Cost Effectiveness of Chimeric Antigen Receptor T-Cell Therapy in Multiply Relapsed or Refractory Adult Large B-Cell Lymphoma

Abstract

PURPOSE Two anti-CD19 chimeric antigen receptor T-cell (CAR-T) therapies are approved for diffuse large B-cell lymphoma, axicabtagene ciloleucel (axi-cel) and tisagenlecleucel; each costs $373,000. We evaluated their cost effectiveness. METHODS We used a decision analytic Markov model informed by recent multicenter, single-arm trials to evaluate axi-cel and tisagenlecleucel in multiply relapsed/refractory, adult, diffuse large B-cell lymphoma from a US health payer perspective over a lifetime horizon. Under a range of plausible long-term effectiveness assumptions, each therapy was compared with salvage chemoimmunotherapy regimens and stem-cell transplantation. Main outcomes were undiscounted life years, discounted lifetime costs, discounted quality-adjusted life years (QALYs), and incremental cost-effectiveness ratio (3% annual discount rate). Sensitivity analyses explored uncertainty. RESULTS In an optimistic scenario, assuming a 40% 5-year progression-free survival (PFS), axi-cel increased life expectancy by 8.2 years at $129,000/QALY gained (95% uncertainty interval, $90,000 to $219,000). At a 30% 5-year PFS, improvements in life expectancy were more modest (6.4 years) and expensive ($159,000/QALY gained [95% uncertainty interval, $105,000 to $284,000]). In an optimistic scenario, assuming a 35% 5-year PFS, tisagenlecleucel increased life expectancy by 4.6 years at $168,000/QALY gained (95% uncertainty interval, $105,000 to $414,000/QALY). At a 25% 5-year PFS, improvements in life expectancy were smaller (3.4 years) and more expensive ($223,000/QALY gained [95% uncertainty interval, $123,000 to $1,170,000/QALY]). Administering CAR-T to all indicated patients would increase US health care costs by approximately $10 billion over 5 years. Price reductions to $250,000 and $200,000, respectively, or payment only for initial complete response (at current prices) would allow axi-cel and tisagenlecleucel to cost less than $150,000/QALY, even at 25% PFS. CONCLUSION At 2018 prices, it is possible that both CAR-T therapies meet a less than $150,000/QALY threshold. This depends on long-term outcomes compared with chemoimmunotherapy and stem-cell transplantation, which are uncertain. Widespread adoption would substantially increase non-Hodgkin lymphoma health care costs. Price reductions or payment for initial response would improve cost effectiveness, even with modest long-term outcomes.

Upsetting the apple CAR‐T (chimeric antigen receptor T‐cell therapy) ‐ sustainability mandates USA innovation

“…Value is clearly evident in paediatric acute leukaemia, where children who would have otherwise succumbed achieved a 66% survival rate at 18‐months 3 . However, the value proposition has been questioned in DLBCL 4 . Further insight is being generated by the accumulation of real‐world outcomes of CAR‐T in the USA 5,6 and in the UK.…”

Psychosocial care for children receiving chimeric antigen receptor (CAR) T‐cell therapy

“…The prevalence of financial hardship is likely similarly underidentified among patients undergoing CAR T‐cell therapies. Although a recent report evaluated the cost‐effectiveness of CAR T‐cell therapies, potential patient‐specific financial toxicities were not examined 42 . However, long‐term financial toxicity is a well‐known complication of surviving childhood cancer.…”

Am individuellen Therapieergebnis orientierte Erstattungsverfahren in der Onkologie: ethische Implikationen am Beispiel der CAR-T-Zelltherapie

“…"Pay for performance", auch P4P abgekürzt, zu Deutsch: erfolgsabhängige Erstattung, bezeichnet im angloamerikanischen Raum Programme, die die Höhe des ärztlichen Gehalts an den globalen Erfolg bei der Behandlung einer bestimmten Erkrankung oder bei der Früherkennung koppeln. Um die Qualität der ärztlichen Versorgung zu verbessern, wird so für den Arzt ein Anreiz geschaffen, in seiner Praxis etablierte Programme einzusetzen, die nachgewiesenermaßen für die Gruppe der Patienten einen Mehrwert darstellen (Lin et al 2019;Matthes 2019). Die Bezeichnung ist jedoch für das hier verhandelte Erstattungsmodell irreführend.…”

“…Das Hauptziel der Therapie mit CAR-T Zellen bei Patienten mit Leukämien und Lymphomen ist die Verlängerung menschlichen Lebens. Dieses Ziel wurde in den pivotalen klinischen Prüfungen der aktuell zugelassenen Präparate gemessen an den Gesamtüberlebenszeiten als primären Endpunkten der jeweiligen Studien erreicht (Lin et al 2019;Neelapu et al 2018).…”