A Systematic Review of the Cost-Effectiveness of Recombinant Factor VIIa (rFVIIa) and Activated Prothrombin Complex Concentrate (aPCC) in the Treatment of Mild to Moderate Bleeding Episodes for Haemophilia Patients with Inhibitors

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 4678-4678
Author(s):  
Chris Knight ◽  
Anne Møller Danø ◽  
Tessa Kennedy-Martin

Abstract Objectives: Although haemophilia patients with inhibitors are rare, the clinical, humanistic and economic consequences associated with this disorder are considerable. The primary treatment for such patients is either rFVIIa or aPCC. The aim of this study was to identify, review and evaluate the quality of the published literature on the relative cost-effectiveness of rFVIIa and aPCC in treating haemophilia patients with inhibitors. Methods: The review concentrates on the model type, the model design, model assumptions, and results. Results: The results of this study suggest that rFVIIa may be the cost-effective alternative to treatment with aPCC due to the superior efficacy of rFVIIa and hence the avoidance of subsequent lines of treatment. In 7 of the 9 studies, rFVIIa had the lower average treatment cost. The adapted modelling framework is similar in all the economic models reviewed, suggesting clinical acceptability of the approach used. The estimates of efficacy varied between the models, especially for aPCC. The efficacy for aPCC derived from retrospective studies was lower than reported in the literature. Sensitivity analysis had been undertaken in the majority of the economic analyses and the results were found to be robust to realistic parameter variations. Only one of the studies was a cost-utility study, showing the lack of measuring health status within this area. The results showed the large impact appropriate treatment can have on the quality of life for haemophilia patients with inhibitors. Conclusions: Ideally, there should be a systematic approach to identifying the relevant data and the lack of data from relevant randomized head-to-head trials is a contributing factor to the variation in efficacy rates and average dosages assumed. However, this systematic review has shown that despite differences in the estimates of efficacy, average dosage required, and unit costs the overall results are robust and appear to favour rFVIIa as the cost-effectiveness treatment for haemophilia patients with inhibitors.

BMJ Open ◽  
2021 ◽  
Vol 11 (9) ◽  
pp. e048141
Author(s):  
Sara Mucherino ◽  
Valentina Lorenzoni ◽  
Valentina Orlando ◽  
Isotta Triulzi ◽  
Marzia Del Re ◽  
...  

IntroductionThe combination of biomarkers and drugs is the subject of growing interest both from regulators, physicians and companies. This study protocol of a systematic review is aimed to describe available literature evidences about the cost-effectiveness, cost-utility or net-monetary benefit of the use of biomarkers in solid tumour as tools for customising immunotherapy to identify what further research needs.Methods and analysisA systematic review of the literature will be carried out according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses statement guidelines. PubMed and Embase will be queried from June 2010 to June 2021. The PICOS model will be applied: target population (P) will be patients with solid tumours treated with immune checkpoint inhibitors (ICIs); the interventions (I) will be test of the immune checkpoint predictive biomarkers; the comparator (C) will be any other targeted or non-targeted therapy; outcomes (O) evaluated will be health economic and clinical implications assessed in terms of incremental cost-effectiveness ratio, net health benefit, net monetary benefit, life years gained, quality of life, etc; study (S) considered will be economic evaluations reporting cost-effectiveness analysis, cost-utility analysis, net-monetary benefit. The quality of the evidence will be graded according to Grading of Recommendations Assessment, Development and Evaluation.Ethics and disseminationThis systematic review will assess the cost-effectiveness implications of using biomarkers in the immunotherapy with ICIs, which may help to understand whether this approach is widespread in real clinical practice. This research is exempt from ethics approval because the work is carried out on published documents. We will disseminate this protocol in a related peer-reviewed journal.PROSPERO registration numberCRD42020201549.


2021 ◽  
Author(s):  
Padraig Dixon ◽  
Edna Keeney ◽  
Jenny C Taylor ◽  
Sarah Wordsworth ◽  
Richard Martin

