Cost of adverse events during treatment with everolimus plus exemestane or single-agent chemotherapy in patients with advanced breast cancer in Western Europe

Objective:

Treatment options for recurrent or progressive hormone receptor-positive (HR+) advanced breast cancer include chemotherapy and everolimus plus exemestane (EVE?+?EXE). This study estimates the costs of managing adverse events (AEs) during EVE?+?EXE therapy and single-agent chemotherapy in Western Europe.

Methods:

An economic model was developed to estimate the per patient cost of managing grade 3/4 AEs for patients who were treated with EVE?+?EXE or chemotherapies. AE rates for patients receiving EVE?+?EXE were collected from the phase III BOLERO-2 trial. AE rates for single-agent chemotherapy, capecitabine, docetaxel, or doxorubicin were collected from published clinical trial data. AEs with at least 2% prevalence for any of the treatments were included in the model. A literature search was conducted to obtain costs of managing each AE, which were then averaged across Western European countries (when available). Per patient costs for managing AEs among patients receiving different therapies were reported in 2012 euros (€).

Results:

The EVE?+?EXE combination had the lowest average per patient cost of managing AEs (€730) compared to all chemotherapies during the first year of treatment (doxorubicin: €1230; capecitabine: €1721; docetaxel: €2390). The most costly adverse event among all patients treated with EVE?+?EXE was anemia (on average €152 per patient). The most costly adverse event among all patients treated with capecitabine, docetaxel, or doxorubicin was lymphocytopenia (€861 per patient), neutropenia (€821 per patient), and leukopenia (€382 per patient), respectively.

Conclusions:

The current model estimates that AE management during the treatment of HR+ advanced breast cancer will cost one-half to one-third less for EVE?+?EXE patients than for chemotherapy patients. The consideration of AE costs could have important implications in the context of healthcare spending for advanced breast cancer treatment.     

Evaluation of the costs and resource use associated with adjuvant chemotherapy for breast cancer in France

Abstract

Objectives:

There is a paucity of recent data on breast cancer costs, particularly on the burden of chemotherapy. The present study was designed to estimate resource use and costs associated with the current standard of care for adjuvant chemotherapy for breast cancer.

Methods:

Costs and resource use were assessed by retrospective analysis of medical records at a single comprehensive cancer care center, Hôpital Tenon, Paris, France. Data were extracted from files of female patients having undergone surgical resection of breast cancer between January–July 2010. Patients were included if they received chemotherapy at the hospital and had medical records available. Patients were followed from the start of adjuvant chemotherapy (including pre-chemotherapy) to the end of treatment. Costs were collected for each resource use item from a societal perspective using standard, published sources and expressed in 2011 Euros (€). Limitations of the analysis included the single-center study design and the use of pre-defined questionnaires on resource use (which may conservatively estimate costs).

Results:

A total of 62 patients were included in the study with a mean age of ～54 years. Most patients had stage II (50.8%) or stage III (40.7%) disease. Anthracycline plus taxane-based chemotherapy regimens were most commonly prescribed (77% of patients). Mean cost of adjuvant chemotherapy was estimated to be ～€15,740 per patient from a societal perspective. The acquisition costs of chemotherapy agents were responsible for 26% of the total, with lost productivity (27%), chemotherapy administration (19%), and adverse events (16%) also contributing substantially.

Conclusions:

Evaluation of costs in patients with non-metastatic breast cancer in France has shown that the costs of adjuvant chemotherapy are substantial. The main components of total cost were the cost of chemotherapy agents, lost productivity, chemotherapy administration, and management and prevention of adverse events.     

Adverse event-related costs for systemic metastatic breast cancer treatment among female Medicaid beneficiaries

Objective: This retrospective study compared the real-world incidence and costs of systemic treatment-related adverse events (AEs) in patients with metastatic breast cancer in a Medicaid population.

Methods: Insurance claims data for adult women who received biologic or chemotherapy (± hormonal therapy) for metastatic breast cancer between 2006–2013 were extracted from the Truven Health MarketScan^® Multi-State Medicaid database. Incidence of AEs (per 100 person years) and average monthly AE-related healthcare costs (per-patient-per-month) during each line of therapy (first or later lines) were estimated. The association between AEs and total all-cause healthcare costs was estimated using multivariable regression.

