bluuebunny/crossref_metadata_2025_split huggingface dataset:
Crowdfunding for lung cancer costs.
Authors: Nguyen Ryan , Hanna Nasser H. , Vater Laura 
Published: May 2019

e18340 Background: Online crowdfunding, where individuals create campaigns to solicit donations, has grown as an avenue to combat the financial toxicities of cancer. The online platform GoFundMe hosts more than 80% of the global market for crowdfunding and has raised over US $5 billion from more than 50 million donors. While medical expenses are the leading cause of crowdfunding campaigns, limited research is available on the use of crowdfunding for cancer costs. Further, no studies have evaluated the use of crowdfunding for patients with lung cancer. Methods: In January 2019, we reviewed the first 200 consecutive campaigns that resulted for “lung cancer” on GoFundMe. Campaigns were included for analysis if their description stated funds were to be used for medical costs for a patient with lung cancer in the United States. Standardized data was collected from each campaign. Descriptive statistics were used to aggregate results. Multivariable linear regression analysis were performed to examine predictors of funds raised, adjusting for campaign duration. Results: The 157 included campaigns raised a total of US $1.2 million (mean $8,364) from 11,919 donors (median 53). Compared to a similar 2018 study, our study showed lung cancer campaigns raised less than breast cancer (mean $16,026) but more than prostate cancer (mean $1,449) campaigns. Nine campaigns that were seeking funds for alternative treatment raised a total of $119,660 (mean $13,296). Narratives of financial need that were significantly associated with greater funds raised were family financial need (+$7,416), medical costs not covered by insurance (+$7,369), and the campaign stating the patient was a never-smoker (+$8,162) (all P values < 0.05). Conclusions: Lung cancer has received less research funding relative to other cancer types and our study suggests a similar disparity with crowdfunding for medical costs. The “blame-the-victim” attitude that contributes to this funding disparity also exists within lung cancer crowdfunding as evidenced by the significantly greater amount of funds raised for campaigns that explicitly stated the patient was a non-smoker. Efforts to study crowdfunding for cancer costs should address the ethical implications of exacerbating funding disparities and funding for alternative treatments.
Understanding differences in total cancer care cost by service site.
Authors: Fields Karen K. , Burkett Jason M , Terrano Cindy , Dukes Floyd , Gorantla Bharat , Salganicoff Marcos , Rao Bharat 
Published: May 2019

e18345 Background: Oncology providers are under mounting pressure to deliver high quality and increasingly complex cancer treatment while simultaneously managing the cost pressures coming from payers and patients alike. Developing an effective strategy to compare costs can be an important tool for assessing and improving performance and cost effectiveness from both a clinical and market competitive standpoint. This comparison can be difficult to achieve in practice, especially between different sites of care and across heterogeneous patient populations. Methods: A large commercial claims dataset with reimbursement data for claims incurred between 2014 and 2017 was used to identify lung cancer patients by site of care. We calculated average annual total cost of care (TCC) for a one year period for patients with a new cancer diagnosis following a one year period of no claims for lung cancer. For study purposes, Moffitt Cancer Center (MCC) was compared to two other peer groups: Large Hospital Systems (LHS) and small hospitals and community-based oncology practices (CBOP). Patients were assigned to each peer group when a majority of claims ( > 70%) were attributed to one of the peer groups. We analyzed overall average annual TCC for all patients and then created a sub-cohort of patients who received surgery within the study period to define early stage patients based on known standard patterns of care for newly diagnosed lung cancer. Results: There were 1249 new lung cancer patients in the study across all three peer groups with an average annual TCC of $134K per patient (range: $110K to $149K). When considering the sub-cohort of early stage patients (n = 396), MCC’s average annual TCC was $124K per patient which was significantly lower than LHS ($152K; p < 0.0499) and CBOP ($167K; p < 0.01). Conclusions: Although claims data generally contain only limited clinical information such as procedures, supplies and diagnosis, this limitation can be address by incorporating known standard of care patterns for cancer treatment to create comparable groups. Using this approach, national claims sets can be leveraged as the basis of a powerful analysis tool for understanding the Total Cost of Cancer care across institutional boundaries while still achieving meaningful comparability of the analyzed patient population.
Real-world demographic and clinical characteristics of patients diagnosed with diffuse large B-cell lymphoma (DLBCL) in the United States (US).
Authors: Yang Xiaoqin , Laliberté François , Germain Guillaume , Raut Monika Kumar , Duh Mei Sheng , Sen Shuvayu , Lejeune Dominique , Cavanaugh Cristi , Desai Kaushal , Armand Phillippe 
Published: May 2019

