Clinical and economic outcomes associated with sequential treatment in patients with advanced renal cell carcinoma (aRCC).

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. 4566-4566
Author(s):  
Meredith M. Regan ◽  
David F. McDermott ◽  
Michael B. Atkins ◽  
Apoorva Ambavane ◽  
Shuo Yang ◽  
...  
Keyword(s):  
Kinase Inhibitors ◽  
Meta Analysis ◽  
Discrete Event ◽  
Cost Effective ◽  
Economic Outcomes ◽  
Sequential Treatment ◽  
New Drugs ◽  
Life Years ◽  
Combination Regimens ◽  
Treatment Sequences

4566 Background: Immuno-oncology therapies (IOs) and tyrosine kinase inhibitors (TKIs) are recommended for the treatment of aRCC. As new drugs and combination regimens emerge, there is interest in gaining a deeper understanding of optimal treatment sequencing. We aimed to assess clinical and economic outcomes associated with treatment sequences for untreated aRCC patients with IMDC intermediate/poor risk. Methods: A discrete event simulation model was developed to estimate the total costs and survival (in life-years; LYs) over patients’ lifetimes when receiving sequential treatment with nivolumab + ipilimumab (N+I), sunitinib (SUN), pazopanib (PAZ), or cabozantinib (CAB) as first-line (1L) treatment, and nivolumab (NIVO), axitinib (AXI), PAZ, CAB, or lenvatinib + everolimus (LEN+EVE) as second line (2L). Efficacy inputs were derived from the CheckMate 214 trial and a network meta-analysis based on available literature. Safety and cost data were obtained from literature and publicly available sources. Results: N+I initiating sequences were estimated to provide longer survival in mean LYs and lower mean costs/LY versus sequences with 1L TKIs (table). The estimates of incremental cost-effectiveness ratio (ICER) for N+I initiating sequences with 2L TKI monotherapy were well below the willingness-to-pay threshold of $50,000. Using 2L LEN+EVE, compared with 2L monotherapies, provided an incremental survival gain but at costs/LY close to $100,000. Conclusions: Use of 1L N+I followed by TKI monotherapy is estimated to provide longer survival while being more cost-effective versus TKIs followed by IOs or sequences cycling TKIs, mainly driven by a longer time to 2L treatment and longer treatment-free survival with N+I. Clinical trials with head-to-head comparisons of treatment sequences would be necessary to validate the findings of the study. [Table: see text]

scholarly journals Clinical and economic outcomes of treatment sequences for intermediate- to poor-risk advanced renal cell carcinoma

Immunotherapy ◽  
2020 ◽  
Vol 12 (1) ◽  
pp. 37-51 ◽  
Author(s):  
Apoorva Ambavane ◽  
Shuo Yang ◽  
Michael B Atkins ◽  
Sumati Rao ◽  
Anshul Shah ◽  
...  
Keyword(s):  
Renal Cell Carcinoma ◽  
Cell Carcinoma ◽  
Renal Cell ◽  
Kinase Inhibitor ◽  
Discrete Event ◽  
Cost Effective ◽  
Advanced Renal Cell Carcinoma ◽  
Poor Risk ◽  
Life Years ◽  
Treatment Sequences

Aim: To assess the cost–effectiveness of treatment sequences for patients with intermediate- to poor-risk advanced renal cell carcinoma. Patients & methods: A discrete event simulation model was developed to estimate patients’ lifetime costs and survival. Efficacy inputs were derived from the CheckMate 214 and CheckMate 025 studies and network meta-analyses. Safety and cost data were obtained from the published literature. Results: The estimated average quality-adjusted life-years (QALYs) gained was the highest on nivolumab + ipilimumab-initiated sequences (3.6–5.3 QALYs) versus tyrosine kinase inhibitor (TKI)-initiated sequences (2.1–3.7 QALYs). Incremental cost per QALY gained for nivolumab + ipilimumab-initiated sequences was below the willingness-to-pay threshold of $150,000 versus other sequences. Conclusion: Immuno-oncology combination therapy followed by TKIs is cost-effective versus TKI sequences followed by immuno-oncology or sequencing TKIs.