Results: A total of 729 metastatic breast cancer patients were analyzed. Hematological (202.3 per 100 person years) and constitutional AEs (289.6 per 100 person years) were the most common class of AEs reported. Unadjusted per-patient-per-month AE-related expenditure by class were highest for hematological AEs ($1524), followed by gastrointestinal ($839) and constitutional AEs ($795), with anemia ($942), nausea/vomiting ($699), and leukopenia/neutropenia ($550) having incurred the highest total AE-related costs. Adjusted total all-cause monthly costs increased with the number of AEs ($19,701 for >7 AEs, $16,264 for 4???6 AEs, and $13,731 for 1???3 AEs) compared to no AEs ($5908) (all p?Conclusions: Among metastatic breast cancer patients treated with systemic therapy in a Medicaid population, AEs were associated with significant increases in costs, which increased with the number of AEs experienced. Therapies associated with a lower incidence of AEs may reduce cost burden and improve patient outcomes.     

Cost impact of oral capecitabine compared to intravenous taxane-based chemotherapy in first-line metastatic breast cancer

Abstract

Objective: Few studies have examined the costs associated with differing first-line chemotherapy regimens in patients with metastatic breast cancer (MBC). This study compares the relative cost impact of women starting first-line chemotherapy with capecitabine versus taxanes.

Methods: Women receiving first-line chemotherapy for MBC from 1998 to 2002 were identified from a hybrid North Carolina Medicaid-claims-tumour registry linked database and Medicare records, and were followed through to 2005 with claims data. Statistical t- and chi-square tests were used to compare baseline characteristics between patients who received first-line chemotherapy with capecitabine versus taxanes. Projected mean costs for 12 months continuous eligibility were estimated using an ordinary least squares linear regression. Overall cost impact of capecitabine after start of therapy was then examined using a multivariate log-linear regression model.

Results: While patients starting taxanes had significantly lower total costs in the pre-index year than patients starting capecitabine (mean: $20,042 vs. $35,538, p<0.001), in the post-index year, the patients on taxanes experienced significantly higher healthcare utilisation and associated costs compared to patients on capecitabine (mean: $43,353 vs. $35,842, p=0.0089). The differences were primarily attributable to lower expenses in chemotherapy related claims and fewer visit days to outpatient settings for patients on capecitabine. After adjustment with propensity scores and other confounders, the capecitabine group was associated with 32% lower healthcare costs compared to the taxane group (p=0.0001).

Conclusions: In this population-based study, women who received capecitabine as first-line treatment for MBC had significantly lower costs compared to women starting taxane therapy.     

Comparison of medical costs and healthcare resource utilization of post-menopausal women with HR+/HER2− metastatic breast cancer receiving everolimus-based therapy or chemotherapy: a retrospective claims database analysis

Objective:

To analyze medical costs and healthcare resource utilization (HRU) associated with everolimus-based therapy or chemotherapy among post-menopausal women with hormone-receptor-positive, human-epidermal-growth-factor-receptor-2-negative (HR+/HER2?) metastatic breast cancer (mBC).

Methods:

Patients with HR+/HER2? mBC who discontinued a non-steroidal aromatase inhibitor and began a new line of treatment with everolimus-based therapy or chemotherapy (index therapy/index date) between July 20, 2012 and April 30, 2014 were identified from two large claims databases. All-cause, BC-related, and adverse event (AE)-related medical costs (in 2014 USD) and all-cause HRU per patient per month (PPPM) were analyzed for both treatment groups across patients’ first four lines of therapies for mBC. Adjusted differences in costs and HRU between the everolimus and chemotherapy treatment group were estimated pooling all lines and using multivariable generalized linear models, accounting for difference in patient characteristics.

