scholarly journals Supporting Formulary Decisions: The Discovery of New Facts or Constructed Evidence?

2016 ◽  
Vol 7 (2) ◽  
Author(s):  
Paul C Langley
Keyword(s):  
Cost Effectiveness ◽  
Lifetime Cost ◽  
Cost Effective ◽  
Evidence Base ◽  
Normal Science ◽  
Critical Question ◽  
Reference Case ◽  
Life Years ◽  
The One ◽  
The Uk

A critical question, given the growing importance of more targeted therapies to support personalized and precision medicine, is the credibility of the evidence base to support formulary decisions and pricing. On the one hand, for those who subscribe to the reference case model of the National Institute of Health and Care Excellence (NICE) in the UK, the decision rests upon the creation of modeled or simulated imaginary worlds and the application of threshold willingness-to-pay cost-per-QALY thresholds. On the other hand, for those who subscribe to the standards of normal science, the decision rests upon the ability to evaluate competing claims, both clinical and cost-effective, in a timeframe that is meaningful to a formulary committee. If we subscribe to the scientific method where the focus is on the discovery of new facts, untestable claims for clinical benefit and cost-effectiveness, such as created claims for lifetime cost per-quality-adjusted discounted life years (QALYs), are properly relegated to the category of pseudoscience. We have no idea, and will never know, whether the claims are right or even if they are wrong. If formulary decisions are to respect the standards of normal science then there has to be a commitment to claims evaluation. A willingness to accept new products provisionally, subject to an agreed protocol to support the evaluation of clinical and cost-effectiveness claims. This dichotomy between the standards of normal science and pseudoscience is explored in the context of published claims for cost-effectiveness and recommendations for product pricing in the US.   Type: Commentary

scholarly journals First-Line Atezolizumab Plus Bevacizumab versus Sorafenib in Hepatocellular Carcinoma: A Cost-Effectiveness Analysis

Cancers ◽  
2021 ◽  
Vol 13 (5) ◽  
pp. 931
Author(s):  
Chi-Leung Chiang ◽  
Sik-Kwan Chan ◽  
Shing-Fung Lee ◽  
Horace Cheuk-Wai Choi
Keyword(s):  
Hepatocellular Carcinoma ◽  
Cost Effectiveness ◽  
Lifetime Cost ◽  
Cost Effective ◽  
First Line ◽  
First Line Therapy ◽  
Payer Perspective ◽  
Life Years ◽  
Using Data

Background: The IMbrave 150 trial revealed that atezolizumab plus bevacizumab (atezo–bev) improves survival in patients with unresectable hepatocellular carcinoma (HCC) (1 year survival rate: 67.2% vs. 54.6%). We assessed the cost-effectiveness of atezo–bev vs. sorafenib as first-line therapy in patients with unresectable HCC from the US payer perspective. Methods: Using data from the IMbrave 150, we developed a Markov model to compare the lifetime cost and efficacy of atezo–bev as first-line systemic therapy in HCC with those of sorafenib. The main outcomes were life-years, quality-adjusted life-years (QALYs), lifetime costs, and incremental cost-effectiveness ratio (ICER). Results: Atezo–bev demonstrated a gain of 0.44 QALYs, with an additional cost of USD 79,074. The ICER of atezo–bev was USD 179,729 per QALY when compared with sorafenib. The model was most sensitive to the overall survival hazard ratio and body weight. If we assumed that all patients at the end of the IMbrave 150 trial were cured of HCC, atezo–bev was cost-effective (ICER USD 53,854 per QALY). However, if all patients followed the Surveillance, Epidemiology, and End Results data, the ICER of atezo–bev was USD 385,857 per QALY. Reducing the price of atezo–bev by 20% and 29% would satisfy the USD 150,000/QALY and 100,000/QALY willingness-to-pay threshold. Moreover, capping the duration of therapy to ≤12 months or reducing the dosage of bev to ≤10 mg/kg would render atezo–bev cost-effective. Conclusions: The long-term effectiveness of atezo–bev is a critical but uncertain determinant of its cost-effectiveness. Price reduction would favorably influence cost-effectiveness, even if long-term clinical outcomes were modest. Further studies to optimize the duration and dosage of therapy are warranted.

scholarly journals A Cost-Effectiveness Analysis of Newborn Screening for Severe Combined Immunodeficiency in the UK

