AbstractTreatment for pulmonary arterial hypertension (PAH) has evolved over the past decade, including approval of new medications and growing evidence to support earlier use of combination therapy. Despite these changes, few studies have assessed real‐world treatment patterns, healthcare resource utilization (HCRU), and costs among people with PAH using recent data. We conducted a retrospective cohort study using administrative claims from the HealthCore Integrated Research Database®. Adult members with claims for a PAH diagnosis, right heart catheterization, and who initiated PAH treatment (index date) between October 1, 2015 and November 30, 2020 were identified. Members had to be continuously enrolled in the health plan for 6 months before the index date (baseline) and ≥30 days after. Treatment patterns, HCRU, and costs were described. A total of 843 members with PAH (mean age 62.3 years, 64.2% female) were included. Only 21.0% of members received combination therapy as their first‐line treatment, while most members (54.6%) received combination therapy as second‐line treatment. All‐cause HCRU remained high after treatment initiation with 58.0% of members having ≥1 hospitalization and 41.3% with ≥1 emergency room visit. Total all‐cause costs declined from $15,117 per patient per month at baseline to $14,201 after treatment initiation, with decreased medical costs ($14,208 vs. $6,349) more than offsetting increased pharmacy costs ($909 vs. $7,852). In summary, despite growing evidence supporting combination therapy, most members with PAH initiated treatment with monotherapy. Total costs decreased following treatment, driven by a reduction in medical costs even with increases in pharmacy costs.

OBJECTIVES:Population-adjusted comparisons of progression-free survival (PFS) from single-arm trials of cancer treatments can be derived using matching-adjusted indirect comparisons (MAICs); however, results are still susceptible to bias, particularly if the trials had different tumor assessment schedules. This study aims to assess the effects of assessment-schedule matching (ASM) on the relative effectiveness on the PFS of avelumab versus approved comparator immunotherapies or chemotherapy after population matching in the second-line (2L) setting for metastatic urothelial carcinoma.METHODS:The MAIC used patient-level data for avelumab from the JAVELIN Solid Tumor trial (NCT01772004). PFS was compared with published curves for other treatments to obtain population-adjusted hazard ratios (HRs). The MAIC was repeated after conducting ASM for differences in tumor assessment scheduled first at 6 weeks for avelumab and durvalumab and at 8 or 9 weeks for other treatments.RESULTS:MAIC adjustment alone altered the HR estimates up to 23%, whereas MAIC plus ASM resulted in up to 32.7% reductions from naive comparisons. Even in cases in which MAIC had little effect, ASM brought an additional change of 11.1% to 15.4%. Overall, the HR range of avelumab versus other treatments changed from 0.83 to 1.25 for naive comparisons to 0.76 to 0.99 after ASM plus MAIC, numerically favoring avelumab.CONCLUSIONS:Small variations in assessment schedules can introduce bias in unanchored indirect treatment comparisons of interval-censored time-to-event outcomes. In this study, adjusted PFS was comparable across second-line urothelial carcinoma treatment options, numerically favoring avelumab versus immunotherapies and chemotherapy agents. Correcting this bias is especially important when HRs are applied in cost-effectiveness models to transition patients between states.

The aim of this study was to identify cost drivers in Pulmonary Arterial Hypertension (PAH) in the US through a systematic literature review of economic and humanistic burden. A systematic literature review was conducted adhering to methodology published by the Cochrane Collaboration and the National Institute for Health and Clinical Excellence (NICE) in the UK. Population, Intervention & Comparators, Outcomes and Study Design (PICOS) criteria were used to identify resource utilization and economic burden studies in PAH published from credible databases (EMBASE, MEDLINE, MEDLINE In-Process) over the last 10 years. A total of 170 publications were extracted with most publications from the US. Total all-cause costs per patient per month (PPPM) in the US (all costs reported in 2019 $USD) were found to range from $2,494 to $11,037, driven primarily by medical costs. Medical costs accounted for a larger portion of total costs than pharmacy costs within both all-cause ($2,113 to $7,625 PPPM) and PAH-related ($293 to $5,086 PPPM) health care costs; among all-cause medical cost components, inpatient admission costs ($1,115 to $2,146 PPPM) contributed the highest costs, followed by outpatient costs ($954 to $1,944 PPPM). Although total costs increased after initiation of PAH therapies, increased pharmacy costs were partially offset by reduced medical costs ($1,506 to $57,409 decreased annual medical costs from baseline). Management of PAH carries a considerable economic burden with the major cost drivers consisting of primarily medical costs. All-cause costs vary widely in current literature, which may be due to heterogeneity in study population and cost components.

