A systematic literature review (SLR) was conducted to identify utilities in acute myeloid leukaemia (AML). SLR methods conformed to National Institute for Health and Care Excellence technology appraisal requirements. Eligible studies were economic evaluations (cost-effectiveness, cost-utility and cost-benefit analyses), utility studies, SLRs and health technology assessments. Nine bibliographic databases and 4 conferences were searched (November 2018) and retrieved 2,278 unique records. Two reviewers independently selected records and one extracted data. Twenty-six studies (28 documents) met inclusion criteria. Data were derived using: EQ-5D (14 studies, including 2 using EQ-5D-5L), TTO (4), VAS (4), DCE (2), HUI2 (1), QLQ-PBM (1), EORTC-8D (1), standard gamble (1), as well as proxy data (1) and data mapped to utility (1, from QLQ-C30). Studies were conducted in Canada, Japan, the Netherlands, UK, USA, and one study was across Europe. Mean utility data were identified for these health states: induction chemotherapy (-0.15 to 0.706), consolidation chemotherapy (-0.11 to 0.71), maintenance (0.81 to 0.95 [median]), complete remission (0.62 to 0.99) [in transplant: 0.61 to 0.71; prior to transplant: 0.826], no relapse (0.83 to 0.90 [median]), temporary remission (0.66), partial remission (0.6574 to 0.7160), relapse (0.1 to 0.79), refractory (-0.1 to 0.568) and transplant (-0.21 to 0.94) [short-term: 0.4; recovery: 0.75 to 0.826; graft versus host disease (GVHD): 0.37 to 0.691; without GVHD: 0.79 to 0.864; long term: 0.94 to 1 [median]]. Disutilities were reported for chemotherapy (0.42) and transplant (0.57). The SLR reported wide variations in utility values across AML health states, with most studies referring to first line treatment with induction/consolidation rather than relapse/refractory. Variations could be due to underlying disease activity within health states, differences utility elicitation methods, and health state definitions. Choice of utility values for decision analytic models should consider these differences to improve validity.

Purpose In advanced non-small cell lung cancer (NSCLC), progressive disease burdens patients considerably. Second-line (2L) chemotherapy improves survival marginally but humanistic outcomes (i.e., quality of life, QOL) are underreported. The impact of 2L therapy remains an important consideration for patients and caregivers, and there have been QOL reviews for 1L, but not 2L, therapies. This review assessed QOL outcomes of approved, guideline-supported 2L chemotherapy with docetaxel, erlotinib, gefitinib, and pemetrexed in advanced NSCLC. Methods Clinical trial reports of approved, guideline-supported 2L or maintenance therapy for NSCLC published from 2000 to 2010 were identified from PubMed/Medline and clinical meetings. Outcomes were stratified by overall QOL impact, domain/symptom-specific effects, effect over time, and subgroup effects. Results Of 145 studies identified, 24 full-text articles were retained. Studies with docetaxel versus best supportive care ( n = 1) and active comparators ( n = 4) reported non-significant overall QOL improvements, as did studies of gefitinib versus placebo and active comparator ( n = 7). Overall QOL improvements were seen for gefitinib versus docetaxel ( n = 2) and gefitinib in a single-arm study ( n = 1). At the symptom level, studies of docetaxel ( n = 4/7), gefitinib ( n = 7/9), and pemetrexed ( n = 1) reported non-significant results. Subgroup analyses indicated improved QOL outcomes for gefitinib-treated responders versus non-responders, worse QOL for gefitinib-treated smokers versus placebo, worse QOL for gefitinib-treated Asian patients versus placebo, and longer time to symptom deterioration in erlotinib versus placebo-treated elderly patients. Conclusions Significant improvements in overall QOL with 2L chemotherapy for advanced NSCLC were infrequent. Single-arm studies and those with less toxic regimens more commonly provided statistically significant improvements in QOL outcomes. Methodological heterogeneity impedes cross-study QOL comparisons.