2019 ◽  
Vol 5 (3) ◽  
pp. 28 ◽  
Author(s):  
Alice Bessey ◽  
James Chilcott ◽  
Joanna Leaviss ◽  
Carmen de la Cruz ◽  
Ruth Wong
Keyword(s):  
Cost Effectiveness ◽  
Severe Combined Immunodeficiency ◽  
Cost Effective ◽  
Sensitivity Analyses ◽  
Combined Immunodeficiency ◽  
Tree Model ◽  
Life Years ◽  
The Uk ◽  
The Cost ◽  
The Impact

Severe combined immunodeficiency (SCID) can be detected through newborn bloodspot screening. In the UK, the National Screening Committee (NSC) requires screening programmes to be cost-effective at standard UK thresholds. To assess the cost-effectiveness of SCID screening for the NSC, a decision-tree model with lifetable estimates of outcomes was built. Model structure and parameterisation were informed by systematic review and expert clinical judgment. A public service perspective was used and lifetime costs and quality-adjusted life years (QALYs) were discounted at 3.5%. Probabilistic, one-way sensitivity analyses and an exploratory disbenefit analysis for the identification of non-SCID patients were conducted. Screening for SCID was estimated to result in an incremental cost-effectiveness ratio (ICER) of £18,222 with a reduction in SCID mortality from 8.1 (5–12) to 1.7 (0.6–4.0) cases per year of screening. Results were sensitive to a number of parameters, including the cost of the screening test, the incidence of SCID and the disbenefit to the healthy at birth and false-positive cases. Screening for SCID is likely to be cost-effective at £20,000 per QALY, key uncertainties relate to the impact on false positives and the impact on the identification of children with non-SCID T Cell lymphopenia.

scholarly journals Challenges in synthesising cost-effectiveness estimates

2020 ◽  
Vol 9 (1) ◽  
Author(s):  
Gemma E. Shields ◽  
Jamie Elvidge
Keyword(s):  
Cost Effectiveness ◽  
Systematic Reviews ◽  
Meta Analysis ◽  
Cost Effective ◽  
Evidence Base ◽  
Decision Makers ◽  
Economic Evaluations ◽  
Quality Adjusted Life Years ◽  
Life Years ◽  
Study Designs

AbstractEconomic evaluations help decision-makers faced with tough decisions on how to allocate resources. Systematic reviews of economic evaluations are useful as they allow readers to assess whether interventions have been demonstrated to be cost effective, the uncertainty in the evidence base, and key limitations or gaps in the evidence base. The synthesis of systematic reviews of economic evaluations commonly takes a narrative approach whereas a meta-analysis is common step for reviews of clinical evidence (e.g. effectiveness or adverse event outcomes). As they are common objectives in other reviews, readers may query why a synthesis has not been attempted for economic outcomes. However, a meta-analysis of incremental cost-effectiveness ratios, costs, or health benefits (including quality-adjusted life years) is fraught with issues largely due to heterogeneity across study designs and methods and further practical challenges. Therefore, meta-analysis is rarely feasible or robust. This commentary outlines these issues, supported by examples from the literature, to support researchers and reviewers considering systematic review of economic evidence.

scholarly journals Cost-effectiveness of an insertable cardiac monitor in a high-risk population in the UK

Open Heart ◽  
2019 ◽  
Vol 6 (1) ◽  
pp. e001037 ◽  
Author(s):  
Claudia I Rinciog ◽  
Laura M Sawyer ◽  
Alexander Diamantopoulos ◽  
Mitchell S V Elkind ◽  
Matthew Reynolds ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
High Risk ◽  
Oral Anticoagulants ◽  
Cost Effective ◽  
Sensitivity Analyses ◽  
High Risk Population ◽  
Risk Population ◽  
Life Years ◽  
The Uk ◽  
The Cost

ObjectiveTo evaluate the cost-effectiveness of insertable cardiac monitors (ICMs) compared with standard of care (SoC) for detecting atrial fibrillation (AF) in patients at high risk of stroke (CHADS2 >2), using a UK National Health Service (NHS) perspective.MethodsUsing patient characteristics and clinical data from the REVEAL AF trial, a Markov model assessed the cost-effectiveness of detecting AF with an ICM compared with SoC. Costs and benefits were extrapolated across modelled patient lifetime. Ischaemic and haemorrhagic strokes, intracranial and extracranial haemorrhages and minor bleeds were modelled. Diagnostic and device costs were included, plus costs of treating stroke and bleeding events and costs of oral anticoagulants (OACs). Costs and health outcomes, measured as quality-adjusted life years (QALYs), were discounted at 3.5% per annum. One-way deterministic and probabilistic sensitivity analyses (PSA) were undertaken.ResultsThe total per-patient cost for ICM was £13 360 versus £11 936 for SoC (namely, annual 24 hours Holter monitoring). ICMs generated a total of 6.50 QALYs versus 6.30 for SoC. The incremental cost-effectiveness ratio (ICER) was £7140/QALY gained, below the £20 000/QALY acceptability threshold. ICMs were cost-effective in 77.4% of PSA simulations. The number of ICMs needed to prevent one stroke was 21 and to cause a major bleed was 37. ICERs were sensitive to assumed proportions of patients initiating or discontinuing OAC after AF diagnosis, type of OAC used and how intense the traditional monitoring was assumed to be under SoC.ConclusionsThe use of ICMs to identify AF in a high-risk population is cost-effective for the UK NHS.

