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Triple-negative breast cancer (TNBC) is associated with a high recurrence risk. However, the magnitude of direct and indirect costs associated with recurrence is lacking in the literature.Adults 18-65 years old diagnosed with TNBC were identified from the OptumHealth Reporting and Insights claims database (1999-2017) and stratified by recurrence. For patients with recurrence, the index date was defined as 30 days before recurrence; for patients without recurrence, it was randomly assigned based on the distribution of time between first treatments and index dates of the recurrence cohort. All-cause and breast cancer-related healthcare resource utilization (HRU), direct and indirect costs, and work loss up to 1 year were compared between cohorts using generalized linear models. Kaplan-Meier analyses and Cox proportional hazards models compared the risk of leaving the workforce.Among the 2340 patients analyzed, mean age was 54 years and 75% of patients had stage 0-2 cancer. Among the 1170 patients with recurrence, 236 were categorized as having metastatic recurrence and 934 as having locoregional recurrence. Relative to patients without recurrence, those with recurrence had significantly higher all-cause and breast cancer-related HRU. For instance, adjusted incidence rates (IRs) for all-cause inpatient admissions were 3.67 and 10.19 times higher for patients with locoregional and metastatic recurrence, respectively (p 0.001). Adjusted all-cause healthcare costs were $8575/month higher for metastatic recurrence and $3609/month higher for locoregional recurrence vs. patients without recurrence (p 0.001). Adjusted IRs for work loss days were approximately two times higher for locoregional and metastatic recurrence vs. without recurrence (p 0.001). Patients with locoregional recurrence incurred $335/month more indirect costs vs. patients without recurrence; those with metastatic recurrence incurred $769/month more (p 0.05). Patients with recurrence had a 63% higher rate of leaving the work force (p = 0.003).The incremental direct and indirect economic burden associated with recurrent TNBC is substantial relative to non-recurrence.

Lay abstract This study included 308 patients with early-stage triple-negative breast cancer treated in the USA at community oncology practices. Patients were female, 18 years or older and diagnosed with stage II, IIIA or IIIB breast cancer between March 2008 and March 2016, and the breast cancer was determined to be triple negative (i.e., negative for estrogen receptors, progesterone receptors and excess HER2 protein). There were 236 patients who received neoadjuvant treatment without subsequent adjuvant treatment (the Neo group) and 72 patients who received neoadjuvant treatment followed by adjuvant treatment (the Neo + adj group). The study looked at healthcare resource use and costs of care during two time periods: from neoadjuvant treatment start date to surgery (Time 1) and after surgery to recurrence or death (Time 2). Average monthly healthcare resource use and average monthly costs of care per patient were higher in Time 1 compared with Time 2 for both groups. These results demonstrate the economic and resource burden of early-stage triple-negative breast cancer especially in the time from neoadjuvant treatment initiation until surgery.

Aim: To analyze therapy for metastatic triple-negative breast cancer (mTNBC), factors contributing to survival and costs. Patients & methods: Using 2010–2016 SEER-Medicare data, we identified women (≥65 years) with mTNBC. Results: Of 302 eligible patients, 152 (50%) received systemic therapy. In multivariable regression analyses, only age

