Efficacy, safety, and cost-minimization analysis of axicabtagene ciloleucel and tisagenlecleucel CAR T-Cell therapies for treatment of relapsed or refractory follicular lymphoma.

Axicabtagene ciloleucel (axi-cel) and tisagenlecleucel (tisa-cel) are chimeric antigen receptor (CAR) T-cell therapies used to treat adult patients with relapsed or refractory follicular lymphoma (rrFL) after two or more lines of systemic therapy. In the absence of head-to-head clinical trials, this study aimed to compare the efficacy, safety, and cost of axi-cel and tisa-cel in the treatment of rrFL after at least two lines of treatment. Overall response rate (ORR) and safety signals were compared using reporting odds ratios (RORs) with 95% confidence intervals (CIs) at p < 0.05. Progression-free survival (PFS), duration of response (DoR), and overall survival (OS) were compared using the Kaplan?Meier method with a log-rank test. Cost and cost-minimization analyses of drug acquisition, drug administration, serious adverse events (AEs), and relapsed management were calculated. Costs were extracted from the IBM-Micromedex Red Book, Centers for Medicare and Medicaid Services, and existing literature. Statistical analyses were conducted using Microsoft Excel and R version 4.0.5. No statistically significant differences were observed between axi-cel and tisa-cel in terms of ORR, DoR, and OS (p > 0.05). PFS was significantly better with tisa-cel (p < 0.05). Axi-cel was significantly associated with higher incidences of CRS, neurologic events, and grade 3-4 AEs than tisa-cel (ROR > 1, p < 0.05). Axi-cel and tisa-cel cost $512,021 and $450,885 per patient, respectively, resulting in savings of US$61,136 with tisa-cel over axi-cel. Tisa-cel appears to have a better safety profile, fewer serious AEs, lower mortality rate, and lower cost than axi-cel.

The Association of Medicaid expansion with prescription drug utilization and expenditure among low-income participants with asthma.

OBJECTIVE: This study estimated the association between the 2014 Medicaid expansion and asthma-related prescription drug utilization and expenditures among low-income adult participants with asthma, including those with uncontrolled asthma, in the United States. METHODS: In this national analysis, using a pooled dataset from 2007-2018 Medical Expenditure Panel Surveys (MEPS), regression discontinuity (D-RD) analyses estimated the association between Medicaid expansion and utilization of and expenditures for asthma-related prescription drugs among participants with asthma aged 26-64 with incomes below vs. at/above 138% of the federal poverty level (FPL). A sub-sample analysis was also conducted among participants with uncontrolled asthma. Utilization and expenditure outcomes were estimated using two-part models with logit as the first part and generalized linear models as the second part. RESULTS: Utilization of and total cost for asthma-related prescription drugs increased by 1.89 fills (p < 0.001) and $306.59 (p < 0.001) among participants with asthma with income below 138% FPL after Medicaid expansion. The utilization and total cost of both short-acting bronchodilators and inhaled corticosteroids (ICSs) increased after Medicaid expansion among participants with asthma with incomes below 138% FPL. Among participants with uncontrolled asthma with incomes below 138% FPL, utilization and expenditures increased after Medicaid expansion for all asthma-related prescription drugs and short-acting bronchodilators. CONCLUSION: Medicaid expansion was associated with increased utilization of and total expenditures for both quick-relief and preventive asthma medications among all low-income participants with asthma, but not with utilization of preventive medications among those with uncontrolled asthma.

Analysis of the Gene Therapies Authorized by the United States Food and Drug Administration and the European Medicines Agency.

BACKGROUND: Gene therapy, altering the genes inside human cells, has recently emerged as an alternative for preventing and treating disease. Concerns have been expressed about the clinical value and the high cost of gene therapies. OBJECTIVE: This study assessed the characteristics of the clinical trials, authorizations, and prices of gene therapies in the United States and the European Union. RESEARCH DESIGN: We collected regulatory information from the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) and manufacturer-listed prices from the United States, UK, and Germany. Descriptive statistics and t tests were conducted in the study. RESULTS: As of January 1, 2022, the FDA and EMA authorized 8 and 10 gene therapies, respectively. The FDA and EMA granted orphan designation to all gene therapies except talimogene laherparepvec. Pivotal clinical trials were nonrandomized, open level, uncontrolled, phase I-III, and included a limited number of patients. Study primary outcomes were mainly surrogate endpoints without demonstration of direct patient benefit. The price of gene therapies at market entry ranged from $200,064 to $2,125,000 million. CONCLUSIONS: Gene therapy is used to treat incurable diseases that affect only a small number of patients (orphan diseases). Based on this, they are approved by the EMA and FDA with insufficient clinical evidence to ensure safety and efficacy, in addition to the high cost.

