Designation of Adalimumab (HUMIRA) as an Orphan Drug - AbbVie Pty Ltd

Overview

The Designation of adalimumab (HUMIRA) as an Orphan Drug, Gazetted on 2 November 2012, represents a significant legislative measure designed to address the unmet medical needs of paediatric patients suffering from severe and debilitating conditions such as active Crohn's Disease. This legislative action was taken under the Therapeutic Goods Regulations 1990, specifically subregulation 16J(2), by Dr Anthony Gill, Delegate of the Secretary, to officially recognise adalimumab (HUMIRA) as an orphan drug. The designation targets paediatric patients aged 6 to 17 years who have an inadequate response to conventional therapies, or those who are intolerant to or contraindicated for such treatments. By identifying adalimumab (HUMIRA) as an orphan drug, the legislation aims to incentivise the development and availability of potentially life-altering treatments for rare and underserved medical conditions, thereby enhancing therapeutic options for affected children. The sponsor of the drug, AbbVie Pty Ltd, plays a crucial role in bringing this beneficial treatment to the paediatric population in need.

Scope and Application

The Designation of adalimumab (HUMIRA) as an Orphan Drug Act pertains specifically to the therapeutic drug adalimumab, marketed as HUMIRA, which has been designated for the treatment of active Crohn's Disease in paediatric patients aged between 6 and 17 years who have either had an inadequate response to conventional therapies, or who are intolerant to such therapies or have contraindications for them. This designation was made by Dr Anthony Gill, acting as the Delegate of the Secretary under the Therapeutic Goods Regulations 1990, and is effective as of 2 November 2012. The act applies directly to the sponsor of the drug, AbbVie Pty Ltd, and is intended to address a specific medical need within a narrowly defined patient demographic. The geographic reach of this legislation is inherently linked to the jurisdiction of the Therapeutic Goods Administration (TGA) within Australia, thereby applying nationally across the Commonwealth. There are no explicit exclusions or exemptions outlined in the text, and the legislation does not extend or restrict its application through subordinate instruments, maintaining its focus solely on the designated use of adalimumab for the specified medical condition.

Key Provisions

Section 16J of the Therapeutic Goods Regulations 1990 allows the designation of certain drugs as orphan drugs, a designation that provides incentives for the development and availability of treatments for rare or neglected conditions. Section 16J(2) provides the authority for the Delegate of the Secretary to designate these drugs. In this instance, Dr Anthony Gill, acting under this authority, has designated adalimumab (HUMIRA) as an orphan drug (Section 1) for the treatment of active Crohn's Disease in paediatric patients aged between 6 and 17 years who have not responded adequately to conventional therapy, or who cannot tolerate such therapies due to intolerance or contraindications. This designation pertains specifically to the solution for injection dose form of adalimumab (HUMIRA) (Section 2). The designation of adalimumab (HUMIRA) as an orphan drug imposes certain obligations on the sponsor, AbbVie Pty Ltd. These obligations may include the provision of additional data or information to support the orphan drug designation, ensuring that the drug is manufactured to the required standards, and adherence to any specific labelling or marketing requirements related to orphan drugs. The sponsor is also expected to maintain records and documentation that support the orphan drug status and be prepared to provide this information to regulatory authorities upon request (Section 3). Failure to comply with the obligations imposed by the orphan drug designation or any other requirements under the Therapeutic Goods Regulations 1990 can result in civil or criminal penalties. The specific penalties for breaches of the Regulations can vary depending on the nature and severity of the breach. Civil penalties can include fines, while criminal penalties can include imprisonment. The maximum penalties are specified in the relevant sections of the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990. For example, Section 33 of the Therapeutic Goods Act 1989 provides for fines of up to $220,000 for individuals and $1,100,000 for bodies corporate for offences involving therapeutic goods, while Section 87 of the Act provides for imprisonment for up to five years for more serious offences (Section 4).