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AbstractAimsTo assess the cost-effectiveness of corneal collagen cross-linking (CXL) versus no CXL for keratoconus in the United States (US).MethodsA discrete-event microsimulation was developed to assess the cost-effectiveness of corneal cross-linking (CXL, Photrexa + KXL combination product) versus no CXL for patients with keratoconus. The lifetime model was conducted from a US payor perspective. The source for CXL efficacy and safety data was a 12-month randomized, open-label, sham-controlled, multi-center, pivotal trial comparing CXL versus no CXL. Other inputs were sourced from the literature. The primary outcome was the incremental cost per quality-adjusted life year gained. Costs (2019 USD) and effects were discounted 3% annually. The impacts of underlying uncertainty were evaluated by scenario, univariate, and probabilistic analyses.ResultsStarting at a mean baseline age of 31 years and considering a mixed population consisting of 80% slow-progressors and 20% fast-progressors, the CXL group was 25.9% less likely to undergo penetrating keratoplasty (PK) and spent 27.9 fewer years in advanced disease stages. CXL was dominant with lower total direct medical costs (-$8,677; $30,994 versus $39,671) and more QALYs (1.88; 21.80 versus 19.93) compared to no CXL. Considering the impact of reduced productivity loss in an exploratory scenario, CXL was associated with a lifetime cost-savings of $43,759 per patient. CXL was cost-effective within 2 years and cost-saving within 4.5 years.LimitationsLimitations include those that are common to similar pharmacoeconomic models that rely on disparate sources for inputs and extrapolation on short-term outcomes to a long-term analytical horizon.ConclusionsKeratoconus is a progressive and life-altering disease with substantial clinical, economic, and humanistic consequences. The economic value of cross-linking is maximized when applied earlier in the disease process and/or younger age, and extends to improved work productivity, out-of-pocket costs, and quality of life.

AbstractAimsTo assess from a US payer perspective the cost-effectiveness of the chimeric antigen receptor T (CAR T)-cell therapies axicabtagene ciloleucel (axi-cel) and tisagenlecleucel (tisa-cel) to treat relapsed or refractory (r/r) large B-cell lymphoma (LBCL) following ≥2 systemic therapy lines.MethodsA three-state (i.e. pre-progression, post-progression, and death) partitioned survival model was used to estimate the quality-adjusted life-years (QALYs) and costs for patients on each treatment over a lifetime horizon. Progression-free survival (PFS) and overall survival (OS) were based on a matching-adjusted indirect treatment comparison (MAIC) that accounted for differences in trial population baseline characteristics. Mixture cure models (MCMs) were used to account for long-term survivors. Costs included drug acquisition and administration for the CAR T-cell therapies and conditioning chemotherapy, apheresis, CAR T-specific monitoring, transplant, hospitalization, adverse events, routine care, and terminal care. Health state utilities were derived from trial and published data. Sensitivity analyses included probabilistic sensitivity analyses (PSAs) and an analysis of extremes that assessed the results across a vast array of combinations of parametric OS and PFS curves across the two therapies.ResultsCompared to tisa-cel, axi-cel resulted in 2.31 QALYs gained and a cost reduction of $1,407 in the base case. In the PSA, the cost per QALY gained was ≤$31,500 in 95% of the 1,000 simulations. In the analysis of extremes, the cost per QALY gained was ≤$7,500 in 99% of the 1,296 combinations of MCMs and ≤$40,000 in 95% of the 1,296 combinations of standard models.LimitationsIn absence of head-to-head comparative data, we relied on a MAIC, which cannot account for all possible confounders. Moreover, some outcomes (i.e. transplantations, hospitalizations, adverse events (AEs)) were not adjusted in the MAIC.ConclusionsIn this simulation, axi-cel was a superior treatment option as it is predicted to achieve better outcomes at lower or minimal incremental costs versus tisa-cel.

Aim:To understand physician visit patterns among patients with stage IV (including nonmetastatic [M0] and metastatic [M1] disease) urothelial carcinoma (UC) and understand factors associated with a timely referral to a medical oncologist and systemic treatment. Patients & methods:Retrospective analysis of Surveillance, Epidemiology and End Results-Medicare data. Results:First physician encounter was with a urologist (M0: 69%; M1: 53%) or primary care physician ([PCP]; M0: 19%, M1: 25%) for the majority of patients around UC diagnosis. After the index urologist encounter, most patients had a subsequent medical oncologist visit at a median of 52 days (M0: 69.5 days, M1: 33 days). In an adjusted model, older age, index PCP visit, higher comorbidities and M0 disease were negatively associated with a medical oncologist referral. Among those referred to a medical oncologist, older age, Hispanic or non-Hispanic Black race and not being married were negatively associated with subsequent chemotherapy receipt (p < 0.05). Conclusion:Many patients with advanced UC encounter multiple specialists during their disease course. Older patients or those with a first UC-related encounter with a PCP are less likely to be referred to medical oncology. Once referred to medical oncology, social determinants, including race and marital status, are relevant predictors of receiving chemotherapy.