Post-stroke infections and preventive antibiotics in stroke: Update of clinical evidence

2018 ◽  
Vol 13 (9) ◽  
pp. 913-920 ◽  
Author(s):  
Jan-Dirk Vermeij ◽  
Willeke F Westendorp ◽  
Diederik van de Beek ◽  
Paul J Nederkoorn
Keyword(s):  
Antibiotic Therapy ◽  
Functional Outcome ◽  
Causal Relation ◽  
Meta Analysis ◽  
Cost Effective ◽  
Related Factors ◽  
Post Stroke ◽  
Life Years ◽  
Large Phase ◽  
Immunological Factors

This review provides an update of evidence on post-stroke infections and the use of preventive antibiotics in stroke. Infection is a common complication after stroke, affecting between 15% and 30% of the patients. The predictors for post-stroke infection can be divided into three categories: clinical factors, anatomical (stroke related) factors and immunological factors. The relation between the occurrence of a post-stroke infection and functional outcome remained subject of debate, but it seems likely that the occurrence of these infections has a causal relation with poor functional outcome and mortality. In the first meta-analysis on preventive antibiotic therapy, almost a decade ago, its beneficial effect on post-stroke infection rate was clear; however, the effect on functional outcome remained uncertain because included studies were small and heterogeneous. Afterwards, three large phase-3 RCTs were published and a Cochrane meta-analysis was performed. It has now become clear that, despite the finding that overall infections are reduced, preventive antibiotic therapy in the acute phase of stroke does neither improve functional outcome, nor decrease mortality rates. This does not yet mean that further research on preventive antibiotics in stroke is useless: the pathophysiology and etiology of post-stroke infections are unclear and the use of preventive antibiotics in specific subgroups of stroke patients could still be very effective. This is currently being studied. Besides, preventive antibiotic therapy might be cost-effective by increasing quality-adjusted life years. Thirdly, research for the upcoming years might put more emphasis on the effect of stroke on immunological alterations.

Is Banning Texturized Implants to Prevent Breast Implant-Associated Anaplastic Large Cell Lymphoma a Rational Decision? A Meta-Analysis and Cost-Effectiveness Study

2019 ◽  
Vol 40 (7) ◽  
pp. 721-731 ◽  
Author(s):  
Stefan V Danilla ◽  
Rocio P Jara ◽  
Felipe Miranda ◽  
Francisco Bencina ◽  
Marcela Aguirre ◽  
...  
Keyword(s):  
Sensitivity Analysis ◽  
Cost Effectiveness ◽  
Cell Lymphoma ◽  
Meta Analysis ◽  
Breast Implant ◽  
Cost Effective ◽  
Large Cell ◽  
Life Years ◽  
Large Cell Lymphoma ◽  
The Cost

Abstract Background Breast implant-associated anaplastic large cell lymphoma (BIA-ALCL) is an emergent disease that threatens patients with texturized breast implants. Major concerns about the safety of these implants are leading to global changes to restrict the utilization of this product. The principal alternative is to perform breast augmentation utilizing smooth implants, given the lack of association with BIA-ALCL. The implications and costs of this intervention are unknown. Objectives The authors of this study determined the cost-effectiveness of smooth implants compared with texturized implants for breast augmentation surgery. Methods A tree decision model was utilized to analyze the cost-effectiveness. Model input parameters were derived from published sources. The capsular contracture (CC) rate was calculated from a meta-analysis. Effectiveness measures were life years, avoided BIA-ALCL, avoided deaths, and avoided reoperations. A sensitivity analysis was performed to test the robustness of the model. Results For avoided BIA-ALCL, the incremental cost was $18,562,003 for smooth implants over texturized implants. The incremental cost-effectiveness ratio was negative for life years, and avoided death and avoided reoperations were negative. The sensitivity analysis revealed that to avoid 1 case of BIA-ALCL, the utilization of smooth implants would be cost-effective for a risk of developing BIA-ALCL equal to or greater than 1:196, and there is a probability of CC with smooth implants equal to or less than 0.096. Conclusions The utilization of smooth implants to prevent BIA-ALCL is not cost-effective. Banning texturized implants to prevent BIA-ALCL may involve additional consequences, which should be considered in light of higher CC rates and more reoperations associated with smooth implants than with texturized implants.

