加拿大卫生部在监管决策中使用真实世界证据（RWE）的情况与基于公开信息的 FDA/EMA 使用情况的比较。
Health Canada Usage of Real World Evidence (RWE) in Regulatory Decision Making compared with FDA/EMA usage based on publicly available information.
机构信息
Catherine Lau, Ph.D., Consultant, Toronto, Ontario, Canada.
Faculty of Pharmacy and Pharmaceutical Sciences, University of Alberta, Edmonton AB, Canada.
出版信息
J Pharm Pharm Sci. 2022;25:227-236. doi: 10.18433/jpps32715.
PURPOSE
Between January 2020 and December 2021, Health Canada provided a Summary Basis of Decision (SBD) for each of 110 products approved, including 29 oncology products and 21 non-oncology orphan drugs. This review sought to gain insight into how Real Word Evidence (RWE) impacts regulatory decision making.
METHODS
SBDs for oncology drugs and non-oncology orphan drugs were reviewed for evidence of use of the RWE or historical data to support regulatory decisions. This information was compared with both FDA and EMA reviews.
RESULTS
For the 29 Health Canada-approved oncology products, 11 were approved with Notice of Compliance with Conditions (NOCc) status. Two NOCc approvals received extensive RWE reviews, while two other approvals briefly mentioned the use of RWE/historical data. Of the 12 NOC approvals, one received RWE reviews. FDA also approved all 29 drugs, 14 of which received extensive comments on RWE and/or historical data and 8 of which mentioned RWE or historical data. EMA approved 25 of the 29 products and provided extensive comments on 10. Four products received a mention of RWE review. The percentages of submissions with RWE/historical reviews conducted by Health Canada, FDA and EMA were 24.1, 75.9 and 56.0 respectively. Of the 21 non-oncology orphan drugs, Health Canada provided priority review status to 11, with extensive RWE comments in 5 and the mention of RWE in 2 of the regular approvals. Two approvals that used third-party data were not included in the comparison. FDA approved 19, and provided extensive RWE assessment on 5 and mentioned use of historical data in 8. EMA approved 17 and provided extensive RWE and historical comments in 7 and mentioned historical data in 4. The percentages of submissions with RWE/historical reviews by Health Canada, FDA and EMA were 36.8, 68.4 and 64.7 respectively.
CONCLUSIONS
Use of Real World Data is common among FDA/EMA reviews and Health Canada used RWE in recent NOCc and orphan drug approvals.
目的
在 2020 年 1 月至 2021 年 12 月期间，加拿大卫生部为 110 种获批产品（包括 29 种肿瘤药物和 21 种非肿瘤孤儿药）中的每一种都提供了综合基本决策（SBD）。本研究旨在深入了解真实世界证据（RWE）如何影响监管决策。
方法
对肿瘤药物和非肿瘤孤儿药物的 SBD 进行审查，以确定是否有使用 RWE 或历史数据来支持监管决策的证据。将这些信息与 FDA 和 EMA 的审查进行了比较。
结果
在加拿大卫生部批准的 29 种肿瘤药物中，有 11 种药物获得了附有条件的合规通知（NOCc）批准。两项 NOCc 批准进行了广泛的 RWE 审查，而另外两项批准则简要提及了 RWE/历史数据的使用。在 12 项 NOC 批准中，有一项获得了 RWE 审查。FDA 也批准了所有 29 种药物，其中 14 种药物的 RWE 和/或历史数据受到了广泛的评论，8 种药物提到了 RWE 或历史数据。EMA 批准了 29 种产品中的 25 种，并对其中 10 种产品的 RWE 进行了详细的评论。有 4 种产品的 RWE 审查情况被提及。加拿大卫生部、FDA 和 EMA 进行 RWE/历史数据审查的提交比例分别为 24.1%、75.9%和 56.0%。在 21 种非肿瘤孤儿药物中，加拿大卫生部对 11 种药物给予了优先审查，其中 5 种药物的 RWE 评论广泛，2 种常规批准药物也提到了 RWE。有两种使用第三方数据的批准未被包括在比较范围内。FDA 批准了 19 种药物，并对其中 5 种药物进行了广泛的 RWE 评估，对 8 种药物提到了使用历史数据。EMA 批准了 17 种药物，并对其中 7 种药物进行了广泛的 RWE 和历史数据评论，对 4 种药物提到了历史数据。加拿大卫生部、FDA 和 EMA 进行 RWE/历史数据审查的提交比例分别为 36.8%、68.4%和 64.7%。
结论
FDA/EMA 审查中经常使用真实世界数据，加拿大卫生部在最近的 NOCc 和孤儿药批准中使用了 RWE。
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