Cytel, Rotterdam, Netherlands.
Cytel, London, UK.
J Comp Eff Res. 2024 Feb;13(2):e230140. doi: 10.57264/cer-2023-0140. Epub 2024 Jan 4.
The drive to expedite patient access for diseases with high unmet treatment needs has come with an increasing use of single-arm trials (SATs), especially in oncology. However, the lack of control arms in such trials creates challenges to assess and demonstrate comparative efficacy. External control (EC) arms can be used to bridge this gap, with various types of sources available to obtain relevant data. To examine the source of ECs in single-arm oncology health technology assessment (HTA) submissions to the National Institute for Health and Care Excellence (NICE) and the Pharmaceutical Benefits Advisory Committee (PBAC) and how this selection was justified by manufacturers and assessed by the respective HTA body. Single-arm oncology HTA submission reports published by NICE (England) and PBAC (Australia) from January 2011 to August 2021 were reviewed, with data qualitatively synthesized to identify themes. Forty-eight oncology submissions using EC arms between 2011 and 2021 were identified, with most submissions encompassing blood and bone marrow cancers (52%). In HTA submissions to NICE and PBAC, the EC arm was typically constructed from a combination of data sources, with the company's justification in data source selection infrequently provided (PBAC [2 out of 19]; NICE [6 out of 29]), although this lack of justification was not heavily criticized by either HTA body. Although HTA bodies such as NICE and PBAC encourage that EC source justification should be provided in submissions, this review found that this is not typically implemented in practice. Guidance is needed to establish best practices as to how EC selection should be documented in HTA submissions.
为满足高未满足治疗需求疾病的患者尽快获得治疗的需求，单臂试验（SAT）的应用越来越多，尤其是在肿瘤学领域。然而，此类试验缺乏对照臂，这给评估和证明比较疗效带来了挑战。外部对照（EC）臂可用于弥补这一差距，有多种来源可获取相关数据。本研究旨在考察英国国家卫生与保健优化研究所（NICE）和澳大利亚药品福利咨询委员会（PBAC）接受的单臂肿瘤学卫生技术评估（HTA）提交材料中 EC 臂的来源，以及制造商如何为其选择提供依据，以及相应的 HTA 机构如何对其进行评估。对 NICE（英格兰）和 PBAC（澳大利亚）自 2011 年 1 月至 2021 年 8 月发表的单臂肿瘤学 HTA 提交报告进行了回顾，对数据进行了定性综合，以确定主题。共确定了 2011 年至 2021 年间使用 EC 臂的 48 份肿瘤学提交材料，其中大多数提交材料涵盖血液和骨髓癌（52%）。在 NICE 和 PBAC 的 HTA 提交材料中，EC 臂通常由多种数据源组合构建，公司对数据源选择的论证很少提供（PBAC [19 项中有 2 项]；NICE [29 项中有 6 项]），尽管这并没有受到两个 HTA 机构的严厉批评。尽管 NICE 和 PBAC 等 HTA 机构鼓励在提交材料中提供 EC 来源论证，但本研究发现，这在实践中通常并未得到实施。需要有指导意见来确定最佳实践，即在 HTA 提交材料中应如何记录 EC 选择。
