Background: Clinical practice guidelines recommend adjuvant therapy for patients with early nonsmall cell lung cancer (eNSCLC), especially those with lymph node metastasis. This study evaluated the prevalence of lymph node examination and its association with adjuvant treatment rates, overall survival (OS), and healthcare costs among United States (US) Medicare patients with resected eNSCLC. Methods: This retrospective observational cohort study used Surveillance, Epidemiology, and End Results cancer registry data linked with Medicare claims data. Eligible patients were aged >= 65 years with newly diagnosed non -small cell lung cancer (NSCLC) stages IA to IIIB [the American Joint Committee on Cancer (AJCC) Cancer Staging Manual , 7th edition] between January 2010 and December 2017 with surgery <= 1 month prior to or <= 12 months after diagnosis. Patients were grouped by lymph node examination status: no examination (pNX), examination and no metastasis (pN0), or metastasis staging in N1 (pN1) or N2 (pN2). OS and costs were evaluated by examination status and number of lymph node examined. OS was analyzed using extended Cox proportional hazards models for specific time periods and time interaction with examination status, and adjusted for patient characteristics. Adjusted post -surgical healthcare costs per patient per month (PPPM) were analyzed using gamma -log regression models. Results: Among the 14,648 patients included in the study, approximately 11% were pNX, whereas most were pN0 (68%), followed by pN1 (11%) and pN2 (10%). Adjuvant treatment rates were higher for pNX (35%) than pN0 (18%), but lower than pN1 (68%) and pN2 (74%) patients (P<0.001). Unadjusted OS for pNX patients was nearly identical to pN2, and significantly worse compared to pN0 and pN1 (P<0.0001). After adjusting for patient characteristics, pNX patients had higher risk of death relative to pN0 patients (P<0.001). Marginal mean adjusted total costs were comparable across pNX ($15,827 PPPM), pN0 ($12,712 PPPM) and pN1 ($17,089 PPPM), but significantly less for pN0 compared to pN2 ($23,566 PPPM) (P=0.002). Conclusions: Inadequate lymph node examination is associated with underutilization of adjuvant treatment and poor OS in resected NSCLC. In the current era of targeted and immunotherapies, lymph node examination is more important than ever, implicating the need for Quality Improvement practices and multidisciplinary coordination.

Randomized trials have demonstrated that anaplastic lymphoma kinase (ALK) tyrosine kinase inhibitors (TKIs) can be safe and efficacious treatments for patients with ALK-positive advanced non-small-cell lung cancer (aNSCLC). However, their safety, tolerability, effectiveness, and patterns of use in real-world patients remain understudied. We sought to assess the overall treatment pattern characteristics, safety, and effectiveness outcomes of real-world patients with ALK-positive aNSCLC receiving ALK TKIs. This retrospective cohort study using electronic health record data included adult patients with ALK-positive aNSCLC receiving ALK TKIs between January 2012 and November 2021 at a large tertiary medical center, University of California, San Francisco (UCSF), with alectinib or crizotinib as the initial ALK TKI therapy. Our primary endpoints included the incidence of treatment changes (treatment dose adjustments, interruptions, and discontinuations) during the initial ALK TKI treatment, the count and type of subsequent treatments, rates of serious adverse events (sAEs), and major adverse events (mAEs) leading to any ALK TKI treatment changes. Secondary endpoints included the hazard ratios (HRs) for median mAE-free survival (mAEFS), real-world progression-free survival (rwPFS), and overall survival (OS) when comparing alectinib with crizotinib. The cohort consisted of 117 adult patients (70 alectinib and 47 crizotinib) with ALK-positive aNSCLC, with 24.8

