ABSTRACT

Introduction: Novel immunotherapeutic agents (e.g. monoclonal antibodies, antibody-drug conjugates, bispecific T-cell engagers) as treatment options for hematologic malignancies continue to emerge. These agents have been used as the standard of care in specific disease states and are associated with high costs. Value assessment of these therapies is of critical importance for coverage and reimbursement decision-making.

Areas covered: We identified 15 immunotherapeutic agents through the U.S. FDA approvals for hematologic malignancies until 2018 and systematically reviewed related cost-effectiveness studies. Additionally, we examined whether drug wastage was accounted for in these studies.

Expert opinion: We reviewed 51 studies for 14 identified immunotherapeutic agents that met the inclusion criteria for this systematic review. Three studies were observational-based, one study was model-based and incorporated observational data. The remaining studies were model-based with the majority of the model parameters extracted from randomized control trials (RCTs). Among 43 model-based economic evaluations, 13 studies accounted for drug wastage. Most of the studies showed favorable incremental cost-effectiveness ratios of immunotherapeutic agents-containing regimens when compared with no immunotherapeutic agents-containing regimens. Alemtuzumab, brentuximab vedotin, and daratumumab were not considered cost-effective across all the studies. Further investigations are warranted to establish the value of recent immunotherapeutic agents for hematologic malignancies.

摘要

简介： 新型免疫治疗剂（例如单克隆抗体、抗体药物偶联物、双特异性 T 细胞接合剂）作为血液系统恶性肿瘤的治疗选择不断出现。这些药物已被用作特定疾病状态的护理标准，并且成本高昂。这些疗法的价值评估对于覆盖和报销决策至关重要。

涵盖领域：截至 2018 年，我们通过美国 FDA 对血液系统恶性肿瘤的批准确定了 15 种免疫治疗药物，并系统地审查了相关的成本效益研究。此外，我们检查了这些研究中是否考虑了药物浪费。

专家意见：我们审查了 51 项研究，涉及 14 种已确定的免疫治疗药物，这些药物符合本系统审查的纳入标准。三项研究是基于观察的，一项研究是基于模型的并结合了观察数据。其余研究是基于模型的，大部分模型参数从随机对照试验 (RCT) 中提取。在 43 项基于模型的经济评估中，13 项研究考虑了药物浪费。大多数研究表明，与不含免疫治疗剂的方案相比，含免疫治疗剂的方案具有有利的增量成本效益比。在所有研究中，阿仑单抗、brentuximab vedotin 和 daratumumab 均未被认为具有成本效益。