within economic evaluation of healthcare programmes. METHODS:A systematic literature review was performed to identify methodological papers on the topic of patient heterogeneity in economic evaluation. Data were obtained using a keyword search of the PubMed database and manual searches. Handbooks were also included. Relevant data were extracted regarding potential sources of patient heterogeneity, in which of the input parameters of an economic evaluation these occur, methods to acknowledge patient heterogeneity and specific concerns associated with this acknowledgement. RESULTS:A total of 20 articles and five handbooks were included. The relevant sources of patient heterogeneity (demographics, preferences and clinical characteristics) and the input parameters where they occurred (baseline risk, treatment effect, health state utility and resource utilization) were combined in a framework. Methods were derived for the design, analysis and presentation phases of an economic evaluation. Concerns related mainly to the danger of false-positive results and equity issues. CONCLUSION:By systematically reviewing current knowledge regarding patient heterogeneity within economic evaluations of healthcare programmes, we provide guidance for future economic evaluations. Guidance is provided on which sources of patient heterogeneity to consider, how to acknowledge them in economic evaluation and potential concerns. The improved acknowledgement of patient heterogeneity in future economic evaluations may well improve the efficiency of healthcare.
journal_name
Pharmacoeconomicsjournal_title
PharmacoEconomicsauthors
Grutters JP,Sculpher M,Briggs AH,Severens JL,Candel MJ,Stahl JE,De Ruysscher D,Boer A,Ramaekers BL,Joore MAdoi
10.1007/s40273-012-0015-4subject
Has Abstractpub_date
2013-02-01 00:00:00pages
111-23issue
2eissn
1170-7690issn
1179-2027journal_volume
31pub_type
杂志文章,评审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: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...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.1007/s40273-016-0397-9
更新日期:2016-08-01 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::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::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::Discrete choice experiments (DCEs) are regularly used in health economics to elicit preferences for healthcare products and programmes. There is growing recognition that DCEs can provide more than information on preferences and, in particular, they have the potential to contribute more directly to outcome measurement ...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200826080-00004
更新日期:2008-01-01 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
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:OBJECTIVE:The objective of this study was to examine the cost effectiveness of fluvoxamine compared with tricyclic antidepressants (TCAs) in the treatment of patients with depressive episodes. DESIGN AND SETTING:A Markov process model was constructed to model the effectiveness, as measured by time without depression, ...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-199814040-00009
更新日期:1998-10-01 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:BACKGROUND AND OBJECTIVE:Glycoprotein (GP) IIb/IIIa receptor inhibitors enhance thrombolysis in patients with acute coronary syndromes. This analysis evaluates the economic impact of abciximab, a GP IIb/IIIa inhibitor, as an adjunct to urokinase in peripheral artery occlusions of less than 6 weeks duration. STUDY DESI...
journal_title:PharmacoEconomics
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.2165/00019053-200220030-00006
更新日期:2002-01-01 00:00:00
abstract:BACKGROUND:The apparent decrease in the rate of approval of new molecular entities has provoked extensive discussion and fears that the productivity of biopharmaceutical research and development has severely declined in recent years. OBJECTIVE:To investigate the extent to which traditional measures of innovative outpu...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-200624002-00008
更新日期:2006-12-01 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: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 willingness-to-pay (WTP) analysis is the first study of its kind undertaken in Australia to support an application for listing of a new drug on the Australian national formulary. The technique offers the advantage of being able to summarise diverse outcomes of therapy in a single unit of measure. Willingness to p...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/00019053-199813030-00009
更新日期:1998-03-01 00:00:00
abstract::Deterministic sensitivity analyses (DSA) remain important to interpret the effect of uncertainties in individual parameters on results of cost-effectiveness analyses. Classic DSA methodologies may lead to wrong conclusions due to a lack of or misleading information regarding marginal effects, non-linearity, likelihood...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-020-00979-3
更新日期:2021-01-01 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::The purpose of this paper is to highlight important considerations in the measurement of health-related quality of life (HR-QOL) in paediatric populations. Considerations specific to the evaluation of HR-QOL in children include children's understanding of the questions being asked, their understanding of their own dis...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-199916060-00002
更新日期:1999-12-01 00:00:00
abstract:BACKGROUND:In many healthcare systems, affordability concerns can lead to restrictions on the use of expensive efficacious therapies. However, there does not appear to be any consensus as to the terminology used to describe affordability, or the thresholds used to determine whether new drugs are affordable. OBJECTIVES...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.1007/s40273-017-0514-4
更新日期:2017-08-01 00:00:00
abstract::The development of evidence to demonstrate 'value for money' is regarded as an important step in facilitating the search for the optimal allocation of limited resources and has become an essential component in healthcare decision making. Real-world evidence collected from de-identified individuals throughout the conti...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-020-00927-1
更新日期:2020-10-01 00:00:00
abstract::To review and critically evaluate published cost-utility analyses (CUAs) pertaining to pharmaceuticals for the past 3 decades. We examined data from the Tufts Medical Center Cost-Effectiveness Analysis Registry (www.cearegistry.org), which contains detailed information on English-language CUAs and their ratios (in $US...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.2165/11312720-000000000-00000
更新日期:2009-01-01 00:00:00
abstract::Cost-effectiveness analysis (CEA) is one of the main tools of economic evaluation. Every CEA is based on a number of assumptions, some of which may not be accurate, introducing uncertainty. Sensitivity analysis (SA) formalizes ways to measure and evaluate this uncertainty. Specific sources of uncertainty in CEA have b...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/11584630-000000000-00000
更新日期:2011-04-01 00:00:00
abstract:OBJECTIVE:The objective of this article was to summarize the findings of all the available studies on alternative pharmacological treatments for asthma and assess their methodological quality, as well as to identify the main drivers of the cost effectiveness of pharmacological treatments for the disease. METHODS:A sys...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-018-0668-8
更新日期:2018-10-01 00:00:00
abstract::Variable compliance with prescribed drug regimens is a leading source of variability in drug response. Specifics differ by drug and disease. The role of variable compliance was clearly defined in 2 trials of lipid-lowering agents, cholestyramine and gemfibrozil, in which exceptionally careful measurements of complianc...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-199915030-00002
更新日期:1999-03-01 00:00:00
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: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::As a consequence of its prevalence, early onset and chronicity, schizophrenia imposes clinical and economic impediments to healthcare practitioners and society alike. Among the many antipsychotics available to treat the symptoms of this devastating illness, clozapine has emerged and differentiated itself from the othe...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-200321010-00001
更新日期:2003-01-01 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::Pharmacoeconomic data may be obtained within the context of randomised clinical trials (RCTs) and from effectiveness studies in the 'real world'. The differences between the 2 types of study design have implications for the types of data that can be obtained and the interpretation of the resulting findings. Because RC...
journal_title:PharmacoEconomics
pub_type: 杂志文章,评审
doi:10.2165/00019053-199915050-00001
更新日期:1999-05-01 00:00:00
abstract:BACKGROUND:Simulation models utilizing real-world data have potential to optimize treatment sequencing strategies for specific patient subpopulations, including when conducting clinical trials is not feasible. We aimed to develop a simulation model to estimate progression-free survival (PFS) and overall survival for fi...
journal_title:PharmacoEconomics
pub_type: 杂志文章
doi:10.1007/s40273-020-00951-1
更新日期:2020-11-01 00:00:00