BACKGROUND:With increased use of immune checkpoint inhibitors (ICIs) among patients with cancer, there is substantial interest in understanding clinical and economic outcomes and management of immune-related adverse events (irAEs).PATIENTS, MATERIALS, AND METHODS:A retrospective study was conducted using Premier Healthcare Database, a U.S. national hospital discharge database, from March 1, 2015, through December 31, 2017. The database comprises more than 880 million inpatient and hospital-based outpatient encounters, with more than 200 million unique patients reported by 966 hospitals. Patients with four solid tumors known to benefit from ICI therapy were included. The list of irAEs assessed was defined a priori per American Society of Clinical Oncology clinical guidelines for irAE management. Baseline irAE-related inpatient and outpatient visits were defined as the first inpatient or hospital-based outpatient visit with discharge diagnosis of any irAE of interest following confirmed ICI usage within 90 days prior to the baseline visit. Patients were followed for 90 days after baseline irAE-related inpatient discharge date or outpatient visit date to assess irAE-related inpatient admissions, all-cause in-hospital mortality, ICI reinitiation, and to determine costs and health care resource utilization.RESULTS:Records from 673,957 patients with four tumor types were reviewed for ICI therapy. Of 13,030 patients receiving ICIs, approximately 40% experienced at least one irAE, with a total of 10,121 irAEs occurring within 90 days of the ICI visit. The most frequent (>1,000 events) irAEs were anemia, impaired ventricular function with heart failure and vasculitis, thrombocytopenia, thyroid conditions, and peripheral edema. As might be expected, compared with those with baseline irAE-related outpatient visits, patients with baseline irAE-related inpatient visits had a significantly higher percentage of irAE-related inpatient admissions (23% vs. 14%) and all-cause in-hospital mortality (22% vs. 6%) and lower reinitiation of ICI therapy (31% vs. 71%). Baseline irAE-related inpatient visits had significantly higher mean costs ($29,477 vs. $5,718) with longer hospital stays (12.6 vs. 7.8 days).CONCLUSION:Findings from a U.S. national hospital discharge database suggest that irAEs in patients treated with ICIs are common, occur in multiples and with greater frequency in those with pre-existing comorbidities. Those with inpatient admissions have poorer outcomes.IMPLICATIONS FOR PRACTICE:The present work addressed the knowledge gap in understanding real-world outcomes of immune-related adverse events (irAEs) associated with immune checkpoint inhibitors (ICIs). Patients who experienced irAEs had significantly higher baseline comorbidities and were more likely to have immune-related or immune-compromised comorbid conditions. Patients with baseline irAE-related hospitalizations were more likely to be rehospitalized and to experience in-hospital mortality and less likely to reinitiate ICI treatment. Real-world patients are more diverse than clinical trials, and clinicians should consider both the efficacy and safety profile of ICI treatments, especially for patients with comorbidity conditions. Close monitoring is needed after patients have experienced an irAE.

Substantial unmet needs exist among patients with metastatic renal cell carcinoma (mRCC). This retrospective study evaluated treatment patterns as well as clinical and economic outcomes associated with first-line monotherapy among patients with mRCC in the USA. Newly diagnosed patients with mRCC initiating at least one first-line therapy (1L) from 1 October 2013 to 31 March 2018 (index date = 1L start date) were identified from the US Veterans Health Administration database. Treatment patterns, clinical outcomes (time to next treatment [TNT] defined by earliest of switch to non-index therapy or re-initiation of index therapy after a more than 90-day gap, time to treatment discontinuation [TTD], overall survival [OS]), and costs were evaluated among patients treated with tyrosine kinase inhibitors (TKI), mammalian target of rapamycin inhibitors (mTOR), immune checkpoint inhibitors (ICI), and other monotherapies. Standard descriptive statistics were presented. The Kaplan–Meier method was used to evaluate clinical outcomes. Of 759 patients (median age 68.0 years), 85.0%, 8.0%, 4.3%, and 2.6% were treated with TKI, mTOR, ICI, or other therapy in 1L, respectively. Advancement rates (to second-line [2L] therapy) ranged from 10.0 (ICI) to 45.1 per 100 person years (TKI). The 12-month OS rates ranged from 47.4% (TKI) to 67.7% (mTOR). The median TNT ranged from 3.8 (mTOR) to 9.6 months (ICI), and median TTD ranged from 2.3 (mTOR) to 4.7 months (TKI). Total all-cause mean costs per patient per month ranged from $12,466 (mTOR) to $19,812 (ICI). These results indicate high unmet medical needs among patients with mRCC treated with 1L monotherapies. Novel combination therapies (e.g., ICI + ICI, ICI + TKI) may improve front-line outcomes for patients with poor prognoses.

