Blood and Marrow Transplant Network, Agency for Clinical Innovation, Darlinghurst, Victoria, Australia.
Pathology. 2011 Oct;43(6):627-34. doi: 10.1097/PAT.0b013e32834b3cfa.
Use of cellular products for therapeutic purposes has predominantly been unregulated in Australia until recently. Transplant of haemopoietic progenitor cells (HPC) for bone marrow regeneration is now a routine treatment for many disorders with an established mechanism of facility accreditation. However, other cellular therapies do not have any form of accreditation, are not well evaluated and may be associated with significant risks. On 31 May 2011 the Therapeutic Goods Administration (TGA) implemented a long heralded regulatory biologicals framework for cell and tissue based therapies. The framework currently excludes human HPC, organs for direct transplantation and reproductive materials which are already covered by various forms of existing peer review and accreditation. This new framework is a practical approach for applying regulation based on the risk of the product to the recipient with four classes of product. Class 1 is reserved for the least regulated products and currently does not contain any proposed products. Class 2 will be for minimally manipulated products which will only require manufacturing compliance and evaluation against product and other mandatory standards before entry onto the Australian Register of Therapeutic Goods (ARTG). Class 3 and 4 products will be more than minimally manipulated and these cells and tissues may be used in a non-homologous manner. Class 3 and 4 products will represent a spectrum of risk where Class 4 therapies will represent the highest potential risk to the recipient, with the same requirements for Class 2 approvals but with additional requirements for comprehensive evaluation of a dossier for quality, safety and efficacy of the product. The extent of this quality, safety and efficacy data will depend upon the nature of the product and its associated risks, but will be more comprehensive for Class 4 as opposed to Class 3 products. The only truly contentious feature of this framework is the extremely high cost for dossier evaluation and the puzzling absence of an orphan drug scheme for biologicals.
直到最近，在澳大利亚，细胞产品的治疗用途主要是不受监管的。造血祖细胞（HPC）移植用于骨髓再生，现已成为许多疾病的常规治疗方法，其设施认证机制已经确立。然而，其他细胞疗法没有任何形式的认证，评估不充分，并且可能存在重大风险。2011 年 5 月 31 日，治疗商品管理局（TGA）对细胞和组织为基础的治疗方法实施了长期以来备受期待的监管生物制品框架。该框架目前不包括人类 HPC、直接用于移植的器官和生殖材料，这些已经涵盖在各种形式的现有同行评审和认证中。这个新框架是一种基于产品对受者风险的实用监管方法，有四个产品类别。第 1 类产品是监管最少的产品，目前没有任何拟议产品。第 2 类产品是经过最小程度处理的产品，只需要符合制造规范，并根据产品和其他强制性标准进行评估，然后才能进入澳大利亚治疗商品登记册（ARTG）。第 3 类和第 4 类产品则需要进行更多的处理，这些细胞和组织可以以非同源的方式使用。第 3 类和第 4 类产品将代表一个风险范围，其中第 4 类治疗方法对受者的潜在风险最高，需要与第 2 类批准相同的要求，但需要对产品质量、安全性和疗效进行全面评估。该质量、安全性和疗效数据的范围将取决于产品的性质及其相关风险，但对于第 4 类产品，其数据将比第 3 类产品更加全面。这个框架唯一真正有争议的特征是文件评估的极高成本，以及令人费解的生物制品孤儿药计划的缺失。
