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Priyanka Gaitonde,1 Viktor Chirikov,2 Sneha Kelkar,2 Bengt Liljas1 1AstraZeneca, Health Economics & Payer Evidence, Gaithersburg, MD, USA; 2OPEN Health Evidence & Access, Bethesda, MD, USACorrespondence: Priyanka Gaitonde, Oncology Market Access and Pricing, AstraZeneca, 200 Orchard Ridge Drive, Gaithersburg, MD, 20878, USA, Email priyanka.gaitonde@astrazeneca.comBackground: To extend the discussion on the use of real-world evidence (RWE) in conveying the clinical value of treatment beyond trial data, the primary objective of this study was to assess if efficacy gains in progression-free survival (PFS) observed in randomized controlled trials (RCT) correlate with efficacy gains in the real-world setting. For this, we assessed the treatment benefit of three tyrosine kinase inhibitors (TKIs) in aNSCLC.Methods: Using matched cohorts identified in the Flatiron Health database (2011– 2020), we mimicked the following cohorts of TKI versus platinum-based chemotherapy (PBC) from the following trials: (1) erlotinib, EURTAC; (2) afatinib, LUX-Lung 3; and (3) crizotinib, PROFILE 1014. Time to treatment discontinuation (TTD) hazard ratio (HR) was used as a proxy for PFS HR, the primary endpoint in the selected RCTs. HRs were calculated via Cox proportional hazard models.Results: Overall, 1,118 patients were included across the three RWE cohorts. Frontline TKI regimens had statistically significantly better real-world TTD than their matched PBC comparator group (HR 0.37, 95% confidence interval [CI] 0.30– 0.44 for erlotinib; HR 0.42, 95% CI 0.32– 0.55 for afatinib; HR 0.37, 95% CI 0.26– 0.53 for crizotinib). The benefit in real-world OS was not different between TKIs and PBC patients, attributed to a high proportion of switching to subsequent therapy. Study findings of relative treatment benefit (HR) for real-world TTD and OS were deemed similar to those for PFS and OS from the pivotal RCTs.Conclusion: The relative treatment effect, measured as real-world TTD HR over the long term, was similar to trial-based PFS HR, implying that the clinical benefit of aNSCLC treatments conveyed in trials translated into the clinical setting. This is important, given that OS data interpretation is limited, even with longer follow-up. Additionally, our RWE analysis endorses TTD as a relevant endpoint to measure clinical benefit.Keywords: non–small cell lung cancer, short- and long-term outcomes, time to treatment discontinuation, reimbursement

Viktor Chirikov,1 Marc Botteman,1 Eric AF Simões2 1OPEN Health, Evidence & Access, Bethesda, MD, USA; 2Department of Pediatrics, University of Colorado School of Medicine, Department of Epidemiology, Center for Global Health, Colorado School of Public Health, and Section of Infectious Disease, Children’s Hospital Colorado, Aurora, CO, USACorrespondence: Viktor Chirikov, OPEN Health Evidence & Access, Bethesda, MD, 20814, USA, Tel +1 240 821 9662, Email ViktorChirikov@openhealthgroup.comBackground: The objective of this study was to estimate the long-term healthcare utilization and cost burden of RSV by chronological age of diagnosis (Year 1, Year 2 and Years 3– 5 cohorts) as well as by gestational age at birth in Japan.Methods: The JMDC database was used to retrospectively identify RSV and control patients between February 1, 2011 and January 31, 2016 and follow them through December 31, 2017. Infants with RSV infection (n = 9028 in Year 1; n = 4929 in Year 2; n = 2004 in Years 3– 5) were matched to controls (n = 17,886; n = 9351; n = 3655, respectively) based on gestational age and year and quarter of birth; controls were assigned the index date (ie, diagnosis) of their respective match. Covariate-balancing propensity score weights were employed adjusting for remaining imbalances between cohorts. The main outcomes were average cumulative rates for all-cause, asthma/wheezing, and respiratory-related hospitalizations, physician and urgent care/emergency visits and associated costs (reported as 2018 ¥JPY) over 36-months of follow-up since index.Results: Healthcare utilization was significantly higher among RSV cases for most comparisons. All-cause average differential cost burden was higher for RSV, compared to controls, among the following cohorts: Year 1 full-term (¥ 277,727); Year 2 preterm (¥ 530,302), late preterm (¥ 270,797), full-term (¥ 238,832); Years 3– 5 preterm (¥ 110,057), late preterm (¥ 486,670), full-term (¥ 289,986). While all-cause costs were similar for preterm and late preterm children in the Year 1 cohort, respiratory- and asthma/wheezing-related attributable costs were substantially higher for RSV.Conclusion: RSV infection had a significant long-term health and economic burden among children infected during their first year of life and later in life. Study findings have import for prevention strategies, currently directed at maternal immunization and monoclonal antibodies for preventing primary RSV infections in the first six months of life and beyond but also for older age not targeted currently.Keywords: respiratory syncytial virus, long-term follow-up, lower respiratory tract infection, prematurity

