INTERACT会议——细胞和基因疗法产品开发的高效途径

科技的进步以及细胞和基因疗法领域的迅猛发展，为患者打开了一个前景无限的医学新纪元。与此同时，为了跟上行业加速发展的步伐，监管机构也有必要为产品开发者提供比以往更明确、更高效的途径。

Advances in scientific knowledge and growth in the cell and gene therapy space have led to a new and exciting era of medicine for patients, as well as a new motivation for regulators to provide clear, efficient pathways for product developers.

基因疗法涉及到转基因等方面的技术应用，包括将外源基因导入患者体内等，以达到治疗、预防或治愈疾病的目的；而细胞疗法则需要将具有相关功能的细胞转移到患者体内。这两类疗法通常针对患有罕见病或重大疾病，且医疗需求尚未得到满足的患者。

Gene therapy involves modifying or introducing genes into a patient’s body with the goal of treating, preventing, or curing a disease, while cell therapy requires transferring cells with relevant function into a patient.

These therapies frequently target diseases in patients that are rare (affecting fewer than 200,000 people in the US), serious, and representing an unmet medical need.

此类药物开发项目通常符合加快产品开发和审查的标准，并且可以从与FDA的早期互动中获益匪浅，从而避免不必要的、成本高昂的潜在临床前研究或其他预备性研究。在此背景下，INTERACT会议——即针对生物制品审评与研究中心（CBER，FDA下属机构）产品的初步有针对性的产品监管建议会议——为生物制品申办方和从业人员提供了一条与CBER进行早期开发讨论的途径。

As a result, programs to develop them often meet the criteria for expedited product development and review and can benefit greatly from early interactions with the FDA to avoid unnecessary and potentially costly preclinical or other preparatory studies.

The INTERACT (INitialTargeted Engagement for Regulatory Advice on CBER ProducTs) meeting offers sponsors of biologics (industry and investigators) an avenue for engagement with CBER in an early development discussion.

图1. 2020年美国药品研究与制造商协会（PhRMA）关于细胞和基因疗法赛道的报告显示，美国有362种疗法处于生物制药公司的临床开发多期阶段。

请注意：图中部分药物属于多个类别。

数据来源：Medicines in Development | 2020 Update: Cell and Gene Therapy

A 2020 PhRMA report on the cell and gene therapy pipeline found 362 therapies in a range of stages in clinical development by biopharmaceutical companies in the United States.

Note: Some medicines are in more than one category.

INTERACT会议的主要目标包括：讨论与早期产品开发相关的重要问题；在新药开发预审会（Pre-IND）前与CBER进行初步的非正式磋商；获取有关化学、制造和控制（CMC），以及药理学和毒理学等方面的初步非约束性建议；讨论新的交付器械、早期产品开发和临床前概念验证；确定需要解决的产品开发关键问题。

The Purpose of INTERACT Meetings

The meeting has several goals:

1.Discuss issues critical to early product development

2.Obtain a preliminary, informal consultation with CBER prior to a Pre-IND meeting

3.Obtain initial, nonbinding advice regarding chemistry, manufacturing, and controls (CMC), pharmacology and toxicology, and/or the clinical aspects of a development program

4.Discuss new delivery devices, early product development, and/or pre-clinical proof-of concept

5.Identify critical development issues or deficiencies to address

如何成功召开INTERACT会议？

召开INTERACT会议并非是为了让申办方向FDA提出开发方案并展开讨论，而是旨在获得有关其提交的预定义计划的反馈。想要开展INTERACT会议，申办方必须向FDA提交一份包含产品信息、建议适应症、产品开发说明，以及拟讨论问题清单等内容的会议申请。FDA会在收到申请后的21个工作日内给予反馈，并在90个工作日内安排会议。

Anatomy of a Successful INTERACT Meeting

An INTERACT meeting is not a forum for a sponsor to present development options to the FDA for discussion. The objective of the meeting is to obtain feedback on a proposed pre-defined program.

First, a sponsor must submit a meeting request and package together (no more than 50 pages) in the following format:

·Product information:

·Proposed indication:

·Product development: History and future plans

·List of questions:

The FDA typically responds to the meeting request within 21 calendar days and schedules the meeting 90 calendar days from receipt.

会议中要讨论哪些与产品开发相关的问题，完全取决于申办方，因为FDA不会主动提供意见或建议。申办方在准备拟讨论问题清单时应注意，重点强调那些关键或棘手的问题，然后在INTERACT会议正式召开时将这些问题列为优先讨论事项，以便留有充足时间得到FDA的相关反馈。

It is up to the sponsor to identify concerning development issues, as the FDA will not provide unsolicited information. A sponsor should highlight those sticky issues in the meeting package then prioritize them for discussion at the beginning of the INTERACT meeting to allow enough time for feedback.

申办方还应注意，每位与会者都必须为会议做好充足准备，因为在仅持续一小时的会议过程中，每一秒都很重要。在会议正式召开前先进行一次会议彩排，让每位与会人员预先演练发言，是一个能提高会议效果的有效方法。

Each participant must be prepared, as every second counts in a one-hour meeting. Lastly, a ‘dry-run’ meeting should be held to practice what each person plans to say during the actual meeting.

在申办方开发细胞和基因疗法产品为代表的创新医疗产品时，FDA能够提供早期指导。他们的意见在引领申办方制定产品初步开发策略方面，具有难能可贵的价值。INTERACT会议是一种获取FDA指导性见解的重要途径，能够在产品开发早期帮助申办方为产品开发计划提高效率并降低成本。

The FDA can help in the development of novel innovative products, and seeking early guidance, particularly when working with cell and gene therapy products, is invaluable when navigating complex or challenging preliminary product development strategies. An INTERACT meeting can provide crucial insight on how to accelerate your program and save time and money in the process.

在充满挑战的细胞和基因疗法产品开发领域，通过INTERACT会议尽早与FDA沟通，有助于申办方迈出通往成功的关键第一步。对于在此领域经验不足的申办方来说，可以向拥有丰富药物开发经验的CRO寻求合作，资深CRO往往与CBER等监管机构关系紧密，能够产品开发各阶段提供有力支持。

In the area of cell and gene therapy product development，sponsors will take the important first step of gaining early communication with the FDA via an INTERACT meeting. For sponsors lack of experience,they can seek cooperation from CROs with extensive drug development experience. Experienced CROs often have a close relationship with CBER and other regulators and can provide strong support at all stages of product development.

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普米尔医药（Premier Research）目前已经充分掌握肿瘤学和血液学、罕见病、神经科学、全球开发注册策略等各方面领域。依托 20 年产品开发经验，公司可以提供从初始策略制定、临床开发再到最终监管申报的一站式全方位服务。公司设有专精于为罕见和挑战性开发项目提供战略、监管和商业层面服务的部门Camargo，在帮助申办方召开INTERACT会议等方面拥有丰富经验。关注普米尔微信Premier Research，了解更多领先行业洞见。