Cost-Effectiveness of Targeted Cancer Therapies for Hematological Malignancies.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 4547-4547
Author(s):  
Ozlem Topaloglu ◽  
Chuck Stevens ◽  
Saurabh Aggarwal
Keyword(s):  
Cost Effectiveness ◽  
Hematological Malignancies ◽  
Conflicts Of Interest ◽  
Variable Cost ◽  
High Dose ◽  
Cancer Therapies ◽  
High Dose Dexamethasone ◽  
Hematological Cancers ◽  
Targeted Cancer Therapies ◽  
The Cost

Abstract Abstract 4547 Targeted cancer therapies for hematological malignancies have rapidly gained uptake in different types of indications, and some have become gold standard of treatment. However, the high cost of these therapies poses challenges for payers, patients and physicians. Methods To understand the cost-effectiveness of these new classes of drugs we reviewed the published cost-effectiveness studies and analyzed their cost/QALY, time horizon, comparators, indications and model approaches. Results Targeted therapies for hematological cancers have much lower and less variable cost/QALY than branded drugs indicated for solid tumors. For example, incremental cost effectiveness ratio (ICER) for imatinib ranges from $40,000 to $45,000/QALY, while ICER for bevacizumab ranges from $125,000 to $325,000/QALY. Secondly, the choice of comparator in some occasions may depend on the country of use, therefore models are required to be customized to local regimens. In one of the cost-effectiveness analysis for bortezomib for refractory multiple myeloma patients, NICE questioned the use of high dose dexamethasone (HDD) as comparator. Third, there is also some variability in methods used by different manufacturers, some used cost/QALY whereas others used cost/LYG. Conclusion: This analysis shows the range, variability and methods used for calculation of ICER values for these targeted cancer therapies and provides lessons for executives and policy makers. Disclosures: No relevant conflicts of interest to declare.

scholarly journals How are we evaluating the cost-effectiveness of companion biomarkers for targeted cancer therapies? A systematic review

BMC Cancer ◽  
2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Mikyung Kelly Seo ◽  
John Cairns
Keyword(s):  
Economic Evaluation ◽  
Cost Effectiveness ◽  
Cochrane Library ◽  
Economic Evaluations ◽  
Health State ◽  
Cancer Therapies ◽  
Companion Diagnostics ◽  
Targeted Cancer Therapies ◽  
Biomarker Testing ◽  
The Cost

Abstract Background Despite the increasing economic assessment of biomarker-guided therapies, no clear agreement exists whether existing methods are sufficient or whether different methods might produce different cost-effectiveness results. This study aims to examine current practices of modeling companion biomarkers when assessing the cost-effectiveness of targeted cancer therapies. It investigates the current methods in modeling the characteristics of companion diagnostics based on existing economic evaluations of biomarker-guided therapies in cancer. Methods A literature search was performed using Medline, Embase, EconLit, Cochrane library for economic evaluations of biomarker-guided therapies with companion diagnostics in cancer. Preferred Reporting Items of Systematic Reviews and Meta-Analyses (PRISMA) guidelines were followed. Studies were selected using pre-specified eligibility criteria based on the PICO framework. To make the included studies more comparable, we qualitatively synthesized the data under nine domains of methods where consensus was deemed lacking. Results Only four of the twenty-two studies included in this review were found to be of good quality with respect to incorporating the characteristics of companion biomarkers in economic evaluations. However, many evaluations focused on a pre-selected patient group rather than including all patients regardless of their biomarker status. Companion biomarker characteristics captured in evaluations were often limited to the cost or the accuracy of the test. Often, only the costs of biomarker testing were modelled. Clinical outcomes and health state utilities were often not included due to the limited data generated by clinical trials. Methods of economic evaluation were not applied consistently in assessments of companion cancer biomarkers for targeted therapies. It was also shown that conflicting cost-effectiveness results were likely depending on what comparator arm was chosen and what comparison structure was designed in the model. Conclusion We found no consistent approach applied in assessing the value of companion biomarker tests and including the characteristics of biomarkers in an economic evaluation of targeted oncology therapies. Currently, many economic evaluations fail to capture the full value of companion biomarkers beyond sensitivity/specificity and cost related to biomarker testing.

