Designation of Bosentan as an orphan drug - Actelion Pharmaceuticals Australia Pty Ltd

Overview

The Therapeutic Goods Act 1989 was enacted by the Commonwealth Parliament to regulate therapeutic goods in Australia, ensuring their safety, quality, and efficacy. The 2014 designation of Bosentan as an orphan drug under this Act, announced by Dr Anthony Gill, Delegate of the Secretary, was intended to address the gap in treatment options for rare diseases by providing incentives for the development of drugs for conditions that affect a small number of people. The policy objective behind this designation is to encourage the research and development of treatments for conditions that might otherwise be overlooked due to their low prevalence. This specific designation aimed to provide access to Bosentan, a treatment for Pulmonary Arterial Hypertension, manufactured by Actelion Pharmaceuticals Australia Pty Ltd, recognising the need for targeted therapies for this rare condition.

Scope and Application

The Therapeutic Goods Act 1989, as amended, governs the regulation of therapeutic goods in Australia, ensuring that they are safe, of high quality, and effective. Under this Act, the Therapeutic Goods Administration (TGA) has the authority to designate certain drugs as orphan drugs, which are medications developed to treat rare medical conditions. Specifically, this designation applies to Bosentan, which has been identified as an orphan drug for the treatment of Pulmonary Arterial Hypertension. This legislative measure applies to the sponsor of the drug, in this case, Actelion Pharmaceuticals Australia Pty Ltd, and pertains to the specific dose form of Bosentan, which is a film-coated tablet. The geographic reach of this Act is national, applying across the Commonwealth of Australia. The Act's application is not restricted by state or territory boundaries, ensuring uniform regulation throughout the country. There are no exclusions or exemptions specified in this particular designation; however, the Act allows for the extension or restriction of its application through subordinate instruments, which may introduce specific conditions or limitations as necessary.

Key Provisions

The key operative sections of this piece of legislation pertain to the designation of Bosentan as an orphan drug for the treatment of Pulmonary Arterial Hypertension (PAH) (section 1). The designation, which is made by Dr Anthony Gill, Delegate of the Secretary for the purposes of section 16J of the Therapeutic Goods Regulations 1990, is effective from 2 December 2014. This designation specifically identifies Bosentan as a film coated tablet, which is a particular dose form, and highlights its sponsor, Actelion Pharmaceuticals Australia Pty Ltd. The Therapeutic Goods Act 1989, through its associated regulations, imposes certain obligations on the parties involved. Actelion Pharmaceuticals Australia Pty Ltd, as the sponsor of Bosentan, must ensure that the drug meets the necessary quality, safety and efficacy standards as set out in the Therapeutic Goods Regulations 1990. The designation of Bosentan as an orphan drug also means that the sponsor is eligible for certain incentives and support, such as reduced fees for the evaluation and registration of the drug, as well as potential tax benefits. The Therapeutic Goods Administration (TGA), which is the entity responsible for regulating therapeutic goods in Australia, must also ensure that the designation process is conducted in accordance with the relevant legislative and regulatory requirements. In the event of non-compliance with the Therapeutic Goods Act 1989 or the Therapeutic Goods Regulations 1990, there may be civil and criminal consequences for the parties involved. For example, failure to comply with the quality, safety and efficacy standards for therapeutic goods may result in the imposition of fines, imprisonment, or both. Additionally, if a person is found to have made a false or misleading representation in relation to a therapeutic good, they may be subject to a civil penalty of up to $222,200 for a corporation and $44,440 for an individual, as well as criminal penalties of up to five years imprisonment or both. It is important for all parties involved to ensure that they are aware of their obligations under the legislation and to take steps to ensure compliance. In summary, this legislation designates Bosentan as an orphan drug for the treatment of PAH, imposes certain obligations on the sponsor and the TGA, and outlines the potential consequences of non-compliance. It is important for all parties involved to understand their obligations and to take steps to ensure compliance with the legislation. Failure to do so may result in significant penalties, including fines, imprisonment, and civil liability.