scholarly journals Forecasting US ivacaftor outcomes and cost in cystic fibrosis patients with the G551D mutation

2016 ◽  
Vol 47 (6) ◽  
pp. 1697-1705 ◽  
Author(s):  
Piyameth Dilokthornsakul ◽  
Ryan N. Hansen ◽  
Jonathan D. Campbell
Keyword(s):  
Cystic Fibrosis ◽  
Usual Care ◽  
Budget Impact ◽  
Lifetime Cost ◽  
Credible Interval ◽  
Genetic Mutation ◽  
Forced Expiratory Volume ◽  
Health States ◽  
Payer Perspective ◽  
Life Years

Ivacaftor, a breakthrough treatment for cystic fibrosis (CF) patients with the G551D genetic mutation, lacks long-term clinical and cost projections. This study forecasted outcomes and cost by comparing ivacaftor plus usual care versus usual care alone.A lifetime Markov model was conducted from a US payer perspective. The model consisted of five health states: 1) forced expiratory volume in 1 s (FEV1) % pred ≥70%, 2) 40%≤ FEV1 % pred <70%, 3) FEV1 % pred <40%, 4) lung transplantation and 5) death. All inputs were extracted from published literature. Budget impact was also estimated. We estimated ivacaftor's improvement in outcomes compared with a non-CF referent population.Ivacaftor was associated with 18.25 (95% credible interval (CrI) 13.71–22.20) additional life-years and 15.03 (95% CrI 11.13–18.73) additional quality-adjusted life-years (QALYs). Ivacaftor was associated with improvements in survival and QALYs equivalent to 68% and 56%, respectively, for the survival and QALY gaps between CF usual care and their non-CF peers. The incremental lifetime cost was $3 374 584. The budget impact was $0.087 per member per month.Ivacaftor increased life-years and QALYs in CF patients with the G551D mutation, and moved morbidity and mortality closer to that of their non-CF peers. Ivacaftor costs much more than usual care, but comes at a relatively limited budget impact.

scholarly journals Cost-effectiveness analysis of a strategy to delay progression to dialysis and death among chronic kidney disease patients in Lima, Peru

2021 ◽  
Vol 19 (1) ◽  
Author(s):  
E. M. Saldarriaga ◽  
J. Bravo-Zúñiga ◽  
Y. Hurtado-Roca ◽  
V. Suarez
Keyword(s):  
Chronic Kidney Disease ◽  
Cost Effectiveness ◽  
Kidney Disease ◽  
Usual Care ◽  
Lifetime Cost ◽  
Standard Of Care ◽  
Health Consequences ◽  
Lack Of Information ◽  
Payer Perspective ◽  
Life Years

Abstract Background The Renal Health Program (RHP) was implemented in 2013 as a secondary prevention strategy to reduce the incidence of patients initiating dialysis and overall mortality. A previous study found that adherent patients have 58% protection against progression to dialysis compared to non-adherent. The main objective of the study was to estimate the lifetime economic and health consequences of the RHP intervention to determine its cost-effectiveness in comparison with usual care. Methods We use a Markov model of three health stages to simulate disease progression among chronic kidney disease patients in Lima, Peru. The simulation time-horizon was 30 years to capture the lifetime cost and health consequences comparing the RHP to usual care. Costs were estimated from the payer perspective using institutional data. Health outcomes included years lived free of dialysis (YL) and quality adjusted life years (QALY). We conducted a probabilistic sensitivity analysis (PSA) to assess the robustness of our estimates against parameter uncertainty. Results We found that the RHP was dominant—cost-saving and more effective—compared to usual care. The RHP was 783USD cheaper than the standard of care and created 0.04 additional QALYs, per person. The Incremental Cost-Effectiveness Ratio (ICER) showed a cost per QALY gained of $21,660USD. In the PSA the RHP was dominant in 996 out of 1000 evaluated scenarios. Conclusions The RHP was cheaper than the standard of care and more effective due to a reduction in the incidence of patients progressing to dialysis, which is a very expensive treatment and many times inaccessible. We aim these results to help in the decision-making process of scaling-up and investment of similar strategies in Peru. Our results help to increase the evidence in Latin America where there is a lack of information in the long-term consequences of clinical-management-based prevention strategies for CKD patients.