Purpose Merkel cell carcinoma (MCC) is a rare, aggressive skin cancer with poor prognosis. This study compared patient characteristics, comorbidities, adverse events (AEs), treatment persistence, healthcare resource utilization (HRU) and costs in patients with metastatic MCC (mMCC) treated with immune checkpoint inhibitors (ICIs) or recommended chemotherapy per 2018 National Comprehensive Cancer Network (NCCN) Guidelines. Patients and Methods A retrospective, observational study was conducted using data from 3/1/2015 through 12/31/2017 from the Premier Healthcare Database, a US hospital discharge database. The study included patients aged ≥12 years with International Classification of Diseases Codes for MCC and metastasis, categorized by their first treatment (index) during the study period (ICI or NCCN-recommended chemotherapy [chemotherapy]). Patient, hospital, and visit characteristics were assessed at the index date and Charlson Comorbidity Index (CCI) score and comorbidities during a 6-month look-back period. Clinical outcomes, including AEs and treatment persistence were assessed over 90 days and HRU and costs over 180 days post-index. Results Of 75 patients with mMCC receiving ICIs (n=37) or chemotherapy (n=38), mean age was ≈73 years, and 21.3% had a history of immune-related (IR) conditions. Overall, ICI- and chemotherapy-treated patients were similar in most baseline characteristics, IR comorbidities, and CCI score. However, more ICI patients (46%) than chemotherapy patients (26%) persisted on treatment over 90-day follow-up, odds ratio (95% CI): 2.04 (0.93, 4.47), P=0.07. Over 180-day follow-up, 33% of patients had an inpatient admission with mean length of stay (LOS) ≈2 days shorter for ICI vs chemotherapy (not statistically significant). Total costs, primarily driven by pharmacy costs, were higher for ICIs than chemotherapy; other departmental costs were similar between treatment groups. Conclusion In a real-world setting, patients with mMCC receiving ICIs had higher treatment persistence over 90 days, shorter inpatient LOS and similar departmental cost (excluding pharmacy cost) than those receiving chemotherapy.

573 Background: Population-adjusted comparisons of PFS from single-arm trials of cancer treatments can be derived using matching-adjusted indirect comparison (MAIC); however, results are still susceptible to bias, particularly if the trials had different tumor assessment schedules (favoring longer intervals). This is shown with an MAIC of avelumab vs atezolizumab, nivolumab, durvalumab, pembrolizumab or chemotherapy in 2L UC. Methods: The MAIC used patient-level data for avelumab from the JAVELIN Solid Tumor trial (NCT01772004) and compared PFS with published curves for other treatments to obtain population-adjusted hazard ratios (HRs). The first assessment was scheduled at 6 weeks for avelumab and durvalumab and at 8 or 9 weeks for the others. The MAIC was repeated after aligning the first assessment time for avelumab to each comparator’s schedule with the Assessment Schedule Matching (ASM) method. Results: Naïve analyses (Table) suggest similar PFS for avelumab vs comparators. MAIC based on population adjustment (PA) altered the HR of avelumab vs atezolizumab and nivolumab (23.0% and 9.2% reductions in HR, respectively) but had little effect in other comparisons. ASM adjustment led to further changes in HR (13.2-32.7% reductions from naïve) favoring avelumab in MAICs where schedules differed. Of note, assessment schedule differences fully accounted for differences in PFS between avelumab and pembrolizumab. Conclusions: Indirect comparisons of PFS from single-arm trials must include adjustment for differences in both population characteristics and tumor assessment schedules. In this 2L UC MAIC, adjustment reversed some naïve comparisons, revealing more favorable PFS with avelumab up to 30%. Clinical trial information: NCT01772004. [Table: see text]

