Health Outcomes and Costs of Rituximab in Combination with Cyclophosphamide, Vincristine and Prednisolone in the Treatment of Patients with Advanced Follicular Lymphoma in Portugal.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 2481-2481
Author(s):  
Paula Braga ◽  
Susana Carvalho ◽  
Marília Gomes ◽  
Lurdes Guerra ◽  
Paulo Lúcio ◽  
...  

Abstract Abstract 2481 Poster Board II-458 Objective: With the pressure on healthcare budgets, it has become increasingly important for healthcare decision makers to consider the value for money of the treatments they reimburse. The objective of this analysis was to evaluate the long term outcomes and costs of rituximab in combination with cyclophosphamide/vincristine/prednisolone chemotherapy regimen (R-CVP) versus CVP alone, in previously untreated patients with indolent Non-Hodgkin Lymphoma (NHL) from the Portuguese National Health System (NHS) perspective. Methods: Cost-effectiveness (Life Years Gained - LYG) and cost-utility analysis (Quality Adjusted Life years – QALYs) were performed for a time horizon of 10 years, according to a Markov economic model with three health states (“progression free survival”, “progression” and “death”) and monthly cycles for a population of previously untreated patients with indolent NHL. Data from a phase III clinical trial (Marcus 2007) was used and expanded to include unpublished 53-month median follow-up data. Survival after first-line therapy was estimated from the Scotland and Newcastle Lymphoma Group registry data and utilities were derived from a study in the UK performed in patients with follicular lymphoma. Resource consumption was estimated by a Portuguese expert panel (Delbecq Panel). Costs were calculated from the Portuguese NHS perspective through official data with prices updated to 2008. Only direct medical costs were considered. Costs and clinical outcomes were discounted at 5% per annum. Deterministic and probabilistic sensitivity analysis were performed around assumptions on the time horizon, costs, utilities and excess mortality rate due to progression applied in the base-case analysis. Results: The 10-year base-case analysis showed a lower total cost per patient with CVP alone (€ 85,838) in comparison with R-CVP (€ 87,774). Life expectancy and quality-adjusted life expectancy per patient were higher with R-CVP (6.361 and 4.166, respectively) than with CVP alone (5.557 and 3.438, respectively), representing increases of 0.804 in LYG and 0.728 (8.7 months) in QALYs gained. The incremental cost per LYG was € 2,407 and the incremental cost per QALY gained was € 2,661. The probabilistic sensitivity analysis confirmed the robustness of the base-case analysis results. Conclusions: This study demonstrates that the combination R-CVP in previously untreated non-Hodgkin lymphoma patients improves life expectancy and is a cost-effective alternative to CVP in Portugal. Disclosures: Braga: Roche Farmacêutica Química, Lda: Employment. Pereira:Roche Farmacêutica Química, Lda.: Employment.

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 4727-4727
Author(s):  
Ahmed Aljabri ◽  
Mahdi Gharaibeh ◽  
Yvonne Huckleberry ◽  
Hussam Kutbi ◽  
Yuval Raz ◽  
...  

Abstract Introduction. The relative efficacy and safety of fondaparinux and argatroban in the management of suspected heparin-induced thrombocytopenia (HIT) have been documented. We conducted a cost-effectiveness analysis of both agents in terms of cost and adverse events averted. Methods. We developed a two-armed decision tree model to simulate a cohort of patients with suspected or confirmed HIT managed with argatroban or fondaparinux, using published efficacy data and probabilities of developing HIT-related VTE or major bleeding. The primary base case analysis considered our institutional cost while the secondary analysis used the Average Wholesale Price. Probabilistic sensitivity analysis (PSA) was performed to confirm findings and one-way sensitivity analysis to evaluate additional scenarios. Results. In the primary base case analysis, fondaparinux dominated argatroban treatment as it was less expensive in managing suspected HIT ($154 vs. $2,064) and more effective in terms of adverse events averted (0.998878 vs. 0.995739). This was confirmed in PSA in terms of both cost ($154 vs. $1,999) and adverse events averted (0.9988711 vs. 0.9957212). Sensitivity analysis showed that the total costs of argatroban would never be less than fondaparinux based on our assumptions. In the secondary analysis, fondaparinux was less expensive ($362 vs. $3722) and more effective in terms of adverse event averted (0.998878 vs. 0.995739). The PSA confirmed these findings both in terms of cost ($343 vs. $3477) adverse events averted. (0.9988711 vs. 0.9957212). Conclusions. The cost savings from a once-daily SC fondaparinux injection with limited laboratory monitoring are more advantageous than IV argatroban infusion that requires continuous titration. Disclosures Off Label Use: Fondaparinux is used as "off-label" for the management of patients with suspected or confirmed HIT.


