scholarly journals The Methodological Quality and Challenges in Conducting Economic Evaluations of Newborn Screening: A Scoping Review

2020 ◽  
Vol 6 (4) ◽  
pp. 94
Author(s):  
Pasquale Cacciatore ◽  
Laurenske A. Visser ◽  
Nasuh Buyukkaramikli ◽  
Catharina P. B. van der Ploeg ◽  
M. Elske van den Akker-van Marle

Cost-effectiveness (CEA) and cost–utility analyses (CUA) have become popular types of economic evaluations (EE) used for evidence-based decision-making in healthcare resource allocation. Newborn screening programs (NBS) can have significant clinical benefits for society, and cost-effectiveness analysis may help to select the optimal strategy among different screening programs, including the no-screening option, on different conditions. These economic analyses of NBS, however, are hindered by several methodological challenges. This study explored the methodological quality in recent NBS economic evaluations and analyzed the main challenges and strategies adopted by researchers to deal with them. A scoping review was conducted according to PRISMA methodology to identify CEAs and CUAs of NBS. The methodological quality of the retrieved studies was assessed quantitatively using a specific guideline for the quality assessment of NBS economic evaluations, by calculating a general score for each EE. Challenges in the studies were then explored using thematic analysis as a qualitative synthesis approach. Thirty-five studies met the inclusion criteria. The quantitative analysis showed that the methodological quality of NBS economic evaluations was heterogeneous. Lack of clear description of items related to results, discussion, and discounting were the most frequent flaws. Methodological challenges in performing EEs of neonatal screenings include the adoption of a long time horizon, the use of quality-adjusted life years as health outcome measure, and the assessment of costs beyond the screening interventions. The results of this review can support future economic evaluation research, aiding researchers to develop a methodological guidance to perform EEs aimed at producing solid results to inform decisions for resource allocation in neonatal screening.

Author(s):  
Anna Parackal ◽  
Karishini Ramamoorthi ◽  
Jean-Eric Tarride

Background: End-of-life care is a driver of increasing healthcare costs; however, palliative care interventions may significantly reduce these costs. Economic evaluations that measure the incremental cost per quality adjusted life years (QALY) are warranted to inform cost-effectiveness of the intervention relative to a comparator and permit evaluation of investment against other therapeutic interventions. Evidence from the literature up to 2011 indicates a scarcity of cost-utility studies in palliative care research. Aim: This literature review evaluates economic studies published between 2011 and 2019 to determine whether the methods of economic evaluations have evolved since 2011. Design and Data Sources: A literature search was completed using CENTRAL, OVID MEDLINE, EMBASE and other sources for publications between 2011 and 2019. Study characteristics, methodology and key findings of publications that met the inclusion criteria were reviewed. Quality of studies were assessed using indicators developed by authors of the previous literature review. Results: 46 papers were included for qualitative synthesis. Among them only 6 studies conducted formal cost-effectiveness evaluations-of these 5 measured QALYs and 1 employed probabilistic analyses. In addition, with the exception of 1 costing analysis, all other economic evaluations undertook a healthcare payer perspective. Quality of evidence were comparable to the previous literature review published in 2011. Conclusion: Despite the small increase in the number of cost-utility studies, the methods of palliative care economic evaluations have not evolved significantly since 2011. More probabilistic cost-utility analyses of palliative care interventions from a societal perspective are necessary to truly evaluate the value for money.


2009 ◽  
Vol 2 (1) ◽  
pp. 1-9 ◽  
Author(s):  
Jenna L. Yoder ◽  
Khalid M. Kamal

