Designation of Equine Antithymocyte Immunoglobulin (ATGAM) as an orphan drug - Pfizer Australia Pty Ltd

Overview

The Therapeutic Goods Act 1989 was enacted to provide for the regulation of therapeutic goods in Australia, aiming to ensure their quality, safety, and efficacy. This Act, administered by the Therapeutic Goods Administration (TGA), forms the legislative framework for the control of therapeutic goods in Australia. The designation of Equine Antithymocyte Immunoglobulin (ATGAM) as an orphan drug under this Act on 8 October 2015 addresses the problem of limited treatment options for rare diseases, specifically aplastic anaemia in this instance. The policy objective is to encourage the development of treatments for conditions that affect a small number of people, by providing certain incentives and streamlined approval processes to pharmaceutical companies. This initiative was undertaken by Dr. Anthony Gill, the Delegate of the Secretary, under subregulation 16J(2) of the Therapeutic Goods Regulations 1990, with Pfizer Australia Pty Ltd as the sponsor of ATGAM.

Scope and Application

The Therapeutic Goods Act 1989 applies to the regulation and oversight of therapeutic goods, including medicines, medical devices, and related substances within Australia. This Act, administered by the Therapeutic Goods Administration (TGA), ensures that therapeutic goods available in Australia meet required standards for quality, safety, and efficacy. The scope of the Act encompasses all therapeutic goods, but its application is notably extended to include the designation of orphan drugs, such as Equine Antithymocyte Immunoglobulin (ATGAM) for the treatment of aplastic anaemia, under specific regulations. The Act applies to individuals and entities involved in the supply, advertising, and manufacturing of therapeutic goods, with particular provisions for entities such as sponsors, manufacturers, and importers. The Therapeutic Goods Regulations 1990, which operate under the Act, further specify the scope and application, including the designation of orphan drugs, thereby ensuring that treatments for rare diseases receive the necessary attention and support. The Act has a national jurisdictional reach, governing therapeutic goods across the Commonwealth of Australia, with specific regulations that may vary by state or territory. Exclusions and exemptions are outlined within the Act and its subordinate regulations, detailing which goods may be exempt from certain controls, such as those for low-risk medical devices. The application of the Act is extended through subordinate instruments, which provide detailed guidance and operational frameworks for the administration and enforcement of the Act’s provisions.

Key Provisions

The Therapeutic Goods Act 1989, as amended by the Therapeutic Goods (Orphan Drugs) Amendment Regulations 2015, designates Equine Antithymocyte Immunoglobulin (ATGAM) as an orphan drug for the treatment of aplastic anaemia (section 16J). This designation, made on 8 October 2015 by Dr Anthony Gill, Delegate of the Secretary, recognises the rarity of the disease and the need for the development of treatments for such conditions. The drug, ATGAM, is to be administered as an injection solution, and the sponsor for this therapeutic good is Pfizer Australia Pty Ltd. This legal instrument is aimed at encouraging the development of drugs for rare diseases by providing certain benefits and incentives. Under the Therapeutic Goods Act 1989 and its associated regulations, the designation of ATGAM as an orphan drug imposes specific obligations on the sponsor, Pfizer Australia Pty Ltd. These obligations include ensuring that the drug is manufactured to the highest quality standards and that clinical trials are conducted to demonstrate the safety and efficacy of the drug for the specified indication. The sponsor must also submit relevant data and information to the Therapeutic Goods Administration for review and approval. Compliance with these obligations is critical for the drug to gain market authorisation and for the sponsor to benefit from the incentives provided under the orphan drug designation. Failure to comply with the requirements set out in the Therapeutic Goods Act 1989 and the Therapeutic Goods (Orphan Drugs) Amendment Regulations 2015 can result in serious legal consequences. The Act provides for both civil and criminal penalties for breaches of its provisions. For instance, section 36 of the Act allows for the imposition of fines up to $222,200 for each offence where a person is found to have supplied a therapeutic good that does not comply with the Act. Additionally, section 41A of the Act allows for imprisonment for up to five years where a person is found to have knowingly made a false or misleading representation in relation to a therapeutic good. These penalties underscore the importance of adhering to the legislative requirements and the potential repercussions of non-compliance.