Survey of Data Package and Sample Size of Comparative Clinical Studies for Biosimilar Developments from PMDA Assessments.

IF 3.1 Q2 PHARMACOLOGY & PHARMACY

Pharmaceutical Medicine Pub Date : 2024-05-01 Epub Date: 2024-04-29 DOI:10.1007/s40290-024-00525-y

Ryosuke Kuribayashi, Aya Hariu, Ayuki Nakano, Yasuhiro Kishioka

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引用次数: 0

Abstract

Background: The Japanese biosimilar guideline requires that the sponsors conduct clinical studies such as comparative pharmacokinetic (PK), pharmacodynamic (PD), or efficacy studies. In each biosimilar development, the sponsors consider the clinical data package, and thus clinical data packages vary among biosimilar developments.

Objectives: The aim of this study was to elucidate the clinical data packages for the biosimilars approved in Japan. The details of clinical data packages and sample size for the regulatory approvals of biosimilars in Japan was reported.

Methods: We surveyed the clinical data packages and sample size based on the Pharmaceuticals and Medical Devices Agency (PMDA) website review reports between 2009 and 2023.

Results: Twenty-four biosimilars have been approved based on the comparative PK and efficacy studies, 10 biosimilars have been approved based on the comparative PK/PD study, and one biosimilar has been approved based on the comparative efficacy study. Regarding the sample size, comparative PK studies were conducted in healthy volunteers or patients for up to 300 cases, although the majority enrolled only 1-100 cases (68.1%, 32/47). Comparative PD studies enrolling 1-30, 31-60, and 61-90 cases totaled 4, 7, and 4 cases, respectively. Finally, comparative efficacy studies enrolling 1-300, 301-600, and 601-900 totaled 6, 10, and 11 cases, respectively. In particular, the oncology and rheumatology areas were the first and second disease areas recruiting 601-900 patients.

Conclusion: Large numbers of patients were enrolled to conduct a comparative efficacy study. Efficient biosimilar development should be considered on the basis of the accumulation of scientific understanding of comparable features of biosimilars and their development.

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从 PMDA 评估中调查生物仿制药开发对比临床研究的数据包和样本量。

背景：日本的生物类似药指南要求申办者开展临床研究，如比较药代动力学（PK）、药效学（PD）或疗效研究。在每个生物类似药开发过程中，申办者都会考虑临床数据包，因此不同生物类似药开发的临床数据包各不相同：本研究旨在阐明日本批准的生物类似药的临床数据包。方法：我们调查了日本批准的生物仿制药的临床数据包和样本量：方法：我们根据药品与医疗器械管理局（PMDA）网站2009年至2023年期间的审查报告调查了临床数据包和样本量：结果：根据PK和疗效比较研究批准了24个生物仿制药，根据PK/PD比较研究批准了10个生物仿制药，根据疗效比较研究批准了1个生物仿制药。在样本量方面，PK 对比研究在健康志愿者或患者中进行，样本量多达 300 例，但大多数研究只纳入了 1-100 例（68.1%，32/47）。1-30 例、31-60 例和 61-90 例的 PD 对比研究分别为 4 例、7 例和 4 例。最后，纳入 1-300、301-600 和 601-900 例的疗效对比研究分别为 6、10 和 11 例。其中，肿瘤和风湿病领域是招募 601-900 例患者的第一和第二大疾病领域：结论：进行疗效比较研究招募了大量患者。应在积累对生物仿制药可比特征及其开发的科学认识的基础上，考虑高效开发生物仿制药。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Pharmaceutical Medicine PHARMACOLOGY & PHARMACY-

CiteScore

5.10

自引率

4.00%

发文量

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.