Background: Continuous oral targeted therapy (OTT) for chronic lymphocytic leukemia (CLL) represents an effective therapy but also a major economic burden on the healthcare system. This study aimed to estimate future direct costs, along with the prevalence, of CLL in the era of continuous OTT in Canada. Methods: The economic burden of OTT was modelled and compared to chemoimmunotherapy (CIT), for CLL treatment. The burden was assessed/projected from 2011 to 2025. For the OTT scenario, CIT was considered the standard of care before 2015, while OTT was considered standard of care for patients with either unmutated immunoglobulin heavy-chain variable (IGHV) or del(17p)/TP53 mutations starting in 2015 and, from 2020 onwards, for all first-line treatments except for patients with mutated IGHV. A Markov model was developed including four health states: watchful-waiting, first-line treatment, relapse and death. Costs of therapy, follow-up/monitoring and adverse events were included. Key clinical parameters were extracted from pivotal clinical trials. Results: As incidence rates and rate of survival are increasing, the prevalence of CLL in Canada is projected to increase 1.8-fold, from 8301 patients in 2011 to 14,654 by 2025. Correspondingly, the total annual costs of CLL management are predicted to increase 15.7-fold, from $60.8 million to $957.5 million during that same period. Conclusions: Although OTT enhances survival for patients with CLL, it is nonetheless associated with an important economic burden due to the projected vast increase in costs from 2011 to 2025. Changes in clinical strategies, such as implementation of a fixed OTT treatment duration, could help alleviate financial burden.

中文翻译：

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口服靶向治疗对加拿大慢性淋巴细胞白血病经济负担的影响

背景：慢性淋巴细胞白血病 (CLL) 的连续口服靶向治疗 (OTT) 是一种有效的治疗方法，但也是医疗保健系统的主要经济负担。本研究旨在估计加拿大持续 OTT 时代 CLL 的未来直接成本以及患病率。方法：对 OTT 的经济负担进行建模，并将其与化学免疫疗法 (CIT) 进行比较，用于 CLL 治疗。对 2011 年至 2025 年的负担进行了评估/预测。 对于 OTT 情景，CIT 被认为是 2015 年之前的护理标准，而 OTT 被认为是具有未突变免疫球蛋白重链变量 (IGHV) 或 del(17p) 的患者的标准护理)/TP53 突变从 2015 年开始，从 2020 年开始，用于所有一线治疗，但 IGHV 突变患者除外。马尔可夫模型包括四种健康状态：观察等待，一线治疗，复发死亡。包括治疗、随访/监测和不良事件的费用。从关键临床试验中提取关键临床参数。结果：随着发病率和生存率的提高，加拿大 CLL 的患病率预计将增加 1.8 倍，从 2011 年的 8301 名患者增加到 2025 年的 14,654 名。相应地，CLL 管理的年度总成本预计将增加 15.7倍，同期从 6080 万美元增加到 9.575 亿美元。结论：虽然 OTT 提高了 CLL 患者的生存率，但由于预计 2011 年至 2025 年的费用将大幅增加，因此它也带来了重要的经济负担。临床策略的变化，例如实施固定的 OTT 治疗持续时间，可能会有所帮助减轻经济负担。