PURPOSE:Clinical trial evidence has affirmed the role for immuno-oncology (IO) treatment for locally advanced or metastatic urothelial carcinoma (la/mUC). This Study informing treatment Pathway dEcision in bladder cAnceR (SPEAR-Bladder) aimed to provide insight into the optimal sequencing of IO treatments among la/mUC patients treated in the US Oncology Network. PATIENTS AND METHODS:This was a retrospective analysis of adult patients with la/mUC who initiated first-line chemotherapy followed by either IO therapy (C-IO subgroup) or chemotherapy (C-C subgroup) between 01/01/2015 and 04/30/2017 and included a potential follow-up period through 06/30/2017. Data were sourced from iKnowMed electronic health records. Patient and treatment characteristics were assessed descriptively, with Kaplan-Meier methods used to evaluate time-to-event outcomes, including overall survival (OS). RESULTS:A total of 117 patients were included in this analysis (median age 69 years, 74.4% male, 88.0% Caucasian): 79 and 38 patients were in the C-IO and C-C subgroups, respectively. The median OS was 19.2 months among patients who received the C-IO sequence and 11.9 months among those who received the C-C treatment sequence. CONCLUSION:These results suggest that patients who received the C-IO treatment sequence had notable improvement in OS compared with those who received the C-C sequence. In light of the rapidly evolving therapeutic landscape, further investigation will be required to determine how best to select the optimal therapeutic regimen and sequencing for patients with la/mUC.

Aim: Retrospectively assessed treatment patterns and clinical and economic outcomes in Merkel cell carcinoma (MCC) patients receiving recommended first-line regimens. Materials & methods: MCC patients newly treated with either immune checkpoint inhibitors (ICIs) or chemotherapies (CTs) were selected from the Veterans Health Administration database (2013-2018); 74 patients (ICIs: 20 and CTs: 54) were selected. Results: Median duration of therapy was 300 days for ICIs and 91 days for CTs. Time to next treatment was 245 and 184 days, respectively. Mean total (per patient per month) costs were $15,306 (ICIs) and $10,957 (CTs), of which 51% and 86%, respectively, were non-MCC therapy-related costs. Conclusion: Despite higher costs, utilization of ICIs in first-line MCC shows clinical advantages over CTs in the real world.

Merkel cell carcinoma (MCC) is a rare and aggressive type of cancer with poor outcomes. To describe treatment patterns, overall survival, and healthcare costs associated with advanced MCC (aMCC) using data from Medicare enrollees who received an aMCC diagnosis in the USA States between 2006 and 2013. Surveillance, Epidemiology, and End Results (SEER)-Medicare data from 2006 to 2013 were used to describe treatment patterns, 1- and 5-year overall survival, and total healthcare costs for the periods 12 months before aMCC diagnosis and 4–12 months afterward in patients aged ≥ 65 years. We identified 257 patients with an aMCC diagnosis, of whom 51% had stage IIIb disease and 49% had stage IV. Within 4 months after diagnosis, 84% of patients (n = 216) received treatment; 45% (n = 115) received surgery, 48% (n = 124) radiation therapy, and 31% (n = 80) chemotherapy. Second-line chemotherapy was administered in 33% of patients (n = 26) receiving first-line chemotherapy. Median overall survival was 27 months in patients whose aMCC was diagnosed at stage IIIb and 12 months in patients whose aMCC was diagnosed at stage IV. Median total 12-month direct healthcare costs were US$48,006 (25th–75th percentile range = US$30,594–US$69,797) per patient. Total costs were highest in patients receiving chemotherapy, either alone or combined with radiation and/or surgery (US$52,854; 25th–75th percentile range = US$34,473–US$71,987). Most patients with aMCC received initial treatment, including surgery, radiation, and/or chemotherapy, and approximately one-third of those receiving chemotherapy received second-line chemotherapy. Total 12-month direct healthcare costs were highest in patients who received chemotherapy alone or combined with radiation and/or surgery. These poor survival results and high treatment costs highlight the need for effective new aMCC therapies.