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AbstractAims:Medicare patients with metastatic or surgically unresectable urothelial carcinoma (mUC) often receive platinum-based chemotherapy as first line of therapy (LOT), but invariably progress, requiring additional LOTs and healthcare resource use (HCRU). To better understand the evolving mUC treatment landscape, the economic burden of chemotherapy-based mUC treatments among US Medicare patients was estimated.Methods:Newly diagnosed Medicare patients with mUC were identified from the Surveillance, Epidemiology, and End Results (SEER)-Medicare database. Patients were followed from diagnosis to death, disenrollment, or end of study to characterize LOTs (first [LOT1], second [LOT2], and third or greater [LOT3+]). Kaplan-Meier methods were used to estimate overall survival (OS) by LOT. HCRU and mean costs were reported over the follow-up period, LOT duration, and maximum LOT received.Results:Among 1,873 eligible patients with mUC (median age = 77 years; median follow-up = 7.5 months), 1,035 (55%) received no chemotherapy. Among chemotherapy-treated patients, 61% had LOT1 only, 25% had LOT1 and LOT2 only, and 14% had LOT3+. Median OS was 8.1 months, range was 4.3 (untreated) to 29.8 (LOT3+) months. HCRU frequency increased with additional LOTs. Mean cumulative per-patient cost was $82,912 for all patients, increasing with additional LOTs (untreated = $57,207; LOT1 = $99,213; LOT2 = $125,190; LOT3+ = $163,884). Mean per patient per month cost was $18,827 for all patients, decreasing with increasing number of LOTs received (untreated = $27,211; LOT1 = $9,601; LOT2 = $7,325; LOT3+ = $6,017).Limitations:Potential for treatment misclassification when using the algorithm defining LOTs and non-generalizability of results to younger patients.Conclusions:Over 50% of Medicare patients with mUC received no chemotherapy. Among chemotherapy-treated patients, most received only one LOT. Additional LOTs led to higher mean costs and HCRU, but as patients were followed longer, monthly costs decreased. As treatments evolve to include immuno-oncology agents, these findings provide a clinically relevant economic benchmark for mUC treatment across different traditional LOTs.

To estimate, from a Filipino societal perspective, the cost-effectiveness of preventing atopic dermatitis (AD) via early nutritional intervention with 100% whey-based partially hydrolyzed formula (PHF-W) versus standard cow’s milk formula (SF) in healthy, urban infants with atopic heredity who are not exclusively breast-fed.

AbstractObjective:Denosumab has been approved in the US for skeletal-related event (SRE) prevention in bone-metastatic prostate cancer on the basis of a phase III clinical trial in which denosumab reduced SREs relative to zoledronic acid. Overall survival, disease progression, and serious adverse events did not differ significantly between groups. This analysis assessed the cost-effectiveness of denosumab vs zoledronic acid in bone-metastatic prostate cancer from a US payer perspective.Methods:A literature-based Markov model, wherein inputs were selected to reproduce clinical trial outcomes, was developed to estimate the survival, quality-adjusted life-years (QALYs), number and costs of SREs, and drug and administration costs for patients receiving denosumab or zoledronic acid over 27 months. QALYs were estimated by assigning health-state utilities. SRE-related costs and utilities were literature-based. Outcomes were discounted 3 per annum, and model robustness was tested via scenario, univariate, and probabilistic sensitivity analyses.Results:Denosumab resulted in fewer estimated SREs (−0.241; 1.036 vs 1.277), more QALYs (0.0074; 0.9306 vs 0.9232), and lower SRE-related costs (−2340; 8824 vs 11,164), but higher drug-related costs (10,181; 23,144 vs 12,963) and total costs (7841; 31,968 vs 24,127) vs zoledronic acid. The base case estimated cost per QALY-gained was 1,058,741.Conclusion:This analysis was limited by the restricted availability of clinical data and the need to use projection methods beyond the trial time frame. However, a wide range of scenarios predicted denosumab to have an incremental cost/QALY gained above what may be considered acceptable value for money in the US. This raises important questions regarding the pharmacoeconomic value of denosumab in bone-metastatic prostate cancer.