scholarly journals Cost-effectiveness analysis of tranexamic acid for the treatment of traumatic brain injury, based on the results of the CRASH-3 randomised trial: a decision modelling approach

2020 ◽  
Vol 5 (9) ◽  
pp. e002716
Author(s):  
Jack Williams ◽  
Ian Roberts ◽  
Haleema Shakur-Still ◽  
Fiona E Lecky ◽  
Rizwana Chaudhri ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Tranexamic Acid ◽  
Randomised Trial ◽  
Cost Effective ◽  
Sensitivity Analyses ◽  
Risk Of Death ◽  
Life Years ◽  
Markov Decision Model ◽  
The Uk ◽  
The Cost

IntroductionAn estimated 69 million traumatic brain injuries (TBI) occur each year worldwide, with most in low-income and middle-income countries. The CRASH-3 randomised trial found that intravenous administration of tranexamic acid within 3 hours of injury reduces head injury deaths in patients sustaining a mild or moderate TBI. We examined the cost-effectiveness of tranexamic acid treatment for TBI.MethodsA Markov decision model was developed to assess the cost-effectiveness of treatment with and without tranexamic acid, in addition to current practice. We modelled the decision in the UK and Pakistan from a health service perspective, over a lifetime time horizon. We used data from the CRASH-3 trial for the risk of death during the trial period (28 days) and patient quality of life, and data from the literature to estimate costs and long-term outcomes post-TBI. We present outcomes as quality-adjusted life years (QALYs) and 2018 costs in pounds for the UK, and US dollars for Pakistan. Incremental cost-effectiveness ratios (ICER) per QALY gained were estimated, and compared with country specific cost-effective thresholds. Deterministic and probabilistic sensitivity analyses were also performed.ResultsTranexamic acid was highly cost-effective for patients with mild TBI and intracranial bleeding or patients with moderate TBI, at £4288 per QALY in the UK, and US$24 per QALY in Pakistan. Tranexamic acid was 99% and 98% cost-effective at the cost-effectiveness thresholds for the UK and Pakistan, respectively, and remained cost-effective across all deterministic sensitivity analyses. Tranexamic acid was even more cost-effective with earlier treatment administration. The cost-effectiveness for those with severe TBI was uncertain.ConclusionEarly administration of tranexamic acid is highly cost-effective for patients with mild or moderate TBI in the UK and Pakistan, relative to the cost-effectiveness thresholds used. The estimated ICERs suggest treatment is likely to be cost-effective across all income settings globally.

Cost-effectiveness of zoledronic acid in the prevention of skeletal-related events in patients with bone metastases from renal cell carcinoma: Comparison between France, Germany, and the United Kingdom

2009 ◽  
Vol 27 (15_suppl) ◽  
pp. 5106-5106
Author(s):  
M. F. Botteman ◽  
S. Kaura
Keyword(s):  
United Kingdom ◽  
Cost Effectiveness ◽  
Zoledronic Acid ◽  
Bone Metastases ◽  
Cost Effective ◽  
Sensitivity Analyses ◽  
Life Years ◽  
Health Related ◽  
Skeletal Related Events ◽  
The Uk