Results:

A total of 3298 patients were included: 902 everolimus-treated patients and 2636 chemotherapy-treated patients. Compared to chemotherapy, everolimus was associated with significantly lower all-cause (adjusted mean difference?=?$3455, p?<?0.01) and BC-related ($2510, p?<?0.01) total medical costs, with inpatient ($1344, p?<?0.01) and outpatient costs ($1048, p?<?0.01) as the main drivers for cost differences. Everolimus was also associated with significantly lower AE-related medical costs ($1730, p?<?0.01), as well as significantly lower HRU (emergency room incidence rate ratio [IRR]?=?0.83; inpatient IRR?=?0.74; inpatient days IRR?=?0.65; outpatient IRR?=?0.71; BC-related outpatient IRR?=?0.57; all p?<?0.01).

Conclusions:

This retrospective claims database analysis of commercially-insured patients with HR+/HER2? mBC in the US showed that everolimus was associated with substantial all-cause, BC-related, and AE-related medical cost savings and less utilization of healthcare resources relative to chemotherapy.     

Cost-effectiveness of ceritinib in previously untreated anaplastic lymphoma kinase-positive metastatic non-small cell lung cancer in the United States

Aims: To assess the cost-effectiveness of first-line ceritinib vs crizotinib and platinum doublet chemotherapy for anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC) from a US third-party payer’s perspective.

Materials and methods: A partitioned survival model with three health states (stable disease, progressive disease, death) was developed over a 20-year time horizon. Ceritinib’s efficacy inputs (progression-free and overall survival) were estimated from ASCEND-4; parametric survival models extrapolated data beyond the trial period. The relative efficacy of ceritinib vs chemotherapy was obtained from ASCEND-4, the relative efficacy of ceritinib vs crizotinib was estimated using a matching-adjusted indirect comparison based on ASCEND-4 and PROFILE 1014. Drug acquisition, treatment administration, adverse event management, and medical costs were obtained from publicly available databases and the literature, and inflated to 2016?US dollars. Treatment-specific stable-state utilities were derived from trials and progressive-state utility from the literature. Incremental costs per quality-adjusted life year (QALY) were estimated for ceritinib vs each comparator. Cost-effectiveness was assessed based on US willingness-to-pay thresholds. Deterministic and probabilistic sensitivity analyses were performed to test model robustness.

Results: In the base case, first-line ceritinib was associated with total direct costs of $299,777 and 3.28 QALYs (from 4.61 life years gained [LYG]) over 20 years. First-line crizotinib and chemotherapy were associated with 2.73 and 2.41 QALYs, 3.92 and 3.53 LYG, and $263,172 and $228,184 total direct costs, respectively. The incremental cost per QALY gained was $66,064 for ceritinib vs crizotinib and $81,645 for ceritinib vs chemotherapy. In the first 2 years following treatment initiation, ceritinib dominated crizotinib by conferring greater health benefits at reduced total costs. Results were robust to deterministic and probabilistic sensitivity analyses.

Limitations: In the absence of head-to-head trials, an indirect comparison method was used.

Conclusions: Ceritinib is cost-effective compared to crizotinib and chemotherapy in the treatment of previously untreated ALK-positive metastatic NCSLC in the US.     

Cost drivers for breast,lung, and colorectal cancer care in a commercially insured population over a 6-month episode: an economic analysis from a health plan perspective

Aims: In the absence of clinical data, accurate identification of cost drivers is needed for economic comparison in an alternate payment model. From a health plan perspective using claims data in a commercial population, the objective was to identify and quantify the effects of cost drivers in economic models of breast, lung, and colorectal cancer costs over a 6-month episode following initial chemotherapy.

Research design and methods: This study analyzed claims data from 9,748 Cigna beneficiaries with diagnosis of breast, lung, and colorectal cancer following initial chemotherapy from January 1, 2014 to December 31, 2015. We used multivariable regression models to quantify the impact of key factors on cost during the initial 6-month cancer care episode.

Results: Metastasis, facility provider affiliation, episode risk group (ERG) risk score, and radiation were cost drivers for all three types of cancer (breast, lung, and colorectal). In addition, younger age (p?p?p?p?p?Conclusions: Value-based reimbursement models in oncology should appropriately account for key cost drivers. Although claims-based methodologies may be further augmented with clinical data, this study recommends adjusting for the factors identified in these models to predict costs in breast, lung, and colorectal cancers.     