Polygenic risk is known to influence susceptibility to cancer. The use of data on polygenic risk, in conjunction with other predictors of future disease status, may offer significant potential for preventative care through risk-stratified screening programmes. An important element in the evaluation of screening programmes is their cost-effectiveness. We undertook a systematic review of papers evaluating the cost-effectiveness of screening interventions informed by polygenic risk scores compared to more conventional screening modalities. We included papers reporting cost-effectiveness outcomes in the English language published as articles or uploaded onto preprint servers with no restriction on date, type of cancer or form of polygenic risk modelled. We excluded papers evaluating screening interventions that did not report cost-effectiveness outcomes or which had a focus on monogenic risk. We evaluated studies using the Quality of Health Economic Studies checklist. Ten studies were included in the review, which investigated three cancers: prostate (n=5), colorectal (n=3) and breast (n=2). All study designs were cost-utility papers implemented as Markov models (n=6) or microsimulations (n=4). Nine of ten papers scored highly (score >75 on a 0-100) scale) when assessed using the Quality of Health Economic Studies checklist. Eight of ten studies concluded that polygenic risk informed cancer screening was likely to be more cost-effective than alternatives. However, the included studies lacked robust external data on the cost of polygenic risk stratification, did not account for how very large volumes of polygenic risk data on individuals would be collected and used, did not consider ancestry-related differences in polygenic risk, and did not fully account for downstream economic sequalae stemming from the use of polygenic risk data in these ways. These topics merit attention in future research on how polygenic risk data might contribute to cost-effective cancer screening.


2007 ◽  
Vol 23 (4) ◽  
pp. 473-479 ◽  
Author(s):  
Pekka Kuukasjärvi ◽  
Pirjo Räsänen ◽  
Antti Malmivaara ◽  
Pasi Aronen ◽  
Harri Sintonen

Objectives:The aim of this study was to systematically review economic analyses comparing drug-eluting stents (DES) to bare metal stents (BMS) in patients who undergo percutaneous coronary intervention to form an overall view about cost-effectiveness of DES and to construct a simple decision analysis model to evaluate the cost–utility of DES.Methods:Electronic databases searched from January 2004 to January 2006 were Cochrane Database of Systematic Reviews; DARE, HTA, EED (NHS CRD); MEDLINE(R) In-Process, Other Non-Indexed Citations, MEDLINE(R). References of the papers identified were checked. We included randomized controlled trials (RCT) or model-based cost-effectiveness analyses comparing DES to BMS in patients with coronary artery disease. The methodological quality of the papers was assessed by Drummond's criteria. Baseline characteristics and results of the studies were extracted and data synthesized descriptively. A decision tree model was constructed to evaluate the cost–utility of DES in comparison to BMS, where health-related quality of life was measured by the 15D.Results:We identified thirteen good-quality economic evaluations. In two of these based on RCTs, DES was found cost-effective. In six studies, it was concluded that DES might probably be a cost-effective strategy in some circumstances, but not as a single strategy, and four studies concluded that DES is not cost-effective. One study did not draw a clear conclusion. In our analysis, the overall incremental cost-effectiveness ratio was €98,827 per quality-adjusted life-years gained. Avoiding one revascularization with DES would cost €4,794, when revascularization with BMS costs €3,260.Conclusions:The evidence is inconsistent of whether DES would be a cost-effective treatment compared with BMS in any healthcare system where evaluated. A marked restenosis risk reduction should be achieved before use of DES is justifiable at present prices. When considering adoption of a new health technology with a high incremental cost within a fixed budget, opportunity cost in terms of untreated patients should be seriously considered as a question of collective ethics.


Author(s):  
Filipa Sampaio ◽  
Inna Feldman ◽  
Tara A. Lavelle ◽  
Norbert Skokauskas

AbstractEconomic evaluations can help decision makers identify what services for children with neurodevelopmental disorders provide best value-for-money. The aim of this paper is to review the best available economic evidence to support decision making for attention deficit-hyperactivity disorder (ADHD) and autism spectrum disorder (ASD) in children and adolescents. We conducted a systematic review of economic evaluations of ADHD and ASD interventions including studies published 2010–2020, identified through Econlit, Medline, PsychINFO, and ERIC databases. Only full economic evaluations comparing two or more options, considering both costs and consequences were included. The quality of the studies was assessed using the Drummond checklist. We identified ten studies of moderate-to-good quality on the cost-effectiveness of treatments for ADHD and two studies of good quality of interventions for ASD. The majority of ADHD studies evaluated pharmacotherapy (n = 8), and two investigated the economic value of psychosocial/behavioral interventions. Both economic evaluations for ASD investigated early and communication interventions. Included studies support the cost-effectiveness of behavioral parenting interventions for younger children with ADHD. Among pharmacotherapies for ADHD, different combinations of stimulant/non-stimulant medications for children were cost-effective at willingness-to-pay thresholds reported in the original papers. Early intervention for children with suspected ASD was cost-effective, but communication-focused therapy for preschool children with ASD was not. Prioritizing more studies in this area would allow decision makers to promote cost-effective and clinically effective interventions for this target group.


