Designation of Tafenoquine (TBA) as an orphan drug - GlaxoSmithKline Australia

Overview

The Therapeutic Goods Act 1989 was enacted by the Parliament of Australia to regulate the import, supply, and advertising of therapeutic goods within the country. One of the key objectives of this Act is to ensure that Australians have access to safe, effective, and quality therapeutic goods. In addressing gaps within the therapeutic landscape, the Act provides a framework for the designation of orphan drugs, which are medications developed for the treatment of rare diseases or conditions that affect a small number of people. The designation of tafenoquine (TBA) as an orphan drug under this Act on 21 April 2017 by Dr Anthony Gill, the Delegate of the Secretary, exemplifies the Act's aim to facilitate the availability of necessary treatments for rare conditions. Tafenoquine (TBA), in the form of a tablet, was designated for the radical cure (prevention of relapse) of Plasmodium vivax malaria, with GlaxoSmithKline Australia Pty Ltd acting as the sponsor for this therapeutic good.

Scope and Application

The Therapeutic Goods Act 1989, through this specific designation, applies to the therapeutic good tafenoquine (TBA) which has been identified for its use in the radical cure of Plasmodium vivax malaria. The Act, managed by the Therapeutic Goods Administration under the Department of Health, designates tafenoquine (TBA) as an orphan drug, a designation given to drugs intended for the treatment, prevention or diagnosis of rare conditions. The sponsor of the drug, GlaxoSmithKline Australia Pty Ltd, is responsible for ensuring compliance with the regulations pertaining to this therapeutic good. This legislation has a national reach, impacting all entities within Australia that are involved in the manufacturing, supply, or prescription of the designated orphan drug. While the Act does not specify exclusions or exemptions for this designation, it operates under the broader scope of the Therapeutic Goods Regulations 1990, which may include provisions for further clarifications and extensions through subordinate instruments.

Key Provisions

The Therapeutic Goods Act 1989, as amended and applied through the Therapeutic Goods Regulations 1990, includes provisions that allow for the designation of certain therapeutic goods as orphan drugs. Section 41-3 of the Act permits the designation of drugs intended for rare medical conditions, specifically those that affect fewer than 5,000 people in Australia. In this instance, the Delegate of the Secretary has exercised their power under section 16J of the Regulations to designate tafenoquine (TBA) as an orphan drug (section 1). This designation is intended to facilitate the development and availability of treatments for rare diseases by providing certain incentives and regulatory advantages. The designation of tafenoquine (TBA) as an orphan drug imposes several obligations on the sponsor, GlaxoSmithKline Australia Pty Ltd. Primarily, the sponsor is tasked with ensuring that the drug meets the necessary standards for safety, efficacy, and quality as outlined in the Therapeutic Goods Act and Regulations. This includes providing comprehensive data and documentation to support the therapeutic claims of the drug, specifically its use in the radical cure (prevention of relapse) of Plasmodium vivax malaria (section 1). The sponsor must also adhere to any specific conditions or requirements set by the Therapeutic Goods Administration (TGA) to maintain the orphan drug designation. Failure to comply with the requirements of the Therapeutic Goods Act and Regulations can result in significant legal consequences. Under section 33DA of the Act, any person who contravenes certain provisions can be subject to civil penalties, including fines up to $222,200 for individuals and $1,111,100 for bodies corporate. Additionally, criminal penalties can apply, with maximum penalties of imprisonment for up to five years, reflecting the seriousness of non-compliance with therapeutic goods laws. These stringent measures are in place to ensure that therapeutic goods available in Australia are safe, effective, and of high quality, thereby protecting public health.