Long-Term Outcomes Affect Cost-Effectiveness of CAR T-Cell Therapy for DLBCL

August 2019, Vol 10, No 4 | Payers’ Perspectives In Oncology: ASCO 2019 Highlights - In the Literature, Lymphoma


The 2 chimeric antigen receptor (CAR) T-cell therapies available so far—axicabtagene ciloleucel (Yescarta) and tisagenlecleucel (Kymriah)—may be considered cost-effective treatments for adults with diffuse large B-cell lymphoma (DLBCL), depending on the long-term outcomes of these patients, according to a recent cost-effective analysis (Lin JK, et al. J Clin Oncol. 2019 Jun 3. Epub ahead of print).

DLBCL is the most common type of non-Hodgkin lymphoma, with approximately 61% of patients achieving long-term remission with first-line chemoimmunotherapy. However, patients who experience relapse or become refractory to multiple lines of therapy have poor prognosis. Axicabtagene ciloleucel and tisagenlecleucel are FDA approved for the treatment of relapsed or refractory DLBCL in adults who have received ≥2 lines of systemic therapy or whose disease relapsed ≤12 months after stem-cell transplantation (SCT). Because the costs of the 2 CAR T-cell therapies are high at their list price of $373,000 each, clinicians need to factor in cost-effectiveness when determining the treatment options for this patient population.

“This work is important and timely, especially for health care payers and patients, because it provides evidence on the long-term value and affordability of resources spent on these innovative, yet extremely costly, CAR-T therapies,” wrote Campbell and Whittington in their editorial (Campbell JD, Whittington MD. J Clin Oncol. 2019 Jun 3. Epub ahead of print) accompanying the study publication.

The researchers evaluated the cost-effectiveness and budget impact for each of the 2 CAR T-cell therapies using a Markov model. The main outcomes were undiscounted life-years, discounted lifetime costs, discounted quality-adjusted life-years (QALYs), and incremental cost-effectiveness.

In a scenario with a 40% rate for a 5-year progression-free survival (PFS), axicabtagene ciloleucel increased life expectancy by 8.2 years at $129,000 per QALY gained. At a 30% 5-year PFS rate, the analysis showed modest improvements in life expectancy by 6.4 years, with a $159,000 cost per QALY gained. For tisagenlecleucel, assuming a 35% rate for a 5-year PFS, life expectancy increased by 4.6 years, with a $168,000 cost per QALY gained. At a 25% rate for a 5-year PFS, improvements in life expectancy were smaller, at 3.4 years and more expensive at $223,000 per QALY gained.

In a budget analysis using 2018 prices, the cost of both CAR T-cell therapies was estimated to increase healthcare spending in the United States during the first 5 years by $12 billion for axicabtagene ciloleucel and by $9 billion for tisagenlecleucel. Price reductions to $250,000 for axicabtagene ciloleucel and $200,000 for tisagenlecleucel, or payment only for initial complete response, would result in a cost of less than $150,000 per QALY for each of the CAR T-cell therapies, even at 25% PFS.

“At 2018 prices, it is possible that both CAR-T therapies meet a less than $150,000 per QALY threshold. This depends on long-term outcomes compared with chemoimmunotherapy and SCT, which are uncertain,” concluded the authors. “Widespread adoption would increase non-Hodgkin lymphoma healthcare costs substantially. Price reductions or payment for initial response would improve cost-effectiveness, even with modest long-term outcomes.”