5106 Background: Zoledronic acid (ZOL) significantly reduces the risk of new skeletal-related events (SREs) in patients (pts) with bone metastases from RCC. This study assessed and compared the cost-effectiveness of ZOL in pts with RCC from French, German, and United Kingdom (UK) societal perspectives. Methods: This analysis was based on a retrospective analysis of RCC pts with bone metastases who were enrolled in a 9-mo trial of ZOL or placebo (PBO) plus concomitant antineoplastic therapy. A model was developed to simulate costs and quality-adjusted life-years (QALYs) experienced by study pts. The model included data and assumptions regarding SRE incidence, mortality, drug and administration costs, SRE costs, reduced quality of life (QOL) because of SREs and bone pain, and therapy duration. SRE costs were estimated using diagnosis-related group tariff information and published literature. Consistent with similar economic analyses, it was assumed that QOL decreased 20% to 80% (depending on SRE type) for 1 mo after each SRE experienced. Sensitivity analyses were performed to test the effects of alternate assumptions, with < 30,000/QALY considered cost-effective. Results: Compared with PBO-treated pts (n = 19), ZOL-treated pts (n = 27) experienced 1.07 fewer SREs/pt and gained discounted QALYs of approximately 0.1563 in France and Germany and 0.1575 in the UK. Discounted SRE-related costs were substantially lower among pts treated with ZOL vs PBO (-4,196 in France, -3,880 in Germany, and -3,355 in the UK). After including drug therapy costs, ZOL saved 1,358, 1,223, and 719 per pt in France, Germany, and the UK, respectively. In multivariate sensitivity analyses, ZOL saved costs in 67% to 77% of cases, depending on the country. ZOL resulted in a cost per QALY gained < 30,000 in approximately 93% of cases. Conclusions: Treatment with ZOL reduces SREs, improves QOL, and lowers health-related costs compared with PBO in French, German, and UK pts with bone metastases from RCC. Use of ZOL in these populations therefore provides health-related cost savings and is a cost-effective use of healthcare resources. [Table: see text]

scholarly journals Economic Evaluation of CYP2C19 Genotype-Guided Antiplatelet Therapy Compared to Universal use of Ticagrelor or Clopidogrel in Qatar

2020 ◽  
Author(s):  
Sawsan Ibrahim AlMukdad ◽  
Hazem Elewa ◽  
Daoud Al-Badriyeh
Keyword(s):  
Cost Effectiveness ◽  
Incremental Cost ◽  
Antiplatelet Therapy ◽  
Cost Effective ◽  
Cyp2c19 Genotype ◽  
Life Years ◽  
One Year ◽  
The One ◽  
Guided Therapy

Background: Patients having CYP2C19 loss-of-function alleles and receiving clopidogrel are at higher risk of adverse cardiovascular outcomes. Ticagrelor is a more effective and expensive antiplatelet that is unaffected by the CYP2C19 polymorphism. The main aim of the current research is to evaluate the cost-effectiveness among CYP2C19 genotype-guided therapy, universal ticagrelor, and universal clopidogrel after a percutaneous coronary intervention (PCI). Methods: A two-part simulation model, including a one-year decision-analytic model and a 20-year followup Markov model, was created to follow the use of (i) universal clopidogrel, (ii) universal ticagrelor, and (iii) genotype-guided antiplatelet therapy. Outcome measures were the incremental cost-effectiveness ratio (ICER, cost/success) and incremental cost-utility ratio (ICUR, cost/qualityadjusted life years [QALY]). Therapy success was defined as survival without myocardial infarction, stroke, cardiovascular death, stent thrombosis, and no therapy discontinuation because of adverse events, i.e. major bleeding and dyspnea. The model was based on a multivariate analysis, and a sensitivity analysis confirmed the robustness of the model outcomes. Results: Against universal clopidogrel, genotype-guided therapy was cost-effective over the one-year duration (ICER, USD 6,102 /success), and dominant over the long-term. Genotype-guided therapy was dominant over universal ticagrelor over the one-year duration and cost-effective over the long term (ICUR, USD 1,383 /QALY). Universal clopidogrel was dominant over ticagrelor over the short term, and cost-effective over the long-term (ICUR, 10,616 /QALY). Conclusion: CYP2C19 genotype-guided therapy appears to be the preferred antiplatelet strategy, followed by universal clopidogrel, and then universal ticagrelor for post-PCI patients in Qatar.

scholarly journals Cost–effectiveness of empagliflozin compared with liraglutide based on cardiovascular outcome trials in Type II diabetes

2020 ◽  
Author(s):  
Mafalda Ramos ◽  
Anastasia Ustyugova ◽  
Nikco Hau ◽  
Mark Lamotte
Keyword(s):  
Cost Effectiveness ◽  
Type Ii Diabetes ◽  
Indirect Comparison ◽  
Cost Effective ◽  
Standard Of Care ◽  
Base Case ◽  
Type Ii ◽  
Cardiovascular Outcome Trials ◽  
Life Years ◽  
The Uk