Budget impact analysis of everolimus for the treatment of hormone receptor positive,human epidermal growth factor receptor-2 negative (HER2−) advanced breast cancer in Kazakhstan

Abstract

Objective:

The aim of this study was to determine the budget impact of everolimus (in combination with letrozole/anastrozole) as a second-line treatment for ER+ HER2? negative advanced and metastatic breast cancer in post-menopausal women.

Research design and methods:

A cumulative cohort model was developed to estimate the 5-year costs associated with introducing everolimus to the Kazakh healthcare system. Two alternative market share scenarios were compared: with everolimus and without everolimus. PFS and OS data were taken from the trial and extrapolated. The background costs of the pre-progressed and post-progressed health states, drug costs and costs associated with adverse events were included in the model.

Results:

The 5-year results from the budget impact analysis demonstrate that the introduction of everolimus leads to a 12% increase in drug costs, a 2% reduction in pre-progression health state costs, a 1% increase in post-progression health state costs, and a 2% reduction in adverse event costs. The net result is only a modest increase in total costs; a 2.69% increase of T201 million, from T7.5 billion to T7.7 billion over a period of 5 years.

Conclusions:

The analysis estimated that, if everolimus were to be introduced to the Kazakh healthcare market for the treatment of ER+ HER2? advanced breast cancer, there would be minimal impact upon overall healthcare expenditure. An increase in drug acquisitions costs was almost exactly offset by a reduction in other healthcare costs, due to improved management of the disease.     

Real-world utilization of molecular diagnostic testing and matched drug therapies in the treatment of metastatic cancers

Aims: To assess the frequency of biopsies and molecular diagnostic testing (human DNA/RNA analysis), anti-cancer drug use (genomically-matched targeted therapy [GMTT], unmatched targeted therapy [UTT], endocrine therapy [ET], and chemotherapy [CT]), and medical service costs among adults with metastatic cancer.

Methods: Adults diagnosed with metastatic breast, non-small cell lung (NSCLC), colorectal, head and neck, ovarian, and uterine cancer (2010Q1–2015Q1) were identified in the OptumHealth Care Solutions claims database and followed from first metastatic diagnosis for ≥1 month and until the end of data availability. Utilization was assessed for each cancer cohort (all and patients aged ≥65 years); per-patient-per-month (PPPM) medical service costs were assessed for all patients. Testing frequency estimates were applied to Surveillance, Epidemiology, and End Results Program data to estimate the number of untested patients (2010–2014).

Results: Patients with metastatic cancer (n?=?8,193; breast [n?=?3,414], NSCLC [n?=?2,231], colorectal [n?=?1,611], head and neck [n?=?511], ovarian [n?=?275], and uterine [n?=?151]) were 63 years old (mean), with 11.1–22.2 months of observation. Biopsy and molecular diagnostic testing frequencies ranged from 7% (uterine) to 73% (ovarian), and from 34% (head and neck) to 52% (breast), respectively. Few were treated with GMTT (breast, 11%; NSCLC, 9%; colorectal, 6%). Treatment with UTT ranged from 0.7% (uterine) to 21% (colorectal). Biopsy, diagnostic testing, and anti-cancer drug therapy were less frequent for those ≥65 years. Medical service costs (PPPM, mean) ranged from $6,618 (head and neck) to $9,940 (ovarian). The estimated number of untested new patients with metastatic cancer was 636,369 (all) and 341,397 (≥65).

Limitations: In addition to the limitations of claims analyses, diagnostic testing frequency may be under-estimated if patients underwent testing prior to study inclusion.

Conclusions: The low frequency of molecular diagnostic testing suggests there are opportunities to better inform management of patients with advanced cancer, particularly decisions to treat with GMTT.     

Retrospective analysis of patients with advanced or metastatic gastric cancer in Colombia

Abstract

Aims: To assess patient and disease characteristics, treatment patterns, and associated costs in patients with advanced or metastatic gastric cancer (A/MGC) in Colombia, in both the public and private hospitals.

