scholarly journals Clinical effectiveness and cost-effectiveness of interventions for the treatment of anogenital warts: systematic review and economic evaluation

2016 ◽  
Vol 20 (24) ◽  
pp. 1-486 ◽  
Author(s):  
Elizabeth Thurgar ◽  
Samantha Barton ◽  
Charlotta Karner ◽  
Steven J Edwards
Keyword(s):  
Systematic Review ◽  
Cost Effectiveness ◽  
Economic Model ◽  
Treatment Options ◽  
Clinical Effectiveness ◽  
Cost Effective ◽  
Evidence Base ◽  
Anogenital Warts ◽  
Ablative Techniques ◽  
The Cost

BackgroundTypically occurring on the external genitalia, anogenital warts (AGWs) are benign epithelial skin lesions caused by human papillomavirus infection. AGWs are usually painless but can be unsightly and physically uncomfortable, and affected people might experience psychological distress. The evidence base on the clinical effectiveness and cost-effectiveness of treatments for AGWs is limited.ObjectivesTo systematically review the evidence on the clinical effectiveness of medical and surgical treatments for AGWs and to develop an economic model to estimate the cost-effectiveness of the treatments.Data sourcesElectronic databases (MEDLINE, MEDLINE In-Process & Other Non-Indexed Citations, EMBASE, The Cochrane Library databases and Web of Science) were searched from inception (or January 2000 for Web of Science) to September 2014. Bibliographies of relevant systematic reviews were hand-searched to identify potentially relevant studies. The World Health Organization International Clinical Trials Registry Platform and ClinicalTrials.gov were searched for ongoing and planned studies.Review methodsA systematic review of the clinical effectiveness literature was carried out according to standard methods and a mixed-treatment comparison (MTC) undertaken. The model implemented for each outcome was that with the lowest deviance information criterion. A de novo economic model was developed to assess cost-effectiveness from the perspective of the UK NHS. The model structure was informed through a systematic review of the economic literature and in consultation with clinical experts. Effectiveness data were obtained from the MTC. Costs were obtained from the literature and standard UK sources.ResultsOf 4232 titles and abstracts screened for inclusion in the review of clinical effectiveness, 60 randomised controlled trials (RCTs) evaluating 19 interventions were included. Analysis by MTC indicated that ablative techniques were typically more effective than topical interventions at completely clearing AGWs at the end of treatment. Podophyllotoxin 0.5% solution (Condyline®, Takeda Pharmaceutical Company Ltd; Warticon®solution, Stiefel Laboratories Ltd) was found to be the most effective topical treatment evaluated. Networks for other outcomes included fewer treatments, which restrict conclusions on the comparative effectiveness of interventions. In total, 84 treatment strategies were assessed using the economic model. Podophyllotoxin 0.5% solution first line followed by carbon dioxide (CO2) laser therapy second line if AGWs did not clear was most likely to be considered a cost-effective use of resources at a willingness to pay of £20,000–30,000 per additional quality-adjusted life-year gained. The result was robust to most sensitivity analyses conducted.LimitationsLimited reporting in identified studies of baseline characteristics for the enrolled population generates uncertainty around the comparability of the study populations and therefore the generalisability of the results to clinical practice. Subgroup analyses were planned based on type, number and size of AGWs, all of which are factors thought to influence treatment effect. Lack of data on clinical effectiveness based on these characteristics precluded analysis of the differential effects of treatments in the subgroups of interest. Despite identification of 60 studies, most comparisons in the MTC are informed by only one RCT. Additionally, lack of head-to-head RCTs comparing key treatments, together with minimal reporting of results in some studies, precluded comprehensive analysis of all treatments for AGWs.ConclusionsThe results generated by the MTC are in agreement with consensus opinion that ablative techniques are clinically more effective at completely clearing AGWs after treatment. However, the evidence base informing the MTC is limited. A head-to-head RCT that evaluates the comparative effectiveness of interventions used in clinical practice would help to discern the potential advantages and disadvantages of the individual treatments. The results of the economic analysis suggest that podophyllotoxin 0.5% solution is likely to represent a cost-effective first-line treatment option. More expensive effective treatments, such as CO2laser therapy or surgery, may represent cost-effective second-line treatment options. No treatment and podophyllin are unlikely to be considered cost-effective treatment options. There is uncertainty around the cost-effectiveness of treatment with imiquimod, trichloroacetic acid and cryotherapy.Study registrationThis study is registered as PROSPERO CRD42013005457.FundingThe National Institute for Health Research Health Technology Assessment programme.