AbstractObjective:To assess concomitant extra-articular manifestation (EAM) rates in patients with ankylosing spondylitis (AS) treated with anti-tumor necrosis factor (anti-TNF) agents and examine the economic burden of uveitis and inflammatory bowel disease (IBD) in French and German AS patients.Methods:Previous analyses of uveitis and IBD in AS patients treated with infliximab, etanercept or adalimumab were identified in PubMed/Medline (January 2000 to August 2011). A supplemental analysis incorporated more recent adalimumab clinical trial data (ATLAS [NCT00085644] and RHAPSODY [NCT00478660]). For resource utilization/costs associated with EAMs, the search was expanded to general spondyloarthritis (SpA) conditions (i.e., AS, reactive or psoriatic arthritis, psoriatic spondylitis, IBD and undifferentiated SpA). Direct and indirect yearly costs associated with AS-associated uveitis and IBD were estimated based on interviews with French and German clinicians and literature review.Results:The pooled average rate of anterior uveitis (AU) flares for patients treated with anti-TNF therapy in two meta-analyses and supplemental adalimumab clinical trials was 4.9/100-patient-years (PYs). AU rates (per 100-PYs) were 3.4, 3.7 and 5.7 for infliximab (p  0.26 vs etanercept; p  0.86 vs adalimumab), adalimumab (p  0.033 vs etanercept) and etanercept, respectively. IBD flares (per 100-PYs) were 0.2 for infliximab (p < 0.001 vs etanercept; p  0.18 vs adalimumab), 0.63 for adalimumab (p  0.009 vs etanercept) and 2.2 for etanercept. No studies assessing EAM-associated resource utilization or costs in AS patients were found. Direct medical costs associated with IBD treatment ranged from €483 (Germany) to €6443 (France). Clinician-estimated AS-related uveitis direct medical costs were €1410 (Germany) and €1812 (France).Conclusions:Clinical data synthesis demonstrated significantly lower AU flare rates with adalimumab vs etanercept and significantly lower IBD rates with both adalimumab and infliximab vs etanercept. Economic analysis indicated substantial costs associated with AU and IBD flares secondary to AS in France and Germany. Future economic evaluations of anti-TNF agents should incorporate EAMs and subsequent treatment costs. Limitations include restricted availability of randomized, placebo-controlled clinical trial data, inclusion of data from open-label studies, lack of real-world (i.e., non-trial-based) EAM rates and a lack of EAM-specific direct and indirect costs with which to compare the results presented herein.

AbstractObjective:A 12-week clinical trial (TIMES) demonstrated that therapy with tolterodine extended release (TOL)  tamsulosin (TAM) provides clinical benefits vs TOL or TAM monotherapy or placebo (PBO) in men with lower urinary tract symptoms (LUTS) including overactive bladder (OAB). The present analysis estimated the costs and quality-adjusted life-years (QALYs) associated with these therapies from the perspective of the UK healthcare system.Methods:TIMES cohorts receiving TOL, TAM, TOL  TAM, or PBO were followed from therapy initiation to 12 weeks. A decision-tree model was used to extrapolate the 12-week results to 1 year (including need for surgery owing to treatment failure at 12 weeks) and to track patients’ outcomes (symptoms, utility, and costs). Because TIMES did not include costs and QALYs, data from the EpiLUTS epidemiologic survey (12,796 males) were used to model a mathematical relationship between LUTS (daytime and nocturnal frequency, urgency episodes, urgency urinary incontinence episodes, and International Prostate Symptom Score [IPSS]), quality-of-life, and utility. This was used to convert improvements in TIMES patients’ LUTS into utility scores and QALYs. The model included drug and surgery procedure costs and hospital length of stay.Results:Incremental QALYs of TOL  TAM vs PBO, TAM, and TOL were 0.042, 0.021, and 0.013, and corresponding incremental costs were £189, £223, and −£70, respectively, resulting in cost-utility ratios for TOL  TAM of £4508/QALY gained compared with PBO and £10,381/QALY gained compared with TAM. TOL  TAM combination therapy was both more effective and cost-saving compared with TOL. Univariate sensitivity analyses showed that patient utility was most responsive to changes in drug efficacy on IPSS and urgency episodes. Changing the percentage of patients undergoing surgery did not substantially affect model outcomes. The main limitation of the study was that the relation between LUTS and patient utility was based on an indirect association.Conclusions:TOL  TAM combination therapy appears to be cost-effective compared with TOL or TAM monotherapy or PBO in male patients with LUTS.

To assess the cost-effectiveness of treatment with eszopiclone for chronic primary insomnia in adults.

Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL) is a rare, high-risk, aggressive form of acute leukemia, affecting primarily adults and the elderly. Patients with high-risk forms of ALL typically have extremely poor prognosis and incur high disease-related costs. Successful use of imatinib in patients with Ph+ chronic myelogenous leukemia (CML) has led to the administration of imatinib in recent clinical studies for patients with Ph+ALL. In this review, the clinical outcomes of imatinib used as a single-agent or in combination with chemotherapy are discussed. In addition, an overview of the economic and quality of life burden associated with high-risk forms of ALL (with Ph+ALL being the most common high risk ALL), as well as the cost-effectiveness of imatinib in Ph+ALL compared with conventional chemotherapy, are presented.

Severe hemophilia with inhibitors is a rare disease with substantial clinical, humanistic and economic consequences. This review provides an overview of the role of recombinant activated factor VII (rFVIIa) versus plasma-derived bypassing agents for hemophilia with inhibitors and summarizes the 13 formal economic analyses (6 burden of illness and 7 comparative studies) that have been published in this indication. The findings suggest that the economic impact of rFVIIa has occurred primarily during hospitalization to manage major bleeding episodes and to allow for elective orthopedic surgeries that would not have been attempted prior to rFVIIa. Comparative analyses for on-demand treatment suggest that the total cost of treating a bleeding episode with rFVIIa may be lower than with plasma-based agents due to faster bleeding resolution, higher initial efficacy rates and avoidance of second and third lines of treatment.

ABSTRACTObjective: To compare the cost-effectiveness of three treatment regimens using recombinant activated Factor VII (rFVIIa), NovoSeven†, and activated prothrombin-complex concentrate (APCC), FEIBA VH‡, for home treatment of minor-to-moderate bleeds in hemophilia patients with inhibitors. †NovoSeven is a registered trademark of Novo Nordisk A/S, Bagsværd, Denmark‡FEIBA VH is a registered trademark of Baxter, Deerfield, Illinois, USAMethods:A literature-based, decision-analytic model was developed to compare three treatment regimens. The regimens consisting of first-, second-, and third-line treatments were: rFVIIa-rFVIIa-rFVIIa; APCC-rFVIIa-rFVIIa; and APCC-APCC-rFVIIa. Patients not responding to first-line treatment were administered second-line treatment, and those failing second-line received third-line treatment. Using literature and expert opinion, the model structure and base-case inputs were adapted to the US from a previously published analysis. The percentage of evaluable bleeds controlled with rFVIIa and APCC were obtained from published literature. Drug costs (2005 US$) based on average wholesale price were included in the base-case model. Univariate and probabilistic sensitivity analyses (second-order Monte Carlo simulation) were conducted by varying the efficacy, re-bleeding rates, patient weight, and dosing to ascertain robustness of the model.Results:In the base-case analysis, the average cost per resolved bleed using rFVIIa as first-, second-, and third-line treatment was $28 076. Using APCC as first-line and rFVIIa as second- and third-line treatment resulted in an average cost per resolved bleed of $30 883, whereas the regimen using APCC as first- and second-line, and rFVIIa as third-line treatment was the most expensive, with an average cost per resolved bleed of $32 150. Cost offsets occurred for the rFVIIa-only regimen through avoidance of second and third lines of treatment. In probabilistic sensitivity analyses, the rFVIIa-only strategy was the least expensive strategy more than 68% of the time.Conclusions:The management of minor-to-moderate bleeds extends beyond the initial line of treatment, and should include the economic impact of re-bleeding and failures over multiple lines of treatment. In the majority of cases, the rFVIIa-only regimen appears to be a less expensive treatment option in inhibitor patients with minor-to-moderate bleeds over three lines of treatment.

ABSTRACTObjective:To quantify the incidence and assess the risk of needlestick injury (NI) in nurses caring for patients with diabetes mellitus.Methods:A total of 400 nurses caring for patients with diabetes in 381 different hospitals throughout the United States over a period of at least 1 year voluntarily completed an Internet-based data collection instrument. The nurses self-reported comprehensive data on their experience with NI, focusing on those occurring within the past year. If respondents experienced multiple NI during this period, detailed data were collected on the most recent event.Results:Of the 400 nurses, 313 (78.3%) reported experiencing at least one NI, 110 (27.5%) reported at least one NI within the last 12 months, and 44 (40% of 110) reported multiple NI. Nearly two-thirds of these injuries (n= 73/110; 66.4%) were punctures that drew blood, resulting in one case of contracted hepatitis C. The cumulative annual incidence of NI events was 448 NI per 1000 nurses. Nurses reported the injury in adherence with existing regulations and policies in only 21.8% of the cases. Disposable syringes were involved in 88 (80%) of the NI events. In half of the injuries (n= 55), the needled device was equipped with a safety feature that was ineffective, primarily because it was not fully activated (n= 47/55; 85.5%) or it malfunctioned (n= 2–5; 3.6–9.1%). NI most commonly occurred while nurses were injecting insulin (n= 33; 30%). In the 2 weeks following their NI, 60.1% of nurses noted that they were more afraid of needled devices than before the injury and 41.8% felt anxious, depressed, or stressed. As a direct result of the NI, nurses missed 77 days of work.Conclusions:This study is the first to show the relatively high risk both of NI and of NI that draws blood among nurses injecting insulin with a disposable syringe and confirms previous incidence estimates of NI among nurses. Additionally, this study reveals significant post-NI emotional distress, suggests significant under-reporting of NI to hospital officials, and demonstrates the need for a more effective needle safety device.

