Regulatory Express_Q4 2019

3.       WHO Releases Draft Guidance on Data Integrity (WHO Oct 2019)

世卫组织发布数据完整性指南草案

Summary:

The World Health Organization (WHO) recently drafted a new guideline on data integrity.  WHO notes that there has been a rise in the number of observations regarding the integrity of data, documentation and record management practices during inspections of GMP, GCP and GLP.  WHO notes possible causes for the increase in data integrity issues as: too much reliance on human practices; the use of computerized systems that are not appropriately managed and validated; and failure to adequately review and manage original data and records.

世界卫生组织（WHO）最近起草了关于数据完整性的新指南。世卫组织注意到，在GMP，GCP和GLP检查期间，关于数据完整性，记录和记录管理实践的发现项有所增加。世卫组织注意到数据完整性问题增加的可能原因是：过分依赖人的行为；使用未经适当管理和验证的计算机化系统；未能充分审查和管理原始数据和记录。

The guidance includes sections covering:

·       Data Integrity

·       Good Documentation Practices

·       Quality Risk Management

·       Management Review

·       Outsourcing

·       Training

·       Computerized System

·       Corrective and Preventive Actions

指南包括以下部分：

·       数据完整性

·       良好的文件规范

·       质量风险管理

·       管理审查

·       外包

·       培训

·       计算机化系统

·       纠正和预防措施

Key Takeaways:

·       This guideline provides information, guidance and recommendations to facilitate compliance with data integrity and record keeping requirements.

·       It recommends a risk-based approach over the life cycle of data and that a data integrity risk assessment should be carried out in order to identify and assess areas of risk.

关键信息：

·       本指导原则提供信息、指导和建议，以促进遵循数据完整性和记录保存的要求。

·       它建议在数据的生命周期中采用基于风险的方法，并应进行数据完整性风险评估，以鉴别和评估风险领域。

References:

WHO Drafts Guideline on Data Integrity for Pharmaceuticals – Regulatory Focus

4.       FDA Releases Final Guidance on Adaptive Design Clinical Trials (FDA Nov 2019)

FDA发布适应性设计临床试验的终版指南 (FDA Nov 2019)

Summary:

The FDA has released a final guidance titled: Adaptive Design Clinical Trials for Drugs and Biologics. This document:

·       Provides guidance on the appropriate use of adaptive designs for clinical trials to provide evidence of the effectiveness and safety of a drug or biologic.

·       Describes important principles for designing, conducting, and reporting the results from an adaptive clinical trial.

·       Advises sponsors on the types of information to submit to facilitate FDA evaluation of clinical trials with adaptive designs, including Bayesian adaptive and complex trials that rely on computer simulations for their design.

FDA发布了最终指南，标题为药物和生物制剂的适应性设计临床试验。本文件：

·       为临床试验适当地使用适应性设计提供指导，以提供药物或生物制剂有效性和安全性的证据。

·       描述了设计，实施和报告适应性临床试验结果的重要原则。

·       对申请人提交的信息类型给出了建议，以帮助FDA对适应性设计的临床试验进行评估，包括依赖计算机模拟进行设计的贝叶斯适应性和复杂试验。

Changes from the October 2018 draft guidance include

1.       Reworking the subsection on Bayesian adaptive designs to clarify the Agency's recommendations

2.       Clarifying the extent of pre-specification required for the rules governing adaptations.

3.       Editorial changes to improve clarity 

2018年10月指南草案的变更包括

1.       修改贝叶斯适应性设计的章节内容以澄清FDA的建议

2.       澄清调整规则所需的预先限定的程度

3.       为提高清晰度而进行的编辑修改

Key Takeaways:

·       The primary focus of this guidance is on adaptive designs for clinical trials intended to support the effectiveness and safety of drugs.

·       The concepts contained in this guidance are also useful for early-phase or exploratory clinical trials as well as trials conducted to satisfy post-marketing commitments or requirements.

关键信息：

·       本指南的主要重点是旨在支持药物有效性和安全性的临床试验的适应性设计。

·       本指南中的概念对于早期或探索性临床试验以及满足上市后承诺或要求的试验也是有参考价值的。