2001 ◽  
Vol 11 (5) ◽  
pp. 1-8 ◽  
Author(s):  
James McInerney ◽  
David A. Gould ◽  
John D. Birkmeyer ◽  
Robert E. Harbaugh

Object Asymptomatic intracranial arteriovenous malformations (AVMs) represent a clinically challenging problem because of the complex decision making that must be undertaken prior to beginning any type of treatment. In addition, the relative infrequency of these lesions means that there is relatively little experience reported in the literature. The authors use a decision-analysis technique to model the considerations that go into determining the treatment of these lesions in an effort to quantify the various risks and overall benefits conferred by the following three treatment strategies: observation/natural history, microsurgery, and stereotactic radiosurgery. Methods The authors conducted a thorough literature search to elucidate the risks and outcomes associated with each treatment option. These values were used to build and run a comprehensive Markov model to determine a base-case analysis. All of the input variables were also subjected to sensitivity analysis to identify the most influential input variables and the crossover points in which favored strategies changed. The base-case analysis suggested that microsurgery was the favored treatment option because this hypothetical cohort accumulated 21.53 quality-adjusted life years (QALYs) over the course of the model compared with the 16.97 QALYs and 16.40 QALYs for stereotctic radiosurgery and observation, respectively. Sensitivity analysis demonstrated that overall major neurological morbidity and mortality were the most influential input variables both perioperatively and during the radiosurgical “latent” period (that is, up to 2 years posttreatment). The maximum acceptable perioperative combined major neurological morbidity and mortality rate was 6.8%. The latent period combined major neurological morbidity and mortality would need to be 0.7% to make radiosurgery favorable in this analysis. Conclusions Results of this decision analysis model suggest that microsurgery in the hands of experienced cerebrovascular surgeons, who can expect a less than 6.8% combined rate of major neurological morbidity and mortality, offers patients a greater overall quality of life over time.


Author(s):  
Matthew D. Bucknor ◽  
Frandics P. Chan ◽  
Jessica Y. Matuoka ◽  
Patti K. Curl ◽  
James G. Kahn

Abstract Objective The aim of this study was to determine if magnetic resonance-guided focused ultrasound (MRgFUS) is cost-effective compared with medication, for refractory pain from bone metastases in the United States. Methods We constructed a Markov state transition model using TreeAge Pro software (TreeAge Software, Inc., Williamstown, MA, USA) to model costs, outcomes, and the cost-effectiveness of a treatment strategy using MRgFUS for palliative treatment of painful bone metastases compared with a Medication Only strategy (Figure 1). Model transition state probabilities, costs (in 2018 US$), and effectiveness data (quality-adjusted life-years [QALYs]) were derived from available literature, local expert opinion, and reimbursement patterns at two U.S. tertiary academic medical centers actively performing MRgFUS. Costs and QALYs, discounted at three percent per year, were accumulated each month over a 24-month time horizon. One-way and probabilistic sensitivity analyses were performed. Results In the base-case analysis, the MRgFUS treatment strategy costs an additional $11,863 over the 2-year time horizon to accumulate additional 0.22 QALYs, equal to a $54,160/QALY ICER, thus making MRgFUS the preferred strategy. One-way sensitivity analyses demonstrate that for the base-case analysis, the crossover point at which Medication Only would instead become the preferred strategy is $23,341 per treatment. Probabilistic sensitivity analyses demonstrate that 67 percent of model iterations supported the conclusion of the base case. Conclusions Our model demonstrates that MRgFUS is cost-effective compared with Medication Only for palliation of painful bone metastases for patients with medically refractory metastatic bone pain across a range of sensitivity analyses.


2022 ◽  
Vol 7 (1) ◽  
pp. 238146832110699
Author(s):  
Vasileios Kontogiannis ◽  
Diarmuid Coughlan ◽  
Mehdi Javanbakht ◽  
Patience Kunonga ◽  
Fiona Beyer ◽  
...  

