Designation of Dexamethasone (Ozurdex) as an orphan drug - Allergan Australia Pty Ltd

Overview

The Therapeutic Goods Act 1989 is the central piece of legislation governing the regulation of therapeutic goods in Australia, enacted to ensure the safety, quality, and efficacy of these products. One specific objective of the Act, as demonstrated by the designation of dexamethasone (Ozurdex) as an orphan drug on 12 April 2017, is to address the gap in treatment options for rare conditions. This action was taken by Dr Anthony Gill, as a Delegate of the Secretary for the Department of Health's Therapeutic Goods Administration, under the authority granted by subregulation 16J(2) of the Therapeutic Goods Regulations 1990. The designation recognises the need for specialised treatments for conditions such as non-infectious uveitis affecting the posterior section of the eye, ensuring that patients with rare diseases have access to potentially life-changing therapies. The sponsor of this therapeutic good, Allergan Australia Pty Ltd, plays a crucial role in the development and supply of this designated orphan drug.

Scope and Application

The Therapeutic Goods Act 1989, administered by the Therapeutic Goods Administration (TGA) under the Commonwealth of Australia's Department of Health, encompasses a broad scope concerning the regulation of therapeutic goods. Specifically, the Act applies to therapeutic goods, which include medicines, medical devices, blood and blood components, tissues, and customised prostheses, ensuring they meet safety, quality, and efficacy standards before being marketed in Australia. The Act applies to entities and individuals involved in the manufacturing, importing, supplying, and advertising of these goods within Australia. The geographic reach of the Act is national, applying uniformly across the Commonwealth, states, and territories. The Act also extends its application through subordinate instruments, such as the Therapeutic Goods Regulations 1990, which provide further detail on specific provisions, including the designation of orphan drugs like dexamethasone (Ozurdex) for rare medical conditions, such as non-infectious uveitis affecting the posterior section of the eye, as specified in the designation. The regulations also establish criteria and processes for such designations, ensuring that the Act's provisions are implemented effectively across the therapeutic goods sector.

Key Provisions

The Therapeutic Goods Act 1989, through the Therapeutic Goods Regulations 1990, empowers the designation of certain drugs as orphan drugs, specifically for rare conditions. In this instance, section 16J(2) of the Regulations allows the Delegate of the Secretary to designate dexamethasone (Ozurdex) as an orphan drug for the treatment of non-infectious uveitis affecting the posterior section of the eye (s 16J(2)). This designation is effective from 12 April 2017, as specified by the Delegate, Dr Anthony Gill, who acts on behalf of the Secretary. The designation pertains to dexamethasone in the form of an intravitreal implant, a specific dose form intended for this medical use. The Act imposes several obligations on the parties involved, primarily the sponsor of the drug, Allergan Australia Pty Ltd. As the sponsor, Allergan Australia is responsible for ensuring that the drug meets the necessary regulatory standards and is safe and effective for its designated use. This includes providing any required documentation and data to support the orphan drug designation and ongoing compliance with the Therapeutic Goods Act and Regulations. The sponsor must also ensure that the drug is manufactured, stored, and distributed in accordance with the stipulated requirements, which include maintaining high standards of quality and safety. Failure to comply with the provisions of the Therapeutic Goods Act and Regulations can result in significant legal consequences. Under the Act, breaches can lead to both civil and criminal penalties. For example, knowingly making a false or misleading representation regarding a therapeutic good can result in a civil penalty of up to $2.1 million for a corporation and $420,000 for an individual, as well as potential imprisonment terms. Criminal offences can also be pursued under the Act, with maximum penalties varying depending on the severity of the breach, which may include substantial fines and imprisonment for directors or officers of the corporation found to be in breach of their duties. These provisions underscore the importance of adherence to the legislative requirements for all parties involved.