scholarly journals The clinical effectiveness and cost-effectiveness of the PROGENSA® prostate cancer antigen 3 assay and the Prostate Health Index in the diagnosis of prostate cancer: a systematic review and economic evaluation

2015 ◽  
Vol 19 (87) ◽  
pp. 1-192 ◽  
Author(s):  
Amanda Nicholson ◽  
James Mahon ◽  
Angela Boland ◽  
Sophie Beale ◽  
Kerry Dwan ◽  
...  
Keyword(s):  
Prostate Cancer ◽  
Systematic Review ◽  
Cost Effectiveness ◽  
Clinical Assessment ◽  
Clinical Effectiveness ◽  
Cost Effective ◽  
Clinical Validity ◽  
Prostate Health Index ◽  
The Cost ◽  
Prostate Health

BackgroundThere is no single definitive test to identify prostate cancer in men. Biopsies are commonly used to obtain samples of prostate tissue for histopathological examination. However, this approach frequently misses cases of cancer, meaning that repeat biopsies may be necessary to obtain a diagnosis. The PROGENSA®prostate cancer antigen 3 (PCA3) assay (Hologic Gen-Probe, Marlborough, MA, USA) and the Prostate Health Index (phi; Beckman Coulter Inc., Brea, CA, USA) are two new tests (a urine test and a blood test, respectively) that are designed to be used to help clinicians decide whether or not to recommend a repeat biopsy.ObjectiveTo evaluate the clinical effectiveness and cost-effectiveness of the PCA3 assay and the phi in the diagnosis of prostate cancer.Data sourcesMultiple publication databases and trial registers were searched in May 2014 (from 2000 to May 2014), including MEDLINE, EMBASE, The Cochrane Library, ISI Web of Science, Medion, Aggressive Research Intelligence Facility database, ClinicalTrials.gov, International Standard Randomised Controlled Trial Number Register and World Health Organization International Clinical Trials Registry Platform.Review methodsThe assessment of clinical effectiveness involved three separate systematic reviews, namely reviews of the analytical validity, the clinical validity of these tests and the clinical utility of these tests. The assessment of cost-effectiveness comprised a systematic review of full economic evaluations and the development of a de novo economic model.SettingThe perspective of the evaluation was the NHS in England and Wales.ParticipantsMen suspected of having prostate cancer for whom the results of an initial prostate biopsy were negative or equivocal.InterventionsThe use of the PCA3 score or phi in combination with existing tests (including histopathology results, prostate-specific antigen level and digital rectal examination), multiparametric magnetic resonance imaging and clinical judgement.ResultsIn addition to documents published by the manufacturers, six studies were identified for inclusion in the analytical validity review. The review identified issues concerning the precision of the PCA3 assay measurements. It also highlighted issues relating to the storage requirements and stability of samples intended for analysis using the phi assay. Fifteen studies met the inclusion criteria for the clinical validity review. These studies reported results for 10 different clinical comparisons. There was insufficient evidence to enable the identification of appropriate test threshold values for use in a clinical setting. In addition, the implications of adding either the PCA3 assay or the phi to clinical assessment were not clear. Furthermore, the addition of the PCA3 assay or the phi to clinical assessment plus magnetic resonance imaging was not found to improve discrimination. No published papers met the inclusion criteria for either the clinical utility review or the cost-effectiveness review. The results from the cost-effectiveness analyses indicated that using either the PCA3 assay or the phi in the NHS was not cost-effective.LimitationsThe main limitations of the systematic review of clinical validity are that the review conclusions are over-reliant on findings from one study, the descriptions of clinical assessment vary widely within reviewed studies and many of the reported results for the clinical validity outcomes do not include either standard errors or confidence intervals.ConclusionsThe clinical benefit of using the PCA3 assay or the phi in combination with existing tests, scans and clinical judgement has not yet been confirmed. The results from the cost-effectiveness analyses indicate that the use of these tests in the NHS would not be cost-effective.Study registrationThis study is registered as PROSPERO CRD42014009595.FundingThe National Institute for Health Research Health Technology Assessment programme.

scholarly journals Systematic review of health economic studies in cranial neurosurgery