Aim: Cost–effectiveness (CE) analysis of empagliflozin+standard of care (SoC) compared with SoC and liraglutide+SoC, in patients with Type II diabetes and established cardiovascular disease, was conducted using evidence from cardiovascular outcomes trials. Methods: The IQVIA Core Diabetes Model was calibrated to predict same outcomes observed in EMPA-REG OUTCOME and LEADER trials. Three-year observed cardiovascular events of SoC, empagliflozin+SoC and liraglutide+SoC were derived from EMPA-REG OUTCOME trial and an indirect comparison. Time horizon was 50 years and the UK payer perspective was taken. Results: Empagliflozin+SoC dominated liraglutide+SoC with greater quality-adjusted life years and reduced costs. Base-case incremental CE ratio of 6428 GBP/QALY was observed for empagliflozin+SoC versus SoC. Conclusion: Results suggest that empagliflozin+SoC is cost effective versus SoC and liraglutide+SoC.

Cost-Effectiveness of Pharmacological Interventions for Smoking Cessation: A Literature Review and a Decision Analytic Analysis

2002 ◽  
Vol 22 (1_suppl) ◽  
pp. 26-37 ◽  
Author(s):  
Fujian Song ◽  
James Raftery ◽  
Paul Aveyard ◽  
Chris Hyde ◽  
Pelham Barton ◽  
...  
Keyword(s):  
Smoking Cessation ◽  
Cost Effectiveness ◽  
Cost Effective ◽  
Decision Analytic Model ◽  
Relative Cost ◽  
Pharmacological Interventions ◽  
Relative Cost Effectiveness ◽  
Life Years ◽  
The Uk ◽  
The Cost

To evaluate the relative cost-effectiveness of nicotine replacement therapy (NRT) and bupropion SR for smoking cessation, the authors reviewed published studies and developed a decision analytic model from the UK National Health Services perspective. Irrespective of the methods or assumptions involved, the results of published studies consistently indicated that NRT for smoking cessation is cost-effective. No published studies have evaluated the relative cost-effectiveness of bupropion SR for smoking cessation. The results of the decision analyses indicated that, as compared with advice or counseling alone, the incremental cost per life-years saved is about $1,441~$3,455 for NRT, $920~$2,150 for bupropion SR, and $1,282~$2,836 for NRT plus bupropion SR. The cost-effectiveness of adding NRT and bupropion SR to advice or counseling for smoking cessation is better than many other accepted health care interventions.

scholarly journals A cost-utility analysis of apalutamide for metastatic castration-sensitive prostate cancer

2021 ◽  
Vol 16 (3) ◽  
Author(s):  
Ambica Parmar ◽  
Narhari Timilshina ◽  
Urban Emmenegger ◽  
Martin Smoragiewicz ◽  
Beate Sander ◽  
...  
Keyword(s):  
Prostate Cancer ◽  
Cost Effectiveness ◽  
Incremental Cost ◽  
Therapeutic Option ◽  
Lifetime Cost ◽  
Cost Effective ◽  
Cost Utility ◽  
Base Case ◽  
Scenario Analyses ◽  
Life Years

Introduction: Earlier application of oral androgen receptor-axis-targeted therapies in patients with metastatic castration-sensitive prostate cancer (mCSPC) has established improvements in overall survival, as compared to androgen deprivation therapy (ADT) alone. Recently, the use of apalutamide plus ADT has demonstrated improvement in mCSPC-related mortality, vs. ADT alone, with an acceptable toxicity profile. However, the cost-effectiveness of this therapeutic option remains unknown. Methods: We used a state-transition model with probabilistic analysis to compare apalutamide + ADT, as compared to ADT alone for mCSPC patients over a time horizon of 20 years. Primary outcomes included expected life-years (LY), quality-adjusted life-years (QALY), lifetime cost (2020 Canadian dollars), and incremental cost-effectiveness ratio (ICER). Parameter and model uncertainties were assessed through scenario analyses. Health outcomes and cost were discounted at 1.5%, as per Canadian guidelines. Results: For the base-case analysis, expected LY for ADT and apalutamide plus ADT were 4.11 and 5.56, respectively (incremental LY 1.45). Expected QALYs were 3.51 for ADT and 4.84 for apalutamide plus ADT (incremental QALYs 1.33); expected lifetime cost was $36 582 and $255 633, respectively (incremental cost $219,051). ICER for apalutamide plus ADT, as compared to ADT alone, was $164 700/QALY. Through scenario analysis, price reductions >50% were required for apalutamide in combination with ADT to be considered cost-effective, at a cost-effectiveness threshold of $100 000/QALY. Conclusions: Apalutamide plus ADT is unlikely to be cost-effective from the Canadian healthcare perspective unless there are substantial reductions in the price of apalutamide treatment.

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