Background: Recurrence is common among patients with early-stage triple-negative breast cancer (ESTNBC). There is minimal real-world evidence describing the patient characteristics and clinical outcomes following recurrence among patients receiving neoadjuvant chemotherapy for ESTNBC. Methods: This retrospective, observational study aimed to describe the demographic and clinical characteristics and clinical outcomes in ESTNBC patients experiencing locoregional recurrence in the US community oncology setting in the Concerto HealthAI Definitive Oncology Dataset. Eligibility criteria included female sex, age 18+ years, diagnosis of stage II, IIIA or IIIB ESTNBC between 3/2008 and 3/2016, and receipt of definitive surgical resection following neoadjuvant systemic therapy. Descriptive methods were used to evaluate patient characteristics and treatment patterns in this population. Locoregional recurrence was defined as recurrence in the same breast and/or regional nodal recurrence as documented by the provider in the medical record. Results: Of 308 patients who received neoadjuvant treatment for ESTNBC, 27.3% patients (n=84) observed recurrence, within which 25.0% (n=21) were locoregional and 75.0% (n=63) were metastatic. All 21 patients with locoregional recurrence were 65 or younger, with mean age of 50.6 (SD 8.7) at initial diagnosis. They were primarily White (47.6%, n=10) or African American (42.9%, n=9). Over half of patients were stage II at initial diagnosis (61.9%, n=13), while 38.1% (n=8) were stage III. The majority had ductal histology (90.5%, n=19) and had Grade 3 tumors (90.5%, n=19). Of the 21 patients with locoregional recurrence, less than one-tenth (9.5%, n=2) had achieved pathologic complete response (pCR) prior to their recurrence, compared to 41.2% (n=127) of the 308 patients receiving neoadjuvant treatment. In terms of treatment following locoregional recurrence, two-thirds of patients received radiation therapy (66.6%, n=14) with median duration of 47.5 days. Over half of patients (57.1%, n=12) had mastectomy following recurrence, while 14.3% (n=3) had partial mastectomy (breast conserving surgery). Most patients (85.7%, n=18) received systemic chemotherapy after recurrence. Median duration of systemic therapy following locoregional recurrence was 108 days. Nearly one-half (47.6%, n=10) had a subsequent metastatic diagnosis and nearly one-third (28.6%, n=6) had a record of death. Median time from locoregional recurrence to metastatic diagnosis was 36.6 months, but median time from locoregional recurrence to death was not reached. Conclusions: Among patients who received neoadjuvant therapy for ESTNBC in the real-world setting, nearly 7% (n=21) experienced locoregional recurrence. Nearly one-fourth of those patients had a prior pCR which potentially suggests a higher risk of recurrence associated with ESTNBC patients. Chemotherapy was the mainstay of treatment following recurrence. Most patients also received radiation therapy and surgery, but despite those nearly one-half of the patients went on to have a subsequent metastatic diagnosis. This probably reflects the limitations of existing treatment modalities for ESTNBC patients. Future studies with a bigger sample size could confirm our findings. Our study provides some benchmark perspective to such future studies. Citation Format: Amin Haiderali, Whitney C. Rhodes, Santosh Gautam, Min Huang, Jan Sieluk, Karen E. Skinner, Lee S. Schwartzberg. Locoregional recurrence in patients with early-stage triple-negative breast cancer receiving neoadjuvant systemic therapy: Patient characteristics and clinical outcomes [abstract]. In: Proceedings of the 2020 San Antonio Breast Cancer Virtual Symposium; 2020 Dec 8-11; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2021;81(4 Suppl):Abstract nr PS13-42.

Objectives There are challenges in conducting a budget impact analysis (BIA) for rare disorders. Through this case study, we present some challenges and limitations of a BIA of managing patients affected with alpha-1 antitrypsin deficiency (AATD). We explored a conceptual basis and barriers for health services researchers interested in quantifying budget impacts of rare disease management program (DMP). Methods We developed a static budget impact cost calculator model in Microsoft Excel, obtaining the clinical impact of a DMP from the literature and translating it into costs using OLDW. Cost inputs and resource use was obtained from 2010 to 2015 claims data using the OLDW. Insurers’ payments were calculated and categorized into the following cost buckets: physician visits, emergency room visits, inpatients stays, augmentation therapy, other prescription drugs costs, and other costs. Results Data were based on 6832 patients with alpha-1 antitrypsin deficiency identified among over 21 million OLDW enrollees observed between January 1, 2010, and December 31, 2015. The introduction of a DMP was estimated to decrease costs of the management of patients with alpha-1 antitrypsin deficiency by $13.5 million over 5 years. The savings attributed to the program over the 5-year time horizon are due to 2555 exacerbations, 5180 emergency room visits, 9342 specialist visits, and 105 358 general practitioner visits avoided. Conclusions A comprehensive DMP for a rare condition might provide cost savings to a health plan. BIAs for rare disease may be more informative if they focus on DMPs rather than on individual drugs.