Despite the increased use of ICIs among cancer patients, there is limited understanding in real-world economic outcomes resulting from ICI use. This study examined the costs and HRU associated with irAEs in patients receiving ICIs. An observational study was conducted using Premier Healthcare Database, a US national hospital discharge database (Mar 2015-Dec 2017). Twenty-eight distinct types of irAEs per the ASCO practice guidelines were identified. Patients with ICD-9/10 codes for non-small cell lung cancer, renal cell carcinoma, urothelial carcinoma, or Merkel cell carcinoma and at least one irAE during the study period were included. Index inpatient or hospital-based outpatient irAE encounters were defined as the first encounter with a confirmed ICI usage during a 90-day look-back period. Patients were then followed for 90 days post-irAE to determine irAE-related costs and HRU. Among 13,030 ICI patients, 51% experienced at least one irAE, with 15,277 distinct irAEs occurred across these patients. 38% of irAE events were inpatient based while the rest were outpatient based encounters. During the follow-up period, the mean±SD cost was $21,219±25,297 for inpatient index irAEs and $5,951±12,654 for outpatient index irAEs, p<0.001. The mean±SD readmission length of stay (LOS) was 9.1±9.4 days for inpatient index irAEs, and 7.7±7.2 days for outpatient index irAEs, p<0.001. Overall, the highest mean costs were for transverse myelitis ($80,976), toxic epidermal necrolysis ($69,115) and myocarditis ($45,341), while the lowest were for acquired hemophilia ($314), adrenal insufficiency ($3,974) and polymyalgia-like syndrome ($4,395). Approximately half of patients receiving an ICI experienced an irAE that required inpatient or hospital-based outpatient treatment. Patients with inpatient index encounters for irAEs subsequently had significantly higher costs and LOS during the follow-up compared to patients with outpatient index encounters. Future studies should evaluate differences in irAEs between different ICIs to enable patient centered treatment choices.

139 Background: MCC is a rare and aggressive skin cancer with annual incidence of 2,500 in the U.S. Checkpoint inhibitors (CPIs) demonstrated durable disease response historically not seen with chemotherapy among MCC patients in clinical trials. Avelumab, a PD-L1 antibody, became the first and only approved treatment for metastatic MCC by the U.S. FDA in March 2017. This study evaluated emerging treatment patterns among MCC patients using the US Veterans Health Administration (VHA) database. Methods: This retrospective study identified newly-diagnosed MCC patients who initiated ≥1 systemic therapy from Oct. 2013 to Jan. 2018 and had continuous enrollment from ≥6 months pre-initial MCC diagnosis date until ≥2 months post-index date with follow-up until the earliest of death, disenrollment, or study end. The index date was defined as the start date of first-line (1L) systemic therapy. A subsequent line of therapy (2L) was defined by the earliest of new therapy addition, regimen switch, or a new/existing regimen after a >60-day gap from the prior cycle end. Duration of 1L therapy was evaluated among patients treated with CPIs, National Cancer Comprehensive Network (NCCN)-recommended chemotherapy (recCTs), and other chemotherapy (oCTs). The median duration of therapy was estimated using the Kaplan Meier method. Results: Of the 120 MCC patients (72% pre-2017) with 1L systemic therapy, 17%, 45%, and 38% were treated with CPIs, recCTs and oCTs, respectively. More than 75% were white men aged ≥65 years with mean baseline Charlson comorbidity index scores ≥5. Carboplatin-etoposide (56%) and cisplatin-etoposide (26%) were the most commonly-used recCTs. The median duration of 1L therapy in patients treated with CPI, recCTs and oCTs was 300, 91, and 21 days, respectively. 2L therapy was initiated in 33% of all 1L patients. Conclusions: Among 1L MCC patients, CPIs treated patients appear to be on treatment longer than both recCTs and oCTs patients. These descriptive findings provide important insights into the emerging real-world treatment patterns amongst MCC patients in the VHA population.

Aim: To conduct a value assessment of an immuno-oncology (IO) therapy for a rare cancer and evaluate whether existing frameworks consider challenges associated with valuing IOs for rare cancers. Materials & methods: Value frameworks developed by American Society of Clinical Oncologists, Memorial Sloan Kettering Cancer Center and National Comprehensive Cancer Network were used to estimate the value of an IO therapy in a rare cancer based on single-arm trial data and retrospective studies. Results: Paucity of direct evidence comparing rare cancer treatments and lack of acceptance of indirect comparisons hinder appropriate value assessment. Measurement of value based on short-term outcomes may not capture the value of IOs, where survival is often characterized by a plateau. Conclusion: Further work is required to factor in nuances associated with rare cancers and guide end users of the frameworks. To capture true value, multiple or more holistic value assessments are required.