Background. Consensus on standardized active surveillance or follow-up care by clinicians is lacking leading to considerable variation in practice across countries. An important structural modelling consideration is that self-examination by patients and their partners can detect melanoma recurrence outside of active surveillance regimes. Objectives. To identify candidate melanoma surveillance strategies for American Joint Committee on Cancer (AJCC) stage I disease and compare them with the current recommended practice in a cost-utility analysis framework. Methods. In consultation with UK clinical experts, a microsimulation model was built in TreeAge Pro 2019 R1.0 (Williamstown, MA, USA) to evaluate surveillance strategies for AJCC stage IA and IB melanoma patients separately. The model incorporated patient behaviors such as self-detection and emergency visits to examine suspicious lesions. A National Health Service (NHS) perspective was taken. Model input parameters were taken from the literature and where data were not available, local expert opinion was sought. Probabilistic sensitivity analysis, one-way sensitivity analysis on pertinent parameters and value of information was performed. Results. In the base-case probabilistic sensitivity analysis, less intensive surveillance strategies for AJCC stage IA and IB had lower total lifetime costs than the current National Institute for Health and Care Excellence (NICE) recommended strategy with similar effectiveness in terms of quality-adjusted life years and thereby likely to be cost-effective. Many strategies had similar effectiveness due to the relatively low chance of recurrence and the high rate of self-detection. Sensitivity and scenario analyses did not change these findings. Conclusions. Our model findings suggest that less resource intensive surveillance may be cost-effective compared with the current NICE surveillance guidelines. However, to advocate convincingly for changes, better evidence is required.


2020 ◽  
Vol 14 (Supplement_1) ◽  
pp. S605-S605
Author(s):  
A De Buck Van Overstraeten ◽  
M Brar ◽  
S Khorasani ◽  
F Dossa ◽  
P Myrelid

Abstract Background Ileorectal anastomosis (IRA) in patients with ulcerative colitis (UC) results in decreased postoperative morbidity and better functional outcome but leads to increased risk for rectal cancer compared with ileal pouch-anal anastomosis (IPAA). This study aims to compare IRA with IPAA in UC, using decision analysis. Methods A Markov simulation model was designed to simulate clinical events of IRA and IPAA over a time horizon of 40 years with time cycles of 1 year (Figure 1). The base case was a 35-year-old patient with ulcerative colitis and relatively preserved rectum. Probabilities and utilities, required to populate the model, were derived from observational studies, identified after a systematic literature search using MEDLINE. Primary outcomes were life years (LY) and quality-adjusted life years (QALY). Deterministic sensitivity analyses were performed to assess the impact of changing variables on the preferred treatment. Monte Carlo probabilistic sensitivity analysis, using 10 000 samples, was performed to account for uncertainty of variables. Prevalence of rectal cancer, IPAA and IRA failure and the stoma rate were calculated at the end of the time horizon, using markov cohort analysis. Results The model resulted in lower LY (36.22 vs. 37.02) and higher QALYs (33.42 vs. 31.57) for IRA. The results of the Monte Carlo probabilistic sensitivity analysis demonstrated that IRA was the preferred treatment option in 63% of the samples, accounting for a clinical significant margin of 0.25 QALY (Figure 2). The model was also sensitive to the utility of IRA, IPAA and end-ileostomy. A higher proportion of IRA patients will develop rectal cancer (7.6% vs. 3.2%) and 43.5% of all IRA patients will end with an ileostomy as opposed to 23.0% of all IPAA patients. The study was limited by characteristics inherent to modeling studies, including assumptions necessary to build the model, data input based on best available but often limited evidence and unavoidable extra- and interpolation of data. Conclusion IRA was the preferred treatment option when quality-adjusted life years was the outcome, with higher life years for IPAA. This model highlights that both surgical strategies are useful in ulcerative colitis patients with relatively spared rectum.


2020 ◽  
Vol 14 (8) ◽  
Author(s):  
Anna Parackal ◽  
Jean-Eric Tarride ◽  
Feng Xie ◽  
Gord Blackhouse ◽  
Jennifer Hoogenes ◽  
...  

Introduction: Recent health technology assessments (HTAs) of robot-assisted radical prostatectomy (RARP) in Ontario and Alberta, Canada, resulted in opposite recommendations, calling into question whether benefits of RARP offset the upfront investment. Therefore, the study objectives were to conduct a cost-utility analysis from a Canadian public payer perspective to determine the cost-effectiveness of RARP. Methods: Using a 10-year time horizon, a five-state Markov model was developed to compare RARP to open radical prostatectomy (ORP). Clinical parameters were derived from Canadian observational studies and a recently published systematic review. Costs, resource utilization, and utility values from recent Canadian sources were used to populate the model. Results were presented in terms of increment costs per quality-adjusted life years (QALYs) gained. A probabilistic analysis was conducted, and uncertainty was represented using cost-effectiveness acceptability curves (CEACs). One-way sensitivity analyses were also conducted. Future costs and QALYs were discounted at 1.5%. Results: Total cost of RARP and ORP were $47 033 and $45 332, respectively. Total estimated QALYs were 7.2047 and 7.1385 for RARP and ORP, respectively. The estimated incremental cost-utility ratio (ICUR) was $25 704 in the base-case analysis. At a willingness-to-pay threshold of $50 000 and $100 000 per QALY gained, the probability of RARP being cost-effective was 0.65 and 0.85, respectively. The model was most sensitive to the time horizon. Conclusions: The results of this analysis suggest that RARP is likely to be cost-effective in this Canadian patient population. The results are consistent with Alberta’s HTA recommendation and other economic evaluations, but challenges Ontario’s reimbursement decision.