Background: For patients with stage II-III early-stage triple-negative breast cancer (ESTNBC), either neoadjuvant or adjuvant administration of chemotherapy is recommended. There is limited evidence describing the real-world patient characteristics and treatment patterns among patients receiving neoadjuvant +/- adjuvant chemotherapy for ESTNBC. Methods: We conducted a retrospective observational study to describe the demographic and clinical characteristics and treatment patterns in ESTNBC patients in the US community oncology setting. Eligibility criteria included female sex, age 18+, diagnosis of stage II, IIIA or IIIB ESTNBC between 3/2008 and 3/2016, receipt of definitive surgical resection, and neoadjuvant systemic therapy with or without adjuvant systemic therapy. Descriptive methods were used to compare demographic and clinical characteristics and treatment patterns among patients who received neoadjuvant systemic therapy only (Neo) versus patients who received neoadjuvant plus adjuvant systemic therapy (Neo+Adj). Results: 308 patients were eligible for the study. There were 236 patients (76.6%) who received Neo, while 72 (23.4%) received Neo+Adj. Mean age was 52.1 (SD 11.3), with 86.4% (n=266) of patients 65 or younger. Patients were predominantly White (56.8%, n=175) followed by African American (35.7%, n=110). Demographic characteristics did not differ by treatment group. The majority of patients were stage II at initial diagnosis (66.6%, n=205) and had ductal histology (93.5%, n=288). Over half of all patients (55.8%, n=172) had mastectomy; 44.2% (n=136) had breast conservation surgery. The majority of patients (79.5%, n=245) had no comorbid conditions and most (82.8%, n=255) had grade 3 tumors. Overall, 42.2% were peri/postmenopausal (n=130) at diagnosis. Distribution of stage at diagnosis was significantly different between groups (p=0.013). The pattern of findings suggests that the Neo group had earlier stage disease. Over one-third of patients with Neo were stage IIA at diagnosis (36.0%, n=85). Nearly one-fifth of patients with Neo+Adj were stage IIIB at diagnosis (19.4%, n=14), compared to 8.5% of patients receiving Neo (n=20). A significant difference in surgery type by treatment group was observed (p=0.035). Patients with Neo had a lower rate of mastectomy (52.5%, n=124) compared to Neo+Adj (66.7%, n=48), likely as a result of earlier stage disease at presentation. Among the Neo group, the most common neoadjuvant regimen was cyclophosphamide, doxorubicin and paclitaxel (59.3%, n=140), followed by cyclophosphamide, doxorubicin and docetaxel (14.4%, n=34). Most (85.2%, n=201) patients receiving Neo received one regimen in the neoadjuvant setting, while 13.1% (n=31) received two regimens. Among the Neo+Adj group, the most common neoadjuvant regimens were cyclophosphamide, doxorubicin and paclitaxel (38.9%, n=28) and cyclophosphamide with doxorubicin (23.6%, n=17), while the most common adjuvant regimens were paclitaxel (22.2%, n=16) and capecitabine (12.5%, n=9). Most (70.8%) patients receiving Neo+Adj received one neoadjuvant regimen followed by a different adjuvant regimen. Conclusions: In this sample of real-world patients with ESTNBC, the majority of patients were diagnosed with stage II disease, with limited or no comorbidities. This evaluation of treatment patterns confirmed that real-world treatment is limited to chemotherapy, which is consistent with the NCCN guidelines. Citation Format: Whitney C Rhodes, Santosh Gautam, Amin Haiderali, Min Huang, Jan Sieluk, Karen E Skinner, Lee S Schwartzberg. Patient characteristics and real-world treatment patterns in patients with early-stage triple-negative breast cancer receiving neoadjuvant chemotherapy [abstract]. In: Proceedings of the 2019 San Antonio Breast Cancer Symposium; 2019 Dec 10-14; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2020;80(4 Suppl):Abstract nr P2-16-33.