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 Should the Government Be Allowed to Take Control over Your Car as Part of a Disaster Management Plan?

2020 ◽  
Vol 17 (21) ◽  
pp. 7780
Author(s):  
Yipeng Lv ◽  
Zafar Zafari ◽  
Boshen Jiao ◽  
Casey Chun ◽  
Lulu Zhang ◽  
...  
Keyword(s):  
Markov Model ◽  
Incremental Cost ◽  
Autonomous Vehicles ◽  
Credible Interval ◽  
The United States ◽  
Status Quo ◽  
Health States ◽  
Life Years ◽  
Hypothetical Scenario ◽  
The Government

Introduction: With the Safety Ensuring Lives Future Deployment and Research in Vehicle Evolution (SELF DRIVE) Act in the United States, there is a growing interest in autonomous vehicles (AVs). One avenue of innovation would be to use them to mobilize and coordinate response efforts during natural disasters. This study uses an earthquake response in an urban, developed setting as a hypothetical example case study. In this hypothetical scenario, private AVs would be mobilized to help rescue victims from collapsed structures. Methods: A Markov model compared an intervention arm with AVs to a status quo arm using a hypothetical cohort of American earthquake victims. The three possible health states were trapped but alive, rescued and alive, and dead. The cycle length of the Markov model was 6 h. Results: The cost of deploying AVs was $90,139 relative to $87,869 in status quo arm. Using AVs produced an incremental cost of $2269 (95% credible interval (CI) = $−12,985–$8959). Victims have 7.33 quality-adjusted life years (QALYs) in the intervention arm compared to 7.20 QALYs in the status quo arm, resulting in an incremental gain of 0.13 (95% CI = −0.73–2.19) QALYs. The incremental cost-effectiveness ratio (ICER) was $16,960/QALY gained (95% CI = cost-saving–$69,065/QALY). Discussion: The mobilization of private AVs in the setting of an earthquake has the potential to save money and reduce the loss of life. AVs may advance emergency management competencies.

scholarly journals Predicting Cost-Effectiveness of Generic Versus Brand Dabigatran Using Pharmacometric Estimates among Patients with Atrial Fibrillation in the United States

2019 ◽  
Author(s):  
Ching-Yu Wang ◽  
Phuong N. Pham ◽  
Sarah Kim ◽  
Karthik Lingineni ◽  
Stephan Schmidt ◽  
...  
Keyword(s):  
Atrial Fibrillation ◽  
Cost Effectiveness ◽  
Lifetime Cost ◽  
Systemic Exposure ◽  
Cost Effective ◽  
The United States ◽  
Payer Perspective ◽  
Life Years ◽  
Generic Medications ◽  
Newer Anticoagulants

ABSTRACTGeneric entry of newer anticoagulants is expected to decrease the costs of atrial fibrillation management. However, when making switches between brand and generic medications, bioequivalence failures are possible. The objectives of this study were to predict and compare the lifetime cost-effectiveness of brand dabigatran with hypothetical future generics. Markov micro-simulations were modified to predict the lifetime costs and quality-adjusted life years of patients on either brand or generic dabigatran from a U.S. private payer perspective. Event rates for generics were predicted using previously developed pharmacokinetic-pharmacodynamic models. The analyses showed that generic dabigatran with lower-than-brand systemic exposure was dominant. Meanwhile, generic dabigatran with extremely high systemic exposure was not cost-effective compared to the brand reference. Cost-effectiveness of generic medications cannot always be assumed as shown in this example. Combined use of pharmacometric and pharmacoeconomic models can assist in decision making between brand and generic pharmacotherapies.