scholarly journals Bortezomib for the treatment of multiple myeloma patients

2009 ◽  
Vol 13 (Suppl 1) ◽  
pp. 29-33 ◽  
Author(s):  
C Green ◽  
J Bryant ◽  
A Takeda ◽  
K Cooper ◽  
A Clegg ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Sensitivity Analysis ◽  
Cost Effectiveness ◽  
Disease Progression ◽  
Survival Curve ◽  
Controlled Trial ◽  
High Dose ◽  
Single Technology Appraisal ◽  
High Dose Dexamethasone ◽  
The Cost

This paper presents a summary of the evidence review group (ERG) report into the clinical effectiveness and cost-effectiveness of bortezomib for the treatment of multiple myeloma patients at first relapse and beyond, in accordance with the licensed indication, based upon the evidence submission from Ortho Biotech to the National Institute for Health and Clinical Excellence (NICE) as part of the single technology appraisal (STA) process. The outcomes stated in the manufacturer’s definition of the decision problem were time to disease progression, response rate, survival and quality of life. The literature searches for clinical and cost-effectiveness studies were adequate and the one randomised controlled trial (RCT) included was of reasonable quality. Results from the RCT suggest that bortezomib increases survival and time to disease progression compared with high-dose dexamethasone (HDD) in multiple myeloma patients who have had a relapse after one to three treatments. Cost-effectiveness analysis based on the same trial and an observational study was reasonable and gave an estimated cost per life-year gained of £30,750, which ranged from £27,957 to £36,747 on sensitivity analysis. An attempt was made to replicate the results of the manufacturer’s model and to compare the results to the Kaplan–Meier survival curve presented in the manufacturer’s submission. In addition, a one-way sensitivity analysis and a probabilistic sensitivity analysis were undertaken, as well as additional scenario analyses. Based on these analyses the ERG suggests that the cost-effectiveness results presented in the manufacturer’s submission may underestimate the cost per life-year gained for bortezomib therapy (versus high-dose dexamethasone) when potential UK practice and scenarios are considered. The guidance issued by NICE in June 2006 as a result of the STA states that bortezomib monotherapy for the treatment of relapsed multiple myeloma is clinically effective compared with HDD but has not been shown to be cost-effective and is not recommended for the treatment of progressive multiple myeloma in patients who have received at least one previous therapy and who have undergone, or are unsuitable for, bone marrow transplantation.

scholarly journals How Are We Evaluating the Cost-Effectiveness of Companion Biomarkers for Targeted Cancer Therapies? A Systematic Review

2021 ◽  
Author(s):  
Mikyung Kelly Seo ◽  
John Cairns
Keyword(s):  
Cost Effectiveness ◽  
Economic Assessment ◽  
Cochrane Library ◽  
Economic Evaluations ◽  
Cancer Therapies ◽  
Pharmaceutical Drugs ◽  
Eligibility Criteria ◽  
Targeted Cancer Therapies ◽  
Biomarker Testing ◽  
The Cost

