Introduction In several studies, the chimeric antigen receptor T‐cell therapy tisagenlecleucel demonstrated encouraging rates of remission and lasting survival benefits in pediatric patients with relapsed/refractory (r/r) acute lymphoblastic leukemia (ALL). We assessed the cost‐effectiveness of tisagenlecleucel (list price: 320 000 EUR) among these patients when compared to clofarabine monotherapy (Clo‐M), clofarabine combination therapy (Clo‐C), and blinatumomab (Blina) from both a healthcare and a societal perspective. We also assessed future medical and future non‐medical consumption costs. Methods A three‐state partitioned survival model was used to simulate a cohort of pediatric patients (12 years of age) through different disease states until the end of life (lifetime horizon). Relevant outcomes were life years, quality‐adjusted life years (QALYs), healthcare costs, societal costs, and the incremental cost‐effectiveness ratio (ICER). Uncertainty was explored through deterministic and probabilistic sensitivity analyses as well as through several scenario analyzes. Results Total discounted costs for tisagenlecleucel were 552 679 EUR from a societal perspective, which was much higher than the total discounted costs from a healthcare perspective (ie, 409 563 EUR). Total discounted societal costs for the comparator regimens ranged between 160 803 EUR for Clo‐M and 267 259 EUR for Blina. Highest QALYs were estimated for tisagenlecleucel (11.26), followed by Blina (2.25), Clo‐C (1.70) and Clo‐M (0.74). Discounted societal ICERs of tisagenlecleucel ranged between 31 682 EUR/QALY for Blina and 37 531 EUR/QALY for Clo‐C and were considered cost‐effective with a willingness‐to‐pay (WTP) threshold of 80 000 EUR/QALY. None of the scenarios exceeded this threshold, and more than 98% of the iterations in the probabilistic sensitivity analysis were cost‐effective. Discussion At the current price and WTP threshold, tisagenlecleucel is cost‐effective from both a healthcare and a societal perspective. Nevertheless, long‐term effectiveness data are needed to validate the several assumptions that were necessary for this model.

Although the Quality of Life Questionnaire Preference-Based Measure and the EORTC-8D appear to have better validity, this study does not provide any strong evidence against the use of the EQ-5D-5L for measuring quality-of-life utilities in acute leukemia. However, our findings need to be confirmed in larger longitudinal studies.

Background Evidence regarding health‐related quality of life (HRQoL) in patients with steroid‐refractory acute graft‐versus‐host disease (SR‐aGvHD) is lacking. Evaluating HRQoL was a secondary objective of the HOVON 113 MSC trial. Here we describe the outcomes of the EQ‐5D‐5L, EORTC QLQ‐C30, and FACT‐BMT for all adult patients who completed these questionnaires at baseline (i.e., before the start of treatment; n = 26). Methods Descriptive statistics were used to describe baseline patient and disease characteristics, EQ‐5D dimension scores and values, EQ VAS scores, EORTC QLQ‐C30 scale/item and summary scores, and FACT‐BMT subscale and total scores. Results The mean EQ‐5D value was 0.36. In total, 96% of the patients reported problems with usual activities, 92% with pain/discomfort, 84% with mobility, 80% with self‐care, and 72% with anxiety/depression. The mean EORTC QLQ‐C30 summary score was 43.50. Mean scale/item scores ranged from 21.79 to 60.00 for functioning scales, from 39.74 to 75.21 for symptom scales, and from 5.33 to 91.67 for single items. The mean FACT‐BMT total score was 75.31. Mean subscale scores ranged from 10.09 for physical well‐being to 23.94 for social/family well‐being. Conclusion Our study showed that HRQoL in patients with SR‐aGvHD is poor. Improving HRQoL and symptom management in these patients should be a top priority.