Designation of Eltrombopag (Revolade) as an orphan drug - GlaxoSmithKline Australia

Overview

The Therapeutic Goods Act 1989 was enacted by the Parliament of Australia to regulate therapeutic goods, including medicines, medical devices, and blood, within Australia. The Act was introduced to ensure that therapeutic goods available in Australia are of an acceptable quality, and that their benefits outweigh their risks. In addressing the specific problem of rare diseases, the Therapeutic Goods Regulations 1990 were later introduced to provide a framework for the designation of orphan drugs, which are medicines developed for the treatment of rare medical conditions. The designation of Eltrombopag (REVOLADE) as an orphan drug for the treatment of chronic idiopathic thrombocytopaenic purpura (ITP) under the authority of the Therapeutic Goods Act 1989 and Therapeutic Goods Regulations 1990, exemplifies the objective of the legislation to support the development and availability of therapeutic goods for rare diseases, ultimately improving access to treatment for patients with these conditions.

Scope and Application

The Therapeutic Goods Act 1989, administered by the Therapeutic Goods Administration (TGA), designates Eltrombopag (REVOLADE) as an orphan drug for the treatment of chronic idiopathic thrombocytopaenic purpura (ITP) with effect from 29 October 2014. This designation applies to Eltrombopag in its powder for oral liquid form, and the sponsor of this drug is GlaxoSmithKline Australia Pty Ltd. The orphan drug designation is a mechanism within the Act that provides incentives to encourage the development of treatments for rare diseases that affect a small number of people. This legislative action applies to the specified pharmaceutical product and its sponsor within the Commonwealth of Australia, and it leverages the Therapeutic Goods Regulations 1990 to implement specific measures for rare disease treatments. The Act's scope is focused on ensuring that orphan drugs receive the necessary support and incentives to address unmet medical needs, without broader implications for other therapeutic goods or industries.

Key Provisions

The Therapeutic Goods Act 1989, through the Therapeutic Goods Regulations 1990, designates Eltrombopag (REVOLADE) as an orphan drug for the treatment of chronic idiopathic thrombocytopaenic purpura (ITP). This designation, under section 16J of the Regulations (subregulation 16J(2)), was made by Dr Anthony Gill, who is the Delegate of the Secretary, on 29 October 2014. The document specifies that the drug's dose form for this particular indication is powder for oral liquid, highlighting the specific nature of its administration. The sponsor of Eltrombopag (REVOLADE) is GlaxoSmithKline Australia Pty Ltd, indicating the entity responsible for the drug's development and marketing within Australia. Under the Therapeutic Goods Act 1989 and its associated Regulations, parties involved with orphan drugs, such as GlaxoSmithKline Australia Pty Ltd, must adhere to stringent regulatory standards. These standards ensure that the therapeutic goods are safe, of high quality, and effective for their intended use. For orphan drugs, the Act and Regulations provide a framework for their designation, which facilitates access to these medicines for rare disease populations and often includes benefits such as priority review and reduced regulatory fees. Breaches of the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990 can result in significant legal consequences. Offences under these laws can lead to both civil and criminal penalties. Civil penalties can include fines, with the maximum penalty varying depending on the specific breach. For example, offences related to the supply of therapeutic goods that do not comply with the Act or Regulations can attract fines up to several thousand dollars. Criminal offences, such as knowingly supplying goods that are unsafe or misrepresented, can result in more severe penalties, including imprisonment. The specific penalties for each offence are detailed within the Act and Regulations, with the maximum penalties often linked to the severity and intent behind the breach.