Abstract Background Despite the increasing economic assessment of biomarker-guided therapies, no clear agreement exists whether existing methods are sufficient or whether different methods might produce different cost-effectiveness results. This study aims to examine current practices of modeling companion biomarkers when assessing the cost-effectiveness of targeted cancer therapies. It highlights the challenges in methods and data requirements faced in the evaluation of biomarker tests which do not necessarily arise with the evaluation of pharmaceutical drugs.Methods A literature search was performed using Medline, Embase, EconLit, Cochrane library. Articles published from 2014 to 2018 were searched. Economic evaluations on biomarker-guided therapies with companion diagnostics in cancer were searched. Preferred Reporting Items of Systematic Reviews and Meta-Analyses (PRISMA) guidelines followed. Studies were selected by pre-specified eligibility criteria using PICO framework. To make studies more comparable, data were synthesized under ten categories of key areas of methods where consensus deemed lacking.Results Eighteen papers were included in this review. Three out of eighteen studies found to be of good quality regarding incorporating the characteristics of companion biomarkers in economic evaluations. However, many evaluations focused on a pre-selected patient group with a specific biomarker status instead of including all patients with a disease regardless of their biomarker status. Companion biomarker characteristics captured in evaluations were often limited to the cost or the accuracy of the test. Often, only the costs of biomarker testing were modelled. Clinical outcomes or utilities were often difficult to include due to the limited data generated by clinical trials. We found that no consistency and consensus existed to the methods of existing economic evaluations of companion cancer biomarkers for targeted therapies. It was also shown that conflicting cost-effectiveness results were likely depending on what comparator arm was chosen and what comparison structure was designed in the model.Conclusion We found that there was no consistent approach applied in assessing the value of biomarkers and including the characteristics of biomarkers in an economic evaluation of targeted oncology therapies. Currently, many EEs fail to capture the full value of companion biomarkers beyond sensitivity/specificity and cost related to biomarker testing.

scholarly journals Cost-Effectiveness Analyses of FDA-Approved Drugs for Cancer Indications, 2015-2017

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 5833-5833 ◽  
Author(s):  
Ohad Oren
Keyword(s):  
Cost Effectiveness ◽  
Full Text ◽  
Conflicts Of Interest ◽  
Economic Evaluations ◽  
Solid Cancer ◽  
Potential Clinical Benefit ◽  
Hematological Cancers ◽  
Anti Cancer ◽  
Approved Drugs ◽  
The Cost

Abstract Background: In addition to a drug's anti-tumor efficacy and tissue toxicity, it is now apparent that affordability is a pivotal factor determining the net therapeutic value of an anti-cancer compound. Cost-Effectiveness Analyses (CEA) are therefore key to appreciating the overall balance between the clinical and economical repercussions of a pharmaceutical agent. We aimed to investigate the cost-effectiveness data of newly approved drugs in cancer care. Methods: The FDA website was reviewed for all drugs approved for any cancer indication between the years 2015-2017. A systematic search of Pubmed and Google Scholar was conducted for Cost-Effectiveness Analyses (CEA) of each of these medications. Average Wholesale Prices were collected from Uptodate.com. Results: 30 drugs were approved for cancer indications in 2015-2017. Each of the approved drugs had an average of 1.27 CEA studies (range 0-6). 4 of the 38 (10.5%) available CEAs were done before approval of an index drug. 18 (60%) of the approved medications had an available published CEA at the time of our search (March, 2018). Partition survival modelling (31.5%) and Markov modelling (26.3%) were the most common methods of analysis. 47.3% (18/38) of the CEAs were thought to reflect a favorable ICER per the analysis's investigators. The nature of the malignancy (solid versus hematologic) did not distinguish between the mean number of a drug's CEAs (1.25; 1.2) but drugs for solid cancer indications were less likely to be associated with a favorable CE assessment compared with hematological cancers (47.8% versus 58.3%). "Expensive" drugs (defined as monthly cost greater than 8,618 dollars) had a lower mean number of CEA studies than "less expensive" drugs (1.09 versus 1.36) and were less likely to be associated with a favorable cost-effectiveness profile (27.2% versus 73.6%). 47.3% (18/38) of the economic evaluations were published as conference proceeding/abstract, 36.8% (14/38) were in the form of a full-text article, and the remainder as journal letters or conference posters (6/38; 15.7%). Conclusion: Insufficient data on the cost-effectiveness profile of novel anti-cancer medications jeopardises our ability to determine their real value. Although these drugs are being routinely used in a large-scale fashion, gaps persist as to their financial harm relative to the associated (potential) clinical benefit. Our study shows that recently-approved anti-cancer drugs have a very small number of CEAs to back their clinical-societal merit. Paradoxically, more costly drugs have fewer CEAs compared with cheaper drugs. Also of note, the majority of the analyses followed the approval of the drug, were published in a non full-text format, and were associated with non-sustainable ICER values. We argue that publication of rigorous, peer-reviewed CEAs should be a mandatory pre-approval step for academia/industry and serve as a pre-requisite to the routine distribution and usage of new cancer-directed medications. Disclosures No relevant conflicts of interest to declare.