Background: Hemophilia A is a bleeding disorder characterized by a deficiency in factor VIII (FVIII). Routine prophylaxis with FVIII products or emicizumab is the recommended approach to management for people with hemophilia A (PwHA). The burden of lifelong prophylaxis treatments to prevent bleeding events for PwHA is substantial, impacting both the patients and the healthcare system. The objective of this study is to estimate the relative cost-effectiveness of these prophylactic therapies for PwHA, including the newly approved efanesoctocog alfa. Methods: A Markov model was developed with four FVIII-based health states (normal clotting function, mild hemophilia, moderate hemophilia, severe hemophilia) and death to compare emicizumab, efanesoctocog alfa, standard half-life (SHL) and other extended half-life (EHL) FVIII products as prophylaxis for PwHA in the United States (US). Advate and Eloctate data were used to represent SHL and EHL, respectively. The model considered a cohort of 38 years old hemophilia A patients with average weight of 94.4 kg treated with prophylaxis. Probability of experiencing each health state was based on each product's pharmacokinetic data, where FVIII activity levels were used to determine disease severity. Other clinical inputs, including annualized bleed rates and adverse event risks, were estimated from clinical trial data. Wholesale acquisition cost (WAC) of drugs and healthcare costs of bleed management and adverse events were included and were estimated from published literature. Utilities by disease severity were derived from published real-world health-related quality of life studies for hemophilia patients. Disutility of infusion was obtained from the published literature. Costs (in 2023 US dollars), quality-adjusted life-year (QALY), and total bleeds were estimated over a lifetime horizon. Cost-effectiveness was estimated as incremental cost per QALY gained. One-way and probabilistic sensitivity analyses were conducted. Results: Over a lifetime, emicizumab was estimated to be less costly (total cost at $17.0 million for emicizumab vs $24.0 million, $19.3 million, and $18.2 million for efanesoctocog alfa, SHL, and EHL) and more effective (18.61 vs 18.58, 16.91, and 17.33 QALY) compared with efanesoctocog alfa, SHL, and EHL FVIII, respectively. Although efanesoctocog led to fewer bleeds (26.58 vs 59.91 bleeds over a lifetime), PwHA on emicizumab were expected to incur fewer bleeds (59.91 vs 198.45, and 108.58 bleeds) compared with EHL and SHL. Results were robust to one-way and probabilistic sensitivity analyses. Conclusions: Findings in this cost-effectiveness analysis suggest that emicizumab is less costly and more effective (i.e., dominant) over a lifetime compared with available FVIII prophylaxis in pediatrics and adult PwHA in the US.

e20567 Background: To evaluate the effect of LN examination status on OS and healthcare costs in patients (pts) with eNSCLC. Methods: Retrospective observational study of pts with resected stage IA-IIIB NSCLC (AJCC 7th ed) from SEER data linked with Medicare claims. Eligible pts were ≥65 years at diagnosis (Jan 2010-Dec 2017), had surgery ≤1 month before or 12 months after diagnosis and were continuously enrolled in Medicare Parts A and B for ≥6 months before diagnosis and in Parts A, B and D up to 6 months post-surgery or date of death. Pts were analyzed by LN examination status: no examination (pNX), examination and no LN metastasis (pN0) and examination with N1 (pN1) or N2 (pN2) metastasis. Extended Cox proportional hazards models presented OS hazard ratios (HRs) for specific time periods. Total healthcare costs per patient per month (PPPM) were analyzed using a gamma-log regression model. Results: A total of 9448 pts were included: 996 pNX (11%), 6457 pN0 (68%), 1003 pN1 (11%) and 992 pN2 (10%). pNX pts were slightly older with lower median income, more comorbidities and more baseline smoking history vs other LN examination status cohorts. Stage distribution in pNX and pN0 pts appeared similar. Grade 3/4 tumors were less prevalent in pNX (n = 301, 30%) and pN0 (n = 1934, 30%) pts relative to pN1 (n = 482, 48%) and pN2 pts (n = 424, 43%; P< 0.001). Unadjusted OS was worse for pN1 and pN2 vs pN0 pts and was similar between pNX and pN2 pts. Adjusted OS showed higher risk of death for pN1 and pN2 vs pN0 pts; this risk decreased over time. pNX pts also had higher risk of death vs pN0 pts. Adjusted marginal mean follow-up costs were lowest for pN0 and highest for pN2 pts (All P < 0.001). Costs were higher, but not statistically significant ( P= 0.591) in pNX vs pN0 pts. The majority of total healthcare costs (62-65%) were due to inpatient services. Conclusions: Stage distribution in pNX pts was more similar to that of pN0 than pN1/2 pts, likely due to understaging from lack of LN examination. However, prognosis in pNX pts was nearly identical to that of pN2 pts, suggesting missed LN metastasis diagnosis in a substantial proportion of pNX pts. Despite worse OS in pNX vs pN0 pts, healthcare costs in pNX pts were not significantly higher, which may reflect underutilization of systemic therapy in pts with resected eNSCLC. [Table: see text]