Background: Standard of care treatment for early-stage triple-negative breast cancer (ESTNBC) is chemotherapy. Minimal research has examined real-world effectiveness outcomes in patients with ESTNBC. Methods: This retrospective observational study evaluated event-free survival (EFS) and overall survival (OS) in patients with ESTNBC from US community oncology practices. Eligible patients were female and age 18+ at the time of stage II, IIIA or IIIB ESTNBC diagnosis that occurred between 3/2008 and 3/2016. All eligible patients received definitive surgery following neoadjuvant systemic therapy, with or without adjuvant systemic therapy. Kaplan-Meier analysis and Cox regression models were used to evaluate EFS and OS from the start of neoadjuvant therapy. EFS events are recurrence, second primary malignancy or death, whichever occurred first. Results: Of 308 patients included in the study, 236 (76.6%) received neoadjuvant only (Neo); 72 (23.4%) received neoadjuvant plus adjuvant systemic therapy (Neo+Adj). Overall, mean age was 52.1 years (SD 11.3), 56.8% were White (n=175), 35.7% African-American (n=110), 7.4% other/undocumented (n=23). Demographic characteristics did not differ between treatment groups. Two-thirds of patients (66.6%, n=205) were stage II at diagnosis, 33.4% (n=103) were stage IIIA or IIIB at diagnosis (22.4% and 11.0%, respectively). Most (82.8%, n=255) had grade 3 tumors and 42.2% were peri/postmenopausal (n=130) at diagnosis. Evidence of pathologic complete response (pCR) was observed in 41.2% of patients (n=127), while 57.5% of patients (n=177) had residual disease, and 1.3% (n=4) had undocumented/other response status. pCR was observed in 47.5% of Neo and 20.8% of Neo+Adj patients. Median EFS for all patients was not reached while mean EFS was 55.6 months (SD 1.6). Stage at diagnosis, surgery type and BMI were significant predictors of an EFS event, with stage II patients having less risk than stage III patients (Hazard ratio [HR]: 0.496 p Median OS for all patients was not reached while mean OS was 70.3 months (SD 1.5). Stage at diagnosis, surgery type and BMI were significant predictors of OS; patients with stage II had a reduced risk of death compared to stage III patients (HR: 0.299, p Conclusions: This real-world study found that stage at diagnosis, surgery type and BMI were significant predictors of EFS and OS events from the start of neoadjuvant treatment. This study highlights the need for improved therapies to extend EFS and OS for patients with ESTNBC. Given the association between pCR and EFS and OS, further analyses will evaluate EFS and OS by pCR status for each group. Time to recurrence analyses are also planned. Citation Format: Whitney C Rhodes, Santosh Gautam, Amin Haiderali, Min Huang, Jan Sieluk, Karen E Skinner, Lee S Schwartzberg. Real-world outcomes in patients receiving neoadjuvant chemotherapy for early-stage triple-negative breast cancer [abstract]. In: Proceedings of the 2019 San Antonio Breast Cancer Symposium; 2019 Dec 10-14; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2020;80(4 Suppl):Abstract nr P2-16-12.

Lay abstract This study included 450 patients with early-stage triple-negative breast cancer treated in the USA at community oncology practices. Patients were female, 18 years or older, diagnosed with stage II, IIIA or IIIB breast cancer between March 2008 and March 2016, and the breast cancer was determined to be triple negative (i.e., negative for estrogen receptors, progesterone receptors and excess HER2 protein). The study looked at the treatments received, whether those treatments worked and the response to treatment at the time of surgery. The study findings align with findings from other studies that complete response in tissue samples is associated with improved clinical outcomes. Triple-negative breast cancer remains challenging to treat, and there is a clear need for innovation in treatment options. Intervening in the early stages of triple-negative breast cancer is critical to improving outcomes.

Aim: To examine real-world treatment patterns and outcomes in neoadjuvant and adjuvant settings for early-stage triple-negative breast cancer (TNBC). Patients & methods: Using the Surveillance, Epidemiology, and End Results-Medicare database, we identified patients (≥65 years) with newly diagnosed stage II/III TNBC in 2010–2015 who had surgery plus neoadjuvant and/or adjuvant (systemic and/or radiation) therapy. Treatment, survival, healthcare resource use and costs were assessed through 2016. Results: Of 1569 patients (>99% women), 6%/74%/20% received neoadjuvant-only/adjuvant-only/both (neo + adj) therapies, respectively. Median overall survival was 23 months/not reached (NR)/78 months, with longer survival at stage II (NR/NR/78 months) than stage III (22/43/38 months). Mean per patient per month costs were $10,620 and $17,872 in neoadjuvant and adjuvant periods. Conclusion: These findings provide insights into clinical and economic outcomes for early-stage TNBC in 2010–2016.

