scholarly journals Cost-Effectiveness Analyses of FDA-Approved Drugs for Cancer Indications, 2015-2017

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 5833-5833 ◽  
Author(s):  
Ohad Oren

Abstract Background: In addition to a drug's anti-tumor efficacy and tissue toxicity, it is now apparent that affordability is a pivotal factor determining the net therapeutic value of an anti-cancer compound. Cost-Effectiveness Analyses (CEA) are therefore key to appreciating the overall balance between the clinical and economical repercussions of a pharmaceutical agent. We aimed to investigate the cost-effectiveness data of newly approved drugs in cancer care. Methods: The FDA website was reviewed for all drugs approved for any cancer indication between the years 2015-2017. A systematic search of Pubmed and Google Scholar was conducted for Cost-Effectiveness Analyses (CEA) of each of these medications. Average Wholesale Prices were collected from Uptodate.com. Results: 30 drugs were approved for cancer indications in 2015-2017. Each of the approved drugs had an average of 1.27 CEA studies (range 0-6). 4 of the 38 (10.5%) available CEAs were done before approval of an index drug. 18 (60%) of the approved medications had an available published CEA at the time of our search (March, 2018). Partition survival modelling (31.5%) and Markov modelling (26.3%) were the most common methods of analysis. 47.3% (18/38) of the CEAs were thought to reflect a favorable ICER per the analysis's investigators. The nature of the malignancy (solid versus hematologic) did not distinguish between the mean number of a drug's CEAs (1.25; 1.2) but drugs for solid cancer indications were less likely to be associated with a favorable CE assessment compared with hematological cancers (47.8% versus 58.3%). "Expensive" drugs (defined as monthly cost greater than 8,618 dollars) had a lower mean number of CEA studies than "less expensive" drugs (1.09 versus 1.36) and were less likely to be associated with a favorable cost-effectiveness profile (27.2% versus 73.6%). 47.3% (18/38) of the economic evaluations were published as conference proceeding/abstract, 36.8% (14/38) were in the form of a full-text article, and the remainder as journal letters or conference posters (6/38; 15.7%). Conclusion: Insufficient data on the cost-effectiveness profile of novel anti-cancer medications jeopardises our ability to determine their real value. Although these drugs are being routinely used in a large-scale fashion, gaps persist as to their financial harm relative to the associated (potential) clinical benefit. Our study shows that recently-approved anti-cancer drugs have a very small number of CEAs to back their clinical-societal merit. Paradoxically, more costly drugs have fewer CEAs compared with cheaper drugs. Also of note, the majority of the analyses followed the approval of the drug, were published in a non full-text format, and were associated with non-sustainable ICER values. We argue that publication of rigorous, peer-reviewed CEAs should be a mandatory pre-approval step for academia/industry and serve as a pre-requisite to the routine distribution and usage of new cancer-directed medications. Disclosures No relevant conflicts of interest to declare.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 4547-4547
Author(s):  
Ozlem Topaloglu ◽  
Chuck Stevens ◽  
Saurabh Aggarwal

Abstract Abstract 4547 Targeted cancer therapies for hematological malignancies have rapidly gained uptake in different types of indications, and some have become gold standard of treatment. However, the high cost of these therapies poses challenges for payers, patients and physicians. Methods To understand the cost-effectiveness of these new classes of drugs we reviewed the published cost-effectiveness studies and analyzed their cost/QALY, time horizon, comparators, indications and model approaches. Results Targeted therapies for hematological cancers have much lower and less variable cost/QALY than branded drugs indicated for solid tumors. For example, incremental cost effectiveness ratio (ICER) for imatinib ranges from $40,000 to $45,000/QALY, while ICER for bevacizumab ranges from $125,000 to $325,000/QALY. Secondly, the choice of comparator in some occasions may depend on the country of use, therefore models are required to be customized to local regimens. In one of the cost-effectiveness analysis for bortezomib for refractory multiple myeloma patients, NICE questioned the use of high dose dexamethasone (HDD) as comparator. Third, there is also some variability in methods used by different manufacturers, some used cost/QALY whereas others used cost/LYG. Conclusion: This analysis shows the range, variability and methods used for calculation of ICER values for these targeted cancer therapies and provides lessons for executives and policy makers. Disclosures: No relevant conflicts of interest to declare.