Background: With the entry of gene therapy into the hemophilia A treatment landscape, healthcare decision makers are re-evaluating the relative value of different prophylaxis options for people with hemophilia A (PwHA) without inhibitors. While there are multiple elements of value to consider when conducting a clinical and economic assessment of treatments for PwHA, this study aims to answer questions about the relative cost-effectiveness of prophylactic therapies. Methods: A Markov model was used to estimate the cost-effectiveness of emicizumab compared to: valoctogene roxaparvovec gene therapy, standard half-life factor VIII (FVIII) , and extended half-life FVIII replacement products. Health states in the model included: life with no arthropathy, life with arthropathy, life after joint replacement surgery, and death. Parameter estimates, including annualized bleed rates and durability of treatment (Table 1), were estimated from clinical trial and real-world data. Costs included the wholesale acquisition cost of drugs, treatment of bleeds, and management of hemophilia-related joint disease, which were estimated from published literature. The cost of gene therapy was assumed to be a one-time cost of $2.5 million (in 2022 US Dollars) per treated patient. Utilities were derived from patient-reported outcomes in published real-world data. Incremental cost-effectiveness (ICER) was estimated in cost per quality-adjusted life-year (QALY) gained, from a payer perspective over a 2-year, 5-year, and a lifetime horizon. Results: Compared to gene therapy, emicizumab was estimated to be less costly (-$1.395 million) with similar effectiveness (0.00 QALY incremental difference) over a 2-year horizon, less costly and more effective over a 5-year horizon (-$125,215; 0.02 QALY), and more costly but cost-effective over a lifetime horizon ($8,945; 0.95 QALY; ICER $9,406/QALY). Compared to standard half-life FVIII prophylaxis and extended half-life FVIII prophylaxis, emicizumab was dominant (less costly and more effective) over each model time horizon. Conclusions: Emicizumab remains a highly cost-effective prophylaxis strategy compared with FVIII prophylaxis in adult PwHA. Uncertainties around the effectiveness and durability of gene therapy make assessment of 2 and 5-year cost-effectiveness challenging, but over a lifetime emicizumab appears to be cost-effective compared with gene therapy for PwHA. Given the uncertainties related to new therapies, additional sensitivity analyses are needed to estimate the value of therapies under different scenarios and to consider other elements of value that are important to PwHA. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

LN examination among eNSCLC patients (pts) is critical in fulfilling the IASLC complete resection criteria and in perioperative chemotherapy decisions. Previous studies have shown that LN non-examination is associated with overall survival rates similar to pts with LN metastasis, presumably due to inadequate adv treatment (tx). This study evaluated the prevalence of LN exam and adv tx in US Medicare eNSCLC pts. This retrospective observational study identified pts with stage IA-IIIB NSCLC (AJCC 7th ed) from SEER data linked with Medicare claims. Pts were ≥65 years at diagnosis between January 2010 and December 2017, had surgery within 1 month prior or 12 months after diagnosis and were continuously enrolled in Medicare Parts A and B ≥6 months before diagnosis. Additional continuous enrollment criteria of Medicare Parts A, B and D ≥6 months post-surgery or up to date of death was required for adv tx outcomes. Pts were grouped by LN exam status: none (pNX), no LN metastasis after LN exam (pN0) or LN metastasis after exam (pN1/2). Adv tx was identified within 6 months or up to death post-surgery. Descriptive statistics were used to summarize results. A total of 14,684 pts were included; 1596 (11%) were pNX, 9943 (68%) were pN0 and 3145 (21%) were pN1/2. The proportion of pNX pts decreased from 14% in 2010 to 8% in 2017. A mean (SD) of 11 (9) LNs were examined (median, 9; IQR, 5-15). Adv chemotherapy was identified in 21% (pNX), 13% (pN0) and 63% (pN1/2) of pts. Wedge resections were performed in 47% (pNX), 18% (pN0) and 11% (pN1/2) of pts. LN examination in Medicare pts with eNSCLC has improved over time with a sufficient number of excised LNs at resection. However, many resections continue to lack LN evaluation and pNX was associated with lower adv