Brain metastases are a frequent complication of many systemic cancers and portend a poor prognosis. This retrospective analysis of health claims data compared survival, treatment and health care utilization and costs in patients with brain metastasis by primary tumor site. Adult commercial and Medicare Advantage enrollees newly diagnosed with brain metastasis in 01 Jan 2004 through 30 Apr 2010 were identified. Inclusion required at least 2 claims that identified the same primary cancer site prior to diagnosis of brain metastasis and no evidence of primary brain tumors. Health care utilization rates and costs were calculated at the patient level for each month of follow-up. Differences among primary cancer site cohorts were assessed by ANOVA (continuous variables), Chi square test (proportions) and the Poisson distribution (utilization rates). The primary cancer cohorts comprised 1,031 lung cancer, 93 melanoma and 395 female breast cancer patients. During the 6 months prior to brain metastasis diagnosis, 59 % of lung cancer patients had no evidence of lymph node involvement or other metastatic disease compared to 55 and 42 % of melanoma and breast cancer patients (P < 0.001). Survival after brain metastasis diagnosis was less than 3 months for 52, 43 and 39 % for lung cancer, breast cancer and melanoma, respectively (P < 0.001). Melanoma patients had the highest rate of inpatient stays and outpatient visits (P ≤ 0.003). Total monthly all-cause costs were: melanoma, $23,426; breast cancer $19,708; lung cancer, $17,007 (P = 0.003). Health care utilization and costs after brain metastasis diagnosis were substantial and differed by primary tumor site.

Aim: To evaluate the disease-specific expenditures incurred by colorectal cancer (CRC) patients with one, two, and three or more lines of therapy. Methods: The Truven Health MarketScan (R) Research Databases were used to identify adults with incident CRC from 2005 to 2009. Healthcare expenditures were measured from initiation through to follow-up and individual therapy line. Results: Among 13,670 CRC patients, 67.5% had one line of therapy, 20.7% had two and 11.8% had three or more. Monthly expenditures averaged US$ 12,523. Monthly first-line therapy costs averaged US$ 12,067, increasing to US$ 13,315 during second-line therapy and to US$ 14,648 during third-line therapy. Patient out-of-pocket expenses were a small (1.9%) contributor to total costs. Conclusion: CRC presents a significant economic burden to payers with important cost differences according to how patients are treated.

AIM:Metastatic breast cancer guidelines contain multiple lines of treatment and regimens; however, little data on therapeutic patterns and costs is available from real-world clinical practice. This descriptive study reports chemotherapy and biologic use, healthcare utilization and costs by line of therapy in a large insured US population. MATERIALS & METHODS:Adult women with newly diagnosed metastatic breast cancer (between 2005 and 2009) were identified from MarketScan® databases containing medical and pharmacy claims of >40 million enrollees insured with >100 US health plans. Descriptive statistics were reported for use, duration and mean per patient per month costs across four lines of therapy. RESULTS:Out of 7767 patients identified (mean [standard deviation] age = 58.2 [12] years), ≥50% received a subsequent line of therapy across the four lines (line 2: n = 4077; line 3: n = 2033; line four: n = 1059). The top two chemotherapies were paclitaxel and capecitabine in lines one and two, and paclitaxel and gemcitabine in lines three and four. The top two biologics were trastuzumab and bevacizumab across the multiple lines of treatments. Duration (mean, standard deviation and median days) varied across multiple lines of treatments: 162.7, 176.9 and 108.0 in line one; 147.5, 146.7 and 99.0 in line two; 139.9, 131.1 and 99.0 in line three; and 130.9, 123.4 and 94.0 in line four, respectively. Mean per patient per month costs decreased with increasing follow-up from US$13,147 (<6 months) to US$11,610 (7-12 months) to US$10,219 (12-24 months) to US$9,192 (24-36 months) to US$7,384 (>36 months). Cumulative costs increased with follow-up, from US$78,882 (<6 months) to US$443,062 (>36 months). CONCLUSION:Longer follow-up, regardless of number of lines of therapy, was associated with higher cumulative, but lower monthly, costs. Delaying progression and improving survival with more individualized treatment regimens may help slow the rate of increasing long-term costs of metastatic breast cancer treatment and care.

