在生物类似开发中,现实世界的证据的实用性
Ramin B Arani1, Jessie Wang1, Dong Pang2
1Biosimilar Biostatistics, Sandoz Pharmaceuticals Inc, Princeton, New Jersey, USA.
Journal of biopharmaceutical statistics
|April 17, 2024
概括
现实世界数据 (RWD) 可以通过建立等价率,潜在地降低试验成本和增加患者获得生物疗法来增强生物类似开发. 这种方法利用现有数据有效支持监管目标.
科学领域:
- 药物经济学和健康经济学
- 生物制药开发 生物制药开发
- 临床试验设计 临床试验设计
背景情况:
- 生物类似开发旨在创建与参考产品非常相似的生物药物,确保可比的质量,安全性和有效性.
- 传统的生物类似药试验需要大量患者来确认临床性能,增加开发时间和成本.
- 大数据分析的进步鼓励将各种数据源,包括真实世界数据 (RWD) 整合到临床试验设计中.
研究的目的:
- 探索RWD在建立,改进或验证生物类似试验设计的等效率 (EQM) 中的利用.
- 提出并说明一种新的匹配方法,用于识别RWD.的可比患者队列.
- 展示RWD如何支持生物类似开发的监管目标,特别是当历史数据有限时.
主要方法:
- 开发了一种匹配方法的变化,以从RWD.中识别匹配的集合.
- 该方法使用共变量和次要终点作为匹配的数据特征.
- 为了说明拟议的匹配方法的应用和有效性,进行了一项案例研究.
主要成果:
- 案例研究表明,从RWD获得的初级终点估计值在已公布结果的1%以内.
- 这表明RWD可以有效地用于证明或估计生物类似开发的等效率.
- 拟议的方法表明,有望在生物类似药和参考产品之间建立可比的临床性能.
结论:
- RWD/现实世界证据 (RWE) 为优化生物类似试验设计和降低成本提供了宝贵的机会.
- 拟议的匹配方法为利用RWD提供了一个强大的框架,以支持生物类似药的监管提交.
- 在生物类似开发中更广泛地采用RWD/RWE可以提高获得基本生物疗法的机会.
相关概念视频
01:16Bioequivalence: Overview
1.0K
Pharmaceutical equivalents, by definition, are drug products with the same active ingredient in the same quantities, encapsulated in identical dosage forms, and intended for the same administration routes. These pharmaceutical equivalents are deemed bioequivalent if the bioavailability of the active entity in the drug preparations is similar. Moreover, pharmaceutical equivalents demonstrating bioequivalence are also regarded as therapeutically equivalent. This means that when used as directed,...
1.0K
01:23Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches
126
Biopharmaceutical studies constitute a vital field aiming to enhance drug delivery methods and refine therapeutic approaches, drawing upon diverse interdisciplinary knowledge. In research methodologies, the choice between controlled and non-controlled studies significantly influences the study's reliability and accuracy.
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast,...
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast,...
126
01:19Pharmacovigilance
830
Post-marketing surveillance is a critical component of pharmaceutical regulation, often uncovering unanticipated adverse drug reactions (ADRs) once a drug is widely used over an extended period.
This process, termed pharmacovigilance, aims to detect, evaluate, and minimize harmful effects related to medication use. The data collection for pharmacovigilance depends on spontaneous reporting systems, where healthcare professionals or patients voluntarily report suspected ADRs.
In some cases, there...
This process, termed pharmacovigilance, aims to detect, evaluate, and minimize harmful effects related to medication use. The data collection for pharmacovigilance depends on spontaneous reporting systems, where healthcare professionals or patients voluntarily report suspected ADRs.
In some cases, there...
830
01:12Analysis of Population Pharmacokinetic Data
254
Analysis of population pharmacokinetic data involves studying the behavior of drugs within diverse populations to understand their pharmacokinetic parameters. Traditional pharmacokinetic methods typically involve collecting samples from a few individuals and estimating these parameters. While these methods are commonly used, they have limitations in capturing the variability in drug response among individuals or heterogeneous populations. Population pharmacokinetics is employed to address these...
254
01:11Clinical Trials: Overview
2.9K
Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
2.9K
01:28Biopharmaceutics and Pharmacokinetics: Overview
2.0K
Understanding drugs, drug products, and their performance in pharmaceutical science is pivotal. Drugs, whether simple molecules or complex compounds, are designed to interact with the body's biological systems to diagnose, treat, or prevent diseases. Drug products include various delivery systems such as tablets, capsules, injections, and inhalers. The performance of these drug products is gauged by their ability to deliver the active ingredient to the desired site of action at the...
2.0K