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.


2020 ◽  
Vol 24 (2) ◽  
pp. 1-180 ◽  
Author(s):  
Nigel Fleeman ◽  
Rachel Houten ◽  
Adrian Bagust ◽  
Marty Richardson ◽  
Sophie Beale ◽  
...  

Background Thyroid cancer is a rare cancer, accounting for only 1% of all malignancies in England and Wales. Differentiated thyroid cancer (DTC) accounts for ≈94% of all thyroid cancers. Patients with DTC often require treatment with radioactive iodine. Treatment for DTC that is refractory to radioactive iodine [radioactive iodine-refractory DTC (RR-DTC)] is often limited to best supportive care (BSC). Objectives We aimed to assess the clinical effectiveness and cost-effectiveness of lenvatinib (Lenvima®; Eisai Ltd, Hertfordshire, UK) and sorafenib (Nexar®; Bayer HealthCare, Leverkusen, Germany) for the treatment of patients with RR-DTC. Data sources EMBASE, MEDLINE, PubMed, The Cochrane Library and EconLit were searched (date range 1999 to 10 January 2017; searched on 10 January 2017). The bibliographies of retrieved citations were also examined. Review methods We searched for randomised controlled trials (RCTs), systematic reviews, prospective observational studies and economic evaluations of lenvatinib or sorafenib. In the absence of relevant economic evaluations, we constructed a de novo economic model to compare the cost-effectiveness of lenvatinib and sorafenib with that of BSC. Results Two RCTs were identified: SELECT (Study of [E7080] LEnvatinib in 131I-refractory differentiated Cancer of the Thyroid) and DECISION (StuDy of sorafEnib in loCally advanced or metastatIc patientS with radioactive Iodine-refractory thyrOid caNcer). Lenvatinib and sorafenib were both reported to improve median progression-free survival (PFS) compared with placebo: 18.3 months (lenvatinib) vs. 3.6 months (placebo) and 10.8 months (sorafenib) vs. 5.8 months (placebo). Patient crossover was high (≥ 75%) in both trials, confounding estimates of overall survival (OS). Using OS data adjusted for crossover, trial authors reported a statistically significant improvement in OS for patients treated with lenvatinib compared with those given placebo (SELECT) but not for patients treated with sorafenib compared with those given placebo (DECISION). Both lenvatinib and sorafenib increased the incidence of adverse events (AEs), and dose reductions were required (for > 60% of patients). The results from nine prospective observational studies and 13 systematic reviews of lenvatinib or sorafenib were broadly comparable to those from the RCTs. Health-related quality-of-life (HRQoL) data were collected only in DECISION. We considered the feasibility of comparing lenvatinib with sorafenib via an indirect comparison but concluded that this would not be appropriate because of differences in trial and participant characteristics, risk profiles of the participants in the placebo arms and because the proportional hazard assumption was violated for five of the six survival outcomes available from the trials. In the base-case economic analysis, using list prices only, the cost-effectiveness comparison of lenvatinib versus BSC yields an incremental cost-effectiveness ratio (ICER) per quality-adjusted life-year (QALY) gained of £65,872, and the comparison of sorafenib versus BSC yields an ICER of £85,644 per QALY gained. The deterministic sensitivity analyses show that none of the variations lowered the base-case ICERs to < £50,000 per QALY gained. Limitations We consider that it is not possible to compare the clinical effectiveness or cost-effectiveness of lenvatinib and sorafenib. Conclusions Compared with placebo/BSC, treatment with lenvatinib or sorafenib results in an improvement in PFS, objective tumour response rate and possibly OS, but dose modifications were required to treat AEs. Both treatments exhibit estimated ICERs of > £50,000 per QALY gained. Further research should include examination of the effects of lenvatinib, sorafenib and BSC (including HRQoL) for both symptomatic and asymptomatic patients, and the positioning of treatments in the treatment pathway. Study registration This study is registered as PROSPERO CRD42017055516. Funding The National Institute for Health Research Health Technology Assessment programme.


