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Cost-Effectiveness of Anti-BCMA Chimeric Antigen Receptor T Cell Therapy in Relapsed/Refractory Multiple Myeloma

•We evaluated the cost-effectiveness of anti-BCMA CAR-T therapy for heavily pretreated relapsed and refractory multiple myeloma patients was assessed using a Markov model incorporating various uncertainties.•The parameter that most significantly impacted cost-effectiveness was the assumption for lon...

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Published in:Transplantation and cellular therapy 2024-01, Vol.30 (1), p.118.e1-118.e15
Main Authors: Yamamoto, Chihiro, Minakata, Daisuke, Yokoyama, Daizo, Furuki, Shuka, Noguchi, Atsuto, Koyama, Shunsuke, Oyama, Takashi, Murahashi, Rui, Nakashima, Hirotomo, Ikeda, Takashi, Kawaguchi, Shin-ichiro, Hyodo, Kazuki, Toda, Yumiko, Ito, Shoko, Nagayama, Takashi, Umino, Kento, Morita, Kaoru, Ashizawa, Masahiro, Ueda, Masuzu, Hatano, Kaoru, Sato, Kazuya, Ohmine, Ken, Fujiwara, Shin-ichiro, Kanda, Yoshinobu
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Language:English
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Summary:•We evaluated the cost-effectiveness of anti-BCMA CAR-T therapy for heavily pretreated relapsed and refractory multiple myeloma patients was assessed using a Markov model incorporating various uncertainties.•The parameter that most significantly impacted cost-effectiveness was the assumption for long-term PFS with CAR-T.•Although anti-BCMA CAR-T is associated with extremely high costs at the start, it has the potential to be cost-effective under the optimistic PFS assumption. Despite its promising outcomes, anti-BCMA chimeric antigen receptor T cell therapy (CAR-T) is the most expensive myeloma treatment developed to date, and its cost-effectiveness is an important issue. This study aimed to assess the cost-effectiveness of anti-BCMA CAR-T compared to standard antimyeloma therapy in patients with relapsed/refractory multiple myeloma. The model included myeloma patients in Japan and the United States who have received ≥3 prior lines of antimyeloma therapy, including immunomodulatory drugs, proteasome inhibitors, and anti-CD38 monoclonal antibodies. A Markov model was constructed to compare the CAR-T strategy, in which patients receive either idecabtagene vicleucel (ide-cel) or ciltacabtagene autoleucel (cilta-cel) followed by 3 lines of multiagent chemotherapy after relapse, and the no CAR-T strategy, in which patients receive only chemotherapy. Data from the LocoMMotion, KarMMa, and CARTITUDE-1 trials were extracted. Several assumptions were made regarding long-term progression-free survival (PFS) with CAR-T. Extensive scenario analyses were made regarding regimens for no CAR-T strategies. The outcome was an incremental cost-effectiveness ratio (ICER) with willingness-to-pay thresholds of ¥7,500,000 in Japan and $150,000 in the United States. When a 5-year PFS of 40% with cilta-cel was assumed, the ICER of the CAR-T strategy versus the no CAR-T strategy was ¥7,603,823 per QALY in Japan and $112,191 per QALY in the United States over a 10-year time horizon. When a 5-year PFS of 15% with ide-cel was assumed, the ICER was ¥20,388,711 per QALY in Japan and $261,678 per QALY in the United States over a 10-year time horizon. The results were highly dependent on the PFS assumption with CAR-T and were robust to changes in most other parameters and scenarios. Although anti-BCMA CAR-T can be cost-effective even under current pricing, a high long-term PFS is necessary.
ISSN:2666-6367
2666-6367
DOI:10.1016/j.jtct.2023.10.001