e18347 Background: DLBCL, the most common type of non-Hodgkin lymphoma in the US, is associated with significant morbidity and mortality. In October 2015, DLBCL was differentiated from other related lymphoma entities with the advent of ICD-10-CM DLBCL-specific codes. With limited real-world data on patients (pts) with DLBCL in the modern treatment era, this study was conducted to characterize these pts. Methods: A retrospective study was conducted using the Optum Clinformatics Data Mart database (01/2013–03/2018). Pts ≥ 18 years of age with ≥ 1 hospitalization or ≥ 2 outpatient visits with an ICD-10-CM diagnosis code for DLBCL (or an antecedent diagnosis of other lymphoma, which may have been assigned before confirmation of DLBCL) after October 1st, 2015 (index date) and no prior ICD-9-CM code for unspecified DLBCL were identified as incident. Pts with an ICD-9-CM code for unspecified DLBCL before October 2015 (index date) were classified as prevalent. At least 12 months of continuous enrollment pre-index date (baseline period) was required. Pts with ICD-10-CM code for primary mediastinal B-cell lymphoma (PMBCL), baseline diagnoses of other malignancies such as Hodgkin lymphoma and multiple myeloma were excluded. Characteristics, including baseline comorbidities, healthcare resource utilization, and costs were assessed. Results: Among 4,074 DLBCL pts (3,201 incident; 873 prevalent), mean age ± standard deviation (SD) was 71 ± 12 years; 46% were female. Incident and prevalent pts had mean Charlson comorbidity index scores of 2.7 and 2.3, respectively. Most common baseline Elixhauser comorbidities were hypertension (68.4%), diabetes (31.1%), and cardiac arrhythmia (25.3%) in incident pts and hypertension (62.5%), diabetes (28.3%), and chronic pulmonary disease (20.6%) in prevalent pts. Mean ± SD number of baseline hospitalizations was 0.32 ± 0.83 and 0.21 ± 0.49 in incident and prevalent pts, respectively. Total mean ± SD baseline healthcare costs (before diagnosis) were $24,621 ± 45,628 for incident pts and $19,137 ± 29,307 for prevalent pts. Conclusions: This study documents substantial co-morbid and economic burden of incident as well as prevalent pts with DLBCL.
Projecting overall survival data for health-economic models in oncology: Do maturity levels impact uncertainty?
Authors: Roze Stéphane , Bertrand Nicolas , Eberst Lauriane , Borget Isabelle 
Published: May 2019

e18350 Background: A lifetime horizon is recommended for health-economic evaluation of anticancer drugs. If overall survival (OS) data is immature, extrapolation of the Kaplan-Meier (KM) curve using distributions is done to obtain long-term data. Depending on OS maturity, the distribution chosen may impact estimation of life expectancy (LE) and of life years gained (LYG) between treatments. This study aimed to estimate the error (on LE and LYG) induced by the choice of extrapolation distributions, for 2 levels of OS maturity (30% and 50%), as compared to LE and LYG at full maturity. Methods: Fifteen phase 3 trials published between 2013 and 2017 containing OS KM curves were selected if maturity > 70% (Full). To test 2 maturity levels, each KM curve was truncated at 30% and 50%. A 3-step process was performed: 1) KM was digitalized, 2) individual patient-data was reproduced using the Guyot algorithm, and 3) 5 parametric survival distributions were fit using the R-Survival package. For each study, the process was done for each treatment arm and each of the 3 maturity levels on the same time horizon, equal to the maximum follow-up of the study. For each curve, the best distribution was chosen by a board of 2 oncologists and 2 health-economists, based on visual inspection, Akaike/Bayesian Information criteria and external validity. Results: Based on the board review of the 90 KM curves, main chosen distributions were Weibull (33%), Log-logistic (32%) and Log-normal (27%). As compared to LE at full maturity, LE was overestimated in 23% and 40% at 30% and 50% maturity, respectively. Mean absolute error was 0.88 at 30% maturity, and decreased to 2.12 at 50% maturity, i.e. estimation was 2.4 times better from 30% to 50% maturity. On average, at 30% maturity versus full, mean percentage of error in LYG was 126.4% and 62.4% at 50% maturity versus full. Conclusions: The use of immature OS data increases the risk of error when projecting long-term LE. Even marginal gains in OS maturity can be translated in more accurate estimations and guide health-economist in developing models.
Cost-effectiveness of programmed cell death protein 1/programmed death-ligand 1 inhibitors (aPD1/aPDL1) varies widely.
Authors: Geisler Benjamin P , Pridham Leesa L. , Monge B. M. Cecilia , Johnson Tali M. , Karnon Jonathan , Sharon Elad 
Published: May 2019