Materials and methods: A total of 145 patients who had received first-line chemotherapy treatment (platinum analog and/or a fluoropyrimidine) and were followed for at least 3 months after the last administration of a first-line cytotoxic agent were eligible for inclusion. Case-report forms were elaborated based on the patients’ medical records from three Colombian hospitals. Estimates of treatment costs were calculated using unit costs from the participating hospitals.

Results: Of the 145 patients, more than half (64.83%) were male, 79.56% were diagnosed with metastatic stage IV disease (mean age = 58.14?years). Prior to MGC diagnosis, 31.71% of the patients being operated on received a total gastrectomy; 66.9% of the patients received a doublet therapy, of which 5-fluorouracil (5-FU) in combination with cisplatin was the standard treatment (14%), followed by combination with leucovorin (12%). Only around 10% of the patients responded to first-line treatment. Out of 41.38% of the patients who received a second-line treatment, 71.67% were still administered a platinum analog and/or fluoropyrimidine. During the follow-up period, 52% of the patients progressed and 20% achieved stable disease. Best supportive care mostly consisted of outpatient visits after last line-therapy (72.41%), palliative radiotherapy (18.6%), and surgery (37.2%).

Limitations and conclusions: Gastric cancer is one of the main causes of cancer-related death in Colombia, as most of the patients are diagnosed at an advanced stage, when prognosis is poor. Treatment patterns are highly heterogeneous. Second-line treatments were mostly initiated with paclitaxel, capecitabine, irinotecan, or cisplatin.     

Cost-effectiveness of six months versus 1-year adjuvant trastuzumab in HER2 positive early breast cancer in Egypt

Abstract

Introduction: Breast cancer is the most prevalent cancer among women in Egypt. Trastuzumab is administered with chemotherapy for patients with HER2-positive advanced breast cancer (HER2?+?ve ABC) in the metastatic and adjuvant settings resulting in improved treatment outcomes, and long-term follow-up. Some studies have evaluated whether equivalent outcomes can be achieved with reduced treatment duration. This study evaluates the cost-effectiveness of 6-month versus 1-year trastuzumab treatments from payer perspective over a 10 year time horizon.

Methods: A half-cycle corrected Markov model was developed with five mutually exclusive health states; patient with HER2?+ve ABC, disease-free survival (DFS), local or regional relapse, metastatic relapse, and death. A cycle length of 6 months was applied, direct medical costs including cost of treatments, day-care, surgery, health states and follow-up visits were collected, and indirect costs such as lost productivity were not estimated. The transition probabilities and utilities were extracted from published literature, and deterministic sensitivity analyses were conducted.

Results: Among the HER2?+ve ABC patient population in Egypt, the total QALYs of the 6-month trastuzumab were estimated to be 2.99 compared with 2.93 for the 1-year trastuzumab which resulted in a difference of 0.06 QALYs. The total costs were EGP 271,647 ($106,947) and EGP 381,248 ($150,097), respectively. These costs yielded an ICER of –109,600 EGP/QALY (–43,149 $/QALY) for the 6-month trastuzumab. The 6-month trastuzumab is a dominant strategy when compared to 1-year trastuzumab, resulting in improved effectiveness at a reduced cost. All analyses results confirmed the dominance of 6-month trastuzumab and our model robustness.

Conclusions: This study concluded that 6-month trastuzumab is a cost-effective option when compared to 1-year trastuzumab in patients with HER2?+ve ABC in Egypt. Our findings provide health care decision makers with additional insights to best allocate available resources concurrently with the improvement of the Egyptian patient’s outcomes.     

Treatment patterns and economic burden of metastatic and recurrent locally-advanced head and neck cancer patients

Abstract

Objective:

To characterize treatment patterns and measure the economic burden associated with metastatic (mHNC) and recurrent, locally-advanced head and neck cancer (rHNC).