Patient-reported outcomes (PRO) instruments currently used for patients with brain metastases (BM) have not been developed with adequate patient input from the appropriate population. The objective of this study was to develop a new PRO in this population. The BASIQ was developed according to the FDA PRO Guidance. A literature review, seven expert interviews, and 19 in-depth interviews with BM patients were conducted to identify the symptoms and impacts of BM important to this population and generate an initial version of the BASIQ. Twenty face-to-face cognitive interviews (CIs) were conducted to assess the content validity of the BASIQ and to assess the understandability, relevance, wording, and importance of items and, if necessary, revise it. The initial 23-item BASIQ included a 7-item event log with a yes/no response (assessing vision, reading, nausea, numbness, needing to stay in bed, falling, fainting), 7-item daily symptom section (assessing severity of headache, memory, balance, physical weakness, dizziness, tiredness, energy) on an 11-point NRS, and a 9-item impact section (assessing speaking certain words, putting ideas into words, staying focused on a topic, walking, understanding words read/heard, following a story in a book/on TV, doing things around the house, bathing, dressing). During the CIs, most of the patients reported that the instrument was easy to understand, of adequate length and format and did not contain any difficult words. Based on the CIs, items were deleted, modified, or included as impacts rather than symptoms. A revised 18-item BASIQ used a 24-hour recall period and an 11-point NRS assessing severity. Robust qualitative methods used to identify the symptoms and impacts of patients with BM led to the development of a much needed PRO measure in BM. Additional qualitative and quantitative research is planned to further support the use of the tool in clinical research.

OBJECTIVE:Although utility-based algorithms have been developed for the Functional Assessment of Cancer Therapy (FACT), their properties are not well known compared with those of generic utility measures such as the EQ-5D. Our objective was to compare EQ-5D and FACT preference-based scores in cancer patients.METHODS:A retrospective analysis was conducted on cross-sectional data collected from 472 cancer patients who completed both FACT-General and the EQ-5D. Preference-based scores were calculated by using published scoring functions for the EQ-5D (Dolan P. Modeling valuations for EuroQol health states. Med Care 1997;35:1095-108; Shaw JW, Johnson JA, Coons SJ. US valuation of the EQ-5D health states: development and testing of the D1 valuation model. Med Care 2005;43:203-20) and FACT (Dobrez D, Cella D, Pickard AS, et al. Estimation of patient preference-based utility weights from the Functional Assessment of Cancer Therapy-General. Value Health 2007;10:266-72; Kind P, Macran S. Eliciting social preference weights for Functional Assessment of Cancer Therapy-Lung health states. Pharmacoeconomics 2005;23:1143-53; Cheung YB, Thumboo J, Gao F, et al. Mapping the English and Chinese versions of the Functional Assessment of Cancer Therapy-General to the EQ-5D utility index. Value Health 2009;12:371-6). Scores were compared on the basis of clinical severity by using Eastern Cooperative Oncology Group performance status ratings by physicians and patients. Relative efficiency of each scoring function was examined by using ratios of F statistics.RESULTS:Mean scores for the overall cohort were lowest when using Kind and Macran's FACT UK societal algorithm (0.55, SD 0.09) and highest when using Dobrez et al.'s FACT US patient algorithm (0.83, SD 0.08). Mean difference scores associated with clinical severity, when extrapolated to quality-adjusted life-years (QALYs), had a range of 0.18 QALYs gained using FACT (Kind and Macran) to 0.45 QALYs gained using the EQ-5D (Dolan). However, relative efficiencies suggested that FACT (Kind and Macran) scores may provide greater statistical power to detect significant differences based on clinical severity.CONCLUSIONS:We found important differences in utilities scores estimated by each algorithm, with FACT-based algorithms tending to underestimate the QALY benefit compared with algorithms based on the EQ-5D. These differences highlight some of the challenges in using disease-specific preference-based measures for decision making despite potentially more relevant disease-specific content.

584 Background: Colorectal cancer patients with EGFRI therapies may develop dermatologic ADRs. ADRs negatively affect patients’ quality of life, require medical care, and may lead to a substantial economic burden. The objective of this study is to assess the economic burden of dermatologic ADRs in patients with colorectal cancer (CRC). Methods: Continuously enrolled adult patients with ≥ 1 CRC diagnosis initiated on an FDA-approved EGFRI therapy (i.e., cetuximab, panitumumab) were selected from a large US claims dataset. Patients were classified into two mutually exclusive cohorts depending on whether or not they had dermatologic ADR during the period following EGFRI initiation. Dermatologic ADRs were identified using ICD-9-CM codes. Patients were observed from the index date up to the end of the health plan enrollment, or data availability, or 90 days after the EGFRI discontinuation, whichever occurred first. Incidence rate ratios (IRRs) for healthcare resource utilization were estimated using Poisson regression models. Incremental costs (2010 USD) were estimated on a monthly basis using generalized linear or two-part models. Multivariate regressions controlled for age, gender, comorbidities, and baseline healthcare utilization and costs. Results: A total of 746 and 1,720 patients were included in the dermatologic ADR and ADR-free cohorts, respectively. After adjusting for confounding factors, compared to patients in the ADR-free cohort, patients in the ADR cohort had higher incidence of emergency room visits (IRR=1.2; p=.009) and higher incidence of outpatient visits (IRR=1.13; p<.001). Moreover, patients in the ADR cohort incurred higher total healthcare costs by $2,532 (p=.009) per patient per month mainly due to higher emergency care costs by $566 (p<.001). Conclusions: Compared to ADR-free patients, dermatologic ADR patients with colorectal cancer incurred a substantial economic burden related to higher healthcare resource utilization and costs. Efforts to improve the dermatologic side effect profile of EGFRI are needed to control this burden.