2018 ◽  
Vol 44 (5) ◽  
pp. E2 ◽  
Author(s):  
Won Hyung A. Ryu ◽  
Michael M. H. Yang ◽  
Sandeep Muram ◽  
W. Bradley Jacobs ◽  
Steven Casha ◽  
...  
Keyword(s):  
Systematic Review ◽  
Ischemic Stroke ◽  
Cost Effectiveness ◽  
Acute Ischemic Stroke ◽  
Cost Effective ◽  
Current Evidence ◽  
Cochrane Library ◽  
Endovascular Thrombectomy ◽  
Life Years ◽  
The Cost

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.

scholarly journals TECHNIQUES FOR DIAGNOSING OSTEOPOROSIS: A SYSTEMATIC REVIEW OF COST-EFFECTIVENESS STUDIES

2014 ◽  
Vol 30 (3) ◽  
pp. 273-281 ◽  
Author(s):  
Davide Minniti ◽  
Ottavio Davini ◽  
Maria Rosaria Gualano ◽  
Maria Michela Gianino
Keyword(s):  
Systematic Review ◽  
Cost Effectiveness ◽  
Postmenopausal Women ◽  
Cost Effective ◽  
Economic Evaluations ◽  
Clear Cut ◽  
Step Procedure ◽  
Database Research ◽  
The Cost ◽  
Effectiveness Studies

Objectives:The study question was whether dual-energy X-ray absorptiometry (DXA) alone is more cost-effective for identifying postmenopausal women with osteoporosis than a two-step procedure with quantitative ultrasound sonography (QUS) plus DXA. To answer this question, a systematic review was performed.Methods:Electronic databases (PubMed, INAHTA, Health Evidence Network, NIHR, the Health Technology Assessment program, the NHS Economic Evaluation Database, Research Papers in Economics, Web of Science, Scopus, and EconLit) were searched for cost-effectiveness publications. Two independent reviewers selected eligible publications based on the inclusion/exclusion criteria. Quality assessment of economic evaluations was undertaken using the Drummond checklist.Results:Seven journal articles and four reports were reviewed. The cost per true positive case diagnosed by DXA was found to be higher than that for diagnosis by QUS+DXA in two articles. In one article it was found to be lower. In three studies, the results were not conclusive. These articles were characterized by the differences in the types of devices, parameters and thresholds on the QUS and DXA tests and the unit costs of the DXA and QUS tests as well as by variability in the sensitivity and specificity of the techniques and the prevalence of osteoporosis.Conclusions:The publications reviewed did not provide clear-cut evidence for drawing conclusions about which screening test may be more cost-effective for identifying postmenopausal women with osteoporosis.

COST-EFFECTIVENESS OF ADHERENCE INTERVENTIONS FOR HIGHLY ACTIVE ANTIRETROVIRAL THERAPY: A SYSTEMATIC REVIEW

2013 ◽  
Vol 29 (3) ◽  
pp. 227-233 ◽  
Author(s):  
Tim Mathes ◽  
Dawid Pieper ◽  
Sunya-Lee Antoine ◽  
Michaela Eikermann
Keyword(s):  
Systematic Review ◽  
Cost Effectiveness ◽  
Antiretroviral Therapy ◽  
Highly Active Antiretroviral Therapy ◽  
Cost Effective ◽  
Sensitivity Analyses ◽  
Baseline Scenario ◽  
Highly Active ◽  
Adherence Interventions ◽  
The Cost

Objectives: The objective of this systematic review was to evaluate the cost-effectiveness of interventions aiming to increase the adherence to highly active antiretroviral therapy (HAART) in HIV-infected patients in developed countries (WHO stratum A).Methods: A systematic search for comparative health economic studies was conducted in the following databases: EMBASE, MEDLINE, NHS Economic Evaluation Database, CINAHL, HEED, and EconLit. The identified publications were selected by two reviewers independently according to predefined inclusion and exclusion criteria. Furthermore, these were evaluated according to a standardized checklist and finally extracted, analyzed, and summarized.Results: After reviewing the abstracts and full texts four relevant studies were identified. Different educational programs were compared as well as the Directly Observed Therapy (DOT). A critical aspect to be considered in particular was the poor transparency of the cost data. In three cost-utility analyses the costs per quality-adjusted life-year (QALY) in the baseline scenario were each under USD 15,000. The sensitivity analyses with a presumed maximum threshold of USD 50,000/QALY showed a predominantly cost-effective result. In one study that examined DOT the costs add up to over USD 150,000/QALY.Conclusions: It seems that adherence interventions for HAART in HIV-infected patients can be cost-effective. Nevertheless, the quality of the included studies is deficient and only a few of the possible adherence interventions are taken into consideration. A final assessment of the cost-effectiveness of adherence interventions in general is, therefore, not possible.

