scholarly journals A Systematic Review of Cost-Effectiveness Analyses of Novel Agents in the Treatment of Multiple Myeloma

Cancers ◽  
2021 ◽  
Vol 13 (22) ◽  
pp. 5606
Author(s):  
Maarten R. Seefat ◽  
David G. J. Cucchi ◽  
Stijn Dirven ◽  
Kaz Groen ◽  
Sonja Zweegman ◽  
...  

Background: Novel therapies for multiple myeloma (MM) promise to improve outcomes but are also associated with substantial increasing costs. Evidence regarding cost-effectiveness of novel treatments is necessary, but a comprehensive up-to-date overview of the cost-effectiveness evidence of novel treatments is currently lacking. Methods: We searched Embase, Medline via Ovid, Web of Science and EconLIT ProQuest to identify all cost-effectiveness evaluations of novel pharmacological treatment of MM reporting cost per quality-adjusted life year (QALY) and cost per life year (LY) gained since 2005. Quality and completeness of reporting was assessed using the Consolidated Health Economic Evaluation Reporting Standards. Results: We identified 13 economic evaluations, comprising 32 comparisons. Our results show that novel agents generate additional LYs (range: 0.311–3.85) and QALYs (range: 0.1–2.85) compared to backbone regimens and 0.02 to 1.10 LYs and 0.01 to 0.91 QALYs for comparisons between regimens containing two novel agents. Lifetime healthcare costs ranged from USD 60,413 to 1,434,937 per patient. The cost-effectiveness ratios per QALY gained ranged from dominating to USD 1,369,062 for novel agents compared with backbone therapies and from dominating to USD 618,018 for comparisons between novel agents. Conclusions: Cost-effectiveness ratios of novel agents were generally above current willingness-to-pay thresholds. To ensure access, cost-effectiveness should be improved or cost-effectiveness ratios above current thresholds should be accepted.

Author(s):  
Zartashia Ghani ◽  
Johan Jarl ◽  
Johan Sanmartin Berglund ◽  
Martin Andersson ◽  
Peter Anderberg

The objective of this study was to critically assess and review empirical evidence on the cost-effectiveness of Mobile Health (mHealth) interventions for older adults. We systematically searched databases such as Pubmed, Scopus, and Cumulative Index of Nursing and Allied Literature (CINAHL) for peer-reviewed economic evaluations published in English from 2007 to 2018. We extracted data on methods and empirical evidence (costs, effects, incremental cost-effectiveness ratio) and assessed if this evidence supported the reported findings in terms of cost-effectiveness. The consolidated health economic evaluation reporting standards (CHEERS) checklist was used to assess the reporting quality of the included studies. Eleven studies were identified and categorized into two groups: complex smartphone communication and simple text-based communication. Substantial heterogeneity among the studies in terms of methodological approaches and types of intervention was observed. The cost-effectiveness of complex smartphone communication interventions cannot be judged due to lack of information. Limited evidence of cost-effectiveness was found for interventions related to simple text-based communications. Comprehensive economic evaluation studies are warranted to assess the cost-effectiveness of mHealth interventions designed for older adults.


Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 5604-5604 ◽  
Author(s):  
Gerry Oster ◽  
Ariel Berger ◽  
Rebecca Bornheimer ◽  
Gary Binder ◽  
Yasir Nagarwala

