scholarly journals ECONOMIC EVALUATION OF CHELATION REGIMENS FOR β--THALASSEMIA MAJOR: A SYSTEMATIC REVIEW

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

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.


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.


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.


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.


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 35 (S1) ◽  
pp. 38-39
Author(s):  
Thomas Plunkett ◽  
Paul Carty ◽  
Michelle O'Neill ◽  
Patricia Harrington ◽  
Susan M Smith ◽  
...  

IntroductionTo inform the development of a national clinical guideline for Chronic Obstructive Pulmonary Disease (COPD), prioritized by the National Clinical Effectiveness Committee in Ireland, a systematic review was conducted to examine the cost-effectiveness of pulmonary rehabilitation programs (PRPs), outreach programs (OPs), and long-term oxygen therapy (LTOT), compared with usual care.MethodsMedline, Embase, the Cochrane Library and grey literature sources were searched up to 19 June 2018. Studies evaluating cost-effectiveness published post-2008 in English were included. Screening, data extraction, and quality assessment using the Consensus Health Economic Criteria and International Society for Pharmacoeconomics questionnaires were conducted independently by two reviewers. Costs were converted to 2017 Irish Euro using consumer price indices for health and purchasing power parity.ResultsFrom 8,661 articles identified, seven studies (one comparing both PRPs and LTOT) were included (PRPs: five; OPs: one; LTOT: two). PRP cost-utility analyses (n = 4) reported conflicting results due to considerable heterogeneity in program and study design, with incremental cost-effectiveness ratios (ICERs) ranging between EUR 12,391 and EUR 509,122 per quality adjusted life-year (QALY) gained. The remaining study investigated hospitalizations avoided and found outpatient and community-based PRPs to be dominant, while home-based PRP produced an ICER of EUR 1,913. OPs were found to be less costly, but also less effective. However, the results of the underpinning trial were neither statistically nor clinically significant. LTOT was found to be cost-effective, with ICERs of EUR 17,603 and EUR 26,936 per QALY gained.ConclusionsApplying a willingness-to-pay threshold of EUR 45,000 per QALY gained, this systematic review found that, compared with usual care, there is inconsistent but generally favorable evidence for PRPs, no clear evidence for the cost-effectiveness of OPs, and that LTOT is likely to be cost-effective. However, there was a lack of methodologically robust studies included in the review and most were not directly transferable to the Irish context.


2019 ◽  
Vol 35 (S1) ◽  
pp. 80-80
Author(s):  
Thomas Plunkett ◽  
Paul Carty ◽  
Michelle O'Neill ◽  
Patricia Harrington ◽  
Susan M Smith ◽  
...  

IntroductionTo inform the development of a national clinical guideline for Chronic Obstructive Pulmonary Disease (COPD), prioritised by the National Clinical Effectiveness Committee (NCEC) in Ireland, a systematic review was conducted to examine the cost-effectiveness of long-acting beta2-agonists (LABAs) in combination with long-acting muscarinic antagonists (LAMAs) compared with LAMA or LABA monotherapy.MethodsMedline, Embase, the Cochrane Library and grey literature sources were searched up to 19 June 2018. Studies evaluating cost-effectiveness published post-2008 in English were included. Screening, data extraction, and quality assessment using the Consensus Health Economic Criteria (CHEC-list) and International Society for Pharmacoeconomics (ISPOR) questionnaires were conducted independently by two reviewers. Costs were adjusted to 2017 Irish Euro using consumer price indices and purchasing power parity as per national guidelines.ResultsFrom a total of 8,661 articles identified, nine studies (all cost-utility analyses) were included in the review. Studies ranged from low to high quality and compared LAMA/LABA combination therapy with LAMA monotherapy. The results reported were mixed, ranging from combination therapy being dominated by (that is, more costly and less effective than) LAMA monotherapy to being dominant (that is, less costly and more effective). However, when excluding low quality, less applicable studies, the remaining six studies reported incremental cost-effectiveness ratios (ICERs) of between EUR 2,770 and EUR 26,462 per quality-adjusted life year (QALY) gained. Only one study additionally compared LABA monotherapy as a comparator, reporting combination therapy to be even more cost-effective than in the LAMA monotherapy comparison.ConclusionsApplying a cost-effectiveness willingness-to-pay threshold of EUR 45,000 per QALY gained, this systematic review found that LAMA/LABA combination therapy is cost-effective compared with LAMA or LABA monotherapy in COPD patients.


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.


2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Sohail Akhtar ◽  
Jamal Abdul Nasir ◽  
Amara Javed ◽  
Mariyam Saleem ◽  
Sundas Sajjad ◽  
...  

Abstract Background The aim of this paper is to investigate the prevalence of diabetes and its associated risk factors in Afghanistan through a systematic review and meta–analysis. Methods A comprehensive literature search was conducted using EMBASE, PubMed, Web of Sciences, Google Scholar and the Cochrane library, carried out from inception to April 312,020, without language restriction. Meta–analysis was performed using DerSimonian and Laird random-effects models with inverse variance weighting. The existence of publication bias was initially assessed by visual inspection of a funnel plot and then tested by the Egger regression test. Subgroup analyses and meta-regression were used to explore potential sources of heterogeneity. This systematic review was reported by following the PRISMA guidelines and the methodological quality of each included study was evaluated using the STROBE guidelines. Results Out of 64 potentially relevant studies, only 06 studies fulfilled the inclusion criteria and were considered for meta-analysis. The pooled prevalence of diabetes in the general population based on population-based studies were 12.13% (95% CI: 8.86–16.24%), based on a pooled sample of 7071 individuals. Results of univariate meta-regression analysis revealed that the prevalence of diabetes increased with mean age, hypertension and obesity. There was no significant association between sex (male vs female), smoking, the methodological quality of included articles or education (illiterate vs literate) and the prevalence of diabetes. Conclusions This meta-analysis reports the 12.13% prevalence of diabetes in Afghanistan,with the highest prevalence in Kandahar and the lowest in Balkh province. The main risk factors include increasing age, obesity and hypertension. Community-based care and preventive training programmes are recommended. Trial registration This review was registered on PROSPERO (registration number CRD42020172624).


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