Designation of Tremelimumab as an orphan drug - AstraZeneca

Overview

The Therapeutic Goods Act 1989, enacted by the Commonwealth Parliament, serves to regulate the supply of therapeutic goods in Australia, ensuring they are of acceptable quality and safety. This Act was introduced to address the need for a robust framework to manage the importation, supply, and registration of therapeutic goods, including medicines, medical devices, and blood products, to safeguard public health. The designation of Tremelimumab as an orphan drug under this Act, as specified in the 2015 Gazette, highlights the legislative intent to support the development of treatments for rare diseases. Dr. Anthony Gill, acting as the Delegate of the Secretary, designated Tremelimumab for the treatment of malignant mesothelioma, underscoring the policy objective to incentivise research and development of therapies for conditions that affect a small patient population. This legislative action reflects the broader aim of the Therapeutic Goods Act to facilitate access to innovative and necessary medical treatments while maintaining stringent regulatory standards.

Scope and Application

The Therapeutic Goods Act 1989, as amended and administered by the Therapeutic Goods Administration (TGA), designates Tremelimumab as an orphan drug specifically for the treatment of malignant mesothelioma. This designation applies to the solution for infusion dose form of Tremelimumab, which is sponsored by AstraZeneca. The Act applies to entities and individuals involved in the development, manufacture, and distribution of therapeutic goods within the Commonwealth of Australia. By designating Tremelimumab as an orphan drug, the Act aims to encourage the development of treatments for rare diseases that otherwise might not receive adequate attention or funding. The geographic reach of this designation is national, encompassing all states and territories within Australia. The Act does not explicitly state any exclusions, exemptions, or thresholds; however, the application for orphan drug designation must meet specific criteria outlined in the Therapeutic Goods Regulations 1990, which may implicitly define eligibility. The application and scope of the orphan drug designation may be further detailed or modified through subordinate instruments under the Regulations.

Key Provisions

The Therapeutic Goods Act 1989, in conjunction with the Therapeutic Goods Regulations 1990, includes provisions for the designation of certain drugs as orphan drugs (section 16J of the Regulations). Specifically, section 16J(2) of the Regulations empowers the Delegate of the Secretary to designate a drug as an orphan drug when it is intended for the treatment of a rare disease or condition. In this case, Tremelimumab has been designated as an orphan drug for the treatment of malignant mesothelioma, a rare form of cancer (section 16J(2)). The decision to designate Tremelimumab as an orphan drug was made by Dr. Anthony Gill, the Delegate of the Secretary, on 26 November 2015. This designation acknowledges the rarity of the condition it treats and facilitates certain benefits and incentives for the sponsor, AstraZeneca. The designation of Tremelimumab as an orphan drug imposes specific obligations on the sponsor, AstraZeneca. These obligations include compliance with any conditions set by the Therapeutic Goods Administration (TGA) to ensure the drug's safety and efficacy for the intended use. The sponsor must also provide any required information or documentation to the TGA, such as clinical trial data and post-market surveillance reports. Furthermore, AstraZeneca must ensure that the drug is manufactured and distributed in accordance with good manufacturing practice (GMP) standards, as outlined in the Therapeutic Goods Regulations. Failure to comply with the obligations imposed by the Therapeutic Goods Act and Regulations can lead to various consequences. For instance, if AstraZeneca does not adhere to the required standards for the manufacture and distribution of Tremelimumab, it may face enforcement actions from the TGA, such as product recalls or fines. Additionally, if the sponsor provides false or misleading information to the TGA, they may be subject to criminal penalties, including fines of up to $222,000 for individuals and $1,110,000 for bodies corporate, as per section 42D of the Therapeutic Goods Act. Furthermore, if the drug is found to be unsafe or ineffective, the TGA may take action to suspend or cancel the drug's registration, which could have significant commercial implications for AstraZeneca.