chemotherapy utilization rates than pN1/2, which further supports that pNX may negatively impact adv tx decisions.Table: 946PVariable, n (%)pNX n=1596pN0 n=9943pN1/2 n=3145Extent of lung cancer resectionPneumonectomy17 (1)150 (2)218 (7)Bilobectomy<11103 (1)71 (2)Lobectomy255 (16)6149 (62)1985 (63)Wedge resection746 (47)1795 (18)347 (11)Segmentectomy134 (8)873 (9)147 (5)Other surgery237 (15)141(1)71 (2)SurgeryRobotic-assisted thoracic surgery31 (2)307 (3)74 (2)Video-assisted thoracic surgery12 (<1)<11<11Thoracotomy192 (12)502 (5)216 (7)Sternotomy19 (1)21 (<1)32 (1)pNX n=996pN0 n=6476pN1/2 n=1999Received adj txChemotherapy211 (21)846 (13)1255 (63)Immunotherapy<11<1121 (1)Targeted therapy25 (3)43 (1)48 (2)Chemoradiation118 (12)197 (3)494 (25)Radiation245 (25)510 (8)652 (33) Open table in a new tab

BACKGROUND: Lung cancer is the leading cause of cancer-related deaths in the United States. Several anaplastic lymphoma kinase (ALK) rearrangement inhibitors have been approved for the treatment of metastatic ALK-positive non-small cell lung cancer (NSCLC). Effective disease management requires an understanding of how these treatments are used in clinical practice, since low treatment adherence and/or early discontinuation have been associated with poor patient outcomes. Owing to the recency of approvals, real-world data on the use of ALK inhibitors in patients with ALKpositive NSCLC are currently limited; this represents a notable gap in our understanding of ALK treatment use. OBJECTIVE: To assess real-world adherence and persistence with ALK inhibitors in patients with ALK-positive NSCLC. METHODS: This retrospective observational study used US commercial claims for patients aged at least 18 years with lung cancer receiving ALK inhibitors (alectinib, brigatinib, ceritinib, crizotinib) between July 1, 2015, and December 31, 2018. Patients' first and any subsequent ALK inhibitor uses were categorized into ALK inhibitor-naive and ALK inhibitor-pretreated cohorts, respectively. Adherence was measured by medication possession ratio and persistence by time from treatment initiation to discontinuation (earliest of a treatment switch or greater than a 60-day gap). Descriptive statistics were used to summarize patient characteristics. Cohort comparisons were made using chi-square tests and t-tests. Persistence and time to next ALK inhibitor were analyzed using Kaplan-Meier methods and the log-rank test. Poisson and Cox regression models of adherence and persistence, respectively, were applied to compare ALK inhibitors. RESULTS: We identified 1,482 patients treated with alectinib (n = 445) or crizotinib (n = 1,037) in the ALK inhibitor-naive cohort; 604, 142, and 134 patients received alectinib, brigatinib, or ceritinib in the ALK inhibitor-pretreated cohort. Adherence during the treatment period (95%-97%) and the proportion of patients with a medication possession ratio of at least 0.8 (92%-95%) were similar for all ALK inhibitors. In the ALK inhibitor-naive cohort, median time to treatment discontinuation with alectinib and crizotinib was 27.1 and 8.8 months, respectively; patients receiving alectinib were 46% less likely to discontinue than patients receiving crizotinib (adjusted hazard ratio [aHR] [95% CI]: 0.54 [0.44-0.65]; P < 0.0001). In the ALK inhibitor-pretreated cohort, the discontinuation risk for alectinib was 64% lower than for ceritinib (aHR [95% CI]: 0.36 [0.27-0.49]; P < 0.0001) and 34% lower than for brigatinib (aHR [95% CI]: 0.66 [0.42-1.02]; P = 0.062). CONCLUSIONS: To our knowledge, this study is the first to address a current research gap by assessing real-world adherence and persistence with ALK inhibitors among patients with ALK-positive NSCLC in real-world clinical practice. Alectinib was associated with longer real-world persistence than other ALK inhibitors, despite similar adherence. Further research with more patients and longer follow-up is needed to link persistence to real-world clinical outcomes. DISCLOSURES: This study was funded by Genentech Inc. Ganti has received research support from Takeda and has provided consulting services to Genentech Inc., AstraZeneca, Flagship Biosciences, Cardinal Health, BioGene, Mirati Therapeutics, Blueprint Medicines, and G1 Therapeutics. Lin, Wong, and Ogale are employees of Genentech Inc. and may own stock in F. Hoffmann-La Roche. Yang was employed by Genentech Inc. at the time of this study. Part of the study findings were presented as a poster at the NCCN 2020 Virtual Annual Conference, April 9, 2020.