Background The timing of efficacy-related clinical events recorded at scheduled study visits in clinical trials are interval censored, with the interval duration pre-determined by the study protocol. Events may happen any time during that interval but can only be detected during a planned or unplanned visit. Disease progression in oncology is a notable example where the time to an event is affected by the schedule of visits within a study. This can become a source of bias when studies with varying assessment schedules are used in unanchored comparisons using methods such as matching-adjusted indirect comparisons. Objective We illustrate assessment-time bias (ATB) in a simulation study based on data from a recent study in second-line treatment for locally advanced or metastatic urothelial carcinoma, and present a method to adjust for differences in assessment schedule when comparing progression-free survival (PFS) against a competing treatment. Methods A multi-state model for death and progression was used to generate simulated death and progression times, from which PFS times were derived. PFS data were also generated for a hypothetical comparator treatment by applying a constant hazard ratio (HR) to the baseline treatment. Simulated PFS times for the two treatments were then aligned to different assessment schedules so that progression events were only observed at set visit times, and the data were analysed to assess the bias and standard error of estimates of HRs between two treatments with and without assessment-schedule matching (ASM). Results ATB is highly affected by the rate of the event at the first assessment time; in our examples, the bias ranged from 3 to 11% as the event rate increased. The proposed method relies on individual-level data from a study and attempts to adjust the timing of progression events to the comparator’s schedule by shifting them forward or backward without altering the patients’ actual follow-up time. The method removed the bias almost completely in all scenarios without affecting the precision of estimates of comparative effectiveness. Conclusions Considering the increasing use of unanchored comparative analyses for novel cancer treatments based on single-arm studies, the proposed method offers a relatively simple means of improving the accuracy of relative benefits of treatments on progression times.

Merkel cell carcinoma (MCC) is a rare and aggressive form of skin cancer, with no US FDA approved treatment until 2017. Avelumab, a PD-L1 antibody, became the first FDA approved treatment for metastatic MCC (mMCC) in March 2017. Treatment guidelines from the National Comprehensive Cancer Network (NCCN) recommend CPIs including avelumab, nivolumab and pembrolizumab as treatment options for mMCC. This study is to assess the real-world treatment patterns and outcomes of mMCC patients receiving CPIs and chemotherapies. An observational study was conducted from March 1, 2015-December 31, 2017 using the Premier Healthcare Database (PHD). mMCC patients age ≥ 12 were identified by ICD-9/10 codes and included if the initial treatment received during the study period was a CPI or chemotherapy per NCCN guidelines. The initial treatment date served as the index date. Thirty-seven mMCC patients received CPIs and 38 chemotherapy, with 84% of patients receiving platinum based chemotherapy. Baseline characteristics were similar between the CPI and chemotherapy group for age, race and Charlson comorbidity index, while the CPI group trended towards having more males and more patients with commercial insurance. More CPI patients stayed on treatments than the chemotherapy group at day 30 (68% vs. 53%, p<0.001), 60 (54% vs. 42%, p<0.001) and 90 (46% vs. 26%, p<0.001). By day 90, 74% of patients who started on chemotherapy switched to a different treatment in comparison to 54% of CPI patients (p<0.001). A comprehensive list of 47 adverse events commonly associated with CPIs and chemotherapies were studied, and the average number of AEs was 1.2±1.5 for the CPI group and 1.6±1.7 for the chemotherapy group (p=0.35). The availability of CPIs for mMCC patients is practice changing. CPI patients treated in hospital-based settings had longer time-on-treatment, less treatment switching, and trended towards fewer adverse events vs. chemotherapy.

OBJECTIVE:To estimate the budget impact of introducing avelumab as a second-line (2L) treatment option for patients with locally advanced or metastatic urothelial cancer (mUC) from the perspective of a US third-party payer (commercial and Medicare).METHODS:A budget impact model (BIM) with a three-year time horizon was developed for avelumab. Efficacy and safety data were sourced from published literature and US package inserts. The analysis was conducted in collaboration with a specialist oncologist who validated clinical assumptions. Costs were based on the number of eligible patients, time-to-treatment failure, overall survival, adverse events (AEs), and projected market shares of various treatments.RESULTS:In a hypothetical commercial health plan of 30,000,000 members, 884 patients were estimated to be eligible for 2L treatment over a three-year time period. Without avelumab, the total cost for treating patients with mUC was estimated to be US$70,268,035. The introduction of avelumab increased total costs by $73,438 (0.10% increase). In a hypothetical Medicare health plan of 30,000,000 beneficiaries, a total of 4,705 patients were estimated to be eligible for 2L treatment. Without avelumab, the total cost for treating patients with mUC was estimated to be $292,923,098 from a Medicare perspective; however, with avelumab, there was an increase of $719,324 (0.25% increase) in total costs. Results of the sensitivity analyses demonstrated a cost-neutral impact across all tested scenarios from both perspectives.CONCLUSION:The BIM estimated that avelumab would have a cost-neutral impact within a US commercial and a Medicare health plan. Overall, avelumab can be an affordable and valuable treatment option for patients with locally advanced or mUC in the 2L setting. These findings demonstrate a consistently favorable budget impact in both populations. Further studies should be conducted to more comprehensively assess the clinical and economic implications of adding avelumab to the treatment armamentarium of 2L mUC.

