Regulatory Express_Q4 2019

1.       FDA Releases Revised Final Guidance on Policy for Device Software Functions and Mobile Medical Applications （FDA Sep. 2019）

FDA发布修订后的设备软件功能和移动医疗应用政策终版指南（FDA Sep. 2019）

Summary:

Given the rapid expansion and broad applicability of software functions deployed on mobile or other general-purpose computing platforms, the FDA is issuing a revised final guidance document to clarify the subset of software functions to which the FDA intends to apply its authority.

鉴于应用在移动或其他通用计算平台上的软件功能的快速扩展和广泛适用性，FDA正在发布修订后的最终指南文件以阐明FDA计划予以监管的软件功能的子集。

Revised final guidance: Policy for Device Software Functions and Mobile Medical Applications. This document supersedes the original version issued February 9, 2015.

·       The guidance explains the agency's oversight of device software functions, including mobile medical apps, as devices and they focus only on the software that presents a greater risk to patients if it doesn't work as intended and on software that causes smartphones, computers, or other mobile platforms to impact the functionality or performance of traditional medical devices.

·       The FDA updated the guidance to reflect changes to the device definition in accordance with Section 3060 of the 21st Century Cures Act, which created a function-specific definition for device.

o   The functions excluded from the device definition are independent of the platform on which they might run.

·       Also, the FDA clarified that the policies for software are function-specific and apply across platforms.

o   Therefore, instances of "mobile application" in the guidance have been changed to "software function."

·       The FDA updated the title of the guidance to "Policy for Device Software Functions and Mobile Medical Applications."

·       Examples of mobile medical apps and software on the FDA web site were incorporated into the appropriate appendices of this document for consistency.

修订后的最终指南: 设备软件功能和移动医疗应用的策略。本文件取代2015年2月9日发布的版本。

·       该指南解释了FDA对设备软件功能（包括移动医疗应用程序）将作为设备进行监管，他们仅关注那些如果不能按预期使用将对患者造成较大风险的软件，以及那些导致智能手机，计算机或其他移动平台对传统医疗器械的功能或性能产生影响的软件。

·       FDA对指南进行的更新基于21世纪Cures法案的3060节，以反映设备定义的变化，该法案对设备创建了一个特定功能的定义。

o   将功能排除在设备的定义之外，独立于它们可能运行的平台。

·       此外，FDA澄清说，软件策略是针对特定功能且适用于跨平台的软件。

o   因此，指南中的“移动应用”实例已改为“软件功能”。

·       FDA将指南标题更新为“设备软件功能和移动医疗应用政策”。

·       FDA网站上移动医疗apps和软件的例子被纳入到本文件的适当附录中以保持一致性。

This document was released as part of a suite of guidances to continue to encourage innovative approaches to the development of digital health tools, and to ensure the agency’s approach to overseeing these technologies advances along with it.  Changes to these documents include clarification that many products previously under enforcement discretion no longer are medical devices under the 21st Century Cures Act, and therefore are not subject to FDA’s regulations and oversight.

 本文件是作为一套指南的一部分发布的，旨在继续鼓励开发数字健康工具的创新方法，并确保监管机构监督这些技术进步的方法。对这些文件的修改包括澄清根据21世纪Cures法案，许多以前被强制执行的产品不再是医疗器械，因此不受FDA的监管和监督。

Key Takeaways:

·       The revised guidance is part of a suite of guidance revisions that offer additional clarity about where the FDA sees its role in advancing safe and effective digital health technologies.

·       FDA intends to apply its regulatory oversight to only those software functions that are medical devices and whose functionality could pose a risk to a patient’s safety if the device were to not function as intended.

关键信息：

·       修订后的指南是一套指南修订的一部分，可以更清楚地说明FDA在推进安全有效的数字健康技术方面的作用。

·       FDA打算将监管监督仅应用于那些医疗设备的软件功能，并且如果设备不能按照预期运作，可能对患者的安全构成风险的软件功能。

2.       European Commission Releases Guideline on GCP for Advanced Therapy Medicinal Products (ATMPs)  (EC 10 Oct 2019)

欧盟委员会发布关于高级治疗药物产品（ATMP）的GCP指南 (EC 10 Oct 2019)

Summary:

To provide for a common framework for the marketing of advanced therapy medicinal products (ATMPs), Regulation (EC) No 1394/2007 was adopted in 2007.  The European Commission (EC) has now released  Guidelines on the good clinical practice (GCP) requirements for Advanced Therapy Medicinal Products (ATMPs).  ATMPs are complex and innovative products that can include gene and cell therapies.

2007年获得通过的第1394/2007号条例(EC)为先进的治疗药物产品（ATMP）的上市提供了一个共同的框架。欧盟委员会（EC）日前发布了先进治疗药物产品良好临床实践（GCP）指南（ATMP）)。ATMPs是复杂创新的产品，包括基因和细胞疗法。

The guideline discusses:

·       clinical trial design and conduct

·       non-clinical studies

·       quality of the investigational ATMPs

·       traceability

·       retention of samples

·       protection of trial subjects

·       safety reporting

·       monitoring

指导原则讨论了：

·       临床试验设计和实施

·       非临床研究

·       研究性ATMPs的质量

·       可追溯性

·       样品的保留

·       保护试验受试者

·       安全性报告

·       监查

Some of the key points in the guidance are:

·       It is recognized that it may not always be feasible to generate relevant non-clinical data before the product is tested in humans.

·       Control groups receiving placebo should not be subjected to a procedure if it presents more than minimal risk and minimal burden.

·       The long-term effects of the product may require specific arrangements for long-term follow-up of the subjects however if the risk of delayed adverse events is low, long-term follow-up is not required.

·       Reporting forms and data capture systems should be adapted to reflect a differentiated causality assessment for each component of the ATMP, the application process and any required concomitant medication.

·       When the investigational ATMP contains cells or tissues of human origin, the traceability from the recipient of the product to the donor of the cells or tissues should be ensured.

指导中的一些要点是：

·       已经认识到，在产品进行人体试验前获得相关的非临床数据可能并不总是可行的。

·       不应对接受安慰剂的对照组实施大于最小风险和大于最小负担的试验程序。

·       该产品的长期影响可能需要对受试者进行长期随访的具体安排，但如果延迟不良事件的风险较低，则不需要长期随访。

·       应调整报告表和数据采集系统，以区分对ATMP各组成部分、应用流程和任何所需的合并用药的因果关系的评估。

·       当研究中的ATMP含有人源细胞或组织时，应确保从产品接受者到细胞或组织供体的可追溯性。

Key Takeaways:

·       ATMPs are complex and innovative products that may pose specific challenges to the design and conduct of clinical trials, these guidelines develop the GCP requirements that are specific to clinical trials conducted with ATMPs.

·       While some ATMP specific considerations relevant to ATMPs are explained in this Guideline, it is stressed that these are non-exhaustive and that the specific content of the cover letter, Protocol, Investigators Brochure (“IB”), and Investigators Medicinal Product Dossier (“IMPD”) is laid down in the Regulation (EU) No 536/2014.

关键信息：

·       ATMP是复杂而创新的产品，可能对临床试验的设计和实施构成特殊挑战，这些指南制定了特定于ATMP临床试验的GCP要求。

·       虽然本指导原则中解释了与ATMP相关的某些ATMPs特定考虑事项，但同时强调了这些并非详尽无遗，申请信的特殊内容、方案、研究者手册（IB）和研究者医学产品档案（IMPD）的具体内容在法规（EU）第536/2014号中列出。

References:

EC Publishes New Guidelines on Good Clinical Practices for ATMPs – Regulatory Focus