e18351 Background: Immunotherapy with aPD1/aPDL1 has been shown to be efficacious for a range of indications. However, cost-effectiveness has only been demonstrated for some. Our goal was to compare incremental costs and effects across Food and Drug Administration (FDA) approved aPD1/aPDL1 using a standardized value framework. Methods: aPD1/aPDL1 randomized controlled trials (RCTs) were obtained from the FDA's 2014-18 approval lists. Restricted mean Overall (OS) and Progression-Free Survival (PFS) were extrapolated to 10 years via best-fitting (Akaike/Bayesian information criterion) parametric function (Kaplan-Meier survival curves digitized via Henley&Hoyle approach). Average wholesale price for experimental and comparator regimens from approval year’s Micromedex Red Book (IBM Watson Health, Cambridge, MA) were applied to either median treatment duration or mean extrapolated PFS, assuming the entire cohort was treated with no dose reductions. For multicenter RCTs with physician’s choice of the comparator, we assumed the regimen most likely used in the U.S. Incremental cost-effectiveness ratios (ICER) were discounted at 3% per annum. Results: Undiscounted OS gains with aPD1/aPDL1 after extrapolation to 10 years ranged from 0.72 to 1.89 LYs as compared to between 0.13 and 0.27 LYs at the end of the RCT. Discounted additional costs for aPD1/aPDL1 ranged from about -$1,700 to $348,312. Crude estimates of the incremental cost per LY gained vary widely, with ICERs for PACIFIC and Impower150 beyond $150,000/LY in both analyses, and additionally Keynote 024 and Checkmate 214 in the analysis that assumed treatment to the end of PFS. Conclusions: Value for money appears to vary widely across indications and comparators, from cost-saving to low-value. Common extrapolation-based analyses of aPD1/aPDL1-based regimens can help elucidate differences in value. The presented analyses are preliminary. Further assessment of extrapolation methods, downstream costs and quality of life effects are required. [Table: see text]
Cost-effectiveness of once weekly carfilzomib 70 mg/m2 plus dexamethasone in patients with relapsed and refractory multiple myeloma in the United States.
Authors: Kumar Shaji , Majer Istvan , Panjabi Sumeet , Malacan Jean , Medhekar Rohan , Campioni Marco , Dimopoulos Meletios A. 
Published: May 2019

e18356 Background: Carfilzomib plus dexamethasone (Kd) dosed once weekly at 70 mg/m2 (QW Kd70) was recently approved in the US for treating patients with relapsed and refractory multiple myeloma (RRMM). To assess the cost-effectiveness (CE) of QW Kd70 vs twice weekly Kd dosed at 27 mg/m2 (BIW Kd27), data from the phase 3 ARROW trial, which directly compared these regimens in patients with 2-3 prior lines of therapy were used. Methods: A partitioned survival model was developed for the CE analysis. Time to treatment discontinuation, progression-free survival, and overall survival (OS) were estimated from the ARROW trial. Long-term OS was extrapolated using Surveillance Epidemiology and End Results registry data after matching characteristics of patients in the registry and ARROW trial. Direct costs were estimated from a US health care payer perspective. Utilities collected in the ARROW trial using the five-level version of the EuroQol questionnaire (EQ-5D-5L) were applied to estimate the quality-adjusted life years (QALYs). Uncertainty was explored using sensitivity analyses. Two subgroups of patients refractory to lenalidomide or bortezomib were assessed. Main outcomes were mean life-years (LYs), QALYs, lifetime costs, and incremental cost-effectiveness ratios (ICERs). Results: For QW Kd70 and BIW Kd27, the model predicted mean LYs of 4.17 and 3.07 years, QALYs of 2.98 and 2.03 years, and mean total lifetime costs of $444,563 and $373,364, respectively. The incremental LYs gain, QALY gain, and incremental costs of QW Kd70 vs BIW Kd27 were estimated to be 1.10 years, 0.95 year, and $71,199, respectively, resulting in an ICER of $64,595 per LY gained and $75,204 per QALY gained. For patients refractory to lenalidomide and bortezomib, similar results were found with ICERs of $79,988 and $76,793, respectively. Conclusions: In line with ARROW trial results, this CE analysis showed that QW Kd70 is expected to provide considerable additional benefit in terms of LYs and QALYs gained compared with BIW Kd27. In the RRMM setting, QW Kd70 is cost-effective with ICERs below accepted willingness to pay thresholds in US and represents an efficient utilization of the health care budget.
Pharmacy cost in cancer.
Authors: Shah Surbhi , Rubin Nathan 
Published: May 2019