Abstract Background There are limited data on the travel burden for cancer patients with rare tumor types, such as Merkel cell carcinoma (MCC). Objective The objective of this study was to understand the travel burden of MCC patients. Methods This study used data from an MCC registry at the Seattle Cancer Care Alliance (SCCA). All MCC patients enrolled at SCCA with a valid 3‐digit ZIP code were included. Patients were followed up from January 1, 2012 until their last follow‐up, death, or end of data (January 1, 2017). Travel burden was measured by one‐way travel distance to SCCA from each patient's 3‐digit ZIP code. Patient demographics, tumor characteristics, and follow‐up visit were evaluated and stratified by one‐way driving distance of ≤300 and >300 miles. Results A total of 391 MCC patients were included (68% men, mean age = 67 years [±SD = ±11 years], 67% residing in the West, and 70% white). At diagnosis, 53% of the patients had Stage III or IV MCC. Mean one‐way distance traveled by patients was 1,137 (median: 813) miles, and 57% of patients traveled >300 miles. Compared to patients who traveled ≤300 miles, those who traveled >300 miles were more likely to be <70 years old (46% vs 65%; P < 0.001), were diagnosed with advanced stage (III or IV) MCC (46% vs 59%; P = 0.01), had shorter follow‐up in the cancer registry (mean: 509 vs 212 days; P < 0.001), and had fewer visits during follow‐up (mean: 5.2 vs 2.5; P < 0.001). Conclusions In this single cancer center study, the majority of MCC patients trav‐eled long distances to receive expert care. Longer travel distances appeared to be associated with younger age, a more advanced stage of cancer at study entry and fewer in‐clinic visits, suggesting that travel burden may impact timely and adequate patient care for this rare cancer.

To assess treatment patterns in first-line (1L) setting among US Veterans Health Administration patients with metastatic renal cell carcinoma (mRCC). This retrospective study identified newly diagnosed adult patients with mRCC who initiated ≥1 line of therapy from 01Oct2013-31Dec2017 (index date=1L therapy start date) and had continuous health plan enrollment from ≥6 months prior to the mRCC diagnosis until ≥3 months post–index date. Second-line (2L) therapy was defined by switch to any non-index therapy. Duration of 1L therapy was measured from index date to the earliest of last prescription’s end date, 2L therapy start date, or follow-up end. Univariate Poisson regression was used to compare patients treated with tyrosine kinase inhibitor (TKI) therapy vs mechanistic target of rapamycin (mTOR), immuno-oncology (IO) therapy, or other therapy, separately, based on rates (in 100-person years) of continuation, permanent discontinuation, interruption, and switch to 2L therapy. Of 809 patients (mean age, 68.4y; 98.4% male; 79.9% white), 85.0%, 7.7%, 4.7%, and 2.6% were treated with TKI, mTOR, IO, or other therapy in 1L, respectively. Patients in the IO cohort were more likely to have baseline COPD (31.6% vs 16.7%; p=0.022) a higher continuation rate (47.7 vs 14.5; p<0.0001) and a lower rate of switching to 2L therapy (13.0 vs 50.3; p=0.0073) vs TKI cohort. Patients received mTOR had a higher rate of permanent discontinuation (49.0 vs 31.0; p=0.0334) and interruption (221.0 vs 122.9; p=0.0002). Duration of 1L therapy (in days) was similar for TKI vs. IO (153.3 vs133.2, p=0.3372), longer for TKI vs. mTOR (153.3 vs 96.9, p=0.0055) and TKI vs. other therapy (153.3 vs 42.9, p<0.0001). Among patients with mRCC, although 1L TKI therapy showed similar duration vs IO, IO therapy demonstrated higher continuation rates vs TKI. Future research should focus on understanding which patients would benefit from IO+TKI.