An Economic Evaluation of a Community-Based Organization Led HIV Self-Testing Program Among Men Who Have Sex With Men in China

2020 ◽  
Author(s):  
Shanzi Huang ◽  
Jason Ong ◽  
Wencan Dai ◽  
Xi He ◽  
Yi Zhou ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Hiv Testing ◽  
Variable Cost ◽  
Community Based ◽  
Self Testing ◽  
Hiv Test ◽  
Community Based Organization ◽  
The Mean ◽  
The Cost ◽  
Mean Cost

Abstract Introduction: HIV self-testing (HIVST) is effective in improving the uptake of HIV testing among key populations. Complementary data on the cost-effectiveness of HIVST is critical for planning and scaling up HIVST. This study aimed to evaluate the cost-effectiveness of a community-based organization (CBO)-led HIVST model implemented in China. Method: A cost-effectiveness analysis (CEA) was conducted by comparing a CBO-led HIVST model with a CBO-led facility-based HIV rapid diagnostics testing (HIV-RDT) model. The full economic cost, including fixed and variable cost, from a health provider perspective using a micro costing approach was estimated. We determined the cost-effectiveness of these two HIV testing models over a two year time horizon (i.e. duration of the programs), and reported costs using US dollars (2020). Results: From January 2017 to December 2018, a total of 4,633 men tested in the HIVST model, and 1,780 men tested in the HIV-RDT model. The total number of new diagnosis was 155 for HIVST and 126 for the HIV-RDT model; the HIV test positivity was 3.3% (95% confidence interval (CI): 2.8-3.9) for the HIVST model and 7.1% (95% CI: 5.9-8.4) for the HIV-RDT model. The mean cost per person tested was $14.57 for HIVST and $24.74 for HIV-RDT. However, the mean cost per diagnosed was higher for HIVST ($435.52) compared with $349.44 for HIV-RDT.Conclusion: Our study confirms that compared to facility-based HIV-RDT, a community-based organization led HIVST program could have a cheaper mean cost per MSM tested for HIV in China. Better targeting of high-risk individuals would further improve the cost-effectiveness of HIVST.

scholarly journals FP728A COMPARISON OF THE COST-EFFECTIVENESS OF HIGH DOSE HEMODIALYSIS (HD) VERSUS CONVENTIONAL IN-CENTER HEMODIALYSIS (ICHD) IN THE NETHERLANDS, FRANCE, AND THE UK

2015 ◽  
Vol 30 (suppl_3) ◽  
pp. iii320-iii320
Author(s):  
Dilip Makhija ◽  
Suzanne Laplante ◽  
Frank Xiaoqing Liu ◽  
Anna Trisia Beby ◽  
Jean-Jacques Dumas ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
The Netherlands ◽  
High Dose ◽  
The Uk ◽  
The Cost

A Three Year Experience at An Inner City Safety-Net Hospital with Immune Thrombocytopenic Purpura (ITP)

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 4684-4684
Author(s):  
Harsha V Poola ◽  
Manila Gaddh ◽  
Samuel N. Ofori ◽  
Moushmi Shah ◽  
Mohammed A. Kassem ◽  
...  
Keyword(s):  
Inner City ◽  
Immune Thrombocytopenic Purpura ◽  
Conflicts Of Interest ◽  
Hepatitis Virus ◽  
Far East ◽  
Safety Net ◽  
High Dose ◽  
Thrombocytopenic Purpura ◽  
High Dose Dexamethasone ◽  
Safety Net Hospital

