Microbiota Transplantation: Recommendations for a Regulatory Framework

 描述(由申请人提供)：在未来，我们对人类微生物群的理解的进步将使微生物群移植(MT)--将细菌从健康个体移植到患者接受者身上以治愈或管理健康状况--变得越来越常见。目前，粪便微生物区系移植(FMT)是一种新出现的治疗艰难梭菌感染的有效方法，也可能有效地治疗其他疾病，目前有很多人对MT感兴趣并有证据支持。FMT给食品和药物管理局(FDA)提出了新的法律和监管问题。虽然FMT是人们关注的焦点，但其他新兴的MT选择包括阴道、皮肤、口腔和前鼻孔移植。这些其他MT选项的使用仍处于初级阶段，文献中几乎没有关于它们的适当监管的讨论。这个 拟议的项目将召集一个多学科工作组，以评估MT的替代监管选择(以FMT为主要重点)，并考虑哪些监管方法将使MT以符合监管有效性的特定标准的方式提供。受邀的工作组将由20-25名专家利益相关者组成，其中包括科学家、临床医生、患者、专业协会、生物伦理学家、政府监管机构、学者、律师和生物技术行业代表。工作组将研究目前如何监管FMT，以及如何监管与MT相似或类似的物质和程序(例如生物制品、血液和血液制品，以及人体器官和组织的移植)，以确定这些监管办法中的一些或全部是否适用于某些或所有形式的MT。工作组将分析每个现有监管制度在多大程度上符合一套标准，以确定监管框架是否适合FMT和其他形式的MT。具体地说，将分析每个监管框架，以确定是否--与某些或所有形式的MT-it有关 将：1)确保该物质/程序的安全性；2)确保该物质/程序的有效性；3)向接受者提供有关该物质/程序的安全性和有效性的可靠信息；4)确保有需要的患者能够适当地获得这些物质/程序；5)确保法规不会不必要地阻碍对MT的研究；以及6)支持与MT相关的公共卫生目标，包括其可能减少医院获得性感染和阻止不安全的家庭使用MT。在项目结束时，工作组将提出一系列关于今后应如何监管MT的建议，这些建议将以一篇或一系列论文的形式发表。

 DESCRIPTION (provided by applicant): In the future, advances in our understanding of the human microbiome will make microbiota transplantation (MT) - transplantation of bacteria from a healthy individual to a patient recipient to cure or manage a health condition - increasingly common. At present, there is much interest and evidence-based support for one type of MT, fecal microbiota transplantation (FMT), a procedure that is emerging as an effective treatment for Clostridium difficile infection and may also be effective in treating other conditions. FMT is raising new legal and regulatory questions for the Food and Drug Administration (FDA). Although FMT is the focus of much attention, other emerging MT options include vaginal, skin, oral, and anterior nares transplantations. Use of these other MT options is still in its infancy an there has been little, if any, discussion in the literature as to their appropriate regulation. The proposed project will convene a multi-disciplinary Working Group to evaluate alternative regulatory options for MT (with FMT as a primary focus) and consider which regulatory approach or approaches would make MT available in a way that meets specific criteria for regulatory effectiveness. The invited Working Group will comprise 20-25 expert stakeholders, including scientists, clinicians, patients, professional associations, bioethicists, government regulators, academics, lawyers, and biotechnology industry representatives. The Working Group will study how FMT is currently regulated and how substances and procedures similar or analogous to MT are regulated (e.g., biological products, blood and blood products, and transplantation of human organs and tissues) to determine if some or all of these regulatory options might be appropriate for some or all forms of MT. The Working Group will analyze the degree to which each existing regulatory scheme meets a set of criteria that determines whether the regulatory framework is appropriate for FMT and other forms of MT. Specifically, each regulatory framework will be analyzed to determine if - in relation to some or all forms of MT - it would: 1) ensure the safety of the substance/procedure; 2) ensure the effectiveness of the substance/procedure; 3) provide reliable information to recipients about the safety and effectiveness of the substance/procedure; 4) ensure that patients who need them have appropriate access to these substances/procedures; 5) ensure that the regulations do not unnecessarily discourage research on MT; and 6) support public health objectives relating to MT, including its potential to mitigate hospital acquired- infections and to discourage unsafe home use of MT. At the conclusion of the project, the Working Group will set forth a series of recommendations regarding how MT should be regulated going forward and these recommendations will be published in a paper or series of papers.