Designation of Riociguat as an Orphan Drug - Bayer Australia Limited

Overview

The designation of Riociguat as an orphan drug was enacted under the Therapeutic Goods Regulations 1990 (the Regulations) by Dr Anthony Gill, the Delegate of the Secretary, on 16 October 2012. This legislative action was introduced to address the need for specialised treatments for rare diseases, specifically for the treatment of Pulmonary Arterial Hypertension (PAH) and Chronic Thromboembolic Pulmonary Hypertension (CTEPH). The policy objective behind this designation was to encourage the development of orphan drugs, which are intended for the treatment of rare medical conditions that affect a small number of people, by providing certain benefits such as streamlined approval processes and market exclusivity. The Therapeutic Goods Regulations, which were enacted by the Australian Parliament, empower the Delegate to make such designations to facilitate access to critical therapies for patients with rare diseases.

Scope and Application

The designation of Riociguat as an orphan drug under the Therapeutic Goods Regulations 1990 applies to the medication Riociguat, which is used for the treatment of specific medical conditions, namely Pulmonary Arterial Hypertension (PAH) and Chronic Thromboembolic Pulmonary Hypertension (CTEPH). This designation is made by Dr. Anthony Gill, a Delegate of the Secretary, and is effective from 16 October 2012. The immediate-release tablets form of Riociguat is the specific dose form recognised for these indications, with Bayer Australia Limited identified as the sponsor of the drug. This designation is confined to the Commonwealth jurisdiction and is intended to provide incentives for the development of treatments for rare diseases. The orphan drug designation does not specify exclusions or exemptions but is contingent on the therapeutic application and the nature of the drug as it pertains to the treatment of rare medical conditions. Any further regulatory specifics or extensions to the application of this designation are to be determined through subordinate instruments as may be prescribed under the Regulations.

Key Provisions

The main operative sections of the legislation, C2012G00118, concern the designation of Riociguat as an orphan drug under the Therapeutic Goods Regulations 1990. Specifically, section 16J of the Regulations empowers the Delegate of the Secretary to designate certain drugs as orphan drugs. In this instance, Dr Anthony Gill, acting under subregulation 16J(2), has designated Riociguat as an orphan drug for the treatment of Pulmonary Arterial Hypertension (PAH) and Chronic Thromboembolic Pulmonary Hypertension (CTEPH), effective from 16 October 2012. This designation applies to Riociguat in the form of immediate-release tablets, with Bayer Australia Limited identified as the sponsor of the drug. The obligations and requirements imposed by this Act primarily rest on the sponsor of the designated orphan drug, Bayer Australia Limited, and the Delegate of the Secretary, Dr Anthony Gill. The sponsor must ensure that Riociguat is manufactured, labelled, and distributed in compliance with the therapeutic goods regulations and standards. Dr Anthony Gill, as the Delegate, has the responsibility of ensuring that the designation process is conducted fairly and in accordance with the provisions of the Therapeutic Goods Regulations. The legislation also outlines potential consequences for non-compliance with the provisions of the Therapeutic Goods Regulations. Although specific offences, penalties, or civil/criminal consequences are not detailed in this particular piece of legislation, general provisions within the Therapeutic Goods Act 1989 and associated regulations may apply. Non-compliance with therapeutic goods regulations could lead to various enforcement actions, including fines, product recalls, and legal proceedings. In severe cases, criminal charges may be brought against individuals or companies responsible for the breach, with penalties potentially including substantial fines and imprisonment. These potential consequences underscore the importance of adhering to the regulations governing therapeutic goods in Australia.