Designation of Regorafenib (Stirvarga) as an Orphan Drug

Overview

The Therapeutic Goods Act 1989 was enacted by the Parliament of Australia to provide a framework for regulating therapeutic goods, including medicines, medical devices, and blood and tissues. The Act aims to protect public health by ensuring that therapeutic goods are of acceptable quality, safety, and efficacy. The designation of regorafenib (STIVARGA) as an orphan drug on 13 July 2016 under the Therapeutic Goods Act 1989 addresses the problem of limited incentives for pharmaceutical companies to develop treatments for rare diseases, such as hepatocellular carcinoma, which affect a small number of people. The policy objective of this designation is to encourage the research, development, and availability of therapeutic goods for rare conditions by providing certain benefits and incentives to sponsors, including priority assessment and potential financial assistance.

Scope and Application

The Therapeutic Goods Act 1989, as amended and supplemented by the Therapeutic Goods Regulations 1990, applies to the designation of specific therapeutic goods within the Australian jurisdiction. In this instance, the Act facilitates the designation of a particular drug, regorafenib (STIVARGA), as an orphan drug for the treatment of hepatocellular carcinoma. This designation applies to the drug in its tablet form, sponsored by Bayer Australia Ltd. The legislation's scope extends to ensuring that orphan drugs, which are those intended for the treatment of rare medical conditions, receive appropriate support and consideration in their development and availability. The Act, administered by the Therapeutic Goods Administration, ensures that the orphan drug designation aligns with the national health objectives and supports the therapeutic needs of patients with rare diseases. This designation is made under the authority of a delegate of the Secretary, ensuring that the process is conducted within the bounds of the relevant regulatory framework and does not extend beyond the specified parameters of the Act.

Key Provisions

The Therapeutic Goods Act 1989, as amended and incorporating regulations such as the Therapeutic Goods Regulations 1990, provides a framework for the regulation of therapeutic goods in Australia. Section 16J of the Regulations allows for the designation of certain therapeutic goods as orphan drugs. In this context, Dr Anthony Gill, acting as a delegate of the Secretary, has designated regorafenib (STIVARGA) as an orphan drug for the treatment of hepatocellular carcinoma (section 16J(2)). This designation, effective from 13 July 2016, recognises the therapeutic need for treatment of rare diseases and provides incentives for the research, development, and availability of such treatments. Entities and parties governed by the Therapeutic Goods Act and the Regulations must comply with the specific requirements set out for orphan drug designation. These include demonstrating the rarity of the condition, the therapeutic benefits of the drug, and the necessity for orphan drug status. The sponsor, Bayer Australia Ltd, must also ensure that the drug, in this case, regorafenib (STIVARGA) in tablet form, meets all necessary safety, quality, and efficacy standards as stipulated under the Act and Regulations. Additionally, they must provide evidence supporting the orphan drug designation to the Therapeutic Goods Administration (TGA) as part of the application process. Failure to comply with the provisions of the Therapeutic Goods Act and the Regulations can result in various civil and criminal consequences. Under the Act, breaches can lead to penalties such as fines and, in severe cases, imprisonment. For instance, knowingly supplying therapeutic goods that do not comply with the Act can result in fines of up to $1,215,000 for individuals and $6,075,000 for corporations, along with potential imprisonment. Additionally, the TGA can take enforcement actions such as recalls, injunctions, or suspension of approval for therapeutic goods found to be non-compliant. These measures are intended to ensure the safety, quality, and efficacy of therapeutic goods available in Australia, thereby protecting public health.