Designation of Defibrotide as an orphan drug - Link Healthcare

Overview

The Therapeutic Goods Act 1989, enacted by the Commonwealth of Australia Parliament, serves as the primary legislation governing the regulation of therapeutic goods within Australia. This Act was introduced to address the need for a comprehensive framework to ensure that therapeutic goods available in Australia are of acceptable quality, safety, and efficacy. One of the key provisions of the Act is its ability to designate certain drugs, such as orphan drugs, which are intended for the treatment of rare diseases. In this context, the Therapeutic Goods Administration (TGA) has the authority to designate Defibrotide as an orphan drug, as demonstrated in the 2013 designation for the treatment of hepatic veno-occlusive disease (VOD). This designation acknowledges the rarity of the condition and the necessity for specialised therapeutic interventions, aligning with the policy objective of facilitating access to critical treatments for rare medical conditions.

Scope and Application

The Therapeutic Goods Act 1989 designates specific therapeutic goods, including drugs, under its regulatory purview, and the Therapeutic Goods Regulations 1990 provide the framework for this regulation. In this instance, Defibrotide has been designated as an orphan drug for the treatment of hepatic veno-occlusive disease (VOD). This designation applies to Defibrotide in its dose form of concentrate for solution for infusion and is effective as of 12 November 2013. The designation is made by Dr Anthony Gill, acting as a Delegate of the Secretary, pursuant to the authority granted under subregulation 16J(2) of the Regulations. The sponsor of Defibrotide, Link Healthcare, will be subject to the obligations and benefits associated with this orphan drug designation. This includes potential incentives and support to facilitate the development and availability of treatments for rare diseases or conditions, such as hepatic VOD. The scope of this designation is limited to the specified therapeutic indication and dose form, and any further extensions or restrictions would be determined through subordinate instruments as necessary.

Key Provisions

The key operative sections of the Therapeutic Goods Act 1989, as applied in this specific context, primarily involve the designation of certain drugs as orphan drugs. Under section 16J of the Therapeutic Goods Regulations 1990, a delegate of the Secretary can designate a drug as an orphan drug. This designation is made under subregulation 16J(2) when it is for the treatment of rare or neglected conditions. In this instance, Defibrotide has been designated as an orphan drug for the treatment of hepatic veno-occlusive disease (VOD) (s. 16J). This is a significant designation as it provides incentives and regulatory support for the development and availability of treatments for rare diseases. The obligations imposed by the Act and the Regulations on the parties involved, particularly the sponsor, include ensuring that the drug meets the necessary standards for safety, efficacy, and quality. For Defibrotide, the sponsor, Link Healthcare, must ensure that the concentrate for solution for infusion is manufactured and supplied according to the required standards. This includes complying with good manufacturing practices and providing adequate information for the drug's use, including its dosage form and intended therapeutic use (s. 16J). The sponsor must also ensure that the drug is appropriately labelled and that all required documentation is submitted to the Therapeutic Goods Administration. Failure to comply with the provisions of the Therapeutic Goods Act 1989 and the Regulations can lead to various civil and criminal consequences. Offences under the Act can include supplying therapeutic goods that do not comply with the standards, which can result in substantial penalties. For example, supplying a therapeutic good that does not comply with the standards can lead to fines up to $222,000 for individuals and $1,110,000 for corporations, as well as potential imprisonment for serious offences (s. 31). Additionally, repeated or serious non-compliance can result in more severe penalties, including higher fines and extended periods of imprisonment. These provisions underscore the importance of adhering to the regulatory requirements to avoid legal repercussions.