Methods:

Administrative claims from Medicare- and privately-insured individuals during 2004–2008 were used in this retrospective database study of patients with advanced HNC. Patients diagnosed with HNC were matched 1:1 to cancer-free controls to measure the incremental economic burden of HNC. Outcomes of interest were measured during the 6 months following the date of a secondary tumor diagnosis for metastatic patients or the date of a diagnosis indicating rHNC. To assess treatment patterns, HNC patients were evaluated for the use frequency of treatments (radiotherapy, chemotherapy and surgery). Costs were reported in 2008 US$ from a third-party payer perspective and were analyzed using generalized linear models and two-part regression models adjusting for differences in age and baseline Charlson Comorbidity Index (excluding cancer diagnoses) between the HNC and control cohorts. Components of cost included inpatient, outpatient and other medical services as well as pharmacy costs.

Results:

The mHNC cohort consisted of 1042 patients and the rHNC cohort included 324 patients. The most common treatments for mHNC patients were supportive care (90.2%), radiation therapy (48.5%), surgery (41.9%) and chemotherapy (38.3%). Patients with rHNC frequently received HNC-related supportive care (71.0%), radiation therapy (67.9%) and chemotherapy (27.2%); HNC-related surgery was infrequent (12.7%) during the study period. The 6-month incremental adjusted total costs were $60,414 per patient for mHNC and $21,141 per patient for rHNC (p?<?0.0001). Approximately 46–58% of the incremental cost was attributable to outpatient visits, 27–37% to inpatient costs and 11–13% to pharmacy, depending on the HNC cohort.

Limitations:

The identification of mHNC/rHNC was based on diagnosis codes and treatment patterns with the limitation of the claims database.

Conclusions:

Metastatic and recurrent, locally-advanced HNC patients frequently receive cancer-related treatments and incur substantial economic burden.     

Indirect costs associated with metastatic breast cancer

Abstract

Objective:

To compare the indirect costs of productivity loss between metastatic breast cancer (MBC) and early stage breast cancer (EBC) patients, as well as their respective family members.

Methods:

The MarketScan® Health and Productivity Management database (2005–2009) was used. Adult BC patients eligible for employee benefits of sick leave and/or short-term disability were identified with ICD-9 codes. Difference in sick leave and short-term disability days was calculated between MBC patients and their propensity score matched EBC cohort and general population (controls) during a 12-month follow-up period. Generalized linear models were used to examine the impact of MBC on indirect costs to patients and their families.

Results:

A total of 139 MBC, 432 EBC, and 820 controls were eligible for sick leave and 432 MBC, 1552 EBC, and 4682 controls were eligible for short-term disability (not mutually exclusive). After matching, no statistical difference was found in sick leave days and the associated costs between MBC and EBC cohorts. However, MBC patients had significantly higher short-term disability costs than EBC patients and controls (MBC: $6166?±?$9194 vs EBC: $3690?±?$6673 vs Controls: $558?±?$2487, both p?<?0.001). MBC patients had more sick leave cost than controls ($2383?±?$5539 vs $1282?±?$2083, p?<?0.05). Controlling for covariates, MBC patients incurred 47% more short-term disability costs vs EBC patients (p?=?0.009). Older patients (p?=?0.002), non-HMO payers (p?<?0.05), or patients not receiving chemotherapy during follow-up (p?<?0.001) were associated with lower short-term disability costs. MBC patients’ families incurred 39.7% (p?=?0.06) higher indirect costs compared to EBC patients’ families after controlling for key covariates.

Conclusion:

Productivity loss and associated costs in MBC patients are substantially higher than EBC patients or the general population. These findings underscore the economic burden of MBC from a US societal perspective. Various treatment regimens should be evaluated to identify opportunities to reduce the disease burden from the societal perspective.     

Healthcare costs,treatment patterns,and resource utilization among pancreatic cancer patients in a managed care population

Abstract

Background:

Pancreatic adenocarcinoma has few effective treatment options and poor survival. The objective of this study was to characterize treatment patterns and estimate the costs and resource use associated with its treatment in a commercially-insured US population.

Methods:

In this retrospective claims-based analysis, individuals ≥18 years old with evidence of pancreatic adenocarcinoma between January 1, 2001 and December 31, 2010 were selected from a managed care database. Treatment phase (either initial non-metastatic or metastatic) was determined using a claims-based algorithm. Patients in the pancreatic cancer population were matched 1:3 to a control population. Resource use (events/person-years), treatment patterns, and healthcare costs (per-patient per-month, PPPM) were determined during a variable length follow-up period (from first pancreatic cancer diagnosis to earliest of death, disenrollment, or study end).

