Department of Research and Development, Netherlands Comprehensive Cancer Organization, Utrecht, the Netherlands.
Erasmus School of Health Policy and Management, Erasmus University Rotterdam, Rotterdam, the Netherlands.
JAMA Netw Open. 2023 Feb 1;6(2):e2256170. doi: 10.1001/jamanetworkopen.2022.56170.
This study found that CDK4/6 inhibitors rapidly reached many eligible patients with metastatic breast cancer and were adopted gradually over time in the Netherlands. Adoption of innovative medicines may be further optimized, and better transparency of the availability of new medicines during different phases of the postapproval access pathway is needed.
DESIGN, SETTING, AND PARTICIPANTS: This cohort study reviewed approval and reimbursement decisions of the CDK4/6 inhibitors palbociclib, ribociclib, and abemaciclib and estimated the number of patients with metastatic breast cancer who were eligible for these medicines compared with the actual use in clinical practice. The study used nationwide claims data that were obtained from the Dutch Hospital Data. Claims and early access data for patients with hormone receptor-positive and ERBB2 (formerly HER2)-negative metastatic breast cancer who were treated with CDK4/6 inhibitors from November 1, 2016, to December 31, 2021, were included.
The number of new cancer medicines that are being approved by regulatory agents is increasing exponentially. Yet little is known about the pace at which these medicines reach eligible patients in daily clinical practice during different phases of the postapproval access pathway.
Description of the postapproval access pathway, monthly number of patients who were treated with CDK4/6 inhibitors in clinical practice, and estimated number of patients who were eligible for treatment. Aggregated claims data were used, and patient characteristics and outcomes data were not collected.
To describe the entire postapproval access pathway of cyclin-dependent kinase 4/6 (CDK4/6) inhibitors in the Netherlands, from regulatory approval to reimbursement and to investigate the adoption of these medicines in clinical practice among patients with metastatic breast cancer.
Three CDK4/6 inhibitors have received European Union-wide regulatory approval for the treatment of HR-positive and ERBB2-negative metastatic breast cancer since November 2016. In the Netherlands, the number of patients who have been treated with these medicines increased to approximately 1847 (based on 1 624 665 claims over the entire study period) from approval to the end of 2021. Reimbursement for these medicines was granted between 9 and 11 months after approval. While awaiting reimbursement decisions, 492 patients received palbociclib, the first approved medicine of this class, via an expanded access program. By the end of the study period, 1616 patients (87%) were treated with palbociclib, whereas 157 patients (7%) received ribociclib, and 74 patients (4%) received abemaciclib. The CKD4/6 inhibitor was combined with an aromatase inhibitor in 708 patients (38%) and with fulvestrant in 1139 patients (62%). The pattern of use over time appeared to be somewhat lower compared with the estimated number of eligible patients (1847 vs 1915 in December 2021), especially in the first 2.5 years after approval.
本研究发现，CDK4/6 抑制剂迅速惠及了许多转移性乳腺癌的合格患者，并且在荷兰逐渐被采用。创新药物的采用可能会进一步优化，并且需要更好地透明化新药物在批准后准入途径的不同阶段的可获得性。
设计、设置和参与者：本队列研究回顾了 CDK4/6 抑制剂 palbociclib、ribociclib 和 abemaciclib 的批准和报销决定，并估计了与实际临床实践中使用相比，有多少转移性乳腺癌患者符合这些药物的条件。该研究使用了全国性的索赔数据，这些数据是从荷兰医院数据中获得的。纳入了 2016 年 11 月 1 日至 2021 年 12 月 31 日期间接受 CDK4/6 抑制剂治疗的激素受体阳性和 ERBB2（以前称为 HER2）阴性转移性乳腺癌患者的索赔和早期准入数据。
监管机构批准的新癌症药物数量呈指数级增长。然而，在批准后准入途径的不同阶段，这些药物在日常临床实践中惠及合格患者的速度知之甚少。
描述批准后准入途径、临床实践中每月接受 CDK4/6 抑制剂治疗的患者数量以及符合治疗条件的患者数量。使用了汇总的索赔数据，并未收集患者特征和结局数据。
描述 CDK4/6 抑制剂在荷兰的整个批准后准入途径，从监管批准到报销，并调查这些药物在转移性乳腺癌患者中的临床实践中的采用情况。
自 2016 年 11 月以来，三种 CDK4/6 抑制剂已获得欧盟范围内用于治疗 HR 阳性和 ERBB2 阴性转移性乳腺癌的监管批准。在荷兰，从批准到 2021 年底，接受这些药物治疗的患者数量增加到大约 1847 人（整个研究期间基于 1624665 份索赔）。这些药物的报销在批准后 9 至 11 个月内获得批准。在等待报销决定期间，492 名患者通过扩大准入计划接受了 palbociclib，这是该类药物中的第一种获批药物。到研究结束时，1616 名患者（87%）接受了 palbociclib 治疗，而 157 名患者（7%）接受了 ribociclib 治疗，74 名患者（4%）接受了 abemaciclib 治疗。在 708 名患者（38%）中，CDK4/6 抑制剂与芳香化酶抑制剂联合使用，在 1139 名患者（62%）中与 fulvestrant 联合使用。随着时间的推移，这种使用模式似乎比估计的合格患者数量（2021 年 12 月的 1847 名与 1915 名）略低，尤其是在批准后的头 2.5 年。
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