Objectives: To explore the use of pharmacoeconomic principles through examination of economic evaluations pertaining to the combination of the monoclonal antibody rituximab with conventional CHOP (cyclophosphamide/doxorubicin/ vincristine/ prednisone) or CHOP-like chemotherapy regimens in patients with non-Hodgkin lymphoma. Methods: A literature search was conducted using Evidence-Based Medical Reviews (EBMR), International Pharmaceutical Abstracts (IPA), and Medline databases to identify all economic studies relating to rituximab in combination with CHOP or CHOP-like regimens. The systematic evaluation also utilized the Quality of Health Economic Studies instrument to assess the quality of each study that was included in the final review. Results: Initially, eight studies were retrieved which included the use of rituximab in non-Hodgkin lymphoma treatment. Of these, four studies were excluded as rituximab was used as a stand-alone treatment option. The remaining four studies involved conventional CHOP therapy versus the combination with rituximab (R-CHOP) in patients with non-Hodgkin lymphoma. One study employed a cost-effectiveness analysis while the remaining three studies used a cost-utility analysis and reported the outcomes in terms of quality-adjusted life years (QALYs). Conclusions: The cost-effectiveness evaluation illustrated the dominance of R-CHOP over CHOP-alone in terms of both lower costs and increased life years gained. The cost-utility of R-CHOP in terms of costs/QALYs were below the accepted threshold of 50,000 in international monetary units. Through examination of evaluation principles employed, it is found that valid results are highly dependent on the input data, assumptions, and sensitivity analyses. Clinical decisionmakers must take into account specific inclusions of costs relevant to their own practice setting.


2020 ◽  
Vol 162 (6) ◽  
pp. 826-838
Author(s):  
Michael Yong ◽  
Jiahe Liang ◽  
Jeromie Ballreich ◽  
Jane Lea ◽  
Brian D. Westerberg ◽  
...  

Objective School hearing screening is a public health intervention that can improve care for children who experience hearing loss that is not detected on or develops after newborn screening. However, implementation of school hearing screening is sporadic and supported by mixed evidence to its economic benefit. This scoping review provides a summary of all published cost-effectiveness studies regarding school hearing screening programs globally. At the time of this review, there were no previously published reviews of a similar nature. Data Sources A structured search was applied to 4 databases: PubMed (Medline), Embase, CINAHL, and Cochrane Library. Review Methods The database search was carried out by 2 independent researchers, and results were reported in accordance with the PRISMA-ScR checklist and the JBI methodology for scoping reviews. Studies that included a cost analysis of screening programs for school-aged children in the school environment were eligible for inclusion. Studies that involved evaluations of only neonatal or preschool programs were excluded. Results Four of the 5 studies that conducted a cost-effectiveness analysis reported that school hearing screening was cost-effective through the calculation of incremental cost-effectiveness ratios (ICERs) via either quality- or disability-adjusted life years. One study reported that a new school hearing screening program dominated the existing program; 2 studies reported ICERs ranging from 1079 to 4304 international dollars; and 1 study reported an ICER of £2445. One study reported that school-entry hearing screening was not cost-effective versus no screening. Conclusion The majority of studies concluded that school hearing screening was cost-effective. However, significant differences in methodology and region-specific estimates of model inputs limit the generalizability of these findings.


Author(s):  
Christopher J Cadham ◽  
Pianpian Cao ◽  
Jinani Jayasekera ◽  
Kathryn L Taylor ◽  
David T Levy ◽  
...  

Abstract Background Guidelines recommend offering cessation interventions to smokers eligible for lung cancer screening, but there is little data comparing specific cessation approaches in this setting. We compared the benefits and costs of different smoking cessation interventions to help screening programs select specific cessation approaches. Methods We conducted a societal-perspective cost-effectiveness analysis using a Cancer Intervention and Surveillance Modeling Network model simulating individuals born in 1960 over their lifetimes. Model inputs were derived from Medicare, national cancer registries, published studies, and micro-costing of cessation interventions. We modeled annual lung cancer screening following 2014 US Preventive Services Task Force guidelines plus cessation interventions offered to current smokers at first screen, including pharmacotherapy only or pharmacotherapy with electronic and/or web-based, telephone, individual, or group counseling. Outcomes included lung cancer cases and deaths, life-years saved, quality-adjusted life-years (QALYs) saved, costs, and incremental cost-effectiveness ratios. Results Compared with screening alone, all cessation interventions decreased cases of and deaths from lung cancer. Compared incrementally, efficient cessation strategies included pharmacotherapy with either web-based cessation ($555 per QALY), telephone counseling ($7562 per QALY), or individual counseling ($35 531 per QALY). Cessation interventions continued to have costs per QALY well below accepted willingness to pay thresholds even with the lowest intervention effects and was more cost-effective in cohorts with higher smoking prevalence. Conclusion All smoking cessation interventions delivered with lung cancer screening are likely to provide benefits at reasonable costs. Because the differences between approaches were small, the choice of intervention should be guided by practical concerns such as staff training and availability.


