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

Overview

The Therapeutic Goods Act 1989, enacted by the Parliament of Australia, was established to regulate therapeutic goods in Australia and ensure their safety, quality, and efficacy. This Act provides the legal framework for the Therapeutic Goods Administration (TGA) to manage the supply and advertising of therapeutic goods, including medicines, medical devices, and blood products. One of the objectives of the Act is to encourage the development of treatments for rare diseases by addressing gaps in the availability of such treatments. In response to this objective, the Act includes provisions for the designation of orphan drugs, which are medicines developed for the treatment of rare medical conditions. This approach aims to facilitate the availability of effective treatments for patients with rare diseases, such as severe aplastic anaemia, by providing incentives to pharmaceutical companies to invest in the development of orphan drugs. The designation of eltrombopag (REVOLADE) as an orphan drug for the treatment of cytopenias in patients with severe aplastic anaemia who have had insufficient response to immunosuppressive therapy is an example of the application of this provision within the Therapeutic Goods Act 1989.

Scope and Application

The Therapeutic Goods Act 1989, as amended and governed by the Therapeutic Goods Administration under the Commonwealth of Australia, designates specific substances and products for various health applications. In this instance, the Act facilitates the classification of eltrombopag (REVOLADE) as an orphan drug, effective from 15 July 2014. This designation is specifically for the treatment of cytopenias in patients with severe aplastic anaemia who have not responded adequately to immunosuppressive therapy. This Act applies to the pharmaceutical entity eltrombopag (REVOLADE) in its tablet form, and the sponsor of this drug is GlaxoSmithKline Australia Pty Ltd. The scope of the Act ensures that the designated orphan drug meets specific criteria under the Therapeutic Goods Regulations 1990, which include the therapeutic benefits for rare conditions and the approval process overseen by a delegate of the Secretary, in this case, Dr Anthony Gill. The legislative framework ensures that such medications are subjected to stringent evaluation and regulation to maintain therapeutic efficacy and safety standards across Australia.

Key Provisions

The primary operative sections of the Therapeutic Goods Act 1989 as applied in this case involve the designation of eltrombopag (REVOLADE) as an orphan drug for the treatment of specific medical conditions. Under section 16J of the Therapeutic Goods Regulations 1990, the Delegate of the Secretary, Dr. Anthony Gill, has designated eltrombopag (REVOLADE) as an orphan drug for the treatment of cytopenias in patients with severe aplastic anaemia who have had an insufficient response to immunosuppressive therapy. This action was taken on 15 July 2014, with the therapeutic product available in tablet form. The sponsor of eltrombopag (REVOLADE) is identified as GlaxoSmithKline Australia Pty Ltd. This designation imposes certain obligations on the parties involved. For instance, the sponsor, GlaxoSmithKline Australia Pty Ltd, must ensure that the therapeutic goods meet the necessary standards and requirements for safety, quality, and efficacy as outlined in the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990. They must also provide relevant information and data to support the use of eltrombopag (REVOLADE) for the specified indication. The Therapeutic Goods Administration (TGA) has the responsibility to monitor and regulate the therapeutic goods to ensure compliance with the legislation. In terms of consequences for non-compliance, the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990 provide for both civil and criminal penalties. For civil penalties, section 92 of the Act allows for fines of up to $222,200 for individuals and $1,111,000 for bodies corporate for offences related to the supply of therapeutic goods that do not comply with the Act or the Regulations. Additionally, section 93A of the Act provides for imprisonment for up to two years or fines of up to $66,650 for individuals and $333,250 for bodies corporate for serious offences. The TGA can also initiate legal proceedings to seek injunctions or other remedies to prevent or address non-compliance with the Act and Regulations.