Aim: Commercial plan coverage policies for multigene panel tests may vary and could result in geographic variation in coverage due to the fragmented nature of the commercial insurance market. This study aimed to characterize the alignment of multigene panel tests coverage policies to that of clinical guidelines, overall and by state. Materials & methods: We reviewed NCCN Guidelines® for four tumors. Public coverage policies were identified via web search. Payer policies included those with the largest or second largest number of commercial lives in each state. Policies were classified as 'more restrictive' or 'consistent' with the guidelines. Results: Of 38 plans/policies reviewed, 71% were classified as 'more restrictive' than the guidelines, with variation in the number of commercial lives by state. Among these, 52% restricted on panel size and 63% restricted in all or select tumors. Conclusion: Most coverage policies were more restrictive. Clinical guideline clarity and state policies may improve alignment to guidelines and geographic variations.

Background: People with hemophilia A (PwHA) face unique challenges in managing their condition, which can impact health-related quality of life. Those who are receiving optimal prophylactic treatment may be able to overcome some of these challenges. Measuring patient-reported outcomes (PROs) could help clinicians and PwHA understand the impact of prophylaxis. Methods: This was a survey designed to assess PROs in adult PwHA receiving prophylactic treatment with either emicizumab or factor VIII (FVIII) replacement therapy. Respondents were recruited through the membership of the Hemophilia Federation of America. To facilitate descriptive comparisons between the experiences of PwHA on either of the two prophylaxis regimens, PwHA with inhibitors were not included. Survey questions focused on outcomes related to healthcare resource use (clinic and emergency department [ED] visits), out-of-pocket expenses, and employment status. Responses are reported in counts and proportions or means with standard deviations (SD). Results: A total of 114 PwHA (60 who reported taking emicizumab and 54 taking FVIII for prophylaxis) responded to the survey. Among them, 89 (78%) received their care at 38 different Hemophilia Treatment Centers across the US. Characteristics of survey respondents are reported in Table 1. Of note, among those receiving emicizumab, 51 (85%) had severe, 7 (12%) had moderate, and 2 (3%) had mild hemophilia A. Among those receiving FVIII, 39 (72%) had severe, 10 (19%) had moderate, and 5 (9%) had mild disease. When asked about healthcare resource use over the past 6 months, PwHA receiving emicizumab reported a mean (SD, range) of 1 (0.9, 0 to 5) clinic visit and 0.1 (0.4, 0 to 2) ED visit, while those receiving FVIII prophylaxis reported a mean of 1.2 (1.4, 0 to 10) clinic visits and 0 (0.2, 0 to 1) ED visits. When asked about out-of-pocket expenses, 19 (32%) of respondents receiving emicizumab and 18 (33%) of those receiving FVIII reported having none. The highest average (SD) hemophilia-related out-of-pocket expenses included lodging (emicizumab vs FVIII: $62 [98] vs $363 [1,997]), over-the-counter medication ($49 [83] vs $125 [433]), prescription medication ($24 [52] vs $58 [249]), and childcare ($27 [44] vs $35 [70]). When asked questions about employment, 22 (37%) of respondents receiving emicizumab and 27 (50%) of those receiving FVIII prophylaxis reported that hemophilia had no impact on their professional or work life. Conversely, 17 (28%) of respondents receiving emicizumab and 17 (32%) of those receiving FVIII reported changing their line of work or career due to hemophilia. Notably, 13 (22%) and 12 (22%) reported having to take a job that offered good health insurance, among those receiving emicizumab and FVIII prophylaxis, respectively. Conclusions: In this survey PwHA receiving prophylactic treatment reported low healthcare resource use, variable out-of-pocket expenses, and a spectrum of hemophilia-related impacts on employment. This study highlights the heterogeneity of experiences among PwHA and potential opportunities for optimization of the patient experience with prophylactic treatment. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Atezolizumab is a programmed death ligand-1 (PD-L1) inhibitor indicated as first-line (1L) monotherapy for metastatic non-small cell lung cancer (mNSCLC) patients whose tumors have high PD-L1 expression (≥50%) without epidermal growth factor receptor or anaplastic lymphoma kinase mutations. This analysis aimed to evaluate the cost-effectiveness of 1L atezolizumab versus pembrolizumab monotherapy for mNSCLC patients with high PD-L1 expression from a US payer perspective. A Microsoft Excel-based Markov model with progression-free, progressive disease (PD), and death states was developed to compare clinical and cost outcomes of atezolizumab versus pembrolizumab monotherapy. Efficacy, safety, and utility data were derived from systematic reviews and network meta-analysis of published cancer immunotherapy agents. Product prescribing information and clinical trials informed dosing and administration. Wholesale acquisition cost (WAC, accessed in March 2020) for drugs were used while other cost inputs were from publicly available fee schedules and peer-reviewed literature. The main outcome was the incremental cost-effectiveness ratio (ICER) expressed as cost per quality-adjusted life-year (QALY) gained. Probabilistic sensitivity analysis (PSA) and one-way sensitivity analysis (OWSA) with 20% variation were performed to address uncertainties around input parameters. In the base case, 1L atezolizumab monotherapy was projected to increase patient’s life expectancy by 0.57 life-years (4.35 vs. 3.78) and 0.41 QALYs (3.17 vs. 2.76) over pembrolizumab monotherapy at an incremental cost of $22,581, resulting in an ICER of $54,549/QALY gained. The PSA demonstrated that atezolizumab had a 43% and 51% probability of being cost-effective at willingness-to-pay thresholds of $100,000 and $150,000, respectively. In the OWSA, results were most sensitive to changes in discount rates for costs and effects. Evidence suggests 1L atezolizumab monotherapy for mNSCLC patients with high PD-L1 expression was cost-effective when compared to pembrolizumab monotherapy. The projected mean ICER ($54,549/QALY) for atezolizumab monotherapy falls below the commonly used US cost-effectiveness thresholds (

e18847 Background: Atezolizumab monotherapy is indicated as 1L treatment for mNSCLC patients with high programmed death ligand-1 (PD-L1) expression (≥ 50%) and without epidermal growth factor receptor or anaplastic lymphoma kinase mutations. This analysis assessed the cost-effectiveness of 1L atezolizumab monotherapy vs. pembrolizumab monotherapy for mNSCLC patients with high PD-L1 expression from a US third-party payer perspective. Methods: A Markov model with progression-free, progressive disease (PD), and death states was developed in Microsoft Excel to compare clinical and cost outcomes of atezolizumab monotherapy vs. pembrolizumab monotherapy. Efficacy, safety, and utility data were derived from systematic reviews and indirect comparisons of the IMpower110, Keynote-024, and Keynote-042 trials. Product prescribing information and clinical trials informed dosing and administration. Wholesale acquisition cost (WAC, accessed in January 2021) for drugs were used while other cost inputs were derived from publicly available fee schedules and peer-reviewed literature. The key outcome of interest was the incremental cost-effectiveness ratio (ICER) expressed as cost per quality-adjusted life-year (QALY) gained. Deterministic sensitivity analysis with 20% variation and probabilistic sensitivity analyses (PSA) were performed to address uncertainties around input parameters. Results: In the base case, 1L atezolizumab monotherapy was projected to increase life expectancy for patients by 0.60 life-years (4.35 vs. 3.75) and 0.47 QALYs (3.46 vs. 2.98) over pembrolizumab monotherapy at an incremental cost of $27,947 (mean total cost: $396,811 vs. $368,864), resulting in an ICER of $58,841/QALY gained. Results of the deterministic sensitivity analysis were most sensitive to changes in discount rates for costs and care costs in the PD state. The PSA showed that the probability of atezolizumab being cost-effective at willingness-to-pay thresholds of $100,000 and $150,000 was 41% and 49%, respectively. Conclusions: First-line atezolizumab monotherapy had 0.6 life-years and 0.47 QALYs gained compared with pembrolizumab monotherapy and was estimated to be cost-effective (ICER $58,841/QALY). As the ICER falls below the US cost-effectiveness thresholds ( < $100,000-$150,000/QALY), clinicians and payers should consider atezolizumab monotherapy as a cost-effective 1L option for mNSCLC patients with high PD-L1 expression.