Abstract Introduction The efficacy and safety of the novel agents, lenalidomide and bortezomib, in previously untreated MM has been demonstrated in several randomized controlled trials (Online-OnlyTs). In this study, we examine the cost-effectiveness of lenalidomide-melphalan–prednisone induction followed by lenalidomide maintenance (MPR-R), and bortezomib-melphalan-prednisone (VMP), respectively, versus melphalan-prednisone (MP), in patients with previously untreated MM. Methods We developed a partitioned-survival model to estimate expected clinical outcomes and costs in newly diagnosed MM patients receiving MPR-R, VMP, or MP as first-line therapy. The model had 3 mutually exclusive health states: (1) “progression-free, alive”; (2) “post-progression, alive”; and (3) “death.”  Progression-free survival (PFS) for MP was estimated by aggregating data across five Online-OnlyTs (Palumbo 2012, San Miguel 2008, Facon 2007, Hulin 2009, Palumbo 2006). Estimates of PFS for MPR-R and VMP were based on an adjusted indirect treatment comparison with MP, using data from MM-015 for MPR-R (Palumbo 2012) and VISTA for VMP (San Miguel 2008). Since many MP patients in both MM-015 and VISTA “crossed over” to lenalidomide and bortezomib following disease progression in these trials, we estimated post-progression survival (PPS) for MPR-R and VMP based on a review of novel agents in MM (Messori 2011), which reported mean PPS of 30.9 months. Costs of MPR-R and VMP were estimated based on actual use of study drug in Online-OnlyTs; costs of adverse events as well as other disease-related costs were estimated based on published data. Health-state utilities also were estimated using published data.  All costs were expressed in 2012 US$.  Cost-effectiveness of MPR-R and VMP versus MP was examined in terms of cost per life-year (LY) gained, cost per quality-adjusted life-year (QALY) gained, and cost per progression-free life-year gained.  Future costs and benefits were discounted at 3% annually. Results Mean estimated PFS was 3.4 years for MPR-R, 2.6 years for VMP, and 1.7 years for MP; corresponding estimates for OS were 6.0 years, 5.2 years, and 4.3 years, respectively (Table 1). Mean total expected lifetime costs (discounted) are reported in the Table. The incremental cost per life-year (LY) gained versus MP was $75,392 for MPR-R and $86,213 for VMP; corresponding estimates of the incremental cost per QALY gained were $91,794 and $106,211, respectively (Figure 1).  The incremental cost per progression-free LY (PFLY) gained versus MP was $70,666 for MPR-R and $80,565 for VMP. Conclusions In patients with previously untreated MM, cost-effectiveness ratios for MPR-R and VMP are well within the range reported for other well-accepted novel therapies in oncology. $/LY: Incremental cost per life-year gained; $/QALY: Incremental cost per quality-adjusted life-year gained; $/PFLY: Incremental cost per progression-free life-year gained Support Funded by Celgene Corporation Disclosures: Oster: Celgene: Research Funding. Off Label Use: Lenalidomide (immunomodulatory agent), bortezomib (proteosome inhibitor), melphalan (alkylator), and prednisone (steroid), are all treatments for multiple myeloma. Berger:Celgene: Research Funding. Bornheimer:Celgene: Research Funding. Binder:Celgene: Employment, Equity Ownership. Nagarwala:Celgene: Employment, Equity Ownership.


BMJ Open ◽  
2018 ◽  
Vol 8 (8) ◽  
pp. e022022 ◽  
Author(s):  
Elizabeth M Camacho ◽  
Gemma E Shields

ObjectivesAnxiety and/or depression during pregnancy or year after childbirth is the most common complication of childbearing. Economic evaluations of interventions for the prevention or treatment of perinatal anxiety and/or depression (PAD) were systematically reviewed with the aim of guiding researchers and commissioners of perinatal mental health services towards potentially cost-effective strategies.MethodsElectronic searches were conducted on the MEDLINE, PsycINFO and NHS Economic Evaluation and Health Technology Assessment databases in September 2017 to identify relevant economic evaluations published since January 2000. Two stages of screening were used with prespecified inclusion/exclusion criteria. A data extraction form was designed prior to the literature search to capture key data. A published checklist was used to assess the quality of publications identified.ResultsOf the 168 non-duplicate citations identified, 8 studies met the inclusion criteria for the review; all but one focussing solely on postnatal depression in mothers. Interventions included prevention (3/8), treatment (3/8) or identification plus treatment (2/8). Two interventions were likely to be cost-effective, both incorporated identification plus treatment. Where the cost per quality-adjusted life year (QALY) gained was reported, interventions ranged from being dominant (cheaper and more effective than usual care) to costing £39 875/QALY.ConclusionsUncertainty and heterogeneity across studies in terms of setting and design make it difficult to make direct comparisons or draw strong conclusions. However, the two interventions incorporating identification plus treatment of perinatal depression were both likely to be cost-effective. Many gaps were identified in the economic evidence, such as the cost-effectiveness of interventions for perinatal anxiety, antenatal depression or interventions for fathers.PROSPERO registration numberCRD42016051133.


