Background Azacitidine is an hypomethylating agent widely adopted for the treatment of acute myeloid leukaemia (AML) in patients who are ineligible for curative-intent chemotherapy. Patients with low bone marrow blast counts (< 30%) experience improved survival with azacitidine, but the benefits are significantly lower in patients with > 30% blasts in the bone marrow. As such, there is uncertainty around the economic benefit of azacitidine in patients with higher blast counts. Objective We present a cost-utility analysis of azacitidine in patients with AML with > 30% blasts to determine the economic value of azacitidine in this patient population from the perspective of a third-party payer. Methods A Markov model was developed with a time horizon of 25 months divided into 22 cycles of 35 days each. The cost utility of azacitidine was compared with that of conventional care regimens (which include best supportive care, low-dose cytarabine and induction chemotherapy). A Canadian public healthcare system perspective was selected. Results In the base case, the incremental cost per quality-adjusted life-year gained (incremental cost-effectiveness ratio [ICER]) for azacitidine compared with conventional care regimens was $Can160,438, year 2018 values. The estimated ICER was insensitive to a longer time horizon but sensitive to the cost of azacitidine and to assumptions relating to survival in both treatment regimens, although the ICER always remained greater than Can$80,000 in all scenarios. Conclusion Azacitidine is unlikely to be cost effective given that the estimated ICER exceeds the willingness to pay commonly used in the Canadian healthcare system.

New therapies in a publicly funded healthcare system are first appraised by health technology assessment agencies that provide funding recommendations to the payers. Treatment with Chimeric Antigen Receptor-T cell (CAR-T) therapy is revolutionizing the management of patients with relapsed/refractory aggressive B-cell lymphoma by providing an effective alternative to the standard of care. Yet, the implementation of CAR-T treatment has a substantial impact on the healthcare system due to its high cost, complex manufacturing process, and requirement for highly specialized services and expertise. CAR-T Cells, as a “living drug”, are fundamentally different from usual medications, and their approvals and funding recommendations pose unique challenges to the health technology agency. In this paper, we explore the specific challenges that face the health technology agencies in reviewing reimbursement recommendations for CAR-T therapy. We take a Canadian perspective and use CAR-T treatment of relapse/refractory aggressive B-cell lymphoma as an example.

Little is known about patient perspectives regarding consent for obtaining extra research-specific bone marrow (BM) samples during the diagnostic procedure for acute leukemia (AL). This study aimed to better understand patient experiences with consenting to provide these samples and identify potential areas for practice improvement. Semi-structured interviews were conducted with patients treated for AL, 4–6 years prior to the interviews, and healthcare professionals involved with obtaining patient consent and sample collection. A total of 17 patients (14 agreed to provide a sample and 3 did not have a sample in the biobank) and 5 healthcare professionals were interviewed, achieving data saturation. Patients supported increasing public knowledge about research and noted the importance of friends and family in providing emotional support and retaining information. Despite time pressure and anxiety, the decision to donate a research sample did not require much deliberation. Proximal factors informing decisions included impact on patient health and family and anticipated, procedure-associated pain; distal factors included altruism and trust in healthcare professionals. Key information included expected pain and management, the purpose of research samples, and sample security and privacy. Our findings suggest that BM research sample collection may be facilitated through optimizing the environment where information is provided and the type of information provided, including pain management options and the value of the samples for current and future research.

Chimeric antigen receptor (CAR) T-cell therapy is a new standard of care for patients with diffuse large B-cell lymphoma (DLBCL); however, studies including healthcare resource utilization (HRU) during routine care are lacking. Accordingly, a population-based study was conducted using linked administrative databases from Ontario, Canada. Patients with DLBCL that failed ≥2 lines of systemic therapy were included. Cox proportional hazard models estimated associations between covariates and overall survival (OS). Logistic, binomial and Poisson regression explored associations between covariates with toxicity and HRU. We identified 308 patients enrolled to receive CAR T-cell therapy of which 255 patients received CAR T-cells (mean age 59 years; 39% female). From the date of CAR T-cell infusion, the median OS was 25.0 months (95% CI, 21.6–28.1 months). Cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome data were available for 155 patients and were reported in 135 (87.1%) and 42 (27.1%) patients, respectively. Of those that received CAR-T cells, 172 patients (67%) were hospitalized with a median length of stay of 5 days (IQR, 0–20) and 243 (95%) had an emergency department visit without hospitalization. Our prospective population-based study demonstrates comparable efficacy and safety of CAR T-cell therapy in the real-world to the pivotal trials and highlights this as an efficacious and relatively safe treatment option for patients with DLBCL in routine clinical care.

Summary: Single‐arm studies demonstrate dramatic improvements in survival in patients with relapsed/refractory (R/R) aggressive B‐cell lymphomas but with significant toxicities and at exceptionally high costs. The objective of this study was to evaluate the real‐world health outcomes of aggressive B‐cell lymphoma patients treated with publicly funded chimeric antigen receptor (CAR) T‐cell therapy in Ontario, Canada, compared to historical controls. We conducted a population‐based retrospective cohort study among patients treated for R/R aggressive B‐cell lymphoma in Ontario between January 2013 and June 2023. The cohort consisted of patients who received commercially available CAR T cells between 1 January 2019 and 30 June 2023 and historical controls who were treated with the standard of care between 1 January 2013 and 31 December 2018, before public funding for CAR T‐cell treatments was available. Inverse probability of treatment weighting (IPTW) was used to balance patients' characteristics across the two groups. The cohort included 314 CAR T‐cell patients and 106 historical controls. The median follow‐up time was 29.6 months (95% confidence interval [CI]: 26.1–33.0), and the median overall survival was 15.1 months (95% CI, 11.1–26.3) and 4.8 months (95% CI, 3.8–6.4) for CAR T‐cell‐treated patients and historical controls (hazard ratio 0.50, 95% CI 0.38–0.65) respectively. These results support the benefit of CAR T‐cell treatment for patients with R/R aggressive B‐cell lymphoma in routine clinical care.