2019 ◽  
Vol 11 (1) ◽  
pp. e2019036 ◽  
Author(s):  
Jialian Li

Background:Deferoxamine (DFO) or Deferiprone (DFP) or Deferasirox (DFX) monotherapy and DFO and DFP combination therapy were four commonly implemented now chelation regimens for the iron overloaded of β-thalassemia major. This systematic review aims to determine the cost-effectiveness of four chelation regimens and provide evidence for the rational use of chelation regimens for β-thalassemia major therapy in clinic.Methods:A systematic literature search in PubMed, EMBASE (Ovid), CENTRAL (Cochrane library), HTAD (Cochrane library), NHS EED (Cochrane library), CBM, CNKI, VIP, and Wanfang was conducted in April 2018. In addition, a manual search was performed. Two researchers, working independently, selected the papers, extracted the data and assessed the methodological quality of the included papers. Each included paper was evaluated using a checklist developed by Drummond et al. Results:The initial number of records was 968, and eight papers met the final eligibility criteria. All the included eight papers were cost-utility analyses. And the methodological quality of these papers was good. Nineteen studies were included in eight papers. Nine studies of DFX versus DFO had contradictory results. Out of the nineteen studies, three studies of DFX versus DFP established that using DFP was cost-effective. Three studies of DFP versus DFO established that using DFP was cost-effective. One study of DFP and DFO combination therapy versus DFO found that using DFO was cost-effective. One study of DFP and DFO combination therapy versus DFP found that using DFP was cost-effective. And there were two studies of DFP and DFO combination therapy versus DFX, but we cannot be sure which one of two chelation regimens was cost-effective. Conclusion:In brief, DFP is the best choice, followed by DFO or DFX, when an iron chelator is to be used alone for β-thalassemia major therapy. All studies that compared DFO and DFP combination therapy with DFO (or DFP or DFX) monotherapy established that the combination therapy with DFO and DFP was not cost-effective. However, due to the low number of related studies, more extensive, high-quality research is required for further analysis and confirmation of our findings. Moreover, the cost effectiveness is not an absolute issue when in different countries(regions) the results are opposite for other countries(regions). The specific region had a substantial influence on the economy of drugs. Key words: β-thalassemia major, Deferoxamine, Deferiprone, Deferasirox, cost-effectiveness, systematic review


2017 ◽  
Vol 21 (80) ◽  
pp. 1-280 ◽  
Author(s):  
Ros Wade ◽  
Stephen Rice ◽  
Alexis Llewellyn ◽  
Eoin Moloney ◽  
Julie Jones-Diette ◽  
...  

BackgroundHyperhidrosis is uncontrollable excessive sweating that occurs at rest, regardless of temperature. The symptoms of hyperhidrosis can significantly affect quality of life. The management of hyperhidrosis is uncertain and variable.ObjectiveTo establish the expected value of undertaking additional research to determine the most effective interventions for the management of refractory primary hyperhidrosis in secondary care.MethodsA systematic review and economic model, including a value-of-information (VOI) analysis. Treatments to be prescribed by dermatologists and minor surgical treatments for hyperhidrosis of the hands, feet and axillae were reviewed; as endoscopic thoracic sympathectomy (ETS) is incontestably an end-of-line treatment, it was not reviewed further. Fifteen databases (e.g. CENTRAL, PubMed and PsycINFO), conference proceedings and trial registers were searched from inception to July 2016. Systematic review methods were followed. Pairwise meta-analyses were conducted for comparisons between botulinum toxin (BTX) injections and placebo for axillary hyperhidrosis, but otherwise, owing to evidence limitations, data were synthesised narratively. A decision-analytic model assessed the cost-effectiveness and VOI of five treatments (iontophoresis, medication, BTX, curettage, ETS) in 64 different sequences for axillary hyperhidrosis only.Results and conclusionsFifty studies were included in the effectiveness review: 32 randomised controlled trials (RCTs), 17 non-RCTs and one large prospective case series. Most studies were small, rated as having a high risk of bias and poorly reported. The interventions assessed in the review were iontophoresis, BTX, anticholinergic medications, curettage and newer energy-based technologies that damage the sweat gland (e.g. laser, microwave). There is moderate-quality evidence of a large statistically significant effect of BTX on axillary hyperhidrosis symptoms, compared with placebo. There was weak but consistent evidence for iontophoresis for palmar hyperhidrosis. Evidence for other interventions was of low or very low quality. For axillary hyperhidrosis cost-effectiveness results indicated that iontophoresis, BTX, medication, curettage and ETS was the most cost-effective sequence (probability 0.8), with an incremental cost-effectiveness ratio of £9304 per quality-adjusted life-year. Uncertainty associated with study bias was not reflected in the economic results. Patients and clinicians attending an end-of-project workshop were satisfied with the sequence of treatments for axillary hyperhidrosis identified as being cost-effective. All patient advisors considered that the Hyperhidrosis Quality of Life Index was superior to other tools commonly used in hyperhidrosis research for assessing quality of life.LimitationsThe evidence for the clinical effectiveness and safety of second-line treatments for primary hyperhidrosis is limited. This meant that there was insufficient evidence to draw conclusions for most interventions assessed and the cost-effectiveness analysis was restricted to hyperhidrosis of the axilla.Future workBased on anecdotal evidence and inference from evidence for the axillae, participants agreed that a trial of BTX (with anaesthesia) compared with iontophoresis for palmar hyperhidrosis would be most useful. The VOI analysis indicates that further research into the effectiveness of existing medications might be worthwhile, but it is unclear that such trials are of clinical importance. Research that established a robust estimate of the annual incidence of axillary hyperhidrosis in the UK population would reduce the uncertainty in future VOI analyses.Study registrationThis study is registered as PROSPERO CRD42015027803.FundingThe National Institute for Health Research Health Technology Assessment programme.


