Designation of Ibuprofen (Pedea) as an Orphan Drug - Emerge Health Pty Ltd

Overview

The Designation of Ibuprofen (PEDEA) as an Orphan Drug, gazetted on 22 May 2013, addresses the specific medical needs of preterm newborn infants less than 34 weeks of gestational age suffering from haemodynamically significant patent ductus arteriosus. This legislative action was taken by Dr Anthony Gill, Delegate of the Secretary, under the authority granted by subregulation 16J(2) of the Therapeutic Goods Regulations 1990. The primary objective of this designation is to encourage the development and availability of treatments for rare conditions that affect a small population, in this case, ensuring that Ibuprofen (PEDEA) in the form of a solution for injection is accessible for the targeted therapeutic use. The sponsor for this orphan drug designation is Emerge Health Pty Ltd, which plays a crucial role in bringing this specialised treatment to the market.

Scope and Application

The Designation of Ibuprofen (PEDEA) as an Orphan Drug Act pertains specifically to the medicinal product Ibuprofen (PEDEA), which has been designated for the treatment of haemodynamically significant patent ductus arteriosus in preterm newborn infants who are less than 34 weeks of gestational age. The legislation designates this specific form of Ibuprofen, a solution for injection, as an orphan drug under the Therapeutic Goods Regulations 1990. This designation is intended to encourage the research, development, and availability of treatments for rare diseases or conditions that affect a small number of individuals, in this case, preterm newborn infants with the specified medical condition. The Act applies to the sponsor of the medicinal product, Emerge Health Pty Ltd, and it is subject to the Commonwealth jurisdiction under Australian law. There are no stated exclusions, exemptions, or thresholds within the text of the legislation itself, although the application and interpretation of the orphan drug designation may be further refined or detailed through subordinate instruments or regulatory guidance.

Key Provisions

This legislation designates Ibuprofen (PEDEA) as an orphan drug (section 1), specifically for the treatment of haemodynamically significant patent ductus arteriosus in preterm newborn infants under 34 weeks of gestational age. The designated form of the drug is a solution for injection (section 1). The sponsor for this drug is Emerge Health Pty Ltd (section 1). The key operative sections of this legislation are those that formally designate the drug as an orphan drug and specify the indication and form of the drug, as well as identifying the sponsor. These sections ensure that the drug is recognised for its intended medical use and that the sponsor is accountable for the drug's development and distribution. The obligations imposed by this Act on the parties involved are primarily centred around the designation and the subsequent responsibilities of the sponsor. The sponsor, Emerge Health Pty Ltd, is tasked with ensuring that the drug meets all regulatory requirements for orphan drug status and is appropriately developed, manufactured, and distributed for the specified indication. The sponsor must also ensure that the drug's use complies with all relevant therapeutic guidelines and that it is used strictly for the designated medical condition. Additionally, the sponsor must maintain records and provide any necessary information to regulatory authorities as required by the Therapeutic Goods Regulations 1990. Any breach of the obligations under this legislation can lead to serious consequences. While the specific penalties for non-compliance are not detailed in this particular legislation, breaches of the Therapeutic Goods Regulations 1990 can result in both civil and criminal penalties. Civil penalties can include fines up to a significant amount, depending on the severity of the breach. Criminal penalties can include fines and imprisonment, with the exact penalties depending on the nature and extent of the offence. The Therapeutic Goods Act 1989 and its associated regulations are designed to ensure that drugs available in Australia are safe, of high quality, and used appropriately, with strict enforcement measures to address any breaches.