2021 ◽  
Vol 103-B (12) ◽  
pp. 1783-1790
Author(s):  
Spencer Montgomery ◽  
Jonathan Bourget-Murray ◽  
Daniel Z. You ◽  
Leo Nherera ◽  
Amir Khoshbin ◽  
...  

Aims Total hip arthroplasty (THA) with dual-mobility components (DM-THA) has been shown to decrease the risk of dislocation in the setting of a displaced neck of femur fracture compared to conventional single-bearing THA (SB-THA). This study assesses if the clinical benefit of a reduced dislocation rate can justify the incremental cost increase of DM-THA compared to SB-THA. Methods Costs and benefits were established for patients aged 75 to 79 years over a five-year time period in the base case from the Canadian Health Payer’s perspective. One-way and probabilistic sensitivity analysis assessed the robustness of the base case model conclusions. Results DM-THA was found to be cost-effective, with an estimated incremental cost-effectiveness ratio (ICER) of CAD $46,556 (£27,074) per quality-adjusted life year (QALY). Sensitivity analysis revealed DM-THA was not cost-effective across all age groups in the first two years. DM-THA becomes cost-effective for those aged under 80 years at time periods from five to 15 years, but was not cost-effective for those aged 80 years and over at any timepoint. To be cost-effective at ten years in the base case, DM-THA must reduce the risk of dislocation compared to SB-THA by at least 62%. Probabilistic sensitivity analysis showed DM-THA was 58% likely to be cost-effective in the base case. Conclusion Treating patients with a displaced femoral neck fracture using DM-THA components may be cost-effective compared to SB-THA in patients aged under 80 years. However, future research will help determine if the modelled rates of adverse events hold true. Surgeons should continue to use clinical judgement and consider individual patients’ physiological age and risk factors for dislocation. Cite this article: Bone Joint J 2021;103-B(12):1783–1790.


BMJ Open ◽  
2021 ◽  
Vol 11 (5) ◽  
pp. e043523
Author(s):  
Zoe McCarroll ◽  
Julia Townson ◽  
Timothy Pickles ◽  
John W Gregory ◽  
Rebecca Playle ◽  
...  

ObjectiveThe aim of this economic evaluation was to assess whether home management could represent a cost-effective strategy in the patient pathway of type 1 diabetes (T1D). This is based on the Delivering Early Care In Diabetes Evaluation trial (ISRCTN78114042), which compared home versus hospital management from diagnosis in childhood diabetes and found no statistically significant difference in glycaemic control at 24 months.DesignCost-effectiveness analysis alongside a randomised controlled trial.SettingEight paediatric diabetes centres in England, Wales and Northern Ireland.Participants203 clinically well children aged under 17 years, with newly diagnosed T1D and their carers.Outcome measuresThe base-case analysis adopted n National Health Service (NHS) perspective. A scenario analysis assessed costs from a broader societal perspective. The incremental cost-effectiveness ratio (ICER), expressed as cost per mmol/mol reduction in glycated haemoglobin (HbA1c), was based on the mean difference in costs between the home and hospital groups, divided by mean differences in effectiveness (HbA1c). Uncertainty was considered in terms of the probability of cost-effectiveness.ResultsAt 24 months postintervention, the base-case analysis showed a difference in costs between home and hospital, in favour of home management (mean difference −£2,217; 95% CI −£2825 to −£1,609; p<0.001). Home care dominated, with an ICER of £7434 (saved) per mmol/mol reduction of HbA1c. The results of the scenario analysis also favoured home management. The greatest driver of cost differences was hospitalisation during the initiation period.ConclusionsHome management from diagnosis of children with T1D who are medically stable represents a less costly approach for the NHS in the UK, without impacting clinical effectiveness.Trial registration numberISRCTN78114042.


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