Phase IV Studies: Some Insights, Clarifications, and Issues.

IF 3.2 Q2 Pharmacology, Toxicology and Pharmaceutics
Bruno Mario Cesana, Elia Mario Biganzoli
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引用次数: 9

Abstract

Background: There is an increasing need to face regulatory aspects as well as ethics and scientific ones in the pharmaceutical research phase after the authorization of a drug. Traditionally, Phase IV studies, after the authorization of a drug to be marketed by the Competent Authority like the Food and Drug Administration (FDA) (in Europe, European Medicine Agency - EMA- through National Procedures or Community Procedures) have been considered mainly aimed to the assessment of the new drug safety profile. However, the sample size calculation for such aim is still an open issue. Moreover, the benefit/risk assessment is a compelling global need.

Methods: This editorial aims to give a fairly exhaustive overview of the main topics currently present in the pharmaceutical research phase after the authorization of a drug. FDA and EMA guidelines are considered under a comparative perspective with a focus on the perspective of "Post Authorization Safety Studies (PASS)" and "Post Authorization Efficacy Studies (PAES)" with critical considerations. Then, the approach of "Explanatory Trials" and "Pragmatic Trials" is proposed under the horizon of Health Technology Assessment (HTA).

Conclusion: Critical remarks are raised against the pure commercial perspective in the proposal of PASS and PAES and on the design of registries which should be planned with relevant objectives to be pursued by appropriate statistical analyses reported in the Statistical Analysis Plan (SAP) of the study protocol. Finally, a particular focus is placed on the work of the Ethical Committees regarding the approval of the observational studies on the safety and the efficacy of the drugs in their pragmatic clinical use.

第四阶段研究:一些见解、澄清和问题。
背景:在药物授权后的药物研究阶段,越来越需要面对监管方面以及伦理和科学方面的问题。传统上,在FDA等主管部门(在欧洲,通过国家程序或共同体程序)批准药物上市后,IV期研究被认为主要是针对新药安全性的评估。然而,这一目标的样本量计算仍然是一个悬而未决的问题。此外,利益/风险评估是一项迫切的全球需求。方法:这篇社论的目的是给出一个相当详尽的概述,目前在药物批准后的药物研究阶段存在的主要问题。FDA和EMA指南是在比较的角度下考虑的,重点是“授权后安全性研究(PASS)”和“授权后有效性研究(PAES)”的角度,并考虑了关键因素。然后,在卫生技术评价(HTA)的视野下,提出了“解释性试验”和“实用性试验”的方法。结论:对PASS和PAES提案中的纯商业观点提出了批评意见,并对注册中心的设计提出了批评意见,注册中心的设计应与研究方案统计分析计划(SAP)中报告的相关统计分析相结合,以实现相关目标。最后,一个特别的重点放在伦理委员会的工作,关于批准药物在实际临床使用中的安全性和有效性的观察性研究。
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来源期刊
Current clinical pharmacology
Current clinical pharmacology PHARMACOLOGY & PHARMACY-
CiteScore
3.60
自引率
0.00%
发文量
0
期刊介绍: Current Clinical Pharmacology publishes frontier reviews on all the latest advances in clinical pharmacology. The journal"s aim is to publish the highest quality review articles in the field. Topics covered include: pharmacokinetics; therapeutic trials; adverse drug reactions; drug interactions; drug metabolism; pharmacoepidemiology; and drug development. The journal is essential reading for all researchers in clinical pharmacology.
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