e12515 Background: Increasingly, there has been a public call for direct patient input into the development of patient reported outcome (PRO) measures of treatment benefit in adult oncology clinical trials. Patient involvement is especially important in conditions where there is poor prognosis and comparative treatment benefit is minimal with standard outcome measures such as overall or progression free survival. To develop a PRO instrument specifically for patients with brain metastases (BM), this study interviewed patients in this population and also reviewed the clinical literature to identify the signs and symptoms relevant to BM. Methods: A literature search was performed inMEDLINE for articles published between 2000 and 2010. Individual in-depth interviews were conducted with 19 patients with BM using open-ended questions to elicit the most frequent and the most bothersome symptoms. Patient inclusion criteria included a diagnosis of BM and being eligible for whole brain radiotherapy. Results: The literature review identified the following symptoms as important: intracranial pressure symptoms (e.g., headache, nausea, vomiting), seizures, and focal neurological symptoms (e.g., paralysis, mental status changes, memory, motor loss/weakness, visual loss/changes). The most frequent symptoms reported by patients in the interviews were: headache (74%), memory loss (63%), vision problems (58%), loss of balance (53%), and physical weakness (53%). Also, the patient interviews identified the following as most bothersome: headache (32%) and vision problems (21%). The patients reported additional concepts not identified in the literature review, including pain, fear, falling, fainting, and difficulty reading. Some symptoms or signs identified in the literature were not mentioned by patients during the interviews such as intracranial hypertension and hemorrhage. Conclusions: Although there was considerable overlap among patient and literature reports, including patient input broadened the comprehensiveness of potential symptoms. The results suggest the added value of collecting direct, primary patient data when developing measures to assess benefits of treatment in BM patients.

e13038 Background: Chemotherapy has been associated with increased risk of fractures1. This study examines the real-world incidence of fractures and healthcare resource use (HRU) that may be associated with CAPN in cancer patients. Methods: A retrospective analysis utilized a national health insurer claims -database (2001-2009), to identify patients ≥18 yrs with a cancer ICD-9-code (140-239) and a chemotherapy drug code (J9xxx). The 1st chemotherapy date was the "index date." Patients with a record of peripheral neuropathy (PN) in the pre-index date were excluded. Patients with a PN post-index were matched with no-PN post-index (non-PN) based on gender, age and index date. Both groups were compared for number of fractures, HRU (hospital outpatient (OP), office, and emergency-room [ER] visits) and all-cause costs in their 365-days post-index period. Time to 1st fracture post-index was compared using Kaplan Meier time to event analysis. Results: Of 34,625 patient meeting the inclusion criteria, 1675 patients (4.3%) formed the PN group and were matched to non-PN group. At baseline, mean age was 54.9 yrs, 62.5% were females, and no difference in % of bone metastasis (p=0.12) between the groups. In PN group, 5.3% (n=87) had a fracture 365-days post-index compared to 3.5% (n=58) in non-PN group (p<0.05). Mean days to fracture from index date in PN group was shorter than the non-PN group (150.9 vs. 153.4, p<0.05). In PN group, annual mean number of OP visit (14.6 vs. 12.0, p<0.0001), ER visit (0.47 vs. 0.30, p<0.001), and office visits (30.4 vs. 23.3, p<0.0001), were higher compared to non-PN group. Annual healthcare cost of PN patients was 21% higher than non-PN patients ($64,578 vs. $53,221) and CAPN-related cost in PN group was estimated to be $5,580 annually. Conclusions: Patients with CAPN were associated with higher incidence of fractures, HRU and cost.

