Designation of Propranolol Hydrochloride as an Orphan Drug - Pierre Fabre Medicament Australia Pty Limited

Overview

The designation of Propranolol Hydrochloride as an orphan drug under the Therapeutic Goods Regulations 1990 was made by Dr Anthony Gill, acting as the Delegate of the Secretary, on 3rd October 2012. This legislative action was taken to address the specific medical need for the treatment of proliferating infantile hemangiomas that require systemic therapy, a rare condition that would not otherwise attract sufficient investment for drug development due to the limited patient population. This designation, which identifies Propranolol Hydrochloride in its oral solution form, was carried out in accordance with subregulation 16J (2) of the Therapeutic Goods Regulations 1990, under the authority granted by the Parliament of Australia. The policy objective behind this designation is to encourage the development of treatments for rare diseases by providing certain incentives, such as expedited review processes and potential market exclusivity, thereby improving access to necessary therapeutic options for patients with rare conditions.

Scope and Application

The Designation of Propranolol Hydrochloride as an Orphan Drug Act pertains specifically to Propranolol Hydrochloride, an orphan drug designated for the treatment of proliferating infantile haemangiomas requiring systemic therapy. This Act applies to the drug in its specified dose form of oral solution, and it is overseen by the sponsor Pierre Fabre Medicament Australia Pty Limited. The legislation operates within the jurisdictional reach of the Therapeutic Goods Regulations 1990, specifically under subregulation 16J (2) which allows the Delegate of the Secretary to make such a designation. This Act is applicable in the Commonwealth of Australia, and it is intended to provide a legislative framework for the use and recognition of Propranolol Hydrochloride as an orphan drug for the specified medical condition. The Act does not extend to other drugs, dose forms, or therapeutic uses, and no exclusions, exemptions, or thresholds are stated within the text of the legislation itself. However, the application and interpretation of the Act may be further defined or extended through subordinate instruments or regulations.

Key Provisions

The key operative sections of the legislation, C2012G00073 (Gazette), designate Propranolol Hydrochloride as an orphan drug for the treatment of proliferating infantile hemangiomas requiring systemic therapy. This designation is made under section 16J of the Therapeutic Goods Regulations 1990 and is effective from 3rd October 2012. The specific form of the drug designated is an oral solution, and the sponsor of this drug is Pierre Fabre Medicament Australia Pty Limited. The legislative instrument outlines the criteria for this designation, which is primarily aimed at encouraging the development of treatments for rare diseases or conditions. The legislation imposes specific obligations on the parties involved. Firstly, the Delegate of the Secretary, in this case Dr Anthony Gill, is required to make the designation under the authority provided by the Therapeutic Goods Regulations 1990. The sponsor of the drug, Pierre Fabre Medicament Australia Pty Limited, must then ensure that the drug is available for the designated use, adhering to the stipulated conditions and requirements of the orphan drug designation. These obligations include compliance with the therapeutic and manufacturing standards set forth by the Therapeutic Goods Administration. Breaching the obligations set out in this legislation can lead to various consequences. While the specific penalties are not detailed in the provided text, under the Therapeutic Goods Regulations 1990, non-compliance could result in enforcement actions by the Therapeutic Goods Administration. These actions may include the imposition of fines, recalls of the designated drug, or other regulatory sanctions. In more severe cases, criminal charges could be pursued, leading to potential imprisonment for individuals involved in the violation of the regulations. The precise penalties would depend on the nature and extent of the breach, as well as any relevant case law or precedents.