For patients with advanced non-small cell lung cancer (aNSCLC) and positive driver mutations (DM)s, NCCN guidelines recommend first-line targeted therapy. Cancer immunotherapies (CIT)s are recommended for patients with ≥ 1% PD-L1 expression and no DM. Our study aimed to describe current aNSCLC testing and treatment patterns in US community settings. Adult (≥ 18 years old) patients with non-squamous aNSCLC who were diagnosed between 10/1/2016-8/31/2019, had ≥ 1 visit within 90 days of advanced diagnosis, and received care in community practices were obtained from Flatiron Health EHR-derived de-identified database. PD-L1 and DM (EGFR, ALK, ROS1, and BRAF) test result date within 90 days of advanced diagnosis were used to identify the presence of biomarker tests. Treatment patterns were described pre and post the first positive DM test results and focused on patients who initiated CITs prior to DM results. A total of 12212 patients with aNSCLC were analyzed. Overall, 72% had evidence of testing both DMs and PD-L1 while 13% and 4% were tested only for DMs or only PD-L1, respectively. Among DM positive patients (n= 1945), 19% initiated treatment (7% CITs, 7% chemo, 5% targeted) prior to receiving their positive test results. Only 26% (n= 35/135) of those who had started CITs prior to positive DM result switched to a targeted therapy after receiving a positive result (median (IQR) time to switch from positive DM result: 1.6 (0.8-5.2) months), while most of these patients had no evidence of new treatment (58%, n= 78/135; median (IQR) follow-up time from positive DM result: 3.6 (1.3-10.2) months). More than half of the patients with EGFR+ or ALK+ aNSCLC who started CITs before DM result have no evidence of receiving targeted therapy even after their positive result date. In this study, 19% of patients with DMs positive aNSCLC had started treatment before DM test results were available. Despite overwhelming clinical evidence supporting the benefits of targeted therapy specific to DMs, most patients who started CITs prior to test results stayed on CITs, rather than switching to a targeted therapy, after the identification of a targetable mutation. It is possible that symptomatic burden precluded waiting for test results, highlighting the need for more expedient testing strategies. Given the short follow-up time for some patients, some of these patients may transition to targeted therapy at a later time, however, there is also the possibility of lost treatment opportunity.

In the phase III ALEX study, alectinib demonstrated superior progression-free survival over crizotinib for treatment-naïve patients with ALK+ NSCLC. This study aims to evaluate health resource utilization (HRU) and costs for patients receiving alectinib or crizotinib as the first ALK inhibitor (ALKi). This retrospective observational study identified patients ≥ 18 years old with lung cancer (ICD-9: 162, ICD-10: C34) receiving their first ALKi between 1/1/2015 and 6/30/2019 from PharMetrics Plus claims database. Patients were required to have continuous medical and pharmacy enrollment 6 months before and 12 months after the first ALKi initiation date (index date) and no prior ALKi treatment during the 6 months pre-index. HRU and per-patient-per-month (PPPM) costs (reported in 2019 USD) during the 12 months post-index were summarized by index ALKi. A multivariate generalized linear model with log-link and gamma distribution was applied to estimate total cost of care, adjusting for baseline characteristics. The study included 53 alectinib and 124 crizotinib patients. In the 12 months post-index, alectinib patients were less likely to have any emergency department (ED) and inpatient visits compared with crizotinib patients (ED: 24.5% vs 40.3%, p=0.044; inpatient: 22.6% vs 41.3%, p=0.019). Although ALKi pharmacy costs were similar (unadjusted PPPM cost: $12,749 vs $12,825, p=0.933), total cost of care was lower for alectinib patients than for crizotinib patients (unadjusted PPPM cost: $18,461 vs $21,307, p=0.069]. After adjusting for baseline characteristics, total cost of care for alectinib patients was 19.5% lower than crizotinib patients (adjusted PPPM cost: $15,536 vs $19,303, p=0.005). In this study, patients receiving alectinib as the first ALKi had approximately 20% lower mean adjusted total cost of care compared with patients receiving crizotinib. This real-world economic evidence supplements clinical benefits from the ALEX study, supporting the use of alectinib as the first ALKi in ALK+ NSCLC patients.

