Designation Of Olaparib As An Orphan Drug - AstraZeneca

Overview

The Therapeutic Goods Act 1989 was enacted to regulate therapeutic goods, including medicines, in Australia, aiming to ensure that these goods are of acceptable quality, safety, and efficacy. This Act was introduced to address the need for a comprehensive legal framework governing the approval, manufacture, and distribution of therapeutic goods to protect public health. Enacted by the Parliament of Australia, the Act provides the Therapeutic Goods Administration (TGA) with the authority to oversee the regulation of therapeutic goods. The policy objective underpinning the Act is to safeguard the health and safety of Australians by ensuring that only therapeutic goods that meet certain standards are available for use in the country. On 15 January 2014, Dr Anthony Gill, as the Delegate of the Secretary for the purposes of the Therapeutic Goods Regulations 1990, designated Olaparib as an orphan drug. This designation recognises Olaparib's role as a treatment for a rare condition, specifically platinum-sensitive relapse BRCA-mutated ovarian, fallopian tube, or primary peritoneal cancer, and acknowledges the importance of incentivising the development of treatments for rare diseases. The sponsor of Olaparib is AstraZeneca Pty Ltd.

Scope and Application

The Therapeutic Goods Act 1989, as designated under the Therapeutic Goods Regulations 1990, includes provisions for the designation of certain medications as orphan drugs, specifically targeting rare diseases and conditions that affect a small population. In this instance, the Therapeutic Goods Administration has designated Olaparib as an orphan drug for the treatment of platinum-sensitive relapsed BRCA-mutated ovarian, fallopian tube, or primary peritoneal cancer in adults who have responded to platinum-based chemotherapy. This designation applies to the sponsor of the drug, AstraZeneca Pty Ltd, and the drug in its capsule form. The Act applies on a Commonwealth level, extending its reach across Australia and ensuring that the regulations are uniformly applied. While the primary legislation provides the framework, the scope and specific application details are further refined through the subordinate instruments, including the designation of particular drugs as orphan drugs, which allows for targeted therapeutic developments and support for rare disease treatments.

Key Provisions

The Therapeutic Goods Act 1989, through the designation of Olaparib as an orphan drug, aims to encourage the development and availability of treatments for rare conditions. Section 42A(1) of the Act outlines the criteria for designating a drug as an orphan drug, which includes the condition being rare and the drug being intended for treatment of that condition. Specifically, in this case, Olaparib is designated as an orphan drug for its use as a monotherapy in the maintenance treatment of adult patients with platinum-sensitive relapse BRCA-mutated ovarian, fallopian tube, or primary peritoneal cancer, as stated in the Regulations under Section 16J(2). This designation acknowledges the rarity of the condition and the need for specific treatments. The obligations imposed by this designation on the parties involved, particularly the sponsor, AstraZeneca Pty Ltd, are significant. They include ensuring that the drug meets the safety, quality, and efficacy standards set by the Therapeutic Goods Administration (TGA). Additionally, the sponsor must comply with any post-market surveillance and reporting requirements to monitor the drug's safety and effectiveness in the real-world setting. The designation also requires the sponsor to provide any necessary information to the TGA to support the orphan drug status and to facilitate the drug's approval and ongoing monitoring. Failure to comply with the obligations and requirements of the Therapeutic Goods Act 1989 and the Regulations can result in serious consequences. For instance, if the sponsor fails to meet the safety and efficacy standards, the TGA has the authority to take action under Section 31 of the Act, which could include the suspension or revocation of the drug’s registration. Additionally, under Section 93 of the Act, any person who contravenes a provision of the Act or the Regulations can be subject to substantial penalties. For example, an individual officer of a corporation can face fines of up to $222,200 for a serious breach or $44,442 for a non-serious breach, while the corporation itself can face even higher penalties, up to $1,111,100 for a serious breach or $222,220 for a non-serious breach. These penalties underscore the importance of strict compliance with the legislative requirements.