e18358 Background: Health care spending in US is highest in the developed world and contributes to up to 1/5 of the GDP. The price escalation is steep and contribution from cancer care is soaring. The cost of medications is deemed to be the leading cause of increased health care spending. In this era of precision medicine, with more effective and better tolerated drugs, patients are using them for longer periods of time, adding to the ever mounting health care spending. Methods: We used a large claims based data set US database MarketScan to explore the economic burden of drug cost in cancer care. Between January 1, 2013 and September 30, 2015 we identified 195,290 enrollees with active cancer. We analyzed the economic burden of medications for overall cancer care by exploring the total cost of care and the pharmacy expenditure by various classes of drugs for these patients. The perspective was that of the health care system as the costs included payments by the insurer and the patient. Results: There were 195,290 active cancer patients in this analysis. Mean age was 61 years, 55% were females. Breast cancer was the most common diagnosis. Mean total cost of care and total drug cost per patient over the study period was $141,415 and $13,579, respectively. The total pharmacy expenditure across all study patients was ~2.5B. Antineoplastic drugs make up the largest portion of the total pharmacy expenditure at 39%. Cost contribution based on drug categories were anti-infective (6%), cardiovascular (6%), central nervous system (including opiates, anti-nausea medications and antidepressants) (7%), blood formulations (including anticoagulants) (8%), hormones (8%) and gastrointestinal drugs (4%) respectively. Conclusions: Based on the real world information from a large insurance claims database, this study quantifies the contribution of various drug classes to the cost of cancer care. Antineoplastic contribute to > 1/3rd of the total pharmacy spending. With increasing trend for immunotherapy and combination therapy drug costs are bound to go up even more steeply. Unless drug prices are regulated, we are looking towards an unsustainable level of growth in the health care spending in cancer care.
Cost effectiveness (CE) of blinatumomab (BLIN) versus inotuzumab ozogamicin (INO) in adult patients with relapsed/refractory (R/R) acute lymphoblastic leukemia (ALL) with no more than one prior salvage therapy (S0/S1) from a United Kingdom health care perspective.
Authors: Delea Thomas E. , Zhang Xinke , Amdahl Jordan , Boyko Diana , Severin Franziska , Campioni Marco , Cong Ze 
Published: May 2019

e18359 Background: To estimate Incremental CE Ratio (ICER) of BLIN vs INO in adults with R/R S0/S1 ALL based on matching-adjusted indirect comparison (MAIC) of TOWER and INO-VATE trials from a UK healthcare perspective. Methods: CE was estimated by a partitioned-survival model with outcomes based on published aggregate data from INO-VATE (cutoff: 01/05/2017) and individual patient data from TOWER weighted to match patients in INO-VATE using MAIC. Five analyses were conducted based on alternative approach for the MAIC (anchored through Standard of Care [SOC] vs unanchored), proportional hazard (PH) assumptions, and reference overall survival (OS) distributions. Rates of complete remission and HSCT, utilities, duration of therapy, and use of subsequent therapies also were MAIC adjusted. Due to inconsistent definitions of event-free survival (EFS) between the trials, EFS was assumed to be equal for BLIN and INO complete responders. Costs were estimated from published sources and included those of medicine and administration, key adverse events, HSCT, salvage therapy, and terminal care. Utilities were based on EORTC-8D values derived from EORTC QLQ-C30 assessments in TOWER. A 50-year time horizon was used. Results: In all analyses, BLIN was more costly and more effective than INO. The ICER for BLIN vs INO ranged from £4,014 to £13,371 per QALY. Conclusions: For various approaches for conducting MAIC of BLIN vs INO, BLIN was highly cost effective vs INO in R/R ALL adults with S0/S1 from a UK healthcare perspective. [Table: see text]
Cost effectiveness of updated laboratory monitoring schedule for immunotherapy regimes.
Authors: Ronan Karine , Che Othman Elly Hanis , McKenna Susan , Anderson Cian , Sheehan David , Griffin Margaret , McDermott Ray , Westrup Jennifer 
Published: May 2019

