Designation of Dichlorphenamide (Keveyis) as an orphan drug - Ranbaxy Australia Pty Ltd

Overview

The Therapeutic Goods Act 1989 was enacted to regulate the provision of therapeutic goods in Australia, ensuring their safety, quality, and efficacy. It was introduced to address the need for a comprehensive framework governing the supply and use of therapeutic goods, including medicines, medical devices, and other related products. The Act empowers the Therapeutic Goods Administration to implement and enforce regulations that safeguard public health. The policy objective is to provide a robust system for the assessment, approval, and monitoring of therapeutic goods to protect consumers from substandard, counterfeit, and unsafe products. The designation of dichlorphenamide (Keveyis) as an orphan drug, announced on 2 August 2016, is a specific instance of this broader legislative intent, aiming to incentivise the development of treatments for rare conditions that otherwise might lack adequate research and development efforts due to limited market potential.

Scope and Application

The Therapeutic Goods Act 1989, as amended, encompasses various provisions that govern the regulation of therapeutic goods within Australia. This Act applies to any person or entity involved in the supply, importation, or advertising of therapeutic goods, including pharmaceuticals, medical devices, and other health-related products. The legislation is enforced by the Therapeutic Goods Administration (TGA), which is part of the Commonwealth of Australia’s Department of Health. It covers a broad spectrum of therapeutic goods, ensuring their safety, quality, and efficacy. The Act's jurisdiction extends nationally, affecting all states and territories within Australia, ensuring a uniform standard across the country. However, certain exclusions apply, such as therapeutic goods used for personal, non-commercial purposes, which are generally not subject to the Act. Additionally, the scope of the Act can be extended or modified through subordinate instruments, such as regulations and guidelines, which provide further detail on specific aspects of therapeutic goods regulation, including the designation of orphan drugs for rare conditions like primary periodic paralysis, as demonstrated in the case of dichlorphenamide (Keveyis).

Key Provisions

The Therapeutic Goods Act 1989, through its Regulations, facilitates the designation of specific drugs as orphan drugs to address unmet medical needs. In this instance, section 16J of the Therapeutic Goods Regulations 1990 empowers the Delegate of the Secretary to designate a drug as an orphan drug (subsection 16J(2)). On 2 August 2016, Dr Anthony Gill, as the Delegate, designated dichlorphenamide (Keveyis) as an orphan drug for the treatment of primary periodic paralysis (section 16J). This designation is specifically for the tablet form of the drug and is sponsored by Ranbaxy Australia Pty Ltd. This legislative action recognises the rarity of primary periodic paralysis and aims to encourage the development of treatments for such conditions. The designation of dichlorphenamide (Keveyis) as an orphan drug imposes certain obligations on the sponsor, Ranbaxy Australia Pty Ltd. These obligations include ensuring that the drug meets the criteria for orphan drug status, such as being intended for a rare condition, and facilitating the drug's development and availability for the targeted condition. The sponsor must also comply with any additional requirements set by the Therapeutic Goods Administration (TGA) related to the orphan drug designation, such as providing necessary documentation and updates on the drug's development and usage. Failure to comply with the provisions of the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990 can result in significant legal consequences. Offences under the Act can lead to civil or criminal penalties, depending on the severity of the breach. For instance, providing false or misleading information about a therapeutic good can result in fines or imprisonment. Specifically, under section 29 of the Act, a person who contravenes a provision of the Act or the Regulations can face fines of up to $222,200 for an individual and up to $1,111,100 for a body corporate, in addition to potential imprisonment terms. These penalties underscore the importance of adhering to the regulatory requirements governing therapeutic goods in Australia.