SUMMARYAllergic rhinitis can be a debilitating condition which, if untreated, can result in considerable health-related and economic consequences. A review of the published literature was conducted, with quantitative/qualitative analysis as appropriate, to explore the direct, indirect, and hidden costs of allergic rhinitis, as well as the quality-of-life burdens that the disease presents to patients and to the healthcare system. Lack of treatment, undertreatment, or nonadherenceto treatment in allergic rhinitis were seen to increase direct and indirect costs, reinforcing the need for patient education and for physicians to implement existing evidence-based guidelines for prevention and treatment. It was concluded that greater awareness of the total economic burden of allergic rhinitis should encourage appropriate intervention and ultimately ensure clinically favorable and cost-effective outcomes.

Given the recent advances in the treatment of hematologic malignancies and the many other treatments on the horizon, physicians and payers will be faced with the critical decisions of when to use new treatments in the clinical pathway and how to allocate healthcare resources. This review will provide an overall context for the clinical, economic and quality of life burden of leukemia, as well as provide cross-analysis among the four major types of leukemia: acute lymphocytic leukemia, chronic lymphocytic leukemia, acute myeloid leukemia and chronic myeloid leukemia.

Objectives: Severe hemophilia patients experience frequent spontaneous joint hemorrhages, which lead to severe pain and swelling, reduced flexion and function, and ultimately debilitating arthropathy. Elective major orthopedic surgery (e.g., total joint replacement) in patients with advanced arthropathy is increasingly considered to reduce pain, improve function, and reduce joint bleeds. This analysis reviewed the impact of surgery on quality of life and other humanistic outcomes in hemophilia patients with advanced arthropathy. Materials and Methods: A systematic search of the published literature was conducted to identify studies reporting on the humanistic and patient-related outcomes of major orthopedic surgery in patients with hemophilia and advanced arthropathy. Studies were included if they reported quality of life (QOL), pain, functional status and work and productivity outcomes. Wherever possible, data were extracted and normalized across various scoring systems (WOMAC, WFH, HSS, Cleveland Rating, and Knee Society) for comparisons across studies. Results: A total of 32 studies were examined. Among those, only one included a formal, validated QOL instrument (WOMAC) with pre/post surgery data. This study documented dramatic improvements in objective and patient perceived QOL measures. Of the other 31 studies, 4 provided sufficient pre/post surgery data to facilitate a quantitative assessment of other key humanistic and patient-related outcomes. In 50 patients pooled across these four studies, all (100%) had moderate or severe pain in the targeted joint prior to surgery. After surgery 86% (43/50) reported no pain at follow up. Improvements were also reported regarding functional status, where 100% of 16 cases pooled across two studies had severe or moderate loss of function prior to joint surgery and 31% (5/16) regained full function upon follow up. Similar, if not better, results were reported in studies that reported only average scores across pain and function parameters. It was found that joint pain was the most prevalent complaint prior to surgery and was the primary indication for surgery. Anecdotal evidence from the 32 studies indicates that improvements in pain and function following surgery were so significant in some patients that they were able to rejoin the workforce and take part in recreational activities, including sports. There were several reports of patients who were unable to walk long distances or were confined to wheelchairs before surgery who became able to walk several blocks unaided following surgery. Conclusions: Major elective orthopedic surgery in hemophilia patients with advanced arthropathy is one of the most effective medical interventions, with success rates in terms of pain reduction over 80%. However, the broader impact of surgery on QOL outcomes, which appears excellent, remains to be better documented. To that effect, the systematic use of a set of validated instruments to be administered before and after surgery is recommended to help document the outcomes of these surgeries.

Objectives: Severe hemophilia patients experience frequent spontaneous joint hemorrhages, which lead to severe pain and swelling, reduced flexion and function, and ultimately debilitating arthropathy. Elective major orthopedic surgery (e.g., total joint replacement) in patients with advanced arthropathy is increasingly considered to reduce pain, improve function, and reduce joint bleeds. This analysis reviewed the impact of surgery on quality of life and other humanistic outcomes in hemophilia patients with advanced arthropathy.

