Estimating the Cost per Clinical Outcome of Second-Line Liso-Cel Versus Autologous Stem Cell Transplantation in Patients with Transplantation-Intended Relapsed/Refractory Large B Cell Lymphoma.

It is important to consider the total cost of care (TCOC) associated with a therapy and clinical benefit for relapsed or refractory (R/R) large B cell lymphoma (LBCL). We estimated the 1-year TCOC and cost per clinical outcome for patients with R/R LBCL treated with second-line lisocabtagene maraleucel (liso-cel) versus autologous stem cell transplantation (ASCT) using data from the TRANSFORM study (ClinicalTrials.gov NCT03575351). A cost per clinical outcome analysis using a Monte Carlo simulation approach was conducted. Cost inputs were generated from a retrospective microcosting analysis of healthcare resource utilization (HCRU). Patient-level data from an interim analysis (March 2021) were used to derive HCRU and clinical inputs. Clinical inputs included median event-free survival (EFS), median progression-free survival (PFS), objective response rate, and complete response (CR) rate. In the intention-to-treat analysis, the mean (standard deviation) TCOC per patient was $550,864 ($173,087) for liso-cel and $413,200 ($290,802) for ASCT. The cost per clinical outcome model estimated a mean cost for liso-cel versus ASCT per EFS month of $57,295 versus $186,369, per PFS month of $40,949 versus $78,797, per overall responder of $653,965 versus $881,804, and per complete responder of $828,045 versus $1,063,822. This economic model shows reductions in mean estimated TCOC per EFS month, PFS month, overall responder, and complete responder with liso-cel versus ASCT owing to the superior efficacy of liso-cel. Although liso-cel-treated patients incurred greater upfront costs, fewer required subsequent therapy, and they accumulated less downstream costs. These results underscore the importance of considering the durability of response and clinical benefit when assessing total costs.

Estimating the Lifetime Medical Cost Burden of an Allogeneic Hematopoietic Cell Transplantation Patient.

Allogeneic hematopoietic cell transplantation (allo-HCT) has the potential for curative outcomes for a variety of hematologic malignancies. Current allo-HCT studies often describe the outcomes and costs in the near term; however, research on the lifetime economic burden post-allo-HCT remains limited. This study was conducted to estimate the average total lifetime direct medical costs of an allo-HCT patient and the potential net monetary savings from an alternative treatment associated with improved graft-versus-host disease (GVHD)-free, relapse-free survival (GRFS). A disease-state model was constructed using a short-term decision tree and a long-term semi-Markov partitioned survival model to estimate the average per-patient lifetime cost and expected quality-adjusted life years (QALYs) for an allo-HCT patient from a US healthcare system perspective. Key clinical inputs included overall survival, GRFS, incidence of both acute and chronic GVHD, relapse of the primary disease, and infections. Cost results were reported as ranges based on varying the percentage of chronic GVHD patients that remained on treatment after 2 years (15% or 39%). Over a lifetime, the average per-patient medical cost of allo-HCT was estimated to range from $942,373 to $1,247,917. The majority of the costs were for chronic GVHD treatment (37% to 53%), followed by the allo-HCT procedure (15% to 19%). The expected lifetime QALYs of an allo-HCT patient were estimated as 4.7. Lifetime per-patient treatment costs often exceed $1,000,000 for allo-HCT patients. Innovative research efforts focused on the reduction or elimination of late complications, particularly chronic GVHD, may provide the greatest value to improved patient outcomes.

A Model to Estimate Cytokine Release Syndrome and Neurological Event Management Costs Associated With CAR T-Cell Therapy.

Chimeric antigen receptor (CAR) T-cell therapies demonstrated efficacy in relapsed/refractory large B-cell lymphoma (LBCL) but are associated with cytokine release syndrome (CRS) and neurological events (NE). We wanted to estimate the total cost of CRS and NE management among patients with relapsed/refractory LBCL treated with lisocabtagene maraleucel (liso-cel), axicabtagene ciloleucel (axi-cel), or tisagenlecleucel (tisa-cel) in the third- or later-line setting. An economic decision tree model was developed using clinical and economic data to estimate a weighted average per-patient adverse event (AE) management cost from a United States health care system perspective in 2020 dollars. In 2 predefined analyses, mean expected cost and 95% confidence intervals of the average treated patient were estimated via Monte Carlo simulations, with per-patient costs for each CAR T-cell therapy further stratified by AE and grade. In the base case, the overall weighted average per-patient cost was $18,718, $47,665, and $42,538 for liso-cel, axi-cel, and tisa-cel, respectively. The weighted average per-patient cost per CRS event was $8213, $20,442, and $26,009 for liso-cel, axi-cel, and tisa-cel, respectively; the weighted average per-patient cost per NE was $10,505, $27,223, and $16,528, respectively. Differences in the base-case scenario estimated total mean costs for liso-cel were -$28,947 and -$23,819 compared with axi-cel and tisa-cel, respectively. In the scenario analysis (alternative cost input), differences in the estimated total mean costs were -$24,498 for liso-cel versus axi-cel, and -$19,326 for liso-cel versus tisa-cel. Across the base case and scenario analysis, liso-cel had the lowest weighted average CRS and NE costs per treated patient compared with axi-cel and tisa-cel owing to lower incidence rates and symptom severity. These findings highlight the economic implications of differences in safety among CAR T-cell therapies.