scholarly journals P758 Ileorectal anastomosis vs. ileal pouch-anal anastomosis for the surgical treatment of ulcerative colitis: A Markov decision analysis

2020 ◽  
Vol 14 (Supplement_1) ◽  
pp. S605-S605
Author(s):  
A De Buck Van Overstraeten ◽  
M Brar ◽  
S Khorasani ◽  
F Dossa ◽  
P Myrelid
Keyword(s):  
Ulcerative Colitis ◽  
Rectal Cancer ◽  
Sensitivity Analysis ◽  
Time Horizon ◽  
Ileal Pouch ◽  
Ileorectal Anastomosis ◽  
Quality Adjusted Life Years ◽  
Life Years ◽  
Anal Anastomosis ◽  
Quality Adjusted Life

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.

scholarly journals Estimating the Financial Impact of Gene Therapy*

2020 ◽  
Author(s):  
Chi Heem Wong ◽  
Dexin Li ◽  
Nina Wang ◽  
Jonathan Gruber ◽  
Rena Conti ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Time Horizon ◽  
Financial Impact ◽  
Age Group ◽  
Patient Access ◽  
Quality Adjusted Life Years ◽  
Gene Therapies ◽  
Life Years ◽  
Expected Benefits ◽  
Quality Adjusted Life

AbstractWe assess the potential financial impact of future gene therapies by identifying the 109 late-stage gene therapy clinical trials currently underway, estimating the prevalence and incidence of their corresponding diseases, developing novel mathematical models of the increase in quality-adjusted life years for each approved gene therapy, and simulating the launch prices and the expected spending of these therapies over a 15-year time horizon. The results of our simulation suggest that an expected total of 1.09 million patients will be treated by gene therapy from January 2020 to December 2034. The expected peak annual spending on these therapies is $25.3 billion, and the total spending from January 2020 to December 2034 is $306 billion. We decompose their annual estimated spending by treated age group as a proxy for U.S. insurance type, and consider the tradeoffs of various methods of payment for these therapies to ensure patient access to their expected benefits.

scholarly journals Cost-Effectiveness Evaluation of Etoricoxib versus Celecoxib and Nonselective NSAIDs in the Treatment of Ankylosing Spondylitis in Norway

2011 ◽  
Vol 2011 ◽  
pp. 1-14 ◽  
Author(s):  
Jeroen P. Jansen ◽  
Stephanie D. Taylor
Keyword(s):  
Cost Effectiveness ◽  
Ankylosing Spondylitis ◽  
Time Horizon ◽  
Cost Effective ◽  
Quality Adjusted Life Year ◽  
Quality Adjusted Life Years ◽  
Life Years ◽  
Cost Effective Treatment ◽  
Ceiling Ratio ◽  
Quality Adjusted Life

Objectives. To evaluate the cost-effectiveness of etoricoxib (90 mg) relative to celecoxib (200/400 mg), and the nonselective NSAIDs naproxen (1000 mg) and diclofenac (150 mg) in the initial treatment of ankylosing spondylitis in Norway.Methods. A previously developed Markov state-transition model was used to estimate costs and benefits associated with initiating treatment with the different competing NSAIDs. Efficacy, gastrointestinal and cardiovascular safety, and resource use data were obtained from the literature. Data from different studies were synthesized and translated into direct costs and quality adjusted life years by means of a Bayesian comprehensive decision modeling approach.Results. Over a 30-year time horizon, etoricoxib is associated with about 0.4 more quality adjusted life years than the other interventions. At 1 year, naproxen is the most cost-saving strategy. However, etoricoxib is cost and quality adjusted life year saving relative to celecoxib, as well as diclofenac and naproxen after 5 years of follow-up. For a willingness-to-pay ceiling ratio of 200,000 Norwegian krones per quality adjusted life year, there is a >95% probability that etoricoxib is the most-cost-effective treatment when a time horizon of 5 or more years is considered.Conclusions. Etoricoxib is the most cost-effective NSAID for initiating treatment of ankylosing spondylitis in Norway.

