Designation of Crizotinib (Xalkori) as an orphan drug - Pfizer Australia Pty Ltd

Overview

The Therapeutic Goods Act 1989 was enacted to regulate the provision of therapeutic goods in Australia, ensuring their quality, safety, and efficacy. The Act was introduced to address the need for a comprehensive framework governing the importation, supply, and advertising of therapeutic goods, including medicines, medical devices, and blood products. This legislative measure was enacted by the Parliament of Australia with the policy objective of protecting public health by ensuring that therapeutic goods are of an acceptable standard and their benefits outweigh any risks. The 2016 designation of Crizotinib (XALKORI) as an orphan drug under the Therapeutic Goods Regulations 1990 is an example of how this Act facilitates the regulation of therapeutic goods to address specific medical needs, in this case, the treatment of ROS1-positive non-small cell lung cancer. This designation recognises the rarity of the condition and aims to encourage the development of new treatments for underserved patient populations.

Scope and Application

The Therapeutic Goods Act 1989, as applied through the Therapeutic Goods Regulations 1990, pertains to the regulation of therapeutic goods in Australia. Specifically, the Act allows for the designation of certain drugs as orphan drugs, which are intended for the treatment of rare medical conditions. In this instance, the Therapeutic Goods Administration has designated Crizotinib (XALKORI), produced by Pfizer Australia Pty Ltd, as an orphan drug for the treatment of ROS1-positive non-small cell lung cancer. This designation is aimed at encouraging the development and availability of therapeutic goods for conditions that affect a small number of people. The application of the Act in this context is limited to the therapeutic good in question, Crizotinib (XALKORI), and its use in the treatment of the specified medical condition. The Act applies across the Commonwealth of Australia, with the Therapeutic Goods Administration exercising its regulatory authority under the Act and the Regulations. The designation of orphan drugs is a specific application of the broader regulatory framework provided by the Therapeutic Goods Act 1989, and does not alter the general scope of the Act, which covers all therapeutic goods within Australia.

Key Provisions

The Therapeutic Goods Act 1989, under the Therapeutic Goods Regulations 1990, has designated Crizotinib (XALKORI) as an orphan drug for the treatment of ROS1-positive non-small cell lung cancer (NSCLC) as outlined in section 16J. This designation is effective from 23 September 2016, and is applied specifically to the capsule form of the drug (16J(2)). The sponsor of Crizotinib (XALKORI) is Pfizer Australia Pty Ltd, and the designation was signed by Dr. Anthony Gill, who is acting as the Delegate of the Secretary for the purposes of the Regulations. The Act imposes certain obligations and requirements on the parties involved, primarily centred around the designation of orphan drugs like Crizotinib (XALKORI). These obligations include ensuring that the drug is appropriately researched, developed, and made available for the specified medical condition. The sponsor, Pfizer Australia Pty Ltd, must comply with these requirements to maintain the orphan drug designation. The sponsor is also responsible for ensuring that the drug is manufactured, stored, and distributed in a manner that meets the standards set by the Therapeutic Goods Administration (TGA). The Therapeutic Goods Act 1989 also provides for the enforcement of its provisions through the imposition of various offences and penalties. Breaches of the Act or Regulations may result in both civil and criminal consequences. For example, knowingly making a false or misleading statement in relation to a therapeutic good could lead to civil penalties of up to $2,200,000 for a corporation or $440,000 for an individual, as well as criminal penalties that may include fines and imprisonment. The Act also provides for the imposition of administrative penalties, such as the cancellation of a therapeutic goods registration or the issuance of a public warning about a particular product. These penalties serve to ensure compliance with the Act and to protect public health and safety.