scholarly journals ANALISIS EFEKTIVITAS BIAYA PASIEN PNEUMONIA BALITA RAWAT INAP DI RUMAH SAKIT BHAYANGKARA MANADO

PHARMACON ◽  
2019 ◽  
Vol 8 (4) ◽  
pp. 968
Author(s):  
Monica D. Lestari ◽  
Gayatri Citraningtyas ◽  
Hosea Jaya Edi
Keyword(s):  
Cost Effectiveness ◽  
Effective Treatment ◽  
Treatment Options ◽  
Cost Effective ◽  
Cost Effectiveness Analysis ◽  
Effectiveness Analysis ◽  
Cost Effective Treatment ◽  
Retrospective Data Collection ◽  
Keywords Antibiotics ◽  
The Cost

ABSTRACTPneumonia is an infectious disease in the lower respiratory tract that affects the lung tissue. Ceftriaxone and Gentamicin antibiotics are the most numerous and good for use in the treatment of pneumonia, but of the two antibiotics is not yet known the options for more cost effective treatment, so it needs to be done the cost effectiveness analysis in order to facilitate the selection of more cost-effective treatment options especially in toddler. This study aims to determine which therapies are more cost-effective than the use of antibiotics Ceftriaxone and Gentamicin in pneumonia patients in the January-December 2018 period in the Bhayangkara Manado Hospital using descriptive research methods with retrospective data collection. The sample in this study were 22 patients, 12 patients using ceftriaxone antibiotics and 10 patients using gentamicin antibiotics. The results showed that pneumonia treatment in infants using Ceftriaxone antibiotics was more cost-effective with ACER ceftriaxone value of Rp. 503,872 / day and ICER value of Rp. 145,588 / day. Keywords : Antibiotics, CEA (Cost-Effectiveness Analysis), Pharmacoeconomy, Toddler Pneumonia. ABSTRAKPneumonia merupakan penyakit infeksi pada saluran pernapasan bagian bawah yang mengenai jaringan paru. Antibiotik Seftriakson dan Gentamisim yang paling banyak dan baik untuk digunakan dalam pengobatan pneumonia, namun dari kedua antibiotik tersebut belum diketahui pilihan terapi yang lebih cost-effective, sehingga perlu dilakukan analisis efektivitas biaya agar dapat mempermudah dalam pemilihan alternatif pengobatan yang lebih cost-effective khususnya pada balita. Penelitian ini bertujuan untuk menentukan terapi yang lebih cost-effective dari penggunaan antibiotik Seftriakson dan Gentamisin pada pasien pneumonia rawat inap periode Januari-Desember 2018 di Rumah Sakit Bhayangkara Manado dengan menggunakan metode penelitian deskriptif dengan pengambilan data secara retrospektif. Sampel pada penelitian ini sebanyak 22 pasien yaitu 12 pasien menggunakan antibiotik Seftriakson dan 10 pasien menggunakan antibiotik Gentamisin. Hasil penelitian menunjukkan pengobatan pneumonia pada balita menggunakan antibiotik Seftriakson lebih cost-effective dengan nilai ACER seftriakson sebesar Rp. 503,872/hari dan nilai ICER sebesar Rp. 145.588/hari. Kata Kunci : Pneumonia Balita, Antibiotik, CEA (Cost-Effectiveness Analysis), Farmakoekonomi

scholarly journals Can polygenic risk scores contribute to cost-effective cancer screening? A systematic review

2021 ◽  
Author(s):  
Padraig Dixon ◽  
Edna Keeney ◽  
Jenny C Taylor ◽  
Sarah Wordsworth ◽  
Richard Martin
Keyword(s):  
Systematic Review ◽  
Health Economic ◽  
Cost Effectiveness ◽  
Cancer Screening ◽  
Cost Effective ◽  
Risk Scores ◽  
Polygenic Risk ◽  
The Cost ◽  
Economic Studies

