Supplementary materials: Justifying the source of external comparators in single-arm oncology health technology submissions: a review of NICE and PBAC assessments

These are peer-reviewed supplementary tables for the article 'Justifying the source of external comparators in single-arm oncology health technology submissions: a review of NICE and PBAC assessments' published in the Journal of Comparative Effectiveness Research.Supplemental Table 1: Summary of included submissionsSupplemental Table 2: Main external control critiques across NICE and PBAC submissions for the same technologiesBackground: 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. Objective: 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. Methods: 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. Results: 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. Conclusion: 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.

本数据集为发表于《比较效果研究杂志》的论文《论单臂临床试验中外部比较者来源的合理性：NICE 和 PBAC 评估综述》的同行评审补充表格。补充表格 1：纳入提交的总结；补充表格 2：NICE 和 PBAC 提交中相同技术的主要外部控制批判。背景：为加速满足高度未满足治疗需求的疾病患者的药物可及性，单臂试验（SATs）的使用量不断增加，尤其是在肿瘤学领域。然而，此类试验缺乏对照组，给评估和证明比较疗效带来了挑战。外部控制（EC）臂可以用来弥补这一差距，并可通过多种途径获取相关数据。目标：审查提交给英国国家健康与临床卓越研究所（NICE）和澳大利亚药品福利咨询委员会（PBAC）的单臂肿瘤学健康技术评估（HTA）提交中外部控制（EC）的来源，以及制造商如何证明这一选择，以及相关 HTA 机构如何评估。方法：对 2011 年 1 月至 2021 年 8 月期间由 NICE（英格兰）和 PBAC（澳大利亚）发布的单臂肿瘤学 HTA 提交报告进行了审查，通过定性合成数据以识别主题。结果：在 2011 年至 2021 年间，共识别出 48 项使用外部控制臂的肿瘤学提交，其中大部分提交涉及血液和骨髓癌（52%）。在提交给 NICE 和 PBAC 的 HTA 中，外部控制臂通常由多种数据源组合而成，公司在数据源选择方面的理由很少提供（PBAC [19 项中的 2 项]；NICE [29 项中的 6 项]），尽管这种缺乏理由并未受到两个 HTA 机构的高度批评。结论：尽管 NICE 和 PBAC 等HTA机构鼓励在提交中提供外部控制来源的合理性说明，但本综述发现，在实践中通常并未实施。需要指导以建立最佳实践，以确定如何在 HTA 提交中记录外部控制的选择。