Importance Management of high-risk non-muscle-invasive bladder cancer (NMIBC) represents a clinical challenge due to high failure rates despite prior bacillus Calmette-Guerin (BCG) therapy. Objective To describe real-world patient characteristics, long-term outcomes, and the economic burden in a population with high-risk NMIBC treated with BCG therapy. Design, Setting, and Participants This retrospective cohort study identified 412 patients with high-risk NMIBC from 63 139 patients diagnosed with bladder cancer who received at least 1 dose of BCG within Department of Veterans Affairs (VA) centers across the US from January 1, 2000, to December 31, 2015. Adequate induction BCG therapy was defined as at least 5 installations, and adequate maintenance BCG therapy was defined as at least 7 installations. Data were analyzed from January 2, 2020, to January 20, 2021. Exposures Intravesical BCG therapy, including adequate induction BCG therapy, was defined as at least 5 intravesical instillations of BCG within 70 days from BCG therapy start date. Adequate maintenance BCG therapy was defined as at least 7 installations of BCG within 274 days of the start (the first instillation) of adequate induction BCG therapy (ie, adequate induction BCG plus some form of additional BCG). Main Outcomes and Measures The Kaplan-Meier method was used to estimate outcomes, including event-free survival. All-cause expenditures were summarized as medians with corresponding interquartile ranges (IQRs) and adjusted to 2019 USD. Results Of the 412 patients who met inclusion criteria, 335 (81%) were male and 77 (19%) were female, with a median age of 67 (IQR, 61-74) years. Follow-up was 2694 person-years. A total of 392 patients (95%) received adequate induction BCG therapy, and 152 (37%) received adequate BCG therapy. For all patients with high-risk NMIBC, the 10-year progression-free survival rate and disease-specific death rate were 78% and 92%, respectively. Patients with carcinoma in situ (Cis) had worse disease-free survival than those without Cis (hazard ratio [HR], 1.85; 95% CI, 1.34-2.56). Total median costs at 1 year were $29 459 (IQR, $14 991-$52 060); at 2 years, $55 267 (IQR, $28 667-$99 846); and at 5 years, $117 361 (IQR, $59 680-$211 298). Patients with progressive disease had significantly higher median 5-year costs ($232 729 [IQR, $151 321-$341 195] vs $94 879 [IQR, $52 498-$172 631]; P

Purpose Regulators approve written medical information for healthcare professionals and consumers, but the consistency of these sources has not been studied. We investigated the consistency of information regarding four cardiovascular risks of attention-deficit/hyperactivity disorder (ADHD) medications approved in four countries. Methods Professional and consumer product labeling for five ADHD medications approved in Australia, Canada, the UK, and the USA were obtained in March/April 2016. Language describing the relationship between medication and elevated blood pressure and/or heart rate, myocardial infarction, stroke, and sudden death was extracted verbatim and classified into one of four categories based on the described relationship between medication and adverse event: “confirmed,” “unconfirmed,” “mixed,” and “not mentioned.” We judged the consistency of messages delivered to healthcare professionals and consumers as either “consistent” or “inconsistent.” Results We obtained 20 healthcare professional labels and 20 corresponding consumer labels for the five ADHD medications registered in all four countries. Not all professional and consumer labeling contained language regarding all four adverse events. Of the 80 theoretically evaluable drug-risk pairs, 38 (48%) were not evaluable because of absence of mention of the adverse event in the consumer label. For the remaining 42, the potential causal relationship was expressed consistently in professional and consumer labeling in 25 (60%) cases. The cardiovascular risk profile was not described consistently across all four countries for any of the five drugs. Conclusions Product labeling provides healthcare professionals and consumers with inconsistent messages regarding the potential causal relationship between stimulant use and specific cardiovascular risks in children and adolescents. Copyright © 2017 John Wiley & Sons, Ltd.

