Designation of Recombinant Human Coagulation Factor IX FC Fusion Protein (rFIXFc) as an Orphan Drug - Biogen Idec Australia Pty Ltd

Overview

The Designation of recombinant human coagulation factor IX Fc fusion protein (rFIXFc) as an Orphan Drug under the Therapeutic Goods Act 1989, enacted in 2012, aims to address the medical needs of individuals suffering from rare conditions, specifically haemophilia B, which is a congenital deficiency of factor IX or Christmas disease. This legislation facilitates the development and availability of specialised treatments for rare diseases by providing certain incentives to pharmaceutical companies. The Therapeutic Goods Administration, through its delegate Dr Anthony Gill, has designated rFIXFc as an orphan drug to ensure that patients with haemophilia B receive appropriate therapeutic options for the control and prevention of haemorrhagic episodes, including during surgical procedures. The intent behind this designation is to encourage research and development of orphan drugs, thereby addressing the specific healthcare needs of a small patient population that might otherwise be overlooked in the broader pharmaceutical market.

Scope and Application

The designation of recombinant human coagulation factor IX Fc fusion protein (rFIXFc) as an orphan drug under the Therapeutic Goods Regulations 1990 applies to this specific pharmaceutical product, intended for the treatment and prevention of haemorrhagic episodes in patients suffering from haemophilia B, a rare genetic disorder resulting in congenital factor IX deficiency or commonly known as Christmas disease. The application of this designation is limited to the use of rFIXFc in controlling and preventing bleeding, including during surgical procedures. This orphan drug designation is granted to address the unmet medical needs associated with this rare condition, and it is applicable to the sponsor of the drug, Biogen Idec Australia Pty Ltd. The geographic reach of this designation is aligned with the national scope of the Therapeutic Goods Regulations 1990, thereby extending its application across the entirety of Australia. Notably, the orphan drug designation process does not impose exclusions or exemptions from other regulatory requirements, and it does not affect the thresholds for safety and efficacy that rFIXFc must meet. The application and scope of this orphan drug designation may be further extended or specified through subordinate instruments under the Regulations.

Key Provisions

The legislation designates recombinant human coagulation factor IX Fc fusion protein (rFIXFc) as an orphan drug under section 16J of the Therapeutic Goods Regulations 1990. Specifically, section 16J(2) of the Regulations empowers the Delegate of the Secretary to make this designation. This designation, effective from 20 September 2012, pertains to the treatment of haemophilia B, a condition also known as congenital factor IX deficiency or Christmas disease. The legislation outlines that rFIXFc is intended for the control and prevention of haemorrhagic episodes, including bleeding during surgical procedures, and is administered as a lyophilised powder for IV injection. The sponsor for this therapeutic product is Biogen Idec Australia Pty Ltd. The obligations under this designation primarily involve the sponsor, Biogen Idec Australia Pty Ltd, ensuring that rFIXFc meets all necessary regulatory standards for orphan drugs. This includes compliance with the therapeutic requirements specified in the Therapeutic Goods Regulations 1990. The sponsor must also ensure that the drug is manufactured and supplied in accordance with good manufacturing practices and that appropriate clinical data support its efficacy and safety for the designated use. Additionally, the sponsor must maintain records and provide documentation as required by the Therapeutic Goods Administration (TGA) to verify compliance with the orphan drug designation. The legislation imposes penalties for non-compliance with the orphan drug designation requirements. Section 33 of the Therapeutic Goods Act 1989 sets out the possible civil and criminal penalties for breaches. Civil penalties may include fines up to $222,222 per offence for corporations, with higher penalties for repeated or serious breaches. Criminal penalties can include fines of up to $1,111,111 and imprisonment for up to 5 years for individuals, and fines of up to $5,555,550 for corporations. These penalties serve as a deterrent against non-compliance and ensure that therapeutic products designated as orphan drugs are used in a manner consistent with the intent of the legislation.