澳大利亚考虑资助的癌症药物的临床获益证据。
Evidence of clinical benefit of cancer medicines considered for funding in Australia.
机构信息
Clinical and Health Sciences, University of South Australia, Adelaide, SA, Australia.
Department of Clinical Pharmacology, Flinders Medical Centre and Flinders University, Bedford Park, SA, Australia.
出版信息
Int J Technol Assess Health Care. 2024 Nov 14;40(1):e55. doi: 10.1017/S0266462324000576.
OBJECTIVES
To describe the type of evidence and the clinical benefit of cancer medicines assessed for funding in Australia by the Pharmaceutical Benefits Advisory Committee (PBAC) and to assess it with the European Society of Medical Oncology Magnitude of Clinical Benefit Scale version 1.1 (ESMO-MCBS).
METHODS
All data on applications submitted to PBAC between 2010 and 2020 were extracted from PBAC Public Summary Documents available online. ESMO-MCBS ratings were retrieved from the ESMO-MCBS website.
RESULTS
Then, 182 cancer indications for 100 cancer medicines were examined by PBAC, including 124 (68.1 percent) for solid tumors and 58 (31.9 percent) for hematological cancers. A total of 137 (75.3 percent) indications were recommended for PBS funding and 40 (21.9 percent) were rejected. Randomized clinical trials (RCTs) were the main source of evidence in 154 indications (84.6 percent), single-arm studies in 28 (15.4 percent) indications. Statistically significant improvement in overall survival (OS) was reported in 80 (44 percent) of the indications, with a median OS gain of 3.0 months (range 0.9-17.0) for solid tumors and 8.2 months (range 1-49.1) for hematological cancers when mature OS data were available. The ESMO-MCBS score was available for 99 solid tumor indications, of which 51 (51.5 percent) showed substantial clinical benefit according to ESMO-MCBS, including 40 (54.1 percent) of PBAC-recommended indications and 9 (42.9 percent) of PBAC-rejected indications. There was no association between the ESMO scoring and PBAC decision.
CONCLUSIONS
Most cancer medicines indications considered by PBAC were supported by RCTs. A minority showed a substantial improvement in OS.
目的
描述澳大利亚药品福利咨询委员会（PBAC）评估的癌症药物的证据类型和临床获益，并使用欧洲肿瘤内科学会临床获益量表 1.1 版（ESMO-MCBS）进行评估。
方法
从 PBAC 在线提供的公共摘要文件中提取 2010 年至 2020 年提交给 PBAC 的所有申请数据。从 ESMO-MCBS 网站获取 ESMO-MCBS 评分。
结果
然后，PBAC 共检查了 100 种癌症药物的 182 种癌症适应证，其中 124 种（68.1%）为实体瘤，58 种（31.9%）为血液系统癌症。共有 137 种（75.3%）适应证被推荐用于 PBS 资助，40 种（21.9%）被拒绝。随机对照试验（RCT）是 154 种适应证（84.6%）的主要证据来源，28 种适应证（15.4%）为单臂研究。在有成熟 OS 数据时，80 种适应证（44%）报告了总生存期（OS）的统计学显著改善，其中实体瘤的中位 OS 获益为 3.0 个月（范围 0.9-17.0），血液系统癌症为 8.2 个月（范围 1-49.1）。99 种实体瘤适应证的 ESMO-MCBS 评分可用，其中 51 种（51.5%）根据 ESMO-MCBS 显示出显著的临床获益，包括 40 种（54.1%）PBAC 推荐适应证和 9 种（42.9%）PBAC 拒绝适应证。ESMO 评分与 PBAC 决策之间没有关联。
结论
PBAC 评估的大多数癌症药物适应证都得到了 RCT 的支持。少数药物在 OS 方面有显著改善。
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