scholarly journals Cost-Effectiveness Analysis of A Program to Delay Progression to Dialysis and Death Among CKD Patients in Lima, Peru

2020 ◽  
Author(s):  
Enrique M Saldarriaga ◽  
Jessica Bravo-Zúñiga ◽  
Yamilee Hurtado-Roca ◽  
Víctor Suarez
Keyword(s):  
Cost Effectiveness ◽  
Cost Saving ◽  
Usual Care ◽  
Economic Value ◽  
Management Intervention ◽  
Lack Of Information ◽  
Case Management Intervention ◽  
Payer Perspective ◽  
Life Years ◽  
The Cost

Abstract Background. The Renal Health Program (RHP) was implemented in 2013 as a secondary prevention strategy to reduce the incidence of patients starting dialysis and overall mortality. A previous study found that adhered patients have 58% protection against progression to dialysis compared to non-adhered. Objective. We aim to estimate the lifetime economic and health consequences of the RHP intervention to determine its cost-effectiveness in comparison with usual care. Methods. We use a Markov model of three health stages to simulate for 30 years the cost associated with RHP and usual care, as well as years lived free of dialysis (YL) and Quality Adjusted Life Years (QALY). Costs were estimated from the payer perspective. We conduct a Probabilistic Sensitivity Analysis (PSA) to assess the robustness of our estimates. Results. We found that the RHP is a cost-saving alternative compared to usual care, with a per person difference of $-782.73in costs and 0.04 in QALYs. The Incremental Cost-Effectiveness Ratio (ICER) per QALY is $-21,660. From a PSA, RHP holds cost-saving under 999 out of 1,000 evaluated scenarios. Discussion. Our results show the lifetime economic value of a case-management intervention for CKD patients to delay its progression to dialysis. The RHP is cost-saving compared to usual care, with a negative ICER per QALY robust to different scenarios. We aim these results to help in the decision-making process of scaling-up and investment of similar strategies in Peru. Our results help to increase the evidence in Latin America where there is a lack of information in the long-term consequences of prevention and early referral strategies in CKD patients.

Cost-effectiveness of denosumab (D) versus zoledronic acid (Z) for skeletal-related event (SRE) reduction in bone-metastatic prostate cancer (mPC) in the United Kingdom.

2012 ◽  
Vol 30 (15_suppl) ◽  
pp. e15189-e15189
Author(s):  
Marc Botteman ◽  
John A. Carter ◽  
Peter Fishman ◽  
David Chandiwana ◽  
Manjinder Bains ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Phase Iii ◽  
List Price ◽  
Base Case ◽  
Health States ◽  
Serious Adverse Events ◽  
Payer Perspective ◽  
Life Years ◽  
Lifetime Analysis ◽  
Skeletal Related Event

e15189 Background: D reduced SREs vs Z in a phase III trial, but no significant differences in overall survival, disease progression, or serious adverse events were reported. The cost-effectiveness of D vs Z in mPC was assessed from a UK payer perspective. Methods: A literature-based Markov model estimated quality-adjusted life-years (QALYs), number and costs of SREs, and drug/administration costs for mPC pts receiving D or Z for 27 mo (trial timeframe) and 60 mo (extended lifetime analysis). Current list prices/injection (£309.86 for D; £174.14 for Z) were used in the base case. As the model’s analytical horizon starts June 2012 and extends beyond generic Z availability in May 2013, a generic Z price (53% < list price, per data for other now-generic bisphosphonates) was used beyond May 2013 in scenario analysis. In other analyses, discounted prices (–30%) were used for both D and Z to assess the effect of price reductions on cost/QALY. Clinical inputs were selected to reproduce trial outcomes. QALYs were estimated by assigning utility weights to time spent in health states (no SRE; SRE; post-SRE; death). SRE costs were £250 to ≥ £6,000/event. Results: Although D may result in fewer SREs, higher QALYs, and lower SRE-related costs (Table), higher acquisition cost of D results in substantially higher costs and may be cost-ineffective in every pricing scenario. Similar results were found for the 27-mo analysis. Conclusions: The incremental cost/QALY gained with D vs Z ranged from £50,000 to £215,000 (> traditional £30,000 threshold), thus raising important questions about D’s value in mPC. [Table: see text]

