Designation of Sorafenib (NEXAVAR) as an Orphan Drug - Bayer Australia Limited

Overview

The Designation of sorafenib (NEXAVAR) as an Orphan Drug Notice, gazetted on 4 March 2013, is a formal declaration under the Therapeutic Goods Regulations 1990. This legislation was enacted to address the need for incentivising the development of treatments for rare diseases, which often do not attract sufficient investment from pharmaceutical companies due to the small number of potential patients. This particular designation was made by Dr Anthony Gill, the Delegate of the Secretary, under subregulation 16J(2) of the Therapeutic Goods Regulations 1990. The policy objective behind the orphan drug designation is to encourage the development and availability of therapeutic goods for conditions that affect a small population, thereby improving access to necessary treatments for patients with rare diseases such as radioactive iodine treatment refractory, locally advanced, or metastatic differentiated thyroid cancer. The sponsor of the designated drug, sorafenib (NEXAVAR), is Bayer Australia Limited.

Scope and Application

The Designation of sorafenib (NEXAVAR) as an orphan drug under the Therapeutic Goods Regulations 1990 applies specifically to the drug sorafenib in its tablet form for the treatment of patients with radioactive iodine treatment (RAI) refractory, locally advanced or metastatic differentiated thyroid cancer (DTC). This designation is a Commonwealth-level decision, made by Dr. Anthony Gill, who is acting as the Delegate of the Secretary under subregulation 16J(2) of the Regulations. The sponsor of the drug, Bayer Australia Limited, plays a key role in this process. This legislation marks sorafenib (NEXAVAR) as an orphan drug, a classification that provides certain incentives and regulatory benefits aimed at encouraging the development and availability of treatments for rare diseases. The scope of the legislation is confined to this particular therapeutic indication and drug formulation, with no mention of broader application to other conditions or entities. The orphan drug designation does not extend or restrict the application through subordinate instruments as per the provided text, but it may be subject to further regulatory conditions or approvals by the Therapeutic Goods Administration.

Key Provisions

The primary operative section of the Gazette C2013G00385 is the designation of sorafenib (NEXAVAR) as an orphan drug (Regulation 16J(2)). This section specifies that sorafenib (NEXAVAR) has been designated for the treatment of patients with radioactive iodine treatment (RAI) refractory, locally advanced or metastatic differentiated thyroid cancer (DTC). The Gazette further clarifies that the approved dose form for this indication is the tablet, and the sponsor of the drug is Bayer Australia Limited. Under this designation, the Act imposes certain obligations and requirements on the parties involved. The sponsor, Bayer Australia Limited, must ensure that the drug is manufactured, labelled, and packaged in accordance with the Therapeutic Goods Regulations 1990. This includes compliance with good manufacturing practices and ensuring that all information provided about the drug is accurate and complete. Additionally, the sponsor is responsible for maintaining records and providing reports to the Therapeutic Goods Administration (TGA) as required. Failure to comply with the obligations and requirements set out in the Act can result in various offences and consequences. The Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990 provide for civil and criminal penalties for breaches. Civil penalties can include fines up to $222,200 for individuals and $1,111,000 for bodies corporate. Criminal penalties can include fines up to $555,500 for individuals and $2,777,500 for bodies corporate, as well as imprisonment for up to five years for serious or repeated offences. The specific penalties depend on the nature and severity of the breach.