Deepinder K Dhaliwal,1 Viktor Chirikov,2 Jordana Schmier,2 Sanika Rege,2 Schalon Newton3 1Department of Ophthalmology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; 2OPEN Health Evidence & Access, Bethesda, MD, USA; 3Trefoil Therapeutics, Inc., San Diego, CA, USACorrespondence: Schalon Newton, Trefoil Therapeutics, Inc., 6330 Nancy Ridge Dr Suite 103, San Diego, CA, 92121, USA, Tel +1 949 678 9572, Email snewton@trefoiltherapeutics.comPurpose: To assess the incremental burden of corneal transplant surgery for US commercially insured patients with Fuchs endothelial corneal dystrophy (FECD) treated with endothelial keratoplasty (EK) compared to controls.Methods: The study design was retrospective cohort using IBM® MarketScan® claims (January 2014–September 2019) and included EK-treated (N=1562) and control patients (N=23,485) having ≥ 12 months’ enrollment before and after diagnosis, who were subsequently matched on select characteristics. The index date was the beginning of the pre-operative period (3 months before EK); synthetic EK index was assigned for controls. All-cause, eye-disease, and complication-related healthcare resource utilization (HCRU) and costs were compared up to 36 months post index. For a small subset of patients, patient data were linked to the Health and Productivity Management supplemental database, which integrates data on productivity loss and disability payments.Results: Matched cohorts included 804 EK-treated and 1453 controls with average age 65.7 years, 1383 (61%) female. Over 12 months of follow-up, all-cause ($41,199 vs $20,222, p< 0.001) and eye-disease related costs ($22,951 vs $1389, p< 0.001) were higher among EK-treated patients than controls. The cost differential increased additionally by $1000–$2000 per annum by 36 months of follow-up. While balanced at baseline, over follow-up EK-treated patients had higher prevalence of glaucoma, elevated intraocular pressure, cataract, cataract surgery, diagnosis of cornea transplant rejection, retinal edema. By 36 month of follow-up, EK-treated patients had 9 more short-term disability days, resulting in $2992 additional burden of disability payments.Conclusion: This study found a higher cost burden among FECD patients receiving EK treatment versus those who did not. With a shift in management of FECD, cost burden estimates generated in this study could serve as an important benchmark for future studies.Keywords: cornea transplantation, cost analysis, glaucoma, retrospective study

Patients in EPCORE NHL-1 with prior CAR T therapy were excluded as ZUMA-1 included only patients without prior CAR T therapy. Efficacy of Subcutaneous Epcoritamab versus Axi-cel in R/R DLBCL CAR T-Naive and CAR T-eligible patients: An Indirect Comparison B Introduction: b Epcoritamab, an off-the-shelf, subcutaneous CD3xCD20 T-cell-engaging bispecific antibody that redirects T cells to eliminate malignant CD20+ B cells, showed single-agent efficacy in patients with relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL) with a manageable safety profile. [Extracted from the article]