Real-world expenditures and survival time after CAR-T treatment for large B-cell lymphoma in Switzerland: a retrospective study using insurance claims data

AIM OF THE STUDY: Newly approved therapies with high and uncertain budget impact pose challenges to public health care systems worldwide. One recent example is chimeric antigen receptor T cell (CAR-T) therapies for adults with large B-cell lymphoma (LBCL). This study’s primary objective is to examine the expenditures of Swiss public payers before, during, and after CAR-T cell therapy in patients with LBCL aged ≥30 years. Its secondary objective is to analyse 24-month survival rates. METHODS: This retrospective observational data analysis used the administrative databases of the Swiss health insurers Concordia, CSS, Groupe Mutuel, Helsana, ÖKK, Sanitas, SWICA, Sympany, and Visana. These health insurers or groups provide mandatory health insurance to approximately 78% of Swiss residents in 2021. Using the relevant procedure codes, we identified CAR-T therapies administered between October 2018 (first approval) and June 2021 (treatment identification cut-off). Patients aged <30 years were excluded because they might be treated for pediatric acute lymphoblastic leukaemia. Expenditures were categorised as pre-infusion, peri-infusion (excluding CAR-T therapy acquisition costs), and post-infusion based on the time of service provision. Overall survival rates were estimated using the Kaplan–Meier method. RESULTS: This study identified 81 patients aged ≥30 years, with a median follow-up period for censored observations of 27 months (interquartile range: 21–31 months). The median age group was 70–74, and 60% of patients were male. Mean healthcare expenditures per patient per month amounted to CHF 8,115–22,564 pre-infusion, CHF 38,490 peri-infusion, and CHF 5,068–11,342 post-infusion. For the total peri- and post-infusion period (i.e. 1-month before infusion to 23 months after infusion), mean healthcare expenditures amounted to CHF 215,737. The 24-month overall survival rate was 48% (95% confidence interval: 38–61%). CONCLUSIONS: Healthcare expenditures after CAR-T cell infusion are relatively high compared to previous estimates of patients with LBCL in the last year of treatment. Further research is needed to understand the drivers behind these post-infusion expenditures. Especially, clinical data should be used to assess the time until disease progression. The analysis of 24-month overall survival is consistent with results from the pivotal trials. Our findings stress the importance of post-approval studies to monitor real-world expenditures and outcomes related to innovative therapies

Dual guidance structure for evaluation of patients with unclear diagnosis in centers for rare diseases (ZSE-DUO): study protocol for a controlled multi-center cohort study

Background: In individuals suffering from a rare disease the diagnostic process and the confirmation of a final diagnosis often extends over many years. Factors contributing to delayed diagnosis include health care professionals' limited knowledge of rare diseases and frequent (co-)occurrence of mental disorders that may complicate and delay the diagnostic process. The ZSE-DUO study aims to assess the benefits of a combination of a physician focusing on somatic aspects with a mental health expert working side by side as a tandem in the diagnostic process. Study design: This multi-center, prospective controlled study has a two-phase cohort design. Methods: Two cohorts of 682 patients each are sequentially recruited from 11 university-based German Centers for Rare Diseases (CRD): the standard care cohort (control, somatic expertise only) and the innovative care cohort (experimental, combined somatic and mental health expertise). Individuals aged 12 years and older presenting with symptoms and signs which are not explained by current diagnoses will be included. Data will be collected prior to the first visit to the CRD's outpatient clinic (T0), at the first visit (T1) and 12 months thereafter (T2). Outcomes: Primary outcome is the percentage of patients with one or more confirmed diagnoses covering the symptomatic spectrum presented. Sample size is calculated to detect a 10 percent increase from 30% in standard care to 40% in the innovative dual expert cohort. Secondary outcomes are (a) time to diagnosis/diagnoses explaining the symptomatology; (b) proportion of patients successfully referred from CRD to standard care; (c) costs of diagnosis including incremental cost effectiveness ratios; (d) predictive value of screening instruments administered at T0 to identify patients with mental disorders; (e) patients' quality of life and evaluation of care; and f) physicians' satisfaction with the innovative care approach. Conclusions: This is the first multi-center study to investigate the effects of a mental health specialist working in tandem with a somatic expert physician in CRDs. If this innovative approach proves successful, it will be made available on a larger scale nationally and promoted internationally. In the best case, ZSE-DUO can significantly shorten the time to diagnosis for a suspected rare disease