2014 ◽  
Vol 2014 ◽  
pp. 1-28 ◽  
Author(s):  
Faith Donald ◽  
Kelley Kilpatrick ◽  
Kim Reid ◽  
Nancy Carter ◽  
Ruth Martin-Misener ◽  
...  

Background. Improved quality of care and control of healthcare costs are important factors influencing decisions to implement nurse practitioner (NP) and clinical nurse specialist (CNS) roles.Objective. To assess the quality of randomized controlled trials (RCTs) evaluating NP and CNS cost-effectiveness (defined broadly to also include studies measuring health resource utilization).Design. Systematic review of RCTs of NP and CNS cost-effectiveness reported between 1980 and July 2012.Results. 4,397 unique records were reviewed. We included 43 RCTs in six groupings, NP-outpatient (n=11), NP-transition (n=5), NP-inpatient (n=2), CNS-outpatient (n=11), CNS-transition (n=13), and CNS-inpatient (n=1). Internal validity was assessed using the Cochrane risk of bias tool; 18 (42%) studies were at low, 17 (39%) were at moderate, and eight (19%) at high risk of bias. Few studies included detailed descriptions of the education, experience, or role of the NPs or CNSs, affecting external validity.Conclusions. We identified 43 RCTs evaluating the cost-effectiveness of NPs and CNSs using criteria that meet current definitions of the roles. Almost half the RCTs were at low risk of bias. Incomplete reporting of study methods and lack of details about NP or CNS education, experience, and role create challenges in consolidating the evidence of the cost-effectiveness of these roles.


2021 ◽  
Vol 9 (3) ◽  
pp. 232596712098753
Author(s):  
Cammille C. Go ◽  
Cynthia Kyin ◽  
Jeffrey W. Chen ◽  
Benjamin G. Domb ◽  
David R. Maldonado

Background: Hip arthroscopy has frequently been shown to produce successful outcomes as a treatment for femoroacetabular impingement (FAI) and labral tears. However, there is less literature on whether the favorable results of hip arthroscopy can justify the costs, especially when compared with a nonoperative treatment. Purpose: To systematically review the cost-effectiveness of hip arthroscopy for treating FAI and labral tears. Study Design: Systematic review; Level of evidence, 3. Methods: PubMed/MEDLINE, Embase, and Cochrane Library databases, and the Tufts University Cost-Effectiveness Analysis Registry were searched to identify articles that reported the cost per quality-adjusted life-year (QALY) generated by hip arthroscopy. The key terms used were “hip arthroscopy,” “cost,” “utility,” and “economic evaluation.” The threshold for cost-effectiveness was set at $50,000/QALY. The Methodological Index for Non-Randomized Studies instrument and Quality of Health Economic Studies (QHES) score were used to determine the quality of the studies. This study was prospectively registered on PROSPERO (CRD42020172991). Results: Six studies that reported the cost-effectiveness of hip arthroscopy were identified, and 5 of these studies compared hip arthroscopy to a nonoperative comparator. These studies were found to have a mean QHES score of 85.2 and a mean cohort age that ranged from 33-37 years. From both a health care system perspective and a societal perspective, 4 studies reported that hip arthroscopy was more costly but resulted in far greater gains than did nonoperative treatment. The preferred treatment strategy was most sensitive to duration of benefit, preoperative osteoarthritis, cost of the arthroscopy, and the improvement in QALYs with hip arthroscopy. Conclusion: In the majority of the studies, hip arthroscopy had a higher initial cost but provided greater gain in QALYs than did a nonoperative treatment. In certain cases, hip arthroscopy can be cost-effective given a long enough duration of benefit and appropriate patient selection. However, there is further need for literature to analyze willingness-to-pay thresholds.