116 Background: Metastatic breast cancer (mBC) guidelines contain multiple lines of treatment (LOTs) and regimen options, however little data on treatment patterns is available from real world clinical practice. This study describes patterns of chemo and biologic therapy use by LOT observed within a large U.S. insured population. Methods: Adult women with newly diagnosed mBC and starting anti-neoplastic treatment were identified in the 2005-2009 MarketScan Database which contains medical, pharmacy claims of > 40 million enrollees insured with >100 health plans across the United States. The index date was the first prescription fill or administration of anti-neoplastic treatment following metastatic diagnosis. A 90-day gap in treatment or initiation of a new regimen defined the end of a LOT. Patient demographics and clinical characteristics were measured at index. Descriptive analyses included the distribution of patients, use of chemo and biologic therapies and their therapy duration, across the first 4 LOTs observed. Results: Of 7,767 patients identified (mean (SD) age=58.2 (11.9) years), ≥50% received a subsequent LOT across the 4 LOTs (2 nd LOT n=4,077, 3 rd LOT n=2,033, 4 th LOT n=1,059). Bone (44%) was the major metastatic site at diagnosis. The days on therapy (mean, SD, median) varied across LOTs: (162.7, 176.9, 108.0) in 1 st , (147.5, 146.7, 99.0) in 2 nd , (139.9, 131.1, 99.0) in 3 rd , and (130.9, 123.4, 94.0) in 4 th LOT. The most common backbone chemotherapies were: paclitaxel (26%), capecitabine (22%), docetaxel (22%) in 1 st ; paclitaxel (28%), capecitabine (18%), gemcitabine (16%) in 2 nd ; paclitaxel (22%), gemcitabine (19%), capecitabine (16%) in 3 rd , and gemcitabine (19%), paclitaxel (18%), capecitabine (16%) in 4 th LOT. Trastuzumab was the most frequently used biologic across all 4 lines (20%, 19%, 19%, 19% in 1 st to 4 th LOT respectively) followed by bevacizumab (20%, 19%, 19%, 19% in 1 st to 4 th LOT respectively). Lapatinib was used in 3 rd (6%) and 4 th LOTs (8%) only. Conclusions: Frequency and duration of chemo and biologic therapy use varied by LOT. The most frequently used agents across the 4 LOTs were paclitaxel (chemo backbones) and trastuzumab (biologics). Number of patients halved with each subsequent LOT.

BACKGROUND:Patients treated with epidermal growth factor receptor inhibitors (EGFRIs) may develop dermatologic adverse drug reactions (ADRs) that may affect patients' quality-of-life, require medical care, and may lead to substantial costs. This study assessed the economic burden of dermatologic ADRs in colorectal cancer (CRC), head and neck cancer (HNC), and non-small cell lung cancer (NSCLC) patients. METHODS:Adult patients with ≥1 diagnosis for the study cancer initiated on EGFRIs indicated for CRC, HNC, and NSCLC were selected from a large commercial database (MarketScan Commercial Database [2000-2010]; Thomas Reuters, New York, NY). For each cancer type, patients were classified into two mutually exclusive cohorts: 'ADR' (patients with ≥1 ADR following EGFRI initiation) and 'ADR-free' (patients without any ADR). Patients were observed from the index date up to the end of continuous healthcare plan enrollment or 90 days after EGFRI discontinuation, whichever occurred first. For each cancer group, the proportion of patients and the incidence rate (IR) of experiencing ≥1 dermatologic ADR were reported. Incidence rate ratios for healthcare resource utilization and monthly incremental costs (2010 USD) were estimated using Poisson regression and generalized linear or two-part models, respectively. RESULTS:Overall, the proportion of patients with ≥1 ADR ranged between 20.5-36.4% across cancer groups (IR ranged between 44.2-57.4 per 100 patient-years). After adjusting for confounders, in each cancer group, ADR patients had higher incidence of healthcare resource utilization, generally driven by higher incidence of emergency room visits and incurred incremental total monthly healthcare costs that ranged between $2284-$3210 across cancer groups. LIMITATIONS:There was no clinical measure of cancer staging and ADR severity in the database. CONCLUSIONS:Results suggest that patients with CRC, NSCLC, and HNC, who may benefit from EGFRI therapies, may also incur a substantial economic burden that is associated with dermatologic ADRs.