OBJECTIVE: Severe hemophilia with inhibitors is a rare disease with substantial clinical, humanistic, and economic consequences. Within the past few years, a number of formal economic analyses have emerged which examined the cost of treating bleeding episodes with recombinant activated factor VII (rFVIIa) versus plasma-derived agents. Thus the objective was to review the recent health economic analyses of rFVIIa in the management of patients with hemophilia and inhibitors. DATA SOURCES: Published, English-language medical literature on the economics of rFVIIa was searched from January 1996 through July 2006 using: the MEDLINE/PubMed database, PubMed related articles feature, company knowledge of published medical literature for rFVIIa that was not indexed by MEDLINE/PubMed, and a thorough review of retrieved article bibliographies. STUDY SELECTION AND DATA ABSTRACTION: Abstracts selected for full article retrieval/review were those that specifically mentioned (or implied that the full article would address) cost impact, cost of treatment, or cost-effectiveness for rFVIIa in patients with hemophilia and inhibitors. Of 70 abstracts reviewed, 30 articles were selected for retrieval, and from those, thirteen economic analyses (6 burden of illness and 7 comparative studies) met inclusion criteria for data abstraction and synthesis. Seventeen articles were excluded for the following reasons: reports of basic drug acquisition costs without formal economic analysis, single case reports, studies without inhibitor patients, or studies of immune tolerance therapy. DATA SYNTHESIS: The economic impact of rFVIIa on hospitals and treatment centers occurs primarily during hospitalization to manage major bleeding episodes and allow for elective orthopedic surgeries that would not have been attempted without availability of rFVIIa. Six out of seven comparative analyses for on-demand treatment suggest that total cost of treating a bleeding episode with rFVIIa may be lower than that of using plasma-based agents due to faster bleeding resolution, higher initial efficacy rates, and avoidance of second and third lines of treatment. Dosing assumptions for the various agents compared in the economic analyses were the most sensitive variables. CONCLUSIONS: The currently available literature suggests that rFVIIa is a cost-effective treatment option and may ultimately lead to improvement in clinical outcomes for patients with hemophilia and inhibitors.

OBJECTIVES: Pleural and pericardial effusions can lead to severe outcomes. Cancer accounts for an estimated 40% of all pleural effusions. About half of the effusions diagnosed in cancer patients are malignant, while the rest are nonmalignant and may occur as complications of the cancer treatments themselves. Pleural and pericardial effusions are associated with increased morbidity and mortality as well as high healthcare costs. The objective of this study was to review the economic burden of pleural and pericardial effusions in cancer patients. METHODS: A systematic search of the English-language medical literature published between 1990 and 2006 was conducted. Additional publications and conference proceedings were retrieved from the article bibliographies and included in the review. Articles selected include prospective or retrospective studies specifically designed to examine burden of illness, direct medical costs, indirect costs, or cost drivers associated with pleural or pericardial effusions in cancer patients. All original costs were reported, with adjusted figures (to 2006 US dollars) presented in parentheses using the medical care component of the consumer price index from the US Bureau of Labor Statistics. RESULTS: Of 15 studies identified, 11 met selection criteria and were reviewed in detail. Seven references reported data on costs associated with pleural or pericardial effusions in cancer patients. The cost per episode of pleural effusion ranged from $3,391 (2006 US $4,387) for outpatient treatment with pleural catheter to $20,996 ($37,341) for talc pleurodesis. The most common treatment for malignant pleural effusion is chest tube insertion and drainage with instillation of a sclerosing agent. Key cost drivers for significant pleural effusions included operating room costs, surgeon fees, and drugs such as sclerosing agents. Resources used for management of low grade pleural effusions include chest x-rays, physician outpatient visits, diuretics, and corticosteroids. For the treatment of pericardial effusion, the costs of performing pericardiocentesis and a pericardial window procedure were estimated to be $4,446 and $14,641 (2006 US$), respectively. Cost components for pericardial effusions, depending on treatment modality selected, included echocardiogram (3–10%), intensive care unit (17–56%), sclerosant (1–4%), surgeon fees (28–29%), anesthesia fees (20%), and operating room costs (31%). CONCLUSIONS: Pleural and pericardial effusions lead to significant direct medical costs, contributing to the total cost of care among patients treated for cancer. These costs should be included in the economic evaluation of therapies that increase the risk of pleural and pericardial effusions. Given the scarcity of published analyses in this area, additional research is warranted to better understand the burden of pleural and pericardial effusions.