e18298 Background: Febrile neutropenia (FN) is a key driver of morbidity and mortality in oncology patients receiving myelotoxic chemotherapy. Identification and prophylaxis per guidelines can result in a substantial reduction of FN risk and is a key oncology quality of care measure. This project evaluated the feasibility of using electronic medical records (EMR) to quantify FN rates and characterize patients at risk for FN. Methods: Search algorithm was used in EMR to identify patients initiating oncologic care between 1/1/2016 and 12/1/2017 at the Masonic Cancer Clinic, University of Minnesota—Fairview Health System, for a lymphoid, gastrointestinal, breast, female genital, or thoracic malignancy. FN event was identified using a diagnosis of neutropenia (ICD-10: D70.x) with associated fever (ICD-10: R50.2-R50.9) or infection (ICD-10: A40.x, A41.x, B95.0-B95.8, B96.0-B96.8, T80.21-T80.29) during the myelotoxic chemotherapy course. FN rates were evaluated overall and by cancer type, disease status, prior chemotherapy use, select comorbidities, age, curative or palliative intent, and treatment plan FN risk. Results: A total of 1,123 patients receiving 1,663 myelotoxic chemotherapy plans were identified. Patients were predominately female (60%) with a mean age of 61 years (SD: 14.1). A total of 66 patients experienced 79 FN events. Of these events, 70 (88.6%) resulted in a hospitalization. The FN rate during the study period was 5.9% (95% CI: 2.3%-10.2%). Factors associated with the highest FN rates were: lymphoid malignancy (9.9%), non-metastatic disease (7.2%), no prior chemotherapy (6.5%), ≥ 3 comorbidities. Conclusions: Our findings indicate it is feasible to estimate FN rates using a search algorithm with a known FN definition in the EMR of a large integrated delivery network. While additional analyses are planned to account for concomitant patient characteristics to fully understand the occurrence of FN, the current project nevertheless demonstrates the ability of estimating FN rates to both measure care quality and allow pursuing quality improvement (QI) initiatives for appropriate FN prophylaxis. The results also have the potential to serve as a benchmark for similar QI projects in other large healthcare systems.

Objectives To examine the association between having a patient-centered medical home (PCMH) and healthcare expenditures and quality of care for children with special health care needs (CSHCN). Methods We conducted a cross-sectional analysis of 8802 CSHCN using the 2008–2012 Medical Expenditure Panel Survey. A PCMH indicator was constructed from survey responses. Inverse probability treatment weighting was applied to balance the cohort. CSHCN’s annual health care expenditures and quality were analyzed using two-part and logistic models, respectively. Results Covariate-adjusted annual total expenditures were similar between CSHCN with and without a PCMH ($4267 vs. $3957, p = 0.285). CSHCN with a PCMH had higher odds of incurring expenditure (OR 1.66, 95% CI 1.22–2.25)—in particular, office-based services and prescriptions (OR 1.46 and 1.36, 95% CI 1.24–1.72 and 1.17–1.58, respectively)—compared to those without a PCMH, without shifting expenditures. When examined in detail, PCMH was associated with lower odds of accessing office-based mental health services (OR 0.80, 95% CI 0.66–0.96), leading to lower expenditures ($106 vs. $137; p = 0.046). PCMH was associated with higher odds of post-operation and immunization visits (OR 1.23 and 1.22, 95% CI 1.05–1.45 and 1.004–1.48, respectively) without changing expenditures. Parents of CSHCN with a PCMH were more likely to report having the best health care quality (OR 2.33, p < 0.001). Conclusions CSHCN who had a PCMH experienced better health care quality and were more likely to access preventive services, with unchanged expenditures. However, they were less likely to use mental health services in office-based settings. As the effects of PCMH varied across services for CSHCN, more research is warranted.

To examine the association between having a patient-centered medical home (PCMH) and healthcare expenditures for children with special health care needs (CSHCN) in the U.S. We conducted a cross-sectional analysis of CSHCN in the 2008-2012 Medical Expenditure Panel Survey (MEPS) databases. MEPS identified CHSCN, while a binary PCMH indicator was constructed from parents’ survey responses following a validated algorithm.1We examined the PCMH effect on CSHCN’s annual healthcare expenditures by payers and patients with a 2-part model using logistic regression and linear regression with log-transformed costs. A total of 8,671 CSHCN were included in the sample. 48.7% of CSHCN reported had a PCMH. Almost all CSHCN (94.8%) incurred positive annual healthcare expenditures. CSHCN with a PCMH had higher odds of incurring any expenditures (OR=1.77, 95% CI: 1.43-2.19) -- in particular, expenditures on office visits (OR=1.47, 95% CI: 1.31-1.65) and prescriptions (OR=1.39, 95% CI: 1.25-1.55) -- compared with those without a PCMH. Conditional on positive expenditures, a PCMH was associated with higher total expenditures, as well as expenditures on outpatient care (6.9% and 28.9%; p=0.03 and p=0.01, respectively). Altogether, annual total expenditures were $3,975 for CSHCN with a PCMH, versus $3,629 for children without one. During the study period, CSHCN with a PCMH had higher healthcare expenditures, in particular on prescription drugs. Further research is needed to identify whether this is a causal effect between PCMHs and health care utilization for CSHCN. Reference: 1. Romaire MA, Bell JF, Grossman DC. Medical home access and health care use and expenditures among children with special health care needs. Archives of pediatrics & adolescent medicine. 2012;166(4):323-330.