2020 ◽  
Vol 14 (8) ◽  
Author(s):  
Anna Parackal ◽  
Jean-Eric Tarride ◽  
Feng Xie ◽  
Gord Blackhouse ◽  
Jennifer Hoogenes ◽  
...  

Introduction: Recent health technology assessments (HTAs) of robot-assisted radical prostatectomy (RARP) in Ontario and Alberta, Canada, resulted in opposite recommendations, calling into question whether benefits of RARP offset the upfront investment. Therefore, the study objectives were to conduct a cost-utility analysis from a Canadian public payer perspective to determine the cost-effectiveness of RARP. Methods: Using a 10-year time horizon, a five-state Markov model was developed to compare RARP to open radical prostatectomy (ORP). Clinical parameters were derived from Canadian observational studies and a recently published systematic review. Costs, resource utilization, and utility values from recent Canadian sources were used to populate the model. Results were presented in terms of increment costs per quality-adjusted life years (QALYs) gained. A probabilistic analysis was conducted, and uncertainty was represented using cost-effectiveness acceptability curves (CEACs). One-way sensitivity analyses were also conducted. Future costs and QALYs were discounted at 1.5%. Results: Total cost of RARP and ORP were $47 033 and $45 332, respectively. Total estimated QALYs were 7.2047 and 7.1385 for RARP and ORP, respectively. The estimated incremental cost-utility ratio (ICUR) was $25 704 in the base-case analysis. At a willingness-to-pay threshold of $50 000 and $100 000 per QALY gained, the probability of RARP being cost-effective was 0.65 and 0.85, respectively. The model was most sensitive to the time horizon. Conclusions: The results of this analysis suggest that RARP is likely to be cost-effective in this Canadian patient population. The results are consistent with Alberta’s HTA recommendation and other economic evaluations, but challenges Ontario’s reimbursement decision.


2012 ◽  
Vol 30 (4) ◽  
pp. 273-285 ◽  
Author(s):  
Song-Yi Kim ◽  
Hyangsook Lee ◽  
Younbyoung Chae ◽  
Hi-Joon Park ◽  
Hyejung Lee

Objective To summarise the evidence on the cost-effectiveness of acupuncture. Methods We identified full economic evaluations such as cost-effectiveness analysis (CEA), cost-utility analysis (CUA) and cost-benefit analysis (CBA) alongside randomised controlled trials (RCTs) that assessed the consequences and costs of acupuncture for any medical condition. Eleven electronic databases were searched up to March 2011 without language restrictions. Eligible RCTs were assessed using the Cochrane criteria for risk of bias and a modified version of the checklist for economic evaluation. The general characteristics and the results of each economic analysis such as incremental cost-effectiveness ratios (ICERs) were extracted. Results Of 17 included studies, nine were CUAs that measured quality-adjusted life years (QALYs) and eight were CEAs that assessed effectiveness of acupuncture based on improvements in clinical symptoms. All CUAs showed that acupuncture with or without usual care was cost-effective compared with waiting list control or usual care alone, with ICERs ranging from ¢3011/QALY (dysmenorrhoea) to ¢22 298/QALY (allergic rhinitis) in German studies, and from £3855/QALY (osteoarthritis) to £9951/QALY (headache) in UK studies. In the CEAs, acupuncture was beneficial at a relatively low cost in six European and Asian studies. All CUAs were well-designed with a low risk of bias, but this was not the case for CEAs. Conclusions Overall, this review demonstrates the cost-effectiveness of acupuncture. Despite such promising results, any generalisation of these results needs to be made with caution given the diversity of diseases and the different status of acupuncture in the various countries.


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.


PLoS ONE ◽  
2021 ◽  
Vol 16 (2) ◽  
pp. e0245963
Author(s):  
Inge J. M. H. Caelers ◽  
Suzanne L. de Kunder ◽  
Kim Rijkers ◽  
Wouter L. W. van Hemert ◽  
Rob A. de Bie ◽  
...  