BACKGROUND The differences in country-specific treatment patterns across Europe for metastatic breast cancer (mBC) patients have not been extensively studied. This study compared the treatment choices in aggregate, as well as by biomarker status, between various lines of therapy in clinical practice in the EU-5 countries among newly diagnosed mBC patients. MATERIALS & METHODS The IMS LifeLink™ Oncology Analyzer database, based on surveys of practicing oncologists, was used to identify mBC patients aged ≥21 years. In this database, sample-level data are projected to national-level estimates for each country using a sample projection technique. RESULTS The prevalence of hormone receptors (71-74%) is quite similar across different countries, while HER2 overexpression varies from 22 (France) to 34% (Italy); chemotherapy combined with HER2-targeted medicine was the mainstay of treatment for HER2(+) patients. The use of HER2-targeted medicine and bevacizumab greatly varied: while they were most frequently used in France, they were least frequently used in the UK. Fewer treatment options existed for triple-negative patients and patients with HER2(+) disease following trastuzumab treatment. Chemotherapy was the treatment choice for triple-negative patients, as these patients do not respond to hormonal therapy and HER2-targeted medicine. CONCLUSION This study found that, while a trastuzumab-based regimen is the preferred option for treating HER2(+) mBC patients in the EU-5, variations in this personalized medicine approach exist between different EU-5 countries. However, fewer treatment options exist for triple-negative and HER2(+) patients after trastuzumab treatment, highlighting the unmet need for these patient subgroups.

According to the FDA Patient-reported outcome (PRO) Guidance, evidence of input from the appropriate population during the development of a PRO measure is critical to determine whether a measure is applicable. A literature review was conducted to identify the symptoms and impacts (concepts) of brain metastases (BM) and the key PRO measures used in BM to compare their development and content validity. Literature searches were conducted using MEDLINE®. PRO measures were reviewed for patient input and the concepts reported in the clinical literature on patients with BM were compared to those included in the identified PRO measures. A total of 34 concepts and seven key PRO measures used in BM (Functional Assessment of Cancer Therapy- General [FACT-G], FACT-Brain Tumor [FACT-Br], FACT-Brain Symptom Index [FBrSI], European Organization for Research and Treatment of Cancer-Quality of Life Questionnaire [EORTC-QLQ-C30], EORTC-Brain cancer module [EORTC QLQ-BN20], the EORTC- palliative care cancer module [EORTC QLQ-C15-PAL] and the M.D. Anderson Symptom Inventory-Brain Tumor Module [MDASI-BT]) were reviewed. The major limitation of all measures reviewed is that the items of these measures were not developed based on qualitative interviews with BM patients. In addition, these measures include concepts that were not related to BM per se as identified in the literature (e.g. itchy skin); some concepts found in the literature were not included in these instruments (e.g. dizziness). Review of the seven PRO measures used in patients with BM found that none of the measures have documented patient input in their development. The gaps between concepts reported in the literature and the PRO measures suggest the need for direct patient input in development of such measures in order to be comprehensive and yet specific to the target population.

Preference-based scoring approaches to measuring health-related quality of life (HRQL) in cancer are proliferating. The objective of this study was to compare preference-based scores estimated by scoring functions for the generic EQ-5D and cancer-specific Functional Assessment of Cancer Therapy (FACT) in terms of differences between algorithms and cancer subtype. Secondary data analysis of patients with advanced cancer (breast, brain, colorectum, hepatobiliary system, lung, and ovary; n=41 to 49 for each subgroup) was conducted. Each patient completed both the EQ-5D and FACT; scores were calculated using scoring functions for EQ-5D (Dolan, Shaw et al), an EQ-5D mapping function (Cheng et al) and FACT (Kind/Macran, Dobrez et al). ECOG performance status rated by physician was used to stratify patients by severity. The relative statistical efficiency (RE) of each algorithm to capture differences in severity was compared using ratios of F-statistics. The rank order of the scores generated by different scoring functions were fairly consistent across cancer subtype, with the lowest mean scores derived from FACT by Kind/Macran (0.52, hepatobiliary, to 0.57, colorectal), and highest mean scores using scoring by Dobrez et al (0.80, hepatobiliary, to 0.85, brain). Within each scoring function, no statistically significant differences in mean scores were found across cancer types. The Dolan algorithm resulted in largest differences in mean scores by severity (ECOG) grades for brain, breast, colorectal and ovarian cancer. The FACT UK societal algorithm by Kind et al had the largest RE for 3 of the cancers (breast, hepatobiliary, and ovarian cancer). Each scoring approach produced different preference-based scores within and across subtype of cancer; extrapolating from ability to discriminate levels of severity EQ-5D scoring functions generally provided scores that would extrapolate to larger QALY benefits compared to FACT-based approaches. No statistically significant differences in the utility scores were observed across the cancer types, but some differences could be considered meaningful; lack of power was a limitation.