Results:

In this study, 5262 pancreatic cancer patients were matched to 15,786 controls. Rates of office visits, inpatient visits, ER visits, and inpatient stays, and mean total all-cause healthcare costs PPPM ($15,480 vs $1001) were significantly higher among cancer patients than controls (all p?<?0.001). Mean inpatient costs were the single largest cost driver ($9917 PPPM). Also, mean total all-cause healthcare costs were significantly higher during the metastatic treatment phase vs the initial treatment phase of non-metastatic disease ($21,637 vs $10,358, p?<?0.001).

Conclusions:

These results indicate that pancreatic cancer imposes a substantial burden on the US healthcare system, and that treatment of more advanced disease is significantly more costly than initial treatment of non-metastatic disease.

Limitations:

Additional research is needed to validate the accuracy of the claims-based algorithms used to identify the treatment phase.     

Confirming the timing of phase-based costing in oncology studies: a case example in advanced melanoma

Aims: The utilization of healthcare services and costs among patients with cancer is often estimated by the phase of care: initial, interim, or terminal. Although their durations are often set arbitrarily, we sought to establish data-driven phases of care using joinpoint regression in an advanced melanoma population as a case example.

Methods: A retrospective claims database study was conducted to assess the costs of advanced melanoma from distant metastasis diagnosis to death during January 2010–September 2014. Joinpoint regression analysis was applied to identify the best-fitting points, where statistically significant changes in the trend of average monthly costs occurred. To identify the initial phase, average monthly costs were modeled from metastasis diagnosis to death; and were modeled backward from death to metastasis diagnosis for the terminal phase. Points of monthly cost trend inflection denoted ending and starting points. The months between represented the interim phase.

Results: A total of 1,671 patients with advanced melanoma who died met the eligibility criteria. Initial phase was identified as the 5-month period starting with diagnosis of metastasis, after which there was a sharp, significant decline in monthly cost trend (monthly percent change [MPC]?=?–13.0%; 95% CI?=?–16.9% to –8.8%). Terminal phase was defined as the 5-month period before death (MPC?=?–14.0%; 95% CI?=?–17.6% to –10.2%).

Limitations: The claims-based algorithm may under-estimate patients due to misclassifications, and may over-estimate terminal phase costs because hospital and emergency visits were used as a death proxy. Also, recently approved therapies were not included, which may under-estimate advanced melanoma costs.

Conclusions: In this advanced melanoma population, optimal duration of the initial and terminal phases of care was 5 months immediately after diagnosis of metastasis and before death, respectively. Joinpoint regression can be used to provide data-supported phase of cancer care durations, but should be combined with clinical judgement.     

Healthcare costs in postmenopausal women with hormone-positive metastatic breast cancer

Abstract

Objectives:

This study examines costs for postmenopausal women with hormone receptor positive (HR+) metastatic breast cancer (mBC).

Methods:

Data were obtained from the IHCIS National Managed Care Benchmark Database from 1/1/2001 to 6/30/2006. Women aged 55–63 years were selected for the study if they met the inclusion criteria, including diagnoses for breast cancer and metastases, and at least two fills for a hormone medication. Patients were followed from the onset of metastases until the earliest date of disenrollment from the health plan or 6/30/2006. Patient characteristics were examined at time of initial diagnoses of metastases, while costs were examined post-diagnosis of metastases and prior to receipt of chemotherapy (pre-chemotherapy initiation period) and from the date of initial receipt of chemotherapy until end of data collection (post-chemotherapy initiation period). Costs were adjusted to account for censoring of the data.

Results:

The study population consisted of 1,266 women; mean (SD) age was 59.05 (2.57) years. Pre-chemotherapy initiation, unadjusted inpatient, outpatient, and drug costs were $4,392, $47,731, and $5,511, while these costs were $4,590, $57,820, and $38,936 per year, respectively, post-chemotherapy initiation. After adjusting for censoring of data, total medical costs were estimated to be $55,555 and $70,587 in the first 12 months and 18 months, respectively in the pre-chemotherapy initiation period. Post-chemotherapy initiation period, 12-month and 18-month adjusted total medical costs were estimated to be $87,638 and $130,738.