BMJ Open ◽  
2021 ◽  
Vol 11 (8) ◽  
pp. e047173
Author(s):  
Madelé van Dyk ◽  
Norma Bulamu ◽  
Chelsea Boylan ◽  
Anna M Mc Laughlin ◽  
Ganessan Kichenadasse ◽  
...  

IntroductionOral anticancer drugs (OADs) have rapidly expanded with more than 70 OADs targeting several molecular targets. Many of the OADs exert an exposure–response relationship but still, a ‘one-size fits-all’ dose is used, ignoring interindividual variability. Several of these OADs share similar mechanisms of actions and thus target the same cancer and has resulted in a substantial research focus on comparing the health benefit of each. However, significantly less is known about the cost–benefit associated with OADs. This paper will provide a protocol to systematically review studies that have evaluated the cost-effectiveness of OADs and their associated individualised dosing interventions.Methods and analysisSystematic review methodology will be applied to identify, select and extract data from published economic evaluation (costs and outcomes/benefits) studies of OADs and their associated individualised dosing interventions. Bibliographic databases (eg, Ovid EMBASE, Ovid MEDLINE) will be used to perform the systematic literature search (between 1 January 2000 and October 2020). Only full economic evaluations will be included, but no restrictions on study outcomes will be applied. The quality of included primary studies will be assessed using the Consolidated Health Economic Evaluation Reporting Standards checklist for reporting economic evaluations. Studies with low-quality evidence will be excluded. A narrative synthesis of the results from the included studies will be undertaken, with a subgroup analysis where appropriate.Ethics and disseminationThis systematic review will not require ethics approval as there will not be any collection of primary data. Findings of this review will be disseminated through publications in peer-reviewed journals, presentations at workshops or conferences and sharing through a media release. Findings from this review will provide evidence to direct and inform policy-makers where cost-neutral strategies may be effective or where dose individualising strategies may be economically beneficial. Additionally, gaps will be identified in the current literature to inform future-related research.PROSPERO registration numberCRD42020218170.Electronic supplemental materialThe online version of this article contains supplemental material, which is available to authorised users.


2019 ◽  
Vol 20 (8) ◽  
pp. 1123-1131
Author(s):  
Nikita M. John ◽  
Stuart J. Wright ◽  
Sean P. Gavan ◽  
Caroline M. Vass

Abstract Background Technological progress has led to changes in the antenatal screening programmes, most significantly the introduction of non-invasive prenatal testing (NIPT). The availability of a new type of testing changes the type of information that the parent(s) require before, during and after screening to mitigate anxiety about the testing process and results. Objectives To identify the extent to which economic evaluations of NIPT have accounted for the need to provide information alongside testing and the associated costs and health outcomes of information provision. Methods A systematic review of economic evaluations of NIPTs (up to February 2018) was conducted. Medline, Embase, CINAHL and PsychINFO were searched using an electronic search strategy combining a published economic search filter (from NHS economic evaluations database) with terms related to NIPT and screening-related technologies. Data were extracted using the Consolidated Health Economic Evaluation Reporting Standards framework and the results were summarised as part of a narrative synthesis. Results A total of 12 economic evaluations were identified. The majority of evaluations (n = 10; 83.3%) involved cost effectiveness analysis. Only four studies (33.3%) included the cost of providing information about NIPT in their economic evaluation. Two studies considered the impact of test results on parents’ quality of life by allowing utility decrements for different outcomes. Some studies suggested that the challenges of valuing information prohibited their inclusion in an economic evaluation. Conclusion Economic evaluations of NIPTs need to account for the costs and outcomes associated with information provision, otherwise estimates of cost effectiveness may prove inaccurate.


Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 4738-4738
Author(s):  
Sarah Perry ◽  
Sylvia McCulloch

Background: Survival outcomes for multiple myeloma have improved dramatically since the introduction of novel therapeutic agents. While these drugs are highly effective in improving quantity and quality of life in patients with multiple myeloma, they are come at a significant financial cost. Cost effectiveness analysis is a commonly used tool to compare drug regimens to maximize value for health care dollars spent. The objective of this review is to assess the economic evidence for novel agents in the treatment of multiple myeloma. Methods: A systematic literature review was conducted to assess the cost effectiveness and cost utility of novel agents in patients with multiple myeloma. The PRISMA checklist was followed. Medline (1946-present), EMBASE (1974-present), and Cochrane Database of Systematic Reviews (2005-present) were searched up to July 2019 for original publications that assessed the economic evidence for novel agents in multiple myeloma. Data was collected for all of the studies that met final inclusion criteria. The incremental cost effectiveness ratio (ICER) was the main outcome. Foreign currencies were converted to US dollars adjusted for inflation. The Quality of Health Economic Studies (QHES) checklist was used to assess study quality. Results: The final search identified 279 records, however, 60 were duplicates. 219 articles were screened, however an additional 201 were excluded for the following reasons: did not meet inclusion criteria (100), systematic reviews or meta-analyses (9), letters to the editor (3), or conference abstracts (88). This left a remaining 19 abstracts for full text review, however full text was not available for 4 articles. Thus, 15 articles were included in the systematic review. Fifteen studies were identified involving novel agents (bortezomib, carfilzomib, thalidomide, lenalidomide, pomalidomide, ixazomib and daratumumab). Three studies assessed the cost effectiveness of novel agents in the frontline setting, 1 assessed the cost effectiveness of maintenance following autologous stem cell transplantation, and the remaining 9 assessed novel agents in the relapsed setting. There was significant heterogeneity in the national perspective of the assessments with the majority being from a payer's perspective in the United States. Economic models varied considerably between the studies with the majority being one of: a partitioned survival analysis, Markov model or discrete event simulation. The time horizon for the models varied between 2 years and a lifetime with the majority being 10 years or longer. Economic evidence was of at least fair quality as assessed by the QHES checklist. The results of the economic analyses are summarized in Table 1. In general, novel agents were considered cost effective when used in both the frontline and relapsed setting. They were cost effective when compared to both steroids and traditional cytotoxic chemotherapy. While combinations of novel agents are clinically effective, cost effectiveness was dependent on individual drug prices as well as one's willingness to pay threshold. The main factors that influence the incremental cost-effectiveness ratio are survival outcomes and drug price. Discussions and Conclusions: This review highlights the need for ongoing research into cost effectiveness of novel agents in multiple myeloma. In general, novel agents are considered cost effective, however; there were a relatively small number of papers ion this topic and conclusions should be drawn with caution regarding the cost effectiveness of specific agents and regimens. Additionally, the studies in this review were quite heterogeneous in terms of the country of analysis, economic model, and study population, which will also influence their generalizability. As well, economic analyses of the same drug regimens showed significant variability, as was the case of lenalidomide maintenance, dependent on data inputs used. Further, the majority of these studies relied on list price for their cost inputs into the model, thus the actual cost effectiveness of a treatment regimen will depend on the final, negotiated drug price, which is often significantly less. Finally, as these models do not account for other factors, such as sequencing and patient and provider preferences, caution must be used when using economic analysis as the sole factor in guiding decision making. Disclosures McCulloch: Celgene: Honoraria; Amgen: Honoraria.


