scholarly journals Cost-effectiveness of treatment optimisation with biomarkers for immunotherapy in solid tumours: a systematic review protocol

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.

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.


2021 ◽  
Vol 11 (1) ◽  
Author(s):  
Olanrewaju Medu ◽  
Adegboyega Lawal ◽  
Doug Coyle ◽  
Kevin Pottie

Abstract Introduction This study reviewed the economic evidence of rapid HIV testing versus conventional HIV testing in low-prevalence high-income countries; evaluated the methodological quality of existing economic evaluations of HIV testing studies; and made recommendations on future economic evaluation directions of HIV testing approaches. Methods A systematic search of selected databases for relevant English language studies published between Jan 1, 2001, and Jan 30, 2019, was conducted. The methodological design quality was assessed using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) and the Drummond tool. We reported the systematic review according to the PRISMA guidelines. Results Five economic evaluations met the eligibility criteria but varied in comparators, evaluation type, perspective, and design. The methodologic quality of the included studies ranged from medium to high. We found evidence to support the cost-effectiveness of rapid HIV testing approaches in low-prevalence high-income countries. Rapid HIV testing was associated with cost per adjusted life year (QALY), ranging from $42,768 to $90,498. Additionally, regardless of HIV prevalence, rapid HIV testing approaches were the most cost-effective option. Conclusions There is evidence for the cost-effectiveness of rapid HIV testing, including the use of saliva-based testing compared to usual care or hospital-based serum testing. Further studies are needed to draw evidence on the relative cost-effectiveness of the distinct options and contexts of rapid HIV testing.


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.


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.


BMJ Open ◽  
2019 ◽  
Vol 9 (4) ◽  
pp. e027490 ◽  
Author(s):  
Giulia Rinaldi ◽  
Alexa Hijazi ◽  
Hassan Haghparast-Bidgoli

IntroductionType 2 diabetes mellitus (T2DM) remains one of the most common chronic diseases of adulthood which creates high degrees of morbidity and mortality worldwide. The incidence of T2DM continues to rise and recently, mHealth interventions have been increasingly used in the prevention, monitoring and management of T2DM. The aim of this study is to systematically review the published evidence on cost and cost-effectiveness of mHealth interventions for T2DM, as well as assess the quality of reporting of the evidence.Methods and analysisA comprehensive review of PubMed, EMBASE, Science Direct and Web of Science of articles published until January 2019 will be conducted. Included studies will be partial or full economic evaluations which provide cost or cost-effectiveness results for mHealth interventions targeting individuals diagnosed with, or at risk of, T2DM. The quality of reporting evidence will be assessed using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist. Results will be presented using a flowchart following the Preferred Reporting Items for Systematic Review and Meta-Analysis Protocols (PRISMA-P) guidelines. Graphical and tabulated representations of the results will be created for both descriptive and numerical results. The cost and cost-effectiveness values will be presented as reported by the original studies as well as converted into international dollars to allow comparability. As we are predicting heterogenous results, we will conduct a narrative and interpretive analysis of the data.Ethics and disseminationNo formal approval or review of ethics is required for this systematic review as it will involve the collection and analysis of secondary data. This protocol follows the current PRISMA-P guidelines. The review will provide information on the cost and cost-effectiveness of mHealth interventions targeting T2DM. These results will be disseminated through publication and submission to conferences for presentations and posters.PROSPERO registration numberCRD42019123476


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.


Author(s):  
Huong Ngoc Quynh Tran ◽  
Emma McMahon ◽  
Marj Moodie ◽  
Jaithri Ananthapavan

Background: While the number of retail interventions with impacts on diet- and/or health-related outcomes is increasing, the economic evaluation literature is limited. This review investigated (i) the cost-effectiveness of health-promoting food retail interventions and (ii) key assumptions adopted in these evaluations. Methods: A systematic review of published academic studies was undertaken (CRD42020153763). Fourteen databases were searched. Eligible studies were identified, analysed, and reported following the Preferred Reporting Items for Systematic Reviews and Meta-Analysis (PRISMA) guidelines. Results: Eight studies that evaluated 30 retail interventions were included in the review. Common outcomes reported were cost per healthy food item purchased/served or cost per disability-adjusted life year (DALY) averted. Four studies undertook cost-utility analyses and half of these studies concluded that retail interventions were cost-effective in improving health outcomes. Most studies did not state any assumptions regarding compensatory behaviour (i.e., purchases/consumption of non-intervention foods or food purchases/consumption from non-intervention settings) and presumed that sales data were indicative of consumption. Conclusion: The cost-effectiveness of retail-based health-promoting interventions is inconclusive. Future health-promoting retail interventions should regularly include an economic evaluation which addresses key assumptions related to compensatory behaviour and the use of sales data as a proxy for consumption.


2020 ◽  
Vol 10 (1) ◽  
Author(s):  
Juliane Andrea Duevel ◽  
Lena Hasemann ◽  
Luz María Peña-Longobardo ◽  
Beatriz Rodríguez-Sánchez ◽  
Isaac Aranda-Reneo ◽  
...  

Abstract Background Depressive disorders are associated with a high burden of disease. However, due to the burden posed by the disease on not only the sufferers, but also on their relatives, there is an ongoing debate about which costs to include and, hence, which perspective should be applied. Therefore, the aim of this paper was to examine whether the change between healthcare payer and societal perspective leads to different conclusions of cost-utility analyses in the case of depression. Methods A systematic literature search was conducted to identify economic evaluations of interventions in depression, launched on Medline and the Cost-Effectiveness Registry of the Tufts University using a ten-year time horizon (2008–2018). In a two-stepped screening process, cost-utility studies were selected by means of specified inclusion and exclusion criteria. Subsequently, relevant findings was extracted and, if not fully stated, calculated by the authors of this work. Results Overall, 53 articles with 92 complete economic evaluations, reporting costs from healthcare payer/provider and societal perspective, were identified. More precisely, 22 estimations (24%) changed their results regarding the cost-effectiveness quadrant when the societal perspective was included. Furthermore, 5% of the ICURs resulted in cost-effectiveness regarding the chosen threshold (2% of them became dominant) when societal costs were included. However, another four estimations (4%) showed the opposite result: these interventions were no longer cost-effective after the inclusion of societal costs. Conclusions Summarising the disparities in results and applied methods, the results show that societal costs might alter the conclusions in cost-utility analyses. Hence, the relevance of the perspectives chosen should be taken into account when carrying out an economic evaluation. This systematic review demonstrates that the results of economic evaluations can be affected by different methods available for estimating non-healthcare costs.


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.


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