Abstract Abstract 4684 Immune Thrombocytopenic Purpura or ITP remains a clinical diagnosis of exclusion. There are numerous treatments, attesting to the fallibility of each. A 3 year experience at an Inner City safety net hospital was analyzed. For inclusion in the study, the hematology service had to have excluded consumption disorders, prior chemotherapy, medication known to cause thrombocytopenia and Viral Infection- HIV and/Hepatitis. 93 patients met these criteria and had platelet counts below 50,000. The median age of the whole group was 49 yrs, with a range of 21 to 78 years. A few were ANA positive. The female preponderance reflects the literature. All Patients were started on Prednisone at 1mg/kg. Three Patients also received IV IGG to hasten the response. There were no intracranial hemorrhages or bleeding described as major. Patients from the Far East had to be excluded for Hepatitis Virus exposure. Results All who did not respond to steroids fully were treated with a second line Rx. i.e, Rituximab, Azathioprine, IV IGG or WIN Rho. One patient received high dose Dexamethasone and responded. Conclusion: Pending the use of TPO agonists, treatment of ITP in adults remains a chronic problem challenging the Hematologist to use as little corticosteroid as possible. Disclosures: No relevant conflicts of interest to declare.

Rituximab and High-Dose Dexamethasone (R-dex) Is Effective In The Treatment Of Relapsed/Refractory Chronic Lymphocytic Leukemia

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 4184-4184
Author(s):  
Martin Simkovic ◽  
David Belada ◽  
Monika Motyckova ◽  
Lukas Smolej ◽  
Pavel Zak
Keyword(s):  
Chronic Lymphocytic Leukemia ◽  
Conflicts Of Interest ◽  
Antimicrobial Prophylaxis ◽  
Univariate Analysis ◽  
Progression Free Survival ◽  
Lymphocytic Leukemia ◽  
Published Data ◽  
High Dose ◽  
High Dose Dexamethasone ◽  
Tertiary Center

Abstract Introduction High-dose methylprednisolone (HDMP) in combination with rituximab is active in the treatment of relapsed/refractory chronic lymphocytic leukemia (CLL) but serious infections are frequent. Recently published data suggest that high-dose dexamethasone might be equally effective to HDMP despite lower cumulative dose. Aims To assess efficacy and safety of high-dose dexamethasone combined with rituximab (R-dex) in relapsed/refractory CLL. Patients and Methods A total of 60 pts (pts) with relapsed/refractory CLL treated at a single tertiary center between September 2008 and October 2012 were included in this retrospective analysis. Basic characteristics are summarized in Table 1. The schedule of R-dex consisted of rituximab 500 mg/m2 i.v. day 1 (375 mg/m2 in cycle 1) and dexamethasone 40 mg orally on days 1-4 and 10-13. Treatment was repeated every 3 weeks for a maximum of 8 cycles. All pts received antimicrobial prophylaxis with sulfamethoxazole/trimethoprim and aciclovir. Results Median number of administered R-dex cycles was 6 (range, 1-8). The overall response (ORR)/complete remissions (CR) were achieved in 75/3%. At the median follow-up of 9 months, median progression-free survival was 8 months and median overall survival 24 months. Significant predictors of short PFS in univariate analysis were bulky lymphadenopathy (p=0.023) and refractoriness to fludarabine (p=0.02). Interestingly, activity of R-dex in bulky fludarabine-refractory CLL was similar to ofatumumab (ORR 62 %, median PFS, 4 months, median OS, 12 months) (Wierda et al., 2010). R-dex was successfully used as a debulking regimen before allogeneic stem cell transplantation in 8 patients. Serious (CTCAE grade III/IV) infections occurred in 29% of patients; 19% pts developed steroid diabetes requiring temporary short-acting insulin. Conclusions Our data show that R-Dex is an active and feasible treatment for patients with relapsed/refractory CLL; however, major infections remain relatively frequent despite combined antimicrobial prophylaxis. In addition, durable responses are infrequent. Therefore, further optimization of this therapeutic approach is warranted. Updated results will be presented. Disclosures: No relevant conflicts of interest to declare.

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