Introduction Management of high-risk non-muscle invasive bladder cancer (NMIBC) represents a clinical challenge due to high failure rates despite prior bacillus Calmette-Guerin (BCG) therapy. We describe real-world patient characteristics, long-term outcomes, as well as the economic burden in a high-risk NMIBC population. Methods We retrospectively identified a random sample of 412 high-risk NMIBC patients who received ≥ 1 dose of BCG within Veterans Affairs (VA) centers from Jan 1, 2000, to Dec 31, 2016. HR NMIBC was defined as high-grade Ta (TaHG), T1, and/or carcinoma-in-situ (CIS). Adequate BCG induction included at least 5 of 6 instillations, and adequate BCG therapy was at least 7 instillations. We used the Kaplan-Meier method to estimate outcomes including event-free survival. All-cause expenditures were summarized as medians with corresponding interquartile ranges (IQR) and adjusted to 2019 USD. Results The total follow-up was 2,694 person-years. At high-risk NMIBC diagnosis, 69 (17%) patients had CIS +/- T1 or TaHG, and 341 (83%) had TaHG or T1, no CIS. A total of 392 (95%) patients received adequate BCG induction and 152 (37%) patients received adequate BCG therapy. Recurrence and progression were observed in 61 (32%) and 71 (17%) patients, respectively. There were 166 deaths during follow-up, of which 27 (7%) patients died from bladder cancer. Total median costs at 1, 2 and 5-year were $29,459 ($14,991-$52,060), $55,267 ($28,667-$99,846), and $117,361 ($59,680-$211,298), respectively. Patients which progressed had significantly higher costs (5-yr, $232,729 vs. $94,879, p Conclusions In an equal access setting, the vast majority of BCG-treated patients with high-risk NMIBC received adequate BCG induction; however, less than 50% of patients received adequate maintenance therapy. Patients with CIS had increased risk of progression which was associated with significantly increased costs up to 5-yrs after diagnosis. These findings associated with $366,857 per patient that underwent subsequent radical cystectomy further highlight the considerable economic burden of managing BCG treated high-risk NMIBC.

e12512 Background: In the US, triple-negative breast cancer (TNBC) represents about 10–20% of breast cancers. Current information about the clinical and economic burden of early-stage TNBC in elderly patients is lacking. Methods: We used the SEER-Medicare database to identify patients with continuous Medicare Parts A/B enrollment, ≥66 years old, newly diagnosed between 2010 - 2015 (followed until 2016) with stage II-III TNBC, who initiated systemic neoadjuvant and/or adjuvant (including chemotherapy and radiation) therapy. Overall survival (OS) and event-free survival (EFS) from diagnosis were estimated using Kaplan-Meier (KM). Healthcare costs were determined during neoadjuvant and adjuvant periods. Results: Of 1569 patients ( > 99% women), 94 (6%) received neoadjuvant therapy, 1162 (74%) received adjuvant therapy, and 313 (20%) received both (neo/adj; Table). Age and race/ethnicity distributions were comparable in the three cohorts. Primary tumor T stage was T1c/T2 for 43%, 83%, and 58% in neoadjuvant, adjuvant, and neo/adj, respectively, and T3 for 14%, 10%, and 15%, respectively. The most common systemic regimens in both neoadjuvant and adjuvant periods were a taxane +/- anthracycline; 21% and 67% of patients in adjuvant and neo/adj cohorts received radiation therapy after surgery. Most claims were for outpatient treatment; hospitalizations were uncommon. The total mean expenditures per patient per month were US$10,620 and $24,408 during neoadjuvant and adjuvant periods, respectively. Conclusions: This study provides insights into patient characteristics, as well as clinical and economic outcomes for elderly patients with early-stage TNBC, treated from 2010-2016 in the US, highlighting the high monetary burden of TNBC and poor outcomes associated with stage III patients. [Table: see text]