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 The Cost-Effectiveness of Lifestyle Interventions for Preventing Diabetes in a Health Resource-Limited Setting

2020 ◽  
Vol 2020 ◽  
pp. 1-8
Author(s):  
Jing Ma ◽  
Xu Wan ◽  
Bin Wu
Keyword(s):  
Cost Effectiveness ◽  
Lifestyle Intervention ◽  
Service Providers ◽  
Cost Effective ◽  
Dominant Strategy ◽  
Economic Outcomes ◽  
Medical Expenditure ◽  
Sensitivity Analyses ◽  
Life Years ◽  
The Cost

Aims. Type 2 diabetes mellitus (T2DM) is a health challenge in China, and the economic outcomes of lifestyle intervention are critically important for policymakers. This study estimates the lifetime economic outcomes of lifestyle intervention among the prediabetic population in the Chinese context. Methods. We developed a mathematical model to compare the cost-effectiveness of lifestyle intervention and no prevention in the prediabetic population. Efficacy and safety, medical expenditure, and utility data were derived from the literature, which was assigned to model variables for estimating the quality-adjusted life-years (QALYs) and costs as well as incremental cost-effectiveness ratios (ICERs). The analysis was conducted from the perspective of Chinese healthcare service providers. One-way and probabilistic sensitivity analyses were performed. Results. Compared with no prevention, lifestyle intervention averted 9.53% of T2DM, which translated into an additional 0.52 QALYs at a saved cost of $700 by substantially reducing the probabilities of macro- and microvascular diseases. This finding indicated that lifestyle intervention was a dominant strategy. The sensitivity analyses showed the model outputs were robust. Conclusions. Lifestyle intervention is a very cost-effective alternative for prediabetic subjects and worth implementing in the Chinese healthcare system to reduce the disease burden related to T2DM.

Health state utility values in schizophrenia: protocol for a systematic review and meta-analysis

2019 ◽  
Vol 22 (4) ◽  
pp. 142-144 ◽  
Author(s):  
David Aceituno ◽  
Mark Pennington ◽  
Barbara Iruretagoyena ◽  
Matthew A Prina ◽  
Paul McCrone
Keyword(s):  
Systematic Review ◽  
Task Force ◽  
Meta Analysis ◽  
Cost Effective ◽  
Health State Utility ◽  
Health State ◽  
Utility Values ◽  
Life Years ◽  
Health State Utility Values

IntroductionCost-effectiveness analyses that use quality-adjusted life-years (QALYs) allow comparing the value for money of interventions across different health problems. Health state utility values (HSUVs) are crucial to calculate QALYs. These are weights attached to a given health state reflecting preferences in health-related quality of life (HRQoL). In schizophrenia, there is extensive evidence about the consequences of this condition on HRQoL. Besides, several interventions have claimed to be cost-effective in terms of QALYs gained. Despite this evidence, a systematic review of HSUVs has not been conducted. Therefore, we aim to synthesise the evidence about HSUVs in schizophrenia.Methods and analysisWe will conduct a systematic review of the literature about HSUVs in people with schizophrenia following the Preferred Reporting Items for Systematic review and Meta-Analysis and the International Society for Pharmacoeconomics and Outcomes Research task force recommendations. The submissions records of eight electronic peer-reviewed databases and three health technology assessment (HTA) agencies will be searched. Quantitative synthesis will be carried out in comparable studies, using random-effects meta-analysis. Heterogeneity will be explored using meta-regression if more than 10 studies per covariate are found. A narrative synthesis and methodological quality of included studies will be also reported.DiscussionThis review will provide a synthesis of the HSUVs estimated for different states experienced by people with schizophrenia. This will inform analysts when calculating QALYs, using values in a more transparent and accountable manner. Finally, it will shed light on evidence gaps and limitations about this measure in mental health.PROSPERO registration numberCRD42019123582.

scholarly journals Cost-Effectiveness of Olaratumab in Combination with Doxorubicin for Patients with Soft Tissue Sarcoma in the United States

