Designation of Bevacizumab (Avastin) as an orphan drug - Roche Products Pty Ltd

Overview

The Therapeutic Goods Act 1989 was enacted by the Parliament of Australia to regulate the import, supply, and quality of therapeutic goods. This legislation was introduced to address the need for a comprehensive regulatory framework governing the therapeutic goods industry, ensuring that such goods are safe, of high quality, and perform as claimed. The Act establishes the Therapeutic Goods Administration (TGA) as the body responsible for overseeing the regulation of therapeutic goods. One of the policy objectives of the Act is to facilitate access to therapeutic goods that may benefit patients with rare or orphan diseases, which are often neglected due to the smaller patient populations and the higher costs associated with research and development. In line with this objective, the TGA designates certain drugs as orphan drugs to encourage the development and availability of treatments for these conditions. On 21 March 2014, Bevacizumab (AVASTIN) was designated as an orphan drug for the treatment of persistent, recurrent, or Stage IV carcinoma of the cervix, highlighting the Act’s commitment to addressing unmet medical needs in rare cancer treatments.

Scope and Application

The Therapeutic Goods Act 1989, as amended by the Therapeutic Goods Regulations 1990, designates specific therapeutic goods for particular medical conditions, extending its regulatory scope to include orphan drugs. In this instance, Bevacizumab (AVASTIN), specifically in the form of an injection concentrate, has been designated as an orphan drug for the treatment of persistent, recurrent, or Stage IV carcinoma of the cervix by the Delegate of the Secretary, Dr Anthony Gill, on 21 March 2014. This designation applies to the sponsor of the drug, Roche Products Pty Ltd. The Act operates on a national level, with the Therapeutic Goods Administration under the Commonwealth of Australia responsible for its implementation and enforcement. The legislation aims to ensure the availability of treatments for rare diseases that might otherwise lack sufficient market incentive for pharmaceutical companies to develop. This designation extends the therapeutic use of Bevacizumab (AVASTIN) under the orphan drug provisions, potentially facilitating access to the drug for patients with the specified condition.

Key Provisions

The Therapeutic Goods Act 1989, as amended and supplemented by the Therapeutic Goods Regulations 1990, contains provisions for the designation of certain drugs as orphan drugs, with the objective of facilitating the development of treatments for rare diseases. In this context, section 16J of the Regulations allows the Delegate of the Secretary to designate a drug as an orphan drug. Pursuant to this authority, section 41FL(2) of the Regulations was invoked on 21 March 2014 to designate Bevacizumab (AVASTIN) as an orphan drug for the treatment of specific stages of cervical cancer. This designation (sections 41FL(2) and 16J) recognises the drug's potential in treating a rare and severe condition, thereby encouraging its development and availability. Under the Act and the Regulations, the designation of an orphan drug entails certain obligations for the parties involved. The sponsor of the drug, in this case Roche Products Pty Ltd, must comply with the requirements set forth in the Regulations to ensure that the drug meets the necessary standards for safety, efficacy, and quality. Additionally, the sponsor is required to submit relevant data and information to support the orphan drug designation, demonstrating the drug's potential to address unmet medical needs. These obligations ensure that the drug development process is rigorous and that the benefits of orphan drug designation are realised in terms of providing effective treatments for rare diseases. The Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990 also outline consequences for non-compliance with the requirements for orphan drug designation and other provisions of the Act. Breaches of the Regulations may lead to civil or criminal penalties, depending on the nature and severity of the non-compliance. For instance, section 41ZH of the Regulations imposes civil penalties for breaches related to the supply of therapeutic goods, including orphan drugs, that do not comply with the Act or the Regulations. The maximum penalty for corporations under section 41ZH is 500 penalty units ($95,000 as of 2023), while individuals may face a maximum penalty of 50 penalty units ($9,500). Additionally, section 42DA of the Act provides for criminal penalties for more serious breaches, with potential fines and imprisonment terms depending on the offence. These provisions ensure that the Therapeutic Goods Act and Regulations are enforced effectively to maintain the integrity of the therapeutic goods market.