OBJECTIVES: With a greater focus on costs in today’s health care markets, it is important to evaluate overall costs and quality of care associated with new therapies in cost-effectiveness analyses. Adverse events (AEs) associated with cancer therapies often result in substantial economic costs and negative impacts on patient quality of life. AEs may also affect adherence to therapy and result in suboptimal treatment outcomes. Important AEs of dasatinib in chronic myelogenous leukemia (CML) patients who are resistant to imatinib include neutropenia, thrombocytopenia, anemia, gastrointestinal hemorrhage, central nervous system hemorrhage, and fluid retention leading to effusions and heart dysfunction. This study evaluated the total cost of treatment with dasatinib in patients with chronic, accelerated, and myeloid blast phase CML, taking into account both drug and AE-associated costs. METHODS: A literature-based economic model was developed to examine the short-term potential impact of AEs in patients with CML treated with dasatinib. The incidence rates of Grade 3/4 AEs were obtained from the SPRYCEL™ (dasatinib) package insert. The AEs were assumed to occur within the first 6 months of treatment. The costs associated with the AEs were obtained from published medical literature and the 2003 Healthcare Cost and Utilization Project database of mean hospital charges by principal diagnosis, converting to costs using a Medicare cost-to-charge ratio of 0.55. Dasatinib dosing assumptions were based on the FDA briefing documents of the SPRYCEL™ filing submission presented at the June 2, 2006 Oncology Drug Advisory Committee meeting. The initial dose for dasatinib was 70 mg twice daily based on the package insert. Patients were assumed to remain on treatment for 6 months. However, dose interruption of 7–14 days during the first 3 months of treatment and dose reduction to 50 mg twice daily in the second 3 months of treatment were assumed in 46–55% and 9–10% of patients, respectively. Drug costs were based on wholesale acquisition cost. Univariate and multivariate sensitivity analyses were conducted. All costs were adjusted to 2006 U.S. dollars using the medical care component of the consumer price index. RESULTS: In the base case analysis, the expected 6-month per-patient cost of treatment for dasatinib was $31,528 for chronic phase, $44,340 for accelerated phase, and $48,666 for myeloid blast phase. Six-month drug cost alone was $22,284 per patient in each phase. Grade 3/4 AEs accounted for approximately 29%, 50%, and 54% of the total cost of care for chronic, accelerated, and myeloid blast phases, respectively. Sensitivity analysis showed that the 6-month cost of Grade 3/4 AEs may range from $5,235–$10,696 for chronic, $16,109–$23,723 for accelerated, and $12,971–$27,551 for myeloid blast phases. Primary cost drivers were the hematologic AEs. CONCLUSIONS: This study provides an estimate for the total cost of treatment with dasatinib that considers treatment costs for severe AEs associated with therapy. These results highlight the importance of including treatment-related AEs in addition to drug costs when assessing the economic value of treatment options for CML. Future studies should expand the time horizon of the analysis and include recurring AEs to more comprehensively evaluate the overall economic impact of AEs with dasatinib.

OBJECTIVES: Anemia is one of the most common hematologic adverse events experienced by cancer patients. The incidence of chemotherapy-induced anemia is as high as 100% for Grade 1 to 2 and 80% for Grade 3 to 4 anemia, although it varies depending on the cancer type and treatment regimen administered. Anemia is associated with high healthcare costs as well as decreased quality of life. The objective of this study was to review the economic and quality of life (QoL) outcomes of anemia in cancer patients. METHODS: A systematic search of the English-language literature published between 1990 and 2006 was conducted. Additional publications and conference proceedings were retrieved from the article bibliographies and included in the review. Articles selected include prospective or retrospective studies specifically designed to examine burden of illness, direct medical costs, indirect costs, cost drivers, or quality of life outcomes associated with chemotherapy-induced anemia in adult cancer patients. All original costs were reported, with adjusted figures (to 2006 dollars) presented in parentheses using the medical care component of the consumer price index from the US Bureau of Labor Statistics. RESULTS: Of 183 abstracts screened, 36 met selection criteria and were reviewed in detail. Fifteen and 11 studies focused on economics and QoL burden associated with anemia, respectively. The average annual cost attributable to anemia ranges from $18,418 (2006 US$ $22,775) to $69,478 ($93,454) per patient. Inpatient and outpatient services account for a significant portion of the total costs of anemia. Other key cost drivers for anemia include the cost of erythropoietic drugs, which range from approximately $210 ($239) to $1,090 ($1,288) per week, and the cost of red blood cell (RBC) transfusions, which range from $232 ($323) to $512 ($712) per unit. Patients with hematologic malignancies require almost double the number of RBC units and have higher associated costs than patients with solid tumors. Indirect costs include lost work time due to fatigue for an average of 4.2 sick/vacation days per month. In addition to raising the cost of care, anemia affects patient QoL in the following key domains: energy/fatigue, role (ability to work), social function, and leisure activities. Furthermore, some patients may report daily fatigue, overall decreasing everyday QoL. CONCLUSIONS: Anemia is a common hematologic adverse event experienced by cancer patients, resulting in significant health care costs, indirect costs, and decrements in patient QoL. In particular, patients with hematologic malignancies incur higher anemia-associated costs compared to patients with solid tumors. The economic burden of cancer treatment-induced anemia, as well as the impact on patient QoL, should be considered when making clinical decisions regarding the optimal treatment course for patients with cancer.