To assess the impact of brain metastasis (BrMets) on health care costs and survival among metastatic NSCLC patients in a geographically diverse commercially insured US population. Retrospective analyses were conducted using a US commercial administrative claims database linking data from a lung cancer registry and mortality records from the Social Security Administration Death Master File (2005-2010). Two cohorts were formed – a) with BrMets, and b) without BrMets. Healthcare cost (hospitalization, ambulatory and pharmacy) and resource use (hospitalization, emergency {ER} and ambulatory visits) were compared using a generalized linear model (diagnosis →end of follow-up); a Cox proportional hazard model estimated impact on survival. All models adjusted for stage at diagnosis, pre-diagnosis comorbidity, age, and gender. A total of 584 metastatic NSCLC patients were included (mean 60.5 years/56.3% male): 247 (42.3%) had claims-based evidence of BrMets and were more likely to have been diagnosed with stage IV disease (62.8% vs. 52.2% without BrMets). Overall survival was shorter among patients with evidence of BrMets (median = 13.5 vs. 17.0 months; HR=1.29, p<001); health plan enrollment duration was similar (median = 11.7 months). With similar lengths of follow-up, average health care costs following diagnosis of BrMets was 23% higher ($184,872 vs. $150,931; p=0.010) after adjustment for other factors. The difference was consistent across resources: 25% higher hospitalization costs $46,871 vs. $37,504; p=.082); 23% higher ambulatory costs, ($121,224 vs. $98,276; p=0.033); 23% higher retail pharmacy costs, ($13,282 vs. $10,774; p=0.118). Patients with BrMets averaged more hospitalizations (2.4 vs. 1.9; p=0.005), ER visits (2.7 vs. 2.2; p=0.067), and ambulatory encounters (111 vs. 92; p=0.005) from initial NSCLC diagnosis to end of follow-up. Intensity of resource use and costs were higher in metastatic NSCLC, especially in BrMets patients. Treatment that improves disease control could reduce the intensity of cost and resource use among NSCLC BrMets patients.

To compare health care resource utilization (HRU) and costs by line of therapy (LOT) among patients with metastatic breast cancer (MBC). MarketScan® databases, January 1, 2005 to December 31, 2009, were used to identify women aged ≥18 with breast cancer (ICD-9-CM of 174.xx). The index date was the first prescription fill or administration of anti-neoplastic agents. Either a 90-day gap in treatment or initiation of a new regimen ended each LOT. Per patient per month (PPPM) expenditures for utilizers of inpatient (IP), outpatient (OP), emergency department visits (ED), MBC-drugs (oral and infused), hormonal, radiology, and supportive therapies across four LOTs (1L-first line, 2L-second, 3L-third, 4L-fourth) were statistically compared. HRU rates (Visits per patient with ≥1 Visit) were also compared. A total of 8494 MBC patients (1L:7,765; 2L:4,077; 3L:2,033, 4L:1,059) were included. Bone metastases were most common (43.9%) at index followed by liver (17.7%) and lung (12.8%). PPPM expenditures for IP (1L:$1,183, 2L:$1,318, 3L: $1,401, 4L:$1,670; p=0.660), OP (1L:$1,751, 2L:$1,624; 3L: $1,626; 4L:$1,626, p=0.413), and ED (1L:$64, 2L:$67, 3L: $73, 4L:$57 p=0.997) were not stastically siginificantly different across the four LOTs. PPPM expenditures for MBC oral-drugs (1L:$460, 2L:$530, 3L:$589, 4L:$743,p=0.37), hormonal (1L:$87, 2L:$65, 3L: $70, and 4L:$55,p=0.388), and radiology therapies (1L:$290,2L:$280,3L: $280,and 4L:$271, p=0.999) were also not statistically different across LOTs. MBC infused-drugs (1L:$4096,2L:$4,607,3L:$4,841, 4L:$4,521,p=0.001) did differ. Within supportives, PPPM across LOTs were stastically different for anti-emetics (1L:$283,2L:$321;3L: $320;4L:$311,p=0.007) and pain medications (1L:$42,2L:$50;3L:$62;4L:$71,p=0.002) but not for IV-bisphosphonates (1L:$406,2L:$412;3L:$419;4L:$410,p=0.964). The mean HRU rates for IP (range1.4-1.4), ED (1.7-1.8), OP hospial (8.4-9.4) office-visit (11.2-12.6), and Other outpatient visits (18.9-20.5,) were similar across LOTs. No significant variation in the PPPM costs of (IP, OP, ED, MBC oral drugs, hormonal, radiology, or IV bisphosphonates) was oberved across four LOTs. LOT costs differed for infused drugs, anti-emetics, and pain medication within this MBC population. Further research is required to explore these variations.

