Designation of Lenvatinib (Not Stated) as an orphan drug - Eisai Australia Pty Ltd

Overview

The Therapeutic Goods Act 1989, enacted to regulate the import, supply, and quality of therapeutic goods in Australia, was introduced to ensure the safety, efficacy, and quality of these goods for public health and safety. This Act was passed by the Australian Parliament and is administered by the Therapeutic Goods Administration (TGA). The designation of Lenvatinib as an orphan drug under this Act, as specified in the 2017 Gazette (C2017G00829), is an important measure to address the gap in treatment options for rare diseases. The policy objective behind this designation is to encourage the development of treatments for conditions that affect a small number of people, thus ensuring that patients with rare diseases have access to necessary therapeutic options. The designation was made by Dr Tim Greenaway, the Delegate of the Secretary, under the authority granted by subregulation 16J(2) of the Therapeutic Goods Regulations 1990, with the intention of advancing medical treatments for hepatocellular carcinoma, a condition for which Lenvatinib has been designated as a treatment.

Scope and Application

The Therapeutic Goods Act 1989, as modified by the designation of Lenvatinib as an orphan drug on 14 July 2017, pertains specifically to the therapeutic substance Lenvatinib, which has been designated for the treatment of hepatocellular carcinoma in the form of hard capsules. This Act applies to the sponsor of the drug, in this instance, Eisai Australia Pty Ltd. The Act operates within the jurisdiction of the Commonwealth of Australia and is administered by the Therapeutic Goods Administration under the Department of Health. The orphan drug designation is intended to encourage the development and availability of treatments for rare diseases or conditions, in this case, hepatocellular carcinoma, by providing certain incentives and streamlined regulatory processes. This legislation does not explicitly state any exclusions, exemptions, or thresholds, but it implicitly excludes other drugs not designated as orphan drugs, thereby focusing its benefits and obligations specifically on Lenvatinib for the specified medical condition. The application and scope of this Act may be further detailed or expanded upon through subordinate instruments, which can provide additional regulatory or procedural guidance.

Key Provisions

The Therapeutic Goods Act 1989 (TGA) provides the legal framework for the regulation of therapeutic goods in Australia, and the designation of Lenvatinib as an orphan drug under section 41Z of the Act is a significant provision. Section 41Z(2) allows for the designation of certain medicines as orphan drugs if they are intended for the treatment of rare diseases or conditions. In this instance, Lenvatinib has been designated as an orphan drug for the treatment of hepatocellular carcinoma, a type of liver cancer that is considered rare (Section 41Z(2)). This designation is a formal recognition of the need for specific therapeutic goods to treat rare conditions and can provide certain benefits, such as streamlined regulatory processes and potential financial incentives for the sponsor. The designation of Lenvatinib as an orphan drug imposes several obligations on the parties involved. Firstly, the sponsor, in this case, Eisai Australia Pty Ltd, must ensure that the therapeutic good is developed and manufactured in accordance with the requirements of the TGA and the Therapeutic Goods Regulations 1990. This includes adhering to good manufacturing practice standards and ensuring that the product is safe, of high quality, and effective for its intended use (Regulation 3.2.1 of the Regulations). Additionally, the sponsor must maintain records and documentation to demonstrate compliance with these obligations (Regulation 3.2.3 of the Regulations). Furthermore, the sponsor must also comply with any conditions imposed by the Therapeutic Goods Administration (TGA) in relation to the orphan drug designation (Section 41Z(4) of the Act). Breaching the obligations imposed by the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990 can result in both civil and criminal penalties. For instance, contravening section 41Z(4) of the Act by failing to comply with the conditions imposed by the TGA in relation to the orphan drug designation may result in a civil penalty of up to $222,200 for a corporation and $44,440 for an individual, as prescribed under section 14D(1) of the Act. In more serious cases, breaches of the Act or Regulations that involve intentional or reckless conduct may also result in criminal penalties, including fines and imprisonment, as provided under section 14D(2) of the Act. Additionally, the TGA may also take regulatory action, such as suspending or cancelling the therapeutic good's registration, which can have significant consequences for the sponsor and the availability of the therapeutic good to patients.