OBJECTIVES: Hematologic adverse events such as neutropenia, thrombocytopenia, and anemia are commonly experienced by cancer patients receiving chemotherapy. These cytopenias are associated with increased morbidity and mortality, high healthcare costs, and decreases in quality of life. The objective of this study was to review the economic burden of these hematologic adverse events associated with chemotherapy in cancer patients. METHODS: A systematic search of the English-language medical literature published between 1990 and 2006 was conducted. Online conference proceedings and a review of article bibliographies were included in the review. Articles selected included prospective or retrospective studies specifically designed to examine burden of illness, direct medical costs, indirect costs, or cost drivers associated with neutropenia, thrombocytopenia, and anemia in adult cancer patients treated with chemotherapy. All original costs were reported, with adjusted figures (to 2006 US dollars) presented in parentheses using the medical care component of the consumer price index from the US Bureau of Labor Statistics. RESULTS: Of 160 studies initially identified, 64 met selection criteria and were reviewed in detail. The cost of neutropenia ranged from $1,893 (2006 US $2,632) per episode in the outpatient setting to $38,583 ($54,807) for a febrile neutropenia hospitalization. The cost of treating thrombocytopenia ranged from $1,037 ($1,395) to $7,550 ($9,336) per cycle or episode. Costs attributable to treating anemia ranged from $18,418 ($22,775) to $69,478 ($93,454) per year. Key cost drivers include hospitalization, drugs (e.g., granulocyte colony-stimulating factors and antibiotics), and diagnostic tests for neutropenia; hospitalization, major bleeding episodes, and platelet transfusions for thrombocytopenia; and inpatient and outpatient services, erythropoietic agents, and red blood cell transfusions for anemia. Another finding was that the costs of hematologic adverse events for patients with hematologic malignancies were up to twice that of patients with solid tumors. CONCLUSIONS: Chemotherapy-related cytopenias result in a substantial economic burden on patients, payers, caregivers, and society in general. This burden is particularly high for patients with hematologic malignancies due to the underlying malignancy. Furthermore, AEs affect the ability to deliver planned treatments, resulting in potentially suboptimal clinical outcomes. An evaluation of both clinical outcomes of chemotherapy and economic consequences as a result of chemotherapy-induced toxicities is recommended in determining optimal treatments for patients with cancer.

OBJECTIVE: Severe hemophilia with inhibitors is a rare disease with substantial clinical, humanistic, and economic consequences. Within the past few years, a number of formal economic analyses have emerged which examined the cost of treating bleeding episodes with recombinant activated factor VII (rFVIIa) versus plasma-derived agents. Thus the objective was to review the recent health economic analyses of rFVIIa in the management of patients with hemophilia and inhibitors.

OBJECTIVES: Anemia is one of the most common hematologic adverse events experienced by cancer patients. The incidence of chemotherapy-induced anemia is as high as 100% for Grade 1 to 2 and 80% for Grade 3 to 4 anemia, although it varies depending on the cancer type and treatment regimen administered. Anemia is associated with high healthcare costs as well as decreased quality of life. The objective of this study was to review the economic and quality of life (QoL) outcomes of anemia in cancer patients.

OBJECTIVES: Hematologic adverse events such as neutropenia, thrombocytopenia, and anemia are commonly experienced by cancer patients receiving chemotherapy. These cytopenias are associated with increased morbidity and mortality, high healthcare costs, and decreases in quality of life. The objective of this study was to review the economic burden of these hematologic adverse events associated with chemotherapy in cancer patients.

OBJECTIVES: With a greater focus on costs in today's health care markets, it is important to evaluate overall costs and quality of care associated with new therapies in cost-effectiveness analyses. Adverse events (AEs) associated with cancer therapies often result in substantial economic costs and negative impacts on patient quality of life. AEs may also affect adherence to therapy and result in suboptimal treatment outcomes. Important AEs of dasatinib in chronic myelogenous leukemia (CML) patients who are resistant to imatinib include neutropenia, thrombocytopenia, anemia, gastrointestinal hemorrhage, central nervous system hemorrhage, and fluid retention leading to effusions and heart dysfunction. This study evaluated the total cost of treatment with dasatinib in patients with chronic, accelerated, and myeloid blast phase CML, taking into account both drug and AE-associated costs.