Health Scope ◽  
2021 ◽  
Vol 10 (2) ◽  
Author(s):  
Maryam Khoramrooz ◽  
Asra Asgharzadeh ◽  
Saeide Alidoost ◽  
Zeynab Foroughi ◽  
Saber Azami ◽  
...  

Context: Stroke is one of the main causes of premature death and disability, imposing significant costs on the healthcare system, especially due to expensive hospital care. Home care service is one of the interventions used in the last two decades to reduce the cost of services provided for stroke patients in different countries. Objectives: The present study aimed to systematically review studies related to the economic evaluation of home care compared to hospital care for stroke patients. Data Sources: A search was conducted between January 1990 and January 2021. PubMed, Scopus, Web of Science, and Embase databases were searched systematically. The Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines were used to select the studies. Data Extraction: To evaluate the quality of studies included in this systematic review, Drummond’s ten-item checklist was used. Results: Five economic evaluation studies were included in this review. The included studies reported different results regarding the effect of home care on improving different indicators and the cost-effectiveness ratio of home care to hospital care. Most previous studies reported that home care is a more cost-effective option for improving many indicators, such as physical function and quality-adjusted life years (QALY), and for reducing mortality and institutionalization, compared to hospital care. Conclusions: Home care is a more cost-effective option than hospital care for stroke patients with regard to some indicators, such as the Barthel index for Activities of Daily Living, Modified Rankin Scale (mRS), quality of life, mortality, and institutionalization. However, there are some exemptions to this conclusion. Due to limitations, such as heterogeneity of interventions in the existing studies, different levels of patients’ disabilities, different perspectives toward economic evaluation, and differences in the healthcare systems of countries, further research is needed according to the context of each country based on clinical trials.


2018 ◽  
Vol 44 (5) ◽  
pp. E2 ◽  
Author(s):  
Won Hyung A. Ryu ◽  
Michael M. H. Yang ◽  
Sandeep Muram ◽  
W. Bradley Jacobs ◽  
Steven Casha ◽  
...  

OBJECTIVEAs the cost of health care continues to increase, there is a growing emphasis on evaluating the relative economic value of treatment options to guide resource allocation. The objective of this systematic review was to evaluate the current evidence regarding the cost-effectiveness of cranial neurosurgery procedures.METHODSThe authors performed a systematic review of the literature using PubMed, EMBASE, and the Cochrane Library, focusing on themes of economic evaluation and cranial neurosurgery following the Preferred Reporting Items for Systematic Reviews and Meta-Analysis (PRISMA) guidelines. Included studies were publications of cost-effectiveness analysis or cost-utility analysis between 1995 and 2017 in which health utility outcomes in life years (LYs), quality-adjusted life years (QALYs), or disability-adjusted life years (DALYs) were used. Three independent reviewers conducted the study appraisal, data abstraction, and quality assessment, with differences resolved by consensus discussion.RESULTSIn total, 3485 citations were reviewed, with 53 studies meeting the inclusion criteria. Of those, 34 studies were published in the last 5 years. The most common subspecialty focus was cerebrovascular (32%), followed by neurooncology (26%) and functional neurosurgery (24%). Twenty-eight (53%) studies, using a willingness to pay threshold of US$50,000 per QALY or LY, found a specific surgical treatment to be cost-effective. In addition, there were 11 (21%) studies that found a specific surgical option to be economically dominant (both cost saving and having superior outcome), including endovascular thrombectomy for acute ischemic stroke, epilepsy surgery for drug-refractory epilepsy, and endoscopic pituitary tumor resection.CONCLUSIONSThere is an increasing number of cost-effectiveness studies in cranial neurosurgery, especially within the last 5 years. Although there are numerous procedures, such as endovascular thrombectomy for acute ischemic stroke, that have been conclusively proven to be cost-effective, there remain promising interventions in current practice that have yet to meet cost-effectiveness thresholds.


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