Introduction The demand for spinal fusion surgery has increased over the last decades. Health care providers should take costs and cost-effectiveness of these surgeries into account. Open transforaminal lumbar interbody fusion (TLIF) and posterior lumbar interbody fusion (PLIF) are two widely used techniques for spinal fusion. Earlier research revealed that TLIF is associated with less blood loss, shorter surgical time and sometimes shorter length of hospital stay, while effectiveness of both techniques on back and/or leg pain are equal. Therefore, TLIF could result in lower costs and be more cost-effective than PLIF. This is the first systematic review comparing direct and indirect (partial) economic evaluations of TLIF with PLIF in adults with lumbar spondylolisthesis. Furthermore, methodological quality of included studies was assessed. Methods Searches were conducted in eight databases for reporting on eligibility criteria; TLIF or PLIF, lumbar spondylolisthesis or lumbar instability, and cost. Costs were converted to United States Dollars with reference year 2020. Study quality was assessed using the bias assessment tool of the Cochrane Handbook for Systematic Reviews of Interventions, the Level of Evidence guidelines of the Oxford Centre for Evidence-based Medicine and the Consensus Health Economic Criteria (CHEC) list. Results Of a total of 693 studies, 16 studies were included. Comparison of TLIF and PLIF could only be made indirectly, since no study compared TLIF and PLIF directly. There was a large heterogeneity in health care and societal perspective costs due to different in-, and exclusion criteria, baseline characteristics and the use of costs or charges in calculations. Health care perspective costs, calculated with hospital costs, ranged from $15,867-$43,217 in TLIF-studies and $32,662 in one PLIF-study. Calculated with hospital charges, it ranged from $8,964-$51,469 in TLIF-studies and $21,838-$93,609 in two PLIF-studies. Societal perspective costs and cost-effectiveness, only mentioned in TLIF-studies, ranged from $5,702/QALY-$48,538/QALY and $50,092/QALY-$90,977/QALY, respectively. Overall quality of studies was low. Conclusions This systematic review shows that TLIF and PLIF are expensive techniques. Moreover, firm conclusions about the preferable technique, based on (partial) economic evaluations, cannot be drawn due to limited studies and heterogeneity. Randomized prospective trials and full economical evaluations with direct TLIF and PLIF comparison are needed to obtain high levels of evidence. Furthermore, development of guidelines to perform adequate economic evaluations, specified for the field of interest, will be useful to minimize heterogeneity and maximize transferability of results. Trial registration Prospero-database registration number: CRD42020196869.


2021 ◽  
pp. jnnp-2021-326344
Author(s):  
Sophy TF Shih ◽  
Michelle Anne Farrar ◽  
Veronica Wiley ◽  
Georgina Chambers

ObjectiveTo assess cost-effectiveness of newborn screening (NBS) for spinal muscular atrophy (SMA) and early treatment with nusinersen or onasemnogene abeparvovec (gene therapy), compared with nusinersen without SMA screening.MethodsInformed by an Australian state-wide SMA NBS programme, a decision analytical model nested with Markov models was constructed to evaluate costs and quality-adjusted life-years (QALYs) from a societal perspective with sensitivity analyses.ResultsBy treating one presymptomatic SMA infant with nusinersen or gene therapy, an additional 9.93 QALYs were gained over 60 years compared with late treatment in clinically diagnosed SMA. The societal cost was $9.8 million for early nusinersen treatment, $4.4 million for early gene therapy and $4.8 million for late nusinersen treatment. Compared with late nusinersen treatment, early gene therapy would be dominant, gaining 9.93 QALYs while saving $360 000; whereas early nusinersen treatment would result in a discounted incremental cost-effectiveness ratio (ICER) of $507 000/QALY.At a population level, compared with no screening and late treatment with nusinersen, NBS and early gene therapy resulted in 0.00085 QALY gained over 60 years and saving $24 per infant screened (85 QALYs gained and $2.4 million saving per 100 000 infants screened). More than three quarters of simulated ICERs by probability sensitivity analyses showed NBS and gene therapy would be dominant or less than $50 000/QALY, compared with no screening and late nusinersen treatment.ConclusionNBS coupled with gene therapy improves the quality and length of life for infants with SMA and would be considered value-for-money from an Australian clinical and policy context.


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