Sarcoma ◽  
2018 ◽  
Vol 2018 ◽  
pp. 1-14 ◽  
Author(s):  
Santiago Zuluaga-Sanchez ◽  
Lisa M. Hess ◽  
Sorrel E. Wolowacz ◽  
Yulia D’yachkova ◽  
Emma Hawe ◽  
...  
Keyword(s):  
United States ◽  
Cost Effectiveness ◽  
Soft Tissue ◽  
Soft Tissue Sarcoma ◽  
Meta Analysis ◽  
Single Agent ◽  
Cost Effective ◽  
Standard Of Care ◽  
The United States ◽  
Life Years

Background. Standard first-line treatments for advanced soft tissue sarcoma (STS) have changed little for 40 years, and outcomes have been poor. Recently, the United States (US) Food and Drug Administration conditionally approved olaratumab in combination with doxorubicin (Olara + Dox) based on a randomized phase II trial that reported a significant 11.8-month improvement in median survival versus single-agent doxorubicin (Dox). The present study investigated the cost-effectiveness of Olara + Dox compared with Dox and five other standard-of-care regimens from the US payer perspective. Methods. An economic model was constructed to estimate costs and outcomes over patients’ lifetimes from start of therapy. Progression-free and overall survival were based on survival analysis of patient-level data and a meta-analysis. Adverse-event rates were based on trials. Costs were from published sources. Results. Olara + Dox resulted in an estimated additional 1.27 life-years (LYs) compared with Dox, with an increase in total expected lifetime costs of $133,653. The incremental cost-effectiveness ratio (ICER) was estimated at $105,408 per LY gained; in a fully incremental analysis, all other regimens were dominated (higher costs and lower LYs or a higher ICER). Conclusion. Olara + Dox is cost-effective for STS treatment compared with Dox and other standard-of-care regimens at willingness-to-pay thresholds of $150,000 per LY and above.

PP146 Cost Effectiveness Of Aripiprazole Orally Disintegrating Tablets For The Treatment Of Schizophrenia In China

2020 ◽  
Vol 36 (S1) ◽  
pp. 17-17
Author(s):  
Ziyi Lin ◽  
Jianwei Xuan
Keyword(s):  
Sensitivity Analysis ◽  
Cost Effectiveness ◽  
Discrete Event ◽  
Economic Value ◽  
Cost Effective ◽  
Brand Names ◽  
Chinese Market ◽  
Local Data ◽  
Oral Tablet ◽  
Life Years

IntroductionAlthough antipsychotic medications have been a cornerstone in the treatment of schizophrenia for decades worldwide, the orally disintegrating tablet (ODT) formulation is a new concept in China. Only four brand names exist in the Chinese market, three of which were launched recently. Patients taking ODTs have a higher rate of medication adherence and consequently experience better treatment outcomes than patients taking the same medication in standard oral tablet (SOT) formulation. This study aimed to analyze the cost effectiveness in China of aripiprazole in ODT form, compared with the SOT forms of aripiprazole and olanzapine.MethodsA discrete-event simulation model was built to represent the one-year progression of schizophrenia. On entry into the model, 100,000 people for each treatment arm were labeled fully adherent, partially adherent, or non-adherent based on medication possession ratios, and then experienced events including relapse, adverse events, changing adherence levels, and treatment switching and quitting. Parameters for adherence rates, medical costs, and utility values were derived from the published literature. The switching pattern was acquired through interviews with fifty-seven Chinese psychiatrists.ResultsThe total annual costs per patient in the aripiprazole-ODT, aripiprazole-SOT, and olanzapine-SOT arms were CNY 9,817 (USD 1,388), CNY 15,278 (USD 2,160), and CNY 10,298 (USD 1,456), respectively. The annual quality-adjusted life-years (QALYs) gained per patient in the aripiprazole-ODT, aripiprazole-SOT, and olanzapine-SOT arms were 0.73, 0.71, and 0.72, respectively. According to the probabilistic sensitivity analysis, the probability of aripiprazole-ODT being cost effective was ninety-nine percent, when compared with aripiprazole-SOT and sixty-nine percent when compared with olanzapine-SOT.ConclusionsAripiprazole-ODT was associated with lower costs and higher gains in QALYs than either aripiprazole-SOT or olanzapine-SOT in patients with schizophrenia in China. While the sensitivity analysis confirmed the robustness of the result that aripiprazole-ODT was better economic value than aripiprazole-SOT, there is some uncertainty in the comparison between aripiprazole-ODT and olanzapine-SOT. The main limitation of this study is that some parameters were sourced from studies on Western populations because of a lack of data in China. Local data on the use of antipsychotics, especially adherence rates, is needed.

