Designation of Ramucirumab (Cyramza) as an Orphan Drug - Eli Lilly

Overview

The Therapeutic Goods Act 1989 was enacted to provide for the regulation of therapeutic goods in Australia, including medicines, medical devices, and other health-related products. The Act was introduced to address the need for a comprehensive framework to ensure the safety, quality, and efficacy of therapeutic goods available to the Australian public. The Therapeutic Goods Administration (TGA), an agency within the Department of Health, is responsible for the administration and enforcement of the Act. The overarching policy objective of the Act is to protect public health by regulating therapeutic goods in a manner that ensures they are of acceptable quality, safety, and efficacy. In the context of the Therapeutic Goods Act 1989, the designation of Ramucirumab (Cyramza) as an orphan drug on 17 September 2013 by Dr Anthony Gill, Delegate of the Secretary, exemplifies the Act's function in facilitating access to innovative treatments for rare diseases. This designation acknowledges the need for specialised therapeutic goods for patients suffering from less common medical conditions, such as advanced gastric cancer or gastro-oesophageal junction adenocarcinoma, and aims to encourage the development of treatments for these conditions by providing certain incentives and streamlined regulatory processes.

Scope and Application

The Therapeutic Goods Act 1989, as amended and regulated by the Therapeutic Goods Administration, pertains to the regulation of therapeutic goods in Australia, encompassing a broad range of products from medicines and medical devices to blood and tissues. This specific designation under the Act designates Ramucirumab (Cyramza) as an orphan drug for the treatment of patients suffering from advanced gastric cancer or gastro-oesophageal junction adenocarcinoma following prior chemotherapy. This designation applies to the sponsor, Eli Lilly, and the particular dose form of the drug, which is a solution for infusion. The orphan drug designation is intended to encourage the development of treatments for rare diseases by offering certain incentives and regulatory support. This legislative action has a nationwide reach within Australia, affecting the pharmaceutical industry and healthcare providers by recognising Ramucirumab (Cyramza) as a critical therapeutic option for a specific patient demographic. The designation does not explicitly state exclusions or exemptions, but it is subject to the overarching provisions and requirements set forth in the Therapeutic Goods Act and the Therapeutic Goods Regulations 1990.

Key Provisions

The Therapeutic Goods Act 1989, under the authority of the Therapeutic Goods Regulations 1990, designates Ramucirumab (Cyramza) as an orphan drug for the treatment of patients suffering from advanced gastric cancer or gastro-oesophageal junction adenocarcinoma who have already undergone prior chemotherapy. This designation, outlined in section 16J of the Regulations, was made on 17 September 2013 by Dr Anthony Gill, who acts as the Delegate of the Secretary under subregulation 16J(2) of the Regulations. Ramucirumab is administered as a solution for infusion, indicating its form and method of delivery. The sponsor of this therapeutic good is Eli Lilly, which is responsible for ensuring that the product meets the regulatory standards set forth by the Therapeutic Goods Administration. The obligations imposed by this designation on the parties involved are significant. The sponsor, Eli Lilly, must ensure that the drug is manufactured, stored, and distributed in compliance with the standards set by the Therapeutic Goods Administration. This includes adhering to Good Manufacturing Practice (GMP) guidelines to ensure the drug's quality and safety. Additionally, any promotional materials for Ramucirumab must be truthful, balanced, and not misleading, as required under the Therapeutic Goods Act. Eli Lilly must also maintain records and documentation to demonstrate compliance with these obligations, and these records must be made available for inspection by regulatory authorities. Breaches of the Therapeutic Goods Act and Regulations can result in severe penalties and consequences. For instance, the manufacture, supply, or advertising of therapeutic goods that do not comply with the Act or Regulations can lead to criminal charges. Under section 33 of the Act, an individual can be fined up to $330,000 or imprisoned for up to five years, or both, for each offence. Additionally, corporations can face substantial fines, with penalties up to $1.65 million for corporate entities. There are also civil penalties for non-compliance, which can include court orders to cease the manufacture or supply of the non-compliant goods. Furthermore, the Therapeutic Goods Administration has the authority to suspend or cancel the Australian Register of Therapeutic Goods (ARTG) listing of a product that does not meet regulatory standards, effectively banning the product from the market.