Lingaratnam Senthil, Hussainy Safeera Yasmeen, Murphy Alexandra, Perrin Cale, Samuvel Melbin, Mehrvarz Elahe, Lim Chiao Xin, Zalcberg John
Pharmacy Department, Peter MacCallum Cancer Centre, Melbourne, Australia.
Sir Peter MacCallum Department of Oncology, University of Melbourne, Melbourne, Australia.
J Pharm Policy Pract. 2024 Aug 21;17(1):2389120. doi: 10.1080/20523211.2024.2389120. eCollection 2024.
Regulatory pathways adopted by the United States Food Drug and Administration (FDA) and Australian Therapeutic Goods Administration (TGA) enable expedited approval of medicines that are thought to offer significant clinical advantage over existing options for severe diseases.
To review Australian accessibility to medicines approved through the FDA breakthrough therapy designation (BTD) process including timelines and approvals by the TGA and Pharmaceutical Benefits Advisory Committee (PBAC) for listing on the Pharmaceutical Benefits Scheme (PBS).
Retrospective review of published reports from the FDA, TGA, PBAC and PBS for BTDs from 1 January 2013-31 August 2023. Uniform data about BTD and milestone dates were collected. Analysis included all BTDs approved by FDA until 31-August-2022. Main outcome measures: Rates of approval by TGA and PBAC, and PBS-listing; and median (interquartile range, IQR) time from FDA submission to FDA approval, and FDA approval to TGA approval, PBAC approval and PBS listing for cancer and non-cancer medicines.
Of 237 BTDs across 156 medicines, 68% were approved by the TGA, and 37% were listed on the PBS. Median (IQR) time from FDA submission to FDA approval was shorter for cancer compared to non-cancer; 179 days (140-210) vs 232 days (181-245), < 0.02. Time from FDA approval to PBS listing was similar for cancer and non-cancer; median 744 days (IQR, 549-1136) and 733 days (IQR 440-960) respectively, with improvements for cancer BTDs noted for 2018-2022 compared to 2013-2017; 566 days (IQR 319-831) vs 880 days (IQR 620-1362), < 0.02 but not for non-cancer BTDs.
BTD medicines are accessible in Australia approximately 2 years after FDA approval. Since 2018, time to PBS listing for cancer therapies improved, mirroring shorter FDA approval times for this category. Further understanding of clinical studies and context by therapeutic area may improve timely and safe access to life-saving medicines.
美国食品药品监督管理局（FDA）和澳大利亚治疗用品管理局（TGA）采用的监管途径能够加快对那些被认为相较于现有重症疾病治疗方案具有显著临床优势的药物的审批。
回顾澳大利亚获取通过FDA突破性疗法认定（BTD）程序获批药物的情况，包括TGA和药品福利咨询委员会（PBAC）批准将其列入药品福利计划（PBS）的时间线和审批情况。
对FDA、TGA、PBAC和PBS于2013年1月1日至2023年8月31日发布的关于BTD的报告进行回顾性审查。收集了关于BTD和关键时间节点的统一数据。分析涵盖了截至2022年8月31日FDA批准的所有BTD。主要观察指标：TGA和PBAC的批准率以及PBS列入情况；从向FDA提交申请到FDA批准、从FDA批准到TGA批准、PBAC批准以及PBS列入癌症和非癌症药物的中位（四分位间距，IQR）时间。
在156种药物的237个BTD中，68%获得了TGA批准，37%被列入PBS。从向FDA提交申请到FDA批准，癌症药物的中位（IQR）时间比非癌症药物短；分别为179天（140 - 210）和232天（181 - 245），P < 0.02。从FDA批准到PBS列入，癌症和非癌症药物的时间相似；中位时间分别为744天（IQR，549 - 1136）和733天（IQR 440 - 960），与2013 - 2017年相比，2018 - 2022年癌症BTD的时间有所改善；分别为566天（IQR 319 - 831）和880天（IQR 620 - 1362），P < 0.02，但非癌症BTD无改善。
BTD药物在FDA批准后约2年可在澳大利亚获取。自2018年以来，癌症治疗药物列入PBS的时间有所改善，这与该类别在FDA的批准时间缩短相呼应。按治疗领域进一步了解临床研究和背景情况可能会改善挽救生命药物的及时和安全获取。