2021 ◽  
Author(s):  
Sergio Marin ◽  
Mateu Serra-Prat ◽  
Omar Ortega ◽  
Pere Clavé

Abstract Background and purpose: Oropharyngeal Dysphagia (OD) affects 40-81% of patients after stroke. A recent systematic review on the costs of OD and it’s main complications showed higher acute and long-term costs for those patients who developed OD, malnutrition and pneumonia after stroke. These results suggest that appropriate management of post-stroke OD could lead to reduction of clinical complications and significant cost savings. The purpose of this systematic review is to assess the available literature exploring the efficiency or cost-effectiveness of available healthcare interventions on the appropriate management of OD. Methods: A systematic review on economic evaluations of health care interventions on post-stroke patients with OD following PRISMA recommendations will be performed. MEDLINE, Embase, the National Health Service Economic Evaluation Database and the Cost-Effectiveness Analysis Registry Database will be searched and a subsequent reference check will be done. English and Spanish literature will be included without date restrictions. Studies will be included if they refer to economic evaluations or studies in which cost savings were reported in post-stroke patients suffering OD. Studies will be excluded if they are partial economic evaluation studies, if they refer to esophageal dysphagia, or if OD is caused by causes different from stroke. Evidence will be presented and synthetized with a narrative method and using tables. Quality evaluation will be done using Consolidated Health Economic Evaluation Reporting Standards (CHEERS) Statement. Discussion: The protocol for this systematic review is the first step to assess the cost-effectiveness of the healthcare interventions that have been described as potential treatments for post-stroke OD. This systematic review will summarize the current evidence on the relation between cost and benefits associated with the appropriate management of OD in post-stroke patients. Systematic review registration: PROSPERO CRD42020136245


2020 ◽  
Author(s):  
Joe Botham ◽  
Amy Clark ◽  
Thomas Steare ◽  
Ruth Stuart ◽  
Sian Oram ◽  
...  

AbstractBackgroundDiagnoses of “personality disorder” are prevalent among people using community secondary mental health services. Whilst the effectiveness of a range of community-based treatments have been considered, as the NHS budget is finite, it is also important to consider the cost-effectiveness of those interventions.AimsTo assess the cost-effectiveness of primary or secondary care community-based interventions for people with complex emotional needs that meet criteria for a diagnosis of “personality disorder” to inform healthcare policy making.MethodSystematic review (PRESPORO #: CRD42020134068) of five databases, supplemented by reference list screening and citation tracking of included papers. We included economic evaluations of interventions for adults with complex emotional needs associated with a diagnosis of ‘personality disorder’ in community mental health settings published between before 18 September 2019. Study quality was assessed using the CHEERS statement. Narrative synthesis was used to summarise study findings.ResultsEighteen studies were included. The studies mainly evaluated psychotherapeutic interventions. Studies were also identified which evaluated altering the setting in which care was delivered and joint crisis plans. No strong economic evidence to support a single intervention or model of community-based care was identified.ConclusionThere is no robust economic evidence to support a single intervention or model of community-based care for people with complex emotional needs. The review identified the strongest evidence for Dialectical Behavioural Therapy with all three identified studies indicating the intervention is likely to be cost-effective in community settings compared to treatment as usual. Further research is needed to provide robust evidence on the cost-effectiveness of community-based interventions upon which decision makers can confidently base guidelines or allocate resources.


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.


2014 ◽  
Vol 30 (3) ◽  
pp. 273-281 ◽  
Author(s):  
Davide Minniti ◽  
Ottavio Davini ◽  
Maria Rosaria Gualano ◽  
Maria Michela Gianino

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.


Author(s):  
Annelies Boonen

Consideration of costs and budgets plays an increasingly important role in decisions on access to innovative technologies. When clinicians want to influence such decisions, it is essential to understand the information on the burden of the disease and the evidence on cost-effectiveness of technologies. This chapter provides guidance to understanding the key methodological principles of economic evaluations, and describes available evidence on these issues in axial spondyloarthritis (axSpA). In the prebiologics era, the cost-of-illness for society of ankylosing spondylitis was slightly lower than for rheumatoid arthritis, and substantially lower than chronic low back pain. Cost of sick leave and work disability accounted for up to 75% of total cost-of-illness. Treatment with biologics increased cost-of-illness substantially, but the important gain in quality-adjusted life years resulted in acceptable cost-effectiveness in patients with active disease. There remains a gap in knowledge about the cost-effectiveness of diagnosing and treating axSpA earlier.


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