Designation of Brigatinib (Alunbrig) as an orphan drug - Takeda Pharmaceuticals Australia Pty Ltd.

Overview

The Therapeutic Goods Act 1989 was enacted by the Parliament of Australia to regulate therapeutic goods, including medicines, medical devices, and blood. This legislation was introduced to address the need for a comprehensive regulatory framework that ensures the safety, quality, and efficacy of therapeutic goods available in the Australian market. The Act provides the Therapeutic Goods Administration (TGA) with the authority to assess, approve, and monitor therapeutic goods to protect public health. The policy objective of the Act is to ensure that therapeutic goods meet the necessary standards before they are made available to consumers, thereby safeguarding the health and well-being of the Australian public. On 28 July 2017, Dr Anthony Gill, as the Delegate of the Secretary, designated brigatinib (ALUNBRIG) as an orphan drug for the treatment of patients with anaplastic lymphoma kinase (ALK) positive non-small cell lung cancer under the provisions of the Therapeutic Goods Regulations 1990. This designation recognises the need for specialised treatments for rare diseases and supports the development of new therapeutic options for patients with limited treatment alternatives.

Scope and Application

The Therapeutic Goods Act 1989, as modified by the designation of brigatinib (ALUNBRIG) as an orphan drug, applies to pharmaceutical products and their sponsors within the Commonwealth of Australia. This specific legislation pertains to the treatment of patients diagnosed with anaplastic lymphoma kinase (ALK) positive non-small cell lung cancer, targeting the film-coated tablet dose form of brigatinib (ALUNBRIG). The Act designates Takeda Pharmaceuticals Australia Pty Ltd as the sponsor responsible for this therapeutic product. The designation is aimed at encouraging the development of treatments for rare diseases or conditions that affect a small number of people in Australia. The geographic reach of this Act is nationwide, ensuring that the therapeutic benefits of brigatinib (ALUNBRIG) are accessible across the entire Commonwealth. This Act does not specify exclusions or exemptions, but its application can be extended or restricted through subordinate instruments, such as regulations or guidelines, issued under the authority of the Therapeutic Goods Administration.

Key Provisions

The Therapeutic Goods Act 1989 (section 16J) and the Therapeutic Goods Regulations 1990 (subregulation 16J(2)) provide the legal framework for the designation of therapeutic goods, including drugs, as orphan drugs. Under this framework, Dr. Anthony Gill, as a delegate of the Secretary, has designated brigatinib (ALUNBRIG) as an orphan drug for the treatment of patients with anaplastic lymphoma kinase (ALK) positive non-small cell lung cancer. This designation, effective from 28 July 2017, recognises the rarity and specific nature of the condition it targets, aiming to encourage the development and availability of treatments for less common diseases. The obligations imposed by this designation on Takeda Pharmaceuticals Australia Pty Ltd, the sponsor of brigatinib (ALUNBRIG), include ensuring that the drug meets the necessary standards for safety, quality, and efficacy as outlined in the Therapeutic Goods Act and Regulations. The sponsor must also provide adequate information to support the orphan drug designation, including data on the drug's clinical efficacy and safety. Additionally, the sponsor must ensure that the drug is manufactured in compliance with good manufacturing practices (GMP) and that any changes to the drug's formulation or manufacturing process are approved by the Therapeutic Goods Administration (TGA). Failure to comply with the obligations and requirements set out in the Therapeutic Goods Act and Regulations can result in significant consequences. Under the Act, breaches may lead to civil or criminal penalties. For instance, section 33 of the Act allows the TGA to take action against any person who contravenes the Act or Regulations, including issuing infringement notices for minor breaches or pursuing more serious charges that could lead to substantial fines. In the case of serious or repeated breaches, the maximum penalty can be up to AUD 1.1 million for individuals and AUD 5.5 million for bodies corporate, as outlined in the Act. Additionally, persistent non-compliance can result in the suspension or cancellation of the therapeutic goods registration, thereby affecting the availability of the drug in the market.