e18363 Background: Patients being treated with immunotherapy undergo frequent laboratory testing to monitor for immune-related adverse events (IRAEs) as per National Cancer Control Programme (NCCP) guidelines in Ireland. Current NCCP guidelines advise pre-treatment testing of thyroid function, glucose and cortisol and follow-up TFTs and glucose levels with each cycle of therapy.1,2,3,4. Endocrinopathies occur in approximately 10% of patients on immunotherapy,5 with most occurring in the first three months of treatment.6 Hypophysitis is a relatively common adverse event associated with ipilimumab and with combination treatments, with incidence rates reported at 10-15%.7,8 Methods: On review of our own cohort and recent publications on incidence rates of IRAEs,5 we adopted updated guidelines for routine laboratory monitoring on immunotherapy. These guidelines incorporate increased monitoring intervals and enhanced surveillance for diabetes mellitus, hypophysitis and adrenal insufficiency. Results: Our proposed schedule advises pre-treatment testing of FBC, U&E, LFTs, TFTs, serum glucose, HbA1c, ACTH and serum cortisol levels. Monitoring then occurs every 3-4 weeks with FBC, LFTs, U&E, TFTs, serum glucose, and routinely testing ACTH and cortisol for Ipilimumab and combination treatments only, for six months duration. After six months, the interval increases to monitoring every 6-8 weeks with the same tests. We have compared the cost of monitoring for one year using our guidelines versus NCCP guidelines,1,2,3,4 using pricing as per a single institution laboratory. We also compared the frequency of monitoring over one year with monitoring versus NCCP guidelines. Conclusions: We conclude that our proposed algorithm for monitoring for IRAEs is cost effective, despite more frequent monitoring of ACTH and cortisol, and carries a lower burden of testing for patients, when compared with monitoring as per current NCCP guidelines. [Table: see text]
Cost-effectiveness (CE) of regorafenib dose optimization schedule in metastatic colorectal cancer.
Authors: Barzi Afsaneh , Cho Sang K , Hay Joel W , Lenz Heinz-Josef , Ou Fang-Shu , Grothey Axel , Bekaii-Saab Tanios S. 
Published: May 2019

e18367 Background: Regorafenib at 160mg daily 21/28 days is a standard therapeutic option in patients with metastatic colorectal cancer. However, the cost of treatment and management of its side-effects is more than the cost-effectiveness threshold for oncology drugs in US and many countries around the world. ReDOS (Regorafenib Dose Optimization Study), is a randomized phase II trial in refractory mCRC and established that weekly dose escalation of regorafenib over the conventional dosing is superior in the composite endpoint of safety and efficacy. We explored the impact of the new dosing strategy on CE of regorafenib. Methods: A Markov model was constructed to compare costs and effectiveness of regorafenib (standard), regorafenib (ReDOS), and best supportive care (BSC). Model inputs for clinical efficacy and safety were from the CORRECT trial for regorafenib (standard) and BSC, and the ReDOS study for regorafenib (ReDOS). The incremental cost-effectiveness ratios (ICERs) were reported to compare treatments. Model robustness was checked with univariate and probabilistic sensitivity analyses. The model used a US payer’s perspective with 5% annual discount rate, and all costs were measured in 2018 US dollars. Results: Regorafenib (ReDOS) dominated regorafenib (standard), producing higher quality-adjusted life years at a lower cost. When compared to BSC, regorafenib (ReDOS) resulted in the ICER of $112,705 while regorafenib (standard) resulted in the ICER of $384,687. The most influential parameters on the ICERs were efficacy and health state utility parameters under univariate sensitivity analysis. In probabilistic sensitivity analysis using cost-effectiveness acceptability curves, regorafenib (ReDOS) was more cost-effective than regorafenib (standard) and BSC in 70% of repetition at the willingness-to-pay threshold of $120,000 per QALY (Quality-Adjusted Life Year) and 98% at a WTP threshold of $150,000. Conclusions: Based on the outcomes demonstrated in the ReDOS study, the dose optimization strategy of regorafenib results in favorable cost-effectiveness under a typical US willingness-to-pay threshold of $150,000 per QALY.