2021 ◽  
Vol 13 (1) ◽  
Author(s):  
Brian Lentz ◽  
Tiffany Fong ◽  
Randall Rhyne ◽  
Nicholas Risko

Abstract Background The use of ultrasound (US) in emergency departments (ED) has become widespread. This includes both traditional US scans performed by radiology departments as well as point-of-care US (POCUS) performed by bedside clinicians. There has been significant interest in better understanding the appropriate use of imaging and where opportunities to enhance cost-effectiveness may exist. The purpose of this systematic review is to identify published evidence surrounding the cost-effectiveness of US in the ED and to grade the quality of that evidence. Methods We performed a systematic review of the literature following Preferred Reporting Items for Systematic Review and Meta-Analyses (PRISMA) guidelines. Studies were considered for inclusion if they were: (1) economic evaluations, (2) studied the clinical use of ultrasound, and (3) took place in an emergency care setting. Included studies were critically appraised using the Consolidated Health Economic Evaluation Reporting Standards checklist. Results We identified 631 potentially relevant articles. Of these, 35 studies met all inclusion criteria and were eligible for data abstraction. In general, studies were supportive of the use of US. In particular, 11 studies formed a strong consensus that US enhanced cost-effectiveness in the investigation of pediatric appendicitis and 6 studies supported enhancements in the evaluation of abdominal trauma. Across the studies, weaknesses in methodology and reporting were common, such as lack of sensitivity analyses and inconsistent reporting of incremental cost-effectiveness ratios. Conclusions The body of existing evidence, though limited, generally demonstrates that the inclusion of US in emergency care settings allows for more cost-effective care. The most definitive evidence for improvements in cost-effectiveness surround the evaluation of pediatric appendicitis, followed by the evaluation of abdominal trauma. POCUS outside of trauma has had mixed results.


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.


Immunotherapy ◽  
2021 ◽  
Author(s):  
Wei Jiang ◽  
Zhichao He ◽  
Tiantian Zhang ◽  
Chongchong Guo ◽  
Jianli Zhao ◽  
...  

Aim: To evaluate the cost–effectiveness of ribociclib plus fulvestrant versus fulvestrant in hormone receptor-positive/human EGF receptor 2-negative advanced breast cancer. Materials & methods: A three-state Markov model was developed to evaluate the costs and effectiveness over 10 years. Direct costs and utility values were obtained from previously published studies. We calculated incremental cost–effectiveness ratio to evaluate the cost–effectiveness at a willingness-to-pay threshold of $150,000 per additional quality-adjusted life year. Results: The incremental cost–effectiveness ratio was $1,073,526 per quality-adjusted life year of ribociclib plus fulvestrant versus fulvestrant. Conclusions: Ribociclib plus fulvestrant is not cost-effective versus fulvestrant in the treatment of advanced hormone receptor-positive/human EGF receptor 2-negative breast cancer. When ribociclib is at 10% of the full price, ribociclib plus fulvestrant could be cost-effective.


2010 ◽  
Vol 196 (5) ◽  
pp. 396-403 ◽  
Author(s):  
Djøra I. Soeteman ◽  
Roel Verheul ◽  
Jos Delimon ◽  
Anke M. M. A. Meerman ◽  
Ellen van den Eijnden ◽  
...  

BackgroundRecommendations on current clinical guidelines are informed by limited economic evidence.AimsA formal economic evaluation of three modalities of psychotherapy for patients with cluster B personality disorders.MethodA probabilistic decision-analytic model to assess the cost-effectiveness of out-patient, day hospital and in-patient psychotherapy over 5 years in terms of cost per recovered patient-year and cost per quality-adjusted life-year (QALY). Analyses were conducted from both societal and payer perspectives.ResultsFrom the societal perspective, the most cost-effective choice switched from out-patient to day hospital psychotherapy at a threshold of €12 274 per recovered patient-year; and from day hospital to in-patient psychotherapy at €113 298. In terms of cost per QALY, the optimal strategy changed at €56 325 and €286 493 per QALY respectively. From the payer perspective, the switch points were at €9895 and €155 797 per recovered patient-year, and €43 427 and €561 188 per QALY.ConclusionsOut-patient psychotherapy and day hospital psychotherapy are the optimal treatments for patients with cluster B personality disorders in terms of cost per recovered patient-year and cost per QALY.


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