Budget Impact Analysis of Idecabtagene Vicleucel for the Treatment of Adult Patients with Relapsed or Refractory Multiple Myeloma in the US.

BACKGROUND: Gene therapy is known to be unaffordable to those who need it the most; however, evidence on the financial impact is limited. OBJECTIVE: This study aimed to estimate the budget impact and affordability of the gene therapy idecabtagene vicleucel for the treatment of adult patients with relapsed or refractory multiple myeloma (rrMM) in the US over a 3-year period from the payer healthcare perspective. METHOD: A budget impact model was developed to estimate the budget impact to the US healthcare plan compared with bortezomib-based maintenance therapy. The target population size was based on a hypothetical 1-million-member plan over a 3-year time horizon with and without idecabtagene vicleucel adoption. The cost of drug acquisition, drug administration, and grade 3-4 adverse events (AEs) were calculated for both scenarios. The budget impact of idecabtagene vicleucel was calculated as the difference in costs for these two scenarios. Costs were extracted from IBM-Micromedex Red Book, Centers for Medicare and Medicaid Services, and the literature. A one-way sensitivity analysis was performed to ensure robustness. RESULTS: An estimated 22 patients with rrMM each year would be eligible for idecabtagene vicleucel. The model projected the annual cost per patient in the first year as $19,449 and $517,528.13 for bortezomib and idecabtagene vicleucel, respectively. Introducing idecabtagene vicleucel was predicted to increase the total budget by $13.4, $13.6, and $14 million in the first, second, and third years. There would be an additional $1.1128, $1.1252, and $1.1486 per member per month (PMPM) when using idecabtagene vicleucel over bortezomib. CONCLUSION: This study suggests that the high cost of the gene therapy idecabtagene vicleucel is justifiable due to the low number of rrMM patients.

The Economic Burden of CAR T Cell Therapies Ciltacabtagene Autoleucel and Idecabtagene Vicleucel for the Treatment of Adult Patients with Relapsed or Refractory Multiple Myeloma in the US.

BACKGROUND: Two chimeric antigen receptor-engineered T (CAR T) cell therapy drugs were recently approved for the treatment of patients with relapsed or refractory multiple myeloma (rrMM). Their financial impact, however, is poorly described. OBJECTIVE: The aim was to evaluate the economic burden of CAR T cell therapies ciltacabtagene autoleucel and idecabtagene vicleucel for the treatment of rrMM patients after at least four lines of therapy, and to compare the annual cost of these CAR T cell therapies over a hypothetical 1-million-member health plan from the US healthcare payer perspective. PATIENTS AND METHODS: The annual economic burden of ciltacabtagene autoleucel and idecabtagene vicleucel was estimated using data from pivotal clinical trials. The costs of drug acquisition, administration, and adverse event (AE) management were extracted from the IBM-Micromedex Red Book online, the Centers for Medicare & Medicaid Services fee schedules, and a review of the literature. We used descriptive statistics for the analysis. RESULTS: The annual costs (US dollars) of drug acquisition, administration, and AE management per patient were $465,000, $60,167, and $40,368 and $419,500, $61,250, and $47,270 for ciltacabtagene autoleucel and idecabtagene vicleucel, respectively. The total annual cost was higher for ciltacabtagene autoleucel ($565,534) than for idecabtagene vicleucel ($528,020). However, the total annual cost in a hypothetical 1-million-member plan was less with ciltacabtagene autoleucel, by $1.8 million. CONCLUSION: This study found that the CAR T cell gene therapies ciltacabtagene autoleucel and idecabtagene vicleucel for rrMM represent a significant economic burden for healthcare payers in the USA.