Cost-Effectiveness of Filgrastim and Pegfilgrastim as Primary Prophylaxis against Febrile Neutropenia in Lymphoma Patients Receiving R-CHOP Chemotherapy.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 2475-2475
Author(s):  
Nina Lathia ◽  
Pierre K Isogai ◽  
Scott Walker ◽  
Matthew C Cheung ◽  
Murray Krahn ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Febrile Neutropenia ◽  
Confidence Interval ◽  
Induction Chemotherapy ◽  
Time Horizon ◽  
Primary Prophylaxis ◽  
Sensitivity Analyses ◽  
Quality Adjusted Life Years ◽  
Life Years ◽  
Quality Adjusted Life

Abstract Abstract 2475 Poster Board II-452 Introduction: Non-Hodgkin Lymphoma (NHL) is the fifth most common type of malignancy in Canada. The most common subtype of NHL is diffuse large B-cell lymphoma (DLBCL). Initial standard treatment for DLBCL includes combination immuno-chemotherapy with rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone (R-CHOP). This regimen is typically administered every 21 days for a total of 6 cycles. Febrile neutropenia (FN) is a serious toxicity of lymphoma chemotherapy and patients with this condition must be treated aggressively as it could lead to complications such as prolonged hospitalization and death. Granulocyte-colony stimulating factors such as filgrastim and pegfilgrastim are efficacious in preventing FN, yet their cost-effectiveness has not been evaluated in the publicly funded Canadian healthcare system. Methods: A Markov model was constructed to evaluate the cost-effectiveness (cost-utility) of filgrastim and pegfilgrastim as primary prophylaxis versus no primary prophylaxis against FN in DLBCL patients receiving induction chemotherapy. Health states included in the model were hospitalization for FN, and receiving chemotherapy with no FN. It was assumed that patients in the no primary prophylaxis arm of the model who experienced a FN episode would receive secondary prophylaxis with filgrastim for all subsequent chemotherapy cycles. The time horizon of the model was 18 weeks, the time period over which the six cycles of chemotherapy are administered. The analysis was conducted from the hospital perspective. Costs are reported in 2009 Canadian dollars and outcomes in quality-adjusted life years (QALY). One-way sensitivity analyses were done on model parameters. A probabilistic sensitivity analysis was done to evaluate overall uncertainty in the model. Results: In the base case analysis costs associated with the no primary prophylaxis, filgrastim and pegfilgrastim interventions were $6044, $9450 and $15899, respectively. Quality-adjusted life years associated with the three interventions were 0.198, 0.200, and 0.202 respectively (over the 18-week model time horizon). The incremental cost-effectiveness ratio (ICER) of filgrastim compared to no primary prophylaxis was estimated to be $1.7 million (M)/QALY [95% confidence interval: -14M/QALY (dominated) to $15M/QALY]; for pegfilgrastim compared to filgrastim the ICER was estimated to be $4.4M/QALY [95% confidence interval: -25M/QALY (dominated) to $22.7M/QALY]. All one-way sensitivity analyses yielded ICERs of greater than $1M/QALY. Conclusions: The ICERs for filgrastim and pegfilgrastim when used as primary prophylaxis against FN in DLBCL patients receiving induction chemotherapy are well above the usually accepted cost-effectiveness threshold of $50,000/QALY. Disclosures: Mittmann: Amgen Canada: Unrestriced educational grant.

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 ◽  
...  
Keyword(s):  
Sensitivity Analysis ◽  
Life Expectancy ◽  
Probabilistic Sensitivity Analysis ◽  
Time Horizon ◽  
Case Analysis ◽  
Base Case ◽  
Base Case Analysis ◽  
Non Hodgkin Lymphoma ◽  
Life Years ◽  
Quality Adjusted Life

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.

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