Limitations:

The use of an administrative claims database necessitates a reliance upon diagnostic codes, age restrictions, and medication use, rather than formal assessments to identify patients with post-hormonal women with breast cancer. Furthermore, such populations of insured patients may not be generalizable to the population as a whole.

Conclusions:

These findings suggest that healthcare resource use and costs – especially in the outpatient setting – are high among women with HR+ metastatic breast cancer.     

Cost of illness associated with Niemann-Pick disease type C in the UK

Abstract

Objective: Niemann-Pick disease type C (NP-C) is a rare and devastating genetic disorder characterised by a range of progressive neurological symptoms, which imposes a burden on patients, family members, the healthcare system and society overall. The objective of this study was to assess direct and indirect costs associated with NP-C in the UK.

Methods: This was a non-interventional, retrospective, cross-sectional cohort study based on responses from patients and/or their carers/guardians recruited from a UK NP-C database. Resource use and direct medical, direct non-medical and indirect costs were evaluated using data collected via postal survey in October 2007, which included a Medical Resource Use questionnaire. Total annual costs per patient were estimated.

Results: In total, 18 Medical Resource Use questionnaires (29% response rate) were received and analysed. The mean total annual cost (SD) of NP-C per patient was £39,168 (£50,315); 46% were direct medical costs, to which home visits and residential care contributed 68% and 15%, respectively. Direct non-medical costs accounted for 24% of the average annual cost per patient, mainly due to specialist education, and indirect costs 30%. If only direct medical costs were considered, the mean annual cost (SD) per patient was reduced to £18,012 (£46,536).

Conclusions: The direct annual per-patient cost of NP-C illness in 2007 appears moderate when compared with other rare and severely disabling diseases. However, cost estimates may be conservative, since findings are limited by a small sample size, low survey response rate and potential recall bias. As demonstrated by this study, a substantial proportion of the cost is shifted from the healthcare system to the patient, family and non-medical providers. These findings highlight the need for treatments that can slow or stop disease progression in NP-C.     

Burden of breast cancer with brain metastasis: a French national hospital database analysis

Abstract

Objective:

Incidence of breast cancer with brain metastases (BCBM) is increasing, especially among patients over-expressing HER2. Epidemiology on this sub-type of cancer is scarce, since cancer registries carry no information on the HER2 status. A retrospective database analysis was conducted to estimate the burden of BCBM, especially among HER2-positive patients in a secondary objective.

Methods:

Patients with a new diagnosis of BCBM carried out between January and December 2008 were identified from the national hospital database using the International Disease Classification. Patients receiving a targeted anti-HER2 therapy were identified from the national pharmacy database. Hospital and pharmacy claims were linked to estimate the burden of HER2-positive patients. Data on hospitalizations were extracted to describe treatment patterns and healthcare costs during a 1-year follow-up. Predictors of treatment cost were analyzed through multi-linear regression analysis.

Results:

Two thousand and ninety-nine BCBM patients were identified (mean age (SD)?=?57.8 (13.6)), of whom 12.2% received a targeted anti-HER2 therapy; 79% of patients had brain metastases associated with extracranial metastases, and the attrition rate reached 82%. Patients received mostly palliative care (47.4%), general medical care (40.6%), and chemotherapy (35.0%). The total annual hospital cost of treatment was 8,426,392€, representing a mean cost of 22,591€ (±14,726) per patient, mainly influenced by extracranial metastases, surgical acts, and HER2-overexpression (p?<?0.0001).

Conclusions:

The database linkage of hospital and pharmacy claims is a relevant approach to identify sub-type of cancer. Chemotherapy was widely used as a systemic treatment for breast cancer rather than for local treatment of brain metastases whose morbi-mortality remains high. The variability of treatment costs suggests clinical heterogeneity and, thus, extensive individualization of protocols.