scholarly journals P424 Economic evaluation of vedolizumab SC for the treatment of moderate-to-severe ulcerative colitis in Canada

2020 ◽  
Vol 14 (Supplement_1) ◽  
pp. S387-S387
Author(s):  
A FISCHER ◽  
M Oppe ◽  
S Stypa ◽  
V Lukyanov ◽  
I Petrakis
Keyword(s):  
Ulcerative Colitis ◽  
Cost Effectiveness ◽  
Meta Analysis ◽  
Cost Effective ◽  
Future Research ◽  
Base Case ◽  
Public Healthcare ◽  
Payer Perspective ◽  
Life Years ◽  
Public Payer

Abstract Background Vedolizumab intravenous (IV), a gut selective humanised monoclonal antibody, is indicated for the management of moderately to severely active Ulcerative Colitis (UC) and has been shown in the only head-to-head clinical trial within UC to be superior to adalimumab (NCT02497469). Furthermore, the novel vedolizumab subcutaneous (SC) has recently been proven to be an effective treatment of UC (NCT02611830). The objective of this study is to estimate the comparative cost-effectiveness of vedolizumab IV with updated efficacy data, and vedolizumab SC for the first time. Both vedolizumab IV and SC have been compared with other publicly reimbursed biologics for the treatment of patients with moderate-to-severe UC from a Canadian public healthcare payer perspective. Methods A hybrid decision tree/Markov model was developed to simulate the clinical course of UC, translating the disease course into costs and quality-adjusted life-years (QALYs). Comparative efficacy of vedolizumab SC, vedolizumab IV, adalimumab, infliximab IV, and golimumab were sourced from a network meta-analysis. Drug and disease management costs (2019 $CAD) were sourced from Ontario public payer schedules of benefits. Utilities were sourced from the literature. Clinical and economic outcomes were projected over a lifetime and discounted at 1.5% annually. Results Within a mixed bio-naïve/experienced population, vedolizumab SC resulted in slightly more QALYs than vedolizumab IV and dominated adalimumab (Table 1); vedolizumab SC yielded an incremental cost-effectiveness ratio (ICER) of $CAD 6,727 per QALY and $CAD 52,673 per QALY relative to golimumab and to infliximab (for which the price of a biosimilar was used), respectively. Further scenario analysis within bio-naïve populations supported the robustness of the base-case results, demonstrating that vedolizumab SC or vedolizumab IV were either dominant or cost-effective across all scenarios. Conclusion Our analysis suggests that vedolizumab SC and vedolizumab IV are a cost-effective therapeutic alternative relative to other biologics for moderate-to-severe UC in Canada. Future research will expand the analysis across all biologic comparators as they are used in the real world.

scholarly journals The impact of pharmaceutical innovation on the burden of disease in Ireland, 2000–2015

2020 ◽  
Vol 42 (4) ◽  
pp. 816-827 ◽  
Author(s):  
Frank R Lichtenberg
Keyword(s):  
Burden Of Disease ◽  
Fixed Effects ◽  
Pharmaceutical Expenditure ◽  
Cost Effective ◽  
Pharmaceutical Innovation ◽  
New Drugs ◽  
Behavioral Risk ◽  
Life Years ◽  
New Chemical Entities ◽  
The Impact

Abstract Background We perform an econometric assessment of the impact that pharmaceutical innovation had on the burden of disease in Ireland. Methods We use a difference-in-differences (or two-way fixed effects) research design: we investigate whether diseases for which more new drugs were launched had larger subsequent reductions in mortality. This design controls for the effects of general economic and societal factors (e.g. income, education, and behavioral risk factors), to the extent that those effects are similar across diseases. Results New Chemical Entities launched during 1983–1997 are estimated to have reduced the total number of disability-adjusted life-years (DALYs) lost in 2015 by about 234 600. Conclusions Pharmaceutical expenditure per DALY gained in 2015 from drugs launched during 1983–1997 was €1137, which indicates that the new drugs launched during 1983–1997 were very cost–effective, overall.

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