从赞助商的角度看监管机构要求的上市后观察性研究对了解日本人体用药产品效益/风险的贡献。

IF 3.1 Q2 PHARMACOLOGY & PHARMACY
Pharmaceutical Medicine Pub Date : 2024-05-01 Epub Date: 2024-03-31 DOI:10.1007/s40290-024-00521-2
Kevin D Wolter, Asayuki Kamatani, Yumiko Suzuki, Takayuki Imaeda, Ramzi Dagher, Allan Safferman, Rod Junor
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引用次数: 0

摘要

背景:在日本,几乎所有新药或新适应症在获得上市许可后,都必须进行上市后研究(PMS)。这些上市后研究的重点是在批准后的一段预定时间内收集一定数量的病例数据,以确认在日本人群中的疗效、安全性和质量。在其他地区,只有在解决特定科学不确定性时才需要进行 PMS,而在日本,无论是否存在特定科学不确定性,通常都需要进行 PMS,因此其科学价值并不明确:目的:确定 2000-2020 年间辉瑞公司在日本为药品和医疗器械管理局(PMDA)重新审查而进行的 PMS 对了解其效益/风险的贡献:对2000-2020年间辉瑞在日本开展的所有上市后研究(PMS)进行回顾性分析。对现有的辉瑞临床研究报告(CSR)和PMDA复审报告(RER)进行了审查,以了解关键的安全性结论。主要分析对象是既有英文CSR,又在PMDA发布的相关RER中对该PMS进行了讨论的PMS子集,这些RER随后由专业翻译供应商翻译成英文。再审结果包含在每份RER中,用于证明该研究对药物效益/风险概况的影响:针对不同治疗领域的 43 种不同药物产品的 79 项 PMS 共招募了 98,035 名患者。这 79 项 PMS 包括 34 项一般药物使用调查 (GDUI) 研究和 45 项特殊调查 (SI) 研究。主要分析涉及 37 项有英文 CSR 和 RER 的 PMS(40,470 名患者);所有这些研究均为观察性设计。就 37 项 PMS 中的 31 项而言,RER 得出的结论是,PMS 中的总体药物不良反应 (ADR) 率略低于 3 期计划。在 37 个 PMS 中,有 28 个报告了未标示的药物不良反应;但是,没有任何一个 PMS 出现需要采取监管行动的新的安全问题。在接受评估的 79 个 PMS 中,PMDA 没有要求对 43 种药物产品中的任何一种采取额外的风险最小化措施。日本的 PMS 数据与之前的全球数据一致,没有证据表明日本患者在安全性方面存在有临床意义的差异。在所有情况下,复审结果均为第 1 类("有用性得到确认"):结论:复审程序没有导致任何受审药物的监管发生变化。日本新药申请(J-NDA)审查和批准程序,包括最初的日本产品标签的实施,确保了批准时可接受的效益/风险,因此应重新考虑对所有产品进行强制性 GDUI 或 SI 研究。如果存在真正的科学不确定性,以至于产品的效益/风险不明确,则有必要进行 PMS。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
A Sponsor's Perspective on the Contribution of Regulatory-Required Observational Post-Marketing Studies to Understanding Human Drug Product Benefit/Risk in Japan.

Background: Following marketing authorization in Japan, for almost all new drugs or new indications, postmarketing studies (PMS) are a regulatory requirement. These PMS focus on accrual of a defined number of cases with data being collected for a predetermined period after approval to confirm efficacy/effectiveness, safety, and quality in the Japanese population. In contrast to other regions where PMS are only required to address a specific scientific uncertainty, in Japan, PMS are often required regardless of any specific scientific uncertainty, and therefore, their scientific value is unclear.

Objectives: To determine the contribution to the understanding of benefit/risk of PMS conducted by Pfizer in Japan over 2000-2020 for Pharmaceuticals and Medical Devices Agency (PMDA) reexamination.

Methods: A retrospective analysis of all Pfizer Japan postmarketing studies (PMS) during 2000-2020 was performed. Available Pfizer clinical study reports (CSRs) and PMDA reexamination reports (RERs) were reviewed for key safety findings. The primary analysis was conducted on the subset of PMS that had both an English CSR and a discussion of that PMS in the relevant RER issued by the PMDA, which was subsequently translated into English by a professional translation vendor. Reexamination outcome is included in each RER and served to demonstrate the impact of the study of the benefit/risk profile of the drug.

Results: A total of 79 PMS for 43 different drug products across therapy areas enrolled a total of 98,035 patients. The 79 PMS comprised 34 general drug use investigation (GDUI) studies and 45 special investigation (SI) studies. The primary analysis involved 37 PMS with a CSR and RER available in English (40,470 patients); all of which were observational in design. For 31 of 37 PMS, the RER concluded the overall adverse drug reaction (ADR) rate in the PMS was nominally lower than in the phase 3 program. Unlabeled ADRs were reported in 28 of 37 PMS; however, no new safety concerns requiring regulatory action arose from any PMS. The PMDA did not require additional risk minimization measures for any of the 43 drug products studied in any of the 79 PMS assessed. Japan PMS data were consistent with prior global data with no evidence of clinically meaningful differences in safety in Japanese patients. In all cases, the reexamination outcome was category 1 ("usefulness is confirmed").

Conclusions: The reexamination process did not result in regulatory changes for any of the examined drugs. The Japan new-drug application (J-NDA) review and approval process, including implementation of the initial Japan product label, assures acceptable benefit/risk at the time of approval such that mandatory GDUI or SI studies for all products should be reconsidered. In the case of genuine scientific uncertainty to the extent that the benefit/risk of the product is not clear, a PMS is warranted.

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来源期刊
Pharmaceutical Medicine
Pharmaceutical Medicine PHARMACOLOGY & PHARMACY-
CiteScore
5.10
自引率
4.00%
发文量
36
期刊介绍: Pharmaceutical Medicine is a specialist discipline concerned with medical aspects of the discovery, development, evaluation, registration, regulation, monitoring, marketing, distribution and pricing of medicines, drug-device and drug-diagnostic combinations. The Journal disseminates information to support the community of professionals working in these highly inter-related functions. Key areas include translational medicine, clinical trial design, pharmacovigilance, clinical toxicology, drug regulation, clinical pharmacology, biostatistics and pharmacoeconomics. The Journal includes:Overviews of contentious or emerging issues.Comprehensive narrative reviews that provide an authoritative source of information on topical issues.Systematic reviews that collate empirical evidence to answer a specific research question, using explicit, systematic methods as outlined by PRISMA statement.Original research articles reporting the results of well-designed studies with a strong link to wider areas of clinical research.Additional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in Pharmaceutical Medicine may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances.All manuscripts are subject to peer review by international experts. Letters to the Editor are welcomed and will be considered for publication.
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