e17031 Background: Management of high-risk non-muscle invasive bladder cancer (HR NMIBC) represents a clinical challenge due to high failure rates despite prior bacillus Calmette-Guérin (BCG) therapy. We describe real-world patient characteristics, long-term outcomes, as well as the economic burden in the HR NMIBC population. Methods: We identified a random sample of 412 HR NMIBC patients who received ≥ 1 dose of BCG within Veterans Affairs (VA) centers across the United States from January 1, 2000, to December 31, 2016. HR NMIBC was defined as high-grade Ta (TaHG), T1, and/or carcinoma-in-situ (CIS). We analyzed the number of BCG instillations received, as well as used the Kaplan-Meier method to estimate event-free survival for cystectomy and bladder cancer-specific death. All-cause expenditures were summarized as medians with corresponding interquartile ranges (IQR) and adjusted to 2019 USD. Results: The median (IQR) age at diagnosis was 67 years (61-74), with most patients being white (84%) and male (81%). At HR NMIBC diagnosis, 69 (17%) patients had CIS +/- T1 or TaHG, and 341 (83%) had TaHG or T1, no CIS. The vast majority of patients [n = 363, (90%)] received six BCG instillations (range: 1-7) within 365 days of the first dose. The total follow-up was 2,694 person-years. From BCG initiation to end of follow-up, the median all-cause expenditures per patient were $358,593 (257,699 – 652,853). Conclusions: In this equal access setting, the vast majority of HR NMIBC patients received 6 instillations of BCG within 1 year, although the interval over which the instillations were given varied among patients. Patients with CIS appeared to have a worse prognosis, as 24% underwent cystectomy, and 13% died of bladder cancer at 10 years of follow-up. These findings also highlight the considerable economic burden of HR NMIBC. [Table: see text]

Background: This study is the first to utilize a large claims database to estimate medical costs of patients with alpha-1 antitrypsin deficiency (AATD) in the United States. Methods: Adult AATD patients were identified from the OptumLabs™ Data Warehouse. Insurer and patient out-of-pocket costs were categorized into the following cost buckets, stratified by augmentation therapy use: physician visits (PV), emergency department visits (ED), inpatient stays (IP), augmentation therapy (AUG), other prescription drug costs (RX), and other costs (OTH). Costs were weighted and adjusted to 2017 U.S. dollars using the medical care component of the consumer price index. Results: The study cohort consisted of9117 AATD patients followed for 53,872 person years observed between 1993 and 2015. The annual costs among AATD patients totaled $127,537 among augmentation therapy users and $15,874 among non-users. The major drivers of annual costs to the insurer among the 7975 patients not on augmentation therapy were: PV: $5352 (37.7%) and IP: $4506 (31.8%). Among the 1142 augmentation users, major annual cost drivers to the insurer were PV: $15,064 (12.3%) and AUG: $82,002 (66.7%). Annual patient out-of-pocket costs were $4601 (AUG: $2084 [45.3%]; RX: $940 [20.4%]) and $1689 (PV: $727 [43.0%]; RX: $589 [34.9%]) among augmentation therapy users and non-users, respectively. Averaged across the entire cohort, the average annual costs per AATD patient were $22,975, paid by insurers ($21,100) and patients ($1875). Conclusions: Annual medical costs among patients with AATD are $127,537 and $15,874 among augmentation therapy users and non-users, respectively, with 75.3% of the cost difference attributable to AUG.

Regulators approve written medical information for healthcare professionals and consumers, but the consistency of these sources has not been studied. We investigated the consistency of information regarding four cardiovascular risks of attention-deficit/hyperactivity disorder (ADHD) medications approved in four countries.Professional and consumer product labeling for five ADHD medications approved in Australia, Canada, the UK, and the USA were obtained in March/April 2016. Language describing the relationship between medication and elevated blood pressure and/or heart rate, myocardial infarction, stroke, and sudden death was extracted verbatim and classified into one of four categories based on the described relationship between medication and adverse event: "confirmed," "unconfirmed," "mixed," and "not mentioned." We judged the consistency of messages delivered to healthcare professionals and consumers as either "consistent" or "inconsistent."We obtained 20 healthcare professional labels and 20 corresponding consumer labels for the five ADHD medications registered in all four countries. Not all professional and consumer labeling contained language regarding all four adverse events. Of the 80 theoretically evaluable drug-risk pairs, 38 (48%) were not evaluable because of absence of mention of the adverse event in the consumer label. For the remaining 42, the potential causal relationship was expressed consistently in professional and consumer labeling in 25 (60%) cases. The cardiovascular risk profile was not described consistently across all four countries for any of the five drugs.Product labeling provides healthcare professionals and consumers with inconsistent messages regarding the potential causal relationship between stimulant use and specific cardiovascular risks in children and adolescents. Copyright © 2017 John WileySons, Ltd.