Although utility-based algorithms have been developed for the Functional Assessment of Cancer Therapy (FACT), their properties are not well-known compared to more widely used utility measures like the EQ-5D. The objective of this study was to compare the properties and relationships between EQ-5D and FACT-based health utility scores in cancer patients. A retrospective analysis was conducted on cross-sectional data collected from 534 cancer patients who completed both FACT-G and EQ-5D. Properties of scores from 3 FACT-based and 2 EQ-5D based algorithms were examined. Known groups comparisons were based on physician and patient-rated ECOG performance status. Relative efficiency (RE) of the utility algorithms was examined using ratios of F-statistics. Mean scores for the overall cohort were lowest using Kind and Macran's FACT UK societal (0.55, SD 0.09), followed by Dolan's EQ-5D UK societal (0.72, SD 0.23), Cheung et al.'s FACT mapped to EQ-5D (0.74, SD 0.11), Shaw et al.'s EQ-5D US societal (0.79, SD 0.15), and highest using Dobrez et al's FACT US patient algorithm (0.83, SD 0.08). When stratified by ECOG status, the largest differences in mean scores were generally observed for EQ-5D UK societal scores and smallest for the FACT-based US patient scores; however, FACT UK societal scores had twice the statistical efficiency of the other algorithms. We found important differences in utilities scores estimated by each algorithm. The FACT-based algorithms tended to underestimate the QALY benefit compared to the EQ-5D, and appeared to driven by a more limited range of scale.

BACKGROUND & AIMS:We assessed the burden of hepatocellular carcinoma (HCC), in terms of mortality and medical care costs, based on analysis of the Surveillance, Epidemiology and End Results (SEER)-Medicare database.METHODS:We analyzed data from the SEER-Medicare database on patients 66 years or older who were diagnosed with primary HCC from 1991 to 2007, entitled for Medicare Parts A and B, and not enrolled in health maintenance organizations (n = 5712). Controls were individuals without HCC, identified from a 5% sample of Medicare beneficiaries residing in SEER areas; they were matched 1:1 with individuals with HCC (cases) for age, sex, race, and geographic region (average age, 75 y; 34.7% female). Kaplan-Meier analysis was used to estimate survival distributions. Costs were reported in 2009 dollars; per-patient-per-month (PPPM) costs were compared between cases and controls using the Wilcoxon rank sum test.RESULTS:The largest proportion of cases had localized disease (38.2%), followed by regional (24.0%), unstaged (20.4%), and distant (17.3%) disease. The median survival times were 5 months for cases and 60 months for controls; they were 3 months for patients with distant disease, 4 months for patients with regional disease, and 9 months for those with localized disease. The mean PPPM costs were $7863 for cases and $1243 for controls (P < .001). These costs were primarily driven by inpatient (mean, $5439 vs $682 without HCC; P < .001) and hospice (mean $554 vs $42 without HCC; P < .001) care. Mean PPPM costs by stage were $7265 for localized disease, $8072 for regional disease, and $9585 for distant disease (P < .001 for trend).CONCLUSIONS:Based on analysis of the SEER-Medicare database, costs for patients with HCC are approximately 6- to 8-fold higher than for those without this cancer. Patients with distant HCC had the greatest costs. These findings highlight that HCC is a substantial medical cost burden for elderly patients.