Designation of Eculizumab (rmc) (Soliris) as an orphan drug - Alexion Pharmaceuticals Australasia

Overview

The Therapeutic Goods Act 1989, enacted by the Commonwealth Parliament, was introduced to regulate the quality, efficacy, and safety of therapeutic goods in Australia. This legislative framework is essential for ensuring that therapeutic products available in Australia meet the required standards. One of the significant aspects of this Act is its ability to designate certain medications as orphan drugs, aimed at addressing medical conditions that affect a small population. The designation facilitates the development and availability of treatments for rare diseases. In this context, Eculizumab (rmc) (SOLIRIS) has been designated as an orphan drug for the prevention of delayed graft function (DGF) after solid organ transplantation, reflecting the policy objective of supporting the development of treatments for rare and neglected conditions. This designation was made under the Therapeutic Goods Regulations 1990 by Dr Anthony Gill, Delegate of the Secretary, on 18 January 2017, with Alexion Pharmaceuticals Australasia as the sponsor.

Scope and Application

The Therapeutic Goods Act 1989 applies to the regulation of therapeutic goods in Australia, encompassing a broad range of products including medicines, medical devices, blood and tissues, and therapeutic appliances. Under this Act, the Therapeutic Goods Administration (TGA) has the authority to regulate these goods to ensure they are safe and of acceptable quality. The legislation applies to both persons and entities involved in the supply, importation, and advertising of therapeutic goods, as well as to the conduct and transactions related to these goods. Geographically, the Act operates at the Commonwealth level, with a uniform application across Australia. However, certain aspects may also be subject to state and territory laws, particularly in relation to the advertising and supply of therapeutic goods. The Act includes various exclusions and exemptions, such as for goods used for personal or domestic purposes, and certain medical devices used within healthcare facilities. Additionally, the application of the Act can be extended or restricted through subordinate instruments, such as regulations and guidelines, which provide further detail on specific requirements and processes for therapeutic goods. In this particular instance, the designation of Eculizumab (rmc) (SOLIRIS) as an orphan drug for the prevention of delayed graft function after solid organ transplantation is a specific application under the broader framework of the Therapeutic Goods Act 1989.

Key Provisions

The Therapeutic Goods Act 1989, through the Therapeutic Goods Regulations 1990, provides for the designation of certain medications as orphan drugs, specifically under subregulation 16J(2) (section 16J of the Regulations). In this instance, Eculizumab (rmc) (SOLIRIS) has been designated as an orphan drug, effective from 18 January 2017. This designation pertains to the drug's use in the prevention of delayed graft function (DGF) following solid organ transplantation, with the specified dose form being a solution for intravenous infusion. The sponsor responsible for this therapeutic good is Alexion Pharmaceuticals Australasia. Entities and individuals governed by the Therapeutic Goods Act and Regulations must comply with the criteria and processes outlined for the designation of orphan drugs. This includes providing evidence and data supporting the drug's therapeutic benefits, rarity of the condition it treats, and the potential public health impact. For orphan drugs, additional benefits may include streamlined registration processes and certain market exclusivity rights. Sponsors, like Alexion Pharmaceuticals Australasia, are required to ensure that the drug meets the necessary safety, quality, and efficacy standards set forth by the Therapeutic Goods Administration (TGA). Breaching the provisions of the Therapeutic Goods Act or Regulations can result in serious legal consequences. For instance, the Act may impose administrative penalties for non-compliance, which can include fines up to a significant amount for both individuals and corporations, as stipulated in the legislation. Additionally, failure to adhere to the regulatory requirements could result in the suspension or revocation of the therapeutic good's registration, thereby preventing its distribution and use in Australia. These penalties underscore the importance of strict compliance with the legislative and regulatory framework governing therapeutic goods.