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.

scholarly journals Clinical effectiveness and cost-effectiveness of issuing longer versus shorter duration (3-month vs. 28-day) prescriptions in patients with chronic conditions: systematic review and economic modelling

2017 ◽  
Vol 21 (78) ◽  
pp. 1-128 ◽  
Author(s):  
Céline Miani ◽  
Adam Martin ◽  
Josephine Exley ◽  
Brett Doble ◽  
Ed Wilson ◽  
...  
Keyword(s):  
Systematic Review ◽  
Primary Care ◽  
Cost Effectiveness ◽  
Health Outcomes ◽  
Chronic Conditions ◽  
Clinical Effectiveness ◽  
Cost Study ◽  
Clinical Scenarios ◽  
Retrospective Cohort Studies ◽  
The Cost

BackgroundTo reduce expenditure on, and wastage of, drugs, some commissioners have encouraged general practitioners to issue shorter prescriptions, typically 28 days in length; however, the evidence base for this recommendation is uncertain.ObjectiveTo evaluate the evidence of the clinical effectiveness and cost-effectiveness of shorter versus longer prescriptions for people with stable chronic conditions treated in primary care.Design/data sourcesThe design of the study comprised three elements. First, a systematic review comparing 28-day prescriptions with longer prescriptions in patients with chronic conditions treated in primary care, evaluating any relevant clinical outcomes, adherence to treatment, costs and cost-effectiveness. Databases searched included MEDLINE (PubMed), EMBASE, Cumulative Index to Nursing and Allied Health Literature, Web of Science and Cochrane Central Register of Controlled Trials. Searches were from database inception to October 2015 (updated search to June 2016 in PubMed). Second, a cost analysis of medication wastage associated with < 60-day and ≥ 60-day prescriptions for five patient cohorts over an 11-year period from the Clinical Practice Research Datalink. Third, a decision model adapting three existing models to predict costs and effects of differing adherence levels associated with 28-day versus 3-month prescriptions in three clinical scenarios.Review methodsIn the systematic review, from 15,257 unique citations, 54 full-text papers were reviewed and 16 studies were included, five of which were abstracts and one of which was an extended conference abstract. None was a randomised controlled trial: 11 were retrospective cohort studies, three were cross-sectional surveys and two were cost studies. No information on health outcomes was available.ResultsAn exploratory meta-analysis based on six retrospective cohort studies suggested that lower adherence was associated with 28-day prescriptions (standardised mean difference –0.45, 95% confidence interval –0.65 to –0.26). The cost analysis showed that a statistically significant increase in medication waste was associated with longer prescription lengths. However, when accounting for dispensing fees and prescriber time, longer prescriptions were found to be cost saving compared with shorter prescriptions. Prescriber time was the largest component of the calculated cost savings to the NHS. The decision modelling suggested that, in all three clinical scenarios, longer prescription lengths were associated with lower costs and higher quality-adjusted life-years.LimitationsThe available evidence was found to be at a moderate to serious risk of bias. All of the studies were conducted in the USA, which was a cause for concern in terms of generalisability to the UK. No evidence of the direct impact of prescription length on health outcomes was found. The cost study could investigate prescriptions issued only; it could not assess patient adherence to those prescriptions. Additionally, the cost study was based on products issued only and did not account for underlying patient diagnoses. A lack of good-quality evidence affected our decision modelling strategy.ConclusionsAlthough the quality of the evidence was poor, this study found that longer prescriptions may be less costly overall, and may be associated with better adherence than 28-day prescriptions in patients with chronic conditions being treated in primary care.Future workThere is a need to more reliably evaluate the impact of differing prescription lengths on adherence, on patient health outcomes and on total costs to the NHS. The priority should be to identify patients with particular conditions or characteristics who should receive shorter or longer prescriptions. To determine the need for any further research, an expected value of perfect information analysis should be performed.Study registrationThis study is registered as PROSPERO CRD42015027042.FundingThe National Institute for Health Research Health Technology Assessment programme.

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
Keyword(s):  
Systematic Review ◽  
Cost Effectiveness ◽  
Factor Viia ◽  
Cost Effective ◽  
Primary Treatment ◽  
Cost Utility ◽  
Economic Consequences ◽  
Modelling Framework ◽  
The Cost

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.

Sign in / Sign up

Export Citation Format

Share Document