Cost-effectiveness of novel antiandrogens (AAs) for treatment of nonmetastatic castrate-resistant prostate cancer (nmCRPC).

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. 5583-5583
Author(s):  
Irbaz Bin Riaz ◽  
Abdulaali Almutairi ◽  
Daenielle K. Lang ◽  
Noureen Asghar ◽  
Anum Riaz ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Lifetime Cost ◽  
Cost Effective ◽  
Cost Utility ◽  
Sensitivity Analyses ◽  
Base Case ◽  
Health States ◽  
Support Unit ◽  
Medication Costs ◽  
Life Years

5583 Background: FDA has approved three novel AAs [Apalutamide(A), Darolutamide(D) and Enzalutamide(E)] in combination with Androgen deprivation therapy ( ADT) for treatment of (nmCRPC) patients (pts). We report the cost-effectiveness of these drugs from the US perspective to help facilitate the choice of these agents for clinical practice. Methods: A life time Markov state-transition model was constructed with three health states (Metastasis-Free Survival[MFS], Metastatic disease, and Death) to compare cost-effectiveness of AA therapies for treatment of nmCRPC based on US healthcare payer perspective. A network meta-analysis of MFS and OS was conducted due to the lack of head to head trials. An approximation of the original individual-level patient time-to-event data were derived from digitized Kaplan-Meier curves for OS and MFS. Weibull distributions was selected as the best fitted model fitted and extrapolated as per the NICE decision support unit recommendations. Medication costs were based on wholesale acquisition cost. Adverse event (AE) grades 3/4 management costs were incorporated in the model. Discount rate of 3% per year was applied to costs and effects. Life years (LYs) and quality adjusted life years (QALYs) for each treatment as well as the incremental cost effectiveness (ICER) and cost utility (ICUR) ratios were estimated. Base case analyses (BCA) and probabilistic sensitivity analyses (PSA) were estimated. Results: The table summarizes the results form BCA analyses. A+ADT offers best gain in LYs (8.37yrs) and QALYs (5.30 yrs) but at higher cost. Conclusions: Apalutamide was associated with gains in LYs and QALYs traded off with higher lifetime cost relative to other AA alternatives. ADT was associated with lower gains in LYs and QALYs traded off with lower lifetime cost relative to other alternatives. Based on a $150,000/QALY threshold pay off, A+ADT is likely more cost effective compared to E+ADT or ADT alone; while E+ ADT may be more cost effective compared to D+ ADT. [Table: see text]

scholarly journals IL-8 correlates with reduced baseline femoral neck bone mineral density in adults with cystic fibrosis: a single center retrospective study

2021 ◽  
Vol 11 (1) ◽  
Author(s):  
Grace Y. Lam ◽  
Sameer Desai ◽  
Joey Fu ◽  
Xun Yang Hu ◽  
Jiah Jang ◽  
...  
Keyword(s):  
Bone Mineral Density ◽  
Cystic Fibrosis ◽  
Femoral Neck ◽  
Bone Mineral ◽  
Forced Expiratory Volume ◽  
Inflammatory Biomarkers ◽  
Mineral Density ◽  
Baseline Serum ◽  
Average Percentage ◽  
System Disease

AbstractCystic fibrosis (CF) is a multi-system disease that is characterized by lung disease due to recurrent airway infection and inflammation. Endocrine complications, such as CF bone disease (CFBD), are increasingly identified as patients are living longer. The cause of CFBD is multifactorial with chronic systemic inflammation theorized to be a contributing factor. Thus, we attempted to identify inflammatory biomarkers that are associated with CFBD. We conducted a retrospective observational study of 56 adult patients with CF with an average percentage predictive forced expiratory volume in one second (ppFEV1) of 73.7% (standard deviation: 30.0) who underwent baseline serum analysis for osteoprotegerin (OPG) and pro-inflammatory biomarkers (IL-1β, IL-6, IL-8 and TNF-α), and had repeated dual-energy x-ray absorptiometry (DXA) scans separated by at least 2 years to examine correlations between serum biomarkers and bone mineral density (BMD) measurements. Univariate linear regression model analysis demonstrated that serum IL-1β and IL-8, but not other pro-inflammatory markers, were negatively correlated with baseline BMD results. However, after accounting for confounding variables, only the relationship between IL-8 and left femoral neck BMD remained statistically significant. Additionally, IL-8 level was associated with BMD decline over time. These results suggest that IL-8 might play a unique role in the pathophysiology of CFBD relative to other pro-inflammatory cytokines but further study is warranted before firm conclusions can be made.

scholarly journals Valuation of the EQ-5D-3L in Russia

2021 ◽  
Author(s):  
Vitaly Omelyanovskiy ◽  
Nuriya Musina ◽  
Svetlana Ratushnyak ◽  
Tatiana Bezdenezhnykh ◽  
Vlada Fediaeva ◽  
...  
Keyword(s):  
Health Problems ◽  
Cost Utility ◽  
Economic Evaluations ◽  
Computer Assisted ◽  
Health State ◽  
Primary Analysis ◽  
Health States ◽  
Value Set ◽  
Model Combining ◽  
Life Years

Abstract Purpose The most widely used generic questionnaire to estimate the quality of life for yielding quality-adjusted life years in economic evaluations is EQ-5D. Country-specific population value sets are required to use EQ-5D in economic evaluations. The aim of this study was to establish an EQ-5D-3L value set for Russia. Methods A representative sample aged 18+ years was recruited from the Russia`s general population. Computer-assisted face–to–face interviews were conducted based on the standardized valuation protocol using EQ-Portable Valuation Technology. Population preferences were elicited utilizing both composite time trade-off (cTTO) and discrete choice experiment (DCE) techniques. To estimate the value set, a hybrid regression model combining cTTO and DCE data was used. Results A total of 300 respondents who successfully completed the interview were included in the primary analysis. 120 (40.0%) respondents reported no health problems of any dimension, and 56 (18.7%) reported moderate health problems in one dimension of the EQ‐5D‐3L. Median self-rated health using EQ‐VAS was 80 with IQR 70–90. Comparing cTTO and DCE-predicted values for 243 health states resulted in a similar pattern. This supports the use of hybrid models. The predicted value based on the preferred model for the worst health state “33333” was −0.503. Mobility dimension had the most significant impact on the utility decrement, and anxiety/depression had the lowest decrement. Conclusion Determining a Russian national value set may be considered the first step towards promoting cost-utility analysis use to increase comparability among studies and improve the transferability of healthcare decision-making in Russia.

A Systematic Review of Cost-Effectiveness Analyses Alongside Randomised Controlled Trials of Acupuncture

2012 ◽  
Vol 30 (4) ◽  
pp. 273-285 ◽  
Author(s):  
Song-Yi Kim ◽  
Hyangsook Lee ◽  
Younbyoung Chae ◽  
Hi-Joon Park ◽  
Hyejung Lee
Keyword(s):  
Cost Effectiveness ◽  
Usual Care ◽  
Randomised Controlled Trials ◽  
Risk Of Bias ◽  
Economic Evaluations ◽  
Controlled Trials